Journal Of Medical Economics[JOURNAL]
Sun
200 papers
Cost comparison of osimertinib plus platinum-pemetrexed versus amivantamab plus lazertinib for the first-line treatment of patients with locally advanced or metastatic epidermal growth factor receptor-mutated non-small cell lung cancer.
Lopes GL, Freeman H, Procter E … +5 more , Upton E, Karia P, Rattigan-Brown Y, Hearmon N, Nieva JJ
J Med Econ · 2026 Dec · PMID 41973054 · Publisher ↗
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 t...
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.
Safeguarding Quality in Health and Medical Science Information Today.
Ratzan SC, Ivic RK, Rabin KH … +13 more , Parker RM, Rubinelli S, Obregon R, Gostin LO, Baker MG, Thomas S, Nutbeam D, Kickbusch I, Payne JG, Ihekweazu V, McCaffery KJ, Street RL, Cooper SC
J Med Econ · 2026 Dec · PMID 41960918 · Publisher ↗
Abstract loading — click title to view on PubMed.
Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with transfusion-dependent -thalassemia in the United States.
Udeze C, Gargano M, Yang H … +5 more , Ogunsile FJ, Li N, Fang H, Jeyakumar S, Lopez A
J Med Econ · 2026 Dec · PMID 41955126 · Publisher ↗
AIMS: Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 with transfusion-dependent β-thalassemia (TDT). Standard of care (...
AIMS: Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 with transfusion-dependent β-thalassemia (TDT). Standard of care (SOC) for TDT includes regular red blood cell transfusions (RBCTs) and iron chelation therapy. This study estimated long-term clinical outcomes and cost-effectiveness of exa-cel vs. SOC among patients with TDT in the US. METHODS: A Markov model was developed to compare the expected lifetime costs and clinical outcomes of patients with TDT treated with exa-cel compared to SOC from the US payer and societal perspectives. The model structure is based on transfusion status, which impacts iron levels and risk of developing TDT-related complications. The model incorporated data from the phase 3 pivotal CLIMB THAL-111 trial and other inputs from literature. Model outcomes included life years (LYs) and quality-adjusted LYs (QALYs) as well as number of RBCTs and proportion of patients developing complications over a lifetime; costs and incremental cost-effectiveness ratios (ICERs) were also estimated. Costs and outcomes were discounted at 3% annually. RESULTS: In the eligible patient population (average age at baseline: 21 years), exa-cel was projected to improve survival by 17.6 years (mean age of death, exa-cel: 64.9 years vs. SOC: 47.3 years), reduce the number of RBCTs received by 425 (exa-cel: 26 vs. SOC: 451), and lower the proportion of patients developing TDT-related complications. Exa-cel was associated with increased discounted costs compared to SOC (exa-cel: $2.9 M vs. SOC: $1.8 M). The ICER per discounted QALY for exa-cel versus SOC was $114,100 from the payer perspective and $55,400 from the societal perspective. CONCLUSIONS: Compared to SOC, exa-cel was projected to considerably reduce the number of RBCTs and TDT-related complications, improve survival, and reduce disease-related costs. Exa-cel is projected to be a cost-effective treatment option for patients with TDT in the US.
Plain language summary looking at the impact of drug management strategies on the incidence and cost of clinical events in patients with non-valvular atrial fibrillation receiving direct oral anticoagulants in the USA.
Subash R, Deeba S, Salter C … +3 more , Dickerson C, Ogunyannwo T, Stawowczyk E
J Med Econ · 2026 Dec · PMID 41955092 · Publisher ↗
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Changes in drug price reduction trends during Japan's transition to an annualized revision environment: a comparative analysis using open data from the national claims database.
Tsuda K, Suzuki A, Nakata Y
J Med Econ · 2026 Dec · PMID 41949918 · Publisher ↗
OBJECTIVE: To address the lack of evidence on the effects of Japan's transition to an annualized revision environment, this study quantified the net change in drug price reduction rates between the biennial and annualize...
OBJECTIVE: To address the lack of evidence on the effects of Japan's transition to an annualized revision environment, this study quantified the net change in drug price reduction rates between the biennial and annualized revision periods, encompassing the introduction of off-year revisions and policy measures, and examined structural heterogeneity across drug types, dosage forms, and therapeutic categories. METHODS: Longitudinal analysis was conducted using the National Database of Health Insurance Claims and Specific Health Checkups (NDB) Open Data. The study period was divided into Pre-period (2016-2019) and Post-period (2020-2023). Linear mixed-effects models (LMMs) were employed to estimate price change rates, adjusting for drug type, dosage form, therapeutic sub-categories, and log-transformed baseline price and prescription volume, with unique drug identifiers included as a random intercept. RESULTS: The analysis included 4,448 drugs from 85 therapeutic sub-categories. Overall, the mean drug price reduction rate significantly accelerated in the Post-period, declining by an additional 4.46 percentage points (pp) compared with the Pre-period ( < 0.001). After covariate adjustment, the primary LMM estimate indicated a net acceleration of -6.65 pp (95% CI: -7.24 to -6.06; < 0.001). The model also revealed pronounced bipolarization: oral drugs showed substantial acceleration in price reduction, with least squares (LS) means decreasing from -6.5% to -17.0% for brand-name drugs and from -15.6% to -27.3% for generics ( < 0.001 for both). Injection products showed divergent trajectories in the primary analysis. Severe price erosion occurred in antitumor agents (-13.3 pp), whereas high price resilience persisted in categories such as anticoagulants and hemodialysis solutions. CONCLUSIONS: Japan's annualized revision environment (2020-2023) was associated with an acceleration in pharmaceutical cost containment, but also with bipolarization across product segments. Marked price erosion in oral drugs, together with pronounced therapeutic heterogeneity, suggests that uniform reductions should be reconsidered to safeguard access and continuity of care.
Cost-effectiveness of pembrolizumab plus chemotherapy for metastatic non-small cell lung cancer: a head-to-head trial vs. real-world comparison.
Teppala S, Koo J, Clarke S … +1 more , Lu CY
J Med Econ · 2026 Dec · PMID 41949826 · Publisher ↗
OBJECTIVES: Real-world evidence (RWE) is increasingly used in health technology assessment (HTA) to address uncertainties surrounding the generalizability of randomized clinical trial (RCT) data. Pembrolizumab plus plati...
OBJECTIVES: Real-world evidence (RWE) is increasingly used in health technology assessment (HTA) to address uncertainties surrounding the generalizability of randomized clinical trial (RCT) data. Pembrolizumab plus platinum-based chemotherapy improves survival in metastatic, non-small cell cancer (mNSCLC); however, existing economic evaluations primarily rely on RCT inputs. This study aimed to provide a within-model comparison of cost-effectiveness estimates derived from RCT and population-based RWE for pembrolizumab plus platinum therapy versus platinum therapy alone. METHODS: A cost-utility analysis was conducted using a semi-Markov approach over a 3-year time horizon. Survival parameters were sourced separately from RCT and RWE datasets. Costs were estimated from the Australian payer perspective. Decision uncertainty was examined through one-way and probabilistic sensitivity analyses. RESULTS: Using RCT-based data, pembrolizumab plus platinum therapy increased total costs by AU$97,661 and provided a gain of 0.29 quality-adjusted life years (QALYs) compared with platinum therapy alone, resulting in an incremental cost-effectiveness ratio (ICER) of AU$336,196/QALY. RWE-based inputs, led to incremental costs of AU$79,471, a gain of 0.16 QALYs, and an ICER of AU$496,007/QALY. Probabilistic analyses indicated a < 5% probability of cost-effectiveness at willingness-to-pay (WTP) thresholds of AU$75,000/QALY for both evidence sources. CONCLUSION: Pembrolizumab plus platinum therapy is unlikely to be cost-effective for mNSCLC. The higher ICER with RWE compared to RCT data suggests that trial-based outcomes may overestimate effectiveness in routine practice. These findings highlight the importance of incorporating RWE into post-approval reassessment for high-cost oncology therapies.
Cost-efficiency modeling of conversion to biosimilar rituximab-based R-CHOP in diffuse large B-cell lymphoma in medicare.
Roth JA, Kratochvil D, Perkins K … +1 more , Zhang W
J Med Econ · 2026 Dec · PMID 41941177 · Publisher ↗
BACKGROUND: Rituximab-pvvr (Ruxience), -abbs (Truxima), and -arrx (Riabni) are biologic therapies approved by the United States Food and Drug Administration (FDA) as biosimilars to originator rituximab (Rituxan). Each is...
BACKGROUND: Rituximab-pvvr (Ruxience), -abbs (Truxima), and -arrx (Riabni) are biologic therapies approved by the United States Food and Drug Administration (FDA) as biosimilars to originator rituximab (Rituxan). Each is approved for the treatment of diffuse large B-cell lymphoma (DLBCL) in combination with cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP). The objective of this study is to explore the cost-efficiency and budget-neutral expanded access of conversion to first-line biosimilar rituximab-based R-CHOP treatment in DLBCL in Medicare. METHODS: We developed a Medicare perspective simulation model of patients treated for DLBCL to estimate the potential cost-savings from converting originator rituximab to rituximab-pvvr, -abbs, -arrx, or a weighted average of these biosimilars. The target patient population was estimated using Medicare enrollment data and Surveillance, Epidemiology, and End Results Program (SEER) Cancer incidence rates in patients age ≥65 years. Costs were derived from 2025 Average Sales Price (ASP) files. Results include per-patient per-month (PPPM) cost savings (vs. originator), total monthly cost savings in the Medicare cohort, and number needed to convert (NNC) to biosimilar to fund additional 100 patient-months of R-CHOP treatment. RESULTS: In the base case strategy with 100% biosimilar conversion and an equally weighted proportion converting to each rituximab biosimilar, PPPM savings were $4,638, full cohort ( = 4,772) monthly savings were $22.1 m, and NNC was 69. In analyses assessing 100% conversion to rituximab-pvvr, -arrx, and -abbs, the PPPM savings were $5,036, $4,450, and $4,429; full cohort monthly savings were $24.0 m, $21.2 m, and $21.1 m; and NNC was 55, 76, and 77 respectively. CONCLUSION: In this first cost-efficiency and expanded access study of biosimilar rituximab in DLBCL in Medicare, we find that conversion to biosimilar-based R-CHOP can result in substantial cost savings relative to originator-based R-CHOP. These biosimilar-associated cost savings could be reinvested to treat thousands of additional DLBCL patients or fund other costs of care in Medicare, on a budget-neutral basis.
The economic burden of moderate-to-severe rheumatoid arthritis in Spain: the CIARA study.
Silva-Fernández L, Balsa Criado A, Sanmartí R … +16 more , Pérez Venegas JJ, López-González R, Castaño Sánchez M, Cáliz Cáliz R, Membrive Jiménez C, Marras Fernández-Cid C, Sánchez-Fernández SÁ, Gómez Castro S, Vegas-Revenga N, Sivera Mascaró F, Mena Vázquez N, Belmonte Á, Ruiz Díaz MÁ, Rejas-Gutiérrez J, De Lossada Juste A, Montoro Álvarez M
J Med Econ · 2026 Dec · PMID 41915412 · Publisher ↗
AIMS: The CIARA (Cost Impact Analysis in Rheumatoid Arthritis patients) study in Spain aimed to assess societal costs (€2022) associated with adult patients with moderate-to-severe rheumatoid arthritis (RA) starting adva...
AIMS: The CIARA (Cost Impact Analysis in Rheumatoid Arthritis patients) study in Spain aimed to assess societal costs (€2022) associated with adult patients with moderate-to-severe rheumatoid arthritis (RA) starting advanced therapies after failing conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) or a first biologics (bDMARD). Costs included those incurred by the Spanish National Health System, indirect costs due to productivity losses, and patient out-of-pocket expenses. Secondary objectives were to compare costs according to patients' disease activity and treatment subgroups, identify variables associated with costs, and conduct a cost-utility analysis (CUA) of switching strategies. METHODS: This prospective, non-interventional, multicenter cohort included adults with active RA (Disease Activity Score in 28 joints with erythrocyte sedimentation rate ≥3.2) starting a bDMARD or a targeted synthetic DMARD (tsDMARD) and followed for 12 months. Total and disaggregated mean costs per patient were estimated across three periods: 6 months before, and 0-to-6 months and 6-to-12 months post-switch. Costs for the 6-month pre-switch were compared with those for the 6-to-12 post-switch. RESULTS: A total of 118 patients (mean age: 54.9 years; 78% women) were included. Mean total cost per patient increased from €6,882 pre-switch to €9,927 at 6-12 months (+44.3%; < 0.001), primarily driven by pharmacological costs (+191%), while out-of-pocket expenses (-55%; = 0.001) and productivity losses (-25%; = 0.394) decreased. Switching from a csDMARD was dominant vs. switching from a first bDMARD (-€11,281/quality-adjusted life year [QALY] gained). Patients who achieved remission at 12 months had lower costs than those with moderate-high activity. The Work Productivity and Activity Impairment (WPAI) productivity impairment subscale and patient satisfaction with care were independently associated with total costs. LIMITATIONS: Observational design, limited power for subgroups, official-actual price gap, and a 1-year horizon. CONCLUSIONS: Switching to advanced therapies reduced out-of-pocket expenses and productivity losses. Although this reduction did not offset higher pharmaceutical spending, it may reflect clinical improvements.
Costs and benefits of whole-exome, whole-transcriptome sequencing versus 50-gene panels for genomic profiling in solid tumors.
Ortendahl JD, Carter GC, Zantema EM … +8 more , Hoag JR, Starodynov A, White KL, Basu GD, Tharayanil A, De La O JP, Hall DW, Baehner FL
J Med Econ · 2026 Dec · PMID 41915379 · Publisher ↗
AIMS: The rapid development of therapies linked to molecular biomarkers has increased the importance of next-generation sequencing (NGS)-based tumor profiling to guide treatment decisions. Technology has enabled more com...
AIMS: The rapid development of therapies linked to molecular biomarkers has increased the importance of next-generation sequencing (NGS)-based tumor profiling to guide treatment decisions. Technology has enabled more comprehensive clinical testing; however, the optimal economic approach deserves investigation. This study evaluated the impact of testing using whole-exome, whole-transcriptome sequencing (WES/WTS) versus 50-gene panel tests from a US payer perspective. MATERIALS AND METHODS: A previously published Microsoft Excel-based model was used to compare WES/WTS and four 50-gene panels for testing within triple-negative breast cancer (TNBC), colorectal cancer (CRC), non-small cell lung cancer (NSCLC), and castrate resistant prostate cancer (CRPC). Genomic alteration prevalence and test sensitivity were based on a previous analysis of WES/WTS results in clinical samples. Model inputs related to the patient population, costs, clinical trial uptake, and market share were based on published literature. Results included the number of patients directed to a different therapy and the per-member per-month (PMPM) impact to a health plan when increasing utilization of WES/WTS testing. RESULTS: In a one-million-member hypothetical plan, 858 patients were eligible for tumor profiling. Across the four 50-gene panel tests, the number of patients switching treatment when using WES/WTS testing was 1-2 (TNBC), 4-6 (CRC), 1-6 (NSCLC), and 3-5 (CRPC). PMPM cost differences when replacing use of 50-gene panels with WES/WTS testing ranged from a cost-savings of $0.0517 in NSCLC to a $0.0268 increase in CRPC. Per-patient costs when using WES/WTS testing were driven by medical and pharmacy costs, with testing representing only 1.1-2.1% of total costs. LIMITATIONS: Limitations include simplifications required in modeling and exclusion of recently approved therapeutic options due to the quickly evolving landscape. CONCLUSIONS: WES/WTS testing resulted in more patients directed to targeted therapies with a minimal budget impact and should be considered in clinical decision-making to improve patient outcomes.
Global burden of disease due to young-onset dementia and the forecast for 2050: update from global burden of disease study 2021.
Park Y, Jeong H, Kim EJ … +3 more , Park S, Lee M, Jakovljevic M
J Med Econ · 2026 Dec · PMID 41894477 · Publisher ↗
OBJECTIVE: The prevalence of young-onset dementia (YOD) is increasing worldwide, leading to greater economic and social burden, necessitating strategic management and prevention. MATERIALS AND METHODS: Using GBD 2021 dat...
OBJECTIVE: The prevalence of young-onset dementia (YOD) is increasing worldwide, leading to greater economic and social burden, necessitating strategic management and prevention. MATERIALS AND METHODS: Using GBD 2021 data, disability-adjusted life years (DALYs) rates were analyzed by age, sex, and risk factors across five age groups. ARIMA and Bayesian models were applied to predict disease burden through 2050. RESULTS: From 1990 to 2021, disease burden increased in both sexes aged ≥55 years, with the greatest rise in the 55-59 group. DALYs rates were consistently higher in females, peaking in the 60-64 group. High fasting plasma glucose was the leading risk factor. Model performance varied by sex and age; applying the best-fitting models indicated a continued increase in burden, particularly among females. CONCLUSIONS: YOD burden has risen over time and is associated with modifiable factors such as high blood glucose and body mass index. The increasing trend is expected to persist, highlighting the need for effective management strategies to reduce future socioeconomic impact.
Cost-effectiveness, public health impact, and budget impact of receipt of Pfizer-BioNTech COVID-19 vaccine, LP.8.1-adapted, 2025/2026 formula among adults aged 18 years and older at high risk for severe outcomes from COVID-19 in the United States.
Yehoshua A, Di Fusco M, Rudolph AE … +4 more , Thoburn E, Lopez SMC, Dodd J, Yarnoff B
J Med Econ · 2026 Dec · PMID 41894410 · Publisher ↗
OBJECTIVES: To assess the cost-effectiveness, public health impact, and budget impact of receiving the LP.8.1-adapted Pfizer-BioNTech COVID-19 Vaccine among United States adults aged ≥ 18 years. METHODS: A previously pub...
OBJECTIVES: To assess the cost-effectiveness, public health impact, and budget impact of receiving the LP.8.1-adapted Pfizer-BioNTech COVID-19 Vaccine among United States adults aged ≥ 18 years. METHODS: A previously published economic model was adapted to compare receiving the LP.8.1-adapted Pfizer-BioNTech COVID-19 Vaccine (2025/2026 formula) versus not in adults aged 18-64 years at high risk of severe COVID-19 and all adults aged ≥ 65 years. Age-specific epidemiological inputs were derived from public health surveillance data. Clinical, cost, and vaccine effectiveness parameters were informed by published literature. The budget impact analysis was based on a hypothetical 1-million-member plan and used a payer perspective. RESULTS: Without vaccination, the model projected 41.5 million new symptomatic cases, 43,681 deaths, 338,252 hospitalizations, $80 billion in total costs, and 1.99 million QALYs lost among adults aged ≥18 years, with the greatest health burden observed among adults aged ≥65 years (73% of hospitalizations and 83% of deaths) and the greatest economic burden in adults aged 18 to 64 years at high risk of severe outcomes (62% of total costs). Compared to no vaccination, vaccination with the LP.8.1-adapted Pfizer-BioNTech COVID-19 vaccine (2025/2026 formula) in the populations aged 18 to 64 years and ≥18 years, respectively was projected to prevent 212,096 and 620,333 cases, 137 and 1,867 deaths, and 1,501 and 12,677 hospitalizations, resulting in incremental costs of $1.1 billion and $567 million, 2,411 and 15,430 LYs gained, 70,493 and 181,137 QALYs gained, ICERs of $16,238 and $3,137 from the societal perspective and $34,022 and $8,059 from the payer perspective. In the budget impact analysis from the payer perspective, vaccination was estimated to result in a modest budget increase. CONCLUSIONS: Vaccinating adults at high risk of severe COVID-19 with the LP.8.1-adapted Pfizer BioNTech COVID-19 Vaccine is projected to be a cost-saving measure from the societal perspective that could reduce the public health and economic burden of COVID-19.
Trends in industry-sponsored clinical trial activity since passage of the Inflation Reduction Act.
Long G
J Med Econ · 2026 Dec · PMID 41880583 · Publisher ↗
BACKGROUND AND OBJECTIVE: By shortening the time after U.S. drug approval when substantial revenue reductions typically occur, the Inflation Reduction Act's (IRA) Drug Price Negotiation Program (DPNP) alters investment i...
BACKGROUND AND OBJECTIVE: By shortening the time after U.S. drug approval when substantial revenue reductions typically occur, the Inflation Reduction Act's (IRA) Drug Price Negotiation Program (DPNP) alters investment incentives. Particular concerns include disincentives for post-approval clinical testing of new indications (begun after U.S. drug approval), and investment in small molecules relative to biologics. Post-approval development faces shortened times to earn returns; small molecule drugs are eligible to be selected for the DPNP four years earlier than biologics. The purpose of this study is to investigate these potential disincentives by quantifying trends in clinical trial starts, before and after IRA passage, for biologics and small molecules, further segmenting trials into those begun prior to U.S. drug approval and those begun later, seeking additional indications (pre-approval and post-approval trials, respectively). METHODS: Combining data from Citeline Trialtrove and Pharmaprojects databases, a linear regression model estimated separately for trials beginning pre- and post-U.S. drug approval yielded best-fit estimates of trends and percentage changes in average monthly Phase I-III industry-sponsored interventional clinical trial starts for the periods before (January 2010-July 2022) and after (August 2022-December 2024) passage of the IRA, in biologic and small molecule drugs. RESULTS: During the first twenty-nine months after IRA passage, the estimated number of small molecule drug clinical trials started monthly dropped by 25.2% (95% CI: -39.0% to -7.1%) and 29.5% (95% CI: -42.4% to -13.2%) for pre-approval trials for new drugs and post-approval trials, respectively. In comparison, for biologics, the model estimates non-significant decreases for pre-approval trials (-2.1%, 95% CI: -9.4% to +5.8%) and post-approval trials (-13.0%, 95% CI: -30.0% to +6.4%). CONCLUSIONS: While descriptive, this study offers early evidence of reductions in industry-sponsored new small molecule pre-approval and post-approval drug trials after IRA passage. These trends should be monitored as responses work their way through drug pipeline investment decisions.
Epidemiology and economic burden of medically attended influenza and influenza-like illness in Germany, 2016-2019.
Meyer AC, Witte J, Batram M … +7 more , Bartelt-Hofer J, Bangert M, Schild M, Marschall U, Greiner W, Schelling J, Damm O
J Med Econ · 2026 Dec · PMID 41880366 · Publisher ↗
AIMS: Older adults and individuals with certain underlying conditions are at elevated risk of severe influenza complications. This study quantifies the burden of influenza and influenza-like illness (ILI) in Germany focu...
AIMS: Older adults and individuals with certain underlying conditions are at elevated risk of severe influenza complications. This study quantifies the burden of influenza and influenza-like illness (ILI) in Germany focusing on these high-risk groups. METHODS: This cohort study is based on claims data of a large statutory health insurance fund. Influenza/ILI was identified through International Classification of Diseases version 10 (ICD-10) codes J09-J11. Among insured individuals, the incidence of medically attended influenza/ILI, hospitalizations, complications, and direct healthcare costs were estimated for three consecutive influenza seasons from 2016-2017 to 2018-2019. Healthcare resource use, complications, and costs attributable to influenza were estimated through comparison with a matched control group. Analyses were stratified by age and prevalence of underlying conditions. RESULTS: Approximately 7 million individuals were included in each season. Influenza/ILI incidence decreased with age, but hospitalizations were most common among older adults. One in five cases aged 80+ years was hospitalized. In all age groups, individuals with underlying conditions had a higher incidence of influenza/ILI and of complications than those without underlying conditions. Direct costs per influenza/ILI case ranged from €133.28 (2016-2017) to €218.99 (2018-2019) and were higher in older adults and in individuals with underlying conditions. LIMITATIONS: Underdiagnosis and misclassification of influenza in administrative claims may occur, particularly in the outpatient setting. Only selected complications were examined; exclusion of cardiovascular and neurological consequences likely results in an underestimation of the influenza/ILI burden. Indirect costs, e.g. through productivity losses are not considered. CONCLUSIONS: Influenza/ILI significantly impacts the German healthcare system, with older adults and individuals with underlying conditions contributing disproportionately to the observed burden. Risks of severe outcomes and direct costs are highest in older adults, particularly those aged 80+ years, while children with underlying conditions also contribute substantially to the observed burden. These groups are important targets for preventive interventions.
Model-based evaluation of colorectal cancer screening effectiveness: three rounds of multitarget stool DNA testing versus one colonoscopy.
Dore M, Ebner DW, Vahdat V … +4 more , Estes C, Ozbay AB, Foster V, Limburg PJ
J Med Econ · 2026 Dec · PMID 41879223 · Publisher ↗
BACKGROUND: Several colorectal cancer (CRC) screening modalities are guideline-recommended in the United States but differ in screening interval and real-world adherence. Accordingly, single-round test performance may no...
BACKGROUND: Several colorectal cancer (CRC) screening modalities are guideline-recommended in the United States but differ in screening interval and real-world adherence. Accordingly, single-round test performance may not reflect cumulative effectiveness over time. This study compared the 10-year longitudinal outcomes of two CRC screening strategies-triennial next-generation multitarget stool DNA testing (ng mt-sDNA) and decennial screening colonoscopy. METHODS: The validated, microsimulation-based Colorectal Cancer and Adenoma Incidence and Mortality (CRC-AIM) model was used to estimate 10-year cumulative outcomes for two guideline-recommended screening strategies: triennial ng mt-sDNA and decennial colonoscopy. Model inputs included test performance and real-world adherence. Outcomes included CRC and precancerous lesions detected, CRC mortality reductions, and life-years gained (LYG). Sensitivity analyses examined the effects of varying screening adherence and follow-up colonoscopy adherence. RESULTS: Over 10 years per 1,000 individuals offered screening, ng mt-sDNA detected 13% more precancerous lesions and 11% more CRC cases than colonoscopy, with a greater proportion of CRCs identified through screening rather than symptomatic detection. ng mt-sDNA achieved greater CRC mortality reduction (33% vs 20%) and 62% more life-years gained, with consistent findings across sensitivity analyses. CONCLUSIONS: With real-world adherence, screening with triennial ng mt-sDNA demonstrates superior cumulative effectiveness compared with decennial colonoscopy, driven by higher adherence and favorable longitudinal performance. These findings support expanded use of noninvasive stool-based screening to reduce CRC mortality and alleviate colonoscopy capacity constraints. Broader adoption of ng mt-sDNA may enhance population-level CRC prevention by increasing participation and improving early detection.
Estimating the public health and economic impact of increased COVID-19 annual vaccination coverage in the 60 years and older population in Spain.
Moreno Guillén S, Redondo Margüello E, Rodríguez García J … +6 more , Aceituno S, Prades M, Yang J, López Rodríguez Á, Molina C, López-Ibáñez de Aldecoa A
J Med Econ · 2026 Dec · PMID 41874460 · Publisher ↗
BACKGROUND: COVID-19 annual vaccination uptake in Spain remains suboptimal. This study aimed to estimate the clinical and economic impact of the 2023/2024 COVID-19 vaccination campaign in individuals aged ≥60 years (scen...
BACKGROUND: COVID-19 annual vaccination uptake in Spain remains suboptimal. This study aimed to estimate the clinical and economic impact of the 2023/2024 COVID-19 vaccination campaign in individuals aged ≥60 years (scenario A: coverage of 33.14% for ages 60-69, 53.15% for 70-79, and 65.32% for ≥80), and to compare it with a hypothetical scenario (scenario B) where coverage reaches the 75% target set by the Spanish Ministry of Health. METHODS: A combined Markov-decision tree model adapted to the Spanish context simulated the weekly progression of the target population through six health states over one year. Infected individuals entered a decision tree reflecting different care pathways (outpatient, hospital ward, ICU with/without invasive mechanical ventilation [IMV], or death), each associated with specific health outcomes and direct costs (€2024). Clinical and economic outcomes were compared between scenarios A and B. Sensitivity analyses explored incremental increases in coverage and age-specific impacts. The analysis was conducted from the National Healthcare System (NHS) perspective. RESULTS: Under scenario A, 378,970 symptomatic infections occurred, leading to 27,611 hospitalizations, 742 ICU admissions (47.3% requiring IMV), and 3,611 deaths. A total of 2,750 quality-adjusted life years (QALYs) were lost, and COVID-19-related care costs reached €240.4 million (85.7% from inpatient care). Scenario B, with 75% coverage, averted -19,409 symptomatic infections, 1,094 hospitalizations, 41 ICU admissions, and 129 deaths, 138 lost QALYs and total cost savings of about €10.5 million. Sensitivity analysis showed how the model is sensitive to sequential increases (10% by 10%) in vaccination rates and highlighted the importance of achieving high vaccination rates, especially in older age groups. CONCLUSIONS: This analysis reveals the significant impact that increasing annual COVID-19 vaccination coverage among the Spanish population over 60 could have in preventing new infections, reducing severe disease consequences, and generating considerable cost savings for the NHS.
Cost-effectiveness of treatment and care of patients with gastrointestinal inflammatory diseases: a systematic review.
Randlová K, Marešová P, Režný L … +5 more , Hruška J, Srdanović S, Guðmundsson GH, Másson E, Kuča K
J Med Econ · 2026 Dec · PMID 41861399 · Publisher ↗
AIMS: The aim of this study was to systematically review and descriptively synthesize published cost-effectiveness evidence for biologic versus non-biologic treatments as well as comparisons among different biologic ther...
AIMS: The aim of this study was to systematically review and descriptively synthesize published cost-effectiveness evidence for biologic versus non-biologic treatments as well as comparisons among different biologic therapies and treatment sequencing strategies, in inflammatory bowel disease, highlighting patterns and variability across disease types, treatment strategies, and study designs. MATERIALS AND METHODS: We conducted a systematic review following the PRISMA guidelines, searching the Web of Science and PubMed databases for studies published between 2013 and 2024. Studies comparing the cost-effectiveness of treatments for inflammatory bowel disease were included. RESULTS: Eighteen studies met the inclusion criteria, covering Crohn's disease and/or ulcerative colitis. Biologic therapies were generally associated with superior health outcomes compared with conventional treatments. Reported quality-adjusted life years (QALYs) ranged from 2.23 to 18.12 for biologic therapies and from 1.69 to 17.99 for non-biologic treatments. Although biologic therapies have higher costs, they are generally considered cost-effective. For Crohn's disease, infliximab was reported as a cost-effective biologic option, while findings for ulcerative colitis varied. Surgical intervention (colectomy) was identified as a cost-effective in selected clinical scenarios. CONCLUSION: Biologic therapies for inflammatory bowel disease are cost-effective, providing significant health benefits that offset higher costs. Substantial methodological heterogeneity and reliance on model-based economic evaluations limit direct comparability across studies. Future economic evaluations should focus on methodological consistency and transparency in assumptions to strengthen the interpretability of cost-effectiveness evidence.
Loss of productivity among commercially insured patients with pulmonary arterial hypertension in the United States.
Watzker A, Ferro C, Dieguez G … +4 more , Girdish C, Alsumali A, Lautsch D, El-Kersh K
J Med Econ · 2026 Dec · PMID 41861398 · Publisher ↗
BACKGROUND: Despite advances in treatment, pulmonary arterial hypertension (PAH) remains a progressive condition with the onset of disease often in working-aged adults, leading to substantial economic burden and increase...
BACKGROUND: Despite advances in treatment, pulmonary arterial hypertension (PAH) remains a progressive condition with the onset of disease often in working-aged adults, leading to substantial economic burden and increased healthcare resource utilization despite the rarity of the disease. The aim of this study was to estimate productivity loss for patients with PAH. METHODS: This was a retrospective cohort study of the Milliman Consolidated Health Cost Guidelines Source Data and the Merative MarketScan Commercial dataset between 01/01/2018 and 09/30/2023. Adult patients diagnosed and treated for PAH between 01/01/2019 and 08/31/2023 were identified and followed through the earliest of end of enrollment, or end of data. Outcomes were described as average workdays lost to receive healthcare services per-patient per-year (PPPY) and further assessed by healthcare setting. The cost of productivity loss was calculated based on the annual median household income. RESULTS: The study included 1,588 commercially insured patients (mean age 52 years, 63% female). Between 2019-2023, the mean number of workdays lost ranged between 21 and 23 PPPY, equating to over 8% of annual workdays. The cost of loss of productivity ranged from $8,828 to $9,599 PPPY (in 2023 dollars). Across all study years, the largest proportion of workdays lost were attributed to emergency room (ER)/observation, followed by office visits, and outpatient facilities, altogether comprising two-thirds of total workdays lost. Inpatient hospitalization-related workday loss was approximately 11% of total workdays lost. Over the study period, the largest change in workdays lost was observed for ER/observation. CONCLUSION: This study demonstrates substantial workdays lost due to health services utilization among patients with PAH, further adding to the overall economic burden of PAH. Quantifying productivity loss in PAH patients provides critical insight into the broader societal costs of the disease, supporting the inclusion of indirect costs in future economic evaluations and healthcare policy decisions.
Cost-effectiveness of pembrolizumab plus chemotherapy vs cemiplimab plus chemotherapy for first-line metastatic non-small cell lung cancer: a US payer perspective using a matching-adjusted indirect comparison.
Huang D, Zhang Y, Babel RA … +4 more , Fan T, Amonkar MM, Vandormael K, Insinga RP
J Med Econ · 2026 Dec · PMID 41861397 · Publisher ↗
AIM: Recent clinical guidelines recommend pembrolizumab plus chemotherapy and cemiplimab plus chemotherapy as key first-line treatment options for metastatic non-small cell lung cancer (mNSCLC). We evaluated the cost-eff...
AIM: Recent clinical guidelines recommend pembrolizumab plus chemotherapy and cemiplimab plus chemotherapy as key first-line treatment options for metastatic non-small cell lung cancer (mNSCLC). We evaluated the cost-effectiveness of these regimens from a US payer perspective. MATERIALS AND METHODS: A partitioned survival model with 1-week cycle, a 20-year horizon, and 3% annual discounting was developed. Clinical efficacy, safety, and utility inputs were derived from the KEYNOTE-189 (non-squamous histology), KEYNOTE-407 (squamous histology) and EMPOWER-Lung 3 Part 2 (both histologies). A matching-adjusted indirect comparison was conducted to compare efficacy outcomes between pembrolizumab plus chemotherapy and cemiplimab plus chemotherapy, with additional adjustment of overall survival to account for differences in subsequent immunotherapy use in control arms. Costs included drug acquisition and administration, adverse events, non-drug disease management, and terminal care. Outcomes included total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Scenario, deterministic, and probabilistic sensitivity analyses were performed. RESULTS: In non-squamous mNSCLC, pembrolizumab plus chemotherapy versus cemiplimab plus chemotherapy increased discounted costs by $43,131 ($335,194 vs $292,062) and QALYs by 0.71 (2.69 vs 1.98), yielding an ICER of $60,957/QALY. In squamous mNSCLC, incremental costs were $34,675 ($276,431 vs $241,756) for a 0.43 QALY gain (2.32 vs 1.89), yielding an ICER of $80,218/QALY. In the pooled mNSCLC population, incremental costs were $41,601 ($324,561 vs $282,960) with 0.66 additional QALYs (2.62 vs 1.97), and resulting ICER of $64,442/QALY. The model is most sensitive to the OS hazard ratio between regimens. Probabilistic sensitivity analysis suggests a high probability that pembrolizumab plus chemotherapy is cost-effective at commonly cited US willingness-to-pay (WTP) thresholds ($100,000/QALY and $150,000/QALY) considering uncertainties. LIMITATIONS AND CONCLUSIONS: After MAIC and subsequent immunotherapy adjustments, pembrolizumab plus chemotherapy showed improved overall survival and progression-free survival versus cemiplimab plus chemotherapy in both non-squamous and squamous metastatic NSCLC at modest incremental cost.
Real-world healthcare resource utilization and clinical outcomes among patients with relapsed/refractory multiple myeloma receiving ciltacabtagene autoleucel after four or more prior lines of therapy in inpatient versus outpatient settings.
Janakiram M, Fan L, Ghosh S … +8 more , Alegria V, Perciavalle M, Emond B, Maitland J, Bixby T, Nagar SP, Qureshi ZP, Dima D
J Med Econ · 2026 Dec · PMID 41848013 · Publisher ↗
BACKGROUND: Ciltacabtagene autoleucel (cilta-cel) has demonstrated remarkable efficacy in relapsed or refractory multiple myeloma (RRMM). Outpatient (OP) administration of cilta-cel is increasingly used to improve access...
BACKGROUND: Ciltacabtagene autoleucel (cilta-cel) has demonstrated remarkable efficacy in relapsed or refractory multiple myeloma (RRMM). Outpatient (OP) administration of cilta-cel is increasingly used to improve access and reduce healthcare resource utilization (HCRU) compared to inpatient (IP) administration. We compared all-cause HCRU and clinical outcomes of IP versus OP administration of cilta-cel in patients with RRMM after ≥4 prior lines of therapy (LOT) in clinical practice. METHODS: We identified adults receiving cilta-cel between 02/28/2022 and 06/30/2024 after ≥4 prior LOT using Komodo Research Database claims data and classified patients into cohorts by setting of administration (IP/OP). All-cause HCRU, treatment-free interval (TFI), overall survival (OS), and clinical events (e.g. cytokine release syndrome [CRS], immune effector cell-associated neurotoxicity syndrome [ICANS]) were assessed. Outcomes were compared using multivariate regression and reported as incidence rate ratios (IRR) with 95% confidence intervals (CI). RESULTS: Among 242 patients, 148 (61.2%) received cilta-cel in IP and 94 (38.8%) in OP. Baseline characteristics were comparable between cohorts. Of patients in the OP cohort, 31.9% did not require an IP admission within 3 months post-infusion. During this period, the OP cohort had significantly fewer IP days per-patient-per-month (2.4 vs. 6.6; IRR [95% CI]: 0.37 [0.28; 0.48], < 0.001) and more OP days (8.5 vs 5.4; IRR [95% CI]: 1.43 [1.26; 1.63], < 0.001) than the IP cohort. From the fourth month post-infusion, no significant differences in HCRU were observed. Rates of CRS and ICANS, and long-term outcomes including 6 and 12 month TFI and OS were similar. CONCLUSION: OP administration of cilta-cel yielded similar effectiveness and safety outcomes relative to IP administration, while significantly reducing IP resource use. These findings support the feasibility of OP administration of cilta-cel in treating RRMM patients and its potential to reduce the burden on the healthcare system.
The full value of mRNA seasonal influenza and endemic-stage COVID-19 combination vaccines: a taxonomy.
Sevilla JP, Knee JS, Burnes D … +5 more , Meier G, Yang J, Di Fusco M, Hu T, Bloom DE
J Med Econ · 2026 Dec · PMID 41843918 · Publisher ↗
AIMS: Seasonal influenza and COVID-19 pose significant ongoing threats to global health. Vaccination remains central to their prevention. Messenger RNA combination influenza and COVID-19 vaccines (mRNA combo vaccines) ar...
AIMS: Seasonal influenza and COVID-19 pose significant ongoing threats to global health. Vaccination remains central to their prevention. Messenger RNA combination influenza and COVID-19 vaccines (mRNA combo vaccines) are in development. Payers will soon need to make value-for-money (VfM) assessments and coverage decisions regarding these vaccines. Value taxonomies play an important role in VfM assessments and coverage decisions. However, no taxonomy exists that captures the full value of mRNA combo vaccines. We aimed to construct a taxonomy of the full value, from a societal perspective, of mRNA combo vaccines in working-age (18-64 years) and older adults (65+ years). METHODS: We (1) performed a targeted literature review (TLR) of existing value taxonomies and value attributes of COVID-19, influenza, other mRNA, and other combination vaccines; and (2) synthesized the value elements found in the TLR into a comprehensive taxonomy specific to mRNA combo vaccines. RESULTS: Of 1851 identified studies, 57 contained relevant value elements. We constructed a taxonomy distinguishing narrow health-related from broader societal values, and traditional from novel values. Value elements in the taxonomy included improved health and reduced treatment costs; improved productivity; improved strain selection, raising vaccine efficacy; greater compliance with vaccine schedules, increasing uptake; improved patient and caregiver health and reduced treatment costs from such greater efficacy and uptake; reduced adverse events, anxiety and vaccination costs from reduced doses; process utilities from increased convenience; higher patient and provider acceptability; increased equity; and health-related R&D spillovers. LIMITATIONS: The TLR was non-systematic. We do not address potential redundancies or the relative importance of different values. CONCLUSIONS: Many value elements in the taxonomy are traditional narrow values and fit within a health payer perspective, but the taxonomy also captures broader societal values. This taxonomy can support more comprehensive valuations of mRNA combo vaccines in national vaccine recommendation and funding decisions.
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