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Meaningful Patient-Focused Drug Development for Rare Disease and Personalized Medicine
Session Chair(s)
Michelle Campbell, PhD
Associate Director, Stakeholder Engagement and Clinical Outcomes, ON, OND, CDER
FDA, United States
This session will provide a platform to discuss measurement challenges and practical considerations for selection of fit-for-purpose clinical outcome assessments (COAs) to support endpoints in rare disease drug development programs.
Learning Objective : 1. Review commonly used metrics to assess treatment benefit, especially considering survival endpoints. 2. Increase awareness and understanding of a novel statistical approach that has shed new light in the assessment of treatment benefit. 3. Describe ongoing efforts to enhance this methodology and make it available to end-users, such as clinical trialists, physicians, and patients.
Speaker(s)
Generalized Pairwise Comparisons for Benefit/Risk Assessment in Personalized Medicine
Marc E. Buyse, DrSc
IDDI, United States
Chief Scientific Officer
Endpoint Selection and Use of Clinical Outcome Assessments (COAs) in Rare Disease Drug Development: A Regulatory Perspective
Lili Garrard, PhD
FDA, United States
Mathematical Statistician, Office of Biostatistics, OTS, CDER
Selection of Fit-for-Purpose COAs to Support Endpoints in Rare Disease
Jean Paty, PhD, MS
IQVIA, United States
Vice President, Patient-Centered Science
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