The US Food and Drug Administration (FDA) has four expedited programs, including the breakthrough therapy designation (BTD). Recently, this program has been criticized. In this feature, we determine whether BTD oncology drugs were truly a breakthrough, based on the outcome of a validated instrument to measure clinical benefit. We find that only a few drugs were likely a breakthrough, indicating that the success rate of the BTD program is somewhat low. Despite this, we believe that programs for fast drug approval do have a place in the current regulatory practice and that the necessary efforts for their improvement should be further explored, especially considering the remaining unmet medical need for patients with cancer.

美国食品和药物管理局 (FDA) 有四个加速计划，包括突破性疗法指定 (BTD)。最近，这个节目受到了批评。在此功能中，我们根据衡量临床益处的经过验证的仪器的结果来确定 BTD 肿瘤药物是否真正具有突破性。我们发现只有少数药物有可能取得突破，说明BTD项目的成功率偏低。尽管如此，我们认为快速药物批准计划在当前的监管实践中确实占有一席之地，应该进一步探索对其改进的必要努力，特别是考虑到癌症患者仍有未满足的医疗需求。