CLINICAL AND ECONOMIC EVALUATION OF ASFOTASE ALFA FOR PEDIATRIC-ONSET HYPOPHOSPHATASIA IN KAZAKHSTAN
Author(s)
Alima Almadiyeva, MScPH, MD1, Aidar Abeuov, MScPH1, Talgat Nurgozhin, PhD, MD, DSc2, Alexandr Kostyuk, PhD, MD2.
1Kazakh Agency for Health Technology Assessment, Astana, Kazakhstan, 2Kazakhstan Association of health technologies assessment, evidence based medicine and pharmacoeconomic research, Astana, Kazakhstan.
1Kazakh Agency for Health Technology Assessment, Astana, Kazakhstan, 2Kazakhstan Association of health technologies assessment, evidence based medicine and pharmacoeconomic research, Astana, Kazakhstan.
OBJECTIVES: This study aimed to conduct a clinical and economic evaluation of asfotase alfa for the treatment of pediatric-onset hypophosphatasia in the Republic of Kazakhstan.
METHODS: A systematic literature review was performed utilizing PubMed, the Cochrane Library, ClinicalTrials.gov, and international health technology assessment databases. A Markov state-transition model with a lifetime horizon was developed from a public healthcare payer perspective to evaluate cost-effectiveness. The model incorporated three distinct health states: hypophosphatasia without invasive ventilation, hypophosphatasia requiring invasive ventilation, and death. Clinical outcomes were translated into quality-adjusted life-years (QALYs) to calculate the incremental cost-effectiveness ratio (ICER). Additionally, a five-year budget impact analysis estimated financial implications within the Kazakhstan healthcare setting.
RESULTS: Asfotase alfa improved five-year overall survival to 84% versus 27% in historical controls for perinatal and infantile-onset patients. Ventilator-free survival improved, with 76% of treated infants successfully extubated compared to 5% in the control cohort. Pediatric patients demonstrated skeletal healing and increased functional mobility, reaching 84% of predicted normal values on mobility assessments. The safety profile was manageable, consisting primarily of mild injection site reactions without severe adverse events. Economically, the therapy yielded an incremental gain of 3.74 QALYs. The calculated ICER was approximately $8.02 million per QALY gained. The budget impact analysis indicated that increased direct drug costs were partially offset by substantial healthcare resource utilization reductions, primarily driven by decreased mechanical ventilation dependency, shorter intensive care unit stays, and fewer orthopedic surgeries.
CONCLUSIONS: Asfotase alfa may improve overall survival and functional independence for patients with pediatric-onset hypophosphatasia. While associated with substantial acquisition costs, the therapy demonstrated a capacity to reduce healthcare resource utilization. These specific findings may inform reimbursement decision-making in Kazakhstan. Given the calculated economic parameters, alternative reimbursement mechanisms may be considered to manage the overall financial impact.
METHODS: A systematic literature review was performed utilizing PubMed, the Cochrane Library, ClinicalTrials.gov, and international health technology assessment databases. A Markov state-transition model with a lifetime horizon was developed from a public healthcare payer perspective to evaluate cost-effectiveness. The model incorporated three distinct health states: hypophosphatasia without invasive ventilation, hypophosphatasia requiring invasive ventilation, and death. Clinical outcomes were translated into quality-adjusted life-years (QALYs) to calculate the incremental cost-effectiveness ratio (ICER). Additionally, a five-year budget impact analysis estimated financial implications within the Kazakhstan healthcare setting.
RESULTS: Asfotase alfa improved five-year overall survival to 84% versus 27% in historical controls for perinatal and infantile-onset patients. Ventilator-free survival improved, with 76% of treated infants successfully extubated compared to 5% in the control cohort. Pediatric patients demonstrated skeletal healing and increased functional mobility, reaching 84% of predicted normal values on mobility assessments. The safety profile was manageable, consisting primarily of mild injection site reactions without severe adverse events. Economically, the therapy yielded an incremental gain of 3.74 QALYs. The calculated ICER was approximately $8.02 million per QALY gained. The budget impact analysis indicated that increased direct drug costs were partially offset by substantial healthcare resource utilization reductions, primarily driven by decreased mechanical ventilation dependency, shorter intensive care unit stays, and fewer orthopedic surgeries.
CONCLUSIONS: Asfotase alfa may improve overall survival and functional independence for patients with pediatric-onset hypophosphatasia. While associated with substantial acquisition costs, the therapy demonstrated a capacity to reduce healthcare resource utilization. These specific findings may inform reimbursement decision-making in Kazakhstan. Given the calculated economic parameters, alternative reimbursement mechanisms may be considered to manage the overall financial impact.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE405
Topic
Clinical Outcomes, Economic Evaluation, Health Policy & Regulatory
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Pediatrics, Rare & Orphan Diseases