Development of statistical methodology for evaluating heterogeneity of treatment effects in basket clinical trials

2023 Fiscal Year Final Research Report

• Project/Area Number: 20K11703
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 60030:Statistical science-related
• Research Institution: Tokyo Medical and Dental University
• Principal Investigator: Hirakawa Akihiro 東京医科歯科大学, 大学院医歯学総合研究科, 教授 (90609330)
• Project Period (FY): 2020-04-01 – 2024-03-31
• Keywords: 臨床試験方法論 / 生物統計学 / ベイズ流アプローチ / がん / バスケットデザイン

Outline of Final Research Achievements

Clinical trials (basket trials) aimed at evaluating drug efficacy across multiple cancer types are increasing. This study focuses on a Bayesian method for estimating response rates by incorporating the homogeneity or heterogeneity of efficacy between different cancer types into the statistical model. We have advanced the research and development of several novel statistical methodologies.

Free Research Field

臨床試験方法論,生物統計学

Academic Significance and Societal Importance of the Research Achievements

本研究では，複数のがん種を対象にがん種横断的に被験薬の治療効果を評価する第2相バスケット試験で用いるベイズ流階層モデルについて研究開発を進め，論文投稿を行った．近年では，FDAが推進するProject Optimusにより，がん早期臨床試験デザインにパラダイムシフトが起こりつつある．本研究の成果は，がん早期臨床試験の成功確率を上げることに寄与するものと考える．