A hypothesis test of feasibility for external pilot trials assessing recruitment, follow-up, and adherence rates.
A hypothesis test of feasibility for external pilot trials assessing recruitment, follow-up, and adherence rates.
复制标题
评估招募、随访和依从率的外部试点试验可行性的假设检验。
DOI：
10.1002/sim.9091
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发表时间：
2021
影响因子：
2
通讯作者：
Wilson DT
中科院分区：
文献类型：
--
作者：
Wilson DT
大型临床试验的效力可能会受到招募率、随访率和依从率低的不利影响。外部试点试验估计这些比率，并通过预先规定的决策规则使用它们来确定最终试验是否可行并应继续进行。关于如何选择这些决策规则或试点样本规模的方法研究很少。在这篇文章中，我们提出了一个确定性试验的可行性假设检验，适用于外部试点数据，并用于作出进展的决定。我们通过计划试验的功效量化可行性，作为招募，随访和依从率的函数。我们使用该指标来定义在试点中进行检验的假设，提出检验统计量，并说明如何计算该检验的错误率，用于具有单一正态分布主要终点的双臂平行组确定性试验的常见场景。我们使用我们的方法来重新设计TIGA‐CUB，这是一项外部试点试验，比较了心理治疗与常规治疗对品行障碍儿童的影响。然后，我们扩展了我们的公式，包括使用试点数据来估计主要终点的标准差，并将其纳入进展决策。
The power of a large clinical trial can be adversely affected by low recruitment, follow‐up, and adherence rates. External pilot trials estimate these rates and use them, via prespecified decision rules, to determine if the definitive trial is feasible and should go ahead. There is little methodological research underpinning how these decision rules, or the sample size of the pilot, should be chosen. In this article we propose a hypothesis test of the feasibility of a definitive trial, to be applied to the external pilot data and used to make progression decisions. We quantify feasibility by the power of the planned trial, as a function of recruitment, follow‐up, and adherence rates. We use this measure to define hypotheses to test in the pilot, propose a test statistic, and show how the error rates of this test can be calculated for the common scenario of a two‐arm parallel group definitive trial with a single normally distributed primary endpoint. We use our method to redesign TIGA‐CUB, an external pilot trial comparing a psychotherapy with treatment as usual for children with conduct disorders. We then extend our formulation to include using the pilot data to estimate the standard deviation of the primary endpoint and incorporate this into the progression decision.