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.

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 a significant challenge with a substantial stumbling block represented by the considerable complexity and variety of clinical manifestations in rare diseases. The Food and Drug Administration (FDA) guidelines highlight the benefits of orphan drug market approval (2). Today, the surrogate endpoints adoption in orphan drug trials overcomes some of the burdens and challenges, minimize trial costs, reduce the number of subjects in clinical trials and the study duration. Thus, surrogate endpoints play a crucial role in orphan drug development (3).

Download the White Paper

Specializing in rare disease, Boston Biotech Clinical Research works with biotech, pharmaceutical, device companies and investors to streamline the clinical trial process. Our experienced team helps each client reach their specific goals by customizing a clinical and regulatory road map of simplified programs and streamlined protocols to meet our clients’ requirements.

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