Genentech, San Francisco, California, USA.
Clin Cancer Res. 2013 Aug 15;19(16):4297-304. doi: 10.1158/1078-0432.CCR-13-0523. Epub 2013 May 29.
In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early-phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the U.S. Food and Drug Administration (FDA) and sponsor would collaborate to determine the best path forward to abbreviate the traditional three-phase approach to drug development. The breakthrough legislation requires that an FDA guidance be drafted that details specific requirements of the bill to aid FDA in implementing requirements of the Act. In this article, we have proposed criteria to define a product as a Breakthrough Therapy, and discussed critical components of the development process that would require flexibility in order to enable expedited development of a Breakthrough Therapy.
2012 年 7 月，美国国会通过了《食品药品监督管理局安全与创新法案》（FDASIA）。推进突破性治疗药物法案被纳入 FDASIA 的一个标题，以加快新的、有潜力的“突破性”药物或治疗方法的临床开发，这些药物或治疗方法在早期研究中显示出显著的反应。利用这一监管途径，一旦有前途的新药候选药物被指定为“突破性治疗药物”，美国食品和药物管理局（FDA）和赞助商将合作确定最佳前进道路，以缩短传统的药物开发三阶段方法。突破性立法要求起草一份 FDA 指南，详细说明法案的具体要求，以帮助 FDA 实施该法案的要求。在本文中，我们提出了定义突破性治疗药物的标准，并讨论了开发过程中需要灵活性的关键组成部分，以便能够加快突破性治疗药物的开发。
