Department of Pharmaceutical Economic and Policy, Chapman University School of Pharmacy, Irvine, CA, USA.
Economic Science Institute, Argyros School of Business and Economics, Chapman University, Orange, CA, USA.
Sci Rep. 2024 Feb 9;14(1):3325. doi: 10.1038/s41598-024-53554-7.
U.S. laws enacted since 1983 have aimed to enhance the development and marketing of new pharmaceutical products. We thoroughly characterized all new molecular entities, therapeutic biologics, and gene and cell therapies approved by the US Food and Drug Administration (FDA) during the period 1980-2022 in the context of these laws and regulations. Throughout the study period, the FDA approved 1355 new pharmaceutical products. The median FDA review time decreased from 26.6 months prior to the Prescription Drug User Fee Act (1992), which authorized the FDA to collect fees from drug companies to 9.9 months after the Food and Drug Administration Safety and Innovation Act (2012), which created new designations that eliminated the requirement for evidence of added therapeutic benefit for FDA expedited drug review. The greatest increase in approvals occurred in antineoplastic and immunomodulating drugs, biologics, and orphan drugs. More than half of new drug approvals benefited from regulatory designations and pathways that did not require addressing unmet medical needs or demonstrating therapeutic benefit over available alternatives. The legislative goal of bringing more drugs to the market faster has been achieved. Further studies are needed to determine the therapeutic value to patients of new drugs approved using expedited approval pathways.
自 1983 年以来，美国颁布的法律旨在促进新药品的开发和营销。我们在这些法律和法规的背景下，全面描述了 1980 年至 2022 年期间，美国食品和药物管理局（FDA）批准的所有新的分子实体、治疗性生物制剂以及基因和细胞疗法。在整个研究期间，FDA 批准了 1355 种新的药品。FDA 审查时间的中位数从《处方药使用者付费法案》（1992 年）之前的 26.6 个月下降到《食品和药物管理局安全和创新法案》（2012 年）之后的 9.9 个月，该法案创建了新的指定，取消了对 FDA 加速药物审查的证据要求，即需要证明附加治疗益处。批准数量最多的是抗肿瘤和免疫调节药物、生物制剂和孤儿药。超过一半的新药批准得益于监管指定和途径，这些指定和途径不需要解决未满足的医疗需求或证明与现有替代药物相比具有治疗益处。更快地将更多药物推向市场的立法目标已经实现。需要进一步研究使用加速批准途径批准的新药对患者的治疗价值。
