We propose an information borrowing strategy for the design and monitoring of phase II basket trials based on the local multisource exchangeability assumption between baskets (disease types). In our proposed local-MEM framework, information borrowing is only allowed to occur locally, i.e., among baskets with similar response rate and the amount of information borrowing is determined by the level of similarity in response rate, whereas baskets not considered similar are not allowed to share information. We construct a two-stage design for phase II basket trials using the proposed strategy. The proposed method is compared to competing Bayesian methods and Simon's two-stage design in a variety of simulation scenarios. We demonstrate the proposed method is able to maintain the family-wise type I error rate at a reasonable level and has desirable basket-wise power compared to Simon's two-stage design. In addition, our method is computationally efficient compared to existing Bayesian methods in that the posterior profiles of interest can be derived explicitly without the need for sampling algorithms.
翻译:我们根据当地多来源交换篮子(疾病类型)的假设,提出了设计和监测第二阶段篮子试验的信息借款战略。在我们拟议的地方-MEM框架内,信息借款只允许在当地进行,即在答复率相似的篮子中,信息借款量取决于答复率的相似程度,而不认为相似的篮子则不允许分享信息。我们用拟议战略为第二阶段篮子试验设计了两阶段设计。拟议方法与相互竞争的Bayesian方法和西蒙在各种模拟情景中的两阶段设计相比较。我们证明,拟议方法能够将家庭一级I型错误率维持在合理的水平,与西蒙的两阶段设计相比,具有可取的篮子能力。此外,我们的方法与巴伊斯现有方法相比,计算效率很高,因为在不需要抽样算法的情况下可以明确得出后方利益简介。