European Medicines Agency, London, UK.
Clin Pharmacol Ther. 2012 Mar;91(3):426-37. doi: 10.1038/clpt.2011.345. Epub 2012 Feb 15.
Traditional drug licensing approaches are based on binary decisions. At the moment of licensing, an experimental therapy is presumptively transformed into a fully vetted, safe, efficacious therapy. By contrast, adaptive licensing (AL) approaches are based on stepwise learning under conditions of acknowledged uncertainty, with iterative phases of data gathering and regulatory evaluation. This approach allows approval to align more closely with patient needs for timely access to new technologies and for data to inform medical decisions. The concept of AL embraces a range of perspectives. Some see AL as an evolutionary step, extending elements that are now in place. Others envision a transformative framework that may require legislative action before implementation. This article summarizes recent AL proposals; discusses how proposals might be translated into practice, with illustrations in different therapeutic areas; and identifies unresolved issues to inform decisions on the design and implementation of AL.
传统的药物许可方法基于二元决策。在许可的那一刻，实验性疗法被假定转化为经过全面审查、安全、有效的疗法。相比之下，适应性许可（AL）方法基于在公认的不确定性条件下的逐步学习，具有数据收集和监管评估的迭代阶段。这种方法允许批准更紧密地符合患者对及时获得新技术的需求，并为医疗决策提供数据。AL 的概念包含了一系列观点。一些人认为 AL 是一个进化的步骤，扩展了现有的元素。另一些人则设想了一个可能需要立法行动才能实施的变革性框架。本文总结了最近的 AL 提案；讨论了如何将提案转化为实践，不同治疗领域的实例；并确定了未解决的问题，以告知关于 AL 的设计和实施的决策。
