Ninth Annual Patient-Reported Outcome Consortium Workshop

April 25 – 26, 2018

Sheraton Silver Spring Hotel
8777 Georgia Avenue
Silver Spring, MD 20910

On April 25-26, 2018 the NINTH 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 address changes associated with the 21st Century Cures Act and PDUFA VI;
  • Describe the development of the three versions of the Diary of Irritable Bowel Syndrome Symptoms (DIBSS);
  • Discuss ways in which clinical trial sponsors and eCOA system providers can work collaboratively to optimize electronic COA data collection in trials;                    
  • Describe results of projects aimed at advancing the science of clinical trial data collection by leveraging available and emerging technologies;
  • Provide multiple stakeholders’ perspectives regarding the challenges and opportunities associated with the application of existing PRO measures in drug development; and
  • Discuss some emerging approaches to outcome assessment in rare diseases and pediatric populations.

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 – Reviewer and Scientific Coordinator, Clinical Outcome Assessments (COA) Qualification Program, COA Staff, 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 UpdateStephen Joel Coons, PhD – Executive Director, Patient-Reported Outcome (PRO) Consortium, Critical Path Institute (C-Path)

8:50–10:20 am

Session 1: Update from FDA Regarding the Clinical Outcome Assessment Qualification Program Moderator: Michelle Campbell, PhD – Reviewer and Scientific Coordinator, COA Qualification Program, COA Staff, OND, CDER, FDA

Presenter:

Elektra Papadopoulos, MD, MPH – Associate Director, COA Staff, OND, CDER, FDA

Panelists:

Laura Lee Johnson, PhD – Acting Director, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences, CDER, FDA

Theresa Mullin, PhD – Associate Director for Strategic Initiatives, CDER, FDA

Q & A

10:20–10:45 am

Break – 25 min

10:45 am–12:15 pm

Session 2: Case Study: The Diary of Irritable Bowel Syndrome Symptoms (DIBSS) Moderator: Jennifer Hanlon, MPH – Associate Director, Study Endpoints, Ironwood Pharmaceuticals

Presenters:

Claire Ervin, MPH – Senior Director, Patient-Centered Outcomes Assessment, RTI Health Solutions

Lori McLeod, PhD – Vice President, Patient-Centered Outcomes Assessment, RTI Health Solutions

Adam Butler – Senior Vice President, Strategic Development, Bracket

Robyn Carson, MPH – Executive Director and Head, Patient-Centered Outcomes Research, Allergan

Panelists:

Stephen Joel Coons, PhD – Executive Director, PRO Consortium, C-Path

Sheri Fehnel, PhD – Vice President, Patient-Centered Outcomes Assessment, RTI Health Solutions

Sarrit Kovacs, PhD – Reviewer, COA Qualification Program, COA Staff, OND, CDER, FDA Q & A

Q & A

12:15–1:15 pm

Lunch – Cedar, Walnut, Persimmon I and Persimmon II Rooms (First Floor)
Day 1 Afternoon Moderator: Elizabeth (Nicki) Bush, MHS – Director, Patient-Focused Outcomes Center of Expertise, Eli Lilly and Company and Industry Co-Director, PRO Consortium

1:15–2:45 pm

Session 3: eCOA: How Do We Get Better Together? Moderator: Jean Paty, PhD – Vice President, Consulting Services, Leading Patient Centered Endpoints Activities, QuintilesIMS

Presenters:

Emily Nash Smyth, PharmD – Senior Research Scientist, Global Patient Outcomes and Real World Evidence, Early Phase Oncology, Eli Lilly and Company

Paul O’Donohoe, MSc – Scientific Lead, eCOA and Mobile Health, Medidata Solutions

Kristina Lowe, BS – Vice President, Business Development, ERT

Katie Zarzar – Senior Manager, Patient-Centered Outcomes Research, Genentech, A Member of the Roche Group

Panelists:

Robyn Carson, MPH – Executive Director and Head, Patient-Centered Outcomes Research, Allergan

Katarina Halling, MSc – Global Head Patient Reported Outcomes, AstraZeneca

Sean Stanton – Chief Executive Officer, Lifecore Solutions

Q & A

2:45–3:10 pm

Break – 25 min

3:10–4:40 pm

Session 4: Advancing the Science of Clinical Trial Data Collection• EQ-5D-5L Study Results

• BYOD Study Results

• IMI PROactive Project Overview

Moderator: Sonya Eremenco, MA – Associate Director, PRO Consortium, C-Path

Presenters:

Jason Lundy, PhD – Principal, Outcometrix

Louise Newton, MSc – Senior Director, Clinical Outcome Assessments, Clinical Outcome Solutions

Niklas Karlsson, PhD – Patient Reported Outcomes Director Respiratory, AstraZeneca

Panelists:

Bill Byrom, PhD – Vice President, Product Strategy and Innovation, CRF Health and Vice Director, ePRO Consortium

Wen-Hung Chen, PhD – Team Leader, COA Staff, OND, CDER, FDA

David Reasner, PhD – Vice President, Data Science and Head, Study Endpoints, Ironwood Pharmaceuticals

Q & A

4:50–4:25 pm

An Overview and Discussion with Members of the Friends of Cancer Research Working Group: Comparative Tolerability Trial Design Alicyn Campbell, MPH – Global Head, Patient Centered Outcomes Research for Oncology, Genentech, A Member of the Roche Group

Lee Jones, MBA – Patient/Research Advocate, Fight Colorectal Cancer, SWOG, Cancer Action Coalition of VA, Cancer Policy and Advocacy Team, Clinical Trials Advisory Panel. Georgetown University Oncology Institutional Review Board

Paul G. Kluetz, MD – Associate Director of Patient Outcomes (Acting), Oncology Center of Excellence, FDA

Mark Stewart, PhD – Senior Science Policy Analyst, Friends of Cancer Research

Q & A

5:25–5:30 pm

Day 1 Closing Remarks
Adjourn

5:30–7:00 pm

Reception and Poster Session – Cedar Room (First Floor)
Asthma Working Group
Cognition Working Group
Depression Working Group
Electronic Patient-Reported Outcome (ePRO) Consortium
Functional Dyspepsia Working Group
Irritable Bowel Syndrome (IBS) Working Group
Multiple Sclerosis Working Group
Myelofibrosis Working Group
Non-Small Cell Lung Cancer (NSCLC) 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: Maria Mattera, MPH – Assistant Director, PRO Consortium, C-Path

8:30 – 10:00 am

Session 5: Why Reinvent the Wheel? Moderator: Maria Mattera, MPH – Assistant Director, PRO Consortium, C-Path

Presenters:

Elizabeth (Nicki) Bush, MHS – Director, Patient-Focused Outcomes Center of Expertise, Eli Lilly and Company and Industry Co-Director, PRO Consortium

Elektra Papadopoulos, MD, MPH – Associate Director, COA Staff, OND, CDER, FDA

Dave Cella, PhD – Professor and Chair, Department of Medical Social Sciences, Feinberg School of Medicine, Northwestern University

Sonya Eremenco, MA – Associate Director, PRO Consortium, C-Path

Panelist:

Billy Dunn, MD – Director, Division of Neurology Products, OND, CDER, FDA

Q & A

10:00–10:25 am

Break – 25 min

10:25–11:55 am

Session 6: Overcoming Challenges in Outcome Measurement in Rare Diseases and Pediatric Populations Moderator: Michelle Campbell, PhD – Reviewer and Scientific Coordinator, COA Qualification Program, COA Staff, OND, CDER, FDA

Presenters:

Nerissa Kreher, MD, MS, MBA – Chief Medical Officer, AVROBIO, Inc.

Bryce Reeve, PhD – Professor and Director of Center for Health Measurement, Duke University School of Medicine

Ebony Dashiell-Aje, PhD – Reviewer, COA Staff, OND, CDER, FDA

Panelist:

Ron Bartek, MA, BS – Co-Founder/Founding President, Friedreich’s Ataxia Research Alliance (FARA)

Q & A

11:55–12:15 pm

Closing Remarks
Adjourn

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