Presentation (CTI)

OBJECTIVES: The National Institute for Health and Care Excellence (NICE) recommends EQ-5D as its preferred method for utility generation. However, in cases where EQ-5D is not appropriate or feasible to collect, manufacturers must turn to alternative methods. Vignette studies - structured descriptive scenarios that describe patient experiences - have emerged as a valuable tool for generating utility values in rare or complex conditions where standardised instruments fall short.
METHODS: A targeted search of NICE’s Technology Appraisal (TA) and Highly Specialised Technologies (HST) guidance was conducted on 11 February 2025 and updated on 26 June 2025. Published appraisals were screened for the use of vignettes in the final economic model. Committee papers and public slides were reviewed to extract commentary from the External Assessment Group (EAG), company responses, and committee conclusions.
RESULTS: A total of 230 appraisals were reviewed (218 STA and 14 HST). Of these, 16 included vignette-based utility generation (8 STA and 8 HST). Therapy areas using vignettes included oncology, infectious diseases, immunology, and rare metabolic disorders. Where EQ-5D was collected in the trial, reasons provided for non-use included lack of validity or sensitivity and lack of data across all health states. EAGs often raised concerns about subjectivity and lack of generalisability, while companies defended vignettes as the only feasible method in these populations. Committees typically accepted vignettes when uncertainty was mitigated through triangulation with other data sources; expert validation and alignment with clinical trial outcomes also helped.
CONCLUSIONS: This review reinforces the continued role of vignette studies in NICE submissions, particularly in rare and complex conditions. While methodological concerns persist, successful appraisals demonstrate that well-constructed vignettes, supported by expert input and transparent methodology, can effectively inform cost-effectiveness decisions. Future work should continue to explore how manufacturers can address EAG and committee concerns to optimise acceptance rates for vignette-based models.