Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands; Health Economics and Health Technology Assessment, Institute of Health and Wellbeing, University of Glasgow, Glasgow, Scotland, UK.
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands; Julius Centre for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
Value Health. 2022 Mar;25(3):390-399. doi: 10.1016/j.jval.2021.09.012. Epub 2021 Dec 10.
Advanced therapy medicinal products (ATMPs) are highly innovative therapies. Their costs and uncertain value claims have raised concerns among health technology assessment (HTA) bodies and payers. Little is known about how underlying considerations in HTA of ATMPs shape assessment and reimbursement recommendations. We aim to identify and assess key considerations that played a role in HTA of ATMPs underlying reimbursement recommendations.
A review of HTA reports was conducted of all authorized ATMPs in Scotland, The Netherlands, and England. Considerations were extracted and categorized into EUnetHTA Core Model domains. Per jurisdiction, considerations were aggregated and key considerations identified (defined as occurring in >1/assessment per jurisdiction). A narrative analysis was conducted comparing key considerations between jurisdictions and different reimbursement recommendations.
We identified 15 ATMPs and 18 HTA reports. In The Netherlands and England most key considerations were identified in clinical effectiveness (EFF) and cost- and economic effectiveness (ECO) domains. In Scotland, the social aspects domain yielded most key considerations, followed by ECO and EFF. More uncertainty in evidence and assessment outcomes was accepted when orphan or end-of-life criteria were applied. A higher percentage of considerations supporting recommendations were identified for products with positive recommendations compared with restricted and negative recommendations.
This is the first empirical review of HTA's using the EUnetHTA Core Model to identify and structure key considerations retrospectively. It provides insights in supporting and opposing considerations for reimbursement of individual products and differences between jurisdictions. Besides the EFF and ECO domain, the social, ethical, and legal domains seem to bear considerable weight in assessment of ATMPs.
先进治疗药物产品（ATMPs）是高度创新的疗法。其成本和不确定的价值主张引起了健康技术评估（HTA）机构和支付方的关注。对于 HTA 中 ATMP 评估和报销建议所依据的基本考虑因素知之甚少。我们旨在确定和评估在 ATMP 的 HTA 中发挥作用的关键考虑因素，这些因素影响了评估和报销建议。
对苏格兰、荷兰和英格兰所有授权的 ATMP 的 HTA 报告进行了审查。提取考虑因素并将其归入 EUnetHTA 核心模型领域。按管辖范围对考虑因素进行汇总，并确定关键考虑因素（定义为每个管辖范围内出现> 1/次评估）。对不同管辖范围和不同报销建议之间的关键考虑因素进行了叙述性分析比较。
我们确定了 15 种 ATMP 和 18 份 HTA 报告。在荷兰和英格兰，大多数关键考虑因素出现在临床有效性（EFF）和成本效益和经济有效性（ECO）领域。在苏格兰，社会方面的领域产生了最多的关键考虑因素，其次是 ECO 和 EFF。当应用孤儿或生命末期标准时，接受了更多证据和评估结果的不确定性。与限制和负面建议相比，支持建议的考虑因素比例更高。
这是首次使用 EUnetHTA 核心模型对 HTA 进行回顾性识别和构建关键考虑因素的实证审查。它提供了有关支持和反对个别产品报销的考虑因素以及不同管辖范围之间差异的见解。除了 EFF 和 ECO 领域外，社会、伦理和法律领域在 ATMP 的评估中似乎具有相当大的权重。
