View Now: 12th Annual Patient-Reported Outcome Consortium Workshop – Held Virtually

April 14–15, 2021

On April 14–15, 2021, the 12th Annual Patient-Reported Outcome Consortium Workshop was held in a virtual format.

The following Workshop Agenda provides an overview of the two-day meeting as well as links to the session recordings and slide decks.  Links to posters summarizing the status of the PRO Consortium’s working groups, the Rare Disease Subcommittee activities, and the ePRO Consortium are located after the agenda.

Agenda – Day 1

11:00–11:20 am Welcome and Patient-Reported Outcome Consortium Update

Overview:  Provides a high-level summary of the recent accomplishments and ongoing activities within the Patient-Reported Outcome (PRO) Consortium

Presenter:

Sonya Eremenco, MA – Director, Patient-Reported Outcome (PRO) Consortium, Critical Path Institute (C-Path)

11:20–12:30 pm Session 1: 2021 FDA Update

Overview:  Provides an update on FDA’s Clinical Outcome Assessment (COA) Qualification Program and other initiatives

Moderator:

Michelle Campbell, PhD – Senior Clinical Analyst for Stakeholder Engagement and Clinical Outcomes, Office of Neuroscience (ON), Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA)

Presenters:

Robyn Bent, RN, MS – Director, Patient Focused Drug Development Program, CDER, FDA

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

Elektra Papadopoulos, MD, MPH – Acting Deputy Director, Division of Clinical Outcome Assessment, Office of Drug Evaluation Sciences (ODES), OND, CDER, FDA

David S. Reasner, PhD – Division Director,  Division of Clinical Outcome Assessment, ODES, OND, CDER, FDA

Q & A

12:30–12:50 pm Break – 20 min  
12:50–2:20 pm Session 2:  Migraine:  A Tale of Two Paths to PRO-Based Product Labeling

Overview:  Discusses the experience of two pharmaceutical firms’ different paths to obtaining FDA-approved PRO-based label claims for novel migraine drugs

Moderator:  

Stephen Joel Coons, PhD – Executive Director, PRO Consortium and Senior Vice President, Clinical Outcome Assessment Program, C-Path

Opening Remarks: 

Billy Dunn, MD – Director, ON, CDER, FDA

Presenters:

Pooja Desai, PhD – Director, US Health Economics, Therapeutic Area Lead – Inflammation, Nephrology and Bone, Amgen

Elizabeth (Nicki) Bush, MHS – Senior Advisor and Head, Patient-Focused Outcomes Center of Expertise, Eli Lilly and Company

Panelists:

Eric Bastings, MD – Acting Director, Division of Neurology I; Deputy Director, ON, CDER, FDA

Nick Kozauer, MD – Director, Division of Neurology II, ON, CDER, FDA

Elektra Papadopoulos, MD, MPH – Acting Deputy Director, Division of Clinical Outcome Assessment, ODES, OND, CDER, FDA

Q & A

2:20–2:30 pm Day 1 Wrap Up  

Agenda – Day 2

11:00–11:20 am eCOA:  Getting Better Together Initiative Update   

Overview:  Provides a brief update on the eCOA:  Getting Better Together Initiative, an ongoing collaboration between the PRO Consortium and ePRO Consortium

Presenter: 

Sonya Eremenco, MA –Director, PRO Consortium and Acting Director, Electronic Patient-Reported Outcome (ePRO) Consortium, C-Path

11:20–12:50 pm Session 3:  Where Are We Headed with Activity Monitors in Clinical Trials?

Overview:  Discusses practical considerations associated with the use of activity monitors to measure efficacy endpoints in clinical trials

Moderator:  

Maria Mattera, MPH – Assistant Director, PRO Consortium, C-Path

Presenters:

Jennifer Goldsack, MChem, MA, MBA – Executive Director, Digital Medicine Society

Bill Byrom, PhD – Vice President, Product Intelligence and Positioning, Signant Health

Jiat Ling Poon, PhD – Principal Research Scientist, Eli Lilly and Company

Milena Anatchkova, PhD – Senior Research Leader, Evidera, Inc.

Panelists:

Andrew Potter, PhD – Mathematical Statistician, Division of Biometrics I, CDER, FDA

Steven Blum, MBA, MA – Asset and Indication Lead, Patient Reported Outcomes Assessment, WWHEOR, Bristol Myers Squibb

Q & A

12:50–1:10 pm Break – 20 min  
1:10–2:40 pm Session 4:  Identifying COAs for Use in Rare Disease Treatment Trials

Overview:  Provides an update regarding the establishment and goals of the Rare Disease COA Consortium and presents results of work accomplished by the PRO Consortium’s Rare Disease Subcommittee

Moderator:

Lindsey Murray, PhD, MPH – Associate Director, PRO Consortium, C-Path

Presenters:

Tori Brooks, MPH – Research Associate II, Mapi Research Trust

Lindsey Murray, PhD, MPH – Associate Director, PRO Consortium, C-Path

Kiera Berggren, MA/CCC-SLP, MS – Research Speech-Language Pathologist, Department of Neurology, Virginia Commonwealth University

Panelists:

Naomi Knoble, PhD – Reviewer, Division of Clinical Outcome Assessment, OND, CDER, FDA

Dawn Phillips, PT, MS, PhD – Director, Clinical Scientist, Outcomes Research, REGENXBIO Inc.

Adam Shaywitz, MD, PhD – Chief Medical Officer, BridgeBio Gene Therapy

Allison Seebald – Senior Research Program Manager, National Organization for Rare Disorders

Q & A

2:40–2:45 pm Workshop Wrap UpSonya Eremenco

Posters summarizing the status of the PRO Consortium’s working groups, the Rare Disease Subcommittee activities, and the ePRO Consortium are available below:

Posters

Chronic Heart Failure Working Group
Cognition Working Group
Depression Working Group 2.0
Functional Dyspepsia Working Group
Irritable Bowel Syndrome Working Group
Multiple Sclerosis Working Group
Pediatric Asthma Working Group
Rheumatoid Arthritis Working Group
Small Cell Lung Cancer Working Group
Rare Disease Subcommittee
ePRO Consortium
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