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).
Prosser LA. Defining the Value of Treatments of Rare Pediatric Conditions. JAMA Pediatr. 2018;172(12):1123–1124. doi:10.1001/jamapediatrics.2018.3343
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