Objectives Novel first-line (1L) combination regimens offer improved efficacy for patients with epidermal growth factor receptor-mutated (EGFRm) locally advanced or metastatic non-small cell lung cancer (NSCLC) versus the standard of care, but their costs have not been comprehensively evaluated. Total and differential costs of 1L osimertinib plus platinum-pemetrexed, intravenous (IV) amivantamab plus lazertinib, and subcutaneous (SC) amivantamab plus lazertinib were estimated from a United States (US) payer perspective. Methods A cost of care model was developed to assess treatment acquisition, administration, disease management, and adverse event (AE) management costs for osimertinib plus platinum-pemetrexed versus IV and SC amivantamab plus lazertinib for patients with EGFRm NSCLC. A one-year time horizon was chosen to allow for direct treatment comparison; a one-year treatment duration was assumed for treat-to-progression regimen components. Results The total first-year cost per-patient for osimertinib plus platinum-pemetrexed (all-payer perspective: $172,600; private perspective: $253,529; Medicare perspective: $84,926) was over twofold lower than for IV amivantamab plus lazertinib (all-payer perspective: $418,176; private perspective: $618,240; Medicare perspective: $201,441) and for SC amivantamab plus lazertinib (all-payer perspective: $419,718; private perspective: $621,166; Medicare perspective: $201,482). This translated to per-patient cost savings of $245,577; $364,711; and $116,514 for osimertinib plus platinum-pemetrexed versus IV amivantamab plus lazertinib and $247,118; $367,637; and $116,555 versus SC amivantamab plus lazertinib for the all-payer, private, and Medicare perspectives, respectively. Limitations Limited data availability necessitated cost conversions across payer perspectives and assumptions for select inputs, including specific concomitant medication durations. Additionally, as any payer-negotiated discounts for treatment are confidential, model treatment acquisition costs reflected list prices. These data limitations contributed to uncertainty surrounding model inputs. Conclusions From a US payer perspective, osimertinib plus platinum-pemetrexed was associated with substantially lower overall costs compared with IV and SC amivantamab plus lazertinib for the 1L treatment of patients with EGFRm locally advanced or metastatic NSCLC.

This retrospective observational cohort study aimed to assess healthcare utilization (HCRU) and costs before and after initiation of treatment with maribavir using a United States (US) administrative claims database to further the understanding of the impact of maribavir in the real world setting. Inovalon Databases were used to identify post-transplant patients with refractory (with or without resistant) CMV treated with maribavir between November 1, 2021 and March 31, 2024 (index = earliest treatment date). HCRU and costs were assessed during variable length periods both prior to and following initiation of maribavir. McNemar's tests were used to evaluate the statistical significance of differences for categorical variables and paired t-tests were used for continuous variables. There were 230 patients eligible for analysis. Compared with the pre-maribavir phase, after the initiation of maribavir there was a significant decrease in the utilization of all-cause acute care services, including inpatient hospital admissions (54.3% v. 30.9%; p < 0.001) and emergency room visits (47.0% v. 36.5%; p = 0.014). There was a significant decrease in the number of outpatient office visits per patient per month (PPPM) (2.33 v. 1.47; p < 0.001) as well as a decrease in the number of all outpatient pharmacy prescriptions PPPM (9.64 v. 7.69; p < 0.001). Total medical costs measured PPPM prior to maribavir initiation were $9,986 compared with $5,480 (p < 0.001) after initiation of maribavir. In real world practice, initiation of maribavir for the treatment of refractory or drug-resistant post-transplant CMV was associated with decreased acute care service utilization, reduced outpatient visits, and lower healthcare costs, resulting in meaningful benefits for both patients and healthcare systems. These reductions in healthcare utilization highlight maribavir's role in optimizing treatment strategies and improving the efficiency of CMV management.

Background Despite clinical efficacy and cost-effectiveness, time to reimbursement for chimeric antigen receptor (CAR) T-cell therapies varies greatly across Europe. We examined these differences, and quantified potential patient benefits with faster access. Methods A targeted literature search collated reimbursement statuses for all approved CAR T-cell therapy indications by 1 October 2024. Time to reimbursement decision (TTRD), defined as time between marketing authorization and published reimbursement decision, was assessed. By benchmarking against the TTRD of the three fastest countries, patient impact, in the form of potential gains in lives, life-years (LYs) and long-term survivors, for the first three approved indications for CAR T-cell therapies were estimated. Each outcome was multiplied by population size, incidence, CAR T-cell eligibility, and market share to obtain population-level impact. Scenario analyses were conducted by examining different TTRD assumptions. Results Across 12 identified indications for CAR T-cell therapy, France, Germany, and Switzerland had the fastest TTRD, compared to Denmark, the Netherlands, and Ireland which had the slowest. Among an estimated annual incident population of 6,594 across 3 L + diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and pediatric acute lymphoblastic leukemia (ALL), 1,199 lives, 1,222 long-term survivorships, and 37,229 LYs, could potentially be gained by ensuring time to access across these countries was equivalent to the fastest three assessors in these 3L + DLBCL, MCL, and pediatric ALL. Conclusion Access to CAR T-cell therapies varied widely across European countries. Faster access to these therapies can result in significant health gains. Ensuring prompt and equitable access to innovative therapies requires proactive alignments between manufacturers and HTA bodies, possibly through mechanisms such alternative pricing models and risk-sharing agreements.

Aims Early detection of hepatocellular carcinoma (HCC) in patients with compensated cirrhosis (CC) is critical for improving prognosis. The GAAD algorithm (gender [biological sex], age, alpha-fetoprotein [AFP], protein induced by vitamin K absence-II [PIVKA-II]) demonstrated good performance for the detection of early-stage HCC. This study aimed to assess the cost-effectiveness of the GAAD algorithm for HCC surveillance in patients with CC in Italy, from the Italian Health Service perspective. Methods A probabilistic micro-simulation Markov model was adapted to the Italian context to estimate lifetime clinical outcomes and costs of CC patients undergoing bi-annual surveillance with ultrasound (US), US+AFP, GAAD, and US+GAAD. Clinical inputs and utility values were derived from Italian real-world data and published literature. Direct healthcare costs were collected from Italian sources. Costs and outcomes were discounted at an annual 3% rate. Sensitivity analyses were conducted to evaluate the uncertainties in input parameters. Results In a simulated cohort of 100,000 CC patients, QALYs and costs per patient were 6.53 and €35,524 for US, 6.56 and €35,825 for US+AFP, 6.57 and €35,423 for GAAD, and 6.58 and €35,939 for US+GAAD. Compared to US and US+AFP, GAAD was dominant, while US+GAAD was cost-effective (ICUR of €9,482 and €10,951 per QALY gained, respectively). At a willingness-to-pay threshold of €30,000, GAAD was the most cost-effective strategy. Sensitivity analyses confirmed the robustness of results. Limitations Assumptions were required to estimate the diagnostic performance of US+GAAD, given the absence of prospective validation data. Some clinical parameters were derived from non-Italian sources, which may limit generalizability. Conclusion GAAD, alone or combined with US, is a cost-effective strategy for HCC surveillance in CC patients in Italy, improving the detection of early-stage disease. Better performance data for US+GAAD is needed to confirm results.

Detecting and treating cancer at an early stage is critical for improving patient survival, quality of life, and health system efficiency. Early diagnosis offers substantial clinical benefits, reduces the need for aggressive treatments associated with advanced disease, and lowers healthcare costs. Despite these benefits, disparities in early-stage detection persist across tumor types due to challenges in screening, public awareness, and the aggressive nature of certain cancers. While early-stage diagnosis generally offers a better prognosis than late-stage detection, disease recurrence remains a significant reality and concern. Many patients experience a relapse of cancer despite initial curative treatment, which adversely affects their survival, quality of life, and financial stability. While effective new treatments for early-stage cancers have emerged, including immunotherapy and targeted therapies, barriers to reimbursement and access persist. One challenge is the absence of mature overall survival data at the time of regulatory and reimbursement approvals for most tumor types, which can result in delayed decision-making, reduced patient access, and worse outcomes. This policy paper combines insights from clinicians, health economists, outcomes researchers, and policy experts to address gaps in early-stage cancer care and provide recommendations to enhance diagnosis rates, reduce the burden of recurrence, and optimize access to innovative treatments. Central to these recommendations is the integration of early cancer care into national cancer control plans, including robust data collection and monitoring, as well as improvements in health literacy. A key factor is the use of early clinical endpoints that measure key outcomes in addition to overall survival, providing timely insights into treatment effectiveness that can guide early regulatory and reimbursement decisions prior to reaching overall survival maturity. This paper is a call to action for relevant stakeholders to take a coordinated approach that optimizes outcomes for cancer patients by promoting early detection and treatment.

Aims Intravenous (IV) lecanemab is approved for the treatment of patients with early Alzheimer’s disease (AD); a subcutaneous (SC) option may offer additional benefits. We assessed the overall value of SC treatments, and direct/indirect outcomes associated with IV and SC lecanemab. Methods and materials For the narrative review, PubMed was searched (February 2025) for studies comparing patient preferences for IV/SC treatment administration published between 2015–2025. Study eligibility was determined using patient, intervention, comparator, outcomes, and study criteria. For the decision-analytic model, a Markov model was developed with four lecanemab treatment scenarios. Scenarios one to three included IV initiation (10 mg/kg biweekly) to month 18, followed by either IV initiation continued (10 mg/kg biweekly), SC maintenance (250 mg weekly) or IV maintenance (10 mg/kg every 4 weeks). Scenario four included SC initiation (500 mg weekly) for an 18-month period, followed by SC maintenance (250 mg weekly). Outcomes were administration time/frequency; patient, caregiver, and healthcare professional time; and caregiver productivity loss. Results Forty-three publications reported patient treatment preferences. Most (88.4%) reported that patients preferred SC over IV. Key reasons for this were time savings (n = 13/43 studies; 30.2%), convenience (n = 11/43; 25.6%), and treatment frequency (n = 12/43; 27.9%). Two studies (n = 2/43; 4.7%) reported an IV preference over SC; for three studies (n = 3/43; 7.0%), treatment preference was driven by administration frequency. Decision-analytic modeling of lecanemab treatment scenarios revealed that IV initiation to IV maintenance had the lowest number of administrations, whereas SC initiation to SC maintenance had the lowest number of treatment hours and caregiver productivity losses. Limitations Caution must be taken when generalizing these results for all AD patients. Conclusions SC treatments show value as a therapeutic option. IV and SC lecanemab availability may offer benefits to patients, caregivers, and society, and improve shared decision making.

Background and Aim Bruton tyrosine kinase inhibitors (BTKis) are the standard of care for patients with relapsed/refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Ibrutinib and acalabrutinib are two commonly used BTKis, though hypertension (HTN) and other cardiovascular medical events of interest (CV MEOIs) can impact treatment tolerability and long-term use. This study compared cardiovascular safety and health care resource use (HCRU) of acalabrutinib versus ibrutinib monotherapy for R/R CLL/SLL in real-world community practice. Methods This retrospective comparative study included 270 RR CLL/SLL patients (90 acalabrutinib; 180 ibrutinib) treated from January 2017 through December 2023 across the ONCare Alliance Network, comprising 30 US community hematology practices. Endpoints included new or worsening of existing HTN, development of CV MEOIs, and treatment discontinuation due to adverse events. HCRU metrics consisted of emergency department visits, hospital admissions, length of stay, specialist consultations, and related medical procedures. Propensity score weighted multivariable logistic regression was used for the comparative analysis on the tolerability endpoints. Results At a median follow-up of 33 months, patients treated with acalabrutinib had significantly fewer HTN events (OR = 0.27, 95% CI = 0.074–0.98; p = .046) and adverse events leading to treatment discontinuation (OR = 0.39, 95% CI = 0.20–0.73; p = .002) compared to those on ibrutinib. Hospital admission rates for MEOIs were lower in the acalabrutinib group (0.20 vs. 0.24 per patient), with a shorter median length of stay (3.0 vs. 6.0 days). Additionally, acalabrutinib patients had fewer specialist consultations (0.11 vs. 0.22 per patient) and associated medical procedures (0.11 vs. 0.18 per patient). Conclusions In R/R CLL/SLL, acalabrutinib demonstrated a more favorable cardiovascular safety profile than ibrutinib, with fewer HTN events and CV MEOIs. This translated into reduced hospital admissions and lower HCRU, supporting acalabrutinib as a potentially better tolerated long-term treatment option in community practice.

Background and aim Pulmonary arterial hypertension (PAH) is a rare, progressive disease associated with significant morbidity and mortality. Recent clinical studies (STELLAR [NCT04576988] and ZENITH [NCT04896008]) showed that adding sotatercept to standard of care improves clinical outcomes in patients with PAH. This study aimed to assess the clinical and economic impact of gradually adding sotatercept to currently available treatments for PAH in Italy. Methods A Markov model was developed to simulate the disease pathway of PAH patients. The model consisted of six health states: low risk PAH, intermediate-low risk PAH, intermediate-high risk PAH, high risk PAH, post lung/heart transplant, and death. Using this model, a cost-consequence analysis was performed to calculate the number of PAH related health events and associated costs over a 3-year time horizon assuming a gradual increase in the use of sotatercept. Primary outcomes included number of hospitalizations, lung/heart transplants, PAH-related deaths, and disease management costs. Sensitivity analyses were conducted to examine model robustness. Results Over a 3-year time horizon, the model predicted a total of 270 deaths, 1 lung/heart transplantation and 430 hospitalizations in the scenario with sotatercept. For the scenario without sotatercept, the model projected a total of 408 deaths, 2 transplants and 720 hospitalizations. Adding sotatercept to background therapy resulted in a reduction of 289 hospitalizations, 1 transplant and 138 deaths. Associated disease management costs were estimated to be €33.8 million in the scenario with sotatercept and €38.2 million in the scenario without sotatercept. Sotatercept plus background therapy was also associated with a reduction in costs of €4.4 million (11.8%). Discussion and conclusions This cost-consequence analysis showed that the introduction of sotatercept as an add-on therapy for the treatment of patients with PAH in Italy has the potential to reduce mortality, hospitalizations and lung/heart transplants, thereby substantially reducing PAH disease management costs.

Objective To inform potential negotiations under the Medicare Drug Price Negotiation Program, this study evaluates the cost-effectiveness of ribociclib versus palbociclib as first-line (1 L) treatment for patients with HR+/HER2– advanced breast cancer (aBC) from the Medicare perspective. Methods A three-way partitioned survival model with states defined by progression-free survival (PFS) and overall survival (OS) was estimated over a lifetime horizon with a 28-day cycle length using data from Phase III MONALEESA-2 (ribociclib) and PALOMA-2 (palbociclib) trials. Relative treatment efficacy was measured using PFS and OS HRs derived from an anchored matching adjusted indirect comparison. Incremental cost-effectiveness ratios (ICERs) were calculated with costs estimated from a Medicare perspective and effectiveness measured using quality adjusted life years (QALYs), life years (LYs), progression-free life years (PFLYs), equal value life years (evLYs), and health years in total (HYT). All effectiveness and cost measures were discounted at 3%. A willingness-to-pay (WTP) threshold of $150,000 per QALY/evLY was used to assess cost-effectiveness. Results Measures of effectiveness consistently favoured ribociclib with estimated gains of 1.40 QALYs, 1.83 LYs, 1.08 PFLYs, 1.59 evLYs, and 1.80 HYT relative to palbociclib. Total costs were comparable, with ribociclib associated with a marginal 1.8% lifetime cost increase. ICERs ranged from $4,697 per LY gained to $7,973 per PFLY gained. At a WTP threshold of $150,000 per evLY, ribociclib demonstrated an approximately 100% probability of being cost-effective relative to palbociclib. Conclusion Ribociclib improved health outcomes with a minimal relative cost increase and is therefore a highly cost-effective 1 L treatment option vs. palbociclib in patients with HR+/HER2– aBC from the Medicare perspective.