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

ABSTRACT

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.

KEYWORDS:

• Paediatrics
• quality of life
• utility
• cost-utility analysis
• NICE

Acknowledgments

The authors wish to thank their former colleague Marwah Hassan for her assistance with the preparation of an initial study of the use of child-specific utilities in NICE appraisals presented at the ISPOR European Congress 2014, from which this Special Report subsequently developed.

Financial & competing interests disclosure

SM Montgomery and JM Kusel are both employees of Costello Medical Consulting Ltd, Cambridge, UK. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.