Honoring the Past, Navigating the Present,
Charting the Future
April 29 – 30, 2014
Sheraton Silver Spring Hotel
8777 Georgia Avenue
Silver Spring, MD 20910
CO-SPONSORED BY:
Critical Path Institute
U.S. Food and Drug Administration
The Patient-Reported Outcome (PRO) Consortium is a public-private partnership established by the Critical Path Institute (C-Path) in cooperation with the U.S. Food and Drug Administration (FDA) and the medical products industry in 2008. The PRO Consortium brings together scientists from C-Path, industry, academia, and regulatory agencies in a pre-competitive environment for the purpose of developing, evaluating, and qualifying PRO instruments for use as primary or secondary endpoint measures in clinical trials designed to evaluate treatment benefit.
On April 29-30, 2014 the FIFTH ANNUAL PATIENT-REPORTED OUTCOME CONSORTIUM WORKSHOP was held in Silver Spring, Maryland. The overall Workshop objectives were to:
- Provide updates on the PRO instrument development and qualification activities within the PRO Consortium’s working groups and beyond
- Discuss lessons learned regarding the practical, scientific, and regulatory issues surrounding the PRO instrument development and qualification process within a collaborative, pre-competitive, multi-stakeholder context
- Discuss the development and implementation of well-defined and reliable clinical outcome assessment (COA) tools for pediatric clinical trials in asthma
- Discuss the technical, scientific, and regulatory issues impacting the planning and implementation of electronic data collection of PRO endpoints in clinical trials
The following Workshop Agenda provides an overview of the day-and-a-half-long meeting as well as links to the slide sets and posters presented.
Workshop Agenda – Day 1
April 29, 2014
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7:30-8:30 am |
Registration and Continental Breakfast – Cypress Grand BallroomDay 1 Morning
Moderator: Ashley F. Slagle, MS, PhD – COA Qualification Scientific Coordinator and Endpoint Reviewer, Study Endpoints and Labeling (SEALD), Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA) |
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8:30-8:50 am |
Welcome and PRO Consortium Update Stephen Joel Coons, PhD — Executive Director, Patient-Reported Outcome (PRO) Consortium, Critical Path Institute (C-Path) |
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8:55-10:25 am |
Session 1: Advancing the Assessment of Outcomes Meaningful to Patients in Drug Development: A Brief History at the FDA and Beyond Moderator: Stephen Joel Coons, PhD – Executive Director, PRO Consortium, C-Path Presenters and Panelists: Robert Temple, MD – Deputy Center Director for Clinical Science and Acting Deputy Director of the Office of Drug Evaluation I, CDER, FDACatherine Acquadro, MD – Scientific Advisor, Mapi Research TrustDonald L. Patrick, PhD, MSPH – Professor and Director, Seattle Quality of Life Group, University of Washington Andrew E. Mulberg, MD, FAAP, CPI – Deputy Director, Division Gastroenterology and Inborn Error Products (DGIEP), OND, CDER, FDATara Symonds, PhD – PRO Lead, Pfizer, Ltd.Laurie Beth Burke, RPh, MPH – Founder, LORA Group, LLC Q & A |
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10:25-10:50 am |
Break – 25 min | |
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10:50-11:20 am |
FDA Update on DDT Qualification Program ShaAvhrée Buckman-Garner, MD, PhD, FAAP – Director, Office of Translational Sciences, CDER, FDA |
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11:25-12:25 pm |
Session 2 – Update on Clinical Outcome Assessment Qualification Program
Moderator: Ashley F. Slagle, MS, PhD – COA Qualification Scientific Coordinator and Endpoint Reviewer, SEALD, OND, CDER, FDA |
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12:25-1:25 pm |
Lunch – Magnolia Ballroom | |
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Day 1 Afternoon Moderator: Risa Hayes, PhD – Research Advisor, Global Patient Outcomes and Real World Evidence, Eli Lilly and Company |
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1:30-2:30 pm |
Session 3 – Lessons Learned from PRO Consortium Along the Path to PRO Instrument Qualification: A Case Study Moderator: Risa Hayes, PhD – Research Advisor, Global Patient Outcomes and Real World Evidence, Eli Lilly and Company Presenters and/or Panelists: Enkeleida Nikaï, MSc, MB – Senior Research Scientist, Global Patient Outcomes and Real World Evidence, Eli Lilly and CompanyApril N. Naegeli, DrPH, MPH – Senior Research Scientist, Global Patient Outcomes and Real World Evidence, Eli Lilly and Company Clifton O. Bingham III, MD – Associate Professor of Medicine, Divisions of Rheumatology and Allergy and Clinical Immunology at Johns Hopkins University; Director, Johns Hopkins Arthritis Center Amye L. Leong, MBA – President and CEO, Healthy Motivation; Director of Strategic Relations, Bone and Joint Decade, the Global Alliance for Musculoskeletal Health Lee S. Simon, MD, FACP, FACR – Principal, SDG LLC Ashley F. Slagle, MS, PhD – COA Qualification Scientific Coordinator and Endpoint Reviewer, SEALD, OND, CDER, FDA Sarah Yim, MD – Supervisory Associate Director, Division of Pulmonary, Allergy, and Rheumatology Products (DPARP), OND, CDER, FDA Q & A |
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2:35-3:35 pm |
Session 4 – Social/Digital Media: The Future of Qualitative Data Collection in the Context of Labeling Moderator: Margaret Rothman, PhD – Senior Director, PRO Group, Janssen Pharmaceutical Companies of Johnson and Johnson Presenters and/or Panelists: Elektra Papadopoulos, MD, MPH – Team Leader, Study Endpoints Team, SEALD, OND, CDER, FDA Q & A |
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3:35-3:55 pm |
Break – 20 min | |
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3:55-5:15 pm |
Session 5 – Developing Pediatric COA Measurement Strategy: A Case Study in Asthma Moderator: Elisabeth Piault-Louis, PharmD, MA – Senior PRO Scientist, Genentec0h Presenters and/or Panelists: Linda Nelsen, MHS – Director, Patient Reported Outcomes, GlaxoSmithKlineLouis Matza, PhD – Senior Research Scientist, Evidera Elektra Papadopoulos, MD, MPH – Team Leader, Study Endpoints Team, SEALD, OND, CDER, FDA Rob Arbuckle, MA, MSc – Director, Endpoint Development & Outcomes Assessment, Adelphi Values Laura Lee Johnson, PhD – Statistician, National Center for Complementary and Alternative Medicine (NCCAM), National Institutes of Health (NIH) Andrew E. Mulberg, MD, FAAP, CPI – Deputy Director, DGIEP, OND, CDER, FDA Hari Cheryl Sachs, MD – Pediatric Team Leader, Pediatric and Maternal Health Staff, OND, CDER, FDA Q & A |
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5:15-5:30 pm |
Day 1 Closing Remarks, Day 2 Preview Adjourn |
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6:00-7:30 pm |
Reception and Poster Session – Magnolia BallroomAsthma Cognition Depression Functional Dyspepsia Irritable Bowel Syndrome (IBS) Non-Small Cell Lung Cancer (NSCLC) Rheumatoid Arthritis |
Workshop Agenda – Day 2
April 30, 2014
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7:30-8:30 am |
Registration and Continental Breakfast – Cypress Grand Ballroom | |
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8:30-8:35 am |
Day 2 Moderator: Ari Gnanasakthy, MSc, MBA – Co-Director, PRO Consortium and Head, Patient Reported Outcomes, Novartis Pharmaceuticals Corporation | |
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8:40-9:40 am |
Session 6 – C-Path Collaborative Efforts to Address Specific ePRO Data Collection Challenges Moderator: Linda S. Deal, MS – Head of Patient-Reported Outcomes, Gastrointestinal Business Unit Lead, Shire Presenters and/or Panelists: Paul O’Donohoe, BSc – Director of Health Outcomes, CRF HealthSarah Fleming, MPH – Manager, Patient Reported Outcomes, Janssen Global Services Alexandra I. Barsdorf, PhD – Associate Director, PRO Center, Pfizer, Inc. Ari Gnanasakthy, MSc, MBA – Co-Director, PRO Consortium and Head, Patient Reported Outcomes, Novartis Pharmaceuticals Corporation Jonathan S. Helfgott, MS – Associate Director for Risk Science (Acting), Office of Scientific Investigations, CDER, FDA Cindy Howry, MS – Vice Director, ePRO Consortium and Vice President, eCOA/ePRO Product Strategy, Y-Prime Q & A |
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9:40-10:10 am |
Break – 25 min | |
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10:10-11:10 am |
Session 7 – Sponsor Infrastructure, Resources and Roles/Positions Needed to Support Successful Execution of ePRO/eCOA Strategies Moderator: Sue Vallow, RPh, MBA, MA – Senior Director, Patient Reported Outcomes, GlaxoSmithKline Presenters and/or Panelists: Sue Vallow, RPh, MBA, MA – Senior Director, Patient Reported Outcomes, GlaxoSmithKlineKathryn Engstrom – Data Scientist – Auto Immune, Eli Lilly and Company Jonathan S. Helfgott, MS – Associate Director for Risk Science (Acting), Office of Scientific Investigations, CDER, FDA Valdo Arnera, MD – General Manager, PHT Corporation Europe Linda S. Deal, MS – Head of Patient-Reported Outcomes, Gastrointestinal Business Unit Lead, Shire Jason Eger – Vice-President, Project Management, ERT Sarah Fleming, MPH – Manager, Patient Reported Outcomes, Janssen Global Services Q & A |
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11:15-12:15 pm |
Session 8 – ePRO: Bring Your Own Device (BYOD) – Application in Clinical Trials Moderator: Jason Lundy, PhD – Director, ePRO Consortium; Associate Director PRO Consortium, C-Path Presenters and/or Panelists:Hannah O’Gorman – ePRO Specialist, Exco InTouchTara Symonds, PhD – PRO Lead, Pfizer, Ltd. Jonathan S. Helfgott, MS – Associate Director for Risk Science (Acting), Office of Scientific Investigations, CDER, FDA Willie Muehlhausen, DVM – Vice President, Head of Innovation, ICON plc Q & A |
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12:15-12:30 pm |
Closing Remarks Adjourn |
