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Defining the Value of Treatments of Rare Pediatric Conditions

Defining the Value of Treatments of Rare Pediatric Conditions Opinion EDITORIAL Lisa A. Prosser, PhD In this issue of JAMA Pediatrics, Whittington et al present a bining these parameters into a 2-way sensitivity analysis would comprehensive analysis of the projected long-term outcomes have provided additional information for decision makers. Using and cost-effectiveness of a chimeric antigen receptor T-cell an alternate functional form yielded slightly less favorable therapy, tisagenlecleucel, for results. Extensive scenario and uncertainty analyses should relapsed or refractory leuke- become standard practice for cost-effectiveness analyses of Related article page 1161 mia in children; this is the first pediatric rare conditions to fully understand the influence of the gene therapy approved by the US Food and Drug Administra- timing of treatment, realization of health benefits, and other tion. The study uses a modeling approach and highlights 2 criti- parameters associated with high uncertainty. cal issues in defining value for an innovative treatment with lim- The choice of a comparator is essential to the accurate esti- ited evidence: the methodological challenges of applying mation of cost-effectiveness. The selection of only 1 compara- economic evaluation techniques to rare pediatric conditions and tor,clofarabine,whenothercomparators,suchasblinatumomab, reliably assessing value and affordability for very costly new are available, is a distinct limitation of the study http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Pediatrics American Medical Association

Defining the Value of Treatments of Rare Pediatric Conditions

JAMA Pediatrics , Volume 172 (12) – Dec 8, 2018

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Publisher
American Medical Association
Copyright
Copyright 2018 American Medical Association. All Rights Reserved.
ISSN
2168-6203
eISSN
2168-6211
DOI
10.1001/jamapediatrics.2018.3343
Publisher site
See Article on Publisher Site

Abstract

Opinion EDITORIAL Lisa A. Prosser, PhD In this issue of JAMA Pediatrics, Whittington et al present a bining these parameters into a 2-way sensitivity analysis would comprehensive analysis of the projected long-term outcomes have provided additional information for decision makers. Using and cost-effectiveness of a chimeric antigen receptor T-cell an alternate functional form yielded slightly less favorable therapy, tisagenlecleucel, for results. Extensive scenario and uncertainty analyses should relapsed or refractory leuke- become standard practice for cost-effectiveness analyses of Related article page 1161 mia in children; this is the first pediatric rare conditions to fully understand the influence of the gene therapy approved by the US Food and Drug Administra- timing of treatment, realization of health benefits, and other tion. The study uses a modeling approach and highlights 2 criti- parameters associated with high uncertainty. cal issues in defining value for an innovative treatment with lim- The choice of a comparator is essential to the accurate esti- ited evidence: the methodological challenges of applying mation of cost-effectiveness. The selection of only 1 compara- economic evaluation techniques to rare pediatric conditions and tor,clofarabine,whenothercomparators,suchasblinatumomab, reliably assessing value and affordability for very costly new are available, is a distinct limitation of the study

Journal

JAMA PediatricsAmerican Medical Association

Published: Dec 8, 2018

References