OBJECTIVES: The National Institute for Health and Care Excellence (NICE) appraisals of highly specialised technologies (HST) to treat very rare conditions were evaluated to identify the included utility values (e.g. patient utility, carer disutility, on-treatment utility increment), the sources of utility values (e.g. clinical trial data, patient surveys, literature review, expert opinion) and how they align to the 2013 NICE reference case, and the opinions of the Evidence Review Group (ERG) and NICE Committee.

METHODS: NICE HST appraisal documents published between January 2015 and May 2018 were reviewed. Details of the technology, indication, source of utility values, ERG and NICE Committee comments on the utility values, and final recommendation were systematically extracted.

RESULTS: In total, seven HST appraisals were reviewed. Two submissions included patient utility values collected directly from UK patients via EQ-5D (NICE’s preferred methodology), from a clinical trial (n=1) or a patient survey (n=1). In other submissions, patient utility values were derived from clinical experts’ EQ-5D ratings of patient health state vignettes (n=1) or from published health-related quality of life data (EQ-5D, n=1; SF-36 mapped to EQ-5D, n=1; non-EQ-5D, n=2). Published carer disutility values were included in two submissions: non-EQ-5D (n=1) or non-EQ-5D in a proxy disease (n=1). On-treatment utility increments were included in three submissions from published EQ-5D trial data (n=2) or a time trade-off study (n=1). All seven submissions also included various other (dis)utility values (e.g. post-treatment health states, adverse events, comparator treatments) derived from various sources (e.g. published estimates, expert opinion, assumptions). In general, the NICE committee accepted assumptions and alternative sources, including using proxy condition data, when considered appropriate by the ERG and patient/clinical experts.

CONCLUSIONS: NICE HST appraisals include a range of utility values and sources, in part due to the lack of evidence in very rare conditions, and NICE are accepting of assumptions and alternative sources.