A systematic review of economic evaluations of advanced therapy medicinal products

Lloyd-Williams, Huw and Hughes, Dyfrig A ORCID: 0000-0001-8247-7459
(2021) A systematic review of economic evaluations of advanced therapy medicinal products. BRITISH JOURNAL OF CLINICAL PHARMACOLOGY, 87 (6). pp. 2428-2443.

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<h4>Aims</h4>Advanced therapy medicinal products (ATMPs) represent a new category of medicinal products with a potential for transformative improvements in health outcomes but at exceptionally high prices. Routine adoption of ATMPs requires robust evidence of their cost-effectiveness.<h4>Methods</h4>A systematic literature review of economic evaluations of ATMPs, including gene therapies, somatic cell therapies and tissue-engineered products, was conducted. Literature was searched using MedLine, Embase, PubMed, Cochrane Register, the NHS Economic Evaluation Database and the grey literature of health technology assessment organisations with search terms relating to ATMPs and economic evaluations. Titles were screened independently by 2 reviewers. Articles deemed to meet the inclusion criteria were screened independently on abstract, and full texts reviewed. Study findings were appraised critically.<h4>Results</h4>4514 articles were identified, of which 23 met the inclusion criteria. There was some evidence supporting the cost-effectiveness of: chimeric antigen receptor T-cell therapy axicabtagene-ciloleucel (Yescarta), embryonic neural stem cells, tumour infiltrating lymphocytes, in vitro expanded myoblast, autologous chondrocyte implantation, ex vivo gene therapy (Strimvelis) and voretigene neparvovec (Luxturna). However, estimates of cost-effectiveness were associated with significant uncertainty and high likelihood of bias, resulting from largely unknown long-term outcomes, a paucity of evidence on health state utilities and extensive modelling assumptions.<h4>Conclusion</h4>There are critical limitations to the economic evidence for ATMPs, most notably in relation to evidence on the durability of treatment effect, and the reliability of opinion-based assumptions necessary when evidence is absent.

Item Type: Article
Uncontrolled Keywords: cell therapy, cost-effectiveness, gene therapy, health technology assessment, regenerative medicine
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Systems, Molecular and Integrative Biology
Depositing User: Symplectic Admin
Date Deposited: 15 Jul 2021 09:01
Last Modified: 18 Jan 2023 21:35
DOI: 10.1111/bcp.14275
Open Access URL: https://doi.org/10.1111/bcp.14275
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URI: https://livrepository.liverpool.ac.uk/id/eprint/3130128