A new clinical trial design considering the efficiency of multiple primary endpoints

2014 Fiscal Year Final Research Report

• Project/Area Number: 25870332
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Statistical science; Epidemiology and preventive medicine
• Research Institution: Kyoto University
• Principal Investigator: SOZU Takashi 京都大学, 医学研究科, 准教授 (80408723)
• Project Period (FY): 2013-04-01 – 2015-03-31
• Keywords: 医薬品開発 / 治験 / 主要評価変数 / エンドポイント / 費用効率 / 多重性 / サンプルサイズ / 群逐次デザイン

Outline of Final Research Achievements

We developed a new design for clinical trials comparing two treatments using multiple primary endpoints in which the number of necessary measurements is determined for each of the endpoints, taking into consideration both the cost and the individual power for each endpoint. We compared the efficiency of the proposed design with that of the conventional design using three variables: (1) the number of participants in the study, (2) the total number of measurements for all endpoints, and (3) the cost of enrolling the participants and obtaining the measurements for all endpoints. The conditions for the proposed design to be useful were found. We extended the proposed design to a group-sequential design.

Free Research Field

医療統計学