Evaluating the Impact of Different Payment Models on the Cost-Effectiveness and Budget Impact of Cell and Gene Therapies: Eladocagene Exuparvovec As a Case Study
Author(s)
Rimma Velikanova, MSc1, Maria Aleneiny, BSc2, Maarten Jacobus Postma, PhD2, Talitha Feenstra, PhD3.
1ASC Academics, Haren, Netherlands, 2University of Groningen, Groningen, Netherlands, 3Groningen University/RIVM, Eelderwolde, Netherlands.
1ASC Academics, Haren, Netherlands, 2University of Groningen, Groningen, Netherlands, 3Groningen University/RIVM, Eelderwolde, Netherlands.
OBJECTIVES: Cell and gene therapies (CGTs) may offer long-lasting clinical benefits for rare conditions; however, their high acquisition costs and limited clinical evidence at launch challenge the conventional reimbursement framework. Alternative payment models have been proposed to ease these pressures. This study assessed three payment models and examined how they influence cost-effectiveness (CE) and budget impact (BI) results, using eladocagene exuparvovec as a case study.
METHODS: A lifetime CE and a 5-year BI analysis were conducted for eladocagene exuparvovec versus best supportive care, treating patients with aromatic L-amino-acid decarboxylase deficiency, from a UK perspective, using three payment models: outcome-based, annuity, and mixed outcome-based instalments, all compared to upfront payment as reference.
RESULTS: All three payment models influenced eladocagene exuparvovec’s economic outcomes relative to an upfront payment. The upfront payment resulted in a total cost of £105.9 million, with an incremental cost-effectiveness ratio (ICER) of £278,365 per quality-adjusted life year (QALY). The outcome-based and mixed models reduced costs to £60.9 million and £58.3 million, lowering the ICER to £147,541 and £139,987. The annuity increased total cost to £107.2 million and raised the ICER to £267,700 due to interest.BI results followed a similar pattern. The upfront payment required £24.2 million in Year 1, exceeding the affordability threshold. The annuity reduced this to £5.2 million yet generated total BI of £31.6 million. The outcome-based model deferred expenditure, with a single £12.8 million payment in Year 5. The mixed instalment model started increasing from £5 million in Year 1 and had a total BI of £15 million.
CONCLUSIONS: In this case, the outcome-based instalment payment model seemed most favourable for financial sustainability and payer value. By combining upfront risk-sharing with payments tied to patient outcomes, it balances short-term affordability with long-term value, reducing budgets and improving ICER, which was still above commonly used thresholds.
METHODS: A lifetime CE and a 5-year BI analysis were conducted for eladocagene exuparvovec versus best supportive care, treating patients with aromatic L-amino-acid decarboxylase deficiency, from a UK perspective, using three payment models: outcome-based, annuity, and mixed outcome-based instalments, all compared to upfront payment as reference.
RESULTS: All three payment models influenced eladocagene exuparvovec’s economic outcomes relative to an upfront payment. The upfront payment resulted in a total cost of £105.9 million, with an incremental cost-effectiveness ratio (ICER) of £278,365 per quality-adjusted life year (QALY). The outcome-based and mixed models reduced costs to £60.9 million and £58.3 million, lowering the ICER to £147,541 and £139,987. The annuity increased total cost to £107.2 million and raised the ICER to £267,700 due to interest.BI results followed a similar pattern. The upfront payment required £24.2 million in Year 1, exceeding the affordability threshold. The annuity reduced this to £5.2 million yet generated total BI of £31.6 million. The outcome-based model deferred expenditure, with a single £12.8 million payment in Year 5. The mixed instalment model started increasing from £5 million in Year 1 and had a total BI of £15 million.
CONCLUSIONS: In this case, the outcome-based instalment payment model seemed most favourable for financial sustainability and payer value. By combining upfront risk-sharing with payments tied to patient outcomes, it balances short-term affordability with long-term value, reducing budgets and improving ICER, which was still above commonly used thresholds.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE444
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Budget Impact Analysis
Disease
Genetic, Regenerative & Curative Therapies, Neurological Disorders, Pediatrics, Rare & Orphan Diseases