OBJECTIVES: Thai pharmaceutical reimbursement list excludes Botulinum toxin type A (BoNT-A), leading to delayed access during optimal age range (2-5 years) and reduced treatment response. This study evaluated cost-utility of two BoNT-A treatments for young children (18-42 months) with ambulatory spastic cerebral palsy (CP).

METHODS: A cohort-based state transition model was constructed using a societal perspective. A 3-month cycle length was used to estimate relevant costs and health outcomes for a lifetime horizon, given a 3% annual discount. Abobotulinum toxin A (Abo) or Onabotulinum toxin A (Ona) combined with standard of care (SoC) were compared to the SoC alone. Direct medical cost was calculated using a generalized linear model based on a five-year retrospective treatment cost from a tertiary hospital. Direct non-medical cost, utility, and transitional probabilities were primarily collected in three tertiary hospitals. A network meta-analysis was employed to estimate the efficacy of each treatment. Lifetime cost, quality-adjusted life years (QALYs) and an incremental cost-effectiveness ratio (ICER) were calculated and compared to the cost-effectiveness threshold of 160,000 THB (US $4,597, where 1 USD = 34.81 THB in 2023) per QALY gained. Probabilistic and one-way sensitivity analyses were performed to estimate parameter uncertainties. Scenario analysis was conducted to estimate ICER for BoNT-A administration during optimal age range.

RESULTS: The Abo or Ona combined with SoC provided 0.0033 and 0.0026 QALYs gained with additional costs of 709 and 1,054 compared to the SoC, respectively. Consequently, Abo had an ICER of 215,009 THB per QALY, while Ona had an ICER of 399,203 THB per QALY. The results were sensitive to the direct non-medical cost of non-responder.

CONCLUSIONS: Combining BoNT-A with SoC was not economically attractive for treating spastic CP in Thailand. Lowering Abo's price by 14% could enhance its effectiveness. Price negotiation process is crucial to ensure financial value and affordability.