Statistical Considerations in Pediatric Cancer Trials: Report of American Statistical Association Biopharmaceutical Section Open Forum Discussions

IF 1.5 4区医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY Statistics in Biopharmaceutical Research Pub Date : 2023-07-25 DOI:10.1080/19466315.2023.2238650

R. Sridhara, Olga V. Marchenko, Qi Jiang, Elizabeth Barksdale, T. Alonzo, Anup K Amatya, David F. Arons, Alex Bliu, Qiuyi Choo, M. Coory, Martha Donoghue, L. Ehrlich, Leonardo Fabio Costa Filho, E. Fox, B. Freidlin, Nancy Goodman, D. Hawkins, D. Häring, Dominik Karres, E. Kolb, Helen Mao, Pallavi S. Mishra Kalyani, A. Naranjo, A. Pappo, M. Posch, Karen L. Price, A. Raven, K. Rantell, Lindsay Renfro, D. Rivera, Pourab Roy, Ming-wei Shan, Richard Simon, Sonia Singh, Malcolm Smith, M. Theoret, Marius Thomas, Z. Thomas, A. Thompson, Hong Tian, Y. Tymofyeyev, Jonathon Vallejo, K. Wathen, Jingjing Ye, R. Pazdur, G. Reaman

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Abstract

Abstract This article provides a summary of discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forum organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group in coordination with the US FDA Oncology Center of Excellence and LUNGevity Foundation on June 24, 2021, and January 13, 2022. Diverse stakeholders engaged in a discussion on how best to use various innovative clinical trial designs in designing future pediatric oncology trials. While standard randomized controlled trials are preferred to evaluate treatment effect in an unbiased manner, given the rarity of pediatric cancers, innovative strategies are needed to promote and assure timely cancer drug development in pediatric populations. The discussions highlighted the need to consider innovative designs with less stringent Type I error specification and Bayesian designs borrowing from external control data, or borrowing treatment effect information from adult data, or both. Such designs are available in the literature and some examples are summarized under the FDA Complex Innovative Trials Design Pilot Program (https://www.fda.gov/drugs/development-resources/complex-innovative-trial-design-meeting-program). Early consultation with global regulatory agencies for pediatric clinical trials can provide a better understanding of different features of the clinical trial design options for successful pediatric cancer drug development.

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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.