United States Payer Perspective Research Articles

Cost-Effectiveness of Lisocabtagene Maraleucel (liso-cel) Versus Axicabtagene Ciloleucel (axi-cel) for Treatment of Relapsed or Refractory (R/R) Large B-Cell Lymphoma (LBCL)

Background: Patients with R/R LBCL after primary treatment have historically had a poor prognosis and few treatment options. Prior research found that the chimeric antigen receptor (CAR) T cell therapy, axi-cel may be cost-effective in comparison with salvage chemotherapy. Liso-cel, a CD19-directed CAR T cell therapy, may have comparable survival and a better safety profile than axi-cel, representing a valuable treatment option in CD19-directed CAR T cell therapy.We developed an economic model assessing cost-effectiveness of liso-cel versus axi-cel for patients with R/R LBCL after ≥ 2 lines of therapy from a commercial United States (US) payer perspective.Methods: Patients entered the model at leukapheresis and were subsequently stratified according to their pre-infusion experience (ie, whether or not they received the CAR T cell infusion due to death or other reasons). The model used a partitioned survival approach to estimate health state occupancy (progression-free survival [PFS], progressive disease, and death) and calculate incremental cost-effectiveness ratios using life-years and quality-adjusted life-years (QALY) over a lifetime horizon.Survival curves from clinical trials of CAR T cell therapies in R/R LBCL exhibit a plateau, indicating a fraction of patients may achieve durable remission after treatment. Parametric survival models that incorporate cure assumptions or hazards that converge to general population mortality have been widely accepted in R/R LBCL by health technology assessment bodies for CAR T cell therapies. Parametric mixture-cure modeling was used to extrapolate overall survival (OS) and PFS data from TRANSCEND NHL 001 (TRANSCEND [NCT02631044]) for liso-cel and ZUMA-1 (NCT02348216) for axi-cel. Survival of cured patients was modeled per the age- and sex-adjusted general population, adjusted by an excess mortality risk. Survival of non-cured patients was estimated using parametric distributions. Given meaningful differences in the study design, eligibility criteria, and baseline patient characteristics between the two trials, comparative efficacy (OS, PFS) and safety (adverse events [AE]) were estimated using matching-adjusted indirect comparison (MAIC). EQ-5D-5L data from TRANSCEND were used to estimate utilities for health states and AEs. Costs included pretreatment (leukapheresis, bridging therapy, and lymphodepleting chemotherapy), acquisition and administration of CAR T-cell therapies and subsequent-line treatments, all-grade AE management, post-infusion hospitalization, monitoring, and end-of-life care.Uncertainty was examined using sensitivity and scenario analyses. Scenarios matching TRANSCEND with ZUMA-1 on bridging therapy and assuming all patients received the CAR T cell infusion were also conducted to explore the impact of potential bias resulting from trial design differences.Results: Following matching and adjustment, the liso-cel effective sample size for indirect comparison was 99 patients. Patients receiving liso-cel achieved improved 5-year survival compared with axi-cel (45% vs 41%, respectively). In the base case, liso-cel dominated axi-cel with nearly equivalent QALYs (0.02) and cost savings of $75,063 (Table 1). The favorable safety profile of liso-cel, including lower rates of cytokine release syndrome and neurological events, was reflected in cost savings (−$35,464) and QALY gains (0.05) when compared with axi-cel. In probabilistic sensitivity analyses, the probability that liso-cel was more effective and less costly than axi-cel was 52% and the probability that liso-cel was cost-effective at a threshold of $100,000 was 78%. Key drivers of cost-effectiveness included the OS cure fraction and the percent of patients experiencing grade 3/4 AEs for axi-cel (Figure). Additionally, 2 scenarios that sought to adjust for differences in trial design both predicted QALY gains and cost savings for liso-cel versus axi-cel (Table 2).Conclusions: This analysis estimated that liso-cel is cost-effective compared with axi-cel from a commercial US payer perspective, generating similar QALYs at lower cost, partly owing to a favorable safety profile associated with liso-cel. [Display omitted] DisclosuresParker: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Liu: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Franco-Villalobos: Bristol Myers Squibb: Consultancy; Other pharmaceutical companies: Consultancy. Proskorovsky: Evidera: Current Employment. Keating: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company.

Cost Effectiveness Analysis of Venetoclax Plus Azacitidine Versus Azacitidine in Newly Diagnosed Adult Patients with Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy from a United States Payer Perspective

Background: The phase 3 VIALE-A trial (NCT02993523) demonstrated that venetoclax plus azacitidine (VEN+AZA) improved overall survival (OS) and led to higher remission rates compared with AZA monotherapy, in patients with newly diagnosed (ND) acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. Based on the results from VIALE-A, VEN+AZA received full United States (US) Food and Drug Administration approval in October 2020 for patients with ND AML aged ≥75 years, or who were ineligible for intensive induction chemotherapy due to comorbidities. This study aims to assess the long-term cost-effectiveness value of the VEN+AZA regimen from the VIALE-A trial from a US third-party payer perspective.Methods: A partitioned survival model with a 28-day cycle was developed to estimate costs and outcomes of treatment with VEN+AZA vs. AZA among patients with ND AML, who are ineligible for intensive chemotherapy, over a lifetime time horizon. The model included three health states: event-free survival (EFS), progressive/relapsed disease, and death. Within the EFS state, patients were further partitioned into time spent in complete remission (CR) or CR with incomplete marrow recovery (CRi), and time spent in non-CR/CRi. Efficacy inputs (OS, EFS, and CR/CRi rate) for both treatment arms were estimated using VIALE-A data. Best-fit parametric models per Akaike information criterion (AIC) were used to extrapolate OS until it reached EFS, and extrapolate EFS for each treatment until Year 5. Patients who remained in EFS after Year 5 were considered cured, and were assumed to have the same mortality as the US general population. Mean time on treatment (ToT) for both regimens was based on the time observed in VIALE-A. The costs for drug acquisition, drug administration for initial and subsequent treatments, subsequent stem cell transplant procedures, adverse events (AEs), and healthcare resource utilization (HRU) associated with each health state were obtained from the literature or publicly available data. All costs were inflated to 2021 US dollars. Utilities for each health state were estimated using EuroQol-5 dimension-5 level (EQ-5D-5L) data from VIALE-A, based on the US crosswalk value set. Information on disutilities due to Grade 3/4 AEs were obtained from the literature. Incremental cost-effectiveness ratios (ICERs) per life year (LY) and quality-adjusted life year (QALY) gained were estimated. Deterministic sensitivity analyses (DSA), scenario analyses and probabilistic sensitivity analyses (PSA) were performed to assess the robustness of the results.Results: Over a lifetime time horizon, compared with AZA, VEN+AZA was associated with an increase of 1.89 LYs (1.10 vs. 2.99, respectively) and 1.45 QALYs (0.84 vs. 2.30, respectively). Patients in the VEN+AZA arm incurred higher total costs ($250,486 vs. $110,034 for patients in the AZA arm). The ICER for VEN+AZA vs. AZA was estimated to be $74,141 per LY gained, and $96,579 per QALY gained. Results from the DSA and scenario analyses supported the base-case findings, with ICERs ranging from $60,922 to $138,554 per QALY gained. The results were most sensitive to alternative approaches for ToT estimation, subsequent treatment HRU costs, cure time point, and the extrapolation approach for EFS. Results from PSA showed that compared with AZA, VEN+AZA was cost-effective in 99.9% of cases at a willingness-to-pay (WTP) threshold of $150,000.Conclusions: Compared with AZA monotherapy, VEN+AZA results in a favorable ICER of $96,579 per QALY gained over a lifetime time horizon. The base-case results suggest that, compared with AZA, VEN+AZA is a cost-effective strategy based on a WTP threshold of $150,000 per QALY gained. Sensitivity analyses support the base-case results. Thus, VEN+AZA offers a cost-effective strategy in the treatment of patients with ND AML who are ineligible for intensive chemotherapy from a US third-party payer perspective. DisclosuresPratz: Agios: Consultancy; Abbvie: Consultancy, Honoraria, Research Funding; University of Pennsylvania: Current Employment; BMS: Consultancy, Honoraria; Novartis: Consultancy; Astellas: Consultancy, Honoraria, Research Funding; Cellgene: Consultancy, Honoraria; Millenium: Research Funding. Chai: Analysis Group, Inc.: Consultancy, Current Employment; Genentech, Inc.: Consultancy. Yin: Analysis Group, Inc.: Consultancy, Current Employment; Genentech, Inc.: Consultancy. Nie: Analysis Group, Inc.: Consultancy, Current Employment; Genentech, Inc.: Consultancy. Montez: Genentech, Inc: Current Employment, Other: May hold equity. Iantuono: Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Downs: Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Ma: Genentech, Inc.: Current Employment, Other: May hold equity.

Cost per Responder Analysis for Pegcetacoplan and Eculizumab in the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria

Background: In the absence of clinical guidelines for paroxysmal nocturnal hemoglobinuria (PNH), the European Society for Blood and Marrow Transplantation (EBMT) developed categories for classifying hematological response to complement inhibitor treatment (per Risitano AM, et al. Front Immunol. 2019;10:1157). These categories are: complete (no transfusions, normal stable Hb, and no evidence of hemolysis); major (no transfusions, normal Hb, but evidence of residual intravascular/extravascular hemolysis); good (no transfusions, but persistent chronic mild anemia); partial (persistent chronic moderate anemia and/or occasional red blood cell transfusions); minor (3-6 transfusions every 6 months); no response (>6 transfusions every 6 months). In the phase 3 PEGASUS trial (NCT03500549), 41 and 39 patients previously treated with C5 inhibitors with Hb<10.5 were randomized for treatment with pegcetacoplan or eculizumab, respectively. In a post hoc analysis of the 16-week PEGASUS data, patients were classified by EBMT category to compare hematologic responses of each treatment (per Risitano AM, et al. Blood. 2020; 136(Suppl1):44-5).Objectives: The objective of this study was to evaluate the number needed to treat (NNT) and treatment costs of pegcetacoplan and eculizumab using EBMT response criteria.Methods: The analysis used a United States payer perspective and included drug acquisition costs and associated intravenous or subcutaneous administration costs. Drug costs were calculated using dosages for each treatment as given in the PEGASUS trial, including dosage and frequency escalations. Weekly dosages were multiplied by each drug's wholesale acquisition costs from RedBook (2020 United States dollars). We calculated the following outcomes over 16 weeks and by treatment: NNT to achieve each level of response, mean cost per treated patient, total costs, mean cost per treated patient per week, cost per complete responder, cost per good responder, and cost spent on patients with partial/minor/no response.Additional analyses were conducted to calculate outcomes for treatment-naïve patients treated with pegcetacoplan using similar post hoc analyses of combined 16-week data from the phase 1b-2a PADDOCK/PALOMINO (NCT02588833 and NCT03593200) trials.Results: Results are summarized in Table 1. Based on response rates, patients with PNH treated with pegcetacoplan had a lower NNT (1.4) to achieve good-to-complete response compared with eculizumab (19.5). NNT to achieve complete response was 2.6 for pegcetacoplan; no patients treated with eculizumab achieved complete response at 16 weeks.For the PEGASUS trial, mean cost per treated patient was $182,762 (with C5 inhibitor loading dose) and $176,504 over 16 weeks for pegcetacoplan and eculizumab, respectively. Among the 41 pegcetacoplan-treated patients, the total costs were $7,493,256 over 16 weeks. Of 41 pegcetacoplan patients, 16 achieved a complete response, and their total costs were $2.9 million over the 16-week period ($468,328 per complete responder). The total 16-week costs were $2.6 million for 14 patients with good response ($535,233 per good responder). For the 39 eculizumab-treated patients, total 16-week costs were $6,883,637. Two of 39 patients achieved a good response, and their total costs were $353,007 ($3,441,818 per good responder). Of the 39 patients, 37 achieved partial, minor, or no response or discontinued treatment, for a total cost of $6.5 million. This resulted in 95% of total treatment costs being spent on inadequate or no response in the eculizumab arm compared with 27% in the pegcetacoplan arm.For treatment-naïve patients treated with pegcetacoplan, the mean cost per complete responder ($347,339) and per good responder ($496,198) over 16 weeks were both lower than the cost per responder for patients treated with either pegcetacoplan or eculizumab after previous C5 inhibitor treatment.Conclusion: Based on the treatment response observed in PEGASUS, the NNT and cost to achieve good or good-to-complete response was lower for pegcetacoplan-treated patients than eculizumab-treated patients, according to EBMT consensus-based hematological response categories. PNH treatment with pegcetacoplan allowed more patients to achieve EBMT defined good-to-complete response and better overall response and is a more efficient use of spending with lower cost per responder in all response categories. [Display omitted] DisclosuresAnderson: Apellis: Consultancy, Research Funding. Talbird: Apellis: Consultancy, Research Funding. Fishman: Apellis: Current Employment, Current equity holder in publicly-traded company.

R codes and Microsoft Excel-based calculations related to manuscript: Quantifying the economic burden of unintended pregnancies due to drug-drug interactions with hormonal contraceptives from the United States payer perspective

R codes and Microsoft Excel-based calculations related to manuscript: Quantifying the economic burden of unintended pregnancies due to drug-drug interactions with hormonal contraceptives from the United States payer perspective

Cost-Effectiveness of Frontline Treatment for Advanced Renal Cell Carcinoma in the Era of Immunotherapies.

Background: Recent randomized controlled trials have demonstrated that immune checkpoint inhibitors (ICIs) improve patient outcomes, but whether these novel agents are cost-effective for untreated advanced renal cell carcinoma (aRCC) remains unclear.Materials and Methods: A microsimulation model was created to project the healthcare costs and outcomes of six strategies (lenvatinib-plus-pembrolizumab, nivolumab-plus-cabozantinib, nivolumab-plus-ipilimumab, pembrolizumab-plus-axitinib, avelumab-plus-axitinib, and sunitinib monotherapy) for patients with aRCC. Transition probability of patients was estimated from CLEAR, CheckMate 9ER, CheckMate 214, KEYNOTE-426, JAVELIN Renal 101, and other data sets by using parametric survival modeling. Lifetime direct medical costs, life years (LYs), quality-adjusted LYs (QALYs), and incremental cost-effectiveness ratios (ICERs) were estimated from a United States payer perspective. One-way and probabilistic sensitivity analyses were performed, along with multiple scenario analyses, to evaluate model uncertainty.Results: Of the six competing strategies, nivolumab-plus-cabozantinib yielded the most significant health outcomes, and the sunitinib strategy was the least expensive option. The cost-effective frontier consisted of the nivolumab-plus-cabozantinib, pembrolizumab-plus-axitinib, and sunitinib strategies, which displayed the ordered ICERs of $81282/QALY for pembrolizumab-plus-axitinib vs sunitinib and $453391/QALY for nivolumab-plus-cabozantinib vs pembrolizumab-plus-axitinib. The rest of the strategies, such as lenvatinib-plus-pembrolizumab, nivolumab-plus-ipilimumab, and avelumab-plus-axitinib, were dominated. The cost of sunitinib drove the model most influentially.Conclusions: For aRCC, the pembrolizumab-plus-axitinib strategy is likely to be the most cost-effective alternative at the willingness-to-pay threshold of $100,000.

Cost-Effectiveness of Daratumumab Plus Lenalidomide Plus Dexamethasone Versus Lenalidomide Plus Dexamethasone for Treatment of Patients with Multiple Myeloma Who Have Received at Least One Prior Therapy: An Analysis of the Pollux Trial

Background: Daratumumab has been approved in the United States (US) for patients with multiple myeloma (MM). In the POLLUX trial of MM patients who had received at least one prior line of therapy, daratumumab plus lenalidomide plus dexamethasone (DRd) showed significant improvements in efficacy compared to lenalidomide plus dexamethasone (Rd) (Dimopoulos, NEJM, 2016). However, limited information is available on the cost-effectiveness of DRd. A decision-analytic model was developed to consider direct medical costs from a US payer perspective and to explore the cost-effectiveness of DRd versus Rd in previously treated MM and in a subgroup of patients with only one prior line of therapy (first relapse).Methods: The model structure took the form of a partitioned survival analysis with three health states: progression-free survival (PFS), progressive disease, and death. A range of parametric survival functions were fitted to patient-level PFS data from the POLLUX trial with (1) selection based on the Akaike and Bayesian information criteria and (2) priority given to the external validity of extrapolations with conservative survival projections. Diagnostic plots were used to assess the validity of the proportional hazards assumption between DRd and Rd. Due to the number of data events and the follow-up time of the trial, overall survival (OS) was not estimated via parametric extrapolation of trial data but rather by its relationship to PFS, based on a retrospective analysis of approximately 23,000 MM patients (Felix, BMC Cancer, 2013). Drug costs were taken from wholesale acquisition costs (Red Book, 2017). Dosing information was extracted from the POLLUX trial. Drug-administration costs were sourced from Centers for Medicare and Medicaid Services, 2017 and health-state costs, subsequent therapy utilization patterns, and utility weights were sourced from published literature. Cost-effectiveness was estimated over a lifetime horizon, with costs and outcomes discounted at 3% per annum. Incremental cost-effectiveness ratios were calculated, specifically the incremental cost per quality-adjusted life-year (QALY) gained and the incremental cost per life-year gained. One-way and probabilistic sensitivity analyses (PSA) were conducted, as well as scenario analyses around parametric curve selection. All analyses were performed for the overall trial population and for the subgroup of patients with a first relapse.Results: The best-fit, conservative parametric function for all patients was Weibull and for first relapse patients, exponential. All results were presented as discounted values (Table 1). In the overall trial population, the mean PFS was estimated to be 4.12 and 1.84 years for DRd and Rd, respectively. DRd resulted in an average of 4.24 additional life-years (8.43 life-years - 4.19 life-years) and 3.66 additional QALYs (7.12 QALYs - 3.46 QALYs) over Rd. Costs increased for DRd; the majority were related to drug acquisition of daratumumab and to longer time on treatment due to a longer PFS period. Incremental cost-effectiveness ratios in the overall trial population were $153,110 per life-year gained and $177,499 per QALY gained. Overall, survival (life-years), PFS, and QALYs were greater in the first relapse subgroup for both DRd and Rd than in the overall trial population. Mean PFS was estimated to be 5.02 and 2.09 years for DRd and Rd, respectively. DRd resulted in an average of 4.83 additional life-years (9.51 life-years - 4.68 life-years) and 4.17 additional QALYs (8.06 QALYs - 3.89 QALYs) over Rd. Incremental cost-effectiveness ratios in the first relapse subgroup were $165,167 per life-year gained and $191,024 per QALY gained. One-way and scenario analyses indicated the most sensitive parameters to be PFS distribution, the relationship between PFS and OS, discount rates, utility weights, and drug-acquisition costs. PSA was summarized in the form of scatter plots and cost-effectiveness acceptability curves.Conclusion: Based on the range of plausible cost-effectiveness ratio thresholds ($183,000-$264,000 per life-year gained and $109,000-$297,000 per QALY gained) reported in the 2003 cost-effectiveness decision rules for the US (Braithwaite, Med Care, 2008), this analysis suggests that DRd is cost-effective compared to Rd in the treatment of previously treated MM. DRd offers a clinically superior and cost-responsible treatment option for previously treated MM. [Display omitted] DisclosuresMaiese:Janssen: Employment; Johnson & Johnson, LLC: Equity Ownership. Graham:Janssen: Research Funding; RTI Health Solutions: Employment. Hawe:RTI Health Solutions: Employment; Janssen: Research Funding. Le Moine:RTI Health Solutions: Employment; Janssen: Research Funding. Abraham:Janssen Oncology (Johnson & Johnson, LLC): Consultancy. Senbetta:Johnson & Johnson, LLC: Equity Ownership; Janssen: Employment.

Cost-Effectiveness of Daratumumab Plus Bortezomib and Dexamethasone Versus Bortezomib Plus Dexamethasone for Treatment of Patients with Multiple Myeloma Who Have Received at Least One Prior Therapy: An Analysis of the Castor Trial

Background: Daratumumab has recently been approved in the United States (US) for patients with multiple myeloma (MM). In the CASTOR clinical trial of patients who had received at least one prior line of therapy, daratumumab plus bortezomib and dexamethasone (DVd) showed significant improvements in clinical efficacy compared to bortezomib plus dexamethasone (Vd) (Palumbo, NEJM, 2016). However, limited information is available on the cost-effectiveness of DVd. A decision-analytic model was developed to consider direct medical costs from a US payer perspective and to explore the cost-effectiveness of DVd versus Vd in previously treated MM and in a subgroup of patients with only one prior line of therapy (first relapse).Methods: The model structure took the form of a partitioned survival analysis with three health states: progression-free survival (PFS), progressive disease, and death. A range of parametric survival functions were fitted to patient-level PFS data from the CASTOR trial with (1) selection based on the Akaike and Bayesian information criteria and (2) priority given to the external validity of extrapolations with conservative survival projections. Diagnostic plots were used to assess the validity of the proportional hazards assumption between DVd and Vd. Due to the number of data events and the follow-up time of the trial, overall survival (OS) was not estimated via parametric extrapolation of trial data but rather by its relationship to PFS, based on a retrospective analysis of approximately 23,000 MM patients (Felix, BMC Cancer, 2013). Drug costs were taken from wholesale acquisition costs (Red Book, 2017). Dosing information was extracted from the CASTOR trial. Drug-administration costs were sourced from Centers for Medicare and Medicaid Services, 2017 and health-state costs, subsequent therapy utilization patterns, and utility weights were sourced from published literature. Cost-effectiveness was estimated over a lifetime horizon, with costs and outcomes discounted 3% per annum. Incremental cost-effectiveness ratios were calculated, specifically the incremental cost per quality-adjusted life-year (QALY) gained and the incremental cost per life-year gained. One-way and probabilistic sensitivity analyses were conducted, as well as scenario analyses around parametric curve selection. All analyses were performed for the overall trial population and for the subgroup of patients with a first relapse.Results: The best-fit, conservative parametric function for all patients was the generalized gamma and for first relapse patients, Weibull. All results were presented as discounted values (Table 1). In the overall trial population, the mean PFS was estimated to be 1.50 and 0.67 years for DVd and Vd, respectively. DVd resulted in an average of 1.92 additional life-years (3.50 life-years - 1.58 life-years) and 1.62 more QALYs (2.88 QALYs - 1.26 QALYs) over Vd. Costs increased for DVd; the majority were related to drug acquisition of daratumumab and to longer time on treatment due to a longer PFS period. Incremental cost-effectiveness ratios in the overall population were $94,446 per life-year gained and $112,338 per QALY gained. Overall, survival (life-years), PFS, and QALYs were greater in the first relapse subgroup for both DVd and Vd than in the overall trial population. Mean PFS was estimated to be 2.56 and 0.80 years for DVd and Vd, respectively. DVd resulted in an average of 3.84 more life-years (5.74 life-years - 1.90 life-years) and 3.23 more QALYs (4.78 QALYs - 1.55 QALYs) over Vd. Incremental cost-effectiveness ratios in the first relapse subgroup were $68,628 per life-year gained and $81,681 per QALY. One-way and scenario analyses indicated the most sensitive parameters to be PFS distribution, the relationship between PFS and OS, discount rates, utility weights, and drug-acquisition costs. PSA was summarized in the form of scatter plots and cost-effectiveness acceptability curves.Conclusion: Based on the range of plausible cost-effectiveness ratio thresholds ($183,000-$264,000 per life-year gained and $109,000-$297,000 per QALY gained) reported in the 2003 cost-effectiveness decision rules for the US (Braithwaite et al., Med Care, 2008), this analysis suggests that DVd is cost-effective compared to Vd in the treatment of previously treated MM. DVd offers a clinically superior and cost-responsible treatment option for previously treated MM. [Display omitted] DisclosuresMaiese:Johnson & Johnson, LLC: Equity Ownership; Janssen: Employment. Graham:RTI Health Solutions: Employment; Janssen: Research Funding. Hawe:RTI Health Solutions: Employment; Janssen: Research Funding. Le Moine:Janssen: Research Funding; RTI Health Solutions: Employment. Abraham:Janssen Oncology (Johnson & Johnson, LLC): Consultancy. Senbetta:Janssen: Employment; Johnson & Johnson, LLC: Equity Ownership.

Evaluation of Tucatinib in HER2-Positive Breast Cancer Patients With Brain Metastases: A United States-Based Cost-Effectiveness Analysis

PurposeTo evaluate the cost-effectiveness of tucatinib in human epidermal growth factor receptor 2 (HER2)-positive breast cancer (BC) patients with brain metastases (BMs) and the subgroup of active BMs from the United States (US) payer perspective. Materials and MethodsA 3-state Markov model was developed to compare the cost-effectiveness of 2 regimens in HER2-positive BC patients with BMs: (1) tucatinib, trastuzumab, and capecitabine (TTC); (2) placebo, trastuzumab, and capecitabine (PTC). And subgroup analysis of active BMs was also performed. Lifetime costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratio (ICER) and incremental net-health benefit (INHB) were estimated. The willingness-to-pay (WTP) threshold was $200,000/QALY. The robustness of the model was tested by sensitivity analyses. Additional scenario analysis was also performed. ResultsCompared with PTC, the ICER yielded by TTC was $418,007.01/QALY and the INHB was -1.08 QALYs in patients with BMs. In the subgroup of active BMs, the ICER and the INHB were $324,465.03/QALY and -0.71 QALY, respectively. The results were most sensitive to the cost of tucatinib. Probabilistic sensitivity analyses suggested that the cost-effective probability of TTC was low at the current WTP threshold in the patients with BMs and the subgroup of active BMs. ConclusionTucatinib is unlikely to be cost-effective in HER2-positive BC patients with BMs from the US payer perspective but shows better economics in patients with active BMs. Selecting a favorable population, reducing the price of tucatinib or offering appropriate drug assistance policies might be considerable options to optimize the cost-effectiveness of tucatinib.

HT2 Evaluation of Tucatinib for HER2-Positive Breast Cancer Patients with Brain Metastases: A United States-Based Cost-Effectiveness Analysis

To evaluate the cost-effectiveness of tucatinib in human epidermal growth factor receptor 2 (HER2)-positive breast cancer (BC) patients with brain metastases (BMs) and the subgroup of active BMs from the United States (US) payer perspective. A three-state Markov model was developed to compare the cost-effectiveness of tucatinib, trastuzumab and capecitabine (TTC) with placebo, trastuzumab and capecitabine (PTC) in HER2-positive BC patients with BMs;subgroup analysis of active BMs was also performed.Pseudo-individual patient data were generated from digitized Kaplan-Meier curves.Costs were derived from official databases and the literature.Health state utility values were consistent with published literature and adjusted by adverse events.Lifetime costs,quality-adjusted life years (QALYs),incremental cost-effectiveness ratio (ICER) and incremental net health benefit (INHB) were estimated.The willingness-to-pay (WTP) threshold was $200,000/QALY.The robustness of the model was tested by sensitivity analysis and scenario analyses were also performed. In patients with BMs,the PTC and TTC strategies cost $87,905.23 and $503,637.21,yielding 0.68 and 1.68 QALYs,respectively.While in the subgroup of active BMs,the two strategies cost $81,968.50 and $451,699.62 and the QALYs were 0.61 and 1.75,respectively.The ICERs yielded by TTC were $418,007.01/QALY and $324,465.03/QALY, and INHBs were -1.08 QALYs and -0.71 QALY, compared with PTC in these two groups,respectively.The results were most sensitive to the cost of tucatinib.Probabilistic sensitivity analysis suggested that the probability of TTC being cost-effective was low at the current WTP threshold in the patients with BMs and the subgroup of active BMs. The additional using tucatinib (TTC) is unlikely to be cost-effective in HER2-positive BC patients with BMs from the US payer perspective,but shows a better economics in patients with active BMs.Therefore, selecting favorable population would be a good way to optimize the cost-effectiveness of tucatinib.To meet the economical demands of public health,it may be a preferable option to reduce the price of tucatinib or offer appropriate drug assistance policies.

PCN43 Cost-Effectiveness of Nivolumab for the Treatment of Advanced Esophageal Squamous Cell Carcinoma Refractory or Intolerant to Previous Chemotherapy: A United-States Payer Perspective

To evaluate the cost-effectiveness of nivolumab versus taxane-based chemotherapy (TbC) for previously treated advanced esophageal squamous cell carcinoma (aESCC) patients from a United States (US) payer perspective using data with minimum 36-months follow-up from the ATTRACTION-3 trial.

Cost-effectiveness (CE) analysis of pembrolizumab as first-line (1L) treatment for recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) in the United States (US).

e18013 Background: Pembrolizumab received FDA approval in 1L R/M HNSCC as monotherapy (P) in patients with combined positive score (CPS) ≥1, and in combination with platinum + 5-Fluorouracil (5-FU) chemotherapy (P + C) in the total population, based on KEYNOTE 048 (KN048) trial. Taking a US payer perspective, we evaluated the CE of P and P + C versus KN048 trial comparator cetuximab + platinum + 5-FU (EXTREME regimen), and other comparators commonly used in the US (i.e. cisplatin+docetaxel+cetuximab [TPEx regimen], platinum + paclitaxel/taxane [Pt + T] and platinum+5-FU [Pt + F]). Methods: A 3-state partitioned-survival model was used to project costs and outcomes over 20 years with a 3% annual discount rate. For P, P + C and EXTREME, health state occupancy was based on KN048 trial results with long-term parametric extrapolation of progression-free survival (PFS) and overall survival (OS) guided by statistical criteria. A network meta-analysis (NMA) was applied to the P and P + C interventions in KN048 to project the PFS and OS outcomes for other comparators. The time on treatment (ToT) for P, P+C and EXTREME was derived from the KN048 trial, whereas for other comparators, ToT was approximated by PFS. Costs of 1L and subsequent treatments, disease management, adverse event, and terminal care were included. Utilities were derived using the EuroQoL five-dimension data from KN048 and a US value set. Results: CE ratios, total and incremental costs and quality-adjusted life years (QALYs) from the base case analysis are summarized in the table below. P was more effective and less costly (i.e. dominant) versus EXTREME and TPEx. P + C was highly cost-effective versus EXTREME ($1,789/QALY) and TPEx regimen (dominant). CE ratios for both P and P + C versus all comparators were below a $100,000/QALY willingness-to-pay threshold. Probabilistic sensitivity analysis also showed a 100% CE probability for P and P + C at the $100,000/QALY threshold. Conclusions: P and P + C are cost-effective treatment options in the US with CE ratios below $100,000/QALY gained versus commonly used therapies in 1L R/M HNSCC.[Table: see text]

PDG20 Cost-effectiveness of Nivolumab in Patients with Pre-treated Advanced Renal Cell Carcinoma (aRCC) in the United States: Impact of >5 Years of Follow-up Data from Checkmate 025

This study compares cost-effectiveness (CE) results when using data from the initial and latest database locks (DBLs) of CheckMate 025 (NCT01668784), a randomized phase 3 trial investigating nivolumab versus everolimus in pre-treated aRCC. The minimum follow-up was 14 months (initial) and 64 months (latest) in these DBLs, respectively. We developed a 3-state (progression-free, progressive disease and death) partitioned survival model to evaluate the CE of nivolumab versus everolimus from a United States payer perspective, with a 25-year time horizon and 3% discounting for costs (2020 US$) and outcomes. Key trial data inputs from the DBLs were progression-free survival (PFS), overall survival (OS), time-to-treatment discontinuation (TTD), treatment response, patient utilities, and subsequent systemic treatment. For comparison, only the inputs derived from the trial were changed in this analysis. Using longer term data resulted in a modest reduction in the incremental cost-utility ratio (ICUR) for nivolumab versus everolimus ($70,490/QALY [incremental cost-effectiveness ratio (ICER): $54,240/LY] versus $72,389/QALY [$55,946/LY] using the initial DBL). The initial DBL had the same (best-fitting) functional forms for survival extrapolations as the latest DBL but underestimated incremental LY and QALY gains with nivolumab by 11.4% and 11.0%, respectively. Based on OS extrapolations from the initial DBL, total LYs for nivolumab and everolimus were 3.1% and 0.6% lower, respectively. Changes in incremental costs (+8.6% with latest DBL) were mainly driven by differences in the use of subsequent treatment and the types of agents given between the two DBLs. Health state utility values, treatment response, and TTD curves between DBLs had limited ICUR influence. The initial DBL conservatively underestimated the incremental lifetime survival benefit for nivolumab versus everolimus when compared with estimates using the latest DBL from CheckMate 025. Higher total incremental costs observed with longer follow-up were offset by increased LY and QALY gains between DBLs.

Cost-Effectiveness of Polatuzumab Vedotin Plus Bendamustine-Rituximab for Transplant-Ineligible Relapsed/Refractory Diffuse Large B-Cell Lymphoma in the United States

Introduction Polatuzumab vedotin (pola), a first-in-class CD79b-targeted antibody-drug conjugate, was recently approved by the United States (US) Food and Drug Administration (June 2019) in combination with bendamustine-rituximab (BR) for the treatment of adults with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) after at least two prior therapies. Approval was based on the results of a randomized Phase II trial (GO29365/NCT02257567) in which pola+BR demonstrated clinically meaningful improvements in complete response (CR) rates (40.0% vs 17.5%, respectively), progression-free survival (PFS; hazard ratio [HR], 0.36; 95% confidence interval [CI]: 0.21-0.63) and overall survival (OS; HR, 0.42; 95% CI: 0.24-0.75) versus BR alone in transplant-ineligible R/R DLBCL (Sehn et al. J Clin Oncol 2020). Objective To evaluate the cost-effectiveness of pola+BR in R/R DLBCL from a US payer's perspective, using 140mg and 30mg single-dose vials of pola and GO29365/NCT02257567 study data. Methods A partitioned survival model was developed to estimate the economic and health-related quality of life impact of pola+BR versus BR alone in transplant-ineligible R/R DLBCL. A lifetime horizon and 3% discount rate were considered for costs and outcomes. Clinical data (PFS, OS, drug utilization, treatment duration and adverse events [AEs]) were sourced from the GO29365/NCT02257567 study. Costs included drug acquisition/administration, AE management, PFS routine care, progressive disease (PD) medical care and end-of-life care. Quality-adjusted life-years (QALYs) were calculated using published utility estimates (Tisagenlecleucel, NICE Report 2019). The base-case analysis assumed OS was informed by PFS (extrapolated) and used a mixture cure rate model to estimate the proportion of putative long-term survivors (pola+BR: 21%; BR: 0% [Sehn et al. ICML 2019]). Deterministic and probabilistic sensitivity analyses (PSA) and scenario analyses (mixture cure rate model with OS modelled separately from PFS, and standard parametric survival models) were performed to assess uncertainty. Results The total cost of pola+BR ($210,418) was $92,329 higher than BR ($118,089), primarily due to higher drug/administration costs ($139,717 vs $47,944, respectively). Pola+BR reflected cost-savings for PD medical care (-$4,849) and end-of-life care (-$2,248) versus BR. Costs due to AE management were $6,484 higher for pola+BR ($21,989) than BR ($15,505). Treatment with pola+BR was associated with an additional 2.57 QALYs versus BR. The incremental cost-effectiveness ratio (ICER) was $35,864/QALY gained. The ICER was most sensitive to survival modelling assumptions, the discount rate considered for effectiveness, and utility in PFS. Results of the PSA showed that pola+BR was cost-effective versus BR in 68% and 97% of cases using a willingness-to-pay threshold of $50,000 and $100,000, respectively. Scenario analyses resulted in ICERs ranging from $41,442/QALY (mixture cure with OS modelled separately from PFS) to $72,166/QALY (standard parametric). Conclusions This updated cost-effectiveness analysis includes 30mg single-dose vials of pola in addition to the original 140mg vials, allowing more precise weight-based dosing. Driven predominantly by higher PFS/OS and the higher estimated proportion of long-term survivors, results suggest that in the US, pola+BR versus BR is cost-effective for the treatment of adults with transplant-ineligible R/R DLBCL. Disclosures Betts: Genentech, Inc.: Research Funding. Felizzi:F. Hoffmann-La Roche Ltd: Current Employment. Dieye:Analysis Group: Current Employment. Li:F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Genentech Inc.: Current Employment. Schulz:F. Hoffmann-La Roche Ltd: Current equity holder in private company; Genentech, Inc.: Current Employment. Hong:Genentech, Inc.: Current Employment; Karuna Therapeutics: Current equity holder in publicly-traded company; Y-mAbs Therapeutics: Current equity holder in publicly-traded company; Astellas Pharma: Ended employment in the past 24 months. Masaquel:Analysis Group: Current Employment.

Cost-effectiveness of filgrastim-sndz as primary prophylaxis (PP) versus secondary prophylaxis (SP) to prevent chemotherapy-induced febrile neutropenia (FN) in breast cancer patients at intermediate risk.

73 Background: According to clinical practice guidelines, the threshold for routine myeloid growth factor (MGF) PP is a high risk (&gt;20%) of developing FN. However, in response to the COVID-19 pandemic, a recent recommendation expands this threshold for using MGF PP to include patients at intermediate risk (10-20%) of developing FN, with the goal of reducing emergency room and hospital visits. Patients with breast cancer receiving potentially curative chemotherapy consisting of docetaxel or paclitaxel (every 21 days) are at an intermediate risk (10-20%) of developing FN. This study evaluates the cost-effectiveness of PP vs. SP using a biosimilar MGF, filgrastim-sndz, in early-stage breast cancer patients at intermediate risk of FN. Methods: A Markov model with a lifetime horizon was constructed to evaluate the total costs and clinical outcomes when using filgrastim-sndz as PP vs. SP in 56 year old early-stage breast cancer patients receiving adjuvant docetaxel (following doxorubicin/cyclophosphamide) every 3 weeks for 4 cycles. Patients had ≥1 FN risk factor (i.e., recent surgery) without the receipt of anti-HER2 therapy, representing a 16% baseline FN risk. Average Sales Price (ASP) calculated from the Centers for Medicare &amp; Medicaid Services July 2020 ASP Drug Pricing File was used as the filgrastim-sndz cost. Incremental cost-effectiveness ratios (ICERs) were calculated for cost per FN event avoided, life-year saved (LYS), and quality-adjusted life-year (QALY) gained from a United States payer perspective. Deterministic and probabilistic sensitivity analyses (PSA) were conducted. Results: Filgrastim-sndz as PP vs. SP provided an additional 0.102 FN events avoided, 0.065 LYS, and 0.056 QALYs at an incremental cost of $2,106. The ICERs were $20,656, $32,624 and $37,333 for cost per FN event avoided, cost per LYS, and cost per QALY gained, respectively. In the PSA, the likelihood of cost-effectiveness at a willingness-to-pay (WTP) threshold of $50,000 per QALY gained was 71.3%. Conclusions: For early-stage breast cancer patients at intermediate risk of FN receiving adjuvant docetaxel with 1 or more risk factors, PP with filgrastim-sndz compared to SP is cost-effective based on a WTP threshold of $50,000/QALY. [Table: see text]

Cost-effectiveness analysis of olaparib as a maintenance monotherapy for patients with newly diagnosed advanced ovarian cancer and a BRCA1/2 mutation: A United States payer perspective

Objective: A phase 3 clinical trial (SOLO1) demonstrated that olaparib as maintenance provided significant improvement in progression-free survival (PFS) in patients with newly diagnosed advanced ovarian cancer and a germline or somatic BRCA mutation. This study evaluates the cost-effectiveness of olaparib as a first-line maintenance therapy versus routine surveillance from a U.S. third-party payer perspective.

Cost-effectiveness of atezolizumab plus chemotherapy for advanced non-small-cell lung cancer.

Background Adding atezolizumab to carboplatin/nab-paclitaxel improved progression-free survival and overall survival in patients with advanced non-squamous non-small-cell lung cancer. However, estimating the economy of atezolizumab/carboplatin/nab-paclitaxel is urgent on account of the high cost of atezolizumab. Objective This study aimed to evaluate the cost-effectiveness of atezolizumab plus carboplatin/nab- paclitaxel for untreated advanced non-squamous non-small-cell lung cancer from the United States payer perspective. Setting This study was based on randomized clinical trial data from the IMpower130 (NCT02367781) published in Lancet Oncology (May 2019). Method A Markov model was constructed to estimate the health expenditure on atezolizumab in combination with carboplatin/nab-paclitaxel for advanced non-small-cell lung cancer treatment. Drug costs were collected from Red Book Wholesale Acquisition Cost, and health state utility values were obtained from the literature. Uncertainty was evaluated via one-way and probabilistic sensitivity analyses. Main outcome measure The main outcomes were cost, life years, quality-adjusted life years, and incremental cost-effectiveness ratio. Results Over a 10-year horizon, atezolizumab/carboplatin/nab-paclitaxel treatment was associated with an expected 1.76 life years and 0.99 quality-adjusted life years compared to the 1.21 life years and 0.67 quality-adjusted life years for carboplatin/nab-paclitaxel alone. Compared to carboplatin/nab-paclitaxel, atezolizumab/carboplatin/nab-paclitaxel produced an incremental cost of $105,617. The resultant incremental cost-effectiveness ratio was $333,199 per quality-adjusted life year, which exceeded the willingness-to-pay threshold of $180,000 per quality-adjusted life year. The price of atezolizumab and utility values were the parameters that greatly impacted the incremental cost-effectiveness ratio. Carboplatin/nab-paclitaxel exhibited 98.6% probability of being a cost-effective treatment option compared to atezolizumab/carboplatin/nab-paclitaxel at a willingness-to-pay of $180,000 per quality-adjusted life year. However, reducing atezolizumab acquisition cost by 43.4% could make atezolizumab/carboplatin/nab-paclitaxel more cost-effective than carboplatin/nab-paclitaxel. Conclusion Adding atezolizumab to carboplatin/nab-paclitaxel was not cost-effective for advanced non-squamous non-small-cell lung cancer in the base-case scenario. Decreasing atezolizumab acquisition cost might enhancethecost-effectiveness.

Cost-effectiveness of filgrastim-sndz as primary prophylaxis (PP) versus secondary prophylaxis (SP) to prevent chemotherapy-induced febrile neutropenia (FN) in non-small cell lung cancer (NSCLC) patients at intermediate risk.

e19401 Background: Patients with nonmetastatic NSCLC receiving platinum-based chemotherapy are at an intermediate risk (10-20%) of developing FN; clinical practice guidelines recommend assessing whether these patients have FN risk factors before considering myeloid growth factor (MGF) prophylaxis. Most NSCLC patients receiving chemotherapy have ≥1 FN risk factors, and while PP leads to lower rates of FN across all cycles, it is more costly compared to SP. This study evaluates the cost-effectiveness of PP vs. SP using a biosimilar MGF, filgrastim-sndz, in NSCLC patients at intermediate risk of FN. Methods: A Markov model with a lifetime horizon was constructed to evaluate the total costs and clinical outcomes of using filgrastim-sndz as PP vs. SP in NSCLC patients 61 years old receiving adjuvant carboplatin/paclitaxel every 3 weeks for 4 cycles. Separate analyses were conducted for patients with 0 FN risk factors (0RF) and with ≥1 FN risk factors (1+RF), representing 11.3% and 18% baseline FN risk, respectively. Incremental cost-effectiveness ratios (ICERs) were calculated for cost per FN event avoided, life-year saved (LYS), and quality-adjusted life-year (QALY) gained from a United States payer perspective. Deterministic and probabilistic sensitivity analyses (PSA) were conducted. Results: For patients with 0RF, use of filgrastim-sndz as PP vs. SP provided an additional 0.056 QALYs (0.079 LYS) at an incremental cost of $3,266. The ICERs were $46,815, $41,555, and $58,531 for cost per FN event avoided, cost per LYS, and cost per QALY gained, respectively. For patients with 1+RF, PP vs. SP added 0.090 QALYs (0.127 LYS) at an incremental cost of $1,605. The ICERs were $13,970, $12,644, and $17,805 for cost per FN event avoided, cost per LYS, and cost per QALY gained, respectively. In the PSA, the likelihood of cost-effectiveness at a willingness-to-pay (WTP) threshold of $50,000 per QALY gained was 31.7% for patients with 0RF and 96.6% for 1+RF. Conclusions: For NSCLC patients at intermediate risk of FN receiving adjuvant carboplatin/paclitaxel with 1+RF, PP with filgrastim-sndz compared to SP is cost-effective based on a WTP threshold of $50,000/QALY. [Table: see text]

Cost-effectiveness analysis of ramucirumab treatment for patients with hepatocellular carcinoma who progressed on sorafenib with α-fetoprotein concentrations of at least 400 ng/ml

Objective: This study aimed to compare the cost-effectiveness of ramucirumab versus placebo for patients with hepatocellular carcinoma who progressed on sorafenib with α-fetoprotein concentrations (AFP) of at least 400 ng/ml in the United States.Methods: A Markov model was constructed to assess the cost-effectiveness of ramucirumab. Health outcomes were measured as quality-adjusted life years (QALYs). With TreeAge software, the disease process was modeled as three health states: progression-free survival (PFS), progressive disease (PD), and death. Costs were extracted from the REACH-2 trial, and utility was derived from published literature. Incremental cost-effectiveness ratios (ICERs) were calculated to compare ramucirumab with placebo. Probabilistic sensitivity analyses were developed to examine the robustness of the results.Results: In the base case analysis, ramucirumab therapy had a cost of $55,508.41 and generated 0.54 QALYs, while placebo therapy had a cost of $761.09 and generated 0.47 QALYs, leading to an additional $54,747.32 in costs and 0.07 QALYs. The ICER was $782,104.57 per QALY, which was much higher than the willingness-to-pay threshold of $100,000 per QALY. According to sensitivity analyses, the utility of PD in the two groups was the dominant parameter influencing the ICER.Conclusion: Although ramucirumab was associated with prolonged survival for patients with advanced hepatocellular carcinoma who progressed on sorafenib treatment with an AFP of at least 400 ng/ml, it is not a cost-effective treatment from a United States payer perspective.

A budget impact analysis of a magnetic sphincter augmentation device for the treatment of medication-refractory mechanical gastroesophageal reflux disease: a United States payer perspective.

Medication-refractory gastroesophageal reflux disease (GERD) is sometimes treated with laparoscopic Nissen fundoplication (LNF); however, this is a non-reversible procedure associated with important side effects and the need for repeat surgery. Removable magnetic sphincter augmentation (MSA) devices are an alternative, effective, and safe treatment option for such patients who have some lower esophageal sphincter function. The objective of this study was to assess the economic impact of introducing MSA technology (i.e., LINX Reflux Management System) into current practice from a US-payer perspective. An economic budget impact model was developed over a 1-year time horizon that compared current treatment of GERD patients who are medically managed (but refractory) or receiving LNF to future treatment of GERD patients that included a mix of patients treated with medical management only, LNF, or MSA. Resources included within the analyses were index procedures (inpatient and outpatient use), reoperations (revisions and removals), readmissions, healthcare visits, diagnostic tests, procedures, and medications. Medicare payment rates were typically used to inform unit costs. Assuming a hypothetical commercial insurance population of 1 million members, the base-case analysis estimated a net cost savings of $111,367 with introduction of the MSA. This translates to a savings of $0.01 per member per month. Results were largely driven by avoided inpatient procedures with use of the MSA device. Alternative analyses exploring the potential impact of increasing surgical volumes predicted that results would remain cost saving if the proportion of MSA market share taken from LNF was ≥ 90%. This study predicts that the introduction of the MSA device would lead to favorable budget impact results for the treatment of medication-refractory mechanical GERD for commercial payers. Future analyses will benefit from inclusion of middle-ground treatments as well as longer time horizons.

CO4 ALEMTUZUMAB VERSUS GENERIC GLATIRAMER ACETATE 20 MG IN PATIENTS WITH RELAPSING FORMS OF MULTIPLE SCLEROSIS: A COST-EFFECTIVENESS STUDY IN THE UNITED STATES

To evaluate the cost-effectiveness of alemtuzumab versus generic glatiramer acetate (GA) 20 mg in patients with relapsing multiple sclerosis (RMS) in the United States (US). A Markov model was developed from a US payer perspective with an annual cycle length and a time-horizon of 20 years. The modeled population represented pooled alemtuzumab-treated patients from the CARE-MS I (NCT00530348) and II (NCT00548405) trials. The British Columbia Multiple Sclerosis cohort data was used in the model to project the natural history of RMS disease progression. Treatment efficacy estimates for both therapies were derived from published network meta-analyses. Deterministic and probabilistic sensitivity analyses were conducted to assess any uncertainty in the model. The base-case analysis results showed that over a 20-year time-horizon, alemtuzumab was less costly compared with generic GA 20 mg ($421,996 vs $895,661). Alemtuzumab-treated patients accumulated a higher number of quality-adjusted life-years (QALYs) compared with those on generic GA 20 mg (8.977 vs 7.845). In addition, patients on alemtuzumab had a lower total number of relapse events compared with patients on generic GA 20 mg (7.06 vs 9.79), irrespective of these relapses leading to hospitalization or not. The probabilistic sensitivity analysis confirmed the base-case results that alemtuzumab dominated generic GA 20 mg as patients accumulated lower total incremental costs (-$476,987; 95% CI: -$579,996 to -$379,029) and higher incremental QALYs (1.1379; 95% CI: 0.4230 to 1.8306). In a scenario where natural history data was sourced from the London Ontario dataset, alemtuzumab continued to be dominant (lower total incremental costs: -$212,222; 95% CI: -$260,900 to -$165,772, and higher incremental QALYs: 0.9442; 95% CI: 0.3635 to 1.5248). Alemtuzumab is a cost-effective disease-modifying therapy for the treatment of patients with RMS when compared with generic GA 20 mg. These findings could help informed disease management decisions for effective RMS treatment.