US Payers Research Articles

Cost-Effectiveness of Fruquintinib for Refractory Metastatic Colorectal Cancer in the USA.

The FRESCO-2 trial established the efficacy and safety of fruquintinib in patients with refractory metastatic colorectal cancer. However, its cost-effectiveness in the US context is not well documented. This study evaluates the cost-effectiveness of fruquintinib versus placebo for this patient population from the perspective of US payers. We developed a partitioned survival model on the basis of patient-level data reconstructed from the survival curves of the FRESCO-2 trial. Parametric estimation was conducted to estimate long-term clinical outcomes and medical costs over a lifetime horizon. Cost inputs and utilities were sourced from public data and previous literature. We used a discount rate of 3.0% per year for both clinical outcomes and costs. We adopted an incremental cost-effectiveness ratio (ICER) threshold of US$100,000 per quality-adjusted life-year (QALY) gained. We performed sensitivity and scenario analyses to examine the robustness of cost-effectiveness results. Fruquintinib treatment resulted in incremental gains of 0.108 life years (LYs) and 0.073 QALYs compared with the placebo, at an additional cost of US$112,294, primarily driven by medication expenses. The ICER for fruquintinib versus placebo was calculated at US$1,037,855 per LY and US$1,546,619 per QALY gained, exceeding the predefined cost-effectiveness threshold. The cost-effectiveness results were robust across all sensitivity and scenario analyses. Despite the survival benefit, fruquintinib was not cost-effective compared with the placebo in patients with refractory metastatic colorectal cancer in the US setting, on the basis of the conventional willingness-to-pay threshold. Our findings may provide a basis for informing the pricing and reimbursement decisions regarding fruquintinib.

Cost-effectiveness of finerenone added to standard of care for patients with type 2 diabetes-related chronic kidney disease in the United States.

To examine the cost-effectiveness of adding finerenone to standard of care (SoC) for treating type 2 diabetes mellitus (T2DM)-related chronic kidney disease (CKD) in the United States. Based on the clinical data analysed by FIDELITY, we referenced the validated FINE-CKD model (Markov model) to evaluate the cost-effectiveness of SoC versus SoC + finerenone from the perspective of US payers. The model was cycled for 35 years in 4-month cycles, with cost and utility values derived from the published literature. The primary outcomes were incremental cost-effectiveness ratio (ICER) and quality-adjusted life years (QALYs). Deterministic and probabilistic sensitivity analyses were conducted to assess the robustness of the base-case results. The treatment strategy of finerenone plus SoC led to gains of 6.95 QALYs and had a lifetime cost of $491 745.31. Compared to SoC, that strategy yielded 0.48 more QALYs at an added cost of $65 305.72. The ICER for finerenone was $135 257.06 per QALY, which is below the willingness-to-pay threshold of United States ($150 000/QALY). The results were sensitive to the hazard ratios associated with the efficacy of finerenone and its cost. Probabilistic sensitivity analyses showed that the probability that finerenone plus SoC would be cost-effective was 57.6%. For patients with T2DM-related CKD, adding finerenone to SoC may be a cost-effective option in the United States. Reasonable price reductions for finerenone could potentially benefit more patients.

Economic evaluation of camrelizumab plus rivoceranib versus sorafenib as first-line therapy for unresectable hepatocellular carcinoma in the United States and China.

Camrelizumab combined with rivoceranib has been proven effective for treating unresectable hepatocellular carcinoma (uHCC). However, their higher prices than sorafenib could impose a substantial economic burden on patients. This study aimed to evaluate the relative cost-effectiveness of the combination of camrelizumab and rivoceranib versus sorafenib as first-line therapy for patients with uHCC from the perspective of the US and Chinese payers. Using data from the CARES-310 trial, a partitioned survival model (PSM) was developed, considering the perspectives of the US and Chinese payers. The model employed a 15-year time horizon and a biweekly cycle. Direct medical costs and utility data were collected from previous studies and open-access databases. Primary outcomes included quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio (ICER). Price simulations, sensitivity analyses, and subgroup analyses were conducted. The ICER for the US and China was $122,388.62/QALY and $30,410.56/QALY, respectively, falling below the willingness-to-pay (WTP) thresholds of $150,000/QALY for the US and $35,898.87/QALY for China. Price simulations indicated the cost-effectiveness of camrelizumab plus rivoceranib when the price of camrelizumab (200mg) remained below $6275.19 in the US and $558.09 in China. The primary determinant of cost-effectiveness in both regions was the cost of camrelizumab. The combination of camrelizumab and rivoceranib is a cost-effective first-line therapy for uHCC in both the US and China. Lowering their prices could significantly influence their cost-effectiveness and accessibility to patients. These findings will guide clinicians in treating uHCC and help decision-makers formulate value-based drug pricing strategies.

Understanding Variation in Negotiated Rates Using Novel Health Plan Price Transparency Data

This study uses health plan price transparency data to examine how negotiated rates for the same service vary within and across US payers and hospitals.

Economic Evaluation of a Novel Lung Cancer Diagnostic in a Population of Patients with a Positive Low-Dose Computed Tomography Result

Background: Early detection of lung cancer is crucial for improving patient outcomes. Although advances in diagnostic technologies have significantly enhanced the ability to identify lung cancer in earlier stages, there are still limitations. The alarming rate of false positives has resulted in unnecessary utilization of medical resources and increased risk of adverse events from invasive procedures. Consequently, there is a critical need for advanced diagnostics after an initial low-dose computed tomography (LDCT) scan. Objectives: This study evaluated the potential cost savings for US payers of CyPath® Lung, a novel diagnostic tool utilizing flow cytometry and machine learning for the early detection of lung cancer, in patients with positive LDCT scans with indeterminate pulmonary nodules (IPNs) ranging from 6 to 29 mm. Methods: A cost offset model was developed to evaluate the net expected savings associated with the use of CyPath® Lung relative to the current standard of care for individuals whose IPNs range from 6 to 29 mm. Perspectives from both Medicare and private payers in a US setting are included, with a 1-year time horizon. Cost calculations included procedure expenses, complication costs, and diagnostic assessment costs per patient. Primary outcomes of this analysis include cost savings per cohort and cost savings per patient. Results: Our analysis showed positive cost savings from a private payer’s perspective, with expected savings of $895 202 311 per cohort and $6460 per patient, across all patients. Scenario analysis resulted in cost savings of $890 829 889 per cohort, and $6429 per patient. Similarly, savings of $378 689 020 per cohort or $2733 per patient were yielded for Medicare payers, across all patients. In addition, scenario analysis accounting for false negative patients from a Medicare payer perspective yielded savings of $376 902 203 per cohort and $2720 per patient. Discussion: The results suggest substantial cost savings, primarily due to reductions in follow-up diagnostic assessments and procedures, and highlight the importance of accurate diagnostic tools in reducing unnecessary healthcare expenditures. Conclusion: CyPath® Lung utilization yields savings for private and Medicare payers relative to the current standard of care in a US setting for individuals with 6 to 20 mm IPNs.

Impact of next-generation sequencing vs polymerase chain reaction testing on payer costs and clinical outcomes throughout the treatment journeys of patients with metastatic non-small cell lung cancer.

For patients with metastatic non-small cell lung cancer (mNSCLC), next-generation sequencing (NGS) biomarker testing has been associated with a faster time to appropriate targeted therapy and more comprehensive testing relative to polymerase chain reaction (PCR) testing. However, the impact on payer costs and clinical outcomes during patients' treatment journeys has not been fully characterized. To assess the costs and clinical outcomes of NGS vs PCR biomarker testing among patients with newly diagnosed de novo mNSCLC from a US payers' perspective. A Markov model assessed costs and clinical outcomes of NGS vs PCR testing from the start of testing up to 3 years after. Patients entered the model after receiving biomarker test results and then initiated first-line (1L) targeted or nontargeted therapy (immunotherapy and/or chemotherapy) depending on actionable mutation detection. A few patients with an actionable mutation were not detected by PCR and inappropriately initiated 1L nontargeted therapy. At each 1-month cycle, patients could remain on treatment with 1L, progress to second line or later (2L+), or die. Literature-based inputs included the rates of progression-free survival (PFS) and overall survival (OS), targeted and nontargeted therapy costs, total costs of testing, and medical costs of 1L, 2L+, and death. Per patient average PFS and OS as well as cumulative costs were reported for NGS and PCR testing. In a modeled population of 100 patients (75% commercial and 25% Medicare), 45.9% of NGS and 40.0% of PCR patients tested positive for an actionable mutation. Relative to PCR, NGS was associated with $7,386 in savings per patient (NGS = $326,154; PCR = $333,540) at 1 year, driven by lower costs of testing, including estimated costs of delayed care and nontargeted therapy initiation before receiving test results (NGS = $8,866; PCR = $16,373). Treatment costs were similar (NGS = $305,644; PCR = $305,283). In the PCR cohort, the per patient costs of inappropriate 1L nontargeted therapy owing to undetected mutations were $6,455, $6,566, and $6,569 over the first 1, 2, and 3 years, respectively. Relative to PCR testing, NGS was associated with $4,060 in savings at 2 years and $1,092 at 3 years. Patients who initiated 1L targeted therapy had an additional 5.4, 8.8, and 10.4 months of PFS and an additional 1.4, 3.6, and 5.3 months of OS over the first 1, 2, and 3 years, respectively, relative to those who inappropriately initiated 1L nontargeted therapy. In this Markov model, as early as year 1, and over 3 years following biomarker testing, patients with newly diagnosed de novo mNSCLC undergoing NGS testing are projected to have cost savings and longer PFS and OS relative to those tested with PCR.

Cost-effectiveness of first-line enfortumab vedotin in addition to pembrolizumab for metastatic urothelial carcinoma in the United States.

The EV-302 trial found that the combination of enfortumab vedotin (EV) with pembrolizumab significantly improved survival for patients with metastatic urothelial carcinoma (mUC). However, given the high cost of the drugs, there is a need to assess its value by considering both efficacy and cost. This study assessed the cost-effectiveness of EV plus pembrolizumab as a first-line treatment for patients with mUC from the perspective of U.S. payers. A Markov model was developed to compare the lifetime costs and effectiveness of EV in combination with pembrolizumab with chemotherapy in the treatment of mUC patients from U.S. payer perspective. Life-years (LYs), quality-adjusted LYs (QALYs), and lifetime costs were estimated. One-way, two-way and probabilistic sensitivity analyses were conducted to evaluate model uncertainty. Additionally, subgroup analyses were performed. Compared to chemotherapy, the combination of EV and pembrolizumab provided an additional 2.10 LYs and 1.72 QALYs, at an incremental cost of $962,240.8 per patient. The incremental cost-effectiveness ratio (ICER) is $558,973 per QALY. Subgroup analysis indicated that patients ineligible for cisplatin treatment had a lower ICER compared to those who were eligible for cisplatin. From the perspective of US payers, at a willingness-to-pay threshold of $150,000 per QALY, the combination of EV and pembrolizumab is estimated to not be cost-effective compared to traditional chemotherapy in the first-line treatment of mUC patients.

Is Bevacizumab a Cost-Effective Regimen for Treating Cerebral Radiation Necrosis in the United States?

BackgroundBevacizumab has been demonstrated to have superior efficacy in the treatment of cerebral radiation necrosis (CRN), but its high cost may exacerbate the disease burden. This study aimed to assess the cost-effectiveness of bevacizumab in comparison to corticosteroids for treating CRN from the US payers’ perspective. MethodsDecision tree models were constructed to simulate the process of bevacizumab and corticosteroids in CRN short-term and long-term therapy. Critical clinical data were derived from the NCT01621880 trial. Costs and utility values were obtained from the US official websites and published literature. The main outcomes were total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses were performed to assess the robustness of the models. ResultsIn the short-term and long-term models, bevacizumab added 0.11 (0.46 vs 0.35) and 0.16 (0.54 vs 0.38) QALYs compared with corticosteroids therapy, with corresponding incremental costs of $12,351 and $23,253, respectively. The resultant ICERs were $112,987/QALY and $150,245/QALY for short-term and long-term treatment, respectively. The one-way sensitivity analysis indicated that utility value of nonrecurrence status, body weight, and bevacizumab price per cycle were the most influential factors for ICER of both models. At the willingness-to-pay threshold of $150,000/QALY in the United States, the probabilities of bevacizumab being cost-effective for CRN short and long-term treatment were 63.9% and 49%, respectively. ConclusionsCompared with corticosteroids, bevacizumab is an economical alternative for CRN short-term treatment from the US payers’ perspective, whereas long-term therapy draws an opposite conclusion.

Evaluating Unmet Needs in Large-Volume Subcutaneous Drug Delivery: US Payer Perspectives on a Novel, Large-Volume On-Body Delivery System.

This research sought to evaluate payer perspectives around OBDS's through semi-structured interviews with 17 US payers representing approximately 227 million covered lives. When asked about the perceived advantages of the novel on-body delivery system (OBDS), 70.6% of payers independently cited simplicity, ease-of-use, and convenience as the most beneficial features, with the potential to replace IV infusion by facilitating at-home HCP- or self-administration being the second most frequent response. Most payers (88.2%) believed that the novel OBDS could fulfill unmet needs such as cost of IV infusion, convenient administration, and improved patient compliance in large-volume SC delivery by enabling safe, easy, self-administration. Nearly all payers (88.2%) stated they would consider covering and managing pharmaceutical products packaged with the novel OBDS, with the remaining payers considering the total cost compared to other routes. A significant proportion of payers (82.4%) acknowledged that a drug delivered via the novel OBDS could warrant a price premium above the cost of the standalone SC drug vial, with half stating the premium would range from 5% to 20% and the other half citing an unspecified premium dependent on the individual drug situation.Conclusions: Given the ability to help address critical unmet needs for the patient and healthcare system, a large proportion of the payers stated that the novel OBDS would fulfill unmet needs and warrant a price premium versus the cost of the standalone SC vial and certainly over the IV counterpart. Future research to quantify the value that OBDS efficiencies could bring to healthcare delivery is warranted.

Real-world clinical and economic outcomes for patients with advanced non-small cell lung cancer enrolled in a clinical trial following comprehensive genomic profiling via liquid biopsy.

Oncology clinical trial enrollment is strongly recommended for patients with cancer who are not eligible for established and approved therapies. Many trials are specific to biomarker-targeted therapies, which are typically managed as specialty pharmacy services. Comprehensive genomic profiling (CGP) of advanced cancers has been shown to detect biomarkers, guide targeted treatment, improve outcomes, and result in the clinical trial enrollment of patients, which is modeled to offset pharmacy costs experienced by US payers, yet payer policy coverage remains inconsistent. A common concern limiting coverage of CGP by payers is the potential of identifying biomarkers beyond guideline-recommended treatments, which creates a perception that insurance companies are being positioned to "pay for research." However, these biomarkers can increase clinical trial eligibility, and specialty pharmacy management may have an interest in maximizing the clinical trial enrollment of members. To investigate if clinical trial enrollment following liquid biopsy CGP for non-small cell lung cancer (NSCLC) is clinically and/or economically impactful from a payer claims perspective. Clinical and economic outcomes were studied using a real-world clinical genomic database (including payer claims data) from patients with NSCLC who enrolled in clinical trials immediately following liquid biopsy CGP (using Guardant360) and matched NSCLC patient controls also tested with liquid biopsy CGP. Real-world overall survival was significantly (log-rank P < 0.0001) better for patients enrolled in clinical trials with similar costs of care, albeit with more outpatient encounters among those enrolled compared with matched controls. The results, together with previous analyses, suggest that, in addition to the clinical benefits associated with targeted therapies directed by CGP and other testing approaches, payers and specialty pharmacy managers may consider clinical trial direction and enrollment as a clinical and economic benefit of liquid biopsy CGP and adopt this into coverage decision frameworks and formularies.

HTA60 What Utilization Management Strategies Does ICER Recommend to US Payers and Policy-Makers to Manage Uncertainty with Gene Therapies?

HTA60 What Utilization Management Strategies Does ICER Recommend to US Payers and Policy-Makers to Manage Uncertainty with Gene Therapies?

Real-world evidence of Pressure-Enabled Drug Delivery for trans-arterial chemoembolization and radioembolization among patients with hepatocellular carcinoma and liver metastases

Objectives Pressure-Enabled Drug Delivery (PEDD), a method using pressure to advance catheter-delivered drug distribution, can improve treatment for hepatocellular carcinoma (HCC) and liver metastases, but real-world evidence is limited. We compared baseline patient characteristics, clinical complexity, and post-procedure healthcare resource utilization (HRUs) and clinical complications for PEDD and non-PEDD procedures. Methods This study used a retrospective, longitudinal, cohort design of claims data from Clarivate’s Real World Data Repository, which includes 98% of US payers with over 300 million unique patients from all US states. We identified patients with a trans-arterial chemoembolization (TACE) or trans-arterial radioembolization (TARE) from 1 January 2019 to 31 December 2022. Subsamples grouped patients with HCC receiving a TARE procedure at their first embolization and patients with metastatic colorectal cancer (CRC) that received a TARE procedure. We reported descriptive comparisons of our full sample of patients with HCC and liver metastases receiving PEDD versus non-PEDD procedures. We then conducted a matching-adjusted comparison of HRUs and clinical complications for PEDD and non-PEDD patients among our subsamples (HCC receiving a TARE procedure at their first embolization and patients with metastatic CRC that received a TARE procedure). Matching was based on baseline demographic and clinical characteristics using coarsened exact matching and propensity-score matching. HRUs included inpatient, outpatient, and emergency department visits. Clinical complications included ascites, cholecystitis, fatigue, gastric ulcer, gastritis, jaundice, LFT increase, lymphopenia, portal hypertension, and post-embolization syndrome. Results PEDD procedures were used on patients with worse baseline disease burdens: baseline Charlson comorbidity index (mean of 6.5 vs. 5.8), any prior clinical complication related to underlying disease (33.7 vs. 31.0%), and prior systemic therapy (22.1% vs. 16.2%). PEDD patients had a greater number of procedural codes indicative of technical complexity for TACE (PEDD mean = 226.3; non-PEDD mean = 134.5; p value <.01) and TARE (PEDD mean = 205.56; non-PEDD mean = 94.8; p value <0.01). Matching-adjusted analyses of patients with HCC and CRC demonstrated comparable HRU and clinical complications for PEDD and non-PEDD procedures post-index. Conclusion Despite higher baseline disease burden and complexity, post-procedure HRU and clinical complications for PEDD patients were similar to non-PEDD patients. The complex baseline clinical profile may reflect selection of challenging cases for PEDD use. Future studies should validate the benefits observed with PEDD embolization in larger samples with greater statistical power.

Cost of genetic testing, delayed care, and suboptimal treatment associated with polymerase chain reaction versus next-generation sequencing biomarker testing for genomic alterations in metastatic non-small cell lung cancer

Aims To assess US payers’ per-patient cost of testing associated with next-generation sequencing (NGS) versus polymerase chain reaction (PCR) biomarker testing strategies among patients with metastatic non-small cell lung cancer (mNSCLC), including costs of testing, delayed care, and suboptimal treatment initiation. Methods A decision tree model considered biomarker testing for genomic alterations using either NGS, sequential PCR testing, or hotspot panel PCR testing. Literature-based model inputs included time-to-test results, costs for testing/medical care, costs of delaying care, costs of immunotherapy [IO]/chemotherapy [CTX] initiation prior to receiving test results, and costs of suboptimal treatment initiation after test results (i.e. costs of first-line IO/CTX in patients with actionable mutations that were undetected by PCR that would have been identified with NGS). The proportion of patients testing positive for a targetable alteration, time to appropriate therapy initiation, and per-patient costs were estimated for NGS and PCR strategies combined. Results In a modeled cohort of 1,000,000 members (25% Medicare, 75% commercial), an estimated 1,119 had mNSCLC and received testing. The proportion of patients testing positive for a targetable alteration was 45.9% for NGS and 40.0% for PCR testing. Mean per-patient costs were lowest for NGS ($8,866) compared to PCR ($18,246), with lower delayed care costs of $1,301 for NGS compared to $3,228 for PCR, and lower costs of IO/CTX initiation prior to receiving test results (NGS: $2,298; PCR:$5,991). Cost savings, reaching $10,496,220 at the 1,000,000-member plan level, were driven by more rapid treatment with appropriate therapy for patients tested with NGS (2.1 weeks) compared to PCR strategies (5.2 weeks). Limitations Model inputs/assumptions were based on published literature or expert opinion. Conclusions NGS testing was associated with greater cost savings versus PCR, driven by more rapid results, shorter time to appropriate therapy initiation, and minimized use of inappropriate therapies while awaiting and after test results.

Budget impact of oral nirmatrelvir/ritonavir in adults at high risk for progression to severe COVID-19 in the United States.

Nirmatrelvir/ritonavir (NMV/r) is indicated for the treatment of mild-to-moderate COVID-19 in adults who are at high risk for progression to severe COVID-19. NMV/r has also been authorized for emergency use by the US Food and Drug Administration for the treatment of mild-to-moderate COVID-19 in pediatric patients (aged 226512 years and weighing at least 40 kg) who are at high risk for progression to severe COVID-19. Understanding the budget impact of introducing NMV/r for the treatment of adults with COVID-19 is of key interest to US payers. To estimate the annual budget impact of introducing NMV/r in a US commercial health plan setting in the current Omicron COVID-19 era. A budget impact model was developed to assess the impact of NMV/r on health care costs in a hypothetical 1-million-member commercial health insurance plan over a 1-year period in the US population; clinical and cost inputs were derived from published literature with a focus on studies in the recent COVID-19 era that included vaccinated population and predominance of the Omicron variant. In the base-case analysis, it was assumed the only effect of NMV/r was a reduction in incidence (not severity) of hospitalization or death; its potential effect on post-COVID conditions was assessed in a scenario analysis. Outcomes included the number of hospitalizations, total cost, per patient per year (PPPY) costs, and per member per month (PMPM) costs. Sensitivity and scenario analyses were conducted to assess uncertainty around key model inputs. An estimated 29,999 adults were eligible and sought treatment with oral antiviral for COVID-19 over 1 year. The availability of NMV/r was estimated to reduce the number of hospitalizations by 647 with a total budget impact of $2,733,745, $91 PPPY, and $0.23 PMPM. NMV/r was cost saving when including post-COVID conditions with a -$1,510,780 total budget impact, a PPPY cost of -$50, and a PMPM cost of -$0.13. Sensitivity analyses indicated results were most sensitive to the risk of hospitalization under supportive care, risk of hospitalization with NMV/r treatment and cost of NMV/r. Treatment with NMV/r in the current COVID-19 era is estimated to result in substantial cost offsets because of reductions in hospitalization and modest budget impact to potential overall cost savings.

A Markov model-based cost-effectiveness analysis comparing zanubrutinib to ibrutinib for treating relapsed and refractory chronic lymphocytic leukemia

ABSTRACT Objective This article examined the cost-effectiveness of zanubrutinib and ibrutinib for managing relapsed and refractory chronic lymphocytic leukemia from the viewpoint of payers in China and the US. Methods Markov models were employed to conduct comparisons. Baseline characteristics and clinical data were extracted from the ALPINE study. The cost-effectiveness outcome indicators encompassed cost, quality-adjusted life years, and the incremental cost-effectiveness ratio. Results The Markov model analysis revealed that the zanubrutinib group incurred an incremental cost per patient of $-24,586.53 compared to the ibrutinib group. The zanubrutinib group exhibited an incremental utility per capita of 0.28 quality-adjusted life years, resulting in an incremental cost-effectiveness ratio of $-88,068.16 per quality-adjusted life year, which is lower than the payment threshold in China. The willingness-to-pay value in China for 2022 was three times the country’s gross domestic product per capita. In the US, patients in the zanubrutinib group experienced per capita incremental costs of $-79,421.56, per capita incremental utility of 0.28 quality-adjusted life years, and an incremental cost-effectiveness ratio of $-284,485.45 per quality-adjusted life year. Conclusion For Chinese payers, zanubrutinib exhibited superior cost-effectiveness compared to ibrutinib. Zanubrutinib proved to be a more affordable option for US payers when considering the payment threshold.

Confronting High Costs And Clinical Uncertainty: Innovative Payment Models For Gene Therapies.

Gene therapies offer potentially life-changing benefits for patients, but their unprecedented high prices exacerbate challenges for reimbursement. Payers must confront high budgetary impacts, as a large up-front payment for each patient makes it difficult to predict and absorb costs. Payers also face considerable clinical uncertainty, as evidence for efficacy and durability is limited at approval. Alternative payment models may address these reimbursement problems and ensure equitable patient access. We developed a taxonomy of possible payment mechanisms for gene therapies, including installments, risk pools, reinsurance, price-volume agreements, expenditure caps, subscriptions, outcomes-based payments and rebates, warranties, population outcomes-based agreements, and coverage with evidence development. We illustrate how these payment models take three main approaches: amortization, which mitigates initial budget impact by spreading payments over time; risk spreading, which makes budgets more predictable by pooling costs with other payers or capping costs based on expected volume; and performance-based payment, which addresses clinical uncertainty by tying prices to patient- or population-level outcomes. We discuss each payment model, its advantages and challenges, and considerations for US payers.

The evolution of payer management of oncology drugs in the United States between 2017 and 2022.

BACKGROUND: Research conducted in 2017 by Runyan et al concerning the current and future management of oncology drugs in the United States formed the basis for this research. The authors concluded that despite the high cost of oncology drugs, US payers relied on traditional management tools to manage the category, although these tools were ineffective at controlling costs. Innovative tools were not common in 2017. OBJECTIVE: To compare findings from the 2017 research with findings from a 2022 payer survey to understand how payer management of oncology drugs changed over 5 years. The study evaluates changing trends in oncology drug management. METHODS: The survey that informed the publication by Runyan et al in 2017 was reviewed, updated, and completed by 21 pharmacy and medical directors across 18 organizations representing 121.9 million covered lives. Both surveys included questions about management tools being employed in oncology and challenges to managing oncology. They used case studies in non-small cell lung cancer and chronic lymphocytic leukemia. These disease areas were chosen again in 2022 because they were included in the 2017 survey and because of the increase in competition in both categories from 2017 to 2022. The payer sample was designed to match the 2017 sample. The research was fielded from March to May 2022. The results were analyzed in Microsoft Excel; basic statistical analysis was conducted. Payers' responses for each question were weighted by the number of reported covered lives at their organization so that the organization's site was represented. RESULTS: On average, payers rated the management priority of oncology as a 5.3 and the budget impact as a 6.3 on a scale of 1 to 7, where 1 was low and 7 was high. Traditional tools remain dominant in this therapeutic area. However, there has been an increase in use of innovative tools. Pathways of care are trending upward since the initial survey in 2017. The Institute for Clinical and Economic Review (ICER) also influences payers' decision-making in oncology more than it did 5 years ago. Despite these shifts, most payers allow for unrestricted access of targeted therapies in non-small cell lung cancer and chronic lymphocytic leukemia, in line with each drug's US Food and Drug Administration-approved label. CONCLUSIONS: The increased use of pathways of care, shifting financial risk to providers, and the influence of ICER should continue to be monitored. Future research should focus on the role of pathways of care, comprehensive, evidence-based treatment protocols, in influencing prescribing decisions of hematologists and oncologists. DISCLOSURES: The authors work for Envision Pharma Group (formerly Two Labs), a company that provides consulting services to the pharmaceutical and biotech industries. As such, clients in these industries pay Envision Pharma Group for their services. This study was funded independently by Envision Pharma Group.

Cost-effectiveness analysis of an orphan drug tebentafusp in patients with metastatic uveal melanoma and a call for value-based pricing.

The normative regimens recommendations for treating metastatic uveal melanoma (mUM) are absent in the US. Recently, a phase III randomized clinical trial revealed that tebentafusp yielded a conspicuously longer overall survival than the control group. Based on the prominent efficacy, this study aimed to assess whether tebentafusp is cost-effective compared to the control group in patients with untreated mUM. A three-state partitioned survival model was developed to assess the costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) from the perspective of US payers. Scenario analyses and sensitivity analyses were conducted to explore the conclusion uncertainty. Compared with control group, tebentafusp therapy yielded an additional 0.47 QALYs (1.19 vs. 0.72 QALYs) and an incremental cost of $444 280 ($633 822 vs. $189 542). The resultant ICER of $953 230/QALY far outweighed the willingness-to-pay threshold of $200 000/QALY. The ICER was always more than $750 000/QALY in all the univariable and probabilistic sensitivity analyses. Scenario analyses indicated that reducing the unit price of tebentafusp to $33.768/µg was associated with a favorable result of tebentafusp being cost-effective. For treatment-naive patients with mUM, the cost of tebentafusp therapy was not worth the improvement in survival benefits at the current price compared to the investigator's choice of therapy. The cost-effectiveness of tebentafusp could be promoted using value-based pricing.

The cost-effectiveness analysis of maintenance olaparib plus Bevacizumab in patients with advanced ovarian cancer: based on the final survival results from PAOLA-1 trial

IntroductionIn 2023, the final PAOLA-1 trial (NCT02477644) survival data were published documenting the benefits of therapy consisting of olaparib plus bevacizumab for patients with advanced ovarian cancer (AOC) as a function of molecular status. In light of these new data, the present study was conducted with the goal of evaluating the cost-effectiveness of olaparib plus bevacizumab for the treatment of the overall AOC patient population and for homologous recombination deficiency (HRD)-positive patients, patients with a breast cancer susceptibility gene (BRCA) mutations, homologous recombination proficiency (HRD)-positive, or patients not harboring BRCA mutations AOC from a US payers perspective.MethodsA Markov state-transition model with a 15-year time horizon was used to evaluate outcomes of patients administered Olaparib plus bevacizumab versus bevacizumab. Life-years (LYs), quality-adjusted LYs (QALYs), and the incremental cost-effectiveness ratio (ICER) values were evaluated in this study in light of a $150,000/QALY of willingness-to-pay (WTP) threshold. The stability of the established model was evaluated through sensitivity analyses.ResultsRelative to bevacizumab alone, Olaparib plus bevacizumab was associated with mean incremental costs and QALYs (LYs) of olaparib plus bevacizumab versus bevacizumab were $293,656 and 1.85 (2.16), $265,668 and 3.34 (4.02), $242,746 and 1.71 (2.06), and $193,792 and 0.97 (1.14) for overall, BRCA mutation-positive, HRD-positive, and HRD-positive BRCA mutation-negative AOC patients, respectively. The corresponding ICER values for these patient subgroups were $158,729 ($136,218), $79,434 ($66,120), $141,636 ($117,747), and $200,595 ($169,733) per QALY (LY) gained Utility value and the price of olaparib were identified in sensitivity analyses as the primary factors influencing these results.ConclusionAt current pricing levels, maintenance treatment with olaparib plus bevacizumab treatment may represent a cost-effective therapeutic option for BRCA mutations and HRD-positive AOC patients in the USA.

Budget Impact of Minimally Invasive Surgical Treatments of Benign Prostatic Hyperplasia: An Analysis of 6 States With Limited New Technology Coverage

Purpose: Prostatic urethral lift (PUL) and water vapor thermal therapy (WVTT) for benign prostatic hyperplasia (BPH) differ in treatment durability and cost of disposables, possibly leading to different long-term costs. This study compares the budget impact of PUL and WVTT for US payers in 6 states (Alabama, New Jersey, New York, Oregon, Pennsylvania, and Washington) over a 5-year time horizon. Materials and Methods: An Excel-based budget impact model was developed to estimate average total medical costs per patient of PUL and WVTT at years 1 and 5 for men with moderate-to-severe BPH. After undergoing an index procedure, men could experience adverse events (AEs) or retreatment while accumulating costs over time. Clinical inputs were derived from PUL and WVTT clinical trials while cost inputs were derived from 2021 Medicare reimbursement rates. Results: At year 1, per-patient costs of PUL ranged from $5690 (Alabama) to $7323 (New Jersey) compared with year 1 costs of WVTT that ranged from $1829 (Alabama) to $2330 (New Jersey). The lower year 1 costs of WVTT stemmed from both lower procedural costs relative to PUL and better durability (lower costs associated with fewer retreatments and AEs). The cost differences between the 2 treatments continued to diverge in years 2 to 5. Through year 5, the cost savings associated with WVTT vs PUL ranged from $4383 (Alabama) to $5649 (New Jersey). Conclusions: Compared with PUL, the use of WVTT could potentially allow payers to cover BPH treatments for more members without compromising clinical outcomes or increasing budgets.