Australian Type 1 Diabetes. Research Resource Map

Transcription

1 Australian Type 1 Diabetes Research Resource Map

2 1 Introduction The Resource Mapping Project has been supported by the Macquarie Group Foundation through its pioneering, long term partnership with the Juvenile Diabetes Research Foundation. Australian Type 1 Diabetes Research Resource Map. The Juvenile Diabetes Research Foundation. Sydney, The Juvenile Diabetes Research Foundation This work is copyright. Apart from any use permitted under the Copyright Act 1968, no part may be reproduced without prior written permission from the Juvenile Diabetes Research Foundation. Requests and enquiries concerning reproduction and rights should be directed to the Juvenile Diabetes Research Foundation, PO Box 183, St Leonards NSW 1590.

3 Contents Executive Summary 2 1 Introduction Background Aim Scope Methodology 5 2 Researchers Research community Clinician researchers 9 3 Funders Academic research career pipeline Governmental funders of type 1 diabetes research in Australia Philanthropic funders of type 1 diabetes research in Australia International funders of type 1 diabetes research in Australia Commercial funding of type 1 diabetes research in Australia 24 4 Infrastructure Animal facilities Patient data Human cells and tissue Clinical trial infrastructure Other infrastructure 43 5 Knowledge and networks Professional groups and meetings Mentoring programs 47 6 Regulatory environment Therapeutic Goods Administration Ethics and frameworks 50 7 Key needs Researcher needs Funding needs Infrastructure needs Knowledge and networks needs 58 8 Next steps 60 9 Appendices Executive Advisory Committee Acknowledgements Abbreviations Contributors to qualitative assessment Infrastructure index 64 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 1

4 Executive Summary The development of prevention strategies, better treatments and eventually a cure for type 1 diabetes requires a clear scientific roadmap, and a diverse, experienced and well resourced scientific community. The unique multi-disciplinary nature of type 1 diabetes research means scientists need a wide range of sophisticated tools and resources as well as a strong collaborative approach to achieve the best results. In Australia, access by the type 1 diabetes research community to relevant research resources has traditionally been fragmented, both geographically and scientifically. In 2010, the Australian Type 1 Diabetes Research Agenda presented a strategic research plan designed to identify Australian research strengths and define research priorities in the field. The Agenda further outlined recommendations for a range of agreed research accelerators and enablers. One of the key recommendations of the Agenda was to undertake a systematic assessment of Australian type 1 diabetes research resources with a focus on facilitating access to such vital resources as research funding, clinical data, tissue specimens and specialised equipment. By doing this, duplication will be reduced and research will become more effective and efficient. The production of scientific output is reliant on four critical resource areas researchers, funders, infrastructure, and knowledge and networks. Using quantitative and qualitative approaches, and including input from researchers from across the entire type 1 diabetes research spectrum, this project has identified resources from each of these four key areas, with a focus on the academic research environment. Each resource is described and reviewed for both current and future accessibility. The scope and impact of the Australian research regulatory environment is also discussed. This document presents the results of the initial comprehensive review and identifies key areas of need, providing a vital source of information for both type 1 diabetes researchers and the funding bodies that support them. Following release, the document will remain open for public consultation from the type 1 diabetes research community in Australia, and submissions of shared resources will be welcomed in order to ensure that the resource map represents a comprehensive and complete resource for the research community. 2 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

5 Introduction 1 1. Introduction 1.1 Background The Australian Type 1 Diabetes Research Agenda 1 was a pioneering document, presenting a strategic research plan for type 1 diabetes research in Australia. The Agenda details Australian research strengths and outlines the globally relevant research objectives in which Australia is best placed to contribute. Further to documenting specific research directions, the Agenda identified recommendations for a range of research accelerators and enablers necessary for research progress across five domains. These are to: Undertake a systematic assessment of Australian type 1 diabetes research resources and diabetes landscape; Establish large, co-ordinated research infrastructure; Facilitate researcher career paths; Support and reward information exchange and collaborative efforts; and Develop the role of funding agencies and organisations. Within the first domain, a specific recommendation was made to map the resources available to type 1 diabetes researchers in Australia, with particular focus on merging, combining and managing access to these data and resources. Compiling a comprehensive resource map, and understanding the needs of the type 1 diabetes research community in this space will better place type 1 diabetes researchers and funders to undertake a number of other recommendations of the Agenda. Such recommendations include the establishment of shared biobanks and databases, appropriate channelling of funding into career support at the neediest career stages, and improving information sharing within the Australian type 1 diabetes research community. This project has comprehensively and systematically mapped the research resources currently available in the field of type 1 diabetes and its complications in Australia, with particular focus on the academic research landscape. Establishment of prevention strategies, better treatments and eventually a cure for type 1 diabetes requires a clear scientific roadmap and a strong scientific community. In today s research environment, scientists need sophisticated tools, resources and a collaborative approach to achieve best results. The production of scientific output is reliant on several resources within the research sphere, this is consistent across the global research community. Researchers, funders, infrastructure and knowledge are all critical and necessary components of research. This document outlines the state of the current research environment within each of these components, along with the impact of the regulatory environment in Australia. A comprehensive map of the resources that are currently utilised by type 1 diabetes researchers in Australia achieves both an increased visibility of available resources for the research community, and allows funders to identify opportunities to increase the stability and accessibility of these resources, and hence their utilisation. Likewise, an understanding of the gaps in resources, both physical and otherwise, allows effective investment into type 1 diabetes research through the support of the areas in most need. 1. Australian Type 1 Diabetes Research Agenda Partnering science, government and the community. 2nd edition. (2010) The Juvenile Diabetes Research Foundation. Available online at AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 3

6 1 Introduction 1.2 Aim The aim of this project is to create an understanding of the resource environment in which type 1 diabetes research takes place in Australia. The scope of the project includes the development of a comprehensive, up-to-date map of the resources currently used in type 1 diabetes research in Australia, as well as providing an insight to the gaps in resources that hinder research progression. This document provides an overview of the resource environment and will be important tool for both researchers and funders of type 1 diabetes research in Australia. Research enablers Research Research outcomes Researchers Funders Infrastructure Hypothesis driven research Publications, patents and discovery Patient benefits Knowledge and networks Regulatory environment Figure 1.1 Factors influencing research outcomes This document maps the four resource areas contributing to research output, with particular reference to type 1 diabetes research in Australia, as well mapping as the regulatory environment that dictates the research environment We anticipate that the Resource Map will be a dynamic document, open to input and consultation from the research community and updated regularly to maintain its relevance and usefulness. 1.3 Scope This project aims to present a comprehensive account of the resources that are available to type 1 diabetes researchers in Australia. For the purposes of the current document, the scope of the project was restricted to the academic setting. As yet, there has been limited analysis of pharmaceutical and biotechnology research into type 1 diabetes in Australia. As such, there is little visibility of the resources available to researchers that stem from the commercial sector. As it is hoped this project will provide an ongoing resource for type 1 diabetes researchers in Australia, the further development of information into the commercial sector will be a priority. 4 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

7 Introduction Methodology A quantitative review was undertaken to determine the basic characteristics of the research community involved in type 1 diabetes research in Australia. The scope was set to include research into the prevention, treatment and cure of type 1 diabetes and diabetes complications including diabetic retinopathy, nephropathy, neuropathy and cardiovascular complications. Researchers were identified through funding notifications from NHRMC, JDRF and other major funders of type 1 diabetes research in Australia from the last five years, and through research group and collaboration networks on researchers institutional profiles online. Over 260 relevant researchers were identified and are included in this analysis. A full list of the researchers included in this analysis can be made available on request. This information was used to inform the analysis of the type 1 diabetes research community in Australia for the Researchers section of this document. A systematic, qualitative assessment was separately undertaken to develop an understanding of the infrastructure environment for type 1 diabetes researchers in Australia. Researchers identified as being currently active in type 1 diabetes research, identifying type 1 diabetes as their primary area of focus and being regular recipients of type 1 diabetes research grants, were contacted for their input into this analysis. Fifty-two researchers were contacted for this review. Of these, 37 were interviewed for this project, 11 did not respond, and four responded, but were unable to schedule interviews, due to time constraints. Overall, this gave a response rate of just over 71%. For the qualitative assessment, site visits and semi-structured interviews, both face to face and by phone, were conducted with researchers and resource managers. Both clinical and basic researchers were contacted, and all areas of Australia were represented. See Appendix 4 for a full list of contributors. This information was used to compile and analyse the current infrastructure environment with particular reference to infrastructure that is shared within the type 1 diabetes research community, as well as informing the Needs section of this document. A consultation and revision process has been undertaken to ensure the accuracy of this document and the relevance of this project to the type 1 diabetes research community. An Executive Advisory Committee was convened to contribute to the development of this document and to inform the progression of this project with respect to the needs of the type 1 diabetes. Limitations In the analysis of the type 1 diabetes research community, there was difficulty capturing junior researchers with the quantitative assessment of type 1 diabetes researchers, as such this analysis represents a bias towards capturing established researchers. More investigation is needed to give an accurate impression of the career stage distribution of type 1 diabetes researchers in Australia. We selected qualitative assessment to allow in-depth exploration of the resources currently used by type 1 diabetes researchers in Australia and identifying what hurdles may lead to underutilisation of these resources. However, the methods used were more likely to identify resources that were already shared or available for sharing. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 5

8 2 Researchers 2. Researchers The community of researchers undertaking type 1 diabetes research in Australia are fundamental to facilitating the quality and impact of research output. A quantitative assessment of a cross-section of the type 1 diabetes research community in Australia was undertaken. The aim of this review was to generate a better understanding of the scope and expertise of the research community, in order to identify the needs of this community and ultimately to improve the environment contributing to research output in the field of type 1 diabetes and diabetes complications in Australia. The type 1 diabetes research community is highly diverse and generates a high diversity of output. This is particularly important in type 1 diabetes research, as the disease affects many organs and research into type 1 diabetes requires a multi-disciplinary approach. Similarly there is a wide spectrum of research in this field from basic research to clinical research. In defining and analysing the type 1 diabetes research community, it is important to note that the overlap between type 1 diabetes research and a range of other disciplines such as type 2 diabetes, cancer, general immunology and more, makes it difficult to clearly delineate and define a type 1 diabetes research community. As such, this analysis includes researchers who pursue research relevant to type 1 diabetes, while it may not be their primary focus. A better understanding of the overlap between disciplines will allow us to identify the needs of the research community better. This section provides an overview of the Australian type 1 diabetes research community with reference to the diversity of this community, including primary research focus, research qualifications and location. 2.1 Research community Research goals prevent, treat and cure Research in type 1 diabetes is multi-disciplinary and can be seen to focus on achieving three research goals: to prevent type 1 diabetes, treat type 1 diabetes or cure type 1 diabetes. With in these goals, research can be broken down into four therapeutic areas, outlined in Table 2.1. These therapeutic areas have defined the scope of this analysis, in order to ensure a comprehensive view of the diverse fields of research contributing to type 1 diabetes and its complications. This analysis has assessed the proportion of 260 type 1 diabetes researchers identified in Australia pursuing research in each of these areas. 6 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

9 Researchers 2 Table 2.1 Research goals and therapeutic areas of type 1 diabetes research Research goal Therapeutic area Description Prevent and cure Immune therapies Research investigating the autoimmune attack against beta cells and abrogating this attack Beta cell therapies Research investigating the replacement or regeneration of beta cells in type 1 diabetes, to restore insulin production Treat Glucose control Research that aims to establish tight blood glucose control for people at all stages of type 1 diabetes Complications therapies Research investigating the prevention, treatment and cure of the complications of type 1 diabetes, including retinopathy, neuropathy, nephropathy and cardiovascular disease Classification into these therapeutic areas shows that the majority of Australian type 1 diabetes researchers are in the field of complications research. This is unsurprising as this field encompasses a wide range of expertise, including nephrology, ophthalmology, neurology and cardiology. Over 130 researchers are pursing research relevant to complications of type 1 diabetes. Research in immune therapies was also highly represented with 83 researches, followed by 43 researchers in beta cell replacement and regeneration therapies, and 21 in the field of glucose control. Considering the aforementioned multi-disciplinary nature of type 1 diabetes, it is unsurprising that a number of researchers captured in this analysis were identified as pursuing research in more than one of these therapeutic areas. 150 Prevent and cure Treat 100 Number of researchers 50 0 Immune therapies Beta cell therapies Complications therapies Glucose control Other Figure 2.1 Distribution of type 1 diabetes researchers in Australia by research goal and therapeutic area AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 7

10 2 Researchers Qualification The majority (75%) of type 1 diabetes researchers in Australia hold a PhD. Of these approximately a quarter also hold a medical degree Bachelor of Medicine, Bachelor of Surgery (MB BS) or Doctor of Medicine (MD). Only 13% of type 1 diabetes researchers in Australia hold a medical degree without further research qualifications such as a PhD. 11.6% Unknown 5.4% PhD MD 5.8% MD 7.6% MB BS 54.0% PhD 15.6% PhD MB BS Figure 2.2 Distribution of type 1 diabetes researchers in Australia by qualification Location The majority of type 1 diabetes researchers in Australia are based in Melbourne (51%) and Sydney (22%). The majority of researchers (97%) were situated in capital cities. There were representations from every state and territory with the exception of the Northern Territory. 3.3% Other 3.6% Brisbane 5.4% Canberra 6.2% Perth 9.1% Adelaide 50.7% Melbourne 21.7% Sydney Figure 2.3 Distribution of type 1 diabetes researchers in Australia by location Institution type The majority of type 1 diabetes researchers in Australia were pursuing research at universities and research institutes, accounting for 85% of the research community analysed. The remainder were situated in hospitals, with a very small proportion working for government agencies. While this analysis gives an overview of the spread of researchers, it must be noted that many researchers are linked across multiple institutions and hence institution types. By comparison, the distribution of National Health and Medical Research Council (NHMRC) Project Grants by institution type shows that there are a noticeably higher number of type 1 diabetes researchers in research institutions compared to the wider health and medical research community supported by NHMRC Project Grants. 8 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

11 Researchers 2 0.4% Government 14.9% Hospital 41.3% Research Institute 43.5% University 2% Other 4% Government 16% Hospital 26% Research Institute 53% University Figure 2.4 Distribution of type 1 diabetes researchers in Australia by institution type (top) and distribution of NHMRC Project Grants by institution type (below) 2.2 Clinician researchers It is difficult to get a sense of how many clinician researchers are involved in type 1 diabetes research in Australia. Approximately a third of type 1 diabetes researchers in Australia have medical qualifications (MD or MB BS) and close to 15% of Australian type 1 diabetes researchers are hospital based. Despite these statistics, it is difficult to ascertain the number of researchers in the field who are clinically active, as this information is not published. Similarly, there is no visibility of commercial and institutional research funding for clinician researchers, which may make up a significant proportion of the total funding commitment to these researchers. Thus, it is difficult to ascertain which clinicians are currently active in type 1 diabetes research. Further to this, many clinician researchers, similarly to the rest of the research community, may pursue type 1 diabetes research, even while this may not be their primary area of focus. Recent canvassing of the type 1 diabetes research community, particularly self-identified clinician researchers, revealed the perception that the current funding environment and high clinical load are leading to a decrease in the number of clinicians becoming involved in research in Australia, and that even currently there are not enough clinician researchers in the field of type 1 diabetes research in Australia. There are a number of clinician research grants that are designed to give protected time to clinicians to dedicate to research, such as the NHMRC Practitioner Fellowship, the RACP/JDRF Australia Fellowship, and the JDRF Early Career Patient-Oriented Diabetes Research Award. The low numbers of these grants awarded a total of 16 NHMRC Practitioner Fellowships were awarded in 2010 across all health and medical disciplines indicates that there is a dearth of funding for this class of researchers. 2. National Health and Medical Research Council (2010) NHMRC Research Funding 2010 Facts Book. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 9

12 3 Funders 3. Funders The majority of type 1 diabetes research in Australia is funded by federal government and philanthropic organisations. The input of the commercial sector (both through pharmaceutical investment and the biotechnology sector) into partnerships with academia has not been assessed in detail in this document. This section focuses on the typical opportunities available to researchers in the field of type 1 diabetes, from both governmental and non-governmental sources. 3.1 Academic research career pipeline The academic researcher career pipeline follows a fairly rigid format whereby there are discrete career stages spanning study to independence as a researcher and research team leader. Funding support for researchers is often restricted to specific career stages. Grants which supply stipends for researchers are particularly important at the early stages of a research career when trainee researchers are dependent on a supervisor or research team leader for research funding. This is reflected in the abundance of people support awards that are geared towards PhD students, post-doctoral researchers, and early career researchers. As researchers gain the experience necessary to pursue an independent research career, research grants become more important to establish independent research programs. As such, the majority of research grants are highly competitive at the level of mid-career to established researchers. Figure 3.1 demonstrates the bias of people support funding to the early career stages, and research funding to the latter end of the career pipeline. 3.2 Governmental funders of type 1 diabetes research in Australia Federal government funding for research, including type 1 diabetes, in Australia is funnelled through four major funding bodies, each with a unique focus and unique funding mechanisms. The funding opportunities that are available to type 1 diabetes researchers in Australia through these funding bodies and governmental departments are outlined in this section. Table 3.1 provides a summary of these funders. 10 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

13 Funders 3 Table 3.1 Relevant governmental funders of type 1 diabetes research in Australia Funding body Focus Relevant grants and programs Australian Research Council Department of Health and Ageing Department of Innovation, Industry, Science and Research National Health and Medical Research Council To deliver policy and programs that advance Australian research and innovation globally and benefit the community To promote, develop, and fund health and aged care services for the Australian public To develop and implement policies and programs to assist industry and to build a competitive and worldclass science, research and innovation system To support health and medical research, develop health advice for the Australian community, health professionals and governments, and to provide advice on ethical behaviour in health care and in the conduct of health and medical research Equipment and Facility Grants Islet Transplantation Program, The Australian Type 1 Diabetes Clinical Research Network Australian Post Graduate Award General grants PhD Scholarship, Early Career Fellowship, Career Development Fellowship, Research Fellowship, Practitioner Fellowship, Project Grant, Program Grant, Development Grant, Strategic Award, National Health Research Enabling Capabilities Scheme, Equipment Grant, Infrastructure Grant, Centres of Clinical Research Excellence scheme Diabetes specific funding Diabetes Vaccine Development Centre, Centre of Clinical Research Excellence in Clinical Science in Diabetes, NHMRC/ JDRF Special Program Grants AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 11

14 3 Funders Figure 3.1 Academic research career pipeline People support programs NHMRC Diabetes specific funders Other Australian funders PhD NHMRC Postgraduate Scholarship JDRF Australia PhD top-up Scholarship Australian Postgraduate Award (Department of Innovation, Industry, Science and Research) JDRF Australia Travel Grant Award Alex Cohen Diabetes Top-up Scholarship (DRF WA) Post-doctoral NHMRC Early Career Fellowship (formerly Training (post-doctoral) Fellowship) JDRF Postdoctoral Fellowship JDRF Advanced Postdoctoral Fellowship JDRF Advanced Postdoctoral Fellowship Transition Award DART Viertel Postdoctoral Fellowship Early career NHMRC Career Development Fellowship (formerly Career Development Award) JDRF Career Development Award Established researcher NHMRC Research Fellowship DRF WA People Support Program NHMRC Practitioner Fellowship 12 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

15 Funders 3 Figure 3.1 Academic research career pipeline Research and infrastructure grants NHMRC Diabetes specific funders Other Australian funders International funders PhD Post-doctoral Early career RACP/JDRF Australia Fellowship Ramaciotti Establishment Grant JDRF Early Career Patient-Oriented Diabetes Research Award Established researcher Development Grant JDRF Priority Research Grant (previously Regular Research Grant) Ramaciotti Biomedical Research Award National Institutes of Health Research Grant Strategic Award JDRF Innovative Grant Financial Markets Foundation for Children Research Grant Centres of Research Excellence Scheme JDRF High priority, Short-Term Bridge Award Rebecca L. Cooper Medical Research Foundation Research (Equipment) Grant Project Grant JDRF Scholar Award Ramaciotti Equipment Grant Program Grant JDRF Strategic Research Agreement Single-Project National Health Research Enabling Capabilities (NHREC) Scheme (replacing Enabling Grant) JDRF Strategic Research Agreement Multi-Project Infrastructure Grant JDRF Strategic Research Agreement Clinical Equipment Grant JDRF Industry Discovery & Development Partnership JDRF Request For Application DART General Research Grant DRF WA Research Grant AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 13

16 3 Funders The Australian Research Council The Australian Research Council 3 (ARC) supports research across all disciplines including science, social sciences and the humanities, with the exception of clinical medicine and dentistry. The ARC does not generally fund medical research, however offers equipment grants for basic research such as the Linkage Infrastructure, Equipment and Facilities scheme, which aims to foster collaboration through supporting national and international research facilities. As such, the ARC indirectly supports researchers in type 1 diabetes research through infrastructure support. Department of Health and Ageing The federal Department of Health and Ageing 4 (DoHA) does not typically fund medical research directly, but supports type 1 diabetes research through the NHMRC as part of the DoHA portfolio. Despite this, DoHA supports large programs offering direct benefits to improve national health. DoHA has funded two large clinical programs in type 1 diabetes through partnerships with JDRF. Both the Islet Transplantation Program and The Australian Type 1 Diabetes Clinical Research Network are funded by DoHA and managed by JDRF. Department of Innovation, Industry, Science and Research The Department of Innovation, Industry, Science and Research 5 offers Australian Postgraduate Awards to support PhD students throughout Australia in all disciplines. While a number of these go to students pursuing health and medical research, it is difficult to ascertain the number of students in the field of type 1 diabetes that are supported through this program. The Australian Postgraduate Award is given to students undertaking a PhD in any field. The Award is $22,860 a year for a period of three years. In 2011, a total of 3,270 Awards were given nationally across all fields. National Health and Medical Research Council The National Health and Medical Research Council 6 (NHMRC) is the primary funder of diabetes research in Australia. A recent survey of type 1 diabetes researchers conducted by JDRF found that 65% of respondents had been supported by NHRMC grants. Figure 3.2 shows the amount of NHMRC funding in type 1 diabetes from It is important to note that the increased funding from is due to the one-off funding of the Diabetes Centres of Clinical Research Excellence, and the NHMRC partnership with JDRF to fund the Diabetes Vaccine Development Centre. It is likely that at the end of the funding period for these two initiatives that NHMRC funding in type 1 diabetes may decrease to the average $10 million at which funding had been stabilised in the previous several years. Available grants and awards People Support Awards Early career At a PhD level, the NHMRC offer the NHMRC Postgraduate Scholarship to students undertaking a PhD in health and medical research. The standard stipend is $22,860, for three years duration. In 2011, there were 131 Scholarships awarded. Success rates for these scholarships were at 43.5% in 2009, there is no more recent data on success rates. The NHMRC Early Career Fellowship (formerly Training (post-doctoral) Fellowship) is offered to postdoctoral researchers awarded a PhD within the last two years. Researchers are only eligible to hold this fellowship once. Recipients will generally have already secured external competitive funding, will have experience as a postdoctoral fellow or research assistant AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

17 Funders 3 $30,000,000 $25,000,000 $20,000,000 $15,000,000 $10,000,000 $5,000,000 $ Figure 3.2 Annual NHMRC research expenditure on type 1 diabetes, excluding diabetes complications from This category includes the Clinical Research Australian Fellowship, Research Fellowships and Translating Research into Practice (TRIP) Fellowships. In 2010, 120 fellowships were awarded, 75 were undertaken in Australia and 45 were undertaken by Australian researchers overseas. The success rate for these fellowships in 2009 was 30%. Mid-career The NHMRC Career Development Fellowship (formerly Career Development Award) is offered at two levels Level 1 Fellowships are for researchers two to seven years post-doctoral and Level 2 Fellowships are for researchers seven to 12 years post-doctoral. These Fellowships include the Clinical Career Development Fellowships available at part-time loads of 30-70%. In 2011, it is anticipated that around 62 Career Development Fellows will be appointed, up from 54 in The success rate for this award was 14.5% in Established researcher NHMRC Research Fellowships are offered at three levels Senior Research Fellow, Principal Research Fellow, Senior Principal Research Fellow. These Fellowships are offered to established researchers with a sustained track record. In 2010, 75 Fellowships were awarded and the success rate for the Fellowships in 2009 was 42.6%. NHMRC Practitioner Fellowships are offered to active clinicians who hold a PhD, to establish a research career in an area linked to their practice. Applicants are expected to have a strong research record and have secured research funding from elsewhere. In 2010, 16 Fellowships were awarded. Available grants and awards Research grants The NHMRC Project Grants Scheme is the main avenue of support for health and medical researchers in Australia. There is no specific limit to the grant amount under this scheme and awards are made for one to five years duration. In 2010, 683 Project Grants were awarded. The success rate for these grants was 23% in 2009, with a further 58% of applications being deemed fundable but unfunded 8. In 2010, the average total amount of Project Grants awarded was approximately $560, National Health and Medical Research Council. List of Datasets and Summary Funding Data. Available online at 8. National Health and Medical Research Council (2010) NHMRC Research Funding 2010 Facts Book. 9. National Health and Medical Research Council. Summary of Annual Funding Commitments 2009 and Available online at AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 15

18 3 Funders NHRMC Program Grants are designed for teams of established researchers to pursue broadly based collaborative research programs. In 2010, 15 Program Grants were awarded at an average of $7.3M per grant. This scheme had a 20.7% success rate in The Development Grant scheme supports the commercial development of health and medical products, processes, procedures and services in the early stages of development. Support through this scheme is designed to bring research to a level that attracts further funding from the private sector. Grants under this scheme are available to researchers and to health and medical research companies. In 2011, 18 Development Grants were awarded, with a 20% success rate 11, none in the field of type 1 diabetes. Strategic awards address priorities identified by the NHMRC on a three year planning cycle. Strategic Grants currently include NHMRC European Union Collaborative Research Grants, the Global Biodiversity Information Facility and the Human Frontier Science Program. In the current cycle, none of these strategic awards are relevant to type 1 diabetes. The Centres of Research Excellence Scheme is offered to teams of researchers to pursue collaborative research and develop capacity in clinical, population health and health services research. The Scheme funds a maximum of $2.5 million for each Centre, duration of five years. The Diabetes Centre of Clinical Research Excellence in Clinical Science was funded in 2007 through this program. To support infrastructure, the NHMRC runs the National Health Research Enabling Capabilities Scheme which replaces the Enabling Grant, and offers Infrastructure Grants and Equipment Grants. Enabling Grants have supported a range of incentives relevant to type 1 diabetes research such as the Australian Childhood Diabetes DNA Repository, the Australian PhenomeBank, and the Australia New Zealand Clinical Trial Registry. 50% 40% Success rates 30% 20% 10% 0% Postgraduate Scholarship Early Career Fellowship Career Development Fellowship Research Fellowship Project Grant Program Grant Figure 3.3 Success rates of selected NHMRC grants for National Health and Medical Research Council (2010) NHMRC Research Funding 2010 Facts Book. 11. National Health and Medical Research Council. Current outcomes of Development Grants funding rounds. Available online at 16 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

19 Funders 3 Case study Comparison to US funding environment The funding environment in Australia is extremely competitive. Comparison of the NHMRC and the National Institutes of Health (NIH) in the US illustrates the difference in available funding addressing the specific needs of Australian researchers. Below is an overview of NHMRC funding and NIH funding throughout the research career pipeline, demonstrating the degree to which NIH offer more people support grants and more research awards in comparison to the NHMRC. PhD Post-doctoral Early career Established researcher NIH National Research Service Award for Individual Predoctoral Fellowship to Promote Diversity in Health-Related Research (F31) National Research Service Award Institutional Research Training Grant (T32) National Research Service Award Institutional Research Training Grant (T32) National Research Service Award for Individual Postdoctoral Fellows (F32) Mentored Research Scientist Award (K01) Mentored Clinical Scientist Research Career Development Award (K08) Mentored Patient-Oriented Research Career Development Award (K23) Research Scholar Development Award (K22) Pathway to Independence Award (K99/R00) Mid-Career Investigator in Patient-Oriented Research (K24) Independent Scientist Award (K02) National Research Service Award for Individual Senior Fellows (F33) Research Project Grant Program (R01) Small Grant Program (R03) Academic Research Enhancement Award (R15) Exploratory/Developmental Research Grant Award (R21) Clinical Trial Planning Grant (R34) Small Business Technology Transfer (R41/R42) Small Business Innovative Research (R43/R44) High Priority, Short-Term Project Award (R56) Research Project Cooperative Agreement (U01) Research Program Project Grant (P01) Exploratory Grant (P20) Center Core Grant (P30) Specialised Center (P50) Resource-Related Research Project (R24) Education Project (R25) NHMRC Postgraduate Scholarship Early Career Fellowship Career Development Fellowship Research Fellowship Practitioner Fellowship Project Grant Development Grant Strategic Award Program Grant Centres of Research Excellence Scheme National Health Research Enabling Capabilities Scheme Infrastructure Grant Equipment Grant Figure 3.4 Comparison of major governmental funding in Australia and the US NIH runs three and sometimes four council rounds yearly in which they accept grant applications, supporting a range of different researcher classes and project types. In addition NIH offers specialised Request for Applications out of cycle, targeting areas of particular interest to the health research. The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is one of the 27 institutes and centres that comprise NIH, and is responsible for conducting and supporting diabetes research, and research into kidney, digestive, metabolic and other endocrine diseases. Success rates for comparable awards are generally higher for NIH grants compared to NHMRC grants. As such, researchers are generally better supported by governmental funding in the US compared to Australia. The overall success rate for Mentored Research Scientist Award (K01) in 2010 was 40% (34% for NIDDK, with 33 out of 96 awarded). Similarly, in 2010, the Mentored Clinical Scientist Research Career Development Award (K08) had a success rate of 44% for NIH, and 50% for NIDDK, with 37 out of 74 awarded by NIDDK 12. By comparison the NHRMC appointed 54 Career Development Fellows in all fields in In 2009, the success rate for this award was 14.5%. Similarly, only 16 NHMRC Practitioner Fellowships were awarded in 2010 to clinicians with a PhD in all health and medical fields, in order to establish a research career 13. Research funding through the basic Research Project Grant (R01) saw 333 grants awarded through NIDDK in 2010, out of a total 1,545 applicants, giving a 22% success rate for the award 14. By comparison, the NHRMC awarded 683 Project Grants in all fields of health and medical research in The 2009 success rate for these awards was 23% in Career Development Awards: Applications, awards, success rates, and funding, by Institute/Center and activity code. Available online at National Health and Medical Research Council (2010) NHMRC Research Funding 2010 Facts Book. 14. Research Project Grants: Success rates by type, activity, and Institute/Center. Available online at National Health and Medical Research Council (2010) NHMRC Research Funding 2010 Facts Book. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 17

20 3 Funders 3.3 Philanthropic funders of type 1 diabetes research in Australia In the academic setting, funding from philanthropic organisations offers significant support to type 1 diabetes research. There are a number of philanthropic and private funding sources in Australia that are available to researchers in type 1 diabetes, some of which are specific to diabetes and type 1 diabetes, and some with a wider scope incorporating type 1 diabetes and its complications. The funding opportunities that are available to type 1 diabetes researchers in Australia through philanthropic and private sources are outlined in this section. The table below provides a summary of these funders. Table 3.2 Private and philanthropic funding of type 1 diabetes research in Australia Funding body Focus Relevant grants and programs Diabetes Australia Research Trust com.au/research/dart/ Financial Markets Foundation for Children forchildren.com.au To support and develop the field of diabetes research in Australia through providing funding towards the prevention, management and cure for diabetes To promote the health and welfare of children of Australia Millennium Grant, Viertel Postdoctoral Fellowship, General Research Grant Financial Markets Foundation for Children Research Grant Institutional funding Support for internal researchers Various Juvenile Diabetes Research Foundation Ramaciotti Foundations ramaciotti/ Rebecca L. Cooper Medical Research Foundation foundation.org.au/ The Diabetes Research Foundation of Western Australia foundation.asn.au To find a cure for type 1 diabetes and its complications through the support of research To support biomedical research To promote and encourage medical research into all fields of the medical sciences, throughout Australia To find a cure for diabetes through support of research in Western Australia Postdoctoral Fellowship, Advanced Postdoctoral Fellowship, Career Development Award, Priority Research Grant (previously Regular Research Grant), Innovative Grant, High Priority Short-Term Bridge Award, Scholar Award, Strategic Research Agreements, Travel Grant Program, Innovation Award, NHMRC PhD Scholarship Top-Up Establishment Grant, Biomedical Research Award, Ramaciotti Medal, Equipment Grant Research (Equipment) Grant Alex Cohen Diabetes Top-up Scholarship, People Support Program 18 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

21 Funders 3 Diabetes Australia Research Trust Diabetes Australia Research Trust 16 (DART) funds research into type 1 diabetes, type 2 diabetes, gestational diabetes and latent autoimmune diabetes in adults. The mission of DART is the prevention, management and cure of all types of diabetes. DART places a focus on enabling new and upcoming researchers in the field of diabetes research through the support of funding. A recent survey conducted by JDRF of researchers active in type 1 diabetes found that 43% of respondents had been supported by DART grants. Available grants and awards DART offers two Millennium Grants yearly, one of which is specific to type 1 diabetes research. The awards are for up to $150,000 over two years. DART offers the Viertel Postdoctoral Fellowship for research in diabetes. The award is offered to a maximum $300,000, for a period of two years. DART General Research Grants are awarded to researchers pursuing research in all types of diabetes. Grants are awarded for one year and are worth $60,000. In 2010, DART awarded ten grants relevant to type 1 diabetes and its complications. In 2009 there were 16 grants relevant to type 1 diabetes and its complications to the total value of $960,000. Financial Markets Foundation for Children Financial Markets Foundation for Children 17 funds health and medical research in areas affecting children in Australia. The foundation has funded a small number of studies in type 1 diabetes over the past several years, with not more than one grant per year generally funding research in the area. Available grants and awards The Foundation offers one and two year Research Grants to a maximum of $100,000 for one year. On average grants are worth $50,000 $80,000 per year. Although the Foundation awarded no grants in type 1 diabetes in 2010, there have been grants relevant to type 1 diabetes in 2009 and previous years. Institutional funding Researchers can often access funding through the institution at which they undertake research, however there is little external visibility of the amount of total funding contributed in this manner to researchers in type 1 diabetes. Institutional funding supports a number of clinical trials in type 1 diabetes in Australia. Some examples include: The use of continuous subcutaneous insulin infusion with real-time continuous glucose monitoring system and low glucose suspend randomised controlled trial at Princess Margaret Hospital for Children; The health and coping in type 1 diabetes trial at the Royal Children s Hospital Melbourne; and The microvascular complications in patients with type 1 diabetes trial at Sydney Diabetes Clinical Research Unit, Royal North Shore Hospital. Juvenile Diabetes Research Foundation Juvenile Diabetes Research Foundation 18 (JDRF) is a major funder of type 1 diabetes and diabetes complications research in Australia with a mission to prevent, treat and cure type 1 diabetes. JDRF funds type 1 diabetes research in the areas of immune therapies, beta cell therapies, glucose control and complications therapies. A recent survey of Australian type 1 diabetes researchers conducted by JDRF found that 82% of respondents had been supported by JDRF funding. Funding for major research grants and fellowships are centralised through JDRF International to ensure international competitiveness and to avoid duplication, however JDRF Australia also locally funds a number of research programs directed at supporting the specific needs of Australian researchers and recognising excellence in research AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 19

22 3 Funders Available grants and awards People Support Awards Postdoctoral Fellowships are offered to researchers not more than five years post PhD, MD or equivalent, with no faculty appointment. Fellowships are for up to US$53,440 per year, for 3 years duration. The Fellowship primarily covers the stipend with $5,500 allocated for research funds. JDRF funded one Fellowship in Australia in 2010 out of a total 25 funded internationally. So far in 2011, three Fellowships have been funded in Australia. The Advanced Postdoctoral Fellowship is offered with the same eligibility and is worth US$90,000 per year, for three years duration. This Fellowship includes funding for a stipend and research funds. Recipients of this Fellowship can apply for the Advanced Postdoctoral Fellowship Transition Award. This one year grant is worth US$110,000 and is offered to people completing a Postdoctoral Fellowship who is in the first year of a faculty appointment. A total of thirteen Advanced Postdoctoral Fellowships were funded internationally in 2010, with none being awarded to Australian researchers. The JDRF Career Development Award is offered to researchers at least three, but no more than seven years post PhD, MD or equivalent. Recipients must hold a faculty appointment to be eligible for this award. The Award is worth US$150,000 per year for five years duration. Funds can go towards a research allowance including a single technician salary. One JDRF Career Development Award was awarded in Australia in 2009 out of a total three awarded that year, and one in 2010 out of a total five awarded that year. Available grants and awards Research Grants The Innovative Grant provides seed funding for highly innovative research with potential to change the current paradigm or to be groundbreaking. Applicants must hold a PhD, MD or equivalent and faculty position. The Grants are for US$110,000, for one year duration. Four Innovative Grants were awarded to Australian researchers in 2010 out of a total 24 awarded internationally. The Early Career Patient-Oriented Diabetes Research Award provides five years of funding to investigators pursuing type 1 diabetes related clinical investigation. Recipients are in the later stages of training and the awards include the ability for recipients to transition to independent appointments. This award has not been awarded to any Australian researches in the past. The High Priority, Short-Term Bridge Award is designed to support the research of investigators whose research is being curtailed or delayed by failure to receive funding. The award is available to researchers who have failed to receive funding but scored within 10% of the funding pay line for a research funding agency up to a year prior. The award is to the value of US$55,000, for one year duration. In 2010, no High Priority, Short-Term Bridge Awards were awarded in Australia, one was awarded in Australia in JDRF offers a range of Strategic Research Agreements single project, multi-project and clinical. These agreements are developed as a partnership between investigators and JDRF scientific staff that support a range of research project, programs, and early stage clinical trials. Partnerships between JDRF and biotech and pharmaceutical companies are supported through Industry Discovery & Development Partnerships Program, which can fund up to US$5M per program. This program aims to promote for-profit interest in JDRF s mission, to develop meaningful relationships with biotech and pharmaceutical partners focused on the discovery, development and commercialisation of therapeutics for type 1 diabetes and its complications. JDRF periodically issues Requests For Applications (RFAs) to solicit applications in specific areas of research. Two grants were awarded in Australia in 2009, and four in 2010 through the Requests For Applications mechanism. 20 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

23 Funders 3 Available grants and awards Australian specific awards JDRF may also offer grants restricted to a specific region or country. Current Australia-restricted opportunities are described in this section. Australia-specific research grants usually result from JDRF partnerships with government or other organisations, and focus on a specific research area. These grants are offered through the RFA mechanism. From a series of basic research grants were awarded to the value of US$3.3 million in the area of islet replacement. These grants were funded through the Islet Transplantation Program, under JDRF management. The most recent round of RFAs released to Australian type 1 diabetes researchers was in the area of clinical research. These RFAs were released through The Australian Type 1 Diabetes Clinical Research Network. JDRF offers up to two PhD Top-Up Scholarships per year, to students with an NHMRC Scholarship in the field of type 1 diabetes. Two Scholarship Top-Ups are awarded per year at a value of $6,000 per year each, on top of the NHMRC Scholarship. This program was supported through a partnership with the Macquarie Group Foundation from 2008 to JDRF offer funding through the Travel Grant Program to researchers in the early stage of their career either PhD students or not more than five years post-doctoral. Grants are a maximum of $4,000 and 50,000 is awarded through this program every year. This program was supported through a partnership with the Macquarie Group Foundation from 2008 to JDRF Australia runs a partnership with the Royal Australian College of Practitioners to offer a clinical researcher Fellowship. The Fellowship is awarded every two years to a clinician researcher pursuing research in type 1 diabetes and its complications and is worth $50,000. Ramaciotti Foundations The Ramaciotti Foundations 19 are one of the largest private contributors to biomedical research in Australia. The Foundations support researchers in biomedical research through a range of mechanisms targeting emerging researchers as well as established researchers. Available grants and awards The Ramaciotti Foundations offer an Establishment Grant for emerging researchers in biomedical research worth up to $75,000, and an Equipment grant to the same value to enable researchers to purchase major equipment. In 2010, the Ramaciotti Foundations awarded one equipment grant to a research group pursuing type 1 diabetes research. The Ramaciotti Foundations offer a major research award every two years. The Biomedical Research Award is a $1 million grant presented to a researcher or group of researchers in the field of biomedical research. The Ramaciotti Medal is offered annually for an outstanding contribution to clinical or biomedical research, or healthcare delivery. The Medal recipient also received a grant of $50,000. The recipient must have previously received support from Ramaciotti Foundations and be peer nominated. Rebecca L. Cooper Medical Research Foundation The Rebecca L. Cooper Medical Research Foundation 20 is a private foundation that supports medical research in a range of fields including biological sciences, physiology, psychology and psychiatry. Available grants and awards The Foundation supports diabetes researchers through the provision of equipment grants and PhD scholarships. Rebecca L. Cooper Medical Research Foundation Research (Equipment) Grants are offered to a maximum of $22,500 to enable researchers to purchase equipment AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 21

24 3 Funders The Diabetes Research Foundation of Western Australia The Diabetes Research Foundation of Western Australia 21 (DRF WA) specifically funds Western Australian researchers in the field of diabetes. DRF WA have a mission to cure diabetes and focus funding support research into the causes of all types of diabetes, diabetes complications and potential cures for diabetes. DRF WA have a strong focus on people support for new and established diabetes researchers in WA with a priority on establishing and supporting scholarships, fellowships and travel grants, as well as directly supporting research. DRF WA established the Centre for Diabetes Research at the Western Australian Institute for Medical Research in 2005, and support a Chair of Diabetes Research. Available grants and awards The Foundation offers the Alex Cohen Diabetes Top-up Scholarship to support PhD students in receipt of an Australian Postgraduate Award at the University of Western Australia, who are undertaking innovative research in the field of diabetes. The foundation further supports a number of researchers at other institutes and hospitals in Western Australia through research grants and a people support program. 3.4 International funders of type 1 diabetes research in Australia Access to international funders is an important avenue for type 1 diabetes researchers in Australia to secure funding for research. There are a range of international funders that have been identified by the type 1 diabetes research community as major financial supporters of Australian type 1 diabetes research. Despite this, the majority of type 1 diabetes researchers in Australia rely on domestic funding sources. There may be a number of reasons for this ranging from a lack of visibility of international funders, lack of understanding of eligibility of international or overseas grants, or a relative lower competitiveness of Australian researchers when applying for international grants. The 2010 Standing Committee on Industry, Science and Innovation report on Australia s International Research Collaboration 22 found that the research community perceived a range of impediments to accessing international funding opportunities. These included the level of bureaucracy involved and a lack of knowledge of funding opportunities and grant schemes. The report recommended that the Department of Industry, Science and Innovation have a role in communicating funding opportunities from the NIH to the Australian research community. NIH offers research grants to internationally based researchers. However, in order to be competitive for these grants, international researchers must offer unusual talent, resources, populations, or environmental conditions that are not readily available in the US, while still offering health benefits to the American population 23. The Special Statutory Funding Program for Type 1 Diabetes Research, administered by NIDDK, specifically allocates funding for type 1 diabetes research. The initial provision for this dedicated funding was made in 1997, and has undergone four rounds of renewal to date. The program has dedicated US$1.89 billion to diabetes research throughout the period of its operation from JDRF played an integral role in securing the extension of this program through to House of Representatives Standing Committee on Industry, Science and Innovation (2010) Australia s International Research Collaboration. Commonwealth of Australia. Canberra. 23. National Institutes of Health. Special Guidance for Foreign Research Opportunities. Available online at Evaluation Report on the Special Statutory Funding Program for Type 1 Diabetes Research. Report to Congress (2010) Department of Health and Human Services, National Institutes of Health, and National Institute of Diabetes and Digestive and Kidney Diseases. 22 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

25 Funders 3 The Program has enabled the creation of over 30 research consortia and clinical trial networks 25 focused on research into type 1 diabetes and its complications, and a range of research resources available to the wider type 1 diabetes research community 26. While some of the funding opportunities through the Program have been restricted to US researchers, a number have been made available to international researchers. There are a range of international funders that offer funding to type 1 diabetes researchers in Australia through internationally competitive grant schemes such as NIH, and others through international clinical trial networks. This section outlines the major international funding sources of type 1 diabetes researchers in Australia. The table below provides a summary of these funders. Table 3.3 International funders of type 1 diabetes research in Australia Funding body Focus Relevant grants and programs Immune Tolerance Network tolerance.org/ National Institutes of Health TrialNet To accelerate the clinical development of immune tolerance therapies To seek fundamental knowledge about the nature and behaviour of living systems and the application of that knowledge to enhance health, lengthen life, and reduce the burdens of illness and disability To prevent, delay and reverse the progression of type 1 diabetes Clinical trial funding Project Grant, Program Grant Clinical trial funding Immune Tolerance Network The Immune Tolerance Network 27 (ITN) is a clinical research consortium sponsored by the National Institute of Allergy and Infectious Diseases and JDRF. ITN supports phase I/II clinical trials of toleranceinducing strategies in a range of immune conditions including type 1 diabetes. Support is available to academic, industry and government investigators. Available grants and awards Whilst the ITN is currently active in Australia, there are no currently funded type 1 diabetes trials domestically. In 2011, ITN has five active trials in type 1 diabetes, all of which are based in the US. ITN accepts proposals through a year round proposal review system. The majority of new clinical trials adopted by the ITN begin as Concept Proposals from independent investigators. Alternatively ITN may solicit proposals through strategic assessments. National Institutes of Health The NIH offers a wide range of people support programs and research grant programs that are available to researchers in the US. A small number of these programs are available to international researchers. Available grants and awards In the past several years, the NIH has funded two grants in type 1 diabetes research in Australia. The total amount of type 1 diabetes research being funded in Australia by the NIH in this period has varied between US$250,000 and US$400,000 per year. In 2010, a total of US$9.2M was awarded by the NIH to Australian researchers AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 23

26 3 Funders Type 1 Diabetes TrialNet Type 1 Diabetes TrialNet 28 is a clinical trial network that conducts type 1 diabetes intervention and prevention trials, funded by NIDDK. There are 18 clinical centres, 15 major affiliates, and multiple affiliated groups throughout North America, Canada, Europe and Australia. Type 1 Diabetes TrialNet welcomes applications for clinical trials from international investigators in academia and industry. Available grants and awards There are currently four open trials internationally two of which are active in Australia. Current TrialNet trials in Australia are the Natural History Study of the Development of Type 1 Diabetes trial and the Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus trial, which are both recruiting in Australia. TrialNet activities in Australia are supported by JDRF funding. TrialNet co-sponsors several studies with ITN and co-sponsors the ANZDiab network with the Diabetes Vaccine Development Centre (DVDC). ANZDiab trials comprise current TrialNet trials active in Australia, the Intranasal Insulin II trial, and the BHT 3021 Proinsulin Plasmid trial. 3.5 Commercial funding of type 1 diabetes research in Australia A number of global pharmaceutical and medical device companies have presence in Australia with the majority of them investing significantly in various research activities including clinical and translational research. Some of those companies are committed to supporting Australian research through various funding programs and mechanisms incorporating grants and fellowships. Research investment in Australia from the commercial sector is, however, heavily weighted to specific therapeutic areas such as oncology and is geared towards pre-clinical research and clinical trials (see below). 12% Basic research 20% Preclinical research 44% Clinical trials 24% Investigator initiated trials Figure 3.5 Industry sponsored clinical research in Australia across all fields in Although Australia has one of the highest incidences of type 1 diabetes in the world and the cost to the health system long term is significant, it remains an area with limited activity within the commercial sector. Many recent developments in the prevention and management of type 1 diabetes have produced clinical trial activity overseas that has not yet extended to Australia. In particular, clinical research activity in the areas of immune therapy and beta cell therapies have to date been limited. In the US, a number of partnerships between JDRF and Industry have been established and continue to be established in order to drive research activity in the areas of immune therapies, beta cell therapies and complications therapies, as well as glucose control. Such partnerships will now need to be translated into the Australian research environment. As mentioned, the resources in the pharmaceutical and biotechnology industries involved in the type 1 diabetes space in Australia remain largely unmapped. Future editions of this resource will be expanded to map this environment and the resources available to researchers Medicines Australia (2010) Medicines Australia Facts Book. 2nd Edition. 24 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

27 Infrastructure 4 4. Infrastructure Infrastructure such as clinical trials support, patient data, clinical samples and animal models are invaluable tools for research. The Australia Type 1 Diabetes Research Agenda 30 identified the area of research enablers and accelerators as of particular importance in achieving research outcomes. A systematic, qualitative analysis was performed on the infrastructure environment for type 1 diabetes researchers in Australia. The aims of this assessment were to capture and collate the major resources utilised by this research community, to understand what the major needs were in this space, and ultimately to improve the environment contributing to research output in the field of type 1 diabetes and diabetes. This section provides an overview of the major infrastructure available to type 1 diabetes researchers in Australia, including commentary on the potential for greater accessibility to these resources. 4.1 Animal facilities Animal facilities are an important resource for the type 1 diabetes research community, particularly at the basic research end of the spectrum. There are a plethora of animal models of type 1 diabetes, some of which are commonly available in Australia and others which are not. This section outlines the animal facilities that are currently available for Australian researchers in the field of type 1 diabetes, including animal models of type 1 diabetes and transplantation. The table below summarises the animal resources currently available to type 1 diabetes researchers in Australia. Table 4.1 Animal facilities and models in Australia relevant to type 1 diabetes research Name Details Accessibility for research Australian BioResources animal housing and breeding facility (NSW) The Gene Mine breeding program (WA) Mouse models of diabetes and its complications (National) The NHMRC Australian PhenomeBank (ACT) The National NHMRC Baboon Colony (NSW) Pig models of xenotransplantation (NSW, VIC, SA) Shared small animal holding and breeding facility Breeding program developing congenic type 1 diabetes mouse strains Various models of type 1 diabetes and its complications Central repository of mouse strains used in medical research Large non-human primate medical research facility Genetically modified pigs, pig-to-baboon models of xenotransplantation Available at a cost to all Australian researchers Available through collaboration Various, depending on institution and strain The database is open to all researchers, cryopreservation services available at cost Available to researchers with ethically approved, feasibility approved studies Animal models and expertise available to collaborators 30. Australian Type 1 Diabetes Research Agenda Partnering science, government and the community. 2nd edition. (2010) The Juvenile Diabetes Research Foundation. Available online at AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 25

28 4 Infrastructure Australian BioResources animal housing and breeding facility Australian BioResources 31 (ABR) is a shared animal holding and breeding facility based in Mossvale, NSW primarily designed for mouse lines. ABR currently houses approximately 40,000 mice for eight institutes and universities. As well as housing and breeding mouse strains, ABR offers cryopreservation to preserve strains when maintenance of live animals is not required. Accessibility The facility owned and operated by Garvan Institute of Medical Research, however is available at a cost for use by all Australian researchers with appropriate institutional and animal ethics accreditation and approvals. The Gene Mine breeding program The Gene Mine breeding program, based at the Centre for Diabetes Research 32 at the Western Australian Institute for Medical Research, aims to produce 1000 congenic mouse strains to locate genetic regions associated with type 1 diabetes. The project has produced mouse strains which have narrowed the regions showing diabetes susceptibility genes and are currently in the process of testing these strains to determine their diabetes status. This project aims to develop a better mouse model for type 1 diabetes. Accessibility These mice are intended to be used for research through direct collaboration. Mouse models of diabetes and its complications There exist a wide range of mouse models of type 1 diabetes and its complications. As well as generic inbred mouse and rat strains, there are several models utilised in Australia that are specific to type 1 diabetes and its complications. Mouse models developed, maintained and used in type 1 diabetes research in Australia include: Non-obese diabetic (NOD) mice, including rotavirus free NOD mice and NOD mice sequenced to ensure 99.9% genetic fidelity; Various genetic modifications on the NOD background including gene knock out and transgenic mice, for example humanised T cell receptor on a NOD background; SCID and NOD SCID mice; RIP-OVA hi and RIP-OVA lo mice expressing ovabulmin under the insulin 2 promoter, and OT-1 and OT-2 mice expressing transgenic T cell receptors from CD8 and CD4 T cells respectively, each specific for different ovalbumin peptides; Models of portal vein and kidney capsule islet transplantation using various C57BL/6 or BALB/c transgenic strains or SCID mice, undergoing chemically induced diabetes; and Various models of kidney disease. Accessibility There are various factors that affect the capacity for different animal models to be shared, some of the hurdles to sharing animals are outlines in the Infrastructure Needs section. There are mechanisms for cataloguing and sharing animals that are available in Australia, which reduce some of these hurdles. One example is the Australian PhenomeBank AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

29 Infrastructure 4 The NHMRC Australian PhenomeBank The NHMRC Australian PhenomeBank 33 (APB), based at the Australian National University in Canberra is a non-profit repository of mouse strains used in medical research. Whilst not specific to type 1 diabetes research, the facility acts as a central database of mouse models including models of type 1 diabetes and its complications. The database allows researchers to consolidate resources by avoiding strain duplication and facilitates research collaboration. As well as maintaining the database, the APB maintains cryopreserved embryos and sperm in order to archive strains submitted by researchers and to facilitate sharing of strains between researchers. The cryopreservation of mouse models circumvents the excessive costs associated with breeding and maintaining mouse strains when not in use for research, without losing the resource. The APB was established with an NHMRC Enabling Grant. Accessibility The facility offers researchers free access to APB database which can be searched for mutations in particular genes, for strains with transgenic alterations and for mice with particular phenotypes. While the sperm cryopreservation service is offered free, there are costs associated with embryo cryopreservation and strain reanimation in order to cover the costs of the facility. The National NHMRC Baboon Colony The National NHMRC Baboon Colony is supported by an NHMRC Enabling Grant. The colony provides access to large non-human primates to support a diverse range of research, including type 1 diabetes. The colony has been a particularly important resource in the development of xenotransplantation research through providing models to assess safety and develop techniques. Accessibility The colony is currently utilised by researchers in type 1 diabetes to develop and research models of wound healing and xenotransplantation. Research proposals are initially assessed for feasibility by the colony veterinarian and director. Access to the colony is only provided once researchers have approval from their local Animal Ethics Committee as well as approval from the Animal Welfare Committee governing the colony. Pig models of xenotransplantation Several research groups in Australia are investigating the potential for xenotransplantation as a therapy for type 1 diabetes. Working in collaboration there have been developed several strains of genetically modified pigs which are currently used in pig-to-baboon models of xenotransplantation, utilising the National Baboon Colony. Researchers are based at St Vincent s Hospital Melbourne, the University of Adelaide, and Westmead Millennium Institute. The strains include the Gal KO, a triple transgenic for CD-55, CD-59 and H transferase on the Gal KO background, with more genetic modifications in process. The resource includes a breeding facility in Victoria however, due to the high cost associated with maintaining large animals, researchers also utilise abattoir-sourced pig uteruses for initial stages of experimentation. Accessibility There is a possibility of sharing this resource with other researchers through collaboration, however the owners of this resource note that it is not currently shared locally. It is noted that this resource is shared with international researchers, both in terms of expertise and tissue. It is also recognised that a major hurdle to sharing this resource is the high cost associated with large animal models, which makes this work unfeasible for a lot of researchers AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 27

30 4 Infrastructure 4.2 Patient data Patient databases range greatly in terms of the information housed and their size and scope. Databases that are relevant to type 1 diabetes research in Australia exist on several levels, from incidence registers covering the majority of people with diabetes in Australia, to international genetics databases, and institutional and project based databases. This section outlines the patient databases that are currently available for Australian researchers in the field of type 1 diabetes, including databases at these different levels. The table below provides a summary of the incidence registers relevant to type 1 diabetes that exist in Australia, and points out their current accessibility for research. Table 4.2 Diabetes incidence registers in Australia Name Details Accessibility for research Australasian Paediatric Endocrine Group state-based incidence registers The National Diabetes Register national-diabetes-register/ 10,000 incident cases of people under 15, some now in adulthood 131,000 people using insulin to treat diabetes There is currently an informal process to access de-identified data from these databases, through APEG Data available for research, cohort available for clinical trial recruitment The National Diabetes Services Scheme 897,867 people with diabetes Cohort available for clinical trial recruitment Australasian Paediatric Endocrine Group state-based registers The Australasian Paediatric Endocrine Group 34 (APEG) maintain a register of incident cases of diabetes in people under fifteen years old to provide secondary ascertainment data on new cases of insulin treated diabetes for the National Diabetes Register. The register has been active since Consent for inclusion in the APEG register is sought when children and adolescents are diagnosed with diabetes. The database currently contains approximately 10,000 records with around 1,000 new incident cases per year. A number of these records are of people with diabetes who have now transitioned into adulthood and adult diabetes centres and as such are not active. The database contains a range of clinical data. Accessibility The register provides statistical information and has been used to publish multiple studies on trends in childhood diabetes across Australia. There is an informal process to access de-identified data from these databases through APEG for research purposes, however the database is currently not heavily utilised for research AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

31 Infrastructure 4 The National Diabetes Register The National Diabetes Register 35 (NDR) is a database of approximately 131,000 people with diabetes who have started insulin treatment from January 1999 and who have consented to be on the database. This includes people with type 1 diabetes, and some people with type 2 diabetes and gestational diabetes. The NDR database is housed at the Australian Institute of Health and Welfare (AIHW) and collates data from the NDSS and the APEG state-based registers. The database includes basic information on age, sex, diabetes diagnosis and insulin treatment, but does not collect clinical information on the registrants. As well as providing information on the national incidence of type 1 diabetes, one of the major functions of the NDR is to provide a database of patient information for epidemiological and clinical studies of type 1 diabetes. Accessibility for research The database is made available to diabetes researchers for specific research projects. While requests for de-identified data may not require ethics approval, there is a general requirement of project approval by both the researcher s institutional ethics committee and the AIHW Ethics Committee. The project must also demonstrate scientific merit, as shown by funding by a competitive peer-reviewed grant process. The NDR can also be used to approach consenting registrants for recruitment into clinical studies. There are associated costs for researchers wishing to access information from the NDR including any costs associated with contacting potential recipients, data-manipulation, project review and an ethics committee review charge. Currently the database is not heavily used for research. While two major projects utilised the NDR in 2009, there were no major projects in 2008 or The National Diabetes Services Scheme database The National Diabetes Services Scheme 36 (NDSS) is an initiative of the Australian Government, administered by Diabetes Australia. The NDSS is in place to deliver subsidised medication and diabetes related products and well as information and support to people with diabetes. Accessibility for research The NDSS is available to researchers as a tool for clinical trials recruitment, by giving them the ability to contact people on the NDSS living with type 1 diabetes who have indicated that they are interested in participating in research. There is potential for the information in the NDSS to be harnessed for use by researchers in epidemiological studies of type 1 diabetes however, currently this capacity has not been utilised. Databases housing information on people with type 1 diabetes may be created around both clinical cohorts and as the result of research projects, as such, the scope of these databases is wide and varied. This section outlines the genetic and clinical databases that are relevant to type 1 diabetes researchers in Australia. Databases covered in this analysis range from large international collections of data to smaller institutionally based databases. Table 4.3 outlines the resources currently available in this space AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 29

32 4 Infrastructure Table 4.3 Clinical and genetic databases relevant to type 1 diabetes research in Australia Name Details Accessibility for research BioGrid Australia Childrens Hospital Westmead clinic database Royal Melbourne Hospital clinic database Royal Prince Alfred Hospital clinic database St Vincent s Hospital Melbourne clinic database St Vincent s Hospital Sydney clinic database T1DBase Type 1 Diabetes Genetics Consortium 59,000 diabetes patient records, across five data owners 4,000 records from paediatric diabetes centre, 1,300 active, 100 incident cases per year Longitudinal database of adults with type 1 diabetes Longitudinal database of 500 adults with type 1 diabetes Database 400 people with type 1 diabetes, clinical database not electronic Longitudinal complications screening database of over 1000 people with diabetes, approximately 30% type 1 Web based resource focused on type 1 diabetes genetics and genomics Genetic database and clinical samples from over 6,000 families with at least two siblings with type 1 diabetes Linked genetic and clinical databases available to researchers with approved studies The clinic database is not currently shared for research purposes The clinic database is available through BioGrid Available to be shared with collaborators Not currently used for research Available to be shared with collaborators A set of datasets and tools, across multiple species is openly available to researchers Prioritised access to contributing centres and to consortium members, delayed access to other researchers BioGrid Australia BioGrid Australia 37 is a non-profit organisation that provides a platform for researchers to access and analyse genetic and clinical data. De-identified patient data is linked to the database and can be made available to authorised clinicians and researchers for research projects. This large clinical database provides the means to improve patient care as well as contributing large datasets for clinical research. Originally set up for cancer research, the platform has recently been adapted by diabetes clinicians and researchers. The power of BioGrid comes from the ability to link together numerous datasets and provide researchers with large blocks of patient data for research. BioGrid was highlighted in the Strategic Roadmap for Australian Research Infrastructure 38 as a possible model for implementing an overarching national bioinformatics system with the ability to link different data types. This was noted as an infrastructure requirement for Australian research Department of Innovation, Industry, Science and Research (2008) Strategic Roadmap for Australian Research Infrastructure. 30 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

33 Infrastructure 4 Accessibility BioGrid contains over 59,000 patient records in type 1 and type 2 diabetes from five centres in Victoria, with the aim of expanding to other states and linking with other databases, such as the NDSS database. Current datasets include clinical data from the Royal Melbourne Hospital, St Vincent s Hospital in Melbourne, as well as data from a number of preventative trials conducted by investigators at the Royal Melbourne Hospital, with multiple datasets including results of complications screening. Datasets are controlled by their owners and applications for use of each dataset must be approved by the data owners as well as by BioGrid. Childrens Hospital Westmead clinic database The clinic database from the Childrens Hospital Westmead diabetes centre currently houses approximately 4,000 records. Although this is a paediatric centre, many of the records housed by the database are from patients who have transitioned to adult clinics and as such are not active on the database. The database incorporates approximately 1,300 active patient records, including around 100 incident cases per year. The database is paired with a small number of clinical samples collected for various projects however, there is currently no routine collection of clinical samples. The clinic hopes to start routine collection of clinical samples to complement the database in the near future. Accessibility The clinic database is not currently shared for research purposes outside of the Westmead research group. Royal Melbourne Hospital clinic database The Royal Melbourne Hospital diabetes clinic maintains a longitudinal patient database. The patient population is approximately adults from urban and rural areas. The database contains information on treatment and complications status. Accessibility The Royal Melbourne Hospital maintains clinical records on BioGrid, the data summary is available on the BioGrid website and researchers can apply to access the data for research. Royal Prince Alfred Hospital clinic database The Royal Prince Alfred Hospital in Sydney has an adult diabetes centre. The centre sees approximately 500 patients a year ranging from age 16 to elderly. The patient makeup has been estimated to be approximately 5% new onset, 40% with established diabetes and 55% with complications. The clinic currently uses an electronic database. The clinic does not currently perform any routine blood collection but is interested in starting this. Accessibility RPAH are interested to participate in clinical trials and the clinic database is available to be shared with collaborators pending any issues surrounding ethics being resolved. St Vincent s Hospital Melbourne clinic database The St Vincent s Hospital Melbourne diabetes centre does not maintain an electronic database of patient data and is currently working to find an appropriate database to house patient records. The centre retains clinical data of approximately 400 people with type 1 diabetes in its database. Accessibility There is limited access to this collection due to institutional ethics. St Vincent s Hospital Sydney clinic database The diabetes clinic at St Vincent s Hospital Sydney currently uses a paper based system, however does maintain a longitudinal database of progressing 18-month complications screening results. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 31

34 4 Infrastructure This database contains records of over 1000 diabetes patients with an estimated 30% having type 1 diabetes. Information is stored in an in-house database developed at the Garvan Institute Accessibility The complications screening database is currently only used for research internally, but there is the possibility of sharing the database with collaborators. T1DBase T1DBase 39 is a resource for researchers in the genetics and genomics of type 1 diabetes, which provides a range of data sets and data analysis tools to support researchers in this field. Datasets include: Annotated genomic sequences for suspected type 1 diabetes susceptibility regions; Genetic and microarray data; Functional annotation of genes active in beta cells; Global datasets, generally from the literature; and T1DGC data is available to T1DGC members through the T1DBase website. As well as data on human genetics, T1DBase provides researchers with a database of congenic NOD mouse strains containing genetic material from a range of other strains. Accessibility A set of datasets and tools, across multiple species is openly available to researchers. The Type 1 Diabetes Genetics Consortium The Type 1 Diabetes Genetics Consortium 40 (T1DGC) is an international research initiative with the aim of identifying genes associated with type 1 diabetes and determining the risk of an individual developing the disease. T1DGC aimed to recruit over 6,000 families with at least two siblings with type 1 diabetes. The T1DGC database contains phenotypic information, such as HLA genotyping and the presence of autoantibodies from these families. As well as data, DNA, serum and plasma samples are available. With the consortiums activities coming to an end, nearly all samples have been transferred to NIDDK Central Repositories. All requests for samples must be submitted through an application to the NIDDK Central Repository, as outlined on their website 41. The T1DGC involved five centres around Australia for recruitment, completed in January T1DGC are now undertaking the transfer of all samples (cell lines, DNA, serum and plasma) to the NIDDK Central Repositories. Accessibility T1DGC prioritises access to contributing centres, then to consortium members, and finally non-members, by placing a lag on data availability. 4.3 Human cells and tissue Similarly to the collection of patient databases, the range of human tissue collections that are available to type 1 diabetes researchers in Australia is wide and varied. Human tissue collections may exist as a biobank or as a sample collection. The NHMRC define biobanks as a collection of human tissue samples that have been established for sharing for research purposes 42. Many of the biobanks and sample collections that exist in Australia are project related, however Australian researchers also have access to a number of international biobanks and sample collections. This section outlines the human cell and tissue collections that are currently utilised for research in type 1 diabetes in Australia. Table 4.2 provides a summary of these resources, highlighting their current availability for research Biobanks Information Paper (2010) National Health and Medical Research Council. Available online at 32 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

35 Infrastructure 4 Table 4.4 Human cell and tissue collections relevant to type 1 diabetes research in Australia Name Details Accessibility for research Antibody screened at risk blood collection Royal Melbourne Hospital (VIC) Australian Childhood Diabetes DNA Repository (WA) Baker IDI Biobank (VIC) core_facilities/biobank/ Diamantina autoimmunity division sample collection (QLD) Environmental determinants blood collection Murdoch Childrens Research Institute (VIC) Human embryonic stem cell lines Monash University (VIC) Islet Transplantation Program (NSW, VIC, SA) LANDMark study biobank and cohort (QLD) opt/research/anterioreye. jsp#landmark Network for Pancreatic Organ donors with Diabetes (International) Princess Margaret Hospital clinical database and sample collection (WA) St Vincent s Hospital Melbourne biobank proposal (VIC) St Vincent s Hospital Melbourne sample collection (VIC) Blood from 20,000+ people in high-risk population screened for autoantibodies and clinical data DNA from saliva samples from 4,500 family trios of a child with diabetes and their biological parents Blood from 6,000+ non-diabetic people, and people with type 1 and type 2 diabetes Piecemeal, different sample types and depth of clinical data from 550 type 1 diabetes and non-diabetic people Blood from 300 children with type 1 diabetes and 600 nondiabetic controls, environmental and genetic data Genetically modified hes cells for type 1 diabetes research Human islet cells, RNA and protein samples, from non-diabetic donors Plasma, serum, immune cells, limited foot biopsy and tear samples from 155 people with type 1 diabetes and 65 nondiabetic controls Pancreata, spleen, lymph nodes and peripheral blood from people with type 1 diabetes or who are positive for islet auto-antibodies Blood, serum and DNA from people with type 1 diabetes This is a proposed resource and will house current tissue samples from SVI as well as establishing ongoing collection Blood and urine samples from adults with type 1 diabetes and nondiabetic controls Data is available through BioGrid. Samples are potentially available to collaborators DNA samples are made available to HREC approved studies Blood cells, serum or DNA samples are made available to HREC approved research Currently not shared and only limited capacity for sharing Potentially available to collaborators Shared with collaborators, restrictions in place for certain research institutions Tissue is made available to type 1 diabetes researchers Samples are available for research with HREC approval Samples allocated to research reviewed for scientific merit and feasibility Potentially available to collaborators Proposed that the resource will be open to external researchers There is limited access due to institutional ethics AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 33

36 4 Infrastructure Antibody screened at risk blood collection This resource, based at the Royal Melbourne Hospital, contains over 20,000 blood samples of direct relatives of people with type 1 diabetes, who have been tested for the presence of autoimmune antibodies. The associated clinical data is housed on the BioGrid Australia database. Accessibility This resource is not currently used for research, however there is potential for sharing. It is noted that this collection contains samples and clinical data from many people with anti-gad antibodies and as such may be useful for research into Latent Autoimmune Diabetes of Adults. Australian Childhood Diabetes DNA Repository The Australian Childhood Diabetes DNA Repository 43 (ACDDR), based at the Western Australian Institute for Medical Research in Perth, was developed in conjunction with T1DGC. The repository houses DNA from saliva samples of 1,500 Australian families with children affected by both type 1 and type 2 diabetes. Samples are collected from four diabetes centres in Perth, Melbourne and Sydney. Each family collection consists of DNA from a child with diabetes and their biological parents, as such the repository houses 4,500 samples in total. ACDDR is supported with an NHMRC Enabling Grant with funding and data collection ending in Accessibility DNA samples are made available to HREC approved studies. NHMRC funded researchers have access to samples for free, while other researchers can obtain samples at a cost. Data from the T1DGC central database is also available through ACDDR. Baker IDI Biobank The Baker IDI Biobank 44 (formerly The Alfred and Baker Gene Bank) is a collection of frozen human blood samples from healthy people, people with type 1 and type 2 diabetes, and people with cardiovascular disease and risk factors. Established in 2000, this resource currently has over 6,000 samples and the collection is growing at samples per year. Accessibility Baker IDI Biobank samples are made available to researchers for HREC approved research requiring human blood cells, serum or DNA. The resource is primarily used internally by researchers at the institute, however there is the capacity to release samples to external researchers. This function is overseen by a steering committee which approves applications for samples from external researchers on a case by case basis AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

37 Infrastructure 4 Diamantina autoimmunity sample collection This resource contains de-identified information and samples from a cohort of approximately 550 children collected through a five year collaboration with Mater Childrens Hospital. The database represents a combination of healthy children, children with type 1 diabetes and the siblings of children with type 1 diabetes. The samples and clinical data captured by this database are varied depending on the specific project for which they were collected. As a result the resource comprises a range of sample types some PBMC, cytoplasmic and nuclear extracts, DNA and RNA, and serum. Likewise, the detail and depth of clinical data associated with the collection ranges from minimal to more detailed, some of the collection is longitudinal. Accessibility Currently, the collection is not shared and the majority of the resource has low potential for sharing. The collection of samples has been relatively piecemeal to satisfy specific project requirements, and as such the collection is not comprehensive. However, the group intends to build this resource over time, providing potential for a shared resource to be established. The database structure was specifically designed for this resource and is very functional, with the ability for data owners to control access to their datasets. There is potential for the database structure to be shared as a platform for biobank management for separate resources, or as a means to consolidate multiple tissue collections. Environmental determinants blood collection This resource, based at the Murdoch Childrens Research Institute, contains blood samples and information from a childhood cohort from the Royal Childrens Hospital in Melbourne. The collection contains stored blood samples from 300 children with type 1 diabetes and 600 control children. The samples are paired with environmental and genetic data. Accessibility This collection is currently only used within this research group, however there is potential for this resource to be made available for research to collaborators. Human embryonic stem cell lines Modified human embryonic stem cell (hes) products, such as a hes cell line that has been genetically modified to produce green fluorescent protein when insulin is expressed, are a valuable tool in research investigating alternatives for beta cell replacement. hes cell lines are developed by researchers at Monash University. Accessibility While the owners of this resource are willing to share hes cell lines with researchers requesting this resource, they have found that a major hurdle to sharing these resources is the stipulations put on hes work by religious institutional oversight boards. Several research institutions in Australia are restricted from pursuing research involving hes, and as such this places a limitation on the accessibility of these resources to researchers in these institutions. Currently, these resources are not widely shared with researchers in Australia. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 35

38 4 Infrastructure The Islet Transplantation Program The Islet Transplantation Program 45 (ITP) supports basic research through direct funding of projects and through the provision of pancreatic and other tissue to external research programs. When criteria are not reached for use of pancreata in the clinical program, the ITP provides human islets to Australian type 1 diabetes researchers with institutional ethics approval, when appropriate consent is obtained from donor families. RNA and protein samples from pancreatic tissue are also available. The majority of pancreata are healthy, however the program has also handled a small number of diabetic pancreata which have been provided for research. Accessibility Currently, between 30 and 50 islet isolations per year being provided to 11 external groups, and non-islet pancreatic tissue is supplied for research into progenitor cells or as control tissue for experiments. The ITP will end in June 2011 as a research program. The clinical program has applied for funding through the Nationally Funded Centres program to support clinical islet transplantation, however there is currently no provision to continue the basic research program which supports the distribution of pancreatic tissue to researchers. LANDMark study biobank and cohort The LANDMark study 46 is a five year longitudinal study from which plasma, serum, buffy coat, foot biopsy and tear samples are collected from a cohort of approximately 320 people, including people with type 1 diabetes and healthy controls. The LANDMark biobank currently houses approximately 10,000 samples from this cohort, including blood, urine and tear. The biobank samples are distributed between Brisbane and Manchester, UK. The Brisbane arm of the biobank currently houses samples from 220 participants (155 from people with type 1 diabetes and 65 controls from the Princess Alexandra Hospital) with approx 26 samples per participant, including baseline and one year follow-up time points. This cohort of patients will be followed for a total of five years. It is projected that the resource will continue to grow at up to 10,000 samples per year, with longitudinal samples added from this cohort for the duration of the five year study. Accessibility With a two year moratorium on the release of biobank samples being lifted in late 2010 the biobank samples are now available to researchers with HREC approval, however the biobank is not currently utilised. Network for Pancreatic Organ donors with Diabetes The Network for Pancreatic Organ donors with Diabetes 47 (npod) based in the USA, was developed by JDRF to collect human tissue and to make it available for type 1 diabetes research at no cost. npod facilitates collection and distribution to researchers a range of human tissue from donors with type 1 diabetes or who are positive for islet auto-antibodies. Tissue made available to researchers includes pancreata, spleen, lymph nodes and peripheral blood. The collection includes subsets of newly diagnosed, and Joslin medallists people who have lived with type 1 diabetes for 50 years or more. Accessibility Currently, four research groups in Australia have grants to access npod samples. Applications for access to tissues are reviewed by npod for scientific merit and feasibility of the proposed research AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

39 Infrastructure 4 Princess Margaret Hospital clinical database and sample collection The Princess Margaret Hospital in Perth is the sole paediatric diabetes centre in Western Australia and as such, has a state-wide population based patient group. For all type 1 diabetes patients, blood samples are taken at diagnosis and samples of blood, serum and DNA are kept for each patient. The value of these samples is enhanced by the coordination of tissue samples with corresponding clinical data from each patient. The clinic maintains a longitudinal database containing information on HLA type, clinical data such as HbA1c, and results of complications screenings which are paired with the clinical samples. Accessibility The collection is generally underutilised. There is currently no formal process for sharing this resource with external researchers, but there is potential for sharing this resource with collaborators. St Vincent s Hospital Melbourne biobank proposal A joint initiative by partners on St Vincent s Hospital campus in Melbourne hopes to establish a biobank to house a range of human tissue samples, specifically relating to diabetes and other autoimmune disorders. The biobank will build on the collection of human tissue currently held by St Vincent s Institute from programs such as the Islet Transplantation Program. St Vincent s Hospital is currently seeking funding to support this initiative to be housed at the National Serology Reference Laboratory at St Vincent s Hospital in Melbourne. Accessibility It is proposed that the resource will be open to external researchers as well as St Vincent s Hospital campus researchers, however the mechanisms for sharing of this resource have not been established as yet. St Vincent s Hospital Melbourne sample collection This collection contains blood and urine samples from adults with type 1 diabetes and non type 1 diabetes controls. The collection supports complications research and as such, the cohort are an average of 40 years old, ranging from years old. As well as urine and blood, skin and ocular advanced glycation end products have been assessed. Due to the ongoing nature of this collection, many samples have been used or partially used, however collection is ongoing. Accessibility There is limited access to these resources due to institutional ethics. 4.4 Clinical trial infrastructure The development and conduct of clinical trials in type 1 diabetes in Australia are currently supported by a range of structures and platforms both within Australia and internationally. While some of these structures may not be specific to type 1 diabetes, they provide relevant resources to the type 1 diabetes research community. This section outlines the range of structures that are currently in place in Australia to support clinical trials in type 1 diabetes. Table 4.5 provides a summary of these resources. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 37

40 4 Infrastructure Table 4.5 Clinical trial structures in Australia relevant to type 1 diabetes research Name Purpose Key Outcomes Australia New Zealand Clinical Trial Registry (National) Diabetes Clinical Centre for Research Excellence (VIC) unimelb.edu.au Diabetes Vaccine Development Centre (NSW) Islet Transplantation Program (NSW, VIC, SA) The Australian Type 1 Diabetes Clinical Research Network (National) NHMRC Clinical Trials Centre (NSW) Princess Margaret Hospital clinical investigations unit (WA) Queensland Clinical Trials Centre (QLD) To provide an online registry of clinical trials in Australia To encourage and promote clinical research in diabetes and its complications To provide a platform to translate type 1 diabetes research into improved clinical outcomes To establish a clinical program of islet transplantation in Australia and support a research program Established in 2010, to better bridge the gap between basic research and clinical therapies development in type 1 diabetes in Australia To achieve best practice in health care and improve outcomes in Australia and internationally through the use of clinical trials research To provide a facility to run type 1 diabetes clinical trials To improve health by designing and managing clinical trials that provide high-quality evidence Searchable online database of registered clinical trials in Australia Six established, and ten supported research projects, eight fellowships for clinician researchers Currently funding and managing two clinical trials Supporting three clinical centres undertaking islet transplantation and sixteen research projects The Network released a Request For Applications in 2010, several trials are under review Supporting clinical trials in cancer, cardiovascular research and other areas Two-room facility in Princess Margaret Hospital Currently supporting fifteen clinical trials The Australian New Zealand Clinical Trials Registry The Australian New Zealand Clinical Trials Registry 48 (ANZCTR) is an online register of clinical trials being undertaken in Australia, New Zealand and globally. The ANZCTR was established in 2005 with an NHMRC Enabling Grant. The National Statement on Ethical Conduct in Human Research 49 states that before beginning the clinical phase of the research, researchers should register clinical trials in a publicly accessible register, however it is not currently mandatory for clinical trials in Australia to be registered National Health and Medical Research Council, Australian Research Council and Australian Vice-Chancellors Committee (2007) National Statement on Ethical Conduct in Human Research. Available online at 38 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

41 Infrastructure 4 Despite this, there exists a range of incentives for registration of trials. Certain funders, including JDRF, state that trial registration is a requirement of funding. In 2004, the International Committee of Medical Journal Editors have made registration of clinical trials a requirement for publication in the journals of this organisation 50, including Annals of Internal Medicine, British Medical Journal, JAMA, New England Journal of Medicine, The Lancet, The Medical Journal of Australia, and more. Available resources The ANZCTR has potential to assist investigators with patient recruitment by making information on the trial publicly and easily accessible to potential participants. The ANZCTR also facilitates information sharing by giving investigators visibility of other trials occurring in the region. A limitation of the registry is that there is no stipulation for trials to be registered at all, to be registered domestically, or on any one stipulated registry. As a result, there are a number of Australian trials that have been registered on other registries such as clinicaltrials.gov, in place of registration on the ANZCTR. Diabetes Clinical Centre for Research Excellence The Diabetes Clinical Centre for Research Excellence 51 (CCRE) was set up in 2007 with a CCRE grant from the NHMRC. The three key objectives of the diabetes CCRE are: Training and research career development; Translation of research into clinical practice; and Fostering new clinical research programs. With an emphasis on clinical treatment, the Diabetes CCRE has been designed to incorporate clinical research into both type 1, and type 2 diabetes. The Diabetes CCRE is currently involved in several longitudinal and epidemiological studies of type 1 and type 2 diabetes, studies in diabetes complications, and studies in islet transplantation. Available resources The Diabetes CCRE is heavily involved in training with the emphasis on providing support for post-doctoral fellows, and encouraging the participation of clinicians in clinical research. The Diabetes CCRE encourages investigators involved with the centre to provide mentorship and guidance to PhD students, postdoctoral fellows and other clinicians. The Diabetes Vaccine Development Centre The Diabetes Vaccine Development Centre 52 (DVDC) is a joint initiative of the NHMRC and JDRF with the purpose of establishing a platform for translational and clinical research in type 1 diabetes prevention and therapy. DVDC aims to identify, evaluate, support and coordinate a range of projects as approved by its Board. DVDC bridges the gap between fundamental research and full scale clinical product development. In 2011, DVDC supports two type 1 diabetes prevention trials and one preclinical study. In Australia, DVDC currently supports the Trial of Intranasal Insulin in Children and Young Adults at Risk of Type 1 Diabetes (INIT II). DVDC also supports a UK study, Islet autoantigen-derived peptides eluted from Human Leucocyte Antigen (HLA) class II molecules as vaccines for the immunotherapy of type 1 diabetes: a safety and proof of concept study in man. Accessibility DVDC has the capability to provide clinical trial support such as data management, and ethics and regulatory process administration on a cost recovery basis to clinical research. DVDC has some capacity to fund new research in prevention and at, or very close to, the clinical trial stage. 50. DeAngelis CD, Drazen JM, Frizelle FA, Haug C, Hoey J, Horton R, Kotzin S, Laine C, Marusic A, Overbeke AJPM, Schroeder TV, Sox HC and Van Der Weyden MB (2004) Clinical trial registration: a statement from the International Committee of Medical Journal Editors. Journal of the American Medical Association. 292(11) AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 39

42 4 Infrastructure Islet Transplantation Program ITP 53 is a major, cohesive clinical and basic research program that directly engages or supports the activities of over 130 researchers across four states. ITP is a joint initiative of JDRF and the NHMRC and the terms of operation of the program were from The ITP clinical program has established a platform of process, technology and expertise as a base for future cellular therapies, such as xenotransplantation or stem cell therapies. Islet transplantation is now being proposed by the clinical sites of ITP for funding through the Nationally Funded Centres program, as a means to continue the clinical program. Accessibility The ITP research program will come to its conclusion in June Islet transplantation as a clinical procedure for a selected group of patients is projected to continue via the Nationally Funded Centres program, however at this stage it is unclear whether this process will limit the number of islets available for research in the future. The Australian Type 1 Diabetes Clinical Research Network The Australian Type 1 Diabetes Clinical Research Network (AT1DCRN) was established by JDRF in 2010 with funds from DoHA, with the aim of better bridging the gap between basic research and clinical therapies development in type 1 diabetes in Australia. The AT1DCRN will solicit, develop, implement and analyse interventional, therapeutic and medical technologies research studies, to evaluate advanced technologies and experimental therapies that are likely to impact disease progression or improve management of type 1 diabetes and its complications. Launched in June 2011, the AT1DCRN will offer clinical trial funding and coordination support and will incorporate a patient referral network to facilitate the recruitment of participants into Australian clinical trials in type 1 diabetes. Accessibility Funding for clinical trials is open to all Australia researchers in type 1 diabetes including commercial and international partnerships. The first RFA was released in December 2010 and proposals submitted in response to the RFA are currently under review, with the outcomes released June The intention of the AT1DCRN is that network members will have ability to access infrastructure support and data collected in the Network. Membership in the Network will be open to Australian type 1 diabetes research community. The first open consultation with the research community is planned for August The operational structures of the AT1DCRN, including data sharing, level of coordination and commercial involvement are in the process of development, and are expected to be confirmed by December NHMRC Clinical Trials Centre The NHMRC Clinical Trials Centre 54 (CTC) is a clinical research organisation that was established in Funding for the CTC comes from the NHMRC, however specific trials are funded by a variety of external bodies. This resource is not specific to type 1 diabetes, but offers infrastructure for clinical trials valuable to this research community AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

43 Infrastructure 4 Available resources As well as running clinical trials, the NHMRC CTC provides a range of services and expertise as a commitment to fostering high quality, investigator initiated clinical trials research. A major service provided by the CTC is CTC Outreach 55 which provides expertise and services to trials run by others, including trial design, randomisation and data analysis. Training and development are offered by the CTC with postgraduate courses in clinical trial research and biostatistics offered. The CTC also runs seminars, short courses and symposia. Princess Margaret Hospital clinical investigations unit The Princess Margaret Hospital in Perth houses a three room clinical investigations unit, currently used in diabetes clinical trials. The unit includes a patient bed, glucose testing equipment and computers. As the unit is specifically designed for diabetes research it is most valuable to researchers in the field. Available resources This resource is currently only used internally. Queensland Clinical Trials Centre The Queensland Clinical Trials Centre 56 (QCTC) designs and manages large scale clinical trials as well as providing expertise to smaller, externally led trials. Like the NHMRC CTC, this resource is not specific to type 1 diabetes. Available resources The services offered by the QCTC include assistance with trial management, randomisation, statistical consulting, database design. QCTC also provides training in all aspects of clinical trial design and management. Short courses are run in conjunction with the NHMRC CTC. QCTC currently has active trials in type 2 diabetes, but has not run any type 1 diabetes trials. QCTC does currently offer clinical trial support to type 1 diabetes researchers in Queensland. Further to Australian specific clinical trial structures, there exist a range of overseas and international resources that are relevant to type 1 diabetes researchers in Australia. The table below summarises the international clinical trial structures that provide resources to type 1 diabetes researchers in Australia. Table 4.6 International clinical trial structures relevant to type 1 diabetes research in Australia Name Purpose Key Outcomes Immune Tolerance Network International clinical trial registries JDRF Canadian Clinical Trials Network TrialNet To accelerate the clinical development of immune tolerance therapies To provide an online registry of clinical trials in the US and worldwide To accelerate made-in-canada solutions to the management, care and cure of type 1 diabetes To prevent, delay and reverse the progression of type 1 diabetes Currently supporting eleven trials, including five in type 1 diabetes Searchable online databases of registered clinical trials in the US and worldwide Support for clinical trials and their coordination Currently supporting four trials in type 1 diabetes AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 41

44 4 Infrastructure Immune Tolerance Network ITN 57 is a clinical trials network incorporating trials in transplantation, autoimmune diseases, and allergy and asthma. The ITN supports its investigators by providing support throughout the range of activities related to clinical trial development, management and analysis. Services include assistance with protocol design, regulatory approval, protocol monitoring, and bioinformatics and data analysis. Available resources The ITN website provides a range of relevant protocols that are made freely available to researchers. The protocols have been developed by the ITN personnel and collaborators and include flow cytometry, RNA preparation, and importantly, cryopreservation protocols which can be helpful in minimising loss of such a valuable resource such as human blood samples. The ITN has a sample sharing program called BioShare 58 that distributes samples collected through ITN trials to researchers, based on scientific review of project applications by the ITN. International clinical trial registries The US based clinical trial registry clinicaltrials.gov 59 and WHO International Clinical Trials Registry Platform (ICTRP) 60 are major clinical trial registries used by Australian researchers. The WHO ICTRP Search Portal collates information from a range of clinical trial registries including ANZCTR, clinicaltrials. gov and ICTRP, as well as other national registries. The value of this global registry is that, because there is no stipulation for Australian clinical trials to be listed on the ANZCTR, this portal compiles data from the range of registries on which they may be listed. This portal allows the searching all multiple databases, including those most commonly used by Australian researchers. Available resources Clinicaltrials.gov, ICTRP and the ICTRP Search Portal are open resources. JDRF Canadian Clinical Trials Network Together with the Federal Economic Development Agency for Southern Ontario, JDRF Canada have developed the JDRF Canadian Clinical Trials Network (CCTN). The CCTN is currently developing several high profile clinical trials in association with leading diabetes researchers at partner universities and medical centres in Southern Ontario. The $34 million CCTN aims to accelerate clinical research in type 1 diabetes, by developing and conducting high profile clinical trials in partnership with leading universities and medical centres in Ontario. The CCTN has a broad mission, spanning advanced technology like the artificial pancreas, through cutting-edge immune therapies and other biological interventions. Available resources Currently, clinical trials are restricted to the province of Ontario. There is a desire to partner with international networks including Australian networks, in order to support international multi-centre trials, but the specific structures for collaboration are not in place as yet AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

45 Infrastructure 4 Type 1 Diabetes TrialNet Type 1 Diabetes TrialNet 61 is an international network of researchers involved in clinical type 1 diabetes research, incorporating 18 research centres around the world, and 12 sites around Australia. TrialNet offers collaboration opportunities for researchers proposing clinical trials in natural history and intervention studies in type 1 diabetes. Available resources TrialNet offers a range of resources to investigators involved with the group, including protocol development and access to samples collected through TrialNet trials. Serum, RNA, and peripheral blood mononuclear cell samples are available from subjects enrolled in the Type 1 Diabetes TrialNet Natural History Study. 4.5 Other infrastructure This section outlines infrastructure identified through this project that is not otherwise categorised. Of particular importance is the sharing of major equipment facilities and expertise, either directly or through sharing platforms. The table below outlines the infrastructure available to researchers in type 1 diabetes that is currently shared within the community. Table 4.7 Other infrastructure relevant to type 1 diabetes research in Australia Name Details Accessibility for research Equipment facilities (national) Rotavirus collection (VIC) Victorian Platform Technologies Network (VIC) platformtechnologies.org/ Varied facilities throughout Australia Genetically modified rotavirus collection Online resource and expertise network Accessibility varies dependant on the facility and institution Shared with collaborators Data available on website and training offered for resource managers Equipment facilities and expertise Many research institutions house specialised large equipment that is routinely used in type 1 diabetes research. Shared equipment facilities vary in terms of the equipment offered, the expertise paired with such facilities and the capacity for sharing the facility with external researchers. Examples of large equipment facilities available to type 1 diabetes researchers in Australia include: Flow cytometry analysis and fluorescence-activated cell sorting; Next Generation sequencing; Proteomics; In vivo imaging such as two-photon imaging in live animals; Genotyping; Microscopy, including confocal microscopy; Ophthalmic testing; and Neurological testing AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 43

46 4 Infrastructure Accessibility There are various factors that affect the capacity for such facilities to be shared; the degree of use within the institution, the owner of the facility (i.e. group owned, institution owned), and the existence of sharing platforms such as the VPTN all contribute. Facilities range from routinely used, to very specialised. The degree of specialisation can play a role in how heavily utilised facilities are and in the potential for sharing these facilities with external resources. It is common for fees associated with the use of these facilities, designed to cover the cost of running the facility, including purchasing reagents and maintaining trained staff. Many facilities have a mechanism in place to prioritise internal researchers, by offering the facility and expertise to external researchers at an inflated fee. Rotavirus collection Researchers at the University of Melbourne house a large collection of fully characterised rotaviruses. The viruses have been adapted to grow in laboratory conditions. The collection is currently being tested to confirm which strains are diabetogenic and genetic mapping is ongoing to establish what genes are associated with the diabetogenic capacity. Accessibility There is potential that this collection can be shared with collaborators. Victorian Platform Technologies Network The Victorian Platform Technologies Network 62 (VPTN) is a network of biomedical research equipment and expertise in Victoria that is available for external use through collaboration or on a fee basis. The VPTN aims to encourage better use of these resources through increased information sharing and to provide quarterly training sessions for member facility managers, with a focus on skills outside the scientific arena. Facility categories include (but are not limited to): Animal models and services; Biobanks; Proteomics and genomics; Flow cytometry; In vivo imaging and microscopy; and Clinical trials and clinical services. Accessibility Listing facilities and expertise on the VPTN website is free. A range of data is made publicly available on the website in order to assist researchers to identify and locate equipment and expertise. The VPTN also supports training programs for facility managers AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

47 Knowledge and networks 5 5. Knowledge and networks Nurturing the current researchers in type 1 diabetes, and attracting new researchers into the field was identified a major aim by the Australia Type 1 Diabetes Research Agenda 63. Research expertise and information sharing within the type 1 diabetes research community are major resources contributing to the scientific output. Due to the multi-disciplinary nature of research in type 1 diabetes and its complications, there exists much varied relevant expertise in Australia relevant to type 1 diabetes research. Mechanisms for sharing and generating knowledge and expertise include a range of means from formal scientific meetings, to the exchange of ideas and information through collaboration, and through both formal and informal mentoring of researchers, particularly during the training stages. Many of the approaches to information sharing, mentoring, networking and collaboration within this community are greatly conventional and the research community has been slow to adapt to incorporate modern technology to help facilitate the flow of information. While this is not specific to the Australian type 1 diabetes research community, better utilisation of telecommunications and internet networking would be particularly beneficial in Australia where geographical isolation is an issue for the majority of researchers, whether on a national or global level. This section provides an overview of the current mechanisms for knowledge sharing and generation in the Australian type 1 diabetes research community, including professional groups and their respective scientific meetings and current opportunities for mentoring for trainee researchers. 5.1 Professional groups and meetings The exchange of information and ideas that occurs at scientific meetings is an integral aspect of how information and ideas are currently shared throughout research communities. In Australia, there are several relevant societies and associations in which researchers in the field of type 1 diabetes research are active, covering the major disciplines involved in type 1 diabetes research. All of these groups hold annual or biannual scientific meetings for clinicians and researchers to present relevant research. The list of relevant professional groups is presented in Table 5.1. Despite the number of relevant societies, there is no local type 1 diabetes-specific professional society or regular scientific meeting in Australia. As such, there are not many formal opportunities for the entire multi-disciplinary community of type 1 diabetes researchers to gather and exchange ideas and information. 63. Australian Type 1 Diabetes Research Agenda Partnering science, government and the community. 2nd edition. (2010) The Juvenile Diabetes Research Foundation. Available online at AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 45

48 5 Knowledge and networks Table 5.1 Professional societies and associations relevant to type 1 diabetes research in Australia Name Australasian Paediatric Endocrine Group Australasian Society for Immunology Australian and New Zealand Society Of Nephrology Australian Diabetes Educators Association Australian Diabetes Society Australian Society for Medical Research Cardiac Society of Australia and New Zealand Endocrine Society of Australia Transplantation Society of Australia and New Zealand Focus To represent those involved in the management and research of children with disorders of the endocrine system including diabetes mellitus To encourage, promote and support the discipline of Immunology in the Australasian region To promote and support the study of the kidney and urinary tract in health and disease, and to ensure the highest professional standards for the practice of nephrology To promote evidenced-based best practice diabetes education to ensure optimal health and well being for all people affected by, and at risk of, diabetes To improve care and outcomes for people with diabetes To foster excellence in health and medical research, expand the interface between basic science and clinical research, and promote community understanding and support To facilitate training, professional development and improve medical practice to enhance the quality of care for patients with cardiovascular disease To represent scientists and clinicians who conduct research and practice in the field of endocrinology To advance the science of transplantation and to foster collaboration with international and other regional societies interested in transplantation 46 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

49 Knowledge and networks Mentoring programs Informal mentoring is an integral aspect of PhD and post-doctoral appointments and is commonly seen to be the responsibility of the direct supervisor of junior researchers in these positions. Often, however, competing priorities of supervisors can mean that trainee researchers suffer a lack of direct mentoring. Components that comprise trainee mentoring at the PhD level include peer-supervisor meetings, formal training programs, formal review processes and participation in academic meetings 64. A number of formal mentoring programs exist, both specifically for type 1 diabetes researchers and more widely within the health and medical research community in general. It has been recommended by the Department of Innovation, Industry, Science and Research, that current mentoring programs be extended and developed to specifically cater for specific target groups with higher needs, such as female researchers 65. Some funders, such as JDRF, stipulate the need for a sponsor who will take responsibility for training recipients of training awards such as Postdoctoral Fellowships and Early Career Patient-Oriented Diabetes Research Awards. Sponsors must be affiliated with an accredited institution full time and be able to provide a training environment conducive to helping the recipient establish a research career. Further details of training, such as formal training or dedicated mentoring/training hours are not stipulated. Other training grants, such as NHMRC Early Career Fellowships specifically include the quality of training in the selection criteria for these fellowships. Specific criteria assessing the quality and relevance of the proposed supervisor and institution to foster the advanced research training of the applicant are included in fellowship grant reviews. There are a limited number of type 1 diabetes specific mentoring programs available to training researchers in the Australia. The Diabetes CCRE fellowship program incorporates a mentorship scheme where a CCRE Fellow is mentored by a Chief Investigator or Associate Investigator not directly related to their research area. General health and medical research focused formal mentoring programs may be accessed by type 1 diabetes researchers. The Australian Society for Medical Research (ASMR) Mentoring Scheme offers ASMR members who are five to 12 years post doctoral an opportunity to be matched with a Career Development Mentor 66. NHMRC TRIP fellowships incorporate access to mentoring through the Mentor Support Program, for career-focussed mentoring from senior Australian healthcare professionals. 64. Leggat PA and Martinez K. (2010) Exploring emerging issues in research higher degree supervision of professional doctorate students in the health sciences. Advances In Health Sciences Education. 15(4) Department of Innovation, Industry, Science and Research (2010) Meeting Australia s Research Workforce Needs: A consultation paper to inform the development of the Australian Government s research workforce strategy AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 47

50 6 Regulatory environment 6. Regulatory environment Underpinning the resource environment in which type 1 diabetes research is conducted in Australia is the regulatory environment in which research takes place. Rather than acting as a resource to facilitate research the regulatory environment shapes what research can be done in Australia, and shapes how research is conducted. The regulatory environment can be viewed as an asset or a hindrance depending on the permissibility of the environment created by governmental policy and frameworks in ethics and regulation, and on the needs of the research community. The regulatory environment affects research across the whole spectrum, from basic research to clinical trials. This section outlines the major frameworks which shape the regulatory environment for type 1 diabetes research in Australia. 6.1 Therapeutic Goods Administration The Therapeutic Goods Administration 67 (TGA) is a division of DoHA, and is responsible for regulating therapeutic goods including medicines, medical devices, blood and blood products. The TGA evaluates therapeutic goods before they are marketed and monitors products once they are on the market, it also assesses the suitability of medicines and medical devices for export from Australia. Any product for which therapeutic claims are made must be listed, registered or included in the Australian Register of Therapeutic Goods (ARTG) before it can be supplied in Australia. The TGA regulates manufacturers of therapeutic goods to ensure they meet acceptable standards of manufacturing quality. It has a team of manufacturing inspectors that audit manufacturing facilities around the world to ensure that products supplied in Australia are of high quality. The TGA administers the Therapeutic Goods Act This legislation provides a framework for a risk management approach that allows the Australian community to have timely access to therapeutic goods which are consistently safe, effective and of high quality. Accessibility and Resources The regulatory framework of the TGA provides the following mechanisms that allow individuals to gain access to therapeutic goods not on the ARTG: Clinical trials via the Clinical Trial Notification Scheme (CTNS) and Clinical Trial Exemption Scheme (CTXS) Special Access Scheme (Categories A & B) Authorised prescribers Importation for personal use A number of TGA publications are available on their website, that provide detailed information on all of the above mechanisms AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

51 Regulatory environment 6 In particular reference to clinical trials, a notification under the CTNS or application under the CTXS is required for all clinical investigational use of a product in Australia, where that use involves: a product not entered on the Australian Register of Therapeutic Goods, including any new formulation of an existing product or any new route of administration; or use of a registered or listed product outside the conditions of its marketing approval. For early phase trials the CTNS is generally acceptable as long as sufficient pre-clinical data is available. Early clinical data must be available for later phase trials to proceed via the CTNS. In Australia, the CTXS is generally only used for new material; new technology or a new concept that has never been evaluated in any country. There are no requirements that applications to the TGA must contain data collected from Australia. Many more clinical trials that do not require a Clinical Trial Notification by virtue of the fact that they are not making use of unapproved therapeutic goods are also conducted every year. It should be noted that unlike other countries, including the US and those in the European Union, under the CTNS all material related to a proposed clinical trial is submitted directly to an Institutional Human Research Ethics Committee (HREC) for review and approval. The institution undertaking the review is the approving authority. The HREC assumes responsibility for assessing the scientific validity of the trial design, safety, efficacy and ethical acceptability, not the TGA. Once approval is granted, the TGA is notified only of the intent to conduct a clinical trial. Acknowledgement from the TGA generally takes 10 days. Such a process provides a favourable regulatory environment in which to conduct clinical research within Australia. From a regulatory perspective, it is also a relatively low cost when compared to other countries. In the context of clinical trials, all regulatory bodies worldwide, including the TGA in Australia, adhere to the International Conference on Harmonisation standards of conduct for clinical trials. These standards comprise the principles of Good Clinical Practice which have their origin in the World Medical Associations Declaration of Helsinki 68. Although the methods for implementing and enforcing these principles vary across regulatory agencies, the end result, it is hoped, are trials that collect high quality, credible data that contribute to the answering of specific scientific questions, whilst most importantly protecting the rights, safety and well being of clinical trial participants. Within the Australian context the TGA has a number of guidance documents including The Australian Clinical Trial Handbook 69 and Human Research Ethics Committees and the Therapeutic Goods Legislation 70. It is important to distinguish between clinical trials and use of a product in an individual patient as part of routine clinical practice. Use of unapproved products in individual patients as part of clinical patient care should be done using the provisions of the Special Access or Authorised Prescriber Scheme and not as a clinical trial. Under the current therapeutic goods legislation, use of unapproved products outside the conditions of marketing approval (so called off label use) is not illegal when done by medical practitioners for individual patients. They do not require Special Access Scheme approval. Clinical trials in which products are used within the conditions of their marketing approval are not subject to CTNS or CTXS requirements but will still need to be approved by an HREC before the trial may commence. All regulatory information, guidelines and applicable application forms can be accessed through the TGA website WMA Declaration of Helsinki Ethical Principles for Medical Research Involving Human Subjects. Available online at The Australian Clinical Trial Handbook (2006) Therapeutic Goods Administration. Available online at Human Research Ethics Committees and the Therapeutic Goods Legislation (2001) Therapeutic Goods Administration. Available online at AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 49

52 6 Regulatory environment 6.2 Ethics and frameworks The NHMRC is Australia s peak government body for supporting health and medical research; for developing health advice for the Australian community, health professionals and governments; and for providing advice on ethical behaviour in health care and in the conduct of health and medical research. The NHMRC issues advice and guidelines on ethics and related issues in the fields of health and human and animal research in accordance with the NHMRC Council Act 1992 and other related legislation. Human research ethics The NHMRC plays a major role in providing guidance and advice to HRECs for the pivotal role they play in reviewing the scientific and ethical aspects of research proposals, and undertaking the chief role of ongoing monitoring of such research and dealing with complaints that arise from such research. The NHMRC National Statement on the Ethical Conduct in Human Research 72 is Australia s primary source of guidance for the conduct of all research that involves human participants. Human research is research conducted, with or without, people, or their data or tissues. It is a statement of ethical principles which provides a national reference point for ethical consideration relevant to all human research. Its purpose is to promote ethically sound human research; to set national standards of ethical conduct for all research involving humans; and to guide institutions, researchers and HRECs in the ethical review and conduct of such research. The National Statement extends to a number of research fields and methods including: Qualitative research methods; Databanks; Interventions and therapies (including clinical and non-clinical trials); Human tissue samples; Human genetics; and Human stem cells. Harmonisation of Multicentre Ethical Review Initiative In 2006, the Australian Health Ministers Advisory Council requested the NHMRC facilitate the development and implementation of a national system where the single ethical review of a HREC would be recognised by all institutions participating in a collaborative research project. By having a single ethical review outcome accepted by collaborating institutions, protection of human participants would be maintained while delays due to the practice of seeking multiple ethical reviews would be mitigated and timelines for research start-up and results would be shortened. From this the Harmonisation of Multicentre Ethical Review Initiative 73 (HoMER) was introduced. As part of this initiative a number of institutions (including public hospitals) have undergone assessment and certification to facilitate single ethical review. The uptake of the national approach for single ethical review will respect institutional autonomy to determine whether research should be conducted at a given site. Advice received from a HREC undertaking the single ethical review will not replace the need for local institutional decision making on matters of research governance AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

53 Regulatory environment 6 Several states have now developed formal systems for streamlining ethical review processes in public health organisations. Other jurisdictions have informal arrangements operating as agreements of acceptance between institutions in the private and the public sectors and between public health organisations and universities. There are a number of national policies, processes, guidance documents and standard forms, including the National Ethics Application Form (NEAF) that are now available 74 and a number of HRECs have also become certified as lead HRECs. A number of HoMER tools are now accessible through the NHMRC website including national policies, procedures, guidance documents and standardised application forms including the NEAF. Animal research ethics In Australia, there are a number of laws which govern the use of animals in research, teaching and product testing in each Australian state. Although there is some variation in the statutory requirements of each state, the Australian Code of Practice for the Care and Use of Animals for Scientific Purposes 75, sets out the common framework of guiding principles. The NHMRC also provides support and advice on animal ethics, including information for Animal Ethics Committees. They have an Animal Welfare Committee set up which provides advice on all matters pertaining to the conduct and ethics on the use of animals for scientific purposes. Xenotransplantation In December 2009, the Council of the NHMRC recommended that clinical trials involving animal to human transplantation (xenotransplantation) only be allowed to proceed, when appropriate regulatory and surveillance frameworks have been put in place. Recent amendments to the Therapeutic Goods Regulations 1990 to create a new regulatory framework for biologicals were passed by Executive Council on 10 March The biologicals framework will commence on 31 May After this date, all products within the scope of the framework will need to comply with the requirements made under the new legislation, subject to relevant transition arrangements. The TGA should be contacted directly to further discuss the implementation of the regulatory framework around xenotransplantation. Gene Technology The Office of the Gene Technology Regulator 76 has been established within the Australian Government Department of Health and Ageing to provide administrative support to the Gene Technology Regulator in the performance of his functions under the Gene Technology Act The Gene Technology Act 2000, which came into force in 2001, introduces a national scheme for the regulation of genetically modified organisms in Australia, in order to protect the health and safety of Australians and the Australian environment by identifying risks posed by or as a result of gene technology, and by managing those risks through regulating certain dealings with genetically modified organisms. The NHMRC also has current publications available on its website specifically related to genetics and gene technology including: Medical Genetic Testing (2010); The Protection of Human Genetic Information in Australia (2003); and Guidelines for Genetic Registers and Associated genetic material (1999) Australian code of practice for the care and use of animals for scientific purposes (2004) National Health and Medical Research Council. Available online at AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 51

54 7 Key needs 7. Key needs The support of research and researchers through stable, well resourced infrastructure is an important part of achieving research outcomes and moving research breakthroughs closer to the patient. Through interviews with researchers and resource managers, several issues were identified that present hurdles to researchers in all fields of type 1 diabetes research. A lack of specific infrastructure was raised as an issue, as was poor utilisation of established resources. This section outlines the major resources needs raised by researchers as pivotal to advance their research and achieve planned research outcomes, with commentary on the major hurdles to improving resource accessibility and utilisation. This assessment uncovered a number of common themes in the infrastructure needs of researchers both in terms of access to physical resources and in terms of improved information flow. These findings are outlined in Figure 7.1 below. Inherent in Australian research environment is the issue of underutilisation of established resources. Issues ranging from lack of information sharing, separation by large geographic distances and project based funding mean that a number of the resources identified in this project are only used locally or by one research group. There is potential that the value of these resources to type 1 diabetes research could be magnified by improving their accessibility to the research community. In addition, fragmentation of the resources prevents large scale, systematic studies as the resources lack common features and standardised protocols for collection and data. Issues preventing better accessibility of the current pool of resources are discussed in this section. 70% 60% % of people surveyed 50% 40% 30% 20% 10% 0% Human tissue Funding Information sharing Collaboration and networking Animal resources Clinical data Clinical trials support Facilities and expertise Data management Other Figure 7.1 Reported resource needs percentage of people surveyed mentioning each category 52 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

55 Key needs Researcher needs This section highlights the needs within the research community, with specific relevance to type 1 diabetes research in Australia. A range of issues, in particular surrounding the realities of pursuing a career in type 1 diabetes research, affect the number and type of researchers who make up this community. Creating a better understanding of the needs of the type 1 diabetes research community allows for the better provision of support for these researchers, and ultimately for increasing the quality and amount of research output in the field. Attrition from academic and main stream research careers A 2008 survey administered to members of the ASMR found that 73% of respondents had considered leaving the field of health and medical research and a further six percent had already left. It was noted that these numbers may be an underestimate as respondents were ASMR members. The reasons for leaving included perceived shortage of funding and lack of career opportunities 77. Levels of attrition from higher research degrees have averaged approximately 17% in recent years for domestic doctoral students in all fields, not specific to medical science or type 1 diabetes research 78. Limitations and needs of clinician researchers The Australian Type 1 Diabetes Research Agenda 79 identified clinician researchers as a group in need of support. It was recommended that research presence in large teaching hospitals should be increased through clinical research programs. It was noted that there was a need for career packages that reserved and rewarded research time for clinicians. A number of the issues surrounding the need for more support for clinician researchers are outlined in a proposal to establish a Victorian Clinician Researcher Network by the Bio21 Cluster Clinician Researcher Working Group. The proposal noted that while clinical research essential for the passage of ideas both ways between basic research and clinical practice, the current health-system allows little time for clinicians to devote to research 80. This was mirrored by perceptions within the research community that while the creation of more part time clinician researcher fellowships would create more opportunities for clinicians to undertake research, lobbying within the hospital system may be necessary to facilitate this as an option for hospital based clinicians. The Clinician Network Proposal noted that the long training period (up to 20 years) can lead to clinician researchers often being uncompetitive for research grants, due to family commitments, age and low publication record relative to age 81. However, there is some difference in opinion within the research community as to whether there are less funding options for clinician researchers, compared to PhD qualified researchers, whether clinician researchers are less competitive for general funding, or whether it is simply the relative instability of research compared to a clinical career. However, there is a shared perception that the combination of a lack of funding and relatively unstable career path makes research an unattractive career choice for medically qualified individuals who may be considering undertaking research. This issue is not necessarily specific to clinician researchers. It was suggested that more funding, particularly directed to people support to create training opportunities, would be beneficial to protect this important subset of type 1 diabetes researchers. 77. Kavallaris M, Meachem SJ, Hulett MD, West CM, Pitt RE, Chesters JJ, Laffan WS, Boreham RR and Khachigian LM (2008) Medical Journal of Australia. 188(9) Department of Innovation, Industry, Science and Research (2010) Meeting Australia s Research Workforce Needs: A consultation paper to inform the development of the Australian Government s research workforce strategy. 79. Australian Type 1 Diabetes Research Agenda Partnering science, government and the community. 2nd edition. (2010) The Juvenile Diabetes Research Foundation. Available online at Clinician Researcher Working Group, Bio21 Cluster (2008) Victorian Clinician Researcher Network proposal. 81. Ibid. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 53

56 7 Key needs 7.2 Funding needs The need for funding was mentioned by 45% of researchers contributing to the qualitative analysis of research infrastructure and resource needs that has been described for this project. This was perceived to be a widespread and ongoing issue and currently exacerbated by a plateauing of the NHMRC budget in recent years, which in absolute terms results in less funds directed to research. Due to a large proportion of grants coming from JDRF in the US, it was brought up that these grants had lost value in Australian dollars due to current strength of the Australian dollar against the US dollar. Career support People support was the most commonly stated need for funding, however this covered a range of specific needs in this area, including: Early career support and mid career support; Support for clinical nurses personnel to perform data management; The need for security and stability of career support, to ensure continuity of research; and Support for training clinician researchers and providing fellowships to this group to protect time for research. Given the relatively few years spent in the training stages, there is an impression that there is over funding in the training stages compared to that offered to mid-career and established researchers. Researchers transitioning from the people support grants that support them through the training stages, to applying for research grants as independent researchers face a very competitive market. The House of Representatives Standing Committee on Industry, Science and Innovation released a report on Australia s International Research Collaboration in The recommendations of the committee included that a proportion of the NHMRC and ARC budgets be set aside for early-career researchers who are first-time awardees. The NHMRC caters for early-career researchers by allocating funds to offer Priming Project Grants to new investigators. In 2010 the NHMRC awarded 53 Priming Project Grants, out of a total 683 Project Grants 83. Due to established researchers relative competitiveness for research funding compared to emerging and mid-career independent researchers, there is a possible gap in funding here for the later group researchers. This comes at a time when competing interests (such as family, financial stability) may draw researchers away from the field, or away from Australia. Grant types The need was raised by multiple researchers for larger, long term grants, again to ensure continuity of research, in particular retaining the research staff and their expertise for longer ongoing projects which can be difficult on standard three year grants. Longer term funding was perceived as necessary for specific non-project related needs, such as maintenance and building of biobanks and database management. Larger grants were also specifically needed by researchers undertaking work with large animal models, which were perceived of being more expensive than other models commonly used by basic researchers, without any specific funding being allocated to this area. The prevalence of project specific funding and the lack of longer term maintenance funding can lead to loss of expertise, in particular with clinical research nurses. Often, these staff are hired for specific clinical trials and can not be maintained long term. As such, valuable expertise is often lost. There was stated a need for funding to support data management to maximise the value of potential data that is not analysed and published due to a lack of funding to create databases and to fund personnel with data management expertise. 82. House of Representatives Standing Committee on Industry, Science and Innovation (2010) Australia s International Research Collaboration. Commonwealth of Australia. Canberra. 83. National Health and Medical Research Council. Summary of Annual Funding Commitments 2009 and Available online at 54 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

57 Key needs 7 Effect of stability of funding on research careers A 2008 survey administered to members of the ASMR found that 73% of respondents were on fixed term appointments. Of the respondents, 57% were directly supported by grants and fellowships and were at risk of losing their job if further funding was not secured. Only 16% did not rely on grants for continuing employment. Three quarters of respondents stated that lack of security had a detrimental effect on their career, and almost all stated that a lack of financial support had a detrimental effect 84. In 2010, the Department of Innovation, Industry, Science and Research reported that there has been a decline in perceived attractiveness of research as a career, perceived low visibility of career paths and concerns around the short term, project based nature of research positions. This short term and project based nature of research funding in Australia was seen to contribute to a perceived lack of stability and continuity in career paths Infrastructure needs This section highlights the infrastructure needs within the type 1 diabetes research community in Australia. A range of needs have arisen from the analysis conducted as a part of this project, and the greatest number of expressed needs were related to accessing infrastructure. Creating a better understanding of the infrastructure needs of the type 1 diabetes research community is important in developing better support of the research community and in removing the hurdles to achieving research outcomes and moving research to the next level. Needs in this area spanned a wide range of issues, spanning basic and clinical research. This section provides an overview of the resource needs expressed by the type 1 diabetes research community. Human tissue samples The most commonly stated resource need of type 1 diabetes researchers in Australia is access to human tissue samples. This need was independently raised by over 60% of researchers contacted. Even with the presence of various smaller biobanks, accessibility to appropriate human tissue samples is seen as difficult or lacking. This need is mirrored in other research communities access to biobanks was highlighted as an infrastructure requirement in the Type 1 Diabetes Research Agenda 86 and the Strategic Roadmap for Australian Research Infrastructure 87. The most commonly stated tissues needed are blood and serum, and pancreatic tissue including isolated islets. The diversity of Australian research in the type 1 diabetes area however, means specialist groups require a range of different sample types. Diabetes complications researchers stated the need for kidney and urine samples. The need for urine samples for studies of metabolites was also raised. Researchers studying type 1 diabetes autoimmunity have stated the need for immune cells such as peripheral blood mononuclear cells, buffy coats separated from whole blood and blood screened for the presence of autoantibodies. There is also a need for gastrointestinal tissue from people with type 1 diabetes. Samples from both healthy controls and people with type 1 diabetes are needed. There is a specific need for tissue samples paired with clinical data for many researchers. Several researchers have particularly noted the need for longitudinal samples. 84. Kavallaris M, Meachem SJ, Hulett MD, West CM, Pitt RE, Chesters JJ, Laffan WS, Boreham RR and Khachigian LM (2008) Medical Journal of Australia. 188(9) Department of Innovation, Industry, Science and Research (2010) Meeting Australia s Research Workforce Needs: A consultation paper to inform the development of the Australian Government s research workforce strategy. 86. Australian Type 1 Diabetes Research Agenda - Partnering science, government and the community. 2nd edition. (2010) The Juvenile Diabetes Research Foundation. Available online at Department of Innovation, Industry, Science and Research (2008) Strategic Roadmap for Australian Research Infrastructure. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 55

58 7 Key needs Due to a lack in funding earmarked for the maintenance of resources such as biobanks and databases, these resources are often funded through project specific grants. This leads to the development of small, project specific resources which are not feasible to maintain in the long term. This funding environment is prohibitive of the development of larger, stable resources that can be shared within the research community. Long term funding is required not only for the development of these resources, but for their long term maintenance. Long term storage of human tissue samples is an important aspect of protecting valuable resources. Without good storage and handling of human tissue samples, the quality of older samples in particular can be compromised. Using clinical samples for research is dependant on having proper informed consent. The level of consent obtained upon sample collection varies widely and is heavily dependent on the specific resource. This may be an issue when it comes to the sharing of clinical samples with the wider research community. This issue could potentially be overcome by the development a centralised resource with standardised consent protocols. It was mentioned that the need for better information sharing and networking contributed to this need. Researchers interested in getting access to clinical samples would benefit from having visibility of what clinicians have an interest in research and are interested in providing human tissue through collaborations. A clear indication of who has access to clinical samples and is open to sharing or collaborating would be valuable to researchers seeking clinical tissue samples. There are currently a number of Australian researchers using samples from international studies and through resources such as npod. These researchers have noted that the logistics involved with importing human tissue samples to Australia hinder research due to the increased time required to access samples and the increased paperwork surrounding import regulations. On top of this it has been mentioned that it is sometimes difficult to secure the quantities of cells required through samples such as these as the resource is shared so widely with a large number of research groups. Animal resources There was a stated need for animal resources including more space for housing. As well as a need for more space for housing small animals (particularly mice), there was a specific need for barrier facilities for housing pigs. This was particularly relevant for work using pig models of xenotransplantation and was seen as an expensive, but necessary step to developing this work to a clinical stage. Issues surrounding the sharing and maintenance of particular resources, for example genetically modified mouse models, include the cost associated maintaining these resources. Research groups developing mouse models for research are often happy to share animals, however are hesitant to spend money and resources on maintaining animals for use by other researchers. Many shared resources are maintained through a cost-covering fee to users, however in the case of animal models, the time and costs associated with maintenance are high and processes for remuneration are not in place. Other needs surrounding animal research infrastructure included a biobank for animal tissue, a shared type 1 diabetes animal repository, and more funding support for the development and sharing of animal models. The APB as previously mentioned, although not type 1 diabetes specific, addresses some of these needs. The value of the APB to type 1 diabetes researchers relies on good visibility to researchers and on comprehensive uptake of its services by the research community and in particular by type 1 diabetes researchers. 56 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

59 Key needs 7 Clinical data The need for clinical data, both independently of human samples and tied to human samples was raised by close to one in five researchers contacted. It was suggested by a range of researchers that it would be beneficial to establish a central repository and database for human tissue samples linked with clinical data. There was a need particularly for access to longitudinal databases, both of people with type 1 diabetes and people at high risk of developing type 1 diabetes. It was raised that there are particular hurdles in creating whole of life databases for people with type 1 diabetes, particularly in the transition from paediatric care to adult care, where continuity is difficult to maintain. The need for access to longitudinal databases, either clinic databases or from clinical research projects was linked to the need for access to longitudinal complications screening databases. Clinical trials support The need for clinical trial support encompassed a range of specific needs. A number of these surrounded the need for assistance in patient recruitment. It was noted that researchers attempting to access the NDSS database to recruit participants for clinical trials encountered a lot of confusion and bureaucracy and it would be beneficial if the process was more streamlined and had increased visibility to researchers. Other specific hurdles mentioned included inadequate space for conducting clinical trials, as well as problems in securing long-term funding for clinical research nurses in order to retain the expertise through more than one clinical trial, when often only having project specific funding to support staff. Patient Referral Networks Within the area of clinical trial support, the need for a type 1 diabetes specific clinical trials registry was highlighted, as well as the need for clinical trial support, in particular assistance with recruitment. Although a range of patient databases that are project or institution specific exist, there is a distinct lack of patient referral services linked to clinical trials. There was an explicit need within the research community for a facility that enables the matching of eligible participants to appropriate clinical trials. The JDRF AT1DCRN plans to fill this gap with the Patient Referral Network (PRN), to be developed in conjunction with the AT1DCRN. The PRN will act not only as a registry of type 1 diabetes clinical trials in Australia, but also as a hub through which potential participants can find information about trials they may be interested in participating in, thus facilitating recruitment. Facilities and expertise Access to facilities and expertise was an issue for a proportion of researchers surveyed. Specific needs mentioned were access to clean-room facilities for human tissue processing, cell imaging equipment and expertise, FACS, electron microscopy and access to bioinformatics and statistics expertise and assistance. Geographic isolation is an issue only in specific circumstances particularly places other than Melbourne and Sydney where there is a high concentration of researchers. Physical facilities may be underutilised because they are not accessible by researchers that would otherwise be interested to use them. Analysis of the research community in Australia has shown that close to three quarters of Australian researchers in the field of type 1 diabetes are located in Melbourne and Sydney. As such, physical resources that are located elsewhere may be underutilised. Similarly, researchers located outside Melbourne and Sydney may have difficulty accessing physical resources in comparison to researchers in these locations. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 57

60 7 Key needs Data management There was a specific need for assistance with data management, both from an expertise perspective and for funding to support data management personnel. There was a perception that it was difficult to secure funding for data management personnel, and difficult to employ data management personnel long term as they were often funded by project grants. Other Poor access to industry and difficulty finding options for commercialisation of research was raised as a hurdle for researchers to bring promising research ideas to the next stage of development. There was a perception that industry was not interested in treatments for type 1 diabetes as it was high risk and with low potential returns. As such, it was reported that it was difficult to generate support for commercialisation of research. The need for support with navigating regulatory and ethics requirements associated with research was raised. It was noted that these requirements consume a large proportion of time, especially for research group leaders and that these requirements and obligations have been increasing over the past several years. It is expected that the HoMER initiative will assist in decreasing this load for multi-centre clinical trials however it is perceived that more support in this arena would facilitate researchers to spend less time devoted to the associated administrative burden of ethics and regulatory considerations. 7.4 Knowledge and networks needs The Australia Type 1 Diabetes Research Agenda 88 identified networking and resource sharing as a major aim to best support type 1 diabetes research in Australia. Supporting and facilitating the flow of information, through both formal and informal mechanisms, will enhance the expertise of the type 1 diabetes research community in Australia in a way that best utilises the current strengths of the community and the resources that are already in place. The needs of the type 1 diabetes research community in Australia in this space fell into two categories: the need for better information sharing within the community, and the need for more opportunities for collaboration within a multi-disciplinary setting. These needs are discussed below. Information sharing The need for better information sharing was raised in several different contexts by approximately one third of researchers interviewed. Researchers identified the need for better information sharing among researchers, in particular the benefit of a national registry or network or type 1 diabetes researchers highlighting their expertise and resources available for shared use. The idea of a centralised registry was brought up in several contexts for specific resources such as type 1 diabetes animal models or antibodies. A potential solution to this issue would be the development of a type 1 diabetes researcher network that allows member visibility of available expertise and resources, facilitating collaboration. This also ties in with the stated need for networking and collaboration opportunities, mentioned below. A lack of understanding of the resources and expertise available to researchers in Australia can lead to the waste of resources and funding. This is an issue both for duplication of resources and underutilisation of resources that may have otherwise been shared. There is the opportunity to improve accessibility to resources and the utilisation of current resources by increasing the visibility of what research resources are available to researchers from expertise, to physical facilities and tissue collections. 88. Australian Type 1 Diabetes Research Agenda Partnering science, government and the community. 2nd edition. (2010) The Juvenile Diabetes Research Foundation. Available online at 58 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

61 Key needs 7 Some researchers identified the need for animal model registries in particular. Although not type 1 diabetes specific, the APB can fulfil this need if the incentives are there for researchers to utilise this resource, and if awareness of the resource is high. The national standardisation of protocols, in particular protocols for the harvesting and processing of human tissue samples was raised as a need. ITN provides a number of standard protocols as used by network members, in particular cryopreservation protocols to ensure high quality and minimal waste of stored human tissue samples. These protocols are an open resource, however there is no tracking of how widely spread they are used for example in cryopreservation for the various collections of human tissue available in Australia. Networking and collaboration Approximately 25% of researchers interviewed stated there was a particular need for opportunities to network with other researchers to facilitate collaboration, whether through face to face meetings for type 1 diabetes researchers, or an online platform through which to access researchers in the field. The need for a regular, national, multi-disciplinary type 1 diabetes specific scientific meeting was explicitly raised by a number of researchers. It has long been recognised that type 1 diabetes research covers a range of different disciplines, between which there has traditionally been little collaboration. There was a perception that a paradigm shift is required to make communication and collaboration between these various fields of type 1 diabetes research the norm. Through this review, it was recognised that while there are various meetings attended by type 1 diabetes researchers in Australia, there are not many opportunities for multidisciplinary researchers in the field of type 1 diabetes to come together on a national level. While it was acknowledged that there was value in attending national and international meetings within a researchers specialty, researchers perceived that there would be additional value in a national type 1 diabetes meeting facilitating networking and communication between researchers outside their direct specialty. It was noted that the one-off multidisciplinary workshop run by JDRF in 2009, as part of the development of the Australian Type 1 Diabetes Research Agenda, was a useful opportunity for fostering new relationships between researchers who otherwise would not have opportunities to come together. The desire for a virtual network for all researchers to connect with each other was raised several times. Further to the need for increased information sharing, the idea of a researcher network was raised as a means to increase communication and opportunities for collaboration between the entire type 1 diabetes research community. There was a perception that creating an online based network, to work together with a national type 1 diabetes meeting, would capitalise on occasional face-to-face meetings. The need for support to bring in international expertise was raised as a concern that due to its geographical isolation, it was difficult and expensive to bring in outside experts network with Australian researchers to enhance local expertise. The idea of earmarking funding specifically for collaborative projects was raised, however the value of earmarked funding would be multiplied by providing a platform through which to initially foster new relationships between potential collaborators to create new collaborations. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 59

62 8 Next steps 8. Next steps Maintaining this document as an up-to-date resource is necessary to ensure it remains relevant and useful to both basic and clinical researchers in type 1 diabetes in Australia. To this end, the document will remain open for public consultation from the type 1 diabetes research community in Australia, and submissions of shared resources will be welcomed in order to ensure that the resource map represents a comprehensive and complete resource for the research community. This document will be developed into a user friendly, online database as an information source for researchers, as a way to increase the visibility of available resources relevant to type 1 diabetes research, facilitate increased utilisation of the current resources identified in this map, and to encourage collaboration within the Australian type 1 diabetes research community. This document provides a comprehensive overview of the resource environment in which type 1 diabetes research is conducted in Australia. In particular, this analysis has revealed a number of areas of need within this environment. The understanding of the resource environment created through this project, and outlined in this document has the potential to increase the efficiency of their investment for funders of type 1 diabetes research resources in Australia. As such, this is a useful resource for funders of type 1 diabetes research across all domains. 60 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

63 Appendices 9 9. Appendices 9.1 Executive Advisory Committee Professor James Best MB BS MD FRACP FRCPath FRCP (Edin) Head, School of Medicine, University of Melbourne VIC Chair of Research Committee, National Health and Medical Research Council Professor Don Chisholm AO MB BS (Hons) FRACP Senior Principal Research Fellow, Garvan Institute of Medical Research NSW Honorary Endocrinologist, St Vincent s Hospital NSW Dr Stuart Mannering PhD Lab Head, Immunology and Diabetes Unit, St Vincent s Institute VIC Dr Dorota Pawlak PhD Head of Research Development Juvenile Diabetes Research Foundation 9.2 Acknowledgements This report was authored by Christine Whittall and Dorota Pawlak PhD. The following individuals are acknowledged for their valuable contribution to this document: Barbara Araneo PhD, Darlene Deecher PhD, Julia Greenstein PhD, Julie Ince-Demetriou PhD, Richard Insel MD, Gayle McNaught, Concepcion Nierras PhD and Mike Wilson. AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 61

64 9 Appendices 9.3 Abbreviations ABR Australian BioResources ACDDR Australian Childhood Diabetes DNA Repository AIHW Australian Institute of Health and Welfare ANZCTR Australian New Zealand Clinical Trials Registry APB Australian PhenomeBank APEG Australasian Paediatric Endocrine Group ARC Australian Research Council ARTG Australian Register of Therapeutic Goods ASMR Australian Society for Medical Research AT1DCRN Australian Type 1 Diabetes Clinical Research Network CCRE Clinical Centre for Research Excellence CCTN Canadian Clinical Trials Network CTC Clinical Trials Centre CTNS Clinical Trial Notification Scheme CTXS Clinical Trial Exemption Scheme DART Diabetes Australia Research Trust DoHA Department of Health and Ageing DRF WA Diabetes Research Foundation of Western Australia DVDC Diabetes Vaccine Development Centre hes human embryonic stem cell HoMER Harmonisation of Multicentre Ethical Review HREC Human Research Ethics Committee ICTRP International Clinical Trials Registry Platform ITN Immune Tolerance Network ITP Islet Transplantation Program JDRF Juvenile Diabetes Research Foundation MB BS Bachelor of Medicine, Bachelor of Surgery MD Doctor of Medicine NDR National Diabetes Register NDSS National Diabetes Services Scheme NEAF National Ethics Application Form NHMRC National Health and Medical Research Council NIDDK National Institute of Diabetes and Digestive and Kidney Diseases NIH National Institutes of Health NOD Non-obese diabetic npod Network for Pancreatic Organ donors with Diabetes PRN Patient Referral Network QCTC Queensland Clinical Trials Centre TGA Therapeutic Goods Administration T1DGC Type 1 Diabetes Genetics Consortium VPTN Victorian Platform Technologies Network 62 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

65 Appendices Contributors to qualitative assessment A/Prof Terri Allen PhD Baker IDI Heart and Diabetes Institute Dr Lois Balmer PhD Western Australia Institute of Medical Research Dr Tom Brodnicki PhD St Vincent s Institute Prof Donald Chisholm MB BS Garvan Institute of Medical Research Prof Peter Colman MB BS Royal Melbourne Hospital A/Prof Barbara Coulson PhD University of Melbourne Prof Peter Cowan PhD St Vincent s Hospital A/Prof Maria Craig PhD MB BS Westmead Children s Hospital and University of Sydney Prof Nathan Efron PhD DSc Institute of Health and Biomedical Innovation A/Prof Assam El Osta PhD Baker IDI Heart and Diabetes Institute Prof Andrew Elefanty PhD MB BS Monash University Mr George Fedesyn Baker IDI Heart and Diabetes Institute A/Prof Josephine Forbes PhD Baker IDI Heart and Diabetes Institute Dr Jenny Gunton MB BS Garvan Institute of Medical Research Prof Annemarie Hennessy PhD MB BS University of Western Sydney Prof Karin Jandeleit-Dahm PhD MD Baker IDI Heart and Diabetes Institute A/Prof Alicia Jenkins MB BS MD St Vincent s Hospital A/Prof Timothy Jones PhD MB BS Princess Margaret Hospital and Institute for Child Health Prof Tom Kay PhD MB BS St Vincent s Hospital and St Vincent s Institute Dr Cecile King PhD Garvan Institute of Medical Research Dr David (Ross) Laybutt PhD Garvan Institute of Medical Research Dr Tom Loudovaris PhD St Vincent s Institute Dr Stuart Mannering PhD St Vincent s Institute Prof Grant Morahan PhD Western Australia Institute of Medical Research Prof Hung Nguyen PhD University of Technology Sydney A/Prof Mark Nottle PhD University of Adelaide Mr Daniel Palamara Australian Institute of Health and Welfare Prof Anne Louise Ponsonby PhD MB BS Murdoch Childrens Research Institute Ms Nicola Pritchard Institute of Health and Biomedical Innovation Dr Charmaine Simeonovic PhD John Curtin School of Medical Research Prof Ann Simpson PhD University of Technology Sydney Prof Ed Stanley PhD Monash University Dr Raymond Steptoe PhD Diamantina Institute for Cancer, Immunology and Metabolic Medicine Dr Helen Thomas PhD St Vincent s Institute Prof Ranjeny Thomas MB BS Diamantina Institute for Cancer, Immunology and Metabolic Medicine Dr Anne Thorburn PhD St Vincent s Institute A/Prof Stephen Twigg PhD MB BS University of Sydney AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP 63

66 9 Appendices Infrastructure index 34 Antibody screened at risk blood collection 28 Australasian Paediatric Endocrine Group state-based registers 26 Australian BioResources animal housing and breeding facility 34 Australian Childhood Diabetes DNA Repository 38 Australian New Zealand Clinical Trials Registry 40 Australian Type 1 Diabetes Clinical Research Network 34 Baker IDI Biobank 30 BioGrid Australia 31 Childrens Hospital Westmead clinic database 39 Diabetes Clinical Centre for Research Excellence 39 Diabetes Vaccine Development Centre 35 Diamantina autoimmunity sample collection 35 Environmental determinants blood collection 43 Equipment facilities and expertise 26 Gene Mine breeding program 35 Human embryonic stem cell lines 42 International clinical trial registries 42 Immune Tolerance Network 36, 40 Islet Transplantation Program 42 JDRF Canadian Clinical Trials Network 36 LANDMark study biobank and cohort 26 Mouse models of diabetes and its complications 29 National Diabetes Register 29 National Diabetes Services Scheme database 27 National NHMRC Baboon Colony 36 Network for Pancreatic Organ donors with Diabetes 27 NHMRC Australian PhenomeBank 40 NHMRC Clinical Trials Centre 27 Pig models of xenotransplantation 37 Princess Margaret Hospital clinical database and sample collection 41 Princess Margaret Hospital clinical investigations unit 41 Queensland Clinical Trials Centre 44 Rotavirus collection 31 Royal Melbourne Hospital clinic database 31 Royal Prince Alfred Hospital clinic database 37 St Vincent s Hospital Melbourne biobank proposal 31 St Vincent s Hospital Melbourne clinic database 37 St Vincent s Hospital Melbourne sample collection 31 St Vincent s Hospital Sydney clinic database 32 T1DBase 32 Type 1 Diabetes Genetics Consortium 43 Type 1 Diabetes TrialNet 44 Victorian Platform Technologies Network 64 AUSTRALIAN TYPE 1 DIABETES RESEARCH RESOURCE MAP

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