ASSESSING METHODOLOGIES FOR HEALTH STATE UTILITIES IN PAEDIATRIC INDICATIONS FOR COST-UTILITY ANALYSES: REVIEW OF NICE TECHNOLOGY APPRAISALS FOR PAEDIATRIC INDICATIONS

Choosing an appropriate methodology for health state utilities is imperative when conducting a cost-utility analysis (CUA). This remains a challenge particularly in paediatric populations where direct utilities (from trials) are often not available. The objective of this review was to assess the methods used for obtaining utilities in prior National Institute for Health and Care Excellence (NICE), UK technology appraisals (TAs) submitted for paediatric indications. A search was conducted on the NICE website in December 2016 to identify the TAs for paediatric indications. TAs which included patients aged <18 years, irrespective of the indication, were included in the review. Revised TAs for which full information was not available were excluded. Data pertaining to basic information about the TA (e.g. intervention, indication), age, and methodology used for measuring utilities, and comments given by Evidence Review Group (ERG) were extracted in a pre-designed extraction grid. Out of a total of 405 TAs, data from 24 TAs for paediatric indications were identified. EQ-5D was used in 11 out of 24 submissions for measuring utilities and mapping from other non-preference based measures to EQ-5D was done in two submissions. ERG did not criticize the use of EQ-5D even though it is not recommended for use in paediatric populations. For submission of CUAs to Health Technology Appraisal bodies like NICE, utility values derived from clinical trials is preferred over values obtained from literature as commented by ERG in one of the submissions. Mapping data to EQ-5D from a non-preference based measure using data collected from patients experiencing the treatment of interest is accepted as an alternative within the NICE reference case. In the absence of direct utility data from trials, mapping data from other measures to EQ-5D for obtaining utilities for CUA may be an appropriate methodology particularly for paediatric indications.