7 th European Conference on Rare Diseases & Orphan Products (ECRD 2014 Berlin) The Rare Disease Puzzle: Bringing the Picture to Life

Transcription

1 Save the Date! 8 10 May 2014 Andel s Hotel, Berlin, Germany 2014 Berlin Brussels May th European Conference on Rare Diseases & Orphan Products (ECRD 2014 Berlin) The Rare Disease Puzzle: Bringing the Picture to Life In the past two decades we have identified the pieces that address the needs of the rare disease community. The challenge remains to combine those pieces into a picture that drives action forward with clarity and energy into a coherent and sustainable strategic plan. ECRD Co-Chairs What is ECRD? Ségolène Aymé Director of International Affairs Orphanet, Chair Topic Advisory Group on Rare Diseases at WHO, Project Leader of Support IRDiRC, Editor in chief of the OJRD (France) Lesley Greene Vice-Chair COMP, Patient Representative, CLIMB & (UK) Wills Hughes-Wilson Chair Task Force Rare Diseases & Orphan Drugs of EuropaBio- EBE, Vice President External Affairs, Chief Patient Access Officer, Swedish Orphan Biovitrum (sobi), Member of DIA Advisory Council Europe (Sweden) The European Conference on Rare Diseases & Orphan Products (ECRD) is the unique platform/forum across all rare diseases, across all European countries, bringing together all stakeholders: patients representatives, academics, health care professionals, industry, payers, regulators and policy makers. ECRD covers research, development of new treatments, health care, social care, information, public health and support at European, national and regional levels. ECRD provides the state of the art of the rare disease environment, monitoring and benchmarking initiatives. Organised by Co-organised by With the support of

2 Why attend ECRD as an Industry Representative? Meet all of the stakeholders shaping your marketplace patients, policy makers, academia, payers and industry colleagues together in one venue Receive the most current facts, figures and trends from the authorities on where we stand on orphan medicinal products, 15 years into the Regulation: what has worked and what can we expect Find out from the regulators and decision-makers about the latest status of current and emerging regulatory tools, as well as frameworks available to support orphan medicinal product development and regulatory approval Evaluate new pathways for collaboration and partnership at all stages of the development chain Discover challenges and opportunities from other stakeholders perspectives Explore emerging pricing, reimbursement and access trends together with those responsible for the markets See how different elements are coming together at both global, regional and country level to support orphan discovery, development and availability Learn how to deal with non-traditional routes to securing availability/access to rare disease treatments Hear from patients first-hand about their needs and experiences in the route to treatment Exchange viewpoints on the position of orphans in times of austerity and what new approaches are underway to address the challenges Understand the state of the art for orphan development as well as trends for the future Hear about challenges and experiences in disease specific registries Get insight into how to sustain attractiveness for companies to invest in rare diseases

3 Why attend ECRD as a Patient Representative/Patient? Learn about the current landscape of policy development on orphan products and rare disease therapies Find out about Centres of Expertise and their important role and relevance in National Plans Get insight into the challenges at national level of disease specific registries Discover inspiring initiatives by providers to address the social challenges of rare diseases Network with fellow rare disease patients, families and all other stakeholders from across Europe Contribute to the discussion on how to improve gene testing, genetic counselling and newborn screening at the European level Contribute to addressing the challenges of healthcare pathways Discover important breakthroughs in science for rare diseases Contribute to the debate on progressive patient access and patient involvement in the benefit-risk assessment Hear about the challenges of off-label use and access Learn about the discovery process of a medicinal product from the point of view of industry and regulators and much more!

4 Why attend ECRD as a Healthcare Professional/Academic? Learn about the current State of the Art of rare diseases Discover the clinical framework specific to rare diseases Understand the opportunities in rare disease collaborative clinical research Contribute to addressing the challenges of healthcare pathways Make the most of new infrastructures and standards to generate knowledge on rare diseases Get insight into the new possibilities for genetic sequencing and its challenges Understand the difficulties and possible solutions for patient access to orphan products and rare disease therapies Network with fellow colleagues, patient representatives, regulators, payers and industry representatives Obtain medical credits by attending ECRD 2014

5 Why attend ECRD as a Policy Maker/Regulator? Contribute to the discussion on EU Policies and national policies for rare diseases Network with rare disease patients as well as Member State representatives, regulators, payers, academia and industry from all over Europe Get the most updated information on the important breakthroughs in medical need driven research for rare diseases Participate in the discussion on improving diagnosis and delivering of adapted care at national level and across European boundaries Share ideas on how to improve the dialogue between all stakeholders to encourage orphan medicinal product development Exchange recommendations on how to address shortages in authorised medicines for rare diseases and how to find solutions Discover inspiring initiatives by patients to address the social challenges of rare diseases Join the coalition in support for the European Year for Rare Diseases in 2019 Contribute to the discussion on how to improve gene testing, genetic counselling and newborn screening at the European level Find out about the regulatory perspectives on private-public partnership for disease specific registries in Europe

6 Call for Poster Abstracts We would like to encourage patient organisations, academics, health care professionals and all other interested parties having conducted research or studies on rare diseases or public health projects to submit a poster abstract to the ECRD The themes mentioned below suggest issues and topics the Programme Committee would like to have addressed and should serve as a valuable guide as you develop your poster abstract. You are invited to submit poster abstracts that fall within the following themes: Theme 1 Improving Healthcare Services: Reports on experiences and best practices; initiatives on how to improve healthcare pathways; advances / breakthroughs for undiagnosed conditions; initiatives on how best to organise diagnostic services at national and European level; experiences and best practices on genetic testing / genetic counselling / new born screening. Theme 2 Knowledge Generation and Dissemination: Reports and outcomes of national and European rare disease specific patient registries; use of hospital computerised systems to increase knowledge of rare diseases; methods of evaluating the quality of the helpline from the point of view of the caller; good clinical practice guidelines; examples of successful awareness-raising campaigns for a rare disease(s). Theme 3 Research from Discovery to Patients: Examples of resource sharing among different stakeholders to help shape rare disease research policy in Europe; examples of public-private initiatives to address the gaps in research and create opportunities; breakthroughs in medical need driven research; examples of social research in the field of rare diseases. Theme 4 State of the Art and Innovative Practices in Orphan Products: examples of new international initiatives having an impact on the development of orphan medicinal products in Europe; recommendations on how to improve access to orphan medicinal products; initiatives on how to stimulate data collection for off-label use and access to therapies; education & training initiatives in medicines development for patients representatives. Theme 5 Emerging Concepts and Future Policies for Rare Disease Therapies: examples of non-traditional routes to securing access/availability to rare disease therapies; new initiatives at both global, regional and country level to support orphan discovery, development and availability; new approaches to address the challenges of developing orphan medicinal products and rare disease therapies in times of austerity; examples of re-purposing of drugs for rare diseases. Theme 6 Beyond Medical Care: research or studies identifying the specific social challenges of rare diseases; innovative solutions, services and programmes that address the social and other non-medical needs of rare disease patients; innovative schemes that may serve as models for promoting independent living; personal experiences of service users; methodologies and programmes for the training of social services providers; research or studies on the benefits for patients and families of respite, therapeutic recreation and adapted housing. Theme 7 Rare Disease Patient Groups Innovations: innovative strategies/approaches/services or projects which could potentially be useful to other patient groups/healthcare professionals/ academia or which deserve to be promoted internationally and recognised by national and European authorities. Theme 8 National Plans and Strategies for Rare Diseases. Theme 9 Other/open topic: what would you like to say about your disease, your actions, you vision? Poster abstract submissions for ECRD 2014 are now open. Please complete the online submission form. An automatic acknowledgement will be sent to the Main Author / Poster Abstract Submitter. If you do not receive an automatic or for any questions, please contact secretariat@ rare-diseases.eu. Poster applicants will be informed of the decision by the Poster Committee by the end of January Submissions: Deadline for Abstract Submission: 15 December 2013 Authors of selected poster abstracts will be notified by the end of January 2014 Guidelines: Abstract submissions accepted in English only. Abstract submissions are limited to 300 words. You may be co-author of any number of abstracts but presenting author of only one abstract. Please indicate if your abstract has already been presented at other events We look forward to your participation. For more information on the Call for Poster Abstracts, please call or secretariat@rare-diseases.eu

7 REGISTRATION FORM Brussels May Berlin 7th European Conference on Rare Diseases & Orphan Products (ECRD 2014 Berlin) 9 10 May 2014 Andel s Hotel, Berlin, Germany Meeting ID# Early-bird rates: Register before 15 February 2014 to benefit from preferential registration rates Special Sessions/meeting taking place on 8 May 2014: General Assembly 09:00-11:00 Open to members only Membership Meeting 11:30-17:00 Open to members only Patient Groups Welcome Reception 18:00-19:00 Open to Patient Groups only I will attend I will attend I will attend Delegate category** *All registration fees include German VAT (19%) Before 15 February 2014* After 15 February 2014* Optional Pre-conference tutorials 1-4 (industry) on 8 May :30-17: Optional Pre-conference tutorials 1-4 (academia) on 8 May :30-17: members ECRD Conference 9-10 May 2014 Patients Organisations (non- members) ECRD Conference 9-10 May Academics/Health care professionals/government workers/policy makers/payers/regulators ECRD Conference 9-10 May Pharmaceutical industry, ERTC members, consultants, investors ECRD Conference 9-10 May Medical students /post-graduate trainees ECRD Conference 9-10 May Please indicate which tutorial number you would like to attend: **Group discount/sme rates available. Please contact ecrd@diaeurope.org for more information. ATTENDEE DETAILS PAYMENT METHODS Credit cards are our preferred payment method PLEASE COMPLETE IN BLOCK CAPITAL LETTERS OR MAKE REGISTRATION EVEN SIMPLER BY ATTACHING THE ATTENDEE S BUSINESS CARD HERE Last Name First Name Company Prof Dr Ms Mr Please charge my credit card - credit card payments by VISA, Mastercard or AMEX can be made by completing the relevant details below. Please note that other types of credit card cannot be accepted. VISA MC AMEX Card N Job Title Address Expiry date Cardholder s name: Postal Code Country Telephone Fax * City Bank transfers: When DIA Europe completes your registration, an will be sent to the address on the registration form with instructions on how to complete the bank transfer. Payments in EURO should be addressed to Account Holder: DIA. including your name, company, Meeting ID# as well as the invoice number to ensure correct allocation of your payment. Payments must be net of all charges and bank charges must be borne by the payer. If you have not received your confirmation within five working days, please contact DIA Europe. *(Required to receive presentation download instructions) Eurordis reserves the right to include your name and affiliation on the attendee list. If you have not received your confirmation within five working days, please contact DIA Europe. By signing below, I confirm that I agree with Eurordis General Terms and Conditions Date Signature The registration fee includes: MEMBERS AND NON-MEMBERS: Access to all capacity-building workshops held on 8 May 2014 Participation in the Patients Group Welcome Reception held on 8 May 2014 Access to all sessions on 9 and 10 May 2014 including the Opening & Plenary Sessions Participation in the Networking event on 9 May 2014 Access to all other networking sessions Access to daily poster sessions featuring up to 100 posters Access to the Exhibit Area featuring exhibiting companies Endless opportunities to network with colleagues from around the globe Complimentary wireless Internet access in public areas of the andel s Hotel Complimentary daily refreshment breaks and lunches Conference bags, programmes and other conference materials ALL OTHER CATEGORIES OF PARTICIPANTS: Access to all sessions on 9 and 10 May 2014 including the Opening & Plenary Sessions Participation in the Networking events on 9 May 2014 and Themed networking sessions Access to daily poster sessions featuring up to 100 posters Access to the Exhibit Area featuring exhibiting companies Endless opportunities to network with colleagues from around the globe Complimentary wireless Internet access in public areas of the andel s Hotel Complimentary refreshment breaks and lunches Conference bags, programmes and other conference materials Pre-conference tutorials are separate ticketed events that require additional registration fees to attend (refer to table above for pricing). Pre-conference tutorials are designed for industry and academic participants but are not closed to other participants. Registration Cancellations/Refunds Cancellation deadline: 25 March, 2014 All cancellations will be charged: Members, non-members, academics, HCP, payers, regulators, government workers, students, postgraduate trainees and policy makers: 25 cancellation fee. Pharma Industry, ERTC members and consultants: 100 cancellation fee. With a WRITTEN notification postmarked, faxed or ed by 25 March 2014, you will be issued a full refund less the processing fee. No refunds will be issued for requests received after the cancellation deadline of 25 March All enquiries should be addressed to: DIA Europe Operations Team from Monday to Friday between 08:00 and 17:00 CET. ecrd@diaeurope.org Tel Fax