Innovation Studies Group, Copernicus Institute of Sustainable Development, Faculty of Geosciences, Utrecht University, the Netherlands.
Innovation Studies Group, Copernicus Institute of Sustainable Development, Faculty of Geosciences, Utrecht University, the Netherlands.
Soc Sci Med. 2019 Feb;222:76-83. doi: 10.1016/j.socscimed.2018.12.025. Epub 2018 Dec 19.
Drug regulatory agencies around the world increasingly implement expedited regulatory pathways allowing for approval of medicines that intend to address unmet medical needs based on lower evidentiary standards than would be conventionally required. Few studies have investigated how companies and regulators utilise these pathways. We therefore conducted a longitudinal analysis of the emergence and implementation of the conditional marketing authorisation (CMA) instrument in the European Union. Drawing on archival documents, procedural data and interviews, we show that there was substantial ambiguity among regulators and companies about how to strike a new balance between evidentiary requirements and patient needs. As ambiguities were left unresolved, parties became reluctant to use CMA and in the majority of procedures did not use the pathway in a prospectively planned fashion. Rather, CMA became an option for regulators and companies to apply when submitted data were not strong enough to justify standard approval. Particularly, incumbent companies profited from this. The results stress the challenges of realising institutional change in drug regulation by showing how interest-driven actors can act upon ambiguities in attempts to shape regulatory outcomes and stretch rule interpretations.
世界各地的药品监管机构越来越多地实施加速监管途径，允许根据低于传统要求的证据标准批准旨在满足未满足的医疗需求的药物。很少有研究调查公司和监管机构如何利用这些途径。因此，我们对欧盟有条件营销授权 (CMA) 工具的出现和实施进行了纵向分析。我们利用档案文件、程序数据和访谈表明，监管机构和公司在如何在证据要求和患者需求之间取得新的平衡方面存在很大的模糊性。由于未解决的模糊性，各方不愿意使用 CMA，并且在大多数程序中没有以前瞻性计划的方式使用该途径。相反，当提交的数据不够强以证明标准批准合理时，CMA 成为监管机构和公司的一种选择。特别是，现有公司从中受益。研究结果强调了通过展示利益驱动的行为者如何在试图塑造监管结果和扩大规则解释时利用模糊性来实现药物监管体制变革的挑战。
