US Payer Research Articles

EE539 Estimating the Lifetime Burden of Illness in Patients with Bronchiolitis Obliterans Syndrome (BOS)

Bronchiolitis Obliterans Syndrome (BOS) is a rare, life-threatening disease where the immune system attacks the airways of the lungs. BOS is estimated to affect approximately 25,000 individuals worldwide and can occur after hematopoietic stem cell transplantation (HSCT) or lung transplantation, with up to half of all lung transplant recipients developing BOS within 5 years of transplantation. BOS is rapidly progressive and irreversible, leading to lung failure after diagnosis, and making it the leading cause of death. The aim of this analysis was to estimate the lifetime economic burden of illness for BOS patients from the perspective of a US Payer.

Cost-effectiveness of the tubeless automated insulin delivery system vs standard of care in the management of type 1 diabetes in the United States.

BACKGROUND: A tubeless, on-body automated insulin delivery (AID) system (Omnipod 5 Automated Insulin Delivery System) demonstrated improved glycated hemoglobin A1c levels and increased time in range (70 mg/dL to 180 mg/dL) for both adults and children with type 1 diabetes in a 13-week multicenter, single-arm study. OBJECTIVE: To assess the cost-effectiveness of the tubeless AID system compared with standard of care (SoC) in the management of type 1 diabetes (T1D) in the United States. METHODS: Cost-effectiveness analyses were conducted from a US payer's perspective, using the IQVIA Core Diabetes Model (version 9.5), with a time horizon of 60 years and an annual discount of 3.0% on both costs and effects. Simulated patients received either tubeless AID or SoC, the latter being defined as either continuous subcutaneous insulin infusion (86% of patients) or multiple daily injections. Two cohorts (children: <18 years; adults: ≥18 years) of patients with T1D and 2 thresholds for nonsevere hypoglycemia (nonsevere hypoglycemia event [NSHE] <54 mg/dL and <70 mg/dL) were considered. Baseline cohort characteristics and treatment effects of different risk factors for tubeless AID were sourced from the clinical trial. Utilities and cost of diabetes-related complications were obtained from published sources. Treatment costs were derived from US national database sources. Scenario analyses and probabilistic sensitivity analyses were performed to test the robustness of the results. RESULTS: Treating children with T1D with tubeless AID, considering an NSHE threshold of less than 54 mg/dL, brings incremental life-years (1.375) and quality-adjusted life-years (QALYs) (1.521) at an incremental cost of $15,099 compared with SoC, resulting in an incremental cost-effectiveness ratio of $9,927 per QALY gained. Similar results were obtained for adults with T1D assuming an NSHE threshold of less than 54 mg/dL (incremental cost-effectiveness ratio = $10,310 per QALY gained). Furthermore, tubeless AID is a dominant treatment option for children and adults with T1D assuming an NSHE threshold of less than 70 mg/dL compared with SoC. The probabilistic sensitivity analyses results showed that compared with SoC, in both children and adults with T1D, tubeless AID was cost-effective in more than 90% of simulations, assuming a willingness-to-pay threshold of $100,000 per QALY gained. The key drivers of the model were the cost of ketoacidosis, duration of treatment effect, threshold of NSHE, and definition of severe hypoglycemia. CONCLUSIONS: The current analyses suggest that the tubeless AID system can be considered a cost-effective treatment compared with SoC in people with T1D from a US payer's perspective. DISCLOSURES: This research was funded by Insulet. Mr Hopley, Ms Boyd, and Mr Swift are full-time Insulet employees and own stock in Insulet Corporation. IQVIA, the employer of Ms Ramos and Dr Lamotte, received consulting fees for this work. Dr Biskupiak is receiving research support and consulting fees from Insulet. Dr Brixner has received consulting fees from Insulet. The University of Utah has received research funding from Insulet. Dr Levy is a consultant with Dexcom and Eli Lilly and has received grant/research support from Insulet, Tandem, Dexcom, and Abbott Diabetes. Dr Forlenza conducted research sponsored by Medtronic, Dexcom, Abbott, Tandem, Insulet, Beta Bionics, and Lilly. He has been speaker/consultant/advisory board member for Medtronic, Dexcom, Abbott, Tandem, Insulet, Beta Bionics, and Lilly.

Budget Impact Analysis of REBYOTA™ (Fecal Microbiota, Live-jslm [FMBL]) for Preventing Recurrent Clostridioides difficile Infection in the US.

Patients with Clostridioides difficile infection (CDI) often experience recurrences (rCDI), which are associated with high morbidity, mortality, and healthcare expenditures. REBYOTA™ (fecal microbiota, live-jslm [FMBL]) is a microbiota-based live biotherapeutic approved for the prevention of rCDI following antibiotic treatment for rCDI. We quantified the budget impact of FMBL during the first 3years following introduction from a third-party US payer perspective. A decision-tree model was used to estimate the budget impact of one-course FMBL by comparing costs under the scenario with FMBL to the scenario without FMBL (standard of care) in patients with one or more (≥ 1) recurrences after a primary episode of CDI and had completed ≥ 1 round of antibiotic treatments. Drug costs, rCDI-related medical costs, and budget impact over 1-3years were estimated in 2022 US dollars. One-way sensitivity analyses were performed. For an insurance plan with a population size of 1,000,000, 468 patients per year were estimated to have ≥ 1 rCDI. The budget impact of one-course FMBL at $9000/course was cost-saving at an» average of -$0.0039 on a per-member-per-month (PMPM) basis, an average of -$8.30 on a per-treated-member-per-month (PTMPM) basis, and a total of -$139,865 on a plan level assuming 5%, 15%, and 20% of patients receive FMBL over 1-3years, respectively. The scenario with FMBL entry was associated with higher drug costs (difference at $0.0474 PMPM; $101.26 PTMPM; $1,706,445 total plan) and lower rCDI-related medical costs (difference at -$0.0513 PMPM; -$109.56 PTMPM; -$1,846,309 total plan). The budget impact of FMBL in patients at first rCDI was cost-saving at -$0.0139 PMPM, -$84.78 PTMPM, corresponding to an annual savings of $500,022. FMBL has a cost-saving budget impact for a US payer, with higher initial drug costs being offset by savings in rCDI-related medical costs. Greater cost saving was found in patients at first recurrence.

Cost-effectiveness analysis of five anti-obesity medications from a US payer's perspective

Background and aimsTo determine the cost-effectiveness of anti-obesity medications (AOM): tirzepatide, semaglutide, liraglutide, phentermine plus topiramate (PpT), and naltrexone plus bupropion (NpB). Methods and resultsFrom a U.S. perspective we developed a Markov model to simulate weight change over a 40-year time horizon using results from clinical studies. According to the body mass index (BMI), cardiovascular diseases, diabetes and mortality risk were the health states considered in the model, being mutually exclusive. Costs of AOM, adverse events, cardiovascular events, and diabetes were included. We applied a 3% per-year discount rate and calculated the incremental cost-effectiveness ratios (ICERs) of cost per quality-adjusted life-year (QALY) gained. Probabilistic sensitivity analyses incorporated uncertainty in input parameters. A deterministic analysis was conducted to determine the robustness of the model. The model included a cohort of 78.2% females with a mean age of 45 years and BMI of 37.1 (SD 4.9) for females and 36.8 (SD 4.9) for males. NpB and PpT were the least costly medications and, all medications differed no more than 0.5 QALYs. Tirzepatide ICER was $355,616 per QALY. Liraglutide and semaglutide options were dominated by PpT. ConclusionCompared to other AOM, PpT was lowest cost treatment with nearly identical QALYs with other agents.

Estimated Societal Value of Lecanemab in Patients with Early Alzheimer's Disease Using Simulation Modeling.

Alzheimer's disease (AD) is a progressive neurodegenerative disorder associated with memory, cognitive, and behavioral deficits, and brings significant economic burden on caregivers and healthcare systems. This study aims to estimate the long-term societal value of lecanemab plus standard of care (SoC) versus SoC alone, corresponding to a range of willingness-to-pay (WTP) thresholds based on the phase III CLARITY AD trial readouts from both the US payer and societal perspectives. An evidence-based model was developed to simulate the effects of lecanemab on disease progression in early AD using interconnected predictive equations based on longitudinal clinical and biomarker data derived from the Alzheimer's Disease Neuroimaging Initiative (ADNI). The model was informed with the results of the phase III CLARITY AD trial and published literature. Key model outcomes included patient life-years (LYs), quality-adjusted life-years (QALYs), and total costs of both the direct and indirect costs of patients and caregivers over a lifetime horizon. Patients treated with lecanemab plus SoC gained an additional 0.62years of life versus SoC alone (6.23years vs. 5.61years). The mean time on lecanemab was 3.91years, and the treatment was associated with an increase in patient QALYs of 0.61 and an increase in total QALYs of 0.64 when both patient and caregiver utilities were considered. The model estimated that the annual value of lecanemab for the US payer perspective was US$18,709-35,678 ($19,710-37,351 for societal perspective) at the WTP threshold of $100,000-200,000 per QALY gained, respectively. Scenario analyses of patient subgroups, time horizon, input sources, treatment stopping rules, and treatment dosing were conducted to explore the impact of alternative assumptions on the model results. The economic study suggested that lecanemab plus SoC would improve health and humanistic (quality of life) outcomes and reduce economic burden for patients and caregivers in early AD.

A value-based budget impact model for dronedarone compared with other rhythm control strategies.

Aim: The budgetary consequences of increasing dronedarone utilization for treatment of atrial fibrillation were evaluated from a US payer perspective. Materials & methods: A budget impact model over a 5-year time horizon was developed, including drug-related costs and risks for long-term clinical outcomes (LTCOs). Treatments included antiarrhythmic drugs (AADs; dronedarone, amiodarone, sotalol, propafenone, dofetilide, flecainide), rate control medications, and ablation. Direct comparisons and temporal and non-temporal combination scenarios investigating treatment order were analyzed as costs per patient per month (PPPM). Results: By projected year 5, costs PPPM for dronedarone versus other AADs decreased by $37.69 due to fewer LTCOs, treatment with dronedarone versus ablation or rate control medications+ablation resulted in cost savings ($359.94 and $370.54, respectively), and AADs placed before ablation decreased PPPM costs by $242 compared with ablation before AADs. Conclusion Increased dronedarone utilization demonstrated incremental cost reductions over time.

Cost-effectiveness of chimeric antigen receptor T-cell therapy in adults with relapsed or refractory follicular lymphoma.

Follicular lymphoma (FL) is traditionally considered treatable but incurable. In March 2021, the US Food and Drug Administration approved the use of chimeric antigen receptor (CAR) T-cell therapy in patients with relapsed or refractory (R/R) FL after ≥2 lines of therapy. Priced at $373 000, CAR T-cell therapy is potentially curative, and its cost-effectiveness compared with other modern R/R FL treatment strategies is unknown. We developed a Markov model to assess the cost-effectiveness of third-line CAR T-cell vs standard of care (SOC) therapies in adults with R/R FL. We estimated progression rates for patients receiving CAR T-cell and SOC therapies from the ZUMA-5 trial and the LEO CReWE study, respectively. We calculated costs, discounted life years, quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER) of CAR T-cell vs SOC therapies with a willingness-to-pay threshold of $150 000 per QALY. Our analysis was conducted from a US payer's perspective over a lifetime horizon. In our base-case model, the cost of the CAR T-cell strategy was $731 682 compared with $458 490 for SOC therapies. However, CAR T-cell therapy was associated with incremental clinical benefit of 1.50 QALYs, resulting in an ICER of $182 127 per QALY. Our model was most sensitive to the utilities associated with CAR T-cell therapy remission and third-line SOC therapies and to the total upfront CAR T-cell therapy cost. Under current pricing, CAR T-cell therapy is unlikely to be cost-effective in unselected patients with FL in the third-line setting. Both randomized clinical trials and longer term clinical follow-up can help clarify the benefits of CAR T-cell therapy and optimal sequencing in patients with FL.

Cost-effectiveness analysis of LungLB for the clinical management of patients with indeterminate pulmonary nodules

BackgroundThere is currently a need for additional diagnostic information to help guide treatment decisions and to properly determine the best treatment pathway for patients identified with indeterminate pulmonary nodules (IPNs). The aim of this study was to demonstrate the incremental cost-effectiveness of LungLB compared to the current clinical diagnostic pathway (CDP) in the management of patients with IPNs, from a US payer’s perspective.MethodsA decision tree and Markov model hybrid was chosen from a payer perspective in the US setting, based on published literature, to assess the incremental cost-effectiveness of LungLB compared to the current CDP in the management of patients with IPNs. Primary endpoints of the analysis include expected costs, life years (LYs), and quality-adjusted life years (QALYs) for each arm of the model, as well as an incremental cost-effectiveness ratio (ICER), which is calculated as the incremental costs per QALY, and net monetary benefit (NMB).ResultsWe find that, with the inclusion of LungLB to the current CDP diagnostic pathway, expected LYs over the typical patient’s lifespan increase by 0.07 years and QALYs increase by 0.06. The average patient in the CDP arm will pay approximately $44,310 over their lifespan, while a patient in the LungLB arm will pay $48,492, resulting in a difference of $4,182. The differentials between the CDP and LungLB arms of the model in costs and QALYs yield an ICER of $75,740 per QALY and an incremental NMB of $1,339.ConclusionThis analysis provides evidence that LungLB, in conjunction with CDP, is a cost-effective alternative compared to the current CDP alone in a US setting for individuals with IPNs.

Cost-effectiveness of sacituzumab govitecan versus chemotherapy in advanced or metastatic triple-negative breast cancer

PurposeThe ASCENT trial demonstrated the efficacy of sacituzumab govitecan for the treatment of advanced or metastatic triple-negative breast cancer (TNBC). The current study evaluated the cost-effectiveness of receiving sacituzumab govitecan compared with standard of care chemotherapy from the United States payer perspective. MethodsA partitioned survival approach was used to project the disease course of advanced or metastatic TNBC. Two survival modes were applied to analyze two groups of patients. The survival data were gathered from the ASCENT trial. Direct medical costs were derived from the data of Centers for Medicare & Medicaid Services. Utility data was collected from the published literature. The incremental cost-utility ratio (ICUR) was the primary outcome that measured the cost-effectiveness of therapy regimen. One-way sensitivity and probabilistic sensitivity analysis were implemented to explore the uncertainty and validate the stability of results. ResultsIn the base-case, the ICUR of sacituzumab govitecan versus chemotherapy is $ 778,771.9/QALY and $ 702,281/QALY for full population group and brain metastatic-negative (BMN) group with the setting of classic survival mode. And in the setting of cure survival mode, the ICUR is $ 506,504.5/QALY for the full population group and $ 274,232.0/QALY for BMN population group. One-way sensitivity analyses revealed that the unit cost of sacituzumab govitecan and body weight were key roles that lower the ICUR value. Probabilistic sensitivity analyses also showed that reducing the unit price of sacituzumab govitecan can improve the likelihood of becoming cost-effective. ConclusionThe cost-effectiveness analysis suggested that from a US payer perspective, sacituzumab govitecan at current price is unlikely to be a preferred option for patients with advanced or metastatic TNBC at a threshold of $ 150,000/QALY.

Cost-effectiveness of immune checkpoint inhibition and targeted treatment in combination as adjuvant treatment of patient with BRAF-mutant advanced melanoma

BackgroundImmune checkpoint inhibitors (ICIs) and targeted treatments have improved the health outcomes of patients with advanced melanoma. However, due to the high cost of novel therapies, it is crucial to evaluate their value by considering both effectiveness and cost. To compare the cost-effectiveness of these novel agents (atezolizumab-vemurafenib-cobimetinib, vemurafenib-plus-cobimetinib, dabrafenib-plus-trametinib, and encorafenib-plus-binimetinib) for first-line treatment of metastatic melanoma with the BRAFV600 mutation.MethodsA patient-level model was developed to project the health outcomes of 4 strategies for patients with advanced melanoma. We estimated transition probabilities from the IMspire150 (ClinicalTrials.gov, NCT02908672), COMBI-AD (NCT01682083), and COLUMBUS (NCT01909453) trials using a parametric survival model. All health outcomes, including direct cost, quality-adjusted life-years (QALYs) and the incremental cost-effectiveness ratio (ICER), were estimated from the US payer perspective. Lifetime cost, QALYs, life-years (LYs), and ICERs were calculated. Univariable and probabilistic sensitivity analyses were performed to test model robustness, along with multiple scenario analyses.ResultsOf the 4 competing strategies, atezolizumab-vemurafenib-cobimetinib produced the best health outcomes, and the vemurafenib-cobimetinib strategy was the least expensive option. Atezolizumab-vemurafenib-cobimetinib, dabrafenib-plus-trametinib, and vemurafenib-cobimetinib formed the cost-effective frontier, indicating that the ordered ICERs were $325,113/QALYs for dabrafenib-plus-trametinib vs. vemurafenib-cobimetinib strategies and $2,247,500/QALYs for atezolizumab-vemurafenib-cobimetinib vs. dabrafenib-plus-trametinib strategies. Encorafenib-plus-binimetinib was dominated by the other 3 competing strategies. The drug price and first-line utility significantly influenced the model utcomes.ConclusionsFor BRAF-mutant advanced melanoma, the vemurafenib-cobimetinib strategy could be considered the most cost-effective treatment at the willingness-to-pay threshold of $150,000.

Cost impact of different treatment regimens of brolucizumab in neovascular age-related macular degeneration: A budget impact analysis.

BACKGROUND: Brolucizumab is a new anti-vascular endothelial growth factor (anti-VEGF) approved for treating neovascular age-related macular degeneration (nAMD). Multiple treatment regimens are available for treating nAMD. These regimens include manufacturer-recommended regimens, pro re nata (PRN) regimens, and treat-and-extend (T&E) regimens, which are based on clinical practice guidelines and data observed in the real-world clinical setting, classified as real-world evidence (RWE). Most budget impact models predict the financial consequences of adding a new drug to the formulary based on the manufacturer-recommended regimen. With different anti-VEGF treatment regimens being used in nAMD by ophthalmologists, it is OBJECTIVE: To estimate the budget impact of different treatment regimens of brolucizumab in nAMD from a US payer perspective. METHODS: A Microsoft Excel-based budget impact model was developed for different treatment regimens of brolucizumab over a 1-year time frame from a US payer perspective. A separate analysis was performed to estimate the budget impact from a US patient population perspective. Model inputs included drug costs, administration costs, physician visit costs, and disease monitoring costs. Outcomes in the budget impact model included the cost per member per month, annual health plan cost, and the US patient population-based annual cost. Based on the prevalence of nAMD in public and commercial health plans, a scenario analysis was conducted on the US population to account for the differences in the drug cost to the public and commercial payers. Further, 1-way sensitivity analyses were conducted to test model assumptions and uncertainty in model inputs. RESULTS: The addition of brolucizumab to the formulary increased the net budgetary impact under PRN and T&E regimens. The maximum increase in expenditure for a hypothetical health plan with 1 million enrollees was associated with the PRN regimen ($824,696), followed by the T&E regimen ($163,101). In contrast, using the manufacturer-recommended and RWE regimens led to an annual saving of $93,068 and $94,170 for the health plan, respectively. In the US patient population model, the introduction of brolucizumab resulted in savings in the manufacturer-recommended ($30.99 million) and RWE regimens ($31.35 million) but led to an increase in annual expenditures for the PRN ($274.58 million) and T&E ($54.30 million) regimens. CONCLUSIONS: Payers need to evaluate the cost impact of different treatment regimens of existing and new anti-VEGFs when making formulary decisions in nAMD management. DISCLOSURES: Mr Siddiqui, Ms Dhumal, Dr Patel, and Dr LeMasters have nothing to disclose. Dr Kamal has received research funding from Cerevel Therapeutics, served as a consultant to Pfizer/Cytel Inc, and received honoraria from Pharmacy Times Continuing Education. Dr Almony has served as a consultant to Cardinal Health and received honoraria from Pharmacy Times Continuing Education and Prime Education.

P36 Can Real-World Evidence (RWE) Drive European, UK, and US Payer Decisions in the Reassessment of Oncology Therapies?

To understand how health technology assessment (HTA) bodies and payers perceive the use and value of RWE when reassessing oncology therapies, and their perceptions on its most desired attributes.

Economic Evaluation of Trastuzumab Deruxtecan in Previously Treated HER2-Low Advanced Breast Cancer in the United States.

Recently, the DESTINY-Breast04 trial revealed that significant benefits in both overall survival (OS) and progression-free survival (PFS) in patients with HER2-low advanced or metastatic breast cancer treated with trastuzumab deruxtecan (T-DXd) compared with chemotherapy. The current study assessed the cost-effectiveness of T-DXd from the perspective of the United States payer. We developed a partitioned survival model to project the disease course of breast cancer. The OS and PFS data were derived from the DESTINY-Breast04 trial. We extrapolate the survival data beyond the follow-up time to assess the long-term survival prognosis. Direct medical costs and utility data were collected. The incremental cost-utility ratio (ICUR) was the primary outcome that evaluated the cost-effectiveness of a therapy regimen. One-way sensitivity and probabilistic sensitivity analysis were implemented to explore the uncertainty of outputs. In the base-case, the ICUR of T-DXd versus chemotherapy is $346,571.8/QALY and $337,789.4/QALY for all patients group and hormone-receptor-positive (HR+) subgroup, respectively. One-way sensitivity analyses revealed that the hazard ratio of OS, the unit cost of T-DXd, and body weight had a relatively large impact on the base-case result. Probabilistic sensitivity analyses showed that the likelihood that T-DXd was cost-effective is 14.5% and 12.6% for all patients group and HR+ subgroup, respectively. The cost-effectiveness analysis suggested that, at current price, trastuzumab deruxtecan is unlikely to be a preferred option for patients with HER2-low breast cancer at a threshold of $150,000/QALY from a US payer perspective.

EE65 Budget Impact of Zanubrutinib for Treatment of Relapsed or Refractory Chronic Lymphocytic Leukemia in the United States

The phase 3 ALPINE trial (NCT03734016) compared the efficacy and safety of ibrutinib, a first-generation Bruton's tyrosine kinase (BTK) inhibitor, with zanubrutinib, a novel highly selective BTK inhibitor, in patients with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The study aimed to conduct a budget impact analysis to estimate the incremental costs associated with using zanubrutinib in CLL/SLL patients from the US payer perspective.

Cost-Effectiveness Analysis of Frontline Treatment with Polatuzumab Vedotin in Diffuse Large B-Cell Lymphoma

Introduction: The POLARIX trial (NCT03274492) recently demonstrated that substituting polatuzumab vedotin (pola), a CD79b-targeted antibody-drug conjugate, for vincristine in standard R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone) chemoimmunotherapy for first-line therapy can prolong progression-free survival in patients with newly diagnosed diffuse large B-cell lymphoma (DLBCL). However, the medication cost of pola-R-CHP (pola, rituximab, cyclophosphamide, doxorubicin, and prednisolone) regimen is substantially higher than that of R-CHOP treatment. It remains unknown whether the lower rate of treatment failure with pola-R-CHP can be translated into reduced subsequent healthcare costs, increased life years (LYs), and improved quality of life, which may offset the higher medication cost. This study aimed to investigate the cost-effectiveness of substituting pola for vincristine in first-line therapy for patients with newly diagnosed DLBCL from a US payer perspective. Methods: We developed a decision analytic model to project lifetime quality-adjusted life years (QALYs) gained, LYs gained, and direct healthcare costs in newly diagnosed DLBCL patients. The model utilized a hypothetical cohort of patients at 65 years (the median age of disease onset). Direct healthcare costs were adjusted to 2021 US dollars, and an annual rate of 3% was used to discount future costs and benefits. Incremental cost-effectiveness ratios (ICERs) were calculated, and a willingness-to-pay (WTP) threshold of USD 150,000 per QALY gained was used as the acceptable level for determining cost-effectiveness. Sensitivity analyses and scenario analyses were conducted to assess the uncertainty of the base-case scenario and input parameters. Results: Pola-R-CHP, compared to standard R-CHOP, was associated with an incremental cost of USD 90,439, an improvement of 0.89 QALYs and 1.14 LYs, leading to an ICER of USD 101,510/QALY. The model was most sensitive to the survival benefit provided by pola-R-CHP, the age of the patients at initial treatment, and the price of pola. The ICER would increase significantly if pola-R-CHP is ineffective in providing long-term remissions. Furthermore, the ICER value increased as patients were older at the beginning of the treatment. When patients were older than 76.5 years at initial treatment, the ICER would exceed the WTP threshold of USD 150,000/QALY. To make the pola-R-CHP regimen cost-effective in all age groups (up to 80 years old), the current price of pola, which was estimated to be USD 19,307 for 170 mg per treatment cycle, needed to be reduced by 20%. In the probabilistic sensitivity analysis, 48% and 73% of the iterations produced ICERs below WTP thresholds of USD 100,000/ QALY and USD 150,000/ QALY, respectively. Conclusion: Our study findings suggest that pola-R-CHP, compared to the standard R-CHOP regimen, could be a cost-effective strategy in treatment-naïve DLBCL patients aged 65 years from a US payer perspective. However, several noticeable factors that could affect the cost-effectiveness profile of pola-R-CHP therapy should be considered in decision-making. The follow-up period of the POLARIX trial was relatively short, and mature survival data would be required to fully evaluate the value of pola in the treatment of newly diagnosed DLBCL.

Cost-Effectiveness Analyses of First Line Ibrutinib Versus Acalabrutinib Versus Zanubrutinib Followed By Second Line Venetoclax Plus Rituximab in Previously Untreated Chronic Lymphocytic Leukemia (CLL) Patients

Background. Targeted therapy with Bruton's tyrosine kinase inhibitors (BTKI) has significantly improved progression-free (PFS) and overall survival (OS) outcomes when compared with chemoimmunotherapy in the front line setting of CLL. Following the approval of the first generation of BTKI (ibrutinib, IBR) in 2016 as first line (1L) treatment of CLL, second generation BTKIs with more favorable safety profiles were approved/recommended in 2020 (acalabrutinib, ACA) and 2021 (zanubrutinib, ZAN). We performed a network meta-analysis (NMA) of these three BTKIs in terms of PFS in support of cost-effectiveness analyses from US payer perspective. Methods. We conducted a NMA of 5 trials (Woyach 2018, Burger 2015, Sharman 2020, Tam 2021, Goede 2014) on the PFS hazard ratios (HR) of the 3 BTKIs and any other CLL interventions that connected in the network. We specified a 5-state Markov model to capture the disease and clinical pathway for CLL patients over a 10 years' time horizon: progression free (PF) on 1L treatment [PF1]; progression before initiating second line (2L) treatment [P1]; PF on 2L treatment [PF2]; progression after 2L treatment [P2]; and death. Patients started PF on 1L treatment with a BTKI and moved to other states based on extracted transition probabilities from lognormal parametric distributions fitted to Kaplan-Meier PFS curves of phase III trials of IBR, ACA and ZAN. Venetoclax plus rituximab (VR) was considered the 2L treatment with PF2, P2 and death in 2L extracted from Seymour 2018. The transition probabilities of PF on 1L treatment and progression after 1L were adjusted based on the NMA results. OS and time to next treatment (TTNT) after first progression were considered the same for the three BTKIs. Costs of regimens (wholesale acquisition cost), adverse event (AE) management, and disease progression were incorporated into the appropriate health states. Utilities of PF1 (0.799), P1 (0.66), PF2 (0.55) and P2 (0.42) were obtained from literature. Annual discount rate of 3.0% was applied. Life years (LY), quality adjusted LY (QALY), and incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) were estimated. Probabilistic sensitivity analyses (PSA; 10,000 iterations) were performed to obtain cost-effectiveness acceptability curves (CEAC). Results. Compared with IBR, ACA (HR=0.54; 95%CI=0.41-0.72) but not ZAN (HR=1.03; 95%CI=0.80-1.34) was associated with a statistically significant lower HR of progression or death. At 10 years, 77% of ACA patients were in PF1 and 9% in PF2, compared to 49% and 28% of ZAN patients, and 50% and 27% of IBR patients. As shown in the table, in base case analyses, total costs of IBR treatment were higher by $104,331 over ACA and by $350,340 over ZAN; with ACA being higher by $246,009 over ZAN. Survival was estimated to be 6.83 LYs for IBR, 7.02 LYs for ACA (+0.19) and 6.82 LYs for ZAN (-0.01). QALYs were estimated to be 5.00 for IBR, 5.46 for ACA (+0.46) and 5.09 for ZAN (+0.09). This yielded negative (cost saving) ICER of $-549,110/LYg and ICUR of $-226,806/QALYg for ACA over IBR. The statistically non-significant survival benefit, the 0.01 LY lost but 0.09 QALY gained, and the lower cost of ZAN over IBR yielded an ICER of $35,034,000/LYg and a negative (cost saving) ICUR of $3,892,666/QALYg. Compared to ZAN, treatment with ACA was associated with incremental costs of $246,009, 0.20 LY and 0.37 QALY differentials, yielding ICER of $1,230,045/LYg yet ICUR of $664,889/QALYg. All estimates were confirmed with negligible differences in PSAs. In a scenario analysis for the PF1 state only, QALY gains were 5.25 for ACA, 4.40 for ZAN and 4.34 for IBR. Considering cost differentials of $-69,138 for ACA and $-352,297 for ZAN relative to IBR, the corresponding ICURs were $-75,975/QALYg and $-5,871,617/QALYg, resp. Conclusion. Clinically over the course of 1L treatment with a BTKI followed by 2L treatment with VR, ACA in 1L is associated with greater reductions in the risk of progression or death compared to IBR and ZAN over 10 years. This benefit comes at lower cost than IBR, which resulted in cost-saving ICUR and ICER estimates; but at higher cost than ZAN, which resulted in an ICUR of ~$665,000 per QALY gained. Proportionately, QALY results came out more favorably than LY results. This rather unusual result is attributable to the better safety profiles and lower AE costs of ACA and ZAN, coupled with lower pricing. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Trametinib for patients with recurrent low-grade serous ovarian cancer: A cost-effectiveness analysis

ObjectiveThe GOG 281/LOGS trial found that trametinib prolonged progression-free survival (PFS) in patients with recurrent low-grade serous ovarian cancer (LGSOC), compared with standard of care (SOC). The current study aimed to evaluate the cost-effectiveness of trametinib versus standard of care for recurrent LGSOC from the US payer perspective. MethodsA Markov model was adopted to compare the cost and effectiveness of trametinib and standard of care group in patients with recurrent LGSOC. Life years (LYs), quality-adjusted LYs (QALYs), lifetime costs, and incremental cost-effectiveness ratios (ICERs) were calculated. One-way, and probabilistic sensitivity analyses were performed to explore the model robustness. ResultTrametinib group provided an additional 0.58 QALYs (1.14 LYs) and an incremental cost of $248,214 compared with the SOC group. The incremental cost-effectiveness ratio was $424,097 per QALY. The results of one-way sensitivity analyses suggested that our model was sensitive to the hazard ratio of OS and PFS between trametinib and SOC group, utility of PFS and the cycle cost of trametinib. Probabilistic sensitivity analyses revealed that there was 6% probability of the trametinib group being cost-effective at a willingness-to-pay (WTP) threshold of $150,000 per QALY. ConclusionsFrom the US payer perspective, trametinib is not cost-effective for patients with recurrent LGSOC at the assumed WTP threshold of $150,000 per QALY. Based on the value standpoint, price reduction of trametinib is expected to improve the cost-effectiveness of trametinib in patients with recurrent LGSOC.

Is adding ribociclib to fulvestrant cost-effective in treating postmenopausal women with HR+/HER2- advanced or metastatic breast cancer? A US payer perspective cost utility analysis.

BACKGROUND: Breast cancer is the most prevalent type of cancer in women in the United States. Ribociclib plus fulvestrant combination therapy gained US Food and Drug Administration approval to treat postmenopausal women with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) advanced or metastatic breast cancer in 2018. OBJECTIVE: To determine the cost-effectiveness of ribociclib plus fulvestrant vs placebo plus fulvestrant therapy in the target population from a US payer perspective. METHODS: A partitioned survival analysis model composed of 3 health states (progression free, progressed disease, and death) was constructed to evaluate the cost-effectiveness of ribociclib plus fulvestrant vs placebo plus fulvestrant. The progression-free survival and the overall survival data points were extracted from published Kaplan-Meier curves in the MONALEESA-3 study and fitted to parametric curves. The safety and efficacy of the treatment was referenced from the MONALEESA-3 trial. Costs were obtained from standard sources including the Red Book for medication costs, Medicare Clinical Laboratory/Physician Fee Schedule for clinical utilization, and the literature for costs of managing adverse events, subsequent therapy, and end-of-life care. Utility and disutility values were obtained from literature to calculate quality-adjusted life-years (QALYs). One-way and probabilistic sensitivity analyses were conducted to test the model robustness. Several scenario analyses were also investigated. RESULTS: In the base case, the ribociclib plus fulvestrant arm was associated with $522,844 and 3.25 QALYs compared with $50,395 and 2.14 QALYs in the placebo plus fulvestrant arm, leading to an incremental cost-effectiveness ratio of $425,951/QALY. The cost of ribociclib had the biggest impact on the model and constituted 84% of the total cost for the ribociclib plus fulvestrant arm. The probabilistic sensitivity analysis projected that the ribociclib plus fulvestrant treatment would have a net benefit over the placebo plus fulvestrant therapy at a willingness-to-pay (WTP) threshold of $405,600/QALY. CONCLUSIONS: At a WTP threshold of $150,000/QALY, the addition of ribociclib to fulvestrant is not considered to be cost-effective in postmenopausal women with HR+/HER2- advanced or metastatic breast cancer. The findings send a strong price signal to the manufacturer and can be used to facilitate payers with price negotiation in making coverage decisions.

Trastuzumab deruxtecan versus chemotherapy for patients with HER2-low advanced breast cancer: A US-based cost-effectiveness analysis.

Background: In recent years, the rise of antibody-drug conjugates (ADCs) has changed the treatment paradigm for patients with HER2-low advanced breast cancer (ABC). DESTINY-Breast04 (NCT03734029) has demonstrated the antitumor activity of trastuzumab deruxtecan (T-DXd). However, the balance between the efficacy and cost of T-DXd remains undefined. Consequently, there is a great need to assess the cost-effectiveness of T-DXd for patients with HER2-low ABC when compared with chemotherapy. Methods: A Markov decision-analytic model with a time horizon of 15years was employed to estimate the costs and clinical efficacy of trials with the administration of T-DXd in contrast to chemotherapy alone as a later-line therapy in a group of patients with hormone receptor-positive (HR+) or negative (HR-) HER2-low ABC. The US payer perspective was taken into account when factors such as medical lifetime expenditure, incremental cost-effectiveness ratios (ICERs), and quality-adjusted life years (QALYs) were calculated. Sensitivity analyses were used to determine the model's stability. A subgroup analysis was also conducted on the HR+/HER2-low cohort. Results: T-DXd was associated with an improvement of 0.543, 0.558, and 0.789 QALYs when compared with treatment with chemotherapy for overall, HR+, and HR- HER2-low patients, respectively. However, incorporating T-DXd into later-line therapy led to increased costs ($161,406, $177,907, and $155,757), which causes the ICER for T-DXd to be $296,873, $318,944, and $197,355 per QALY. The cost of T-DXd and the patient's weight were the most influential factors for ICER. T-DXd being the dominant strategy is about 1.5%, 0.5%, and 28.0% in overall, HR+, and HR- HER2-low ABC patients, respectively. In addition, the T-DXd regimen was not cost-effective in all subgroups. Conclusion: Compared with chemotherapy, T-DXd was not cost-effective for patients with HER2-low ABC in the United States. However, it can provide more health benefits to patients with HR+/HER2-low ABC.

Trastuzumab deruxtecan versus trastuzumab emtansine for patients with human epidermal growth factor receptor 2-positive metastatic breast cancer: A cost-effectiveness analysis

BackgroundDESTINY-Breast03 (NCT03529110) was the first global phase III study to assess the antitumor activity of trastuzumab deruxtecan (T-DXd) compared to trastuzumab emtansine (T-DM1) in 2022. However, the balance between efficacy and cost of T-DXd remains unclear. As a result, the present study's goal is to investigate the cost-effectiveness of T-DXd vs T-DM1 as a second-line treatment for patients with HER2-positive MBC from the US and Chinese payer's perspectives. MethodsA Markov model with a 20-year time horizon was developed to evaluate the overall cost of patient treatment, incremental cost-effectiveness ratio (ICER), quality-adjusted life-years (QALYs), and life-years (LYs) in the US and China at WTP levels of 150,000/QALY and 37,653/QALY, respectively (3 times GDP per capita in 2021). Key data were gathered from the US government's official website, the Xiangya Hospital of Central South University, and published literature. To determine the model's stability, a sensitivity analysis was performed. A subgroup analysis was also implemented. ResultsCompared with T-DM1, treatment with T-DXd generated an additional 1.672 QALYs (2.796 LYs), resulting in an ICER of $13,342/QALY (US) and $186,017/QALY (China). The cost of drugs is the most influential factor in the American and Chinese models. Subgroup analysis revealed that the T-DXd and T-DM1 regimens were more cost-effective at reducing the risk of death in the US and Chinese HER2-positive MBC patients. ConclusionT-DXd as second-line treatment could gain more health benefits for HER2-positive MBC patients in comparison with T-DM1, which is considered to be cost-effective in the US but not in China.