Development of new endpoints and analysis methods for clinical trials in emerging infectious diseases

2023 Fiscal Year Final Research Report

• Project/Area Number: 21K10296
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 58010:Medical management and medical sociology-related
• Research Institution: National Center for Global Health and Medicine
• Principal Investigator: Uemura Yukari 国立研究開発法人国立国際医療研究センター, 臨床研究センター, 臨床研究センター データサイエンス部 生物統計研究室 室長 (80548537)
• Co-Investigator(Kenkyū-buntansha): 坂巻 顕太郎 横浜市立大学, 附属市民総合医療センター, 助教 (30644819)
• Project Period (FY): 2021-04-01 – 2024-03-31
• Keywords: 主要評価項目 / 新興感染症 / 検出力 / 臨床試験

Outline of Final Research Achievements

n drug development during emerging infectious disease outbreaks, it is important to simultaneously enroll patients with a wide range of disease severity into clinical trials and obtain rapid approval, given the limited information available on infectious diseases and drugs. However, because emerging infectious diseases have both worsening and improving clinical courses, it is difficult to define appropriate primary endpoints that can validate treatment efficacy.
In fact, clinical trials of COVID-19 have revealed that a variety of endpoints have been established. A prioritized composite outcome was considered to be effective in evaluating a comprehensive treatment effect that summarizes the clinical course in both directions, and its operational characteristics were evaluated through simulation experiments.

Free Research Field

生物統計学

Academic Significance and Societal Importance of the Research Achievements

本研究は新興感染症領域における臨床試験に対し、悪化と改善の両方向の臨床経過を包括的な評価可能なprioritized composite outcome（PCE）を主要評価項目と設定することを提案した。悪化と改善の両方向の臨床的効果を評価する他の方法として、複数評価項目を設定して多重性により考慮する方法や競合リスクを用いた方法が考えうるが、それら方法とPCEの動作特性の違いについてシミレーション実験を通じて評価した。