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Abstract:
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Developing therapeutic products for very rare diseases presents enormous challenges in clinical trial design and analysis. In order for a drug or biologic product to be approved in the United States, it needs to be shown to be safe and effective. In this talk, I will present case examples of rare disease products reviewed by the Center for Biologics Evaluation & Research at the U.S. Food and Drug Administration. I will focus on design features that facilitated determinations of safety and effectiveness based on very small clinical trials, sometimes involving as few as a dozen subjects. Finally, I will discuss some Bayesian and adaptive approaches designed specifically to assess the safety of products for small populations.
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