论文标题
使用反应自适应前向Gittins指数规则的临床试验治疗差异的新型统计检验
A Novel Statistical Test for Treatment Differences in Clinical Trials using a Response Adaptive Forward Looking Gittins Index Rule
论文作者
论文摘要
反应自适应临床试验的最常见目标是寻求确保试验中的患者有很大的机会通过根据积累数据来改变分配的机会来获得最佳的治疗方法。由于治疗分配的高度不平衡，在研究结束时测试治疗差异时，从常见的角度来看，产生良好患者利益特性的方法具有低功率。在这项工作中，我们根据协变量调整后的响应自适应随机随机分配概率（通过前视GITTINS指数规则（CARA-FLGI））开发了用于治疗差异的替代测试。在模拟中评估了新型测试的性能，以进行两臂研究，然后说明了其在多臂研究中的应用。当使用此类非侧反应适应时，提出的测试显着提高了传统Fisher精确测试的功率。我们还发现，测试的功率接近在同等随机分配下Fisher精确测试的功率。
The most common objective for response adaptive clinical trials is to seek to ensure that patients within a trial have a high chance of receiving the best treatment available by altering the chance of allocation on the basis of accumulating data. Approaches which yield good patient benefit properties suffer from low power from a frequentist perspective when testing for a treatment difference at the end of the study due to the high imbalance in treatment allocations. In this work we develop an alternative pairwise test for treatment difference on the basis of allocation probabilities of the covariate-adjusted response-adaptive randomization with forward looking Gittins index rule (CARA-FLGI). The performance of the novel test is evaluated in simulations for two-armed studies and then its applications to multi-armed studies is illustrated. The proposed test has markedly improved power over the traditional Fisher exact test when this class of non-myopic response adaptation is used. We also find that the test's power is close to the power of a Fisher exact test under equal randomization.
