CDE issued “Guidelines on Clinical Trial Statistics of Drug Products for Rare Diseases (Interim)”

In order to encourage the development of drugs for rare diseases and guide applicants to improve R&D efficiency from the perspective of clinical research methodology, the Drug Evaluation Center organized the formulation of the "Statistical Guidelines for Clinical Research of Drugs for Rare Diseases (Trial)" (see attachment). According to the requirements of the "Notice of the General Office of the National Drug Administration on the Issuance of the Procedure for the Release of Drug Technical Guidelines" (Drug Supervision General Drug Administration [2020] No. 9), after review and approval by the National Drug Administration, it is now issued and will be implemented from the date of issuance.

This is hereby announced.

Attachment: Statistical Guidelines for Clinical Research of Drugs for Rare Diseases (Trial)

Drug Evaluation Center of the National Drug Administration

June 2, 2022

For specific article links, please see: Statistical Guidelines for Clinical Research of Drugs for Rare Diseases (Trial)