A post-market economic evaluation of Bortezomib, Lenalidomide and Dexamethasone versus pre-funding standard of care for newly diagnosed multiple myeloma using registry data

Background and Objective: Health technology assessments traditionally rely on clinical trial data, leaving uncertainties about real-world cost effectiveness. This post-market economic evaluation used registry data to estimate the real-world cost effectiveness of bortezomib, lenalidomide and dexamethasone (VRd) versus standard of care as it existed prior to VRd funding for newly diagnosed, transplant eligible and ineligible multiple myeloma, as subsidised by the Australian government in 2019. Methods: We conducted the economic evaluation from the perspective of the Australian healthcare system using the EpiMAP Myeloma model, a discrete event simulation model powered by risk equations based on data from the Australia & New Zealand Myeloma and Related Diseases Registry. This approach captured individual patient heterogeneity and treatment pathways through up to nine lines of therapy. We assessed differences in quality-adjusted life-years and costs over a lifetime horizon, discounting both at the standard Australian rate of 5% per annum. Costs were valued in 2025 Australian dollars and non-parametric bootstrapping was used to quantify parameter uncertainty. Results: Brtezomib, lenalidomide and dexamethasone was associated with 0.16 incremental quality-adjusted life-years (95% confidence interval [CI] 0.10, 0.21) and A$16K incremental costs (95% CI A$12K, A$120K). Improved response to therapy with VRd was predicted to marginally increase receipt of autologous stem cell transplantation by 1.1% (95% CI 0.6, 1.7), significantly increase receipt of maintenance therapy by 13.8% (95% CI 10.4, 17.3) and marginally decrease the proportion of patients progressing to subsequent lines. None of the bootstrap iterations fell below the traditional A$50K/quality-adjusted life-year threshold. Conclusions: The 2019 decision to universally fund VRd for newly diagnosed multiple myeloma did not result in a cost-effective allocation of healthcare resources when judged against the traditional A$50K/quality-adjusted life-year threshold. Our findings provide nuanced insights into the real-world cost effectiveness of VRd, highlighting how post-market evaluations can inform refinement of funding decisions for complex therapeutic interventions.