Medicines Evaluation Board, Utrecht, The Netherlands.
Medicines Evaluation Board, Utrecht, The Netherlands.
Drug Discov Today. 2020 Sep;25(9):1580-1584. doi: 10.1016/j.drudis.2020.06.009. Epub 2020 Jun 17.
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）有四个加速审批项目，包括突破性疗法认定（BTD）。最近，这个项目受到了批评。在这篇特写中，我们根据一种经过验证的衡量临床获益的工具的结果，确定 BTD 肿瘤药物是否真的是一种突破。我们发现，只有少数药物可能是突破，这表明 BTD 计划的成功率有些低。尽管如此，我们认为快速药物审批程序在当前的监管实践中还是有一席之地的，应该进一步探索改进这些程序的必要措施，尤其是考虑到癌症患者仍存在未满足的医疗需求。
