Clinical and Economic Factors Underlying QALY Weights and Severity Modifiers in NICE Health Technology Evaluations: An Analysis of Recent Appraisals
Author(s)
George Seleiro, MEng, PhD1, Anjali Menon, BSc, MSc2, Keval Haria, BA, MSc2, John D Whalen, BSc, MBA3, Joanne Beatrice Tutein Nolthenius, BSc, MSc3.
1Costello Medical, Cambridge, United Kingdom, 2Costello Medical, London, United Kingdom, 3Pharming Group N.V., Leiden, Netherlands.
1Costello Medical, Cambridge, United Kingdom, 2Costello Medical, London, United Kingdom, 3Pharming Group N.V., Leiden, Netherlands.
OBJECTIVES: Acknowledging that strict adherence to a cost-effectiveness threshold may not capture the full value of health technologies, National Institute for Health and Care Excellence (NICE) committees may apply decision modifiers in severe diseases: severity modifiers in single technology appraisals (STAs, since 2022) and quality-adjusted life year (QALY) weights in highly specialised technology (HST) evaluations (since 2017). This research sought to identify factors underlying the application of these decision modifiers in NICE evaluations.
METHODS: The NICE website was searched for the 30 most recently published STAs and HSTs, which were reviewed to extract clinical and economic factors underlying severity modifiers and QALY weights.
RESULTS: QALY weights were applied in 16/30 HSTs, including five enzyme replacement therapies and five gene therapies; seven in metabolic disorders and four in neurological conditions. QALY weights were not applied and not reported in 11/30 and 3/30 HSTs, respectively. A negative survival impact of the condition was a key driver of the application of QALY weights, with NICE concluding such an impact in 11/16 HSTs incorporating QALY weights, compared with 1/11 HSTs without. High QALY weights (≥2) were applied in 4/16 HSTs, of which three reported on metabolic disorders. QALY weights applied by committees usually differed from those in company submissions and were reduced by committees in 2/16 HSTs due to uncertainty in QALY gains. A 1.5% discount rate was accepted in 4/16 HSTs with QALY weights, versus 0/11 without. Severity modifiers were applied in 11/30 STAs, including seven in oncology; in six, supportive care was the modelled comparator. In 3/11 STAs, the highest severity weight (x1.7) was applied.
CONCLUSIONS: The highest weights were applied in similarly low proportions of HSTs and STAs, with highly variable factors influencing these weights. However, one consistent factor driving QALY weights across HSTs was the negative impact of the condition on survival.
METHODS: The NICE website was searched for the 30 most recently published STAs and HSTs, which were reviewed to extract clinical and economic factors underlying severity modifiers and QALY weights.
RESULTS: QALY weights were applied in 16/30 HSTs, including five enzyme replacement therapies and five gene therapies; seven in metabolic disorders and four in neurological conditions. QALY weights were not applied and not reported in 11/30 and 3/30 HSTs, respectively. A negative survival impact of the condition was a key driver of the application of QALY weights, with NICE concluding such an impact in 11/16 HSTs incorporating QALY weights, compared with 1/11 HSTs without. High QALY weights (≥2) were applied in 4/16 HSTs, of which three reported on metabolic disorders. QALY weights applied by committees usually differed from those in company submissions and were reduced by committees in 2/16 HSTs due to uncertainty in QALY gains. A 1.5% discount rate was accepted in 4/16 HSTs with QALY weights, versus 0/11 without. Severity modifiers were applied in 11/30 STAs, including seven in oncology; in six, supportive care was the modelled comparator. In 3/11 STAs, the highest severity weight (x1.7) was applied.
CONCLUSIONS: The highest weights were applied in similarly low proportions of HSTs and STAs, with highly variable factors influencing these weights. However, one consistent factor driving QALY weights across HSTs was the negative impact of the condition on survival.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA74
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases