OBJECTIVES : EQ-5D is widely used in health technology assessments in a variety of conditions, but it is important to consider whether it is appropriate for ultra-rare diseases. This review examines the use of EQ-5D and other HRQoL measures in NICE highly specialised technologies (HST) evaluations and the committee’s conclusions on their appropriateness.

METHODS : Published HST evaluations were reviewed in February 2020, to inform the ongoing NICE methods review. Data extracted included disease area, population, EQ-5D data availability and whether they were collected in the clinical trials, other HRQoL measures presented, and committee’s conclusions about the appropriateness of EQ-5D and other measures.

RESULTS : Twelve evaluations were reviewed. Results show that EQ-5D data were either collected in the trials (17%) or available from the literature (33%) in half of cases. In a quarter, other generic measures, such as the SF-36 or HUI3 (25%) were used instead of, or presented alongside, EQ-5D. Alternative methods were sometimes used to obtain EQ-5D values, such as clinicians completing EQ-5D for vignettes (33%) or using mapping algorithms to derive EQ-5D values from another measure (8%). The evaluation documents do not record any instances of the committee being presented with data to demonstrate EQ-5D being inappropriate (e.g. poor responsiveness or lack of validity). Where the committee did accept modelling based on alternative HRQoL measures, this was because there was no EQ-5D data available.

CONCLUSIONS : In HST evaluations, lack of EQ-5D has been a bigger issue than concerns about its appropriateness. Where EQ-5D was not available, other HRQoL measures have informed committee’s decisions. Where EQ-5D was available, the committee often noted concerns about the methods used to derive the data and the robustness of the estimates. The challenges and uncertainties around measuring HRQoL in ultra-rare diseases are important for committees to consider but is not a barrier to decision-making.