Statistical Considerations for the Design of Rare Disease Clinical Investigations; Establishment of a Public Docket; Request for Information and Comments
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Document details are published in the U.S. Federal Register; SPEQ surfaces them for reference and is not affiliated with or endorsed by the FDA or the Office of the Federal Register. The authoritative text is on federalregister.gov.
The Food and Drug Administration (FDA, the Agency, or we) is establishing a public docket to collect feedback on statistical considerations for rare disease clinical investigations. This docket is open in conjunction with a Rare disease Innovation, Science, and Exploration (RISE) Workshop on the same topic. Feedback is welcome both on the attached pre-read documents and on the content covered in the Workshop itself.
Abstract as published in the Federal Register.
The comment window is still open until November 13, 2026. The comment window is open. Check the document itself for what comments are being solicited on and what weight they carry. Submit comments through the Federal Register document below.
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