What is a Special Interest Group (SIG)?

PATIENT REPORTED OUTCOMES RESEARCH SPECIAL INTEREST GROUP Open Meeting 20 th Annual European Congress November 6, 2017 Glasgow, Scotland What is a Special Interest Group (SIG)? Member-driven group interested in a specific topic SIG membership is open to all ISPOR members PURPOSE Monitor & share information related to the topic To engage ISPOR members in projects that advance health economics and outcomes research and the use of this research in health care decisions 2 1

New Special Interest Group Structure Special Interest Group Chair, Past Chair, Chair-elect Key Project Working Group* (Co-chairs) (i.e. manuscript) Member Engagement Working Group (Co-chairs) (i.e. Webinars SIG Membership * Working group formally known as Leadership Group 3 Governance Chair elect Chair Past Chair (chair will move to this position next year) Each chair serves a 1-year term Working Group Co-Chairs Key Project Serve until the project is complete (average 24 months) Member Engagement Projects Serves for at least the year To be considered for chair positions please submit your current CV to ttesoro@ispor.org 4 2

Chair, Past Chair, Chair-elect Responsibilities Provide overall direction and leadership Identify the topics the SIG members would like to address Work with the Working Group co-chairs to ensure the timeliness of the key project Categorizing topics into long, short term projects Address any issues within the group Identify the appropriate platforms for delivery 5 Chair, Past Chair, Chair-elect Responsibilities Provide quarterly updates to the SIG membership Report the progress of the SIG projects Recruit new SIG members Current activities, new developments in field via the yearly business and project plan to HSPC 6 3

Working Group The Working Group provides leadership and contributes to the development of the project Each SIG will have at least 2 Working Groups Key Project (i.e. Manuscript in Value in Health or Value Outcomes & Spotlight, Short Courses) Member Engagement (i.e. webinars, virtual issue panel, workshop, etc.) SIG membership is open to all stakeholders in all geographic areas 7 Key Project Working Group Manuscript: Value in Health or Value Outcomes & Spotlight Short Courses Tools for Website The Working Group Co-chairs will be responsible for: Leading the project and teleconference Ensuring adherence to ISPOR processes and working group deadlines Address any conflicts or issues 8 4

Member Engagement Working Group Webinar/speaker Journal club Technical workshop series Presentations on Working Group products ISPOR forums on Key Project, or other presentations Other organization presentations Encouraged to be creative The Working Group Co-chairs will be responsible for: Work with SIG chairs to identify member engagement topics Identifying speakers, articles, topics for member engagement activities Ensuring that all member engagement activities take place 9 SIG Benefit Share and exchange information with other members from different stakeholder groups Networking Collaborating with HEOR experts Contribute to the Science Develop ISPOR educational content 10 5

Publication Ethics Ten years ago, the ISPOR Board of Directors instituted a Scientific and Health Policy Group publication rule: No member of an ISPOR Task Force or Special Interest Group should publish any material from an upcoming report, public presentation, or project deliverable without first consulting the larger group for permission prior to submission and publication." ISPOR Code of Ethics: Members should NOT publish any material relating to the activities of an ISPOR Task Force, Special Interest Group or other ISPOR group at any stage, without first consulting fellow group members / co-authors for permission. 11 QUESTIONS? Thank You 6

Next Steps Identify: Interest in Topic List of Potential Research Topics Which topics are: Key Project Member Engagement Project Submit proposal to HSPC 13 Differentiating patient engagement from PRO goals (Bryan Bennett) Patient engagement goal To facilitate the involvement of patient representatives in the research process. The group will define key terms, and examine existing theoretical frameworks of patient engagement in health research and outcomes process. Patient reported outcomes goal To identify new and best methodological advances in the field of patient reported outcome instrument development and use. The group will liaise with patient engagement group in terms of patient involvement but has a distinct focus on implementation and methods. It may also produce materials that could be of use in other psychometric clinical outcomes assessments. 14 7

Potential Research Topics 1. RW data and PRO (Angela Rylands; 10 mins) 2. PRO results simplification (Katja Rüdell; 10 mins) 3. PRO analyses and psychometric methods (Stacie Hudgens; 10 mins) 15 Key Projects of the PRO group 1. RW data and PRO (Angela Rylands; 10 mins) 2. PRO results simplification (Katja Rüdell; 10 mins) 3. PRO analyses and psychometric methods (Stacie Hudgens; 10 mins) 16 8

Who is talking about patient centred outcomes (PCO) data collected via real work research (Angela Rylands) Developing a Framework for Regulatory Use of Real- World Evidence Conference theme Embracing Complexity: Using PROs to Generate RWE Why is there increasing interest in PCO in the real world? Patients: - Track symptoms over time - In turn can benefit patients quality of life - Lengthen survival - Shared decision making Patients Physicians Physicians: - Clinical effectiveness from the patients perspective - Enhance patient-clinician communication -Increased data for comparative effectiveness research Payers: - Reduce emergency department use - Increased data for resource use or cost-benefit assessments Payers Regulators Regulators: - Use of novel RWE trials and methodologies - Want to understand role of RW data to measure drug effectiveness 9

What challenges are there with PCO data in the real world? Standards for PCO in RW to define; appropriate frequency of data collection minimising missing data rules for analysing PCO data in RW User Guides for PCO data collection in the RW Add to the ISOQOL 2011 guidelines for implementing PROMs in clinical practice Systematically integrate PCO data collection into routine clinical care / RW Standardisation of methods for integrating PCO instruments into clinical workflow PCO data collection via online patient portals? Information in a flexible user-configurable format? Education and incentives around collecting PCO data in the RW PCO data interpretation for clinicians / health care providers how do providers with the flow of information Define of best in class PCO instruments in RW what constitutes the responsiveness of a PRO in a RW stetting? Key project 1 PCO in the Real World This key project aims to address the use of PCO in real world studies. It is necessary to identify the role of PCO in RW studies and to determine if there are any methodological differences in the real world verses clinical trials. The deliverables from this key project would include the following: Envisaged steps: Submission of a forum at a forthcoming ISPOR conference to discuss the use of PCO in RWE Development of a PCO RWE implementation guidelines that will aggregate the issues around RWE or clinical use of PRO and inclusion in medical records. Development of a manuscript for submission to Value in Health setting the standards for the use of PCO data collection in RW research. 10

Simplification and reduction of PRO results complexity (Katja Rüdell) PRO data is very complex (similar to nutritional values) There are many different components to consider (the absolute values at baseline, the distribution and change over time) ISPOR presentation from 2016 lots of different tools available to researchers so it gets very difficult for the average researcher consumer to understand what it means Not all teams or consumers will have a PRO researcher at hand to help them interpret the data. What to do next? Nutritional intake is equally complex Key project 2 Simplifying PRO results This key project aims to simplify PRO data to such an extent that it becomes accessible to patients, researchers and clinicians Envisaged steps: A) Engaging with patients/caregivers to discuss what information they would expect to see in findings from clinical trial results. We envisage this could be done through the following ways: A brief literature review to identify currently how information is presented to patients. Conduct online focus groups to identify what information is most relevant and useful to patients that considers: a) Patient Reported Content that should be displayed on the label/ in the product description or in manuscripts 1. Symptoms, 2. Side effects, 3. Functioning / QOL and/ or 4. Intrusiveness /complexity of the medical treatment). b) Patient relevant therapeutic effects 1. Onset of effect (how long will it take to see the onset on symptoms or side effects?), 2. Duration of effect (how long will I experience the benefit of symptom relief?) 3. Strength of effect (will this medication help me have a meaningful improvement?) 11

Key project 2 B) The development of education materials for patients and caregivers to better understand patient reported effects that are observed from a trial within a quick study of a label or a research publication. Graphical displays that speak to this will be tested with patients. It is envisaged that this work will aim to simplify the information and make symptoms, side effects, functioning and quality of life findings accessible to patients, caregivers and payers. We will partner with a group of caregivers and patients initially to discuss their evidence requirements and provide solutions that address these issues. The meetings will be held as webinars so that patients from different countries can attend. Development of a manuscript outlining the activities a. and b. above for submission in Value in Health Development of PRO results template slide deck for distribution amongst ISPOR members Key project 3 Statistical and Psychometric Analytic Solutions (Stacie Hudgens) This key project aims to synthesize cross-sectional and longitudinal statistical and psychometric methods into a best practice guide for analysis and visualization of patient outcomes data Stacie is Chief Executive Officer and Strategic Lead at Clinical Outcomes Solutions with over 20 years of Pharmaceutical industry, academic and consulting experience and has a specialism in Quantitative Science. She is currently the co-chair of the Psychometric Special Interest Group at ISOQOL. 12

Key project 3 Statistical and Psychometric Analytic Solutions (Stacie Hudgens) Current Guideline Documents COSMIN (COnsensus-based Standards for the selection of health Measurement Instruments) PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) MOOSE (Meta-analysis Of Observational Studies in Epidemiology) STARD (Standards for Reporting Diagnostic Accuracy) CONSORT (CONsolidated Standards Of Reporting Trials) Lacks guidance on reporting of PROs PRO data increasingly used for clinical decision making as well as comparative effectiveness, health policy, and reimbursement decisions Trials that incorporate PROs as an outcome should therefore be designed, analysed, and reported well. Key project 3 Statistical and Psychometric Analytic Solutions (Stacie Hudgens) Current Guideline Documents CONSORT PRO 2013 extension of the CONSORT 2010 checklist to facilitate optimal reporting of RCTs in which PROs are the primary or secondary endpoints Developed based on the methodological framework for guideline development proposed by the EQUATOR network (Enhancing the QUAlity and Transparency Of health Research) Provides guidance for authors of trials describing PROs Five CONSORT PRO checklist items 1) PROs identified as 1 or 2 outcomes in the abstract 2) Description of PRO hypotheses and relevant domains provided 3) Evidence of PRO reliability and validity be provided or cited 4) Statistical approaches for dealing with missing data explicitly stated 5) PRO-specific limitations and generalizability to other populations and clinical practice be discussed Calvert M, Blazeby J, Altman DG, et al. Reporting of Patient-Reported Outcomes in Randomized Trials: The CONSORT PRO Extension. JAMA 2013; 309(8): 814-822 13

Key project 3 Statistical and Psychometric Analytic Solutions (Stacie Hudgens) Best practice guide for analysis and visualization of patient outcomes data Envisaged steps: Conduct a brief literature review of published statistical and clinical trial patient outcomes data to identify statistical and psychometric parameters commonly used to present as well as interpret data Based on findings from the literature review, conduct a web-based survey circulated to ISPOR membership Prepare data visualization graphics to guide the interpretation of complex analyses for non-statisticians and psychometricians used in published clinical trial results Outputs: Development of best practice guide(s) for analysis and interpretation of patient outcomes data (manuscript or slide deck for ISPOR distribution) one for statistical analysis and one for psychometric analysis Member Engagement Project The PRO working group has identified 3 key projects which will all require ISPOR member use and engagement. It is therefore currently envisaged that member engagement will be driven from these projects. Additionally in the field of PRO research the use of electronic devices to collect PRO data and translation is very crucial for efficient and robust data collection we therefore envisage that we will also collaborate with CPATH consortiums epro group and run webinars on new and innovative translation methods as well as epro advances. (Sue Vallow will act as liaison with CPath) 28 14

Interested in participating in the leadership group forming the SIG projects please contact Theresa Tesoro ttesoro@ispor.org 29 15