COST EFFECTIVENESS OF ONASEMNOGENE ABEPARVOVEC IN THE TREATMENT OF PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY IN TURKIYE
Author(s)
Mehtap Tatar, PhD1, Vildan Goknur Haliloglu, Prof. Dr.2, Tugce Ozbalas, Sr., BA, BSc, MBA3, Ebru Alnigenis, M.D3.
1Director, Polar Health Economics and Policy Consultancy, Ankara, Turkey, 2Hacettepe University Faculty of Medicine, Ankara, Turkey, 3Novartis Pharma Turkey, Istanbul, Turkey.
1Director, Polar Health Economics and Policy Consultancy, Ankara, Turkey, 2Hacettepe University Faculty of Medicine, Ankara, Turkey, 3Novartis Pharma Turkey, Istanbul, Turkey.
OBJECTIVES: Spinal Muscular Atrophy (SMA)is a rare, genetic, neuromuscular disease. Presymptomatic SMA refers to the detection of SMA often through newborn screening before the onset of symptoms. The objective of this study is to estimate the cost effectiveness of onasemnogene abeparvovec for the treatment of presymptomatic SMA compared to nusinersen and risdiplam in Turkiye.
METHODS: A Markov model was developed to estimate the lifetime health effects and costs of onasemnogene abeparvovec compared with nusinersen and risdiplam for infants with genetically confirmed, presymptomatic SMA with a genotype predictive of SMA (i.e. those with two and three copies of the SMN2 gene). The health states in the model were broad range of normal development, delayed walker, experiences later onset SMA, sitter, non-sitter, non-sitter (permanent assisted ventilation) and death. The analysis was conducted from the Turkish payer perspective (The Social Security Institution-SSI) with a life-time horizon. Costs of treating SMA were calculated using the reimbursement prices of the SSI. All costs and outcomes were discounted at 3%.
RESULTS: The cost-effectiveness analysis results for the combined cohort (2 and 3 gene copied cohorts) comparing onasemnogene abeparvovec with nusinersen estimated that the lifetime cost of treating presymptomatic SMA was lower for onasemnogene abeparvovec (53,534,935 TRY vs 119,625,986 TRY) with higher life years (30.41 vs 30.38) and QALYs (27.55 vs 27.13). Similar results were found in comparison with risdiplam as well. The cost of treatment was lower for onasemnogene abeparvovec compared with risdiplam (53,534,935 TRY vs 169,388,586TRY) with higher life years (30.41 vs 29.95) and QALYs (27.55 vs 23.62). Sensitivity analysis results indicated that the findings were robust. With these results onasemnogene abeparvovec dominated both nusinersen and risdiplam in the treatment of presymptomatic SMA in Turkiye.
CONCLUSIONS: Onasemnogene abeparvovec is a cost-effective option for the treatment of presymptomatic SMA in Turkiye.
METHODS: A Markov model was developed to estimate the lifetime health effects and costs of onasemnogene abeparvovec compared with nusinersen and risdiplam for infants with genetically confirmed, presymptomatic SMA with a genotype predictive of SMA (i.e. those with two and three copies of the SMN2 gene). The health states in the model were broad range of normal development, delayed walker, experiences later onset SMA, sitter, non-sitter, non-sitter (permanent assisted ventilation) and death. The analysis was conducted from the Turkish payer perspective (The Social Security Institution-SSI) with a life-time horizon. Costs of treating SMA were calculated using the reimbursement prices of the SSI. All costs and outcomes were discounted at 3%.
RESULTS: The cost-effectiveness analysis results for the combined cohort (2 and 3 gene copied cohorts) comparing onasemnogene abeparvovec with nusinersen estimated that the lifetime cost of treating presymptomatic SMA was lower for onasemnogene abeparvovec (53,534,935 TRY vs 119,625,986 TRY) with higher life years (30.41 vs 30.38) and QALYs (27.55 vs 27.13). Similar results were found in comparison with risdiplam as well. The cost of treatment was lower for onasemnogene abeparvovec compared with risdiplam (53,534,935 TRY vs 169,388,586TRY) with higher life years (30.41 vs 29.95) and QALYs (27.55 vs 23.62). Sensitivity analysis results indicated that the findings were robust. With these results onasemnogene abeparvovec dominated both nusinersen and risdiplam in the treatment of presymptomatic SMA in Turkiye.
CONCLUSIONS: Onasemnogene abeparvovec is a cost-effective option for the treatment of presymptomatic SMA in Turkiye.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE720
Topic
Economic Evaluation
Topic Subcategory
Trial-Based Economic Evaluation
Disease
Neurological Disorders, Rare & Orphan Diseases