A novel, out-of-pocket, cost-effectiveness analysis comparing a frontline niraparib-for-all to a biomarker-based strategy in patients with advanced ovarian cancer

Objectives: Niraparib has been FDA approved as frontline maintenance therapy (FMT) for advanced-stage ovarian cancer regardless of biomarker status. A recently published preferences study demonstrated that, on average, ovarian cancer patients are willing to pay (WTP) $424 per month for an additional 6 months of progression-free survival (PFS) and $6 per month if the PFS benefit is only 2 months. The objective of this study is to determine whether a niraparib-for-all FMT approach is cost-effective compared to a biomarker-based strategy (BBS) from a patient out-of-pocket (OOP) cost perspective. Methods: A Markov-based decision model simulating the publicly available PRIMA trial results was used to evaluate the cost-effectiveness of two FMT strategies from a patient perspective: (1) niraparib for all (niraparib-for-all) and (2) BBS, defined as niraparib only for deleterious germline/somatic BRCA mutations and homologous recombination deficient (HRD)+ tumors. Monthly OOP drug costs were estimated for commercially insured beneficiaries with ovarian cancer using MarketScan (2014 - 2017) and for Medicare beneficiaries using Surveillance, Epidemiology, and End Results (SEER)-Medicare (2014-2016). The median OOP cost for any PARP inhibitor given during the time period were aggregated. OOP costs fortoxicities were incorporated into the model as emergency department copay costs. For comparison to previously reported patient WTP data, cost-effectiveness was reported as the incremental cost-effectiveness ratio (ICER) in U.S. dollars per quality-adjusted progression-free month (QA-PFM) gained between strategies. Results: The median monthly OOP cost of PARP maintenance was $43 for commercially insured beneficiaries and $694 for Medicare beneficiaries. In the model, niraparib-for-all was more costly ($638 versus $362 for commercial insurance; $5841 vs $1029 for Medicare) and more effective (11-month PFS vs 9.6-month PFS) than BBS. For patients with commercial insurance, niraparib-for-all had an ICER of $200/QA-PFM compared to BBS, and could be considered potentially cost-effective compared to a previously published patient WTP of $424/QA-PFM. For patients with Medicare, niraparib-for-all had an ICER of $3,213/QA-PFM compared to BBS and is not cost-effective. A monthly OOP cost of $90 or less wound render niraparib-for-all cost-effective based on the published WTP threshold. Conclusions: For patients with private insurance, accounting for only OOP costs and assuming a PFS benefit of approximately 6 months, niraparib-for-all could be considered cost-effective, falling within patients’ WTP, with an ICER of $200/QA-PFM compared to BBS. However, for patients with Medicare, both strategies incur considerably higher OOP expenses, and niraparib-for-all cannot be considered cost-effective. These results highlight the stark differences in financial burden on patients depending on their insurance coverage. Future cost-effectiveness studies should incorporate patient OOP costs and willingness to pay so that relative financial burden can be used to facilitate treatment conversations.