论文标题
确认性两阶段自适应程序的特性，用于评估平均生物等效性
Properties of a confirmatory two-stage adaptive procedure for assessing average bioequivalence
论文作者
论文摘要
我们研究了一种确认性的两阶段自适应程序，用于评估平均生物等效性，并为其理论特性提供一些见解。有效地，我们进行了两项单方面测试（TOST），以对宣布平均生物等效性涉及的两种传统的无效杂种中的每一个中的每一个都做出总体决策。基于相应的阶段p值对，将测试分别作为每个假设的组合测试。该过程的特征包括内置徒劳，样本量重新评估以及同时评估平均生物等效性相对于多个终点的能力，同时控制家庭误差率。为了促进试验结束时的推论，我们还得出了与每个单方面假设所达成的决策相匹配的总体置信度限制，并提供了理论确保其适当性。在计划一项研究的背景下，通过模拟来评估该性能，以比较特应性皮炎的生物治疗的两种不同的施用。
We investigate a confirmatory two stage adaptive procedure for assessing average bioequivalence and provide some insights to its theoretical properties. Effectively, we perform Two One-Sided Tests (TOST) to reach overall decision about each of the two traditional null-hypotheses involved in declaring average bioequivalence. The tests are performed as combination tests separately for each hypothesis based on the corresponding pair of stagewise p-values. Features of the procedure include a built in futility, sample size reassessment, and the ability to simultaneously assess average bioequivalence with respect to multiple endpoints while controlling the familywise error rate. To facilitate inference at the end of a trial we also derive overall confidence limits that match the decision reached on each one sided hypothesis and provide theory ensuring their appropriateness. The performance is assessed by simulation in the context of planning a study to compare two different administrations of a biologic treatment of atopic dermatitis.
