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
A hypothesis test of feasibility for external pilot trials assessing recruitment, follow-up and adherence rates
摘要：
一项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该继续进行。关于如何选择这些决策规则或试点的样本量，几乎没有方法学研究。在本文中，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力量，作为招募，随访和依从率的函数来量化可行性。我们使用该指标来定义在试点中进行检验的假设，提出了一个检验统计量，并展示了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，一个外部试点试验，比较心理治疗与常规治疗行为障碍儿童。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。并将其纳入进展决定中。
英文摘要：
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 pre-specified 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 paper 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 re-design 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.
机构、总结、中文摘要均由AI分析而来，可能存在错误，请以论文正文为准。
