Cost-effectiveness and budget impact of Luspatercept in Thai adults with transfusion-dependent beta-thalassaemia: a multi-state Markov model based on national claims data

Information from the abstract

Background Luspatercept is a first-in-class erythroid maturation agent that reduces transfusion burden in adults with transfusion-dependent beta-thalassaemia (TDT). However, its cost-effectiveness has not been evaluated in middle-income settings. This study aimed to evaluate the cost-effectiveness and budget impact of Luspatercept for TDT in Thai adults.Methods A hybrid decision tree and lifetime Markov cohort model was developed to compare Luspatercept plus best supportive care (BSC) with BSC alone from a societal perspective. Age-specific 12-week transition probabilities across three transfusion burden states (low, medium, and high) and death were derived from a multi-state Markov survival model fitted to national administrative claims data. Treatment efficacy was based on the phase 3 BELIEVE trial. Three treatment eligibility scenarios were evaluated: universal (all patients), targeted (medium and high transfusion burden), and restricted (high transfusion burden only). Deterministic, probabilistic, threshold price, and budget impact analyses were performed.Results In the universal scenario, Luspatercept produced an incremental gain of 0.205 QALYs and an additional cost of 955,053 THB (29,048 US$) per patient, resulting in an incremental cost-effectiveness ratio (ICER) of 4,650,427 THB/QALY (141,442 US$/QALY). This value exceeds the Thai willingness-to-pay threshold of 160,000 THB/QALY by approximately 29-fold. Restricting eligibility to patients with higher transfusion burden did not improve cost-effectiveness; in the restricted scenario, Luspatercept was dominated. A price reduction of 90.5% would be required for Luspatercept to become cost-effective, which would also reduce the five-year cumulative budget impact from 2,505 million THB to 296 million THB.Conclusion Luspatercept is not cost-effective for TDT in Thailand at its current price across all scenarios evaluated. A substantial price reduction would be necessary, though this would bring the budget impact to a manageable level. These findings may help inform pricing and reimbursement decisions in Thailand and other middle-income countries.

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Related topics: Hemoglobinopathies and Related Disorders · Myeloproliferative Neoplasms: Diagnosis and Treatment · Blood groups and transfusion