Designing and conducting adaptive trials to evaluate interventions in health services and implementation research: practical considerations.
Designing and conducting adaptive trials to evaluate interventions in health services and implementation research: practical considerations.
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设计和进行适应性试验以评估卫生服务干预措施和实施研究：实际考虑。
DOI：
10.1136/bmjmed-2022-000158
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发表时间：
2022
期刊：
影响因子：
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通讯作者：
Trippa,Lorenzo
中科院分区：
文献类型：
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作者：
Lauffenburger,JulieC;Choudhry,NiteeshK;Russo,Massimiliano;Glynn,RobertJ;Ventz,Steffen;Trippa,Lorenzo
随机对照临床试验被广泛认为是评估医疗保健干预措施的功效或效果的首选方法。适应性试验随着研究的进行而发生变化，例如修改分配概率或消除可能无效的治疗组。这些设计已广泛用于药物发现研究，但也可用于卫生服务和实施研究，但使用很少。在本文中，我们使用正在进行的适应性试验和两个已完成的平行小组研究作为激励示例，以强调在卫生服务和实施研究中使用适应性试验设计时的潜在优点、缺点和重要考虑因素。我们还调查了如果使用适应性原则进行两个完成的平行组试验对功效和研究持续时间的影响。与传统试验设计相比，适应性设计通常可以评估更多干预措施，调整参与者分配概率（例如，实现协变量平衡），并识别可能同意入组的参与者。这些功能可以减少进行试验所需的资源。然而，适应性试验具有潜在的缺点和需要考虑的实际问题，最值得注意的是：可以快速测量和提取的结果（例如，需要相当长的时间从数据源测量的长期结果可能具有挑战性）、最小的缺失数据和时间趋势。总之，适应性设计是一种很有前途的方法，可以帮助确定如何最好地将基于证据的干预措施实施到卫生服务和实施研究的现实世界实践中。
Randomised controlled clinical trials are widely considered the preferred method for evaluating the efficacy or effectiveness of interventions in healthcare. Adaptive trials incorporate changes as the study proceeds, such as modifying allocation probabilities or eliminating treatment arms that are likely to be ineffective. These designs have been widely used in drug discovery studies but can also be useful in health services and implementation research and have been minimally used. In this article, we use an ongoing adaptive trial and two completed parallel group studies as motivating examples to highlight the potential advantages, disadvantages, and important considerations when using adaptive trial designs in health services and implementation research. We also investigate the impact on power and the study duration if the two completed parallel group trials had instead been conducted using adaptive principles. Compared with traditional trial designs, adaptive designs can often allow the evaluation of more interventions, adjust participant allocation probabilities (eg, to achieve covariate balance), and identify participants who are likely to agree to enrol. These features could reduce resources needed to conduct a trial. However, adaptive trials have potential disadvantages and practical aspects that need to be considered, most notably: outcomes that can be rapidly measured and extracted (eg, long term outcomes that take considerable time to measure from data sources can be challenging), minimal missing data, and time trends. In conclusion, adaptive designs are a promising approach to help identify how best to implement evidence based interventions into real world practice in health services and implementation research.