Key Considerations in the Health Technology Assessment of Advanced Therapy Medicinal Products in Scotland, The Netherlands, and England

• Published in: Value in health 2022-03, Vol.25 (3), p.390-399
• Main Authors: ten Ham, Renske M.T., Frederix, Geert W.J., Wu, Olivia, Goettsch, Wim, Leufkens, Hubert G.M., Klungel, Olaf H., Hoekman, Jarno
• Format: Article
• Language: English
• Subjects: advanced therapies; advanced therapy medicinal products; cell therapy; Cost-Benefit Analysis - methods; Cost-Benefit Analysis - statistics & numerical data; Ethical Analysis; Europe; gene therapy; health technology assessment; Humans; Insurance, Health, Reimbursement - economics; Retrospective Studies; Technology Assessment, Biomedical - methods; Technology Assessment, Biomedical - statistics & numerical data; Therapies, Investigational - economics; Therapies, Investigational - ethics; Uncertainty
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2021.09.012

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. •Advanced therapy medicinal products (ATMPs) are highly innovative therapies. Their high costs and uncertain value claims raise concerns among health technology assessment bodies and payers, yet little is known about the experiences with health technology assessment of ATMPs.•Key considerations underlying the reimbursement recommendations of ATMPs were identified and assessed in Scotland, The Netherlands, and England using the EUnetHTA Core Model.•This study provides insights in supporting and opposing considerations for individual products and on a member state level. Besides the therapeutic eff