TY - JOUR

T1 - A Systematic Review of Economic Evaluations of Advanced Therapy Medicinal Products

AU - Lloyd Williams, Huw

AU - Hughes, Dyfrig

N1 - Innovate UK. Grant Number: WBS‐ITT‐36837

PY - 2021/6

Y1 - 2021/6

N2 - BACKGROUND 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 HTA organisations with search terms relating to ATMPs and economic evaluations. Titles were screened independently by two reviewers. Articles deemed to meet the inclusion criteria were screened independently on abstract, and full texts reviewed. Study findings were appraised critically.RESULTS 4,514 articles were identified, of which 23 met the inclusion criteria. There was some evidence supporting the cost-effectiveness of: CAR 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.CONCLUSIONS 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.

AB - BACKGROUND 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 HTA organisations with search terms relating to ATMPs and economic evaluations. Titles were screened independently by two reviewers. Articles deemed to meet the inclusion criteria were screened independently on abstract, and full texts reviewed. Study findings were appraised critically.RESULTS 4,514 articles were identified, of which 23 met the inclusion criteria. There was some evidence supporting the cost-effectiveness of: CAR 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.CONCLUSIONS 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.

KW - cell therapy

KW - cost-effectiveness

KW - gene therapy

KW - health technology assessment

KW - regenerative medicine

U2 - 10.1111/bcp.14275

DO - 10.1111/bcp.14275

M3 - Review article

VL - 87

SP - 2428

EP - 2443

JO - British Journal of Clinical Pharmacology

JF - British Journal of Clinical Pharmacology

SN - 0306-5251

IS - 6

ER -