Inside the Mind of the DMC: A Review of Principles and Issues with Case Studies.

IF 2 4区 医学 Q4 MEDICAL INFORMATICS Therapeutic innovation & regulatory science Pub Date : 2024-12-17 DOI:10.1007/s43441-024-00720-8
Lizhao Ge, Toshimitsu Hamasaki, Scott R Evans
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Abstract

A data monitoring committee (DMC) can have an extremely challenging job. Stop a trial too soon, and results are inconclusive and the trial fails to obtain answers to important questions that could inform future clinical practice. Stop a trial too late, and trial participants are exposed to potentially harmful or ineffective interventions longer than necessary. Securing convincing and conclusive evidence and the ethical responsibility to current and future patients are weighed carefully during DMC deliberations. The ability to interpret complex information, and appreciation of issues affecting scientific integrity, are critical for the DMC to protect trial participants and public trust. Challenges faced by and issues of prudence faced by DMCs are discussed including interim analysis issues, assessing the totality of information with statistical boundaries as guidelines, interpretation of composite and surrogate outcomes, reactions to early trends, benefit:risk assessment, landscape changes, subgroup analyses, composing information for a comprehensive understanding of patient-centric effects, and evaluating the value of additional data. Case studies illustrate how DMCs addressed the challenges.

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数据监控委员会(DMC)的工作极具挑战性。过早停止一项试验,试验结果就会不确定,试验也就无法回答一些重要问题,而这些问题可以为未来的临床实践提供参考。试验停止得太晚,试验参与者接受潜在有害或无效干预的时间就会超过必要的时间。在 DMC 审议期间,要仔细权衡确保获得令人信服的确凿证据以及对当前和未来患者的道德责任。解读复杂信息的能力以及对影响科学诚信问题的认识,对于 DMC 保护试验参与者和公众信任至关重要。本文讨论了 DMC 所面临的挑战和需要谨慎处理的问题,包括中期分析问题、以统计学界限为准则评估全部信息、解释综合结果和替代结果、对早期趋势的反应、获益:风险评估、环境变化、亚组分析、综合信息以全面了解以患者为中心的效果,以及评估额外数据的价值。案例研究说明了 DMC 如何应对这些挑战。
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
Therapeutic innovation & regulatory science
Therapeutic innovation & regulatory science MEDICAL INFORMATICS-PHARMACOLOGY & PHARMACY
CiteScore
3.40
自引率
13.30%
发文量
127
期刊介绍: Therapeutic Innovation & Regulatory Science (TIRS) is the official scientific journal of DIA that strives to advance medical product discovery, development, regulation, and use through the publication of peer-reviewed original and review articles, commentaries, and letters to the editor across the spectrum of converting biomedical science into practical solutions to advance human health. The focus areas of the journal are as follows: Biostatistics Clinical Trials Product Development and Innovation Global Perspectives Policy Regulatory Science Product Safety Special Populations
期刊最新文献
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