-
学术期刊
-
学术著作
-
精品图书
-
投稿须知
International Medical Research and Translation (IMRT) June 2026, Vol.2, No.3
The Application of Synchronous Verification in the Production of Rare Disease Drugs
Hai Tong
Shanghai SynTheAll Pharmaceutical Co., Ltd., Shanghai 201507, China
Abstract: Concurrent verification involves collecting verification data simultaneously during commercial production, and is mainly used for the production of rare disease drugs where there is an urgent clinical need and traditional pre-verification cannot be adopted. This paper takes the concurrent verification project of the orphan drug Elamipretide for treating Barth syndrome as an example, systematically introducing the implementation difficulties, quality control methods and core achievements of concurrent verification in the production of rare disease drugs. The practice shows that this method successfully completed the first concurrent verification project of the enterprise, filled the experience gap in the verification of rare disease drugs, helped the client obtain FDA priority review and be approved for market launch, and provided practical and feasible technical references for pharmaceutical enterprises to conduct concurrent verification of rare disease drugs.
Keywords: concurrent verification, rare disease drugs, orphan drugs, quality risk management, cGMP
