Department of Gastrointestinal Medical Oncology, Investigational Cancer Therapeutics, and Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA.
Clin Cancer Res. 2013 Aug 15;19(16):4305-8. doi: 10.1158/1078-0432.CCR-13-1428. Epub 2013 Jul 5.
Recent advances in biotechnology have led to discoveries resulting in major improvements in the therapy of refractory malignancies, although most advanced cancers remain incurable. Thus, there is global consensus around the need to streamline the drug approval process for effective agents. Accelerated Approval and Breakthrough Therapy Designation hold the promise of making new treatments available sooner through the use of smaller studies using intermediate endpoints. Here, we consider the inherent limitations of smaller studies and discuss the strategies for hastening oncology drug development while maintaining high-efficacy standards.
生物技术的最新进展带来了重大发现，使难治性恶性肿瘤的治疗得到了显著改善，尽管大多数晚期癌症仍然无法治愈。因此，全球范围内都达成了共识，需要简化药物审批流程，以确保有效药物的批准。加速审批和突破性治疗指定有望通过使用较小的研究和中间终点来更快地提供新的治疗方法。在这里，我们考虑了较小研究的固有局限性，并讨论了在保持高效标准的同时加快肿瘤药物开发的策略。
