Minimizing Patient Burden Through the Use of Historical Subject-Level Data in Innovative Confirmatory Clinical Trials: Review of Methods and Opportunities

作     者：Lim, Jessica Walley, Rosalind Yuan, Jiacheng Liu, Jeen Dabral, Abhishek Best, Nicky Grieve, Andrew Hampson, Lisa Wolfram, Josephine Woodward, Phil Yong, Florence Zhang, Xiang Bowen, Ed 

作者机构：GlaxoSmithKline Clin Stat Collegeville PA USA UCB Ctr Excellence Stat Innovat Slough Berks England Allergan Stat Sci & Programming Irvine CA USA GlaxoSmithKline Adv Biostat & Data Analyt Ctr Excellence Uxbridge Middx England Novartis Pharma AG Stat Methodol & Consulting Basel Switzerland Astellas Data Sci Biostat Leiden Netherlands Pfizer Biostat Worldwide Res & Dev Cambridge MA USA Eli Lilly & Co Global Stat Sci Indianapolis IN 46285 USA GlaxoSmithKline R&D Data Ctr Excellence Collegeville PA USA 

出 版 物：《THERAPEUTIC INNOVATION & REGULATORY SCIENCE》 (药物信息杂志)

年 卷 期：2018年第52卷第5期

页      面：546-559页

核心收录：

学科分类：1007[医学-药学(可授医学、理学学位)] 1001[医学-基础医学(可授医学、理学学位)] 10[医学] 

基　　金：UK Medical Research Council [MR/M013510/1] TransCelerate BioPharma Inc MRC [MR/M013510/1] Funding Source: UKRI 

主　　题：TransCelerate historical controls propensity score Bayesian informative prior 

摘      要：The goal of clinical trial research is to deliver safe and efficacious new treatments to patients in need in a timely and cost-effective manner. There is precedent in using historical control data to reduce the number of concurrent control subjects required in developing medicines for rare diseases and other areas of unmet need. The purpose of this paper is to provide a review for a regulatory and industry audience of the current state of relevant statistical methods, and of the uptake of these approaches and the opportunities for broader use of historical data in confirmatory clinical trials. General principles to consider when incorporating historical control data in a new trial are presented. Bayesian and frequentist approaches are outlined including how the operating characteristics for such a trial can be obtained. Finally, examples of approved new treatments that incorporated historical controls in their confirmatory trials are presented.