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Tagged: Surrogate Endpoints

There remains continued interest in the use of surrogate endpoints as primary measures of the effectiveness of investigational drugs in definitive drug trials. BBCR’s published White Paper – Surrogate Endpoints for an Accelerated Orphan Drug Approval – remains a valuable resource in the area of orphan drug development.

September 20th, 2023 | Surrogate Endpoints

Rare Diseases are conditions that affect less than 200,000 people in the U.S. However, over 7,000 Rare Diseases affect more than 400,000,000 people worldwide, including ~25 million in the U.S. and ~30 million in Europe (1). In recent years, the number of orphan drugs approved has increased significantly. However, developing and marketing orphan drugs remains […]

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