Xia Yi, Cui Lu, Yang Bo
a Eisai, Inc. , Woodcliff Lake , New Jersey , USA.
J Biopharm Stat. 2014;24(5):1102-14. doi: 10.1080/10543406.2014.931410.
Accelerated approval by the Food and Drug Administration (FDA), under the agency's Fast Track review designation, allows early approval of drugs to treat serious diseases and fill an unmet medical need based on a surrogate endpoint. In May 2012, FDA issued a draft Guidance for Industry on the accelerated approval of breast cancer drugs based on the surrogate endpoint "pathologic complete response" (pCR). The research reported in this article investigates potential issues in designing clinical studies for pCR-based accelerated approval. The correlation between pCR and long-term survival was investigated. Two sample comparisons based on a conditional survival model under different assumptions were performed and are discussed along with simulation results. The findings from this research may shed some light on the implementation of the FDA draft guidance.
美国食品药品监督管理局（FDA）根据其快速通道审评指定进行的加速批准，允许基于替代终点对治疗严重疾病且满足未满足医疗需求的药物进行早期批准。2012年5月，FDA发布了一份关于基于替代终点“病理完全缓解”（pCR）加速批准乳腺癌药物的行业指南草案。本文报道的研究调查了基于pCR的加速批准临床研究设计中的潜在问题。研究了pCR与长期生存之间的相关性。基于不同假设在条件生存模型下进行了两个样本比较，并结合模拟结果进行了讨论。本研究结果可能为FDA指南草案的实施提供一些启示。
