Journal Of Medical Economics[JOURNAL]
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200 papers
Modeling the cost-effectiveness of the next-generation COVID-19 mRNA-1283 vaccine in the United States.
Fust K, Kohli M, Joshi K … +5 more , Cartier S, Lee A, Van de Velde N, Weinstein M, Beck E
J Med Econ · 2026 Dec · PMID 41764030 · Publisher ↗
AIMS: COVID-19 disease burden in United States (US) adults ≥65 years and persons with underlying medical conditions remains high. This modeling study estimated the cost-effectiveness of the next-generation COVID-19 mRNA-...
AIMS: COVID-19 disease burden in United States (US) adults ≥65 years and persons with underlying medical conditions remains high. This modeling study estimated the cost-effectiveness of the next-generation COVID-19 mRNA-1283 vaccine in persons aged 12-64 at high risk of severe COVID-19 outcomes and all adults ≥65 years. METHODS: mRNA-1283 was compared with no annual vaccination and originally licensed mRNA vaccines mRNA-1273 and BNT162b2. Analyses were conducted using a static decision-analytic model (1-year horizon). Vaccine effectiveness (VE) against infection and hospitalization for mRNA-1283 versus no vaccination was based on relative VE (rVE) from the Phase 3 pivotal randomized controlled trial comparing mRNA-1283 against mRNA-1273, and mRNA-1273 real-world data. rVE estimates for mRNA-1283 versus BNT162b2 were based on an indirect treatment comparison. The societal incremental cost per quality-adjusted life-year (QALY) gained and the benefit-cost ratio (BCR) were calculated. RESULTS: During the 2025/2026 season, a single dose of mRNA-1283 was estimated to yield an incremental cost per QALY gained of $16,247 compared with no vaccine. The BCR for the base case strategy ranged from $2.16-9.74 returned for every $1 spent for mRNA-1283. mRNA-1283 was estimated to dominate originally-licensed mRNA-COVID-19 vaccines in analyses of the target population. Results were sensitive to COVID-19 incidence, hospitalization rates, post-discharge mortality rates, and VE. LIMITATIONS: The real-world effectiveness and safety of mRNA-1283 have not yet been established, and relative VE estimates should be validated with real-world data. Full year 2025/2026 COVID-19 incidence and vaccine uptake in the US is uncertain. CONCLUSIONS: Study results suggest mRNA-1283 may represent a highly cost-effective strategy (considering a $100,000-150,000 per QALY willingness-to-pay threshold) to reduce COVID-19 burden. Based on rVE assumptions made, mRNA-1283 was estimated to dominate originally-licensed mRNA vaccines in this recommended population. mRNA-1283 may provide a valuable option to optimize US COVID-19 immunization programs and protect those most vulnerable.
Publisher's note.
J Med Econ · 2026 Dec · PMID 41763986 · Publisher ↗
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Breaking barriers in women's pelvic health: claims-based economic analysis and healthcare utilization of an AI care program compared to usual care.
Pereira AP, M Seet A, Domingues B … +11 more , Janela D, Pradhan A, C Areias A, Tong X, Bento V, Yanamadala V, Cohen SP, Atherton J, Dias Correia F, Belz L, Costa F
J Med Econ · 2026 Dec · PMID 41733548 · Publisher ↗
AIM: To compare healthcare utilization and spending among women enrolled in an employer-sponsored, artificial intelligence (AI) structured pelvic care program with those receiving usual in-person care for pelvic floor dy...
AIM: To compare healthcare utilization and spending among women enrolled in an employer-sponsored, artificial intelligence (AI) structured pelvic care program with those receiving usual in-person care for pelvic floor dysfunction (PFD) in routine clinical settings. METHODS: This retrospective payor-perspective economic evaluation used exact and propensity score-matched cohorts derived from a third-party U.S. nationwide claims database from July 2022 to May 2025. Eligible participants were adult females with a pelvic-related condition, at least 24 months of continuous health-insurance coverage, and a minimum of one pelvic claim in the prior year. Intervention group (IG) comprised women who participated in the AI pelvic care program (consisting of biofeedback-mediated pelvic floor muscle training asynchronously monitored by a physical therapist specialized in pelvic health). Comparator group (CG) included women who sought a medical or physical therapy evaluation visit for PFD. Self-reported clinical outcomes available for the IG were assessed using latent-basis growth analysis. RESULTS: The matched cohort included 602 women (301 per group). Relative to CG, IG patients had substantially lower healthcare spending over 12 months, with mean gross per-person pelvic-related savings of $3,082.4 (95% CI $1,270.2 to $4,894.7, <.001). Savings were primarily associated with fewer surgical procedures (per-person difference of $2,534.2; 95% CI $831.2 to $4,237.2, =.004), with differences also noted in medical office visits and imaging utilization. IG participants demonstrated significant improvements in pelvic floor symptom burden, work productivity, and mental health. LIMITATIONS: Claims-based analyses cannot exclude unmeasured confounding, misclassification, or selection bias. The one-year follow-up limits assessment of long-term economic impact. CONCLUSIONS: Participation in this AI pelvic care program was associated with markedly lower healthcare utilization and spending compared with usual care, largely linked to fewer surgical interventions. These findings highlight the potential of accessible, guideline-concordant AI pelvic care to lessen healthcare spending associated with PFD and inform payor-oriented care delivery models.
Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United States.
Lopez A, Gargano M, Yang H … +5 more , Ogunsile FJ, Li N, Xie Y, Jeyakumar S, Udeze C
J Med Econ · 2026 Dec · PMID 41730016 · Publisher ↗
OBJECTIVE: Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 years with sickle cell disease (SCD) with recurrent vaso-occl...
OBJECTIVE: Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 years with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). Standard of care (SOC) for SCD includes symptomatic care, hydroxyurea and/or red blood cell transfusions. This study estimated the long-term clinical outcomes and cost-effectiveness of exa-cel relative to SOC among patients with SCD with recurrent VOCs. METHODS: A Markov model was used to compare the expected lifetime costs and clinical outcomes of patients with SCD with recurrent VOCs treated with exa-cel versus SOC from the US payer and societal perspectives. The model structure is based on disease severity, characterized by VOC frequency, which impacts the risk of developing SCD-related complications and mortality. The model incorporated data from the phase 3 pivotal CLIMB SCD-121 trial alongside published literature. Model outcomes included number of VOCs and other acute complications, proportion of patients developing chronic complications, life years (LYs), quality-adjusted LYs (QALYs), costs, and incremental cost-effectiveness ratios (ICERs). RESULTS: Over a lifetime horizon, exa-cel was projected to improve survival by 30.8 years (mean age of death, exa-cel: 74.5 vs. SOC: 43.6), reduce the number of VOC events by 77 (7 vs. 84), and reduce undiscounted disease-related costs by $3.34 M ($0.55 M vs. $3.89 M) compared to treatment with SOC. Patients treated with exa-cel also were less likely to experience acute complications or develop chronic complications compared to SOC. The ICER per discounted QALY for exa-cel versus SOC was $16,800 from the payer perspective; exa-cel was dominant (less costly, more effective than SOC) from the societal perspective. CONCLUSIONS: Compared to SOC, exa-cel was projected to considerably reduce the number of VOCs, improve survival, and reduce disease-related costs in patients with SCD. Exa-cel was projected to be a cost-effective treatment option.
Economic evaluation of perioperative pembrolizumab plus standard of care as treatment for resectable locally advanced head and neck squamous cell carcinoma in the United States.
Qian F, Bensimon AG, Tzontcheva A … +9 more , Benjamin K, Uppaluri R, Adkins D, Johnson G, Fernan C, Zheng D, Tang Y, Chafamo B, Muston D
J Med Econ · 2026 Dec · PMID 41730003 · Publisher ↗
AIMS: In the phase 3 KEYNOTE-689 trial (NCT03765918) among patients with resectable locally advanced head and neck squamous cell carcinoma (LA HNSCC), perioperative pembrolizumab (pembrolizumab before surgery, then conti...
AIMS: In the phase 3 KEYNOTE-689 trial (NCT03765918) among patients with resectable locally advanced head and neck squamous cell carcinoma (LA HNSCC), perioperative pembrolizumab (pembrolizumab before surgery, then continued with standard-of-care [SOC] radiotherapy +/- cisplatin after surgery followed by pembrolizumab alone) significantly prolonged event-free survival vs. SOC alone, both in the intention-to-treat population and PD-L1 combined positive score (CPS) ≥1 subgroup. Perioperative pembrolizumab + SOC received Food and Drug Administration approval in June 2025 for resectable LA HNSCC expressing PD-L1 (CPS ≥ 1). The present study evaluated the cost-effectiveness of perioperative pembrolizumab + SOC versus SOC in this indication, from a US healthcare payer perspective. MATERIALS AND METHODS: A Markov cohort model with four states (event-free, local recurrence, incurable recurrence/progression, death) was developed to estimate lifetime costs, life years (LYs), and quality-adjusted LYs (QALYs) with 3% annual discounting. Transition probabilities were fitted to patient-level time-to-event data from KEYNOTE-689 through parametric multistate modelling. Costs of initial and subsequent treatment, adverse events, disease management, and terminal care were estimated in 2025$ based on trial results, drug labels, public databases, and literature. Utilities were derived through analyses of EQ-5D-5L data collected in KEYNOTE-689. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Compared to SOC, perioperative pembrolizumab + SOC increased total costs by $82,311 and provided gains of 1.47 QALYs and 1.77 LYs. Incremental cost-effectiveness ratios of perioperative pembrolizumab + SOC vs. SOC were $55,863/QALY and $46,406/LY. Higher initial treatment costs of perioperative pembrolizumab (incurred mainly in Year 1) were partially offset by lower recurrence-related costs. At the typical $150,000/QALY threshold, perioperative pembrolizumab + SOC was cost-effective in 96% of probabilistic simulations. LIMITATIONS: Survival extrapolations beyond the available trial period are subject to uncertainty. CONCLUSIONS: Perioperative pembrolizumab + SOC was found to be cost-effective versus SOC for the treatment of resectable LA HNSCC with CPS ≥ 1.
Real-world treatment switching and healthcare costs of onabotulinumtoxinA and calcitonin gene-related peptide monoclonal antibodies in Medicare patients with chronic migraine: a retrospective claims analysis.
Dong Y, Mao Z, Dominguez A … +1 more , Park TJ
J Med Econ · 2026 Dec · PMID 41725292 · Publisher ↗
AIMS: Evaluate treatment switching and healthcare costs of onabotulinumtoxinA (onabotA) compared to calcitonin gene-related peptide (CGRP) monoclonal antibodies (mAbs) for the preventive treatment of chronic migraine (CM...
AIMS: Evaluate treatment switching and healthcare costs of onabotulinumtoxinA (onabotA) compared to calcitonin gene-related peptide (CGRP) monoclonal antibodies (mAbs) for the preventive treatment of chronic migraine (CM). MATERIALS AND METHODS: Adult patients with CM who initiated onabotA or a CGRP mAb between 1 October 2021 and 31 December 2023 were identified from the Optum de-identified Clinformatics Data Mart database. Index date was the first recorded treatment claim and patients must have had continuous Medicare coverage 12-months pre- and post-index period. Treatment switching, defined as ≥1 claim for a different branded migraine preventive treatment in the 12 months post-index period, was evaluated. Two additional treatment switch definitions were evaluated. All-cause healthcare resource utilization and costs were evaluated over the 12-month follow-up. Multivariable logistic regression adjusted for baseline characteristic differences when comparing odds of switching between onabotA and CGRP mAbs. Patient information on CM severity was not available in the database and not reported. RESULTS: Of 887 patients identified, 367 initiated onabotA and 520 a CGRP mAb as index treatment. After 12 months of follow-up, 8.7% of onabotA users and 18.3% of CGRP mAb users initiated a different branded migraine preventive treatment. After adjusting for differences in baseline characteristics, CGRP mAb users had 134% higher odds of switching treatment during the 12-month follow-up compared to onabotA users (OR, 2.34; 95% CI: 1.49, 3.67, < 0.001), or 9.3% absolute risk difference. Results for additional treatment switch definitions were consistent. OnabotA and CGRP mAb users had comparable all-cause healthcare costs during the 12-month follow-up. LIMITATIONS: Outcomes could only be adjusted for known and observed confounders, which could introduce bias between comparators. CONCLUSIONS: Patients with CM on a CGRP mAb were significantly more likely to switch to a different branded migraine preventive treatment within 12 months of treatment initiation compared to those on onabotA. Total costs were comparable between treatments.
Plain language summary examining clinical event related costs associated with changing medication from apixaban to rivaroxaban in people with non-valvular atrial fibrillation in the USA and Germany.
Subash R, Duan C, Hines DM … +4 more , Zhang M, Kongnakorn T, Dworatzek E, Kisser A
J Med Econ · 2026 Dec · PMID 41725198 · Publisher ↗
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Economic burden of advanced non-small cell lung cancer (NSCLC): a systematic literature review.
Jovanoski N, Kaur G, Shukla H … +2 more , Chana N, Kharawala S
J Med Econ · 2026 Dec · PMID 41723781 · Publisher ↗
AIMS: A systematic review of the economic burden of advanced non-small-cell lung cancer (NSCLC). METHODS: Articles from 2011 onwards reporting the economic burden of locally advanced (stage IIIB/C)/metastatic (stage IV)...
AIMS: A systematic review of the economic burden of advanced non-small-cell lung cancer (NSCLC). METHODS: Articles from 2011 onwards reporting the economic burden of locally advanced (stage IIIB/C)/metastatic (stage IV) NSCLC were identified through systematic and supplementary searches. Outcomes included hospitalizations, emergency department (ED) and outpatient visits, and direct and indirect costs, amongst others. RESULTS: Across 50 publications (43 studies), patients with advanced NSCLC had high rates of healthcare resource utilization (HCRU), with most reporting hospitalization (ranging from 13.0% to 98.2% of patients), ED visits (2.5% to 83.1%), outpatient visits (74.6% to 100.0%), and diagnostic or monitoring tests (45.9% to 92.0%). HCRU (hospitalizations, ED visits and pharmacy visits) appeared to be lower with immunotherapy as compared to chemotherapy. Brain/central nervous system (CNS) metastases were the major clinical factor influencing HCRU. Mean direct costs ranged from US$5,647 (Brazil) to US$158,908 (US) over 12-24 months, and were generally higher in the US, Korea, Germany, and the UK (vs. Brazil, France, and Italy). The main direct cost drivers were drug-related costs (9.5-76.0% of total), overall outpatient costs (39-70.6%), and inpatient costs (5.0-58.1%). Costs were higher for chemotherapy than for immunotherapy. In China, indirect medical costs were US$1,413 per case. In general, mean total healthcare costs were higher for metastatic disease. Disease severity/diagnosis, presence of brain/CNS metastases, targeted therapy and chemotherapy (vs. immunotherapy) and the presence of comorbidities were the main factors influencing higher costs. LIMITATIONS AND CONCLUSIONS: Patients with advanced NSCLC had high rates of HCRU, and costs were substantial, though varying greatly across countries. HCRU and costs were higher in patients with brain/CNS metastases. Since this was a qualitative review, no formal quantitative synthesis was attempted. Costs reported in different currencies and heterogeneity across studies limited comparability. Finally, a single reviewer extracted data.
The healthcare resource utilization and costs associated with metabolic dysfunction-associated steatohepatitis among Medicare beneficiaries: a retrospective cohort study.
Albarmawi H, Tan A, Aly A … +5 more , Hoovler A, Zhong C, Vaccaro J, Harton J, Mantry P
J Med Econ · 2026 Dec · PMID 41719199 · Publisher ↗
AIMS: The main objective of this study was to estimate the incremental healthcare resource utilization (HCRU) and costs attributable to metabolic dysfunction-associated steatohepatitis (MASH) from a Medicare fee-for-serv...
AIMS: The main objective of this study was to estimate the incremental healthcare resource utilization (HCRU) and costs attributable to metabolic dysfunction-associated steatohepatitis (MASH) from a Medicare fee-for-service perspective by comparing beneficiaries diagnosed with MASH with those not diagnosed with MASH. MATERIAL AND METHODS: This observational study used 100% Medicare fee-for-service claims data from January 1, 2016, through December 31, 2022. The study population was stratified in cohorts based on MASH status. In the main analysis, the MASH cohort included all beneficiaries diagnosed with MASH, while the non-MASH cohort comprised a random sample of beneficiaries without a MASH diagnosis, matched in size to the MASH cohort. To compare the 2 cohorts, stabilized inverse probability of treatment weighting (IPTW) was used to adjust for differences in baseline covariates, including selected cardiometabolic conditions. Reported outcomes included all-cause, cardiovascular-related, and liver-related HCRU and costs after IPTW. RESULTS: The study included 128 622 beneficiaries in the MASH cohort and 128 579 beneficiaries in the non-MASH cohort. After IPTW, MASH was associated with higher all-cause HCRU rates, particularly for inpatient hospitalizations (rate ratio, 1.36; 95% CI, 1.33-1.39). This increase appeared to be driven by liver-related hospitalizations (rate ratio, 10.41; 95% CI, 9.40-11.42). Consistent with HCRU findings, mean total cost per patient per year was higher for MASH compared with non-MASH ($27 816 vs $25 666; mean cost difference, $2150; 95% CI, $1673-$2627). LIMITATIONS: The HCRU and cost attributed to MASH could be underestimated because of MASH underdiagnosis and underreporting, as well as potential overadjustment for MASH-driven comorbidities in the IPTW model. CONCLUSIONS: Among Medicare fee-for-service beneficiaries aged 66 years and older, MASH was associated with significantly greater HCRU and costs, even after adjustment for cardiometabolic and other comorbidities. The higher HCRU and costs are likely driven by the management of liver disease, which may include cirrhosis and hepatic decompensation.
Clinical and economic benefits of combined genetic and genomic testing strategies to guide treatment decisions for patients with HR+/HER2- breast cancer in the US.
Gouldson M, Le Q, Millen S … +8 more , Racz J, Arrick B, Hartzfeld D, Heald B, Eymere S, De La O JP, Berdunov V, Cuyun Carter G
J Med Econ · 2026 Dec · PMID 41711467 · Publisher ↗
BACKGROUND: In the US, breast cancer is the most common female cancer. Personalized treatment strategies, informed by genomic assays and germline genetic testing (GGT), can optimize therapeutic decisions and improve pati...
BACKGROUND: In the US, breast cancer is the most common female cancer. Personalized treatment strategies, informed by genomic assays and germline genetic testing (GGT), can optimize therapeutic decisions and improve patient outcomes along the hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer treatment pathway. PURPOSE: To evaluate the cost-effectiveness of combining molecular tools, including the 21-gene assay, GGT, and comprehensive genomic profiling (CGP) when compared to no testing, to inform the treatment of patients with HR+/HER2- breast cancer from the US societal perspective. METHODS: A health economic model was developed which stratified patients with node-negative (N0) and node-positive (1-3 positive nodes) (N1) early-stage breast cancer according to the 21-gene assay and GGT. Long-term outcomes were simulated using a Markov model. CGP was used to stratify patients who progressed to advanced disease for actionable genomic alterations. Clinical inputs and costs were sourced from published literature. A clinician survey was used for inputs to reflect current US clinical practice. RESULTS: Over a lifetime horizon, the full testing strategy was dominant (more effective and cost-saving) in N0 patients and cost-effective in N1 (ICER = $54,734/QALY) patients. The model estimated quality-adjusted life year (QALY) gains of 0.322 and cost savings of $7,168 for N0 patients and 0.130 QALYs gain and additional costs of $7,109 for N1 patients. Sensitivity analyses supported the robustness of the analysis. CONCLUSION: Compared to no testing, the full testing strategy of genetic and genomic testing was more effective at a lower cost or was cost-effective, supporting the goal to increase the survival and the quality-of-life of women with breast cancer along the cancer care continuum.
Correction.
J Med Econ · 2026 Dec · PMID 41708479 · Publisher ↗
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Health state utilities for relapsed or refractory large B-cell lymphoma treatments: a time trade-off study in the Japanese general population.
Fuji S, Ando H, Takaura K … +6 more , Ishii K, Kawaguchi I, Alhasani S, Matsuyama F, Murata T, Liao L
J Med Econ · 2026 Dec · PMID 41705966 · Publisher ↗
AIM: Conventional therapies in Japan for relapsed or refractory large B-cell lymphoma (r/r LBCL) require intravenous infusion (IV), while epcoritamab is administered subcutaneously. Despite the expected quality of life (...
AIM: Conventional therapies in Japan for relapsed or refractory large B-cell lymphoma (r/r LBCL) require intravenous infusion (IV), while epcoritamab is administered subcutaneously. Despite the expected quality of life (QOL) improvement from reduced drug administration burden, limited data exists on QOL for common r/r LBCL treatment modalities. This study aimed to estimate the impact of drug administration on QOL (i.e. process utility) across treatments for r/r LBCL using responses from the Japanese general population. METHODS: An in-person questionnaire survey using the vignette-based time trade-off method was conducted. Participants from the Japanese general population, aged 20 years or older who were residing in Japan at the time of screening, valued vignettes representing therapy routes and schedules of epcoritamab monotherapy, salvage chemotherapy (R-ICE and Pola-BR as representatives) or no treatment. These vignettes were developed based on previous studies, clinical trial data, and expert opinions. Descriptive statistics were calculated for each vignette's utility values and the differences in utility between vignettes. RESULTS: The survey included 308 participants (mean age: 45.5 years; female: 50%). Utility for epcoritamab monotherapy was 0.459 for Cycle 1, 0.585 for Cycle 2-3, 0.642 for Cycle 4-9 and 0.678 for Cycle 10+ and beyond. Utilities for R-ICE were 0.151 for Cycle 1 and 0.380 for Cycle 2+; for Pola-BR were 0.216 for Cycle 1 and 0.490 for Cycle 2+. Relative to Pola-BR and R-ICE, epcoritamab was associated with higher utility values at all corresponding cycles, with differences of 0.243 and 0.307 in Cycle 1 and 0.094 and 0.205 in Cycles 2-3, respectively. CONCLUSIONS: This study provides early economic insights, based on general population preferences, suggesting that epcoritamab's subcutaneous injection and outpatient administration beyond Cycle 2 may be associated with improved QOL compared to other therapies, regardless of efficacy and safety.
Time to access matters: patient gains from faster CAR T-cell reimbursement in Europe.
Tan YZ, Breislin E, Woods M … +7 more , Madin-Warburton M, Gladwell D, Zang A, Vadgama S, Doble B, Wiesinger A, Sola-Morales O
J Med Econ · 2026 Dec · PMID 41705912 · Publisher ↗
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 potenti...
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.
Delta price cost-effectiveness analysis of PCV21 vs PCV20 use in adults aged ≥65 years in Switzerland.
Simuzingili M, Favre-Bulle A, Mutschler T … +3 more , Yi Z, Tsoumani E, Owusu-Edusei K
J Med Econ · 2026 Dec · PMID 41703735 · Publisher ↗
INTRODUCTION: This study evaluated the health and economic impacts of the 21-valent pneumococcal conjugate vaccine (PCV21) compared to the 20-valent pneumococcal conjugate vaccine (PCV20) in Swiss adults aged 65 years an...
INTRODUCTION: This study evaluated the health and economic impacts of the 21-valent pneumococcal conjugate vaccine (PCV21) compared to the 20-valent pneumococcal conjugate vaccine (PCV20) in Swiss adults aged 65 years and older using the delta-price approach. METHOD: A published state-transition Markov model was used to track health and economic outcomes of invasive pneumococcal disease (IPD), inpatient non-bacteremic pneumococcal pneumonia (NBPP), and post-meningitis sequelae (PMS). Estimated quality-adjusted life years (QALYs)and cost outcomes were discounted at 3%. All costs were presented in 2024 Swiss Franc (CHF). Price premiums were estimated as the difference between the prices of PCV21 and PCV20, and the maximum premiums at which PCV21 remains cost-saving and cost-effective (at a willingness-to-pay threshold of CHF 40,000) were reported. A scenario analysis was conducted to include a pneumococcal polysaccharide vaccine 23-valent (PPSV23) and 13-valent pneumococcal conjugate vaccine (PCV13) vaccine-experienced population. Sensitivity analysis identified the input categories that were most influential on the price premiums. RESULTS: Overall, PCV21 averted more cases of IPD, NBPP, PMS and associated deaths, and saved more direct costs than PCV20. Compared to PCV20, PCV21 was cost-saving until a price premium of CHF 25.10 and cost-effective up to a price premium of CHF 88.01. In the scenario analysis, PCV21 was cost-saving up to a price premium of CHF 25.38, and cost-effective up to a price premium of CHF 88.68. Vaccine effectiveness and disease costs were the most influential inputs on the price premiums. CONCLUSION: PCV21 provides greater health and economic benefits than PCV20 and is cost-effective over a range of price premiums.
Health and productivity benefits of anti-PD-(L)1 agents for early-stage cancer treatment in Hungary.
Ladino D, Patterson K, Várnai M … +3 more , Balogh É, Khurana V, Aguiar-Ibáñez R
J Med Econ · 2026 Dec · PMID 41703729 · Publisher ↗
AIM: Anti-PD-(L)1 agents, inhibitors of programmed cell death protein 1 (PD-1) or its ligand (PD-L1), are established therapies that improve cancer management as well as the disease and societal burden of specific metast...
AIM: Anti-PD-(L)1 agents, inhibitors of programmed cell death protein 1 (PD-1) or its ligand (PD-L1), are established therapies that improve cancer management as well as the disease and societal burden of specific metastatic and early-stage cancers. The aim of the study was to determine the impact of adopting anti-PD-(L)1 agents for the treatment of all eligible patients with early-stage cancers versus reserving anti-PD-(L)1 agents for patients with metastatic disease alone in Hungary. METHODS: This study evaluated two scenarios, one where anti-PD-(L)1 agents were used to treat all eligible early-stage disease case (ESD scenario) of melanoma (stage IIB-C and III), renal cell carcinoma (RCC), and triple-negative breast cancer (TNBC) versus a reference scenario where anti-PD-(L)1 agents were only used to treat metastatic disease cases in Hungary (2024-2033). A Markov-modeling approach estimated the health outcomes and productivity losses from each scenario from a societal perspective. Outcomes included recurrence-/event-/disease-free life-years, total life-years, quality-adjusted life-years (QALYs), productive years (patients and caregivers), recurrences/events, active treatments for metastatic disease, and deaths. The cumulative health and productivity impact of ESD treatment with anti-PD-(L)1 agents in Hungary was the difference in health and productivity outcomes between the ESD and reference scenarios for the time horizon of the model. RESULTS: ESD treatment with anti-PD-(L)1 agents was estimated to increase recurrence-/event-/disease-free life-years (+13.8%), total life-years (+3.7%), and QALYs (+4.7%), as well as productive work years for patients (+39.6%) and caregivers (+27.6%). Concurrently, there would be fewer recurrences/events (-31.0%), active treatments for metastatic disease (-34.0%), post-recurrence deaths (-30.3%), and total deaths (-23.1%). CONCLUSION: Investing in anti-PD-(L)1 agents for early-stage disease may not only increase the life expectancy and QALYs for patients in Hungary but also increase productive work years for both patients and caregivers in Hungary. In addition, it may also help to reduce metastatic disease treatments and cancer-related deaths.
The economic value of self-administered subcutaneous immunoglobulin G (SCIg) in Canada: a scoping review.
Cheung A, Manuel M, Mac S … +3 more , Fakhraei R, Giangregorio A, Betschel SD
J Med Econ · 2026 Dec · PMID 41701183 · Publisher ↗
BACKGROUND: Subcutaneous immunoglobulin (SCIg) is a treatment available in Canada for patients requiring antibody replacement. While standard-of-care involves in-hospital intravenous Ig (IVIg) treatment, SCIg allows for...
BACKGROUND: Subcutaneous immunoglobulin (SCIg) is a treatment available in Canada for patients requiring antibody replacement. While standard-of-care involves in-hospital intravenous Ig (IVIg) treatment, SCIg allows for home-based administration and fewer clinic visits, representing a common treatment choice. This study summarized the economic value of SCIg IVIg treatment in Canada. METHODS: A scoping review was conducted using Medline and Embase, supplemented by grey literature searches. Eligibility criteria were clinical studies and economic evaluations reporting cost and healthcare resource use (HCRU) outcomes of SCIg or IVIg treatment in Canada (any condition). Types and estimates of costs (Canadian dollars [CAN$]), cost-effectiveness, and HCRU were summarized. Estimates from economic models were inflated to 2025 CAN$ and standardized as cost savings per-patient per-year. RESULTS: From 824 database abstracts and 10 grey literature citations, 21 eligible articles were included; articles without Canadian economic data for IVIg or SCIg were excluded. Lower costs and HCRU were reported for SCIg IVIg (=7 studies), where reductions in total cost ranged from $1,795 to $5,386/patient/year (uninflated). Staffing time and lost productivity were also lower; SCIg required 45-57 fewer nursing hours/patient/year, and 51-82 fewer parental hours/patient/year. Lower costs with SCIg were driven by reduced infusion materials, staff time, transportation to clinics, and lost productivity. Across economic models, annual cost savings were substantial after switching patients from IVIg to SCIg; post-inflation estimates averaged from $1,947 to $7,152 per patient. In a cost-utility analysis, SCIg was less costly while providing greater quality-adjusted life-years than hospital-based IVIg, even when hospital/staff costs for IVIg were lowered by 50%. CONCLUSION: SCIg represents an important treatment option both for alleviating burden to the Canadian healthcare system and for patients. Substantial cost savings were estimated by switching from IVIg to SCIg. These findings provide evidence to support healthcare decision-making from an economic perspective.
Cost-effectiveness of the GAAD algorithm for hepatocellular carcinoma surveillance of patients with compensated cirrhosis: a model-based analysis using Italian real-world data.
Porta C, Pradelli L, Cirotto G … +7 more , Majorini MT, Garay OU, Calvaruso V, Fasano T, Napoli L, Foschi FG, Lampertico P
J Med Econ · 2026 Dec · PMID 41701165 · Publisher ↗
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...
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.
Indirect treatment comparison of DVRd plus DR maintenance (PERSEUS study) versus DVTd or VTd with or without lenalidomide maintenance in transplant-eligible patients with previously untreated multiple myeloma.
Dimopoulos MA, Boccadoro M, Einsele H … +20 more , Rodriguez-Otero P, Hulin C, Perrot A, Leleu X, Caillot D, Facon T, Mina R, Gay F, Broijl A, Hajek R, Spencer A, Schjesvold F, Zweegman S, Nair S, Ammann E, He J, Nobrega M, Rowe M, Sitthi-Amorn A, Sonneveld P
J Med Econ · 2026 Dec · PMID 41701105 · Publisher ↗
AIMS: To compare the effectiveness of induction and consolidation with daratumumab, bortezomib, lenalidomide, and dexamethasone (DVRd) plus maintenance therapy with daratumumab and lenalidomide (DR) with 2 treatment regi...
AIMS: To compare the effectiveness of induction and consolidation with daratumumab, bortezomib, lenalidomide, and dexamethasone (DVRd) plus maintenance therapy with daratumumab and lenalidomide (DR) with 2 treatment regimens that are widely used for transplant-eligible newly diagnosed multiple myeloma (TE NDMM): bortezomib, thalidomide, and dexamethasone (VTd) or daratumumab-VTd (DVTd) followed by observation (Obs) or lenalidomide maintenance. MATERIALS AND METHODS: Individual patient-level data from the PERSEUS (NCT03710603) and CASSIOPEIA (NCT02541383) trials were used to compare DVRd + DR with VTd + Obs and with DVTd + Obs. Inverse probability of treatment weighting was used to adjust for differences in baseline patient characteristics between the two trials; inverse probability of censoring weighting was used to adjust for the second randomization of CASSIOPEIA. Data from the CASSIOPEIA and Myeloma XI (EudraCT 2009-010956-93) trials were combined to model outcomes associated with lenalidomide (R) maintenance following induction with DVTd or VTd. RESULTS: DVRd + DR showed superior progression-free survival compared with DVTd + Obs (hazard ratio, 0.39 [95% CI = 0.26-0.59]), VTd + Obs (0.17 [95% CI = 0.12-0.25]), DVTd + R (0.62 [95% CI = 0.41-0.92]), and VTd + R (0.29 [95% CI = 0.18-0.43]). Sensitivity analyses showed results were consistent with the base case. LIMITATIONS: The indirect treatment comparison was unanchored due to a lack of common comparators and relied on external data from the Myeloma XI trial to model outcomes associated with DVTd or VTd followed by R maintenance. Despite best efforts to identify and adjust for important treatment effect modifiers, there is a potential for residual confounding. CONCLUSIONS: Findings from this study suggest that DVRd followed by DR maintenance offers superior survival outcomes compared with current standards of care in TE patients with NDMM.
Cost-effectiveness and budget impact of PFO closure: Cardioform vs Amplatzer and medical therapy for secondary stroke prevention in Australia.
Starmer G, Sharpe R, Wong A … +4 more , ElMarkaby H, Louwsma T, Camuglia B, Fox D
J Med Econ · 2026 Dec · PMID 41661066 · Publisher ↗
AIM: The objective of this study was to evaluate the value of three secondary stroke prevention strategies in Australia: the Cardioform and Amplatzer Patent Foramen Ovale (PFO) closure devices, and medical management. MA...
AIM: The objective of this study was to evaluate the value of three secondary stroke prevention strategies in Australia: the Cardioform and Amplatzer Patent Foramen Ovale (PFO) closure devices, and medical management. MATERIALS AND METHODS: An eight-state Markov model was employed to simulate a cohort of 1,000 patients with a history of PFO-associated stroke over a five-year time horizon. Treatment strategies included Cardioform, Amplatzer, and medical therapy alone. Effectiveness data were derived from the REDUCE and RESPECT trials, a matching-adjusted indirect comparison (MAIC), and prior cost-effectiveness studies. Costs, presented from an Australian healthcare perspective and expressed in 2023 AUD, were used to calculate quality-adjusted life-years (QALYs), strokes prevented, the incremental cost-effectiveness ratio (ICER), and net monetary benefit (NMB). RESULTS: Compared to Amplatzer, treatment with Cardioform yielded cost savings to the Australian health care systems (NMB of AUD 3.7 million) and improved patient outcomes (yielded 26.48 additional QALYs and prevented 28 more recurrent strokes). Relative to medical therapy alone, Cardioform resulted in improved patient outcomes and was cost-effective, with an ICER of $11,784/QALY. Cardioform provides an NMB of AUD 14.3 million and yielded 374.5 additional QALYs beside preventing 67 more strokes compared to medical therapy alone. CONCLUSION: Cardioform appears more cost-effective in the prevention of secondary PFO-associated strokes, supporting its adoption in clinical practice.
Rationale and recommendations for improving early-stage oncology diagnosis, treatment, and access.
Aguiar-Ibáñez R, Goldschmidt D, Zhou ZY … +10 more , Eales J, Peters S, Cardoso F, Ciani O, Arunachalam A, Haiderali A, Roediger A, Black CM, Martinez E, Garrison LP
J Med Econ · 2026 Dec · PMID 41653456 · Publisher ↗
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 aggres...
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.
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