10 March 2020 - Advanced therapy medicinal products represent a new category of medicinal products with a potential for transformative improvements in health outcomes but at exceptionally high prices. Routine adoption requires robust evidence of their cost‐effectiveness.
A systematic literature review of economic evaluations of advanced therapy medicinal products, including gene therapies, somatic cell therapies, and tissue‐engineered products, was conducted.
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).