Department of General Medical Oncology, University Hospitals Leuven, KU Leuven, Leuven Cancer Institute, Leuven, Belgium
BMJ Open. 2021 Oct 25;11(10):e047294. doi: 10.1136/bmjopen-2020-047294.
The role and use of independent data monitoring committees (IDMCs) has evolved over the past decades. The Food and Drug Administration and European Medicines Agency have issued guidelines on the role and functioning of such committees. In general, data monitoring committees are recommended for large, often randomised clinical trials involving life-threatening diseases, studies performed in vulnerable populations or where the experimental intervention can potentially harm the trial participant. Such committees play an important role in trials evaluating treatments with the potential to prolong life or reduce the risk of major adverse health outcomes.Typically, oncology clinical trials fall within these recommendations, as they are often large, randomised, multicentric protocols aiming at improving survival outcomes by exploring the use of study treatments that may be associated with a significant risk of serious, even life-threatening adverse events. IDMCs are required for National Cancer Institute phase III randomised trials, European Organisation for Research and Treatment of Cancer phase II/III trials with formal interim analyses, early-stopping rules or adaptive studies. The primary role of an IDMC of ensuring the safety of study participants and maintaining clinical trial integrity is particularly important in oncology trials, due to the nature of the disease, the potential for treatment toxicity and for instilling confidence that the clinical trial data are reliable. A clear understanding by IDMC members of the natural course of the disease, treatment landscape, importance and relevance of certain adverse events in trial participants, clinical trial methodology in general and stopping rules for oncology trials in particular, is crucial for the functioning of an IDMC.It is recommended that IDMC members should be experienced trialists, have a track record of strong clinical, statistical and/or methodological expertise and the required level of independence, as they play a highly important role in the protection of study participants, and in commercially and strategically important go/no decisions. Ideally, IDMC members should have relevant experience or have some training, mentorship or guidelines.
独立数据监测委员会（IDMC）的作用和用途在过去几十年中不断发展。美国食品和药物管理局和欧洲药品管理局已经发布了关于此类委员会的作用和功能的指南。一般来说，建议在涉及危及生命的疾病、在弱势群体中进行的研究或实验性干预可能对试验参与者造成潜在伤害的大型、通常是随机临床试验中使用数据监测委员会。此类委员会在评估有延长生命或降低主要不良健康结果风险的治疗方法的试验中发挥着重要作用。通常，肿瘤学临床试验符合这些建议，因为它们通常是大型、随机、多中心的方案，旨在通过探索可能与严重甚至危及生命的不良事件相关的研究治疗方法来改善生存结果。国家癌症研究所 III 期随机试验、欧洲癌症研究与治疗组织 II/III 期试验（具有正式中期分析、提前终止规则或适应性研究）需要 IDMC。IDMC 的主要作用是确保研究参与者的安全并维护临床试验的完整性，这在肿瘤学试验中尤为重要，因为疾病的性质、治疗毒性的可能性以及让人们相信临床试验数据是可靠的，这一点非常重要。IDMC 成员对疾病的自然病程、治疗现状、试验参与者中某些不良事件的重要性和相关性、一般临床试验方法以及肿瘤学试验的停止规则有清晰的了解，这对于 IDMC 的运作至关重要。建议 IDMC 成员应该是经验丰富的试验人员，具有强大的临床、统计和/或方法学专业知识和所需的独立性记录，因为他们在保护研究参与者以及在商业和战略上重要的是/否决策方面发挥着非常重要的作用。理想情况下，IDMC 成员应该具有相关经验，或者具有一些培训、指导或指南。
