Key considerations in the health technology assessment of advanced therapy medicinal products in Scotland, The Netherlands, and England

ten Ham, R. M.T., Frederix, G. W.J., Wu, O. , Goettsch, W., Leufkens, H. G.M., Klungel, O. H. and Hoekman, J. (2022) Key considerations in the health technology assessment of advanced therapy medicinal products in Scotland, The Netherlands, and England. Value in Health, 25(3), pp. 390-399. (doi: 10.1016/j.jval.2021.09.012) (PMID:35227451)

Abstract

Objectives: 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. Methods: 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. Results: 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. Conclusions: 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.

• Item Type: Articles
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Wu, Professor Olivia
• Authors: ten Ham, R. M.T., Frederix, G. W.J., Wu, O., Goettsch, W., Leufkens, H. G.M., Klungel, O. H., and Hoekman, J.
• College/School: College of Medical Veterinary and Life Sciences > School of Health & Wellbeing > Health Economics and Health Technology Assessment
• Journal Name: Value in Health
• Publisher: Elsevier
• ISSN: 1098-3015
• ISSN (Online): 1524-4733
• Published Online: 10 December 2021
• Copyright Holders: Copyright © 2021 ISPOR-The Professional Society for Health Economics and Outcomes Research
• First Published: First published in Value in Health 25(3): 390-399
• Publisher Policy: Reproduced under a Creative Commons License