Department of Pharmaceutical Sciences, University of Milan, G. Colombo 71, 20133 Milan, Italy.
Department of Pharmaceutical Sciences, University of Milan, G. Colombo 71, 20133 Milan, Italy.
Int J Pharm. 2023 Jul 25;642:123193. doi: 10.1016/j.ijpharm.2023.123193. Epub 2023 Jun 30.
For unmet medical needs, the European Union has established fast-track regulatory pathways for ensuring patients' access to essential treatments. It is the case of the Conditional Marketing Authorisation (CMA) and the Authorisation under "Exceptional Circumstances" (EXC), which can be granted even if the clinical part of a medicinal product's dossier is not yet complete. The article aims to discuss the peculiarity of such regulatory pathways and assess the impact of their application on products' market access and penetration. A review of the regulatory history of medicines authorised with EXC or CMA has been performed on European Institutional databases (e.g., EMA portal, Union Register). Excluding vaccines, 71 CMAs and 51 EXCs were granted in the EU from 2002 and 2006, respectively, to 2022. Most CMAs have been released for the treatment of different types of tumours, while most of EXCs for alimentary tract and metabolism diseases, especially in the paediatric population, addressing unmet medical needs. Therefore, both regulatory pathways are effective for placing on the market essential medicines, preserving the initial positive benefit-risk balance. However, on average, CMAs are converted into "normal" authorisations only after a time which is significantly longer than the provided one-year renewal period, suggesting that such a regulatory pathway is still far from optimized.
对于未满足的医疗需求，欧盟建立了快速监管途径，以确保患者能够获得基本治疗。这就是有条件的上市许可（Conditional Marketing Authorization，CMA）和“特殊情况下”的上市许可（Authorization under "Exceptional Circumstances"，EXC），即使药品档案的临床部分尚未完成，也可以授予这些许可。本文旨在讨论这些监管途径的特殊性，并评估其应用对产品市场准入和渗透的影响。在欧洲机构数据库（如 EMA 门户、联盟登记册）上对通过 EXC 或 CMA 获得许可的药物的监管历史进行了审查。不包括疫苗，从 2002 年到 2006 年，欧盟分别批准了 71 项 CMA 和 51 项 EXC，截至 2022 年。大多数 CMA 是为治疗不同类型的肿瘤而发放的，而大多数 EXC 是为治疗胃肠道和代谢疾病而发放的，特别是在儿科人群中，以满足未满足的医疗需求。因此，这两种监管途径都能有效地将基本药物投放市场，保持最初的积极风险效益平衡。然而，平均而言，CMA 仅在经过明显长于一年的续期期限后才转换为“正常”许可，这表明这种监管途径仍远未得到优化。
