Tenth Annual Patient-Reported Outcome Consortium Workshop

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
Chronic Heart Failure Working Group
Cognition Working Group
Depression Working Group
Functional Dyspepsia Working Group
Electronic Patient-Reported Outcome Consortium
Irritable Bowel Syndrome Working Group
Multiple Sclerosis Working Group
Pediatric Asthma Working Group
Rheumatoid Arthritis 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

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