The prevalence of child-specific utilities in NICE appraisals for paediatric indications: rise of the economic orphans?

摘要

Children are not mini-adults, and thus require studies to be conducted in the population of interest to inform decisions about their care. The paucity of such studies for clinical efficacy lead them to be termed therapeutic orphans'. Following the introduction of the fourth hurdle' of reimbursement approval on the basis of cost-utility analysis, utility data is now a key requirement for patients to access treatments in England and many other countries. This special report considers whether a paucity of utility valuation studies in children may have made them economic orphans' as well and presents results of a review of NICE appraisals as a window on this problem over time.