Clinical and economic value of onabotulinumtoxinA vs oral pharmacotherapies in overactive bladder in the United States

Aims To evaluate the clinical and economic value of onabotulinumtoxinA (onabotA) vs oral pharmacotherapies for adults with OAB inadequately managed with anticholinergics from a United States (US) Medicare + societal perspective over 1 year.Materials and methods A Markov model-based 1-year cost per responder (CPR) analysis was conducted for patients achieving complete response (100% reduction in urinary incontinence episodes [i.e. complete dryness]). Comparators included onabotA and oral therapies (vibegron [brand], mirabegron 25/50 mg [brand and generic], and anticholinergics [generic]), using inputs for efficacy, costs, and healthcare resource use. The primary outcome was CPR for a complete response, with secondary outcomes including number of urinary incontinence episodes per patient and number needed to treat (NNT). Uncertainty was evaluated through one-way and probabilistic sensitivity analyses. Scenario analyses assessed results using commercial costs and longer-term effects for selected outcomes.Results In the base-case analysis, onabotA had the highest complete response rate (23.5%), resulting in the lowest NNT and CPR (4.3; $18,646) vs oral therapies (response 10.9–12.1%; NNT 8.3–9.2; CPR $21,941–$49,524). Scenario and sensitivity analyses were consistent with these findings and supported the base-case results.Limitations Model inputs were primarily based on clinical trial data and may not fully reflect real-world practice; differences across trials may limit comparability. The stringent responder definition, use of non-US productivity estimates, 1-year time horizon, and exclusion of neuromodulation therapies may further limit generalizability and scope.Conclusions OnabotA demonstrated favorable clinical and economic value relative to oral pharmacotherapies that was consistent across sensitivity and scenario analyses. About 1 in 4 patients treated with onabotA could achieve a 100% response over 1 year compared to 1 in at least 8 with oral pharmacotherapies. These findings support onabotA as a treatment option with demonstrated clinical and economic value for patients who do not achieve adequate symptom relief with oral OAB therapies.