LAUSR

Importance There is an increasing body of literature that supports the use of rituximab as a first-line steroid-sparing agent in pemphigus vulgaris. However, the cost of rituximab is substantial compared with conventional agents, and there are limited health economic data to justify its use. Objective To evaluate the cost-effectiveness of rituximab biosimilars relative to mycophenolate mofetil as a first-line steroid-sparing agent for moderate to severe pemphigus vulgaris. Design, Setting, and Participants A cost-utility analysis over a 24-month time horizon was conducted from the perspective of the Australian health care sector using a modeled cohort of treatment-naive adult patients with moderate to severe pemphigus vulgaris. A Markov cohort model was constructed to simulate disease progression following first-line treatment with rituximab biosimilars or mycophenolate mofetil. The simulated cohort transitioned between controlled disease, uncontrolled disease, and death. Efficacy and utility data were obtained from available published literature. Cost data were primarily obtained from published government data. One-way and probabilistic sensitivity analyses were performed to assess uncertainty. Primary outcomes were the changes in cost and quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) over the 24 months. Interventions Rituximab biosimilars and mycophenolate mofetil. Results The simulated cohort of treatment-naive patients had a mean age of 50.8 years, a female-to-male ratio of 1.24, and moderate to severe disease as classified by the Harman criteria. First-line rituximab biosimilars were associated with a cost reduction of AU$639 and an improvement of 0.07 QALYs compared with mycophenolate mofetil, resulting in an ICER of -AU$8818/QALY. Rituximab biosimilars were therefore more effective and less costly compared with mycophenolate mofetil. Sensitivity analyses demonstrated that rituximab biosimilars remained cost-effective across a range of values for cost, utility, and transition probability input parameters and willingness-to-pay thresholds. Conclusions and Relevance In this cost-utility analysis, rituximab biosimilars were cost-effective compared with mycophenolate mofetil for moderate to severe pemphigus vulgaris. Further investigation into its cost-effectiveness over a longer time horizon is necessary, but the favorable results of this study suggest that the high acquisition costs of rituximab biosimilars may be offset by its effectiveness and provide economic evidence in support of its listing on the Pharmaceutical Benefits Scheme for pemphigus vulgaris.