Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK.
Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK.
Contemp Clin Trials. 2022 Jan;112:106625. doi: 10.1016/j.cct.2021.106625. Epub 2021 Nov 15.
Platform designs - master protocols that allow for new treatment arms to be added over time - have gained considerable attention in recent years. Between 2001 and 2019, 16 platform trials were initiated globally. The COVID-19 pandemic seems to have provided a new motivation for these designs. We conducted a rapid review to quantify and describe platform trials used in COVID-19.
We cross-referenced PubMed, ClinicalTrials.gov, and the Cytel COVID-19 Clinical Trials Tracker to identify platform trials, defined by their stated ability to add future arms.
We identified 58 COVID-19 platform trials globally registered between January 2020 and May 2021. According to trial registries, 16 trials have added new therapies (median 3, IQR 4) and 11 have dropped arms (median 3, IQR 2.5). About 50% of trials publicly share their protocol, and 31 trials (53%) intend to share trial data. Forty-nine trials (84%) explicitly report adaptive features, and 21 trials (36%) state Bayesian methods.
During the pandemic, there has been a surge in the number of platform trials compared to historical use. While transparency in statistical methods and clarity of data sharing policies needs improvement, platform trials appear particularly well-suited for rapid evidence generation. Trials secured funding quickly and many succeeded in adding new therapies in a short time period, thus demonstrating the potential for these trial designs to be implemented beyond the pandemic. The evidence gathered here may provide ample insight to further inform operational, statistical, and regulatory aspects of future platform trial conduct.
平台设计——允许随着时间的推移添加新治疗方案的主协议——近年来受到了广泛关注。在 2001 年至 2019 年期间，全球启动了 16 项平台试验。COVID-19 大流行似乎为这些设计提供了新的动力。我们进行了一项快速审查，以量化和描述用于 COVID-19 的平台试验。
我们通过交叉引用 PubMed、ClinicalTrials.gov 和 Cytel COVID-19 临床试验追踪器来识别平台试验，这些试验通过其能够添加未来方案的能力来定义。
我们在全球范围内确定了 58 项 COVID-19 平台试验，这些试验于 2020 年 1 月至 2021 年 5 月期间注册。根据试验注册处的信息，有 16 项试验已经添加了新的治疗方法（中位数 3，IQR 4），11 项试验已经取消了方案（中位数 3，IQR 2.5）。大约 50%的试验公开分享了他们的方案，31 项试验（53%）计划分享试验数据。49 项试验（84%）明确报告了适应性特征，21 项试验（36%）表示使用贝叶斯方法。
在大流行期间，与历史使用相比，平台试验的数量激增。虽然统计方法的透明度和数据共享政策的清晰度需要改进，但平台试验似乎特别适合快速生成证据。试验迅速获得资金，并在短时间内成功添加了新的治疗方法，这表明这些试验设计有可能在大流行之外得到实施。这里收集的证据可能为进一步告知未来平台试验的操作、统计和监管方面提供充分的见解。
