This article investigates patterns of pharmaceutical development activity around the 2012 creation of the FDA’s Breakthrough Therapy Designation (BTD). The BTD introduced regulatory flexibility and support to avoid ethical challenges created by experimental therapies of exceptional performance in early stage clinical trials. We argue that the program’s design indirectly created substantial incentives for the industry to pursue the designation. Consistent with this hypothesis, our evidence links the creation of the program with a substantial increase in the number of new drug indications entering the clinical trial process. This surge in introductions has resulted in a discernible increase in approvals, which has manifested with a lag and may strengthen in the future. Countering theoretical predictions, BTD incentives have not led to increased risk taking in project selection.

中文翻译：

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规范伦理实验：突破性疗法指定对药物研发的影响

本文调查了 2012 年 FDA 突破性疗法认定 (BTD) 设立以来的药物开发活动模式。BTD 引入了监管灵活性和支持，以避免早期临床试验中表现出色的实验疗法带来的伦理挑战。我们认为，该计划的设计间接地为该行业追求这一称号创造了巨大的动力。与这一假设相一致的是，我们的证据表明该计划的创建与进入临床试验过程的新药适应症数量的大幅增加有关。引进数量的激增导致批准数量明显增加，但这种增加存在滞后性，并且在未来可能会加强。与理论预测相反，BTD 激励措施并未导致项目选择中的风险增加。