Real-world cost-effectiveness of pertuzumab (P) with trastuzumab + chemo (T+Chemo) in patients (pts) with metastatic breast cancer (MBC): A population-based retrospective cohort study by the Canadian Real-world Evidence for Value in Cancer Drugs (CanREValue) collaboration.

1048 Background: Addition of P to T+chemo for MBC pts has been shown to improve overall survival (OS) in a pivotal randomized trial (hazard ratio [HR] = 0.66, 95% CI: 0.52, 0.84) (Baselga et al., NEJM 2012). In Canada, the manufacturer submission to the health technology assessment agency estimated that P produced 0.64 life years gained (LYG) with an incremental cost-effectiveness ratio (ICER) of $187,376/LYG over 10 years (CADTH-pCODR, 2013). This retrospective cohort analysis aims to determine the comparative real-world population-based effectiveness and cost-effectiveness of P among MBC pts in Ontario, Canada. Methods: MBC pts were identified from the Ontario Cancer Registry and linked to the New Drug Funding Program database to identify receipt of treatment between 1/1/2008 and 3/31/2018. Cases received P-T-chemo after universal public funding of P (Nov 2013) and controls received T-chemo before. Demographic (age, socioeconomic, rurality) and clinical (comorbidities, prior adjuvant treatments, prior breast cancer surgery, prior radiation, stage at diagnosis, ER/PR status) characteristics were identified from linked admin databases balanced between cases and controls using propensity score matching. Kaplan-Meier methods and Cox regressions accounting for matched pairs were used to estimate median OS and HR. 5-year mean total costs from the public health system perspective were estimated from admin claims databases using established direct statistical methods and adjusted for censoring of both cost and effectiveness using inverse probability weighting. ICERs and 95% bootstrapped CIs were calculated, along with incremental net benefit (INB) at various willingness-to-pay values using net benefit regression. Results: We identified 1,823 MBC pts with 912 cases and 911 controls (mean age = 55 years), of which 579 pairs were matched. Cases had improved OS (HR = 0.66; 95% CI: 0.57, 0.78), with median 3.4 years, compared to controls median OS of 2.1. P provided an additional 0.63 (95% CI: 0.48 – 0.84) LYG at an incremental cost of $196,622 (95% CI: $180,774, $219,172), with a mean ICER = $312,147/LYG (95% CI: $260,752, $375,492). At threshold of $100,000/LYG, the INB was -$133,632 (95% CI: -$151,525, -$115,739) with < 1% probability of being cost-effective. Key drivers of incremental cost increase between groups included drug and cancer clinic costs. Conclusions: The addition of P to T-chemo for MBC increased survival but at significant costs. The ICER based on direct real-world data was higher than the initial economic model due to higher total costs for pts receiving P. This study demonstrated feasibility to derive ICER from person-level real-world data to inform cancer drug life-cycle health technology reassessment.

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