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Clinical Outcomes Assessments as Study Endpoints in Rare Diseases
Session Chair(s)
Susan Martin
Head, Patient Reported Outcomes
RTI Health Solutions, United States
Selecting and identifying study endpoints in Rare Disease (RD) clinical trials can present challenges to drug developers. In many RDs there is a lack of precedence and even a void of existing instruments for evaluating treatment benefit in the context of a high unmet medical need for a small patient population. As a result, standard methods for study endpoints development and use must be adapted in such a manner that preserves scientific best practices, e.g. of the FDA PRO Guidance, yet accommodates the special circumstances of RD. The following sessions will address these challenges and offer suggestions to be discussed by a panel of drug development specialists from Industry, FDA and patient advocacy groups.
Program Committee Representative: Linda Deal, Pfizer
Speaker(s)
1:00-1:05PM Welcome/Overview
Susan Martin
RTI Health Solutions, United States
Head, Patient Reported Outcomes
1:05-1:25PM Commercial Considerations for Study Endpoints in RD
Harris Kaplan, MBA
Red Team Associates, Div of Healogix, United States
Managing Partner
1:25-1:45PM Clinical Challenges and Opportunities in Study Endpoint Development for RD
Annabel Barbier, MD, PhD
Agios Pharmaceuticals Inc, United States
Vice President, Clinical Development Rare Genetic Diseases
1:45-2:10PM Regulatory Considerations for Clinical Outcome Assessment Selection in RD
Elektra Papadopoulos, MD, MPH
AbbVie, United States
Director, Patient Experience Data and Strategy
2:10PM-2:25PM Networking Break
All Participants
United States
2:25-2:50PM RD Endpoint Strategy and Implementation
Linda S. Deal, MS
Pfizer Inc., United States
Head of Patient-Centered Outcomes Measurement
2:50-3:10PM Working with Patient Advocacy Organizations to Implement a RD Strategy
Marianne Clancy, MPA
Cure HHT, United States
Executive Director
3:10-3:45PM RD Endpoint Evaluation, Analysis and Interpretation
Lori McLeod, PhD
RTI Health Solutions, United States
Head, Psychometrics
3:45-4:20PM Panel Discussion: All Session Speakers and Additional Panelist
Lawrence Charnas, MD, PhD
Novartis Institutes For Biomedical Research, United States
Senior Translational Medicine Expert Musculoskeletal Diseases
4:20-4:30PM Wrap-up/Session Adjourned
Susan Martin
RTI Health Solutions, United States
Head, Patient Reported Outcomes
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