A limited number of gene therapy medicinal products (GTMPs) have received marketing authorization (MA), of which some have been withdrawn, and even less have gained reimbursement. Many challenges that complicate GTMP market access can occur across multiple jurisdictions and decision-making contexts, but some reimbursement challenges are specific to jurisdictions. The importance of these challenges vary according to the specific therapy being developed, the country where market access is sought, and the efforts made by developers, regulators and payers to implement solutions to overcome these barriers. This review could alert developers to challenges associated with GTMP MA and how to address them.

有限数量的基因治疗药物（GTMP）获得了上市许可（MA），其中一些已被撤回，获得报销的更少。许多使 GTMP 市场准入复杂化的挑战可能发生在多个司法管辖区和决策环境中，但一些报销挑战是特定于司法管辖区的。这些挑战的重要性因正在开发的特定疗法、寻求市场准入的国家以及开发商、监管机构和付款人为实施解决方案以克服这些障碍而做出的努力而异。该审查可以提醒开发人员注意与 GTMP MA 相关的挑战以及如何解决这些挑战。