Center for Outcomes and Measurement, College of Rehabilitation Sciences, Thomas Jefferson University, Philadelphia, PA, USA.
University of Colorado, Denver, CO, USA.
Spinal Cord. 2020 Dec;58(12):1235-1248. doi: 10.1038/s41393-020-00547-8. Epub 2020 Sep 16.
Narrative review.
To provide an overview of adaptive trial designs, and describe how adaptive methods can address persistent challenges encountered by randomized controlled trials of people with spinal cord injury (SCI).
With few exceptions, adaptive methodologies have not been incorporated into clinical trial designs of people with SCI. Adaptive methods provide an opportunity to address high study costs, slow recruitment, and excessive amount of time needed to carry out the trial. The availability of existing SCI registries are well poised to support modeling and simulation, both of which are used extensively in adaptive trial designs. Eight initiatives for immediate advancement of adaptive methods in SCI were identified.
Although successfully applied in other fields, adaptive clinical trial designs in SCI clinical trial programs have been narrow in scope and few in number. Immediate application of several adaptive methods offers opportunity to improve efficiency of SCI trials. Concerted effort is needed by all stakeholders to advance adaptive clinical trial design methodology in SCI.
叙述性评论。
提供适应性试验设计概述，并描述适应性方法如何解决脊髓损伤（SCI）患者随机对照试验中遇到的持续挑战。
除了少数例外，适应性方法尚未纳入 SCI 患者的临床试验设计中。适应性方法提供了一种机会，可以解决高研究成本、招募缓慢以及开展试验所需的大量时间问题。现有的 SCI 登记处可很好地支持建模和模拟，这两者在适应性试验设计中都得到了广泛应用。确定了八项即时推进 SCI 适应性方法的倡议。
尽管在其他领域得到了成功应用，但 SCI 临床试验计划中的适应性临床试验设计范围狭窄，数量有限。立即应用几种适应性方法为提高 SCI 试验的效率提供了机会。所有利益相关者都需要共同努力，推进 SCI 中的适应性临床试验设计方法。
