1 Baylor Scott & White Health, Temple, Texas, and University of Texas, Health Outcomes and Pharmacy Practice Division, Austin.
J Manag Care Spec Pharm. 2018 Jul;24(7):677-681. doi: 10.18553/jmcp.2018.24.7.677.
Enacted in December 2016, the 21st Century Cures Act is a pivotal piece of legislation that will influence the FDA drug and device approval process for the foreseeable future. Although this legislation received overwhelming support by members of Congress for much needed budgetary increases for the FDA and other national health organizations, there is much controversy over certain provisions that potentially diminish the robustness of the evidence base required for drug and medical device approvals. This article discusses the key provisions from "Title III - Development" under "Division A - 21st Century Cures" related to drug development and, specifically, explores those aspects that address patient-focused drug development, use of surrogate endpoints in clinical trials, modernization of trial design, and use of real-world evidence for decision making and health care economic information. In addition, specific legislation on regulatory changes is discussed pertaining to antimicrobial stewardship. Essentially, summarized interpretations are presented here of the provisions covering the aforementioned topics, along with insights into potential intended and unintended consequences for U.S. health care payers, health care providers, government entities, and product manufacturers.
No outside funding supported this writing. Goble is completing fellowship training sponsored by Novartis Pharmaceuticals.
21 世纪治愈法案于 2016 年 12 月颁布，是一项具有重大意义的法规，将在可预见的未来影响 FDA 的药品和器械审批流程。尽管这项立法得到了国会议员的压倒性支持，为 FDA 和其他国家卫生组织提供了急需的预算增加，但其中一些条款存在争议，这些条款可能会削弱药品和医疗器械批准所需的证据基础的稳健性。本文讨论了“第 21 世纪治愈法案”的“发展”标题下“A 部分-21 世纪治愈”中的关键条款，这些条款与药物开发有关，特别是探讨了那些关注患者的药物开发、临床试验中替代终点的使用、试验设计的现代化以及真实世界证据在决策和医疗保健经济信息中的应用。此外，还讨论了与抗菌药物管理有关的监管改革的具体立法。本质上，本文对涵盖上述主题的条款进行了总结性解释，并深入探讨了这些条款对美国医疗保健支付者、医疗保健提供者、政府实体和产品制造商可能产生的预期和非预期影响。
本文写作没有外部资金支持。Goble 正在接受诺华制药公司赞助的奖学金培训。
