Research on Clinical Trial Information Used in Biosimilar Development in Japan

Abstract

 The proliferation of biosimilars has garnered significant attention, with their development being highly sought after. However, there is a paucity of systematic information demonstrating the comparability evaluation of biosimilars to biotechnology-derived drugs already approved in Japan as new active ingredient-containing pharmaceuticals. This study aimed to elucidate trends in clinical trials conducted for biosimilar development by investigating and analyzing clinical trial information for currently approved biosimilars. We examined 31 biosimilars approved in Japan by March 2023, extracting data from review reports on trial phase, sample size, number of Japanese participants in multi-regional clinical trials (MRCTs), trial locations (domestic/international), endpoints, and observation periods. All 31 products were approved using Japanese data. Of the 18 products approved in Japan subsequent to overseas approval, 14 were approved using information from overseas sources, either in studies to clinical pharmacology or studies to verify efficacy equivalence. Between 2017 and 2022, 12 products (50.0％) were approved based on MRCTs. On January 25, 2024, The Ministry of Health, Labour and Welfare of Japan issued “Questions and Answers (Q & A) on Guidelines for Ensuring the Quality, Efficacy, and Safety of Biosimilars” (Administrative Notice, January 25, 2024). This notice is expected to expedite the time to marketing approval through increased participation in MRCTs and modifications to the criteria for utilizing Japanese data. Our study elucidates the current landscape of biosimilar development in Japan and the characteristics of associated clinical trials, underscoring the necessity for vigilant monitoring of future trends.