ANNUAL RARE DISEASE SCIENTIFIC WORKSHOP
Improving the Clinical Development Process
Workshop 7: "Incorporating the Patient Perspective in Rare Disease Drug Development"
Hosted By:
EveryLife Foundation for Rare Diseases
Willard Hotel Washington, D.C.
Tuesday, September 15, 2015
AGENDA
Workshop Overall Goal: Create a usable, scientifically rigorous, framework for industry to
incorporate patient input in rare disease drug development in a quantitative and objective way that is
acceptable to the FDA
7:45 AM
Breakfast & Registration
8:30 AM
Welcome, Creating a Structured Process for Patient Engagement
Emil Kakkis, MD, Ph.D., President & Founder, EveryLife Foundation for Rare
Diseases, President & CEO, Ultragenyx
Session A: Overview of Policies and Practices
8:45 AM
Rare Disease Drug Development & Patient Perspective Initiatives at FDA
Jonathan C. Goldsmith, MD, FACP, Associate Director, Rare Diseases Program,
OND/CDER, Food and Drug Administration
9:00 AM
Patient-Focused Drug Development: Advancing the Science of Patient Input
Sara Eggers, Ph.D., Office of Program and Strategic Analysis/Office of Strategic
Programs, CDER, Food and Drug Administration
9:15 AM
The Role of Patient Advocates in NCATS and the Rare Disease Clinical
Research Network
Philip J. (PJ) Brooks, Ph.D., Division of Clinical Innovation, National Center for
Advancing Translational Sciences (NCATS), National Institutes of Health
9:35 AM
From Passengers to Co-Pilots
Kimberly McCleary, Managing Director, FasterCures
9:55 AM
Benefit-Risk Preference Methods: What Makes Rare Disease Unique
Brett Hauber, Ph.D., Senior Economist and VP, Health Preference Assessment,
RTI Health Solutions
10:15 AM
Questions & Discussion
10:35 AM
Break (15 Minutes)
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September 15, 2015
EveryLife Foundation Annual Scientific Workshop
Session B: Best Practices
Session B 1: Patient Organizations Leading the Way
10:50 AM
Patient Focused Drug Development 2.0
Pat Furlong, President and CEO, Parent Project Muscular Dystrophy
11:10 AM
Integrating the Patient Experience into Lung Cancer Policy, Treatment
and Research
Andrea Ferris, President, Lungevity Foundation
11:30 AM
Changing the Policy Landscape to Achieve a Patient-Driven Ecosystem
Marc Boutin, Chief Executive Officer, National Health Council
11:50 PM
Patient First Drug Development from Pre-Clinical to Post-Market
Sally Okun, Vice President of Advocacy, Policy & Patient Safety, PatientsLikeMe
12:10 PM
Questions & Discussion
12:30 PM
Lunch (45 Minutes)
Session B 2: Examples from Large and Small Drug Sponsors
1:15 PM
Collaborating with Patients: Learning from Psoriasis and Other Diseases
Marilyn Metcalf, Ph.D., Senior Director, Benefit Risk Evaluation, Chief Medical
Office, GlaxoSmithKline
1:35 PM
Value of Patient Input to Inform Endpoint Development: Examples from
GNE Myopathy and MPS 7
Alexandra Bowden Ph.D., Clinical Outcomes Research and Evaluation,
Ultragenyx Pharmaceutical Inc.
1:50 PM
Development of a Patient Survey to Inform the Design of Clinical Trials for
Children and Adults with XLH
Alison Skrinar, Ph.D., Senior Director, Clinical Outcomes Research and
Evaluation, Ultragenyx Pharmaceutical, Inc.
2:05 PM
Partnership with Breast Cancer Patients to Quantify the True Burden of
Disease and Improve Patient-Outcome Measures
Jennifer A. Petersen, MPH, Principal Scientist - Patient-Centered Outcomes
Research, Genentech, A Member of the Roche Group
2:25 PM
Questions & Discussion
2:45 PM
Break (15 Minutes)
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September 15, 2015
EveryLife Foundation Annual Scientific Workshop
Session C: Sample Frameworks & Implementation
3:00 PM
A Framework for Incorporating Information on Patient Preferences
Regarding Benefit and Risk into Regulatory Assessments of New Medical
Technology
Stephanie Christopher, Program Manager, Patient Centered Benefit Risk
Assessment and Clinical Trials Innovation & Reform,
Medical Device Innovation Consortium (MDIC)
3:20 PM
A Lifecycle Approach to FDA’s Structured Benefit-Risk Assessment
Framework
Cartier Esham, Ph.D., Executive Vice President, Emerging Companies,
Biotechnology Industry Organization
3:40 PM
Developing Therapies for Patients with Patients- Shifting the Paradigm of
Innovation
Roslyn F. Schneider, MD, MSc, Global Patient Affairs Lead, Pfizer Inc.
4:00 PM
Operationalizing Patient Engagement in Research
Suzanne Schrandt, Deputy Director of Patient Engagement
Patient-Centered Outcomes Research Institute (PCORI)
4:20 PM
Questions & Discussion
4:40 PM
Summary, Future Directions and Closing Remarks
Emil Kakkis, MD, Ph.D., President & Founder, EveryLife Foundation for Rare
Diseases, President & CEO, Ultragenyx
4:55 PM
END CONFERENCE
Thank you to our Sponsors:
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