Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY.
Delphi Diagnostics, Baltimore, MD.
Med Care. 2022 Apr 1;60(4):287-293. doi: 10.1097/MLR.0000000000001692.
The Prescription Drug User Fee Act (PDUFA) is due for reauthorization in 2022. Beyond creating the user fee program which now generates a majority of the Food and Drug Administration (FDA) Human Drugs Program budget, PDUFA has made numerous additional changes to FDA policy during its 29-year history. FDA's budgetary dependence on user fees may advantage the industry in negotiating favorable policy changes through PDUFA.
The full texts of all prior PDUFA reauthorization bills and all submitted public comments and meeting minutes for the 2022 reauthorization were reviewed. Provisions affecting FDA regulatory authority and processes were identified.
PDUFA legislation has instituted a broad range of changes to FDA policy, including evidentiary standards for drug approval, accelerated pathways for approval, industry involvement in FDA decision-making, rules regarding industry information dissemination to providers, and market entry of generic drugs. Negotiations over the 2022 reauthorization suggest that industry priorities include increased application of real-world evidence, regulatory certainty, and increased communication between FDA and industry during the drug application process.
The need for PDUFA reauthorization every 5 years has created a recurring legislative vehicle through which far-ranging changes to FDA have been enacted, reshaping the agency's interactions and relationship with the regulated industry. The majority of policy changes enacted through PDUFA legislation have favored industry through decreasing regulatory standards, shortening approval times, and increasing industry involvement in FDA decision-making. FDA's budgetary dependence on the industry, the urgency of each PDUFA reauthorization's passage to maintain uninterrupted funding, and the industry's required participation in PDUFA negotiations may advantage the industry.
《处方药使用者付费法案》（PDUFA）将于 2022 年重新授权。除了创建目前为食品和药物管理局（FDA）人类药物计划预算提供大部分资金的用户收费计划外，PDUFA 在其 29 年的历史中还对 FDA 政策进行了许多其他更改。FDA 对用户收费的预算依赖可能使行业在通过 PDUFA 协商有利的政策变更方面具有优势。
审查了所有先前的 PDUFA 重新授权法案的全文以及 2022 年重新授权的所有提交的公开意见和会议记录。确定了影响 FDA 监管权力和程序的规定。
PDUFA 立法对 FDA 政策进行了广泛的更改，包括药物批准的证据标准、批准的加速途径、行业参与 FDA 决策、关于行业向提供者传播信息的规则以及仿制药的市场准入。关于 2022 年重新授权的谈判表明，行业的优先事项包括增加实际证据的应用、监管确定性以及在药物申请过程中增加 FDA 和行业之间的沟通。
每 5 年需要重新授权 PDUFA，这为立法机构提供了一个经常性的立法手段，通过该手段对 FDA 进行了广泛的更改，重塑了该机构与受监管行业的互动和关系。通过 PDUFA 立法制定的大多数政策变更都通过降低监管标准、缩短批准时间以及增加行业对 FDA 决策的参与，使行业受益。FDA 对行业的预算依赖、每次 PDUFA 重新授权的紧迫性以维持不间断的资金以及行业在 PDUFA 谈判中的必要参与可能使行业受益。
