FDA Grand Rounds - Statistical Considerations for Drug Development in Rare Disease

FDA Center for Drug Evaluation and Research — Office of Biostatistics Grand Rounds · February 5, 2026

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Opening and speaker introduction

Presentation

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Rakesh Raghuanshi (Office of the Chief Scientist) opened FDA Grand Rounds and introduced the presenter, naming 'Doctor Emily Morris' from the Office of Biostatistics as today's speaker and noting no reported conflicts. He turned the floor to the presenter.

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Natalie Morris, a statistician in the FDA Center for Drug Evaluation and Research Office of Biostatistics, presented methods and simulation results for adaptive trial designs in rare-disease drug development, highlighting adaptive treatment-duration designs as a widely applicable approach and urging simulations and prespecification to preserve trial integrity.

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Transcript

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