Program

Sessions

Session I: Introduction to Core Outcome Sets

Monday, 27 July | 1:15 pm – 2:45 pm

 

Description:

Core outcome sets can provide value by creating a common understanding of important treatment outcomes for a specific disease area across a range of settings. In this session, speakers from different areas of interest will describe key issues in the development, evaluation, and use of core outcome sets.

Speakers:

Elektra Papadopoulos, MD MPH, U.S. Food and Drug Administration, Silver Spring, Maryland, United States

Elektra Papadopoulos, MD MPH, leads the Division of Clinical Outcome Assessment in the Office of New Drugs in the Center for Drug Evaluation and Research (CDER) at FDA. The Division provides consultation to CDER’s therapeutic area review divisions as well as other FDA Centers on clinical outcome assessments (COAs) including their development and validation for use as study endpoints in medical product development. She has participated in the development of FDA policy related to COAs including methodologic and disease-specific guidance documents and leads CDER’s COA Drug Development Tool Qualification Program.

 

Donna Messner, PhD, Center for Medical Technology Policy, Baltimore, Maryland, United States

Donna Messner, PhD, is President and CEO of the non-profit Center for Medical Technology Policy (CMTP), where she leads programs to promote clinical evidence development that is patient-centered, stakeholder-informed, and relevant to real-world decision-making. She leads CMTP’s multi-stakeholder Green Park Collaborative (GPC) and developed GPC’s program for core outcome sets (COS), including methods for prioritizing patient-important outcomes in COS. She was previously the Gordon Cain Fellow in Technology, Policy, and Entrepreneurship at the Chemical Heritage Foundation; a fellow in University of Pennsylvania’s Penn Center for the Integration of Genetic Health Care Technologies; and a long-time laboratory director and senior scientist.

 

Clifton Bingham

Session II: Methodological Approaches for Development of Core Outcome Sets

Monday, 27 July | 3:00 pm – 4:30 pm

 

Description:

Methodological approaches to developing core outcome sets vary widely for a number of reasons, such as differences in project scope, varying definitions of an “outcome,” differing approaches to combining similar concepts to reduce redundancies, differing approaches to undertaking identification of concepts and measures and of evaluating measures, and other attributes. In this session, speakers will describe the implications of these varied approaches using examples from their experience and discuss how some methodological pitfalls can be avoided.

Speakers:

Lee Aiyegbusi, PhD, Centre for Patient-Reported Outcome Research (CPROR), University of Birmingham, Birmingham, United Kingdom

Dr. Lee Aiyegbusi is a Research Fellow and a Deputy Director at the Centre for Patient-Reported Outcome Research (CPROR), University of Birmingham, UK. He is currently exploring the development and use of core outcome sets in inflammatory diseases and the regulatory requirements for drug approvals and labeling. His research interests also include patient public involvement in research and the optimization of research impact. His previous research focused on the use of Patient-Reported Outcome Measures in the routine management of patients with advanced chronic kidney disease. He was involved in the development of a renal ePROM system currently being piloted.

 

Jakob Bjørner, MD PhD, Optum Patient Insights, Johnston, Rhode Island, United States

Dr. Jakob Bjørner is Chief Science Officer at Optum Outcomes, Professor of Epidemiology at the Danish National Research Centre for the Working Environment, and Honorary Professor at the University of Copenhagen. He specializes in applying modern psychometric theory to the measurement of health outcomes. In collaboration with John E. Ware Jr. and the science team at Optum Outcomes (formerly QualityMetric), Dr. Bjørner developed the DYNHA® computerized adaptive testing software. They have subsequently developed several computerized adaptive tests of generic and disease-specific patient-reported health outcomes. A physician by training, he also holds a doctorate in medical sociology from the University of Copenhagen.

 

Paula Williamson

Session III: Stakeholder Perspectives on Core Outcome Sets

Tuesday, 28 July | 9:15 am – 10:45 am

 

Description:

In the context of medical product development and health outcomes assessment, it is critically important to understand whether the outcome measures and endpoints adequately capture treatment benefit in the target population, for the needs of various key stakeholders. Stakeholders may, at times, have varying perspectives on what might constitute meaningful information to support their needs. During this session, speakers representing the patient, payer, and regulatory perspectives will discuss their views on and experiences with core outcome sets.

Speakers:

Eleanor Perfetto, PhD, National Health Council, Washington, D.C., United States

Dr. Eleanor M. Perfetto named Executive Vice President in January 2019. She also holds a part-time faculty appointment at the University of Maryland, Baltimore School of Pharmacy where she is Professor of Pharmaceutical Health Service Research. Her research and policy work primarily focus on patient engagement in comparative effectiveness and patient centered-outcomes research, medical product development; patient-reported outcome selection and development; and health care quality. Dr. Perfetto holds BS and MS degrees in pharmacy from the University of Rhode Island, and a PhD from the University of North Carolina School of Public Health with concentrations in health policy and epidemiology.

 

Sally Okun, RN MMHS, UnitedHealth Group Research & Development, White Stone, Virginia, United States

Sally Okun joined UnitedHealth Group R&D in 2020. Previously she spent 12 years at PatientsLikeMe where she developed the site’s patient voice ontology, drug safety platform, FDA research collaboration and the Ethics and Compliance Advisory Board. She has been a leader in bringing the voice of patients and public participation into research, care and policy on a national and global level. As an RN she practiced as a community-based palliative care specialist for much of her career. She completed her graduate work at the Heller School for Social Policy & Management and was a 2010 NLM Fellow in Biomedical Informatics.

 

Robyn Bent, U.S. Food and Drug Administration, Silver Spring, Maryland, United States

Robyn Bent joined the US FDA in 2019 as the director of CDER’s Patient-Focused Drug Development (PFDD) Initiative, an effort to systematically obtain patient input and facilitate the incorporation of meaningful patient input into drug development and regulatory decision making. Prior to joining FDA, she was a Chief Scientific Program Specialist at the National Institutes of Health. Captain Bent has extensive experience in clinical trial design, conduct, and oversight. She earned a Bachelor of Science in Nursing from The Catholic University of America and a Master of Science degree from the George Washington University.

 

Tom Valuck, MD JD, Discern Health, Washington, D.C., United States

Dr. Tom Valuck is a Partner at Discern Health. He is a thought leader on healthcare system transformation and helps lead the firm’s clients achieve better health and health care outcomes at lower cost. Before joining Discern, Dr. Valuck served as Senior Vice President for Strategic Partnerships at the National Quality Foundation (NQF) where he oversaw the Measure Applications Partnership (MAP) and the National Priorities Partnership (NPP). Dr. Valuck also served as a Senior Advisor and Medical Officer at the Centers for Medicare and Medicaid Services, where he advised senior agency leadership regarding Medicare payment and quality of care.

Session IV: Future Challenges with Core Outcome Sets

Tuesday, 28 July | 1:30 pm – 3:00 pm

 

Description:

While core outcome sets hold promise to standardize our understanding of treatment outcomes, challenges remain with respect to defining the best methods to define COS, gaining agreement across stakeholder perspectives, efficient means of implementation and maintenance of the COS.  Speakers familiar with developing, promoting, and using core outcomes sets will reflect on the challenges that need to be addressed before the full value of COS can be realized.

Speakers:

Donna Messner, PhD, Center for Medical Technology Policy, Baltimore, Maryland, United States

Donna Messner, PhD, is President and CEO of the non-profit Center for Medical Technology Policy (CMTP), where she leads programs to promote clinical evidence development that is patient-centered, stakeholder-informed, and relevant to real-world decision-making. She leads CMTP’s multi-stakeholder Green Park Collaborative (GPC) and developed GPC’s program for core outcome sets (COS), including methods for prioritizing patient-important outcomes in COS. She was previously the Gordon Cain Fellow in Technology, Policy, and Entrepreneurship at the Chemical Heritage Foundation; a fellow in University of Pennsylvania’s Penn Center for the Integration of Genetic Health Care Technologies; and a long-time laboratory director and senior scientist.

 

Chris Sidey-Gibbons, PhD, MD Anderson Cancer Center, Houston, Texas, United States

Dr. Sidey-Gibbons is a data scientist, psychometrician, and health science researcher. His research focuses on the development, evaluation, and implementation of tools to collect, analyze, and report patient data. He is Associate Professor and Deputy Chair at the Department of Symptom Research at MD Anderson Cancer Center and Health Director for the University of Cambridge Psychometrics Center Concerto Platform.

 

Laura Lee Johnson

Workshops

Workshop I: Methodological Approaches for Development of Core Outcome Sets

Monday, 27 July | 4:30 pm – 5:30 pm

Workshop II: Examining the Impact of Stakeholder Perspectives on COS

Tuesday, 28 July | 11:00 am – 12:00 pm

 

Description

After learning about the various stakeholder perspectives that factor into the development of a core outcome set, meeting attendees will break into small groups for a brief workshop session. Groups will be provided with a brief case study describing a single disease area. Each group will be assigned a role (payer, patient, regulatory) and asked to consider how they might approach developing a COS for the specific disease area. Groups should consider the unique concerns and key concepts of interest of their stakeholder group, challenges their group might face, potential allies and adversaries, etc. After discussion, one person from each group will report, giving the attendees a chance to compare how different groups approached the same case study.

Schedule of Events

Monday, 27 July 2020

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1:00 PM – 1:15 PM

Welcome & Introduction

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1:15 PM – 2:45 PM

Session I: Introduction to Core Outcome Sets

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2:45 PM – 3:15 PM

Coffee Break

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3:15 PM – 4:45 PM

Session II: Methodological Approaches for Development of Core Outcome Sets

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4:45 PM – 5:45 PM

Workshop I: Methodological Approaches for Development of Core Outcome Sets

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5:45 PM – 6:45 PM

Welcome Reception

Tuesday, 28 July 2020

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8:30 AM – 9:00 AM

Breakfast

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9:00 AM – 9:15 AM

Welcome to Day 2

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9:15 AM – 10:45 AM

Session III: Stakeholder Perspectives on Core Outcome Sets

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10:45 AM – 11:15 AM

Coffee Break

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11:15 AM – 12:15 PM

Workshop II: Examining the Impact of Stakeholder Perspectives on COS

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12:15 PM – 12:45 PM

Workshop II Recap

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12:45 PM – 1:45 PM

Lunch

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1:45 PM – 3:15 PM

Session IV: Future Challenges with Core Outcome Sets

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3:15 PM – 3:30 PM

Symposium wrap up

The International Society for Quality of Life Research (ISOQOL) is a global community of researchers, clinicians, health care professionals, industry professionals, consultants, and patient research partners advancing health related quality of life research (HRQL).

Together, we are creating a future in which patient perspective is integral to health research, care and policy.