论文标题
基于新近的试验的活动臂试验的样本量计算
Sample Size Calculation for Active-Arm Trial with Counterfactual Incidence Based on Recency Assay
论文作者
论文摘要
在过去的十年中，在生物医学剂的发展中取得了巨大进展，这些生物医学预防前预防（PREP）可预防HIV。为了扩大产品和交付方法的选择，正在开发新的药物和递送方法。鉴于基于ARV的PREP产品成为当前或将来的护理标准的高疗效，未来的非效率试验将需要大量参与者和可能不可行的长时间随访时间。这激发了对反事实估计的构建，该估计值近似于未接受准备的随机并发对照组的发病率。我们提出了一种方法，该方法是招募一系列预期的预备使用者，并增加对HIV感染持续时间的实验室标记的HIV筛查，以表明最近的感染。我们讨论了这些数据将对反事实HIV发病率进行估计的假设，并开发样本量和功率计算，以将研究与研究剂观察到的发病率进行比较。
The past decade has seen tremendous progress in the development of biomedical agents that are effective as pre-exposure prophylaxis (PrEP) for HIV prevention. To expand the choice of products and delivery methods, new medications and delivery methods are under development. Future trials of non-inferiority, given the high efficacy of ARV-based PrEP products as they become current or future standard of care, would require a large number of participants and long follow-up time that may not be feasible. This motivates the construction of a counterfactual estimate that approximates incidence for a randomized concurrent control group receiving no PrEP. We propose an approach that is to enroll a cohort of prospective PrEP users and augment screening for HIV with laboratory markers of duration of HIV infection to indicate recent infections. We discuss the assumptions under which these data would yield an estimate of the counterfactual HIV incidence and develop sample size and power calculations for comparisons to incidence observed on an investigational PrEP agent.
