A systematic review of economic evaluations of advanced therapy medicinal products.
Br J Clin Pharmacol
; 87(6): 2428-2443, 2021 06.
Article
em En
| MEDLINE
| ID: mdl-32154598
ABSTRACT
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 abstract, 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.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Avaliação da Tecnologia Biomédica
Tipo de estudo:
Health_economic_evaluation
/
Health_technology_assessment
/
Prognostic_studies
/
Systematic_reviews
Idioma:
En
Revista:
Br J Clin Pharmacol
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Reino Unido