Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators

IF 1.5 4区医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY

Statistics in Biopharmaceutical Research Pub Date : 2023-01-09 DOI:10.1080/19466315.2023.2166099

Olga V. Marchenko, R. Sridhara, Qi Jiang, Elizabeth Barksdale, Y. Ando, D. D. Alwis, Katie Brown, L. Fernandes, M. V. van Bussel, Qiuyi Choo, M. Coory, E. Garrett-Mayer, T. Gwise, Lorenzo Hess, Rong Liu, S. Mandrekar, D. Ouellet, J. Pinheiro, M. Posch, N. Rahman, K. Rantell, A. Raven, Sarem Sarem, S. Sen, M. Shah, Y. Shen, Richard Simon, M. Theoret, Ying Yuan, R. Pazdur

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引用次数: 0

Abstract

Abstract The article provides a summary of discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forums on March 18th, June 10th, and July 8th of 2021, organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group in coordination with the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence and the LUNGevity Foundation. Diverse stakeholders including oncologists, patient advocates, experts from regulatory agencies across the world, academicians, and representatives from the pharmaceutical industry engaged in a lively discussion on strategies for and designs of dose-optimization studies in cancer drug development. Dose-optimization is one of the major challenges in oncology drug development. The discussions were focused on considerations in designing dose-optimization studies of products for treatment of cancer patients in pre-approval and post-approval stages. Presenters and panelists discussed diverse ideas and methods and agreed that a shift in paradigm is required in oncology drug development that should improve dose optimization while not unnecessarily delaying patient access to potentially efficacious new treatments.

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设计癌症药物开发中的剂量优化研究:与监管机构的讨论

摘要:本文提供了2021年3月18日、6月10日和7月8日美国统计协会(ASA)生物制药(BIOP)分会公开论坛的讨论总结，该论坛由ASA BIOP肿瘤科学工作组与美国食品和药物管理局(FDA)肿瘤卓越中心和LUNGevity基金会协调组织。包括肿瘤学家、患者倡导者、来自世界各地监管机构的专家、学者和制药行业代表在内的各种利益相关者就癌症药物开发中剂量优化研究的策略和设计进行了热烈的讨论。剂量优化是肿瘤药物开发的主要挑战之一。讨论的重点是在批准前和批准后阶段设计用于治疗癌症患者的产品的剂量优化研究的考虑因素。演讲者和小组成员讨论了不同的想法和方法，并一致认为肿瘤药物开发需要转变模式，以改善剂量优化，同时不会不必要地延迟患者获得潜在有效的新治疗方法。

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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.