A systematic review of economic evaluations of advanced therapy medicinal products

• Published in: British journal of clinical pharmacology 2021-06, Vol.87 (6), p.2428-2443
• Main Authors: Lloyd‐Williams, Huw, Hughes, Dyfrig A.
• Format: Article
• Language: English
• Subjects: Advanced Therapy Medicinal Products ‐ Systematic Review and Meta‐analysis; cell therapy; cost‐effectiveness; gene therapy; health technology assessment; regenerative medicine; Systematic Review and Meta‐analysis
• ISSN: 0306-5251; 1365-2125
• DOI: 10.1111/bcp.14275

Aims 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. Methods 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 , and full texts reviewed. Study findings were appraised critically. Results 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. Conclusion 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.