Interpretation of key points of the technical guidelines for clinical trials of drugs for the treatment of primary biliary cholangitis

Abstract

There are limited drug options in the field of primary biliary cholangitis, so there is a great clinical need. In recent years, research and development of PBC treatment medications have been active domestically and internationally, and clinical trials have been conducted on multiple drugs with distinct targets. Therefore, on February 13, 2023, the State Drug Administration issued the "Technical Guidelines for Clinical Trials of Drugs for the Treatment of Primary Biliary Cholangitis" in order to guide and standardize the clinical trials of drugs for the treatment of PBC. This article briefly summarizes the key points of the guiding principles, focuses on the difficulties of clinical evaluation of drugs, discusses the key elements of clinical trials such as the selection of test populations and efficacy endpoints, and introduces the determination process through literature searches and expert discussion methods combined with reviewer experience and scientific considerations.