Defining the value of treatments of rare pediatric conditions
Menée dans un contexte américain à partir de données de 3 études incluant des enfants et jeunes adultes (âgés de moins de 25 ans) atteints d'une leucémie lymphoblastique aiguë à cellules B réfractaire ou récidivante, cette étude analyse le rapport coût-efficacité d'une immunothérapie à base de lymphocytes CAR-T (tisagenlecleucel)
In this issue of JAMA Pediatrics, Whittington et al1 present a comprehensive analysis of the projected long-term outcomes and cost-effectiveness of a chimeric antigen receptor T-cell therapy, tisagenlecleucel, for relapsed or refractory leukemia in children; this is the first gene therapy approved by the US Food and Drug Administration. The study uses a modeling approach and highlights 2 critical issues in defining value for an innovative treatment with limited evidence: the methodological challenges of applying economic evaluation techniques to rare pediatric conditions and reliably assessing value and affordability for very costly new treatments (in this case, $475 000 per patient).
JAMA Pediatrics , éditorial, 2017