Thursday, 10 September | 9:00 am – 10:30 am EDT

 

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:

Clifton (Bing) Bingham, MD, Johns Hopkins, Baltimore, Maryland, United States

Dr. Bingham is a rheumatologist and Professor of Medicine at Johns Hopkins University, where he directs the Arthritis Center and serves as Deputy Director of Research for the Division of Rheumatology. He is also on the executive committee for OMERACT (Outcome Measures in Rheumatology), an international multi-stakeholder organization established to identify outcomes for clinical trials, including Core Set development, critical domain definition, and optimal instrument selection. He has a longstanding interest in outcome assessment in rheumatic diseases, especially PROs and PROMIS measures and how these can be incorporated in clinical trials, observational studies, and clinical practice settings.

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.

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.

Thursday, 10 September | 10:30 am – 12:00 pm EDT

 

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:

Paula Williamson, PhD FRSS FMedSci, COMET Initiative Management Group, University of Liverpool, Liverpool, United Kingdom

Paula Williamson is Professor of Biostatistics at the University of Liverpool, and was Head of the Department of Biostatistics from 2002 until 2018. Her research has focussed on clinical trials, meta-analysis, and health outcome selection. She leads the MRC/NIHR Trials Methodology Research Partnership. She is an NIHR Senior Investigator, a Fellow of the Academy of Medical Sciences, and gave the Bradford Hill Lecture in 2017. Paula co-founded the COMET (Core Outcome Measures in Effectiveness Trials) Initiative in 2010, to improve the quality and relevance of health research to decision makers including patients, health professionals, regulators and policymakers.

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.

Thursday, 17 September | 9:00 am – 10:30 am EDT

 

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:

Sally Okun, RN MMHS, Independent Consultant, White Stone, Virginia, United States

Sally Okun, an independent consultant focused on person-generated data and patient/public involvement in care, research and policy, was until recently the Director, Policy and Ethics at UnitedHealth Group Research & Development. 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.

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.

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.

Thursday, 17 September | 10:30 am – 12:00 pm EDT

 

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:

Laura Lee Johnson, PhD, U.S. Food and Drug Administration, Silver Spring, Maryland, United States

Laura Lee Johnson, PhD, is a division director and the Patient Focused Drug Development liaison for the Office of Biostatistics at the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER). She specializes in design, logistics, implementation, and analysis of research studies ranging from clinical outcome assessment (COA) qualification to trials of all sizes. She works across FDA on patient focused drug development initiatives. Prior to working at the FDA she spent over a decade at the U.S. National Institutes of Health working on and overseeing clinical research and research support programs.

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.