Clinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation

IF 1.5 4区医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY

Statistics in Biopharmaceutical Research Pub Date : 2023-07-03 DOI:10.1080/19466315.2022.2081601

Alexei C. Ionan, M. Paterniti, D. Mehrotra, John Scott, B. Ratitch, S. Collins, S. Gomatam, L. Nie, K. Rufibach, F. Bretz

引用次数: 2

Abstract

Abstract The ICH E9 (R1) Addendum on “Estimands and Sensitivity Analysis in Clinical Trials (Step 4)” was finalized in November 2019 and subsequently implemented by many regulatory agencies, including FDA (May 2021). This article is based on a session organized to cover experience implementing the estimand framework, including its use, impact on drug/biologic development, common challenges and ways to address them, as well as keys to productive interdisciplinary collaboration.

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ICH E9(R1)评估框架实施的临床和统计观点

ICH E9 (R1)附录“临床试验中的估计和敏感性分析(步骤4)”于2019年11月定稿，随后由包括FDA在内的许多监管机构实施(2021年5月)。这篇文章是基于一个会议的基础上组织的，该会议涵盖了实施评估框架的经验，包括它的使用，对药物/生物开发的影响，共同的挑战和解决这些挑战的方法，以及有效的跨学科合作的关键。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Statistics in Biopharmaceutical Research MATHEMATICAL & COMPUTATIONAL BIOLOGY-STATISTICS & PROBABILITY

CiteScore

3.90

自引率

16.70%

发文量

期刊介绍： Statistics in Biopharmaceutical Research ( SBR), publishes articles that focus on the needs of researchers and applied statisticians in biopharmaceutical industries; academic biostatisticians from schools of medicine, veterinary medicine, public health, and pharmacy; statisticians and quantitative analysts working in regulatory agencies (e.g., U.S. Food and Drug Administration and its counterpart in other countries); statisticians with an interest in adopting methodology presented in this journal to their own fields; and nonstatisticians with an interest in applying statistical methods to biopharmaceutical problems. Statistics in Biopharmaceutical Research accepts papers that discuss appropriate statistical methodology and information regarding the use of statistics in all phases of research, development, and practice in the pharmaceutical, biopharmaceutical, device, and diagnostics industries. Articles should focus on the development of novel statistical methods, novel applications of current methods, or the innovative application of statistical principles that can be used by statistical practitioners in these disciplines. Areas of application may include statistical methods for drug discovery, including papers that address issues of multiplicity, sequential trials, adaptive designs, etc.; preclinical and clinical studies; genomics and proteomics; bioassay; biomarkers and surrogate markers; models and analyses of drug history, including pharmacoeconomics, product life cycle, detection of adverse events in clinical studies, and postmarketing risk assessment; regulatory guidelines, including issues of standardization of terminology (e.g., CDISC), tolerance and specification limits related to pharmaceutical practice, and novel methods of drug approval; and detection of adverse events in clinical and toxicological studies. Tutorial articles also are welcome. Articles should include demonstrable evidence of the usefulness of this methodology (presumably by means of an application). The Editorial Board of SBR intends to ensure that the journal continually provides important, useful, and timely information. To accomplish this, the board strives to attract outstanding articles by seeing that each submission receives a careful, thorough, and prompt review. Authors can choose to publish gold open access in this journal.