Journal of Pharmaceutical Policy and Practice · Available online 3 Jul 2026 · In press · DOI 10.1080/20523211.2026.2691449
Khachen Kongpakwattana, Peerapon Wong, Nattiya Teawtrakul, Pimlak Charoenkwan, Nilawan Upakdee
Background Transfusion-dependent beta-thalassemia requires lifelong treatment, yet economic evidence in adult patients remains limited, particularly for direct non-medical and indirect costs in high-prevalence, middle-income settings. This study aimed to estimate the annual cost-of-illness and health utility, and to examine their associations with clinical and sociodemographic factors among Thai adults with transfusion-dependent beta-thalassemia.Methods Adult patients with beta-thalassemia major or beta-thalassemia/hemoglobin E disease receiving regular transfusions at two university-affiliated tertiary hospitals in Thailand were enrolled. Sociodemographic data, health utility (EQ-5D-5L), and productivity losses (Work Productivity and Activity Impairment questionnaire) were collected via structured interviews. Healthcare resource utilisation over the preceding 12 months was obtained from hospital records. Costs were annualised and analyzed from a societal perspective. Generalised linear models were used to identify predictors of costs and health utility.Results Eighty-two patients were included. The mean total annual societal cost was $5,093 (95% CI: $4,467–$5,926) per patient. Patients with medium-to-high transfusion burden incurred significantly higher median costs than those with low transfusion burden ($5,260 vs. $3,841; p = 0.020). Direct medical costs were the primary cost driver (60%), largely attributable to outpatient visits, blood transfusions, and iron chelation therapy, followed by indirect costs (26%), with productivity losses driven mainly by presenteeism. Blood transfusion intensity and iron chelation therapy use were significant determinants of total costs (p < 0.05), providing actionable parameters for budget impact forecasting. Mean health utility was 0.912 (95% CI: 0.890–0.934), yet differences across insurance schemes suggested potential inequities in access to optimal care.Conclusion Adult transfusion-dependent beta-thalassemia imposes a substantial economic burden in Thailand, driven largely by direct medical costs. Identification of key cost drivers provides relevant evidence to inform healthcare planning, resource allocation, and policy efforts to address care inequities in high-prevalence settings.
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Kongpakwattana, K., Wong, P., Teawtrakul, N., et al. (2026). Economic burden and health utility of transfusion-dependent beta-thalassemia in Thai adults: a societal perspective. Journal of Pharmaceutical Policy and Practice. https://doi.org/10.1080/20523211.2026.2691449