Statistical approaches for optimizing the design and analysis of clinical trials

2023 Fiscal Year Final Research Report

• Project/Area Number: 20K18900
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 58010:Medical management and medical sociology-related
• Research Institution: National Cardiovascular Center Research Institute
• Principal Investigator: Asakura Koko 国立研究開発法人国立循環器病研究センター, 病院, 室長 (70738690)
• Project Period (FY): 2020-04-01 – 2024-03-31
• Keywords: 臨床試験 / 多重性 / 中間解析 / 適応的デザイン

Outline of Final Research Achievements

In this research, we proposed statistical methods for optimising the adaptive design of a clinical trial to evaluate the efficacy of new treatments. Specifically, we investigated methods for study design and interim decision making for adaptive hypothesis selection, analysis plan optimisation and small clinical trial planning.
The applicability of the proposed method to actual clinical trials was evaluated by examining the performance of the proposed method in terms of power and sample size under Type I error control.

Free Research Field

生物統計学

Academic Significance and Societal Importance of the Research Achievements

臨床試験の効率化を目的としたデザインや解析に関する統計的方法が活発に議論されてきたが，各々の試験に最適なデザインとその設定の選択に関し，一致した見解をもって即時に実施可能な状態とは言い難い．そのために最適な統計手法を実地で選択・活用できる環境の整備が望まれている．
本研究により，試験の主要なゴールは一貫させたもとで，試験途中で検討する仮説の数や順序または重みを最適化することが可能となり，既存の手法とは異なる方向性で試験の成功確率および効率性を高めることが期待できる．