Abstract

Background:Treatment options for multiple myeloma (MM) have evolved from chemotherapeutic agents to novel agents, including bortezomib (V), thalidomide (T), and lenalidomide (R). The combination regimens have become standard of care in the first‐line (1L) and second‐line (2L) anti‐MM therapy worldwide. However, there are only few studies focusing on economic impact from these novel treatments.Aims:To evaluate and compare the economic impact, in terms of one‐year healthcare resource utilization (HCRU) and expenditure, among different regimens after the commencement of 1L and 2L anti‐MM therapy.Methods:Patients with newly diagnosed MM (NDMM) during 2009‐2015 were identified from the Taiwan National Health Insurance Research Database. Only those who had received the 1L and 2L anti‐MM therapy respectively since Jun and Dec 2012 (when reimbursement for novel agents started) were enrolled. Treatment regimens were categorized as: V+T/R‐based (as reference for comparison), V‐based, T/R‐based and non‐V/T/R‐based. HCRU, in terms of count of visit to outpatient department (OPD), emergency room (ER), and hospitalization (IPD), related to the treatment categories in a year was compared using multivariable negative binomial regression. The expenditure was analyzed using multivariable generalized linear model with gamma distribution and presented by per patient per month. Two‐stage model was used to handle excess zeros with 95% confidence intervals (CIs) calculated by bootstrapping.Results:A total of 3,434 NDMM patients were identified, and 1,939 and 1,534 patients who had respectively received 1L and 2L anti‐MM therapy were enrolled for HCRU/expenditure analysis. In the 1L patients eligible for AuHSCT (N = 379; 19.5%) and receiving V+T/R‐based regimens, the mean (±SD) number of times visiting OPD, ER and IPD in one year were 32.6 ± 13.6, 1.2 ± 1.6 and 3.0 ± 1.4, respectively. Although the visit frequencies were not significantly different compared to the other regimens, there was a trend toward fewer OPD visits in non‐V/T/R‐based regimens (adj. incidence rate ratio [aIRR] 0.78; P = .07). The OPD expenditure was significantly higher in V+T/R‐based regimens (€2,279 ± 768) than the others, with adj. cost ratios of 0.82, 0.76 and 0.55 for V‐, T‐ and non‐V/T/R‐based regimens, respectively. In the 1L patients ineligible for AuHSCT (N = 1,560) and receiving V+T/R‐based regimens, the number of OPD, ER and IPD visits were 30.7 ± 20.0, 1.9 ± 2.7 and 1.9 ± 2.6, respectively. Significantly fewer OPD and ER visits were observed in non‐V/T/R‐based regimens (aIRR, 0.68 and 0.74; both P<.01). The OPD expenditure were also significantly less than V+T/R‐based regimens (€552 ± 846 vs. €2,301 ± 1,281; adj. cost ratio 0.22, 95% CI 0.18‐0.26). In contrast, IPD expenditure was significantly higher in patients with non‐V/T/R‐based regimens (€4,115 ± 3,273 vs. €1,973 ± 3,362; adj. cost ratio 1.40, 95% CI 1.10‐1.76). For patients who had received 2L anti‐MM therapy, there were significantly more frequent OPD visits in those with V+T/R‐ or V‐based regimens than T‐, R‐ and non‐V/T/R‐based regimens. Nevertheless, more ER and IPD visits were found in patients with R‐based regimens (aIRR 2.06 and 1.99, respectively; both P<.0001), and so as the related expenditure (adj. cost ratio 2.26 [1.29‐3.60] and 1.48 [1.06‐2.03], respectively).Summary/Conclusion:Various anti‐MM regimens containing novel agents have substantially impacted the HCRU and related expenditure, especially in 1L patients ineligible for AuHSCT and in 2L patients. This study provides a rationale for conducting further cost‐effectiveness analysis.

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