Addressing Payer Concerns: Opportunities for HEOR in Orphan Drug Development
Author(s)
Catalin Gorceag, MSc1, Tatiana O. Bessonova, MSc2, Oliver Smith, MSc1, McVin Cheen, PhD2, Weiwei Xu, PhD, MD2.
1IQVIA, London, United Kingdom, 2IQVIA, Amsterdam, Netherlands.
1IQVIA, London, United Kingdom, 2IQVIA, Amsterdam, Netherlands.
OBJECTIVES: To assess health technology assessment bodies’ (HTAb) criticisms of the orphan drugs evidence package.
METHODS: Completed National Institute for Health and Care Excellence (NICE) appraisals of highly specialised technologies (HSTs) for endocrine and metabolic disorders since 2023 were identified. Appraisals of the same technologies identified from NICE were expanded to Canada’s Drug Agency (CDA-AMC), Scottish Medicines Consortium (SMC) and Pharmaceutical Benefits Advisory Committee (PBAC) of Australia. Key criticisms were identified across population, clinical and economic value domains.
RESULTS: Eight NICE, four SMC, three CDA-AMC and four PBAC reports were reviewed. Key population-related criticisms included baseline imbalances between treatment arms and inappropriate subgroup classification/ underrepresentation (n=10, 53%). Clinical evidence criticisms included inappropriate validation of surrogate endpoints (n=5, 26%) and reliance on non-disease specific health-related quality of life (HRQoL) (n=6, 32%). Economic evidence critiques highlighted utility value uncertainty (use of vignette studies and carer/ bereavement disutility) (n=11, 58%) and overreliance on expert opinion/ surrogate endpoints as model parameters (n=5, 26%). NICE permitted the use of carer disutility in economic models of paediatric patients (only in genetic disorders with perinatal, infantile, or juvenile onset [n=6, 32%]), while PBAC and CDA-AMC highlighted uncertainties associated with surrogate endpoints. No substantial difference between HTAb’s were observed in the population and clinical value domains.
CONCLUSIONS: Strategies addressing payer evidence needs should be implemented early in the technology’s lifecycle to optimise HTA outcomes. Scientific advice alongside HTA analysis of analogue diseases can identify relevant subgroups and optimise design of pivotal trials. Ensuring balanced randomisation across treatment arms based on key prognostic factors to minimise bias in the evidence. Surrogate endpoint validation helps demonstrate the clinical and economic value of the technology amid high uncertainty. Validated disease specific HRQoL tools in the absence of tools in suitable proxy conditions can reduce model uncertainties and reliance on vignette studies.
METHODS: Completed National Institute for Health and Care Excellence (NICE) appraisals of highly specialised technologies (HSTs) for endocrine and metabolic disorders since 2023 were identified. Appraisals of the same technologies identified from NICE were expanded to Canada’s Drug Agency (CDA-AMC), Scottish Medicines Consortium (SMC) and Pharmaceutical Benefits Advisory Committee (PBAC) of Australia. Key criticisms were identified across population, clinical and economic value domains.
RESULTS: Eight NICE, four SMC, three CDA-AMC and four PBAC reports were reviewed. Key population-related criticisms included baseline imbalances between treatment arms and inappropriate subgroup classification/ underrepresentation (n=10, 53%). Clinical evidence criticisms included inappropriate validation of surrogate endpoints (n=5, 26%) and reliance on non-disease specific health-related quality of life (HRQoL) (n=6, 32%). Economic evidence critiques highlighted utility value uncertainty (use of vignette studies and carer/ bereavement disutility) (n=11, 58%) and overreliance on expert opinion/ surrogate endpoints as model parameters (n=5, 26%). NICE permitted the use of carer disutility in economic models of paediatric patients (only in genetic disorders with perinatal, infantile, or juvenile onset [n=6, 32%]), while PBAC and CDA-AMC highlighted uncertainties associated with surrogate endpoints. No substantial difference between HTAb’s were observed in the population and clinical value domains.
CONCLUSIONS: Strategies addressing payer evidence needs should be implemented early in the technology’s lifecycle to optimise HTA outcomes. Scientific advice alongside HTA analysis of analogue diseases can identify relevant subgroups and optimise design of pivotal trials. Ensuring balanced randomisation across treatment arms based on key prognostic factors to minimise bias in the evidence. Surrogate endpoint validation helps demonstrate the clinical and economic value of the technology amid high uncertainty. Validated disease specific HRQoL tools in the absence of tools in suitable proxy conditions can reduce model uncertainties and reliance on vignette studies.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA23
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases