OBJECTIVES: In Australia, the reimbursement of botulinum neurotoxin-A (BoNT-A) on the Pharmaceutical Benefits Scheme (PBS) for the treatment of moderate to severe spasticity of the upper limb following a stroke (PSS-UL) is restricted to four treatment cycles per upper limb per lifetime. The objective of this study was to examine the cost-effectiveness of extending the treatment beyond four treatments among patients with an adequate response to previous treatment cycles.

METHODS: A Markov state transition model was developed to perform a cost-utility analysis (CUA) of extending the use of incobotulinumtoxin-A beyond the current restriction. The Markov model followed patients in 12-weekly cycles for five years, estimating the proportion of patients with or without response over this period in each of the modelled treatment arms. A post-hoc analysis was performed to estimate probabilities of achieving and maintaining response over time. The perspective of the analysis was the Australian healthcare system meaning only direct healthcare costs were included. Utility values by response status were derived from EQ-5D data from a published double-blind placebo-controlled study. The primary outcome measure was the incremental cost per quality-adjusted life year (QALY). Univariate and probabilistic sensitivity analyses were conducted.

RESULTS: The incremental cost per QALY gained of continued use of incobotulinumtoxin-A beyond the current restriction of four treatments was AUD$59,659.

CONCLUSIONS: Continuing the incobotulinumtoxin-A treatment beyond four cycles can be cost-effective. Careful patient selection is required so treatment is targeted at those with the greatest likelihood to continue to respond to multiple treatment cycles.