PRO86 Approaches to the Collection of Utility Values for RARE Diseases: A CROSS-Country Comparison

Abstract

To further characterise utility data collection methods in (ultra-)orphan indications by identifying published utility studies from the EU5 and Canada, and comparing methodologies reported in submissions to the National Institute for Health and Care Excellence (NICE) with the Scottish Medicines Consortium (SMC) and Canadian Agency for Drugs and Technologies in Health (CADTH). A previously conducted review was expanded to identify utility studies from EU5 countries/Canada published between January 2014–June 2020 and additional highly specialised technology (HST) appraisals published on the NICE website since the original search in May 2019.1 SMC and CADTH websites were then searched for equivalent published appraisals corresponding to all available HST submissions. In total, 126/611 articles from the expanded review were included. 31.0% (39/126) and 16.7% (21/126) collected utilities using observational or interventional studies, respectively. Additional study designs included surveys (13.5% [17/126]), systematic literature reviews (12.7% [16/126]), and/or vignette studies (4.8% [6/126]). Nine SMC and 11 CADTH appraisals corresponding to 17 completed/ongoing HST appraisals were identified, with 7 appraisals being identified on all three websites. In line with previous findings, submissions often reported utility data from interventional studies (15/17 [88.2%] HST, 5/9 [55.6%] SMC and 8/11 [72.7%] CADTH); however, the proportion of appraisals using these data for economic evaluations varied (3/15 [20.0%] HST, 4/5 [80.0%] SMC and 4/8 [50.0%] CADTH). Amongst common alternative methods used to supplement utility data for economic evaluations were vignette studies (4/17 [23.5%] HST, 2/9 [22.2%] SMC and 2/11[18.2%] CADTH). Appraisals for very rare diseases across Canada, England and Scotland rely to different degrees on alternative methods for collection of utilities, corresponding with the variety of published utility study types. There remains a need for further research on the appropriate application of alternative utility collection methods in rare diseases.