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 Update Stephen 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 |
