Journal of pharmaceutical policy and practice · 2026
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.
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Kongpakwattana Khachen, Wong Peerapon, Teawtrakul Nattiya, Charoenkwan Pimlak, Upakdee Nilawan
Аннотация
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.
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.