COST-EFFECTIVENESS OF NEWBORN SCREENING FOR SPINAL MUSCULAR ATROPHY IN CHINA
Author(s)
Taoran Liu, PhD, YANGYANG GAO, PhD, Wai-kit Ming, MBA, MPH, PhD, MD.
Department of Infectious Diseases and Public Health, City University of Hong Kong, Hong Kong, Hong Kong.
Department of Infectious Diseases and Public Health, City University of Hong Kong, Hong Kong, Hong Kong.
OBJECTIVES: To estimate the cost-effectiveness of SMA NBS followed by early treatment compared with no NBS followed by symptomatic diagnosis and treatment in China, using both a 3-year short-term health-state model and a lifetime extrapolation model.
METHODS: A decision-analytic model was developed for an annual Chinese newborn cohort of 7,920,000 infants. The model combined a screening decision tree, a 3-year health-state model with 6-month cycles, and lifetime extrapolation conditional on health state at 36 months. Health states included permanent ventilation, not sitting, sitting, walking with assistance, broad range of normal development (BRND)/walking unsupported, and death. The base-case health-system perspective included first-tier screening, confirmatory testing, symptomatic diagnosis, drug acquisition and administration, health-state costs, and terminal care. The China-practical base case included Nusinersen and Risdiplam but excluded gene therapy because routine newborn pricing and reimbursement pathways were not available at the time of model parameterization.
RESULTS: Deletion-based NBS identified 767.93 true-positive cases and generated 791.92 false-positive first-tier screens requiring confirmatory testing. At 36 months, NBS shifted outcomes away from permanent ventilation, sitting-only status, walking with assistance, and death, and increased the number of children in the BRND/walking unsupported state. Over the 3-year horizon, NBS generated 347.00 additional QALYs and 56.89 additional life-years at an incremental cost of CNY 163.50 million, resulting in an ICER of CNY 471,195 per QALY. Over the lifetime horizon, NBS generated 3,472.78 additional QALYs and 2,031.00 additional life-years at an incremental cost of CNY 380.65 million, resulting in an ICER of CNY 109,611 per QALY. First-tier screening cost was a key affordability driver: the lifetime ICER was CNY 86,805 per QALY at CNY 15 per newborn and CNY 166,626 per QALY at CNY 50 per newborn.
CONCLUSIONS: SMA NBS may be cost-effective at the three times GDP-per-capita threshold, particularly if first-tier screening can be delivered at a low marginal cost.
METHODS: A decision-analytic model was developed for an annual Chinese newborn cohort of 7,920,000 infants. The model combined a screening decision tree, a 3-year health-state model with 6-month cycles, and lifetime extrapolation conditional on health state at 36 months. Health states included permanent ventilation, not sitting, sitting, walking with assistance, broad range of normal development (BRND)/walking unsupported, and death. The base-case health-system perspective included first-tier screening, confirmatory testing, symptomatic diagnosis, drug acquisition and administration, health-state costs, and terminal care. The China-practical base case included Nusinersen and Risdiplam but excluded gene therapy because routine newborn pricing and reimbursement pathways were not available at the time of model parameterization.
RESULTS: Deletion-based NBS identified 767.93 true-positive cases and generated 791.92 false-positive first-tier screens requiring confirmatory testing. At 36 months, NBS shifted outcomes away from permanent ventilation, sitting-only status, walking with assistance, and death, and increased the number of children in the BRND/walking unsupported state. Over the 3-year horizon, NBS generated 347.00 additional QALYs and 56.89 additional life-years at an incremental cost of CNY 163.50 million, resulting in an ICER of CNY 471,195 per QALY. Over the lifetime horizon, NBS generated 3,472.78 additional QALYs and 2,031.00 additional life-years at an incremental cost of CNY 380.65 million, resulting in an ICER of CNY 109,611 per QALY. First-tier screening cost was a key affordability driver: the lifetime ICER was CNY 86,805 per QALY at CNY 15 per newborn and CNY 166,626 per QALY at CNY 50 per newborn.
CONCLUSIONS: SMA NBS may be cost-effective at the three times GDP-per-capita threshold, particularly if first-tier screening can be delivered at a low marginal cost.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE559
Topic
Economic Evaluation, Epidemiology & Public Health, Health Service Delivery & Process of Care
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Pediatrics