ECONOMIC ORPHANS? THE PREVALENCE OF CHILD-SPECIFIC UTILITIES IN NICE APPRAISALS FOR PAEDIATRIC INDICATIONS
Author(s)
Montgomery S, Hassan M, Kusel J
Costello Medical Consulting Ltd., Cambridge, UK
Presentation Documents
OBJECTIVES Children have been termed “therapeutic orphans” due to the paucity of age-specific therapeutic data. Here we review the extent to which utility data derived from under-18s were used to inform National Institute for Health and Care Excellence (NICE) Technology Appraisals (TAs) providing cost-effectiveness guidance in paediatric indications, in line with the NICE reference case. METHODS All 311 published TAs up to April 2014 were initially sifted to identify therapeutic recommendations for children. Identified TAs were reviewed to determine if a cost-utility analysis (CUA) was performed. For each CUA, the published TA along with the manufacturer’s submission (single TAs) or the assessment report (multiple TAs) were examined to determine the origin of the utilities used. RESULTS Of 35 published TAs reviewed, 27 analysed cost-per-QALY and made recommendations for treatment of under-18s. Of these, 17 used adult utilities, 1 of which attempted to adjust the adult values for children; 3 considered child and adult populations as one, with child-derived data used within the overall model inputs for the whole population, 1 of which adjusted both child and adult utilities by age. Only 6 studies used child-specific utilities: 1 assumed a specified change from treatment on a generic QoL instrument, 2 used parent-reported utilities on a generic QoL instrument, 1 used parent-reported utilities mapped from a disease-specific scale and 2 used child-reported utilities mapped from a disease-specific scale. One MTA contained diverging submissions, 1 adult-derived and 1 child-reported. No trends over time in the types of utilities used were apparent from visual examination of the results. CONCLUSIONS Despite NICE’s reference case specifying that utilities should be measured in the population in question, children may also be termed “economic orphans” with the majority of cost-utility submissions applying adult-derived utilities to paediatric indications and no trend away from this apparent over time.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
QA1
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Multiple Diseases, Pediatrics