THE HIDDEN COST OF ACCESS: INTEGRATING HTA, EVIDENCE GENERATION, AND MANAGED ENTRY AGREEMENT REQUIREMENTS INTO COST-BASED PRICING MODELS FOR CELL AND GENE THERAPIES
Author(s)
Rimma Velikanova, MSc1, Leo Mulder, MSc2, Maarten Postma, PhD3.
1Asc Academics, Groningen, Netherlands, 2University Medical Center Groningen, University of Groningen, Groningen, Netherlands, 3University of Groningen, Groningen, Netherlands.
1Asc Academics, Groningen, Netherlands, 2University Medical Center Groningen, University of Groningen, Groningen, Netherlands, 3University of Groningen, Groningen, Netherlands.
OBJECTIVES: CGTs are frequently criticised for high prices; cost-based pricing models focus on R&D, manufacturing, and regulatory expenditures. Costs after regulatory approval, including HTA requirements, evidence generation, long-term monitoring, pricing negotiations, and MEAs, are not included. The objective of this study was to identify and quantify these hidden Market Access (MA) costs and assess their impact on cost-based pricing estimates for CGTs.
METHODS: The study uses a three-step mixed evidence approach. First, a pragmatic literature review identified development, manufacturing, regulatory, and MA costs using academic literature, annual reports, SEC filings, HTA reports, and industry sources. Second, qualitative document analysis of EMA, NICE, and ZIN assessments for Zolgensma, Abecma, and Carvykti mapped post-approval requirements to manufacturers’ activities, thereby deriving hidden cost categories, including long-term monitoring and pricing processes. Third, these components were integrated into an extended cost-based pricing model incorporating risk-adjusted R&D costs, manufacturing costs, cost of capital, MA costs, and scenario analyses to estimate per-patient price impacts.
RESULTS: HTA evaluations identified post-approval requirements generating additional manufacturer activities beyond approval, including clinical studies, follow-up periods, revised cost-effectiveness models, comparative effectiveness analyses, and outcome-based reimbursement arrangements. Hidden cost estimates included evidence generation USD 4.0 to 15.0 million), long-term monitoring USD 2.8 to 10.9 million), and economic modelling 2.7 per cent of total R&D expenditure. Incorporating costs resulted in per-patient prices of €538,602 for Zolgensma, €148,778 for Abecma, and €159,444 for Carvykti. For Zolgensma, the estimate exceeded the prior cost-based price that excluded MA costs by 41.6%.
CONCLUSIONS: Current pricing models may underestimate the total cost burden of CGTs by excluding post-approval MA requirements. HTA-driven evidence generation, monitoring obligations, and reimbursement processes may be important cost drivers in cost-based pricing analyses. Incorporating hidden costs may improve transparency and provide a realistic understanding of economic challenges.
METHODS: The study uses a three-step mixed evidence approach. First, a pragmatic literature review identified development, manufacturing, regulatory, and MA costs using academic literature, annual reports, SEC filings, HTA reports, and industry sources. Second, qualitative document analysis of EMA, NICE, and ZIN assessments for Zolgensma, Abecma, and Carvykti mapped post-approval requirements to manufacturers’ activities, thereby deriving hidden cost categories, including long-term monitoring and pricing processes. Third, these components were integrated into an extended cost-based pricing model incorporating risk-adjusted R&D costs, manufacturing costs, cost of capital, MA costs, and scenario analyses to estimate per-patient price impacts.
RESULTS: HTA evaluations identified post-approval requirements generating additional manufacturer activities beyond approval, including clinical studies, follow-up periods, revised cost-effectiveness models, comparative effectiveness analyses, and outcome-based reimbursement arrangements. Hidden cost estimates included evidence generation USD 4.0 to 15.0 million), long-term monitoring USD 2.8 to 10.9 million), and economic modelling 2.7 per cent of total R&D expenditure. Incorporating costs resulted in per-patient prices of €538,602 for Zolgensma, €148,778 for Abecma, and €159,444 for Carvykti. For Zolgensma, the estimate exceeded the prior cost-based price that excluded MA costs by 41.6%.
CONCLUSIONS: Current pricing models may underestimate the total cost burden of CGTs by excluding post-approval MA requirements. HTA-driven evidence generation, monitoring obligations, and reimbursement processes may be important cost drivers in cost-based pricing analyses. Incorporating hidden costs may improve transparency and provide a realistic understanding of economic challenges.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE722
Topic
Economic Evaluation, Health Policy & Regulatory, Organizational Practices
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Genetic, Regenerative & Curative Therapies, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases