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Official NIH NLM MEDLINE / PubMed Citation • PMID: 42989979 • DOI: 10.1080/13696998.2026.2755187

Cost-effectiveness of onabotulinumtoxinA versus topiramate in chronic migraine in the US: an economic analysis of the FORWARD randomized trial.

Journal of medical economics • 2026 Dec • Vol. 29 (1) : 2685-2699 • ISSN 1941-837X
Authors: Naoko Ronquest, Yiwei Zhang, Tae Jin Park, Kelly Campbell, Seri Anderson
RTI Health Solutions, Durham, NC, USA.

Verbatim Formal Study Abstract

AIMS

Assess the cost-effectiveness of onabotulinumtoxinA (OnabotA) versus topiramate for the preventive treatment of chronic migraine in US adult patients from a payer perspective.

METHODS

A multistate Markov model evaluated the incremental costs per headache-day avoided and quality adjusted life-year (QALY) over a 2-year period in patients treated with OnabotA versus topiramate. The FORWARD trial data informed model inputs, including reductions in headache-days, while patients receive OnabotA/topiramate. Treatment discontinuation for topiramate was modeled with data from FORWARD and a randomized controlled trial (RCT) to test a range of values for topiramate. Costs of drug acquisition, administration, and monitoring were calculated from prescribing information and physician fee schedules. The model estimated costs of healthcare resource utilization (HCRU) and acute migraine treatment by response level. Sensitivity and scenario analyses explored cost drivers and assessed model robustness.

RESULTS

Our model estimated that OnabotA-treated patients experience 122.56 fewer headache-days (0.34 years) than topiramate-treated patients over the 2-year time horizon. With an incremental cost of $4,639, the incremental cost per headache-day avoided was ∼ $37.85/day, and the incremental cost per QALY gained was $63,646. The estimated cost per avoided headache-day was less than half for patients treated with OnabotA in comparison with topiramate ($116 vs. $246). Sensitivity analyses showed that the incremental cost per headache-day avoided remained below $50 and the incremental cost per QALY gained remained below $100,000 across all tested ranges. Results were similar when using a lower, RCT-supported discontinuation rate for topiramate. In scenario analyses, a longer time horizon and a greater price discount for OnabotA substantially reduced the incremental cost-effectiveness ratios.

CONCLUSION

OnabotA was a cost-effective preventive treatment of chronic migraine versus topiramate. Although OnabotA had higher drug acquisition costs, these were offset by reduced HCRU costs due to fewer migraines, resulting in a low incremental cost per headache-day avoided versus topiramate.

ABSTRACT

Chronic migraine is a neurological disease affecting approximately 80 million people worldwide, substantially diminishing quality of life and imposing a sizable economic burden. Preventive medications, including onabotulinumtoxinA (OnabotA) injections and oral topiramate, are widely used in clinical practice. Although prior studies have evaluated the costs of modern preventive treatments of chronic migraine, direct cost comparisons between these branded treatments and lower cost generic alternatives remain limited. Using the FORWARD clinical trial data, we evaluated the cost-effectiveness of OnabotA versus generic topiramate in US adult patients. The analysis incorporates direct cost components, including drug acquisition, administration, and monitoring, as well as HCRU and acute migraine treatment costs while patients are on treatment and after discontinuation. Although OnabotA carries a higher drug acquisition cost, the analysis demonstrates meaningful cost offsets driven by reduced HCRU associated with improved migraine control (e.g. $859 due to less HCRU, $1,799 due to less use of acute medication in 2 years). As a result, given that OnabotA results in fewer days with headaches, over time, it may be considered a more cost-effective option than topiramate.

Publication Classification: Journal Article Randomized Controlled Trial
NLM MeSH Descriptors: Humans Migraine Disorders Topiramate Botulinum Toxins, Type A Cost-Benefit Analysis Cost-Effectiveness Analysis Quality-Adjusted Life Years Markov Chains Female Adult Chronic Disease Fructose United States Male Middle Aged Health Services