Day 1 Morning Moderator:

Michelle Campbell, PhD – Associate Director, Stakeholder Engagement and Clinical Outcomes, Office of Neuroscience (ON), Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA)

Welcome and Patient-Reported Outcome Consortium Update

Overview: Provide a high-level summary of the recent accomplishments and ongoing activities within the Patient-Reported Outcome Consortium

Presenter:

Sonya Eremenco, MA – Executive Director, Patient-Reported Outcome (PRO) Consortium, Critical Path Institute (C-Path)

Session 1: FDA Update

Overview: Provide an update on FDA’s Clinical Outcome Assessment Qualification Program and other FDA initiatives

Moderator:

Michelle Campbell, PhD – Associate Director, Stakeholder Engagement and Clinical Outcomes, Office of Neuroscience, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Presenters:

Robyn Bent, RN, MS – Director, Patient-Focused Drug Development Program, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Selena Daniels, PharmD, PhD – Clinical Outcome Assessment Team Leader, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Lili Garrad, PhD – Lead Mathematical Statistician Team Leader, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Laura Lee Johnson, PhD – Director, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Jessica Mavadia-Shukla, PhD – Program Director, Medical Device Development Tools, Center for Devices and Radiological Health, U.S. Food and Drug Administration

David S. Reasner, PhD – Division Director, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Q & A

Session 2: Patient Experience Data: Use in Regulatory Decision Making and Labeling

Overview: Discuss patient experience data and its use in regulatory decision making

Moderator:

Sonya Eremenco, MA – Executive Director, Patient-Reported Outcome Consortium, Critical Path Institute

Presenters and Panelists:

Robyn Bent, RN, MS – Director, Patient-Focused Drug Development Program, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Michelle Campbell, PhD – Associate Director, Stakeholder Engagement and Clinical Outcomes, Office of Neuroscience, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Ellyn Kodroff, BS – President and Founder, CURED (Campaign Urging Research for Eosinophilic Diseases) Nfp

Sarrit Kovacs, PhD – Clinical Reviewer, Division of Gastroenterology, Office of Immunology and Inflammation, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Marc Yale –Advocacy and Research Coordinator, International Pemphigus Pemphigoid Foundation (IPPF)

Q & A

Day 1 Afternoon Moderator:

Josephine Norquist, MS – Executive Director, Patient-Centered Endpoints & Strategy Lead, Merck & Co., Inc. and Industry Co-Director, Patient-Reported Outcome Consortium

Session 3: Qualitative Methods in Clinical Trials: Opportunities and Challenges

Overview: Discuss methodological approaches to qualitative data collection in clinical trials, and the opportunities and challenges involved

Moderator:

Maria Mattera, MPH – Scientific Director, Patient-Reported Outcome Consortium, Critical Path Institute

Presenters:

Asha Hareendran, PhD – Patient Centred Outcomes Research (PCOR) Excellence Lead, UCB Biopharma Srl

Nicola Williamson, MSc – Associate Director, Patient-Centered Outcomes, Adelphi Values

Jane R. Wells, MSc – Clinical Outcomes Assessment Lead, Sanofi

Calvin N. Ho, PhD – Associate Director, Patient Centered Science, AstraZeneca

Bellinda King-Kallimanis, PhD – Director of Patient-Focused Research, LUNGevity Foundation

Panelists:

Naomi Knoble, PhD – Associate Director of Rare Disease Measurement Science, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences, Office of New Drugs, Center for Drug Evaluation Research, U.S. Food and Drug Administration

Marc Yale – Advocacy and Research Coordinator, International Pemphigus Pemphigoid Foundation (IPPF)

Q & A

Session 4: Applying Methodologies for Estimating Meaningful Within-person Change Thresholds: Considerations and Alternative Approaches

Overview: Examine four examples that advance the estimation and use of meaningful within-person change thresholds

Moderator:

Rebecca M. Speck, PhD, MPH – Clinical Outcome Assessment Scientist, Eli Lilly and Company

Presenters:

Elizabeth (Nicki) Bush, MHS – Senior Director, Endpoints and Measurement Strategy, Janssen Pharmaceutical Companies of Johnson & Johnson

Devin Peipert, PhD – Assistant Professor of Medical Social Sciences, Northwestern University

Karon Cook, PhD – Research Professor (Retired), Feinberg School of Medicine, Northwestern University

Bill Byrom, PhD – Vice President, Product Intelligence and Positioning, and Principal, eCOA Science, Signant Health

Panelists:

Selena Daniels, PharmD, PhD – Clinical Outcome Assessment Team Leader, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Monica Morell, PhD – Patient-Focused Statistical Support Reviewer, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Q & A

Day 1 Closing Remarks

Sonya Eremenco, MA – Executive Director, Patient-Reported Outcome Consortium, Critical Path Institute

Adjourn

Reception and Poster Session – Magnolia Ballroom

Chronic Heart Failure Working Group
Cognition Working Group
Depression Working Group 2.0
Functional Dyspepsia Working Group
Irritable Bowel Syndrome Working Group
Multiple Sclerosis Working Group
Pediatric Asthma Working Group
Rheumatoid Arthritis Working Group
Small Cell Lung Cancer Working Group
Rare Disease COA Consortium
eCOA Consortium

Day 2 Moderator:

Lindsey Murray, PhD, MPH – Executive Director, Rare Disease Clinical Outcome Assessment Consortium, Critical Path Institute

Session 5: Strategies for Use of COAs in Rare Disease Pediatric Populations

Overview: Explore methodological challenges in research involving rare pediatric populations, especially in children less than 5 years of age

Moderator:

Lindsey Murray, PhD, MPH – Executive Director, Rare Disease Clinical Outcome Assessment Consortium, Critical Path Institute

Presenters:

Lindsey Murray, PhD, MPH – Executive Director, Rare Disease Clinical Outcome Assessment Consortium, Critical Path Institute

Dawn Phillips, PT, MS, PhD – Director, Clinical Scientist, Outcomes Research, REGENXBIO Inc.

Ebony Dashiell-Aje, PhD – Executive Director & Head, Patient Centered Outcomes Science, BioMarin Pharmaceutical, Inc.

Naomi Knoble, PhD – Associate Director of Rare Disease Measurement Science, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences, Office of New Drugs, Center for Drug Evaluation Research, U.S. Food and Drug Administration

Panelists:

Teresa Buracchio, MD – Director (Acting), Office of Neuroscience, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Emily Freilich, MD – Deputy Director (Acting), Division of Neurology I, Office of Neuroscience, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Cara O’Neill, MD – Chief Science Officer, Cure Sanfilippo Foundation

Q & A

Session 6: eCOA: COA Program Projects and Collaborations Update

Overview: Explore two collaborative projects leveraging sensor-based measurement of activity and discuss the challenges involved in assessing algorithms for deriving interpretable metrics from raw sensor data

Moderator:

Scottie Kern, BSc (Hons) – Executive Director, Electronic Clinical Outcome Assessment Consortium, Critical Path Institute

Presenters:

Scottie Kern, BSc (Hons) – Executive Director, Electronic Clinical Outcome Assessment Consortium, Critical Path Institute

Josephine Norquist, MS – Executive Director, Patient-Centered Endpoints & Strategy Lead, Merck & Co., Inc. and Industry Co-Director, Patient-Reported Outcome Consortium

Christine Guo, PhD – Chief Scientific Officer, ActiGraph

Ronenn Roubenoff, MD, MHS – Global Head, Translational Medicine Discovery & Profiling, Novartis Institutes for Biomedical Research

Gül Erdemli, MD, PhD – Global Program Regulatory Director, Novartis Pharmaceuticals Corporation

Panelist:

David S. Reasner, PhD – Division Director, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Q & A

Workshop Wrap Up

Sonya Eremenco, MA – Patient-Reported Outcome Consortium, Critical Path Institute

Cheryl D. Coon, PhD – Vice President, Clinical Outcome Assessment Program, Critical Path Institute

Adjourn