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)

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

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

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

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

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