April 24 – 25, 2019
Sheraton Silver Spring Hotel
8777 Georgia Avenue
Silver Spring, MD 20910
On April 24-25, 2019 the Tenth Annual Patient-Reported Outcome Consortium Workshop was held in Silver Spring, Maryland. The overall Workshop objectives were to:
- Provide an update on FDA’s Clinical Outcome Assessment (COA) Qualification Program and other initiatives;
- Describe new and emerging approaches to the assessment of clinical outcomes in rare diseases;
- Discuss efforts to qualify a performance outcome assessment tool for early Alzheimer’s disease;
- Explore the construction of clinical endpoints using activity monitor data in chronic heart failure trials;
- Discuss how sponsors and eCOA providers can work together to optimize COA data collection in trials; and
- Describe ways to use patient input to estimate meaningful within-patient change at the scale score level.
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.
Agenda – Day 1
|
7:30–8:30 am |
Registration and Continental Breakfast – Cypress Ballroom |
| Day 1 Morning Moderator: Michelle Campbell, PhD – Senior Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, Division of Neurology Products (DNP), Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA) | |
|
8:30–8:50 am |
Welcome and Patient-Reported Outcome Consortium Update
Stephen Joel Coons, PhD – Executive Director, Patient-Reported Outcome (PRO) Consortium, Program Officer, Clinical Outcome Assessment Program, Critical Path Institute (C-Path) |
|
8:50–10:20 am |
Session 1: Update from FDA
Moderator: Michelle Campbell, PhD – Senior Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, DNP, OND, CDER, FDA Presenters:Peter Stein, MD – Director, OND, CDER, FDAElektra Papadopoulos, MD, MPH – Associate Director, Clinical Outcome Assessments (COA) Staff, OND, CDER, FDA Michelle Campbell, PhD – Senior Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, DNP, OND, CDER, FDA Q & A |
|
10:20–10:45 am |
Break – 25 min |
|
10:45 am–12:15 pm |
Session 2: Leveraging Information that Can Inform the Evaluation of Clinical Benefit in Rare Diseases
Moderator: Michelle Campbell, PhD – Senior Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, DNP, OND, CDER, FDA Presenters:Lucas Kempf, MD – Associate Director, Rare Diseases Program (acting), OND, CDER, FDADylan Trundell, MSc – Senior Outcomes Research Scientist, Patient-Centered Outcomes Research, Roche Mindy Leffler, MEd – President, Casimir Panelists: Billy Dunn, MD – Director, Division of Neurology Products, OND, CDER, FDA Lili Garrard, PhD – Senior Statistical Reviewer, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences (OTS), CDER, FDA Montserrat Vera-Llonch, MD, MPH, MSc – Senior Director, Global Outcomes Research and Epidemiology, Takeda Q & A |
|
12:15–1:15 pm |
Lunch – Magnolia Ballroom, Elm I and Elm II |
| Day 1 Afternoon Moderator: Linda M. Nelsen, MHS – Senior Director and Head, Patient Centered Outcomes, Value Evidence and Outcomes, GlaxoSmithKline and Industry Co-Director, PRO Consortium | |
|
1:15–2:45 pm |
Session 3: Cognition Working Group Case Study
Moderator: Katy Benjamin, PhD – Director, HEOR, Patient Centered Outcomes, AbbVie Inc. Presenters:Richard Keefe, PhD – CEO, VeraSci, Inc., Professor of Psychiatry, Psychology and Neurosciences, Duke University Medical CenterPhilip D. Harvey, PhD – Leonard M. Miller Professor of Psychiatry, University of Miami Miller School of Medicine Katy Benjamin, PhD – Director, HEOR, Patient Centered Outcomes, AbbVie Inc. Panelist: Billy Dunn, MD – Director, Division of Neurology Products, OND, CDER, FDA Q & A |
|
2:45–3:10 pm |
Break – 25 min |
|
3:10–4:40 pm |
Session 4: Endpoint Construction from Activity Monitor Data: Chronic Heart Failure
Moderator: Maria Mattera, MPH – Assistant Director, PRO Consortium, C-Path Presenters:Chad Gwaltney, PhD – President, Gwaltney ConsultingMaria Mattera, MPH – Assistant Director, PRO Consortium, C-Path Jeremiah (Jay) Trudeau, PhD – Director, Patient-Reported Outcomes, Janssen Global Services Wayne Amchin, RAC, MIA, MPA – Senior Consumer Safety Officer, Division of Cardiovascular and Renal Products, OND, CDER, FDA Bill Byrom, PhD – Vice President of Product Strategy and Innovation, CRF Bracket Panelist: Ebony Dashiell-Aje, PhD – Reviewer, COA Staff, OND, CDER, FDA Q & A |
|
4:40–5:00 pm |
Day 1 Closing Remarks – Linda Nelsen and Sonya Eremenco Adjourn |
|
5:00–5:30 pm |
Open |
|
5:30–7:00 pm |
Reception and Poster Session – Magnolia Ballroom
Asthma Working Group |
Agenda – Day 2
|
7:30–8:30 am |
Registration and Continental Breakfast – Cypress Ballroom |
| Day 2 Moderator: Sonya Eremenco, MA – Associate Director, PRO Consortium and Acting Director, Electronic Patient-Reported Outcome (ePRO) Consortium, C-Path | |
|
8:30–10:00 am |
Session 5: eCOA – Continuing to Get Better Together
Moderator: Sonya Eremenco, MA – Associate Director, PRO Consortium and Acting Director, ePRO Consortium, C-Path Presenters:Katherine Zarzar – Senior Manager, Patient-Centered Outcomes Research, Genentech, A Member of the Roche Group Paul O’Donohoe, MSc – Scientific Lead, eCOA and Mobile Health, Medidata Solutions and Vice Director, ePRO Consortium Alexandra (Alex) Barsdorf, PhD – Director, Clinical Outcome Assessments, Clinical Outcomes Solutions Megan Turner – Scientist, COA Implementation, Value Evidence and Outcomes, GlaxoSmithKline Patricia (Trish) Shepherd Delong, MS – Manager, Patient-Reported Outcomes, Global Commercial Strategy Organization (GCSO), Janssen Andres Escallon, DM – Director, eCOA Clinical Data Management, ERT Panelist: David Reasner, PhD – Vice President, Data Science and Head, Study Endpoints, Ironwood Pharmaceuticals Q & A |
|
10:00–10:25 am |
Break – 25 min |
|
10:25–11:55 am |
Session 6: Using Patient Input to Estimate Clinically Meaningful Within-Patient Change at the Scale Score Level
Moderator: Lori McLeod, PhD – Vice President, Patient-Centered Outcomes Assessment, RTI Health Solutions Presenters:Jean Paty PhD – Vice President and Head of Patient Centered Endpoints, IQVIAKate Sully, PhD – Senior Research Manager, Patient-Centered Outcomes, Adelphi Values Kelly McCarrier, PhD, MPH – Director and Qualitative Research Lead, Pharmerit International Cheryl D. Coon, PhD – Principal, Outcometrix Panelists: Scott Komo, DrPH – Team Leader, Division of Biometrics III, Office of Biostatistics, OTS, CDER, FDA Michelle Campbell, PhD – Senior Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, DNP, OND, CDER, FDA Q & A |
|
11:55 am–12:15 pm |
Closing Remarks – Stephen Coons
Adjourn |
