Cost-Utility Analysis of Including X-linked Adrenoleukodystrophy in the Spanish Newborn Screening Program
Author(s)
Lucía Pedrosa Pérez, PhD1, Carlos Martin-Saborido, PhD1, Iñaki Imaz-Iglesia, PhD, MD2, Cristina Valcárcel-Nazco, MSc3, Carlos Sánchez-Piedra, PhD1, Esther García Carpintero, PhD1, Lourdes Rodríguez-Rojas, MD1, Montserrat Carmona, PhD1.
1Health Technology Assessment Agency, Instituto de Salud Carlos III, Madrid, Spain, 2National School of Health, Instituto de Salud Carlos III, Madrid, Spain, 3Evaluation Unit of the Canary Islands Health Service, Canary Islands Health Service, Santa Cruz de Tenerife, Spain.
1Health Technology Assessment Agency, Instituto de Salud Carlos III, Madrid, Spain, 2National School of Health, Instituto de Salud Carlos III, Madrid, Spain, 3Evaluation Unit of the Canary Islands Health Service, Canary Islands Health Service, Santa Cruz de Tenerife, Spain.
OBJECTIVES: X-linked adrenoleukodystrophy (X-ALD) is the most common peroxisomal disorder with a strikingly heterogeneous clinical spectrum, including childhood cerebral form, which is highly lethal in boys without hematopoietic stem cell transplantation before symptom onset. Therefore, early diagnosis of X-ALD is critical to administer pre-symptomatic treatment that reduces mortality and improves quality of life. This research aims to assess the cost-effectiveness of implementing universal newborn screening (NBS) for X-ALD in Spain.
METHODS: A cost-utility analysis was conducted using a lifetime decision-tree model, parameterized through a systematic review. From the perspective of the Spanish National Health System (NHS), direct healthcare costs were included, expressed in 2024 euros. The social perspective additionally considered disease-related non-healthcare costs (personal care, housing, goods and services, transportation, and educational and maintenance support) and opportunity costs for patients and their families in Spanish context. A 3% annual discount rate was applied to both costs and health outcomes. Deterministic and probabilistic sensitivity analyses were performed.
RESULTS: Compared to no screening, the NBS strategy for X-ALD resulted in cost savings and increased utility from both the NHS and social perspectives. From the NHS perspective, the costs difference was -€0.71 and the incremental utility was 0.0000093 QALYs. From the social perspective, the costs difference was -€5.36 with the same utility gain (0.0000093 QALYs). Assuming a willingness-to-pay threshold of €27,000 per QALY, the probability that NBS is cost-effective was 97.69% from the NHS perspective and 99.38% from the social perspective.
CONCLUSIONS: Including X-ALD in the Spanish NBS program is a cost-effective strategy from both NHS and social perspectives.
METHODS: A cost-utility analysis was conducted using a lifetime decision-tree model, parameterized through a systematic review. From the perspective of the Spanish National Health System (NHS), direct healthcare costs were included, expressed in 2024 euros. The social perspective additionally considered disease-related non-healthcare costs (personal care, housing, goods and services, transportation, and educational and maintenance support) and opportunity costs for patients and their families in Spanish context. A 3% annual discount rate was applied to both costs and health outcomes. Deterministic and probabilistic sensitivity analyses were performed.
RESULTS: Compared to no screening, the NBS strategy for X-ALD resulted in cost savings and increased utility from both the NHS and social perspectives. From the NHS perspective, the costs difference was -€0.71 and the incremental utility was 0.0000093 QALYs. From the social perspective, the costs difference was -€5.36 with the same utility gain (0.0000093 QALYs). Assuming a willingness-to-pay threshold of €27,000 per QALY, the probability that NBS is cost-effective was 97.69% from the NHS perspective and 99.38% from the social perspective.
CONCLUSIONS: Including X-ALD in the Spanish NBS program is a cost-effective strategy from both NHS and social perspectives.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA100
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Neurological Disorders, Pediatrics, Rare & Orphan Diseases