Sixth Annual
Patient-Reported Outcome Consortium Workshop

Partners in Progress: Sharing the Vision,

Shaping the Future


April 29 – 30, 2015

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

 

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, 2015 the SIXTH ANNUAL PATIENT-REPORTED OUTCOME CONSORTIUM WORKSHOP was held in Silver Spring, Maryland. The overall Workshop objectives were to:

  • Discuss how all stakeholders can work together to improve patient-focused drug development
  • Provide updates on the PRO instrument development and qualification activities within the PRO Consortium’s working groups
  • Describe the qualitative and quantitative research that led to development of the Asthma Daily Symptom Diary (ADSD) within the Asthma Working Group
  • Discuss the development and implementation of well-defined and reliable clinical outcome assessment (COA) tools for pediatric clinical trials
  • Discuss ways to optimize qualitative and quantitative research to more efficiently generate evidence supporting the content validity of new PRO instruments

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, 2015

7:30-8:30 am

Registration and Continental Breakfast – Cypress  Ballroom

Day 1 Morning Moderator:  Ashley F. Slagle, MS, PhD Clinical Outcome Assessment (COA) Qualification Scientific Coordinator and Endpoint Reviewer, Study Endpoints and Labeling Development (SEALD), Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA)

 

8:30-8:45 am

Welcome and Patient-Reported Outcome Consortium Update
Stephen Joel Coons, PhD — Executive Director, Patient-Reported Outcome (PRO) Consortium, Critical Path Institute (C-Path)

8:45-9:45 am

Session 1: Stakeholder Collaboration to Improve Patient-Centered Drug Development

Moderator: Ashley F. Slagle, MS, PhD – COA Qualification Scientific Coordinator and Endpoint Reviewer, SEALD, OND, CDER, FDA

Presenters and Panelists:

Janet W. Maynard, MD, MHS – Clinical Team Leader, Division of Pulmonary, Allergy, and Rheumatology Products (DPARP), OND, CDER, FDA

Elektra Papadopoulos, MD, MPH – Acting Associate Director, Study Endpoints Team, SEALD, OND, CDER, FDA

Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium

Cynthia A. Bens
– Vice President, Public Policy, Alliance for Aging Research

9:45-10:10 am

Session 2: COA Qualification and Study Endpoints Update

Presenter:

Ashley F. Slagle, MS, PhD   – COA Qualification Scientific Coordinator and Endpoint Reviewer, SEALD, OND, CDER, FDA

 

 

10:10-10:30 am

Break – 20 min 

10:30-11:15 am

Session 3  – The Asthma Working Group: On the Path to Success

Moderator:  Josephine M. Norquist, MS  Patient-Reported Outcomes Specialist, Merck Sharp & Dohme, Corp

Presenters and Panelists: 

Linda Nelsen, MHS – Director, Patient Reported Outcomes, GlaxoSmithKline

Adam Gater, MSc – Director, Endpoint Development and Outcomes Assessment, Adelphi Values

Elektra Papadopoulos, MD, MPH  – Acting Associate Director, Study Endpoints Team, SEALD, OND, CDER, FDA

Q & A

 

11:15-12:15 pm

Session 4: Patient-Centric Endpoints in Oncology 

Moderator:  Katarina Halling, MSc    Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium

Presenters and Panelists: 

Cindy Geoghegan –  Patient Advocate and Principal, Patient and Partners LLC

Paul G. Kluetz, MD – Acting Deputy Director, Office of Hematology and Oncology Products (OHOP), OND, CDER, FDA

Ethan Basch, MD, MSc – Director, Cancer Outcomes Research Program, University of North Carolina at Chapel Hill

Q & A

 

12:15 – 1:15 pm

Lunch – Elm I, Elm II and Magnolia Ballroom

 

Day 1 Afternoon Moderator: Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium

1:15 – 2:15 pm

Session 5: Optimizing Qualitative and Quantitative Research: How to Make the Process More Efficient

Moderator: J. Jason Lundy, PhD – Principal, Outcometrix

Presenters and Panelists:

J. Jason Lundy, PhD – Principal, Outcometrix

Stacie Hudgens, MA (AbD) – Strategic Lead, Quantitative Science, Clinical Outcomes Solutions

R.J. Wirth, PhD – Managing Partner, Vector Psychometric Group, LLC

Wen-Hung Chen, PhD – Reviewer, Study Endpoints, SEALD, OND, CDER, FDA

Q & A

2:15 – 3:15 pm

Session 6: Interpreting Change in Scores on COA Endpoint Measures

Moderator: Cheryl D. Coon, PhD – Director, Healthcare Analytics, Adelphi Values

Presenters and Panelists:

Joseph C. Cappelleri, PhD, MPH, MS – Senior Director of Biostatistics, Pfizer Inc.

Cheryl D. Coon, PhD – Director, Healthcare Analytics, Adelphi Values

Scott Komo, DrPH – Senior Statistical Reviewer, Office of Biostatistics, CDER, FDA

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

Q & A

 

3:15 – 3:45 pm

Break – 30 min

3:45 – 4:45 pm

Session 7: Thinking with the End in Mind: From COA Instrument to Endpoint

Moderator: Jean Paty, PhD – Principal Advisory Services, Quintiles

Presenters and Panelists:

Paul G. Kluetz, MD – Acting Deputy Director, OHOP, OND, CDER, FDA

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

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

Elisabeth (Liz) Piault-Louis, PharmD, MA – Principal Outcomes Research Scientist, Oncology Genentech, a member of the Roche Group

Q & A

 

4:45 – 5:15 pm

Day 1 Closing Remarks, Day 2 Preview
Adjourn

 

5:30 – 7:00 pm

Reception and Poster Session – Magnolia Ballroom

Asthma
Cognition
Depression
Functional Dyspepsia
Irritable Bowel Syndrome (IBS)
Non-Small Cell Lung Cancer (NSCLC)

 

 

Workshop Agenda – Day 2
April 30, 2015

7:30-8:30 am

Registration and Continental Breakfast – Cypress Ballroom

 

8:30 – 9:00 am

Session 8: Results from “Literature Review to Determine Empirical Basis for Response Scale Selection in Patient-Reported Outcome Instrument Development”

Presenter:
Katharine S. Gries, PharmD, PhD – Senior Research Associate, Evidera

Q & A

9:00 – 10:00 am

Session 9: Practical Considerations in Implementing a Pediatric COA Measurement Strategy: A Case Study in Functional Constipation

Moderator: Sarrit Kovacs, PhD – Study Endpoints Reviewer, SEALD, OND, CDER, FDA

Presenters and Panelists:

Andrew E. Mulberg, MD, FAAP, CPI – Deputy Director, Division Gastroenterology and Inborn Errors Products (DGIEP), OND, CDER, FDA

Diane Turner-Bowker, PhD – Engagement Leader I, Quintiles

Gina Calarco (Smith), MPH, RN, CCRC – Associate Director and Deputy Head of the Pediatric Center of Excellence, Quintiles

Jean Paty, PhD – Principal Advisory Services, Quintiles

Q & A

10:00 – 10:30am

Break – 30 min

 

10:30 – 11:30 am

Session 10: ePRO Science and Innovation: BYOD Approaches and Equivalence Across Administration Modalities

Moderator: Susan Vallow, RPh, MBA, MA – Senior Director, Patient Focused Outcomes, GlaxoSmithKline

Presenters and Panelists:

Willie Muehlhausen, DVM – Vice President, eCOA and Innovation, ICON plc

Chad Gwaltney, PhD – Chief Scientist and Regulatory Advisor, Endpoints, ERT

Virginia (Gini) Kwitkowski, MS, RN, ACNP-BC – Clinical Team Leader and Associate Director for Labeling, Division of Hematology Products, OHOP, OND, CDER, FDA

Cindy Howry, MS – Vice President, Product Strategy and Innovation, YPrime and Vice Director, ePRO Consortium

Sheila Rocchio, MBA – Vice President, Marketing and Strategy, PHT

Q & A

11:30 – 12:15 pm

Session 11: The Relevance of Patient-Reported Endpoints to Payers and Regulators: Is there Common Ground?

Moderator: Peter C. Trask, PhD, MPH – Principal Scientist, Patient Centered Outcomes Research, Genentech

Presenters and Panelists:

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

Vasudha Bal, MSc, MBA – Director, Patient Reported Outcomes, Novartis Pharmaceuticals Corporation

Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium

Selena R. Daniels, PharmD, MS – Study Endpoints Reviewer, SEALD, OND, CDER, FDA

Alan L. Shields, PhD – Vice President, Endpoint Development and Outcomes Assessment, Adelphi Values

Robin S. Turpin, PhD – Director and Head, HEOR, U.S. Medical and Scientific Affairs, Takeda Pharmaceuticals, USA

Q & A

12:15-12:30 pm

Closing Remarks

 

12:30 pm

Adjourn

 
Copies of the Workshop’s slide presentations will be available on the Critical Path Institute’s website (https://www.c-path.org/PRO.cfm) after May 30, 2015.