The impact of rarity in NICE’s health technology appraisals

Abstract Background In the absence of a framework designed to evaluate medicines for rare diseases in UK, most orphan are appraised by National Institute Health and Care Excellence (NICE) through Single Technology Appraisal (STA) process. Results An analysis STA appraisals non-orphan revealed that were subject significantly longer mean time NICE process than [370 days (n = 44) vs. 277 118), p < 0.0001]. A higher proportion STAs required more one Committee Meeting (ACM) versus STAs, disadvantaged worse outcomes with respect positive recommendations those assessed Highly Specialised evaluation (HST). Conclusions The uncertainties inherent developing may contribute these disadvantages. Improved understanding challenges drug development clearer guidance decision makers on navigating uncertainty HTA promote greater equity access across common conditions.