The Cost-Effectiveness of Atidarsagene Autotemcel (arsa-cel) for the Treatment of Metachromatic Leukodystrophy (MLD) in Spain

Author(s)

Bean K1, Olaye A2, Miller B3, Jensen I3, Muduma G2, Giró A4, Pang F5, Badia X4
1Orchard Therapeutics, Hook, HAM, UK, 2Orchard Therapeutics, London, UK, 3Precision AQ, Boston, MA, USA, 4Omakase Consulting S.L., Barcelona, Spain, 5Orchard Therapeutics, London, LON, UK

OBJECTIVES: MLD is an ultra-rare, rapidly progressive neurodegenerative disease which leads to motor and cognitive decline and premature death. The aim of this study was to determine the cost-effectiveness of an autologous ex vivo gene therapy, arsa-cel, compared to standard of care for the treatment of early-onset MLD from the Spanish National Health System (MHS) and societal perspective.

METHODS: A cost-effectiveness model was developed based on a partitioned-survival framework and Markov structure to model disease progression in MLD using the seven stage Gross Motor Function MLD scale (GMFC-MLD) in patients with Late Infantile (LI; age at symptom-onset ≤30 months) and Early Juvenile (EJ; age at symptom-onset 30 months to <7 years) MLD. Health state transitions were based on patient-level data from arsa-cel clinical trials. A lifetime time horizon was adopted; costs and outcomes were discounted at 3% per annum. Healthcare resource use consumption and unit cost data from EsSalud were used to calculate costs by GMFC-MLD health state. Utilities were derived from a UK vignette study based on time trade-off valuations of health state descriptions and an algorithm involving the Spanish EQ-5D tariff to reflect the preferences of the Spanish general population. Caregiver utilities and societal inputs were calculated from an international caregiver study and median annual earnings in Spain.

RESULTS: For the combined MLD population (pre-symptomatic LI and EJ, and early symptomatic EJ), the base case analysis indicated that arsa-cel is associated with discounted incremental gains vs. BSC of 20.76 QALYs. Based on the list price of arsa-cel, the incremental cost-effectiveness ratio for the combined MLD population treated with arsa-cel vs. standard of care was €128,387/QALY gained and €106,522/QALY gained from a payer and societal perspective, respectively. Sub-group analyses indicate that arsa-cel is most cost-effective in patients treated pre-symptomatically.

CONCLUSIONS: Arsa-cel has shown to be cost-effective for the Spanish NHS versus BSC.

Conference/Value in Health Info

2024-11, ISPOR Europe 2024, Barcelona, Spain

Value in Health, Volume 27, Issue 12, S2 (December 2024)

Code

EE434

Topic

Economic Evaluation

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis

Disease

Genetic, Regenerative & Curative Therapies, Pediatrics, Rare & Orphan Diseases

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