European Medicines Agency, 7 Westferry Circus, London, UK.
Nat Rev Drug Discov. 2010 Apr;9(4):277-91. doi: 10.1038/nrd3079. Epub 2010 Feb 26.
Drug regulatory agencies have traditionally assessed the quality, safety and efficacy of drugs, and the current paradigm dictates that a new drug should be licensed when the benefits outweigh the risks. By contrast, third-party payers base their reimbursement decisions predominantly on the health benefits of the drug relative to existing treatment options (termed relative efficacy; RE). Over the past decade, the role of payers has become more prominent, and time-to-market no longer means time-to-licensing but time-to-reimbursement. Companies now have to satisfy the sometimes divergent needs of both regulators and payers, and to address RE during the pre-marketing stages. This article describes the current political background to the RE debate and presents the scientific and methodological challenges as they relate to RE assessment. In addition, we explain the impact of RE on drug development, and speculate on future developments and actions that are likely to be required from key players.
药品监管机构传统上负责评估药品的质量、安全性和疗效，目前的模式规定，当收益超过风险时，新药应获得许可。相比之下，第三方支付者主要根据药物相对于现有治疗选择的健康益处来做出报销决定（称为相对疗效；RE）。在过去的十年中，支付者的作用变得更加突出，上市时间不再意味着获得许可的时间，而是获得报销的时间。现在，公司必须满足监管机构和支付者有时不同的需求，并在上市前阶段解决 RE 问题。本文描述了 RE 辩论的当前政治背景，并介绍了与 RE 评估相关的科学和方法学挑战。此外，我们解释了 RE 对药物开发的影响，并推测关键参与者可能需要采取的未来发展和行动。
