• Defining the Value of Treatments of Rare Pediatric Conditions

    12 days ago - By JAMA Pediatrics

    In this issue of JAMA Pediatrics, Whittington et al 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...
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