Journal Of Medical Economics[JOURNAL]
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200 papers
Health state utilities associated with obstructive sleep apnea: preferences of patients with OSA and obesity in the UK and the US.
Stewart KD, Matza LS, Dunn JP … +4 more , Neff LM, Niemeyer A, Boye KS, Shinde S
J Med Econ · 2025 Dec · PMID 41089051 · Publisher ↗
INTRODUCTION: Obstructive sleep apnea (OSA) is characterized by repeated episodes of airway obstruction during sleep, resulting in intermittent hypoxia, disrupted sleep, and reduced quality of life. Obesity management me...
INTRODUCTION: Obstructive sleep apnea (OSA) is characterized by repeated episodes of airway obstruction during sleep, resulting in intermittent hypoxia, disrupted sleep, and reduced quality of life. Obesity management medications may provide new options for patients with OSA and obesity. Health state utilities will be needed as inputs in economic modeling conducted to inform decision-making about these medications for treatment of OSA. The purpose of this study was to estimate utilities associated with OSA severity based on preferences of individuals with OSA and obesity. METHODS: Participants with OSA and obesity (BMI ≥ 30) in the UK and US valued health state vignettes in time trade-off interviews. Vignettes described four levels of OSA severity defined by apnea-hypopnea index: remission (<5), mild (5-14.9), moderate (15-29.9), and severe (≥30). Participants were instructed to imagine having the option to use non-pharmacologic treatment for OSA when valuing the health states. Utilities were first estimated for OSA with these treatment options. Then, for health states where participants imagined using positive airway pressure or another device, they were asked to value the vignettes a second time without this treatment to estimate utilities associated with symptoms of OSA at each severity level. RESULTS: A total of 208 participants (UK = 105; US = 103) completed interviews (50.0% male; mean age = 53.6y). Mean (SD) utilities ranged from 0.93 (0.08) for mild OSA to 0.85 (0.15) for severe OSA when considering health states with the option to use non-pharmacologic treatment. When participants imagined living with the symptoms of OSA without the non-pharmacologic treatment option, mean utilities ranged from 0.89 (0.13) for mild OSA to 0.68 (0.27) for severe OSA. DISCUSSION: Results provide insight into preferences associated with OSA among people with OSA and obesity. The health state utilities estimated in this study may be useful in cost-effectiveness models evaluating treatments for OSA in individuals with obesity.
Foslevodopa/foscarbidopa (LDp/CDp) in advanced Parkinson's disease (aPD): demonstration of savings from a societal perspective in the UK.
Chaudhuri KR, Bergmann L, Belsey J … +2 more , Boodhna T, Leoncini E
J Med Econ · 2025 Dec · PMID 41054384 · Publisher ↗
AIMS: In advanced Parkinson's disease (aPD), adequate 24-hour control of OFF-time may not be achievable using oral/transdermal therapies. Clinical trials of foslevodopa/foscarbidopa (LDp/CDP) demonstrate meaningful reduc...
AIMS: In advanced Parkinson's disease (aPD), adequate 24-hour control of OFF-time may not be achievable using oral/transdermal therapies. Clinical trials of foslevodopa/foscarbidopa (LDp/CDP) demonstrate meaningful reductions in OFF-time and OFF-related sleep disturbance in aPD. Previous analyses have only considered direct medical costs: this analysis considers a broader societal perspective (direct non-medical costs, informal care, loss of earnings, productivity, and tax). METHODS: Inputs for the societal impact model were taken from a cost-utility model comparing LDp/CDp with best medical treatment (BMT), accepted by the UK National Institute of Health and Care Excellence (NICE). Quintiles of normalized OFF-time across a 16-hour waking day in each treatment group were applied to literature-based estimates for direct medical, non-medical, and indirect costs. The resulting state-specific cost estimates were applied to the modelled aPD patient population. RESULTS: The model estimates the potential UK population for LDp/CDp at 17,505. Continuous 24-hour delivery of LDp/CDp results in greater time spent in OFF-time states 0-1 (0-4 h of OFF-time/16-hour waking day) vs BMT alone. Net costs in year 1, if all eligible patients receive LDp/CDp, are £45.5 M. Cumulative net savings thereafter are £47.0 M by year 2, rising to £166.1 M by year 3, £261.9 M by year 4 and £312.2 M by year 5. Results are robust in scenario analyses (excluding costs of sleep disturbance, earnings loss, productivity, and tax loss). LIMITATIONS: A NICE-accepted model was used as the economic modelling basis for the societal impact model. However, much of the data was derived from Adelphi datasets, with the potential for inconsistent definitions. CONCLUSION: When considered from a societal perspective, the use of LDp/CDp in aPD patients inadequately controlled on oral therapy, is associated with a reduction in medium-term healthcare and societal costs vs BMT.
Cost-effectiveness of enfortumab vedotin plus pembrolizumab as a first-line treatment of locally advanced or metastatic urothelial carcinoma in the United States.
Meng Y, Zhang S, Aout M … +7 more , Babcock A, Li H, Lai Y, Brand-Wiita S, Notinger S, Bavle A, Mamtani R
J Med Econ · 2025 Dec · PMID 41039930 · Publisher ↗
OBJECTIVE: Enfortumab vedotin plus pembrolizumab (EV+Pem) was recently approved in the United States as the new first-line standard of care for locally advanced or metastatic urothelial carcinoma (la/mUC). We assessed th...
OBJECTIVE: Enfortumab vedotin plus pembrolizumab (EV+Pem) was recently approved in the United States as the new first-line standard of care for locally advanced or metastatic urothelial carcinoma (la/mUC). We assessed the cost-effectiveness of EV+Pem as first-line treatment for la/mUC from a US third-party payer perspective, using the most recent database lock (data cutoff: 8 August 2024) from the Phase III EV302/KEYNOTE-A39 study (NCT04223856). METHODS: A partitioned survival model assessed the cost-effectiveness of EV+Pem versus gemcitabine and cisplatin/carboplatin followed by maintenance avelumab if not progressed (GP/GC ± Ave) in la/mUC patients. Standard and flexible spline parametric models were fitted to patient-level overall survival (OS) and progression-free survival (PFS) data from EV-302. Treatment discontinuation, utilities, and other clinical inputs were also based on EV-302. Cost inputs were sourced from literature or public databases. A cisplatin-eligible (Cis-E) subpopulation analysis compared EV+Pem versus nivolumab plus gemcitabine and cisplatin (Nivo + GP) and other platinum-based chemotherapies. A network meta-analysis based on the EV-302 and CheckMate-901 studies informed the OS and PFS hazard ratios for Nivo + GP versus EV+Pem. RESULTS: For the overall population, EV+Pem was projected to extend 2.58 life-years (LYs), or 1.76 quality-adjusted life-years (QALYs), versus GP/GC ± Ave, with an incremental cost-effectiveness ratio (ICER) of $180,631/QALY. Probabilistic sensitivity analyses (PSA) showed that EV+Pem had a 77% probability of being more cost-effective versus GP/GC ± Ave at a willingness-to-pay (WTP) threshold of $200,000/QALY. For the Cis-E subpopulation, EV+Pem was projected to extend 2.37 LYs (1.45 QALYs) versus Nivo + GP, with an ICER of $136,475/QALY. The PSA showed that EV+Pem had a 59% probability of being most cost-effective among all the treatment regimens considered, including platinum-based chemotherapies, at a WTP threshold of $200,000/QALY. CONCLUSIONS: EV+Pem is cost-effective compared to platinum-based regimens for the treatment of previously untreated la/mUC from a US third party payer perspective at a willingness-to-pay threshold of $200,000/QALY.
A budget impact analysis of the Dario Diabetes Solution for type 2 diabetes mellitus in a US managed care population.
Wilson L, Martin C, Tripathi S … +4 more , Dixit H, Miller J, Wang R, Lee WC
J Med Econ · 2025 Dec · PMID 41024731 · Publisher ↗
AIMS: To estimate the potential budget impact following adoption of the Dario Diabetes Solution (DDS), a combined all-in-one smart glucose monitor with cloud-based app integration, in combination with standard of care (S...
AIMS: To estimate the potential budget impact following adoption of the Dario Diabetes Solution (DDS), a combined all-in-one smart glucose monitor with cloud-based app integration, in combination with standard of care (SoC) for patients with type 2 diabetes mellitus (T2D) versus SoC alone. METHODS: Applying the findings of the retrospective cohort from real world data (RWD) studies of T2D patients' clinical outcomes and healthcare r251esource utilization (HCRU), a population-based budget impact model was developed. Budget impact was estimated with disaggregated costs calculated in categories, including medication, inpatient (IP) and emergency room (ER), office visit, and all-cause total costs. One-way sensitivity analysis (OWSA) was conducted to quantify the uncertainty in the model input parameters. Eight unique scenarios were analyzed, including the Medicare perspective, a two-year time horizon, and glycemic control. RESULTS: Analysis of the base case (1-year, commercial perspective, inclusion of device, application, and costs pertaining to consumable, medication, and health care resource utilization) resulted in net cost savings of $9,652,498 in a plan population of 1 million. Net budget impact for scenarios ranged from savings of $39,216,721, for the Medicare-only perspective instead of commercial, to $251,554 when restricting the population to patients who moved from poor-to-good glycemic control (HbA1C ≥ 8% mg/dl to < 8%). CONCLUSIONS: Despite cost estimation using remittance data and a relatively short time horizon (one year), our budget impact analysis demonstrated substantial cost savings with the introduction of DDS, driven by reduced HCRU for patients using DDS, who displayed improved adherence and glycemic control.
Frequency, duration, and cost of pulmonary exacerbations among patients with bronchiectasis.
Mohanty M, Leiras C, Sun H … +4 more , Rau R, Fastenau J, Feliciano J, Devarajan SR
J Med Econ · 2025 Dec · PMID 41017477 · Publisher ↗
BACKGROUND: In administrative claims database studies of bronchiectasis, pulmonary exacerbations are usually defined using a fixed period for their start and end, which prevents assessment of exacerbation duration and th...
BACKGROUND: In administrative claims database studies of bronchiectasis, pulmonary exacerbations are usually defined using a fixed period for their start and end, which prevents assessment of exacerbation duration and thereby limits assessment of exacerbation characteristics. Here, we applied a novel cost-based algorithm to characterize exacerbations. METHODS: This cohort study used the Merative MarketScan Commercial Claims and Encounters database, 1-Jan-2016 to 31-Dec-2022. Patients ≥18 years with bronchiectasis (≥2 outpatient or ≥1 inpatient claim with bronchiectasis; no cystic fibrosis) had 12 months of continuous enrollment before (baseline) and ≥12 months after (follow-up) index (first bronchiectasis claim). Cost-based exacerbations were identified by compound score of week with highest percentage all-cause cost increase during follow-up compared with baseline weekly maintenance all-cause cost, and week with highest absolute weekly cost during follow-up. Exacerbation duration was the period with significantly higher weekly cost difference during follow-up than mean baseline weekly cost. Cost-based exacerbations were compared with exacerbations identified using a traditional claims-based definition. RESULTS: Of 9,005 patients with bronchiectasis, 6,033 had 49,750 cost-based exacerbations during 2.5 years median follow-up. Mean (SD) cost-based exacerbation duration was 3.4 (8.6) weeks (median [Q1, Q3] 1 [1, 3] weeks). During follow-up, 82.8% patients had ≥3 cost-based exacerbations, and 67.5% patients needed hospitalization/intravenous antibiotic treatment for an exacerbation. Mean respiratory costs were higher for the first cost-based exacerbation ($7,738) than the second ($5,429). Annual respiratory costs were $14,116 for patients with (vs. $3,390 without) cost-based exacerbations. Overall, 95.7% patients with cost-based exacerbations had ≥1 claims-based exacerbation; 51.0% cost-based exacerbations met the claims-based definition. LIMITATIONS: Cost-based exacerbations may not represent true exacerbations, because cost increases could also result from worsening comorbidities or other clinical events. CONCLUSIONS: Exacerbations identified using a cost-based algorithm frequently lasted >3 weeks. Patients with cost-based exacerbations had higher healthcare costs, particularly respiratory costs, than those without.
Addendum.
J Med Econ · 2025 Dec · PMID 41017396 · Publisher ↗
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The incremental net monetary benefit of higher-valent pneumococcal conjugate vaccines for adults: a systematic review and meta-analysis of economic evaluations.
Lee H, Cho JY, Vietri J … +2 more , Dawson E, Chaiyakunapruk N
J Med Econ · 2025 Dec · PMID 40994424 · Publisher ↗
AIMS: This study aimed to evaluate the cost-effectiveness of newly developed higher-valent pneumococcal conjugate vaccines (PCVs) - including PCV15, PCV20, and PCV21 - for adult populations through a systematic review an...
AIMS: This study aimed to evaluate the cost-effectiveness of newly developed higher-valent pneumococcal conjugate vaccines (PCVs) - including PCV15, PCV20, and PCV21 - for adult populations through a systematic review and meta-analysis of economic evaluations (MAEE), providing pooled incremental net benefits (INBs) as a quantitative summary across studies. METHODS: Economic evaluation studies of PCV15, PCV20, or PCV21 were identified through database searches (PubMed, EMBASE, EconLit, Tufts CEA registry, HTA database, and NITAG resource center) through November 2024. INBs were calculated and pooled using a random-effects model, with positive INB indicating cost-effectiveness. Outcomes were stratified by population age (≥ 18, ≥ 50 and ≥ 60 years), perspective (healthcare and societal), and country income level. Sensitivity analyses assessed the impact of funding sources, imputed variance, and incomplete sensitivity analysis. RESULTS: Twenty-seven studies were included, with 14 comparisons eligible for meta-analysis. PCV20 was the most frequently studied vaccine. From a healthcare perspective, PCV20 was significantly cost-effective compared to PCV15 followed by PPSV23 (PCV15 + PPSV23) in ≥ 18 population (INB = 19.3; 95% CI = 14.2-24.4) and ≥ 60 population (44.3; 21.1-67.4). A similar trend was observed when compared against PPSV23 alone (269.3; 96.4-442.1 and 161.2; 3.5-318.9). Among ≥ 18 population, PCV20 showed favorable INBs compared to PCV13 + PPSV23 (241.7; -5.6-488.9; healthcare perspective, and 171.4; -44.2-386.9; societal perspective), and no vaccine (65.6; -35.7-167.0; healthcare perspective). Among ≥ 60 population, PCV20 was significantly cost-effective compared to PCV15 alone (37.5; 28.0-47.0), but not compared to no vaccine (-13.3; -122.0-95.5). PCV15 and PCV21 had few meta-analyzable comparisons. CONCLUSIONS: Higher-valent PCVs appear cost-effective in most adult populations and settings, especially in high-income countries and from the healthcare perspective. This meta-analysis provides a rigorous quantitative synthesis to support vaccine policy decisions.
Cost drivers and delays in recovery following rotator cuff repair: insights from a national claims database.
Pill S, Beckley SJ, Karim M … +2 more , Stinton SK, Branch TP
J Med Econ · 2025 Dec · PMID 40986644 · Publisher ↗
AIMS: This study aimed to establish a real-world benchmark of recovery following rotator cuff repair (RCR) using healthcare claims data. Secondary objectives included determining the effect of comorbidities and complicat...
AIMS: This study aimed to establish a real-world benchmark of recovery following rotator cuff repair (RCR) using healthcare claims data. Secondary objectives included determining the effect of comorbidities and complications such as joint contracture, additional procedures, and rehospitalizations on the recovery timeline and costs. MATERIALS AND METHODS: Healthcare claims data from the IBM MarketScan Commercial Claims and Encounters Database (2015-2018) were reviewed to determine costs and recovery time after RCR. Costs and recovery duration (index surgery to last therapy claim) were calculated. Subgroup analyses assessed the effects of comorbidities (diabetes, obesity, peripheral vascular disease, cardiovascular disease) and postoperative events (revision, motion restoring surgery (MRS), complication-related surgery, and nonoperative hospitalization) on outcomes. Perioperative complications including joint fibrosis/contracture, infection, and pulmonary embolus were also reported. Descriptive statistics including medians with interquartile ranges (IQR) were reported. RESULTS: In the 14,947 patients included in analysis, median index surgery cost was $11,454 (IQR = $8,169-$17,204). Median recovery was 153 days (IQR = 79-683). Development of postoperative shoulder contracture or adhesive capsulitis added a median of 162 recovery days and nearly doubled costs. Patients requiring surgery for a complication had 3.5-fold longer recoveries and 5-fold higher costs than those without complications. MRS increased recovery time and costs nearly 3-fold, and patients undergoing MRS were 7 times more likely to require arthroplasty. Comorbidities extended recovery by 30-90 days, modestly increased costs, and were associated with a 2-3 times higher frequency of pulmonary embolism. LIMITATIONS: Claims data may be affected by coding inconsistencies, lack of clinical detail, and inability to capture medication costs or outcomes beyond the last therapy claim. CONCLUSIONS: This study defined a benchmark for recovery after RCR and found that complications including contracture and motion restoring surgery substantially increased recovery time and costs. These benchmarks can guide earlier identification of patients at risk for delayed recovery and help in evaluating strategies to reduce economic burden and improve outcomes.
Economics of corneal cross-linking for keratoconus treatment.
Rapuano CJ, Lindstrom RL, Donnenfeld E … +4 more , Berdahl JP, Thompson V, Kratochvil D, Carter J
J Med Econ · 2025 Dec · PMID 40981503 · Publisher ↗
AIM: To optimize the real-world applicability of a health economic model estimating the cost-effectiveness of corneal cross-linking for the treatment of keratoconus in the United States. MATERIALS AND METHODS: A previous...
AIM: To optimize the real-world applicability of a health economic model estimating the cost-effectiveness of corneal cross-linking for the treatment of keratoconus in the United States. MATERIALS AND METHODS: A previously reported discrete-event simulation (DES) model was adapted to reassess the cost-effectiveness of corneal cross-linking (CXL) from the US payer perspective. The simulation of keratoconus progression, which is a key driver of model outcomes, was remodeled to more accurately reflect the real-world relationship between age and the rate of disease progression. All costs were updated to reflect 2025 values. We simulated 4,000 eyes of 2,000 patients diagnosed with keratoconus to compare the lifetime cost and quality-adjusted life years (QALYs) of those treated with CXL vs conventional management. RESULTS: Given updated cost and disease progression parameters, CXL resulted in lower direct medical costs of $38,897 and 2.97 incremental QALYs over a lifetime treatment horizon. Economically justifiable price (EJP) analysis demonstrated that CXL remained cost-effective up to a drug acquisition cost of $172,369 at a $100,000/QALY cost-effectiveness threshold and $246,549 at a $150,000/QALY cost-effectiveness threshold. CONCLUSIONS: After modeling a conservative cost of corneal transplantation and incorporating an age-dependent disease progression rate, our evaluation confirmed that CXL was the dominant (i.e. cost-saving and cost-effective) treatment strategy, primarily due to greatly improved patient outcomes (i.e. benefits strongly correlated with visual acuity). As novel and less invasive methods (e.g. transepithelial CXL) enter the market, it is expected that the demand for corneal cross-linking will increase given its significant clinical and economic value.
Cost-effectiveness analysis of universal hypothyroidism screening in the general population aged 30-65 years in Spain.
Zafón Llopis C, Vicente López O, Cruz Martos MÁ … +6 more , de Los Santos Real H, Alcalá Revilla B, Ojeda Rife O, Pérez Troncoso D, Citoler Berdala B, Cucalón Arenal JM
J Med Econ · 2025 Dec · PMID 40971307 · Publisher ↗
AIMS: Hypothyroidism is an endocrine disorder that often begins in a subclinical form but can lead to non-specific symptoms and cardiovascular problems. Its prevalence is higher among women, and a significant proportion...
AIMS: Hypothyroidism is an endocrine disorder that often begins in a subclinical form but can lead to non-specific symptoms and cardiovascular problems. Its prevalence is higher among women, and a significant proportion of cases remain undiagnosed. While previous studies assessed screening in specific populations (e.g. pregnant women, older adults), this study evaluates the cost-effectiveness of population-wide screening in adults aged 30-65 from the Spanish National Health System (NHS) perspective. MATERIALS AND METHODS: A cost-effectiveness Markov model was developed, simulating seven health states: subclinical hypothyroidism (undiagnosed and controlled), overt hypothyroidism (undiagnosed and controlled), euthyroid state, cardiovascular event, and death. Two strategies were compared: population-based screening no screening. Model inputs-transition probabilities, prevalence, costs, utilities, and screening effectiveness-were obtained from published literature. A panel of four clinical experts validated the model structure and assumptions. Lifetime costs and quality-adjusted life-years (QALYs) were estimated, and the incremental cost-effectiveness ratio (ICER) was calculated. Probabilistic, sensitivity, and scenario analyses were conducted. RESULTS: Population-based screening for hypothyroidism in individuals aged 30-65 resulted in an incremental cost of €34.7 million and 6,037 QALYs gained over 35 years, yielding an ICER of €5,745/QALY, significantly below the Spanish willingness-to-pay threshold (€21,000/QALY). Screening also resulted in 33,215 additional diagnoses of subclinical hypothyroidism and 6,870 fewer cases of overt hypothyroidism. It was cost-effective in 99% of probabilistic simulations and under all tested screening intervals (1-5 years). LIMITATIONS AND CONCLUSIONS: Key limitations include the use of constant transition probabilities and some inputs from international sources. Nonetheless, expert validation supports the model's relevance. The analysis adopts a conservative approach, excluding potential additional benefits like hyperthyroidism detection or integration with routine bloodwork, which could improve cost-effectiveness. Overall, hypothyroidism screening is a cost-effective strategy for the Spanish NHS, improving early detection, preventing progression, and enhancing quality of life in a frequently underdiagnosed population.
Estimating the societal economic burden of food allergy in the United States.
Warren C, Whittington MD, Bilaver L … +8 more , Kratochvil D, Liu R, Seetasith A, Ko S, Garmo V, Kowal S, Gupta S, Gupta R
J Med Econ · 2025 Dec · PMID 40960448 · Publisher ↗
AIM: Food allergy (FA) imposes a large burden on patients/caregivers, but there is limited recent information on the societal economic burden of FA. We aimed to quantify this burden in the United States (US) using a heal...
AIM: Food allergy (FA) imposes a large burden on patients/caregivers, but there is limited recent information on the societal economic burden of FA. We aimed to quantify this burden in the United States (US) using a health economic population cost exercise. METHODS: A prevalence-based model was adopted to estimate the US population with FA and the annual societal/patient costs of FA (in 2024 US$). The Markov model consisted of 3 mutually exclusive health states: "avoidance (with sensitivity)," "full tolerance," and death. Inputs included published data on epidemiology, clinical outcomes, and estimated costs. The model focuses on a comprehensive societal scenario. Scenario analyses utilized a conservative costing approach, determined if the estimated burden exhibited heterogeneity across underserved populations of race/ethnicity and household income, and assessed the impact of allergen desensitization on estimated costs. RESULTS: The model estimated 16,678,832 people with ≥1 physician-diagnosed FA, an annual total societal cost of $370.8 billion, and an annual cost per patient of $22,234, which was higher for children and adolescents than adults. Societal cost was primarily attributed to indirect costs (92.7% of total cost). In the scenario analysis, conservative total societal cost was $39.6 billion; Hispanic, African American, and other races/multiracial patients had ∼3% increased cost relative to White patients; direct medical cost for the low-income group was 39.1% higher than for the high-income group; and total cost was reduced by ∼4% for each additional 10% of the population that entered the model as desensitized. CONCLUSIONS: Using a population cost exercise that incorporated a range of epidemiologically plausible explanations, we estimated the annual total societal cost of FA in the US may approach $370.8 billion, mostly from indirect costs borne by patients/caregivers during daily living. This assessment may assist population-level healthcare decision-makers in investing in measures that reduce economic burden, valuing interventions, and considering approaches to reduce economic/health disparities for underserved populations.
Evaluating the broad societal value of pembrolizumab in women's cancer in Brazil.
Wijenayake N, Ladino D, Patterson K … +4 more , Rosim M, Rojas Rojas M, Davies A, Young K
J Med Econ · 2025 Dec · PMID 40959987 · Publisher ↗
AIMS: Our study analyzes the cost-effectiveness of pembrolizumab in early-stage triple-negative breast cancer (eTNBC) and persistent, recurrent, or metastatic cervical cancer (CC) referred to here as "women's cancer" in...
AIMS: Our study analyzes the cost-effectiveness of pembrolizumab in early-stage triple-negative breast cancer (eTNBC) and persistent, recurrent, or metastatic cervical cancer (CC) referred to here as "women's cancer" in a Brazilian setting. We incorporate the value elements described in the third Special Task Force Report by the Professional Society for Health Economics and Outcomes Research and additional novel elements considered relevant to women's cancer. METHODS: We analyzed the incremental cost-effectiveness ratio (ICER) of pembrolizumab-based therapy in treating eTNBC and CC over a lifetime across the three perspectives: traditional payer perspective (TPP), traditional societal perspective (TSP), and broad societal perspective (BSP). Existing analyses previously used for health technology assessment included a four health state Markov model for eTNBC and a three health state Markov model for CC. These models were expanded to include productivity for the TSP along with caregiver burden, insurance value, value of hope, real option value, severity of disease, out-of-pocket expenses, and fertility treatment costs for the BSP. Data sources included the corresponding clinical trials and a targeted literature review. A single standard of care (SoC) comparator, using indication prevalence weighting and market share, was used to formulate the overall ICER. Probabilistic sensitivity analysis and scenario analyses were conducted. RESULTS: Pembrolizumab-based therapy generated an ICER over three times less with the BSP (R$45,180), compared with TPP (R$139,083) and TSP (R$141,152). The largest driver of results was the inclusion of insurance value, which substantially impacted quality-adjusted life years (QALYs). However, results excluding insurance value still generated an ICER of R$74,291, R$64,792 less than the TPP. Probabilistic results were consistent with the deterministic analysis. CONCLUSION: A broader analysis perspective significantly increased the estimated value of treating women's cancers compared with the TPP, indicating the perspective used by health technology bodies likely does not capture the full societal value of therapeutics.
Cost-utility analysis of icosapent ethyl in patients with high triglyceride levels and recent acute coronary syndrome in Catalonia.
Comin-Colet J, Freixa R, García-Muñoz N … +7 more , Mallén-Alberdi M, Montaño J, Gaugris S, Mistry C, Tilley L, Vitale G, López S
J Med Econ · 2025 Dec · PMID 40957613 · Publisher ↗
OBJECTIVE: To evaluate the cost-effectiveness of icosapent ethyl (IPE) as an adjunct to standard of care (SoC) for reducing cardiovascular (CV) events in statin-treated adults with elevated triglycerides (TG ≥ 150 mg/dL)...
OBJECTIVE: To evaluate the cost-effectiveness of icosapent ethyl (IPE) as an adjunct to standard of care (SoC) for reducing cardiovascular (CV) events in statin-treated adults with elevated triglycerides (TG ≥ 150 mg/dL), established CV disease, and a recent acute coronary syndrome (ACS) in Catalonia, Spain. METHODS: A partitioned survival model was developed to simulate the natural history of CV events over a 20-year horizon from the Catalan healthcare payer perspective. The model incorporated clinical efficacy and safety data from a analysis of REDUCE-IT, a global CV outcomes trial with IPE, local treatment patterns, and Spanish-specific cost data. Outcomes were expressed in quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs). Deterministic, probabilistic, and scenario sensitivity analyses were conducted to assess model robustness. RESULTS: In the base case, IPE plus SoC resulted in an incremental gain of 0.57 QALYs and €8,287 in additional costs compared to SoC alone, yielding an ICER of €14,543/QALY gained-well below the commonly accepted willingness-to-pay threshold of €30,000/QALY in Spain. Probabilistic sensitivity analysis showed that IPE was cost-effective in 75.3% of simulations and dominant in 15.4%. Scenario analyses confirmed the robustness of results across different time horizons and discount rates. Key drivers of cost-effectiveness were the selected efficacy curves for IPE and its per-cycle treatment cost. CONCLUSIONS: IPE appears to be a cost-effective intervention for high-risk patients with elevated TG and recent ACS in Catalonia. While limitations related to model assumptions, data extrapolation, and partial adaptation to local clinical practice exist, the findings remain consistent with international evidence and suggest that IPE could be a cost-effective intervention in Catalonia, offering a valuable opportunity to optimize healthcare resource allocation in the management of high-risk CV populations.
Correction.
J Med Econ · 2025 Dec · PMID 40957609 · Publisher ↗
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Assessment of unmet clinical needs and healthcare resource use among statin-treated patients with or at risk of developing atherosclerotic cardiovascular disease.
Ortiz N, Shehata J, Smart J … +5 more , Leinwand B, Ali A, Katariya D, Dicklin MR, Hsieh A
J Med Econ · 2025 Dec · PMID 40955449 · Publisher ↗
AIM: Atherosclerotic cardiovascular disease (ASCVD) imposes considerable clinical and economic burdens. ASCVD prevention seeks to control low-density lipoprotein cholesterol (LDL-C) using statins as first-line treatment....
AIM: Atherosclerotic cardiovascular disease (ASCVD) imposes considerable clinical and economic burdens. ASCVD prevention seeks to control low-density lipoprotein cholesterol (LDL-C) using statins as first-line treatment. This retrospective US cohort study assessed unmet clinical needs and healthcare resource use among statin users in primary and secondary prevention. MATERIALS AND METHODS: MarketScan administrative claims 2017-2021 were leveraged and linked to laboratory data to identify patients with hypercholesterolemia followed for 2 years. Numbers of statin-treated hypercholesterolemia patients in primary prevention, very high-risk or not very high-risk secondary prevention, and their LDL-C goal achievement, were estimated and inflated to national estimates, along with annualized healthcare resource utilization and costs. Cardiovascular events according to LDL-C goal attainment were also assessed. RESULTS: Almost 125,000 statin-treated patients did not meet LDL-C goals. Data inflated to US national estimates suggested approximately 72 million (M) patients have hypercholesterolemia: 43 M primary prevention (∼40% above goal), 9.8 M very high-risk secondary prevention (∼78% above goal), and 9.1 M not very high-risk secondary prevention (∼60% above goal) are treated with statins, and 9.5 M are untreated (∼84% above goal). Managing LDL-C to goal was associated with a 50% reduction in the proportion of patients with a cardiovascular event. Patients utilizing high-cost healthcare services and annualized healthcare costs increased from primary to secondary prevention, and from not very high- to very high-risk secondary prevention. LIMITATIONS AND CONCLUSIONS: Prevention is an essential component of any effort to improve population health and ultimately reduce spending. While some prevention efforts are cost-saving, some strategies that improve health will increase total spending. Nonetheless, millions of people in the US taking statins do not achieve LDL-C goals, indicating a significant clinical burden among those with, or at risk for, ASCVD, resulting in substantial healthcare resource use and costs.
Navigating the economic value of treatment sequencing in large B-cell lymphoma: insights into optimizing value and patient outcomes of CAR T-cell and other available therapies in Brazil.
Nabhan S, Kievit B, Blissett R … +1 more , Ball G
J Med Econ · 2025 Dec · PMID 40955441 · Publisher ↗
BACKGROUND: The advent of chimeric antigen receptor t-cell therapy (CAR T) has ushered in a new treatment paradigm in relapsed or refractory (R/R) diffuse large b-cell lymphoma (DLBCL) in which patients have the potentia...
BACKGROUND: The advent of chimeric antigen receptor t-cell therapy (CAR T) has ushered in a new treatment paradigm in relapsed or refractory (R/R) diffuse large b-cell lymphoma (DLBCL) in which patients have the potential to be cured of their disease. However, as a result of non-clinical barriers and eligibility misinterpretations, patients may not receive CAR T therapy and are instead misallocated to treatment pathways which may result in suboptimal costs and outcomes. We sought to quantify the financial impact of these misallocations across the treatment sequences of therapies available to patients with R/R DLBCL in Brazil. METHODS: We developed a patient-level discrete event simulation model to compare treatment options within the sequence of available therapies for DLBCL in Brazil. A cost offset analysis was performed to analyze the financial impact of possible DLBCL treatment sequences with axicabtagene ciloleucel (axi-cel), epcoritamab, and standard-of-care (SoC) therapies. Sensitivity analyses were conducted to examine costs and outcomes for axi-cel compared to tisagenlecleucel (tisa-cel), another available CAR T therapy in Brazil. RESULTS: We found that using axi-cel before epcoritamab in the 3L setting was cost-saving compared to using epcoritamab before axi-cel. Compared to both epcoritamab and SoC therapies, cost offsets were observed across all treatment lines due to the prolonged survival of treating with axi-cel compared to other options, and savings were greater when axi-cel was administered earlier in the treatment sequence. Sensitivity analyses demonstrated that treating with axi-cel in 3L was less expensive than treating with tisa-cel. CONCLUSION: Administering CAR T therapies like axi-cel before epcoritamab or SoC in R/R DLBCL can lead to substantial cost offsets as fewer patients ultimately progress to subsequent treatment lines. Optimal R/R DLBCL outcomes and costs can be achieved in Brazil through maximizing the potential for cure by treating eligible patients with axi-cel ahead of other available therapies.
Budget impact of introducing an omalizumab biosimilar in 23 European countries.
Jang M, Lee J, Yi S … +1 more , Kwon TS
J Med Econ · 2025 Dec · PMID 40955168 · Publisher ↗
BACKGROUND: Omalizumab, a monoclonal antibody indicated for severe persistent allergic asthma, chronic spontaneous urticaria (CSU), and chronic rhinosinusitis with nasal polyps (CRSwNP), imposes a financial burden on Eur...
BACKGROUND: Omalizumab, a monoclonal antibody indicated for severe persistent allergic asthma, chronic spontaneous urticaria (CSU), and chronic rhinosinusitis with nasal polyps (CRSwNP), imposes a financial burden on European healthcare systems due to its high cost, with expenditures exceeding €787 million across 23 countries in 2024. Introducing an omalizumab biosimilar offers a promising avenue to reduce expenditure while maintaining clinical outcomes. OBJECTIVE: To evaluate the budgetary impact of introducing an omalizumab biosimilar for severe persistent allergic asthma, CSU, and CRSwNP across 23 European countries from a payer's perspective. METHODS: A biosimilar market share uptake-based budget impact model (BIM) was developed in Microsoft Excel to compare two scenarios over 5 years: one without biosimilar introduction and one with gradual adoption. Market share evolution was modeled using country-specific biosimilar uptake as a proxy, while omalizumab usage growth was estimated from historical compound annual growth rates. Drug acquisition costs came from national price databases, assuming a 30% discount for the biosimilar. Sensitivity analysis applying 50% and 70% discount rates was conducted to reflect reimbursed cost from the payer's perspective. RESULTS: Introducing an omalizumab biosimilar is projected to save €40 million in the first year at a 30% discounted price compared with using only the originator across 23 countries. Over 5 years, cumulative savings could reach €640 million, potentially enabling treatment for ∼96,000 additional patients per year from the derived budget savings. These estimates are a conservative baseline because the model does not incorporate competitive price erosion of the originator following biosimilar entry; in real-world settings, savings are typically higher when originator prices decline in response to competition. CONCLUSION: Adopting an omalizumab biosimilar in European healthcare systems could yield substantial cost savings, enhance patient access, and reduce fiscal pressure. This analysis supports biosimilars as a strategic tool for sustainable biologic therapy access in Europe.
Budget impact analysis of skytrofa for the treatment of pediatric growth hormone deficiency in a US health plan.
Boller E, Raveendran S, Smith A … +3 more , Noori W, Miner P, Kleintjens J
J Med Econ · 2025 Dec · PMID 40944302 · Publisher ↗
BACKGROUND: Daily administration of growth hormone (GH) can be challenging for patients with pediatric growth hormone deficiency (pGHD) and their caregivers. Lonapegsomatropin, a prodrug of somatropin, is administered on...
BACKGROUND: Daily administration of growth hormone (GH) can be challenging for patients with pediatric growth hormone deficiency (pGHD) and their caregivers. Lonapegsomatropin, a prodrug of somatropin, is administered once weekly and designed to provide sustained release of active, unmodified somatropin. International guidelines recognize the burden associated with daily injections and suggest long-acting GH to improve adherence and outcomes. The design of the auto-injector for lonapegsomatropin results in zero medication wastage leading to potential cost savings. OBJECTIVE: To estimate the budget impact to a US health plan of introducing lonapegsomatropin to a market with existing daily and weekly GH treatments and to identify possible cost savings associated with its use. METHODS: The budget impact model (BIM) was developed to compare the costs associated with two hypothetical scenarios for pediatric pGHD: one without lonapegsomatropin, where daily and weekly GHs are available, and one with lonapegsomatropin introduced to the market alongside daily and weekly GHs. The model combined per-patient costs with expected market shares for GHs to estimate the total financial expenditure for both scenarios over a five-year period, simulating costs for a US payer covering one million lives. This analysis included daily somatropin, and weekly somatrogon and somapacitan, as comparators. RESULTS: The introduction of lonapegsomatropin resulted in a cumulative total saving of $959,629 over a 5-year time horizon, with cost savings of $40,314 in Year 1, increasing to $373,258 in Year 5. The estimated average cost savings were $3,283 per patient per month (PPPM) and $0.08 per member per month (PMPM). Scenario and sensitivity analyses demonstrated that the cost-savings with lonapegsomatropin are robust across various assumptions, including alternative market share and dosing scenarios. The primary drivers of the cost reduction were decreased acquisition costs for daily somatropin and the elimination of wastage for weekly GHs. CONCLUSIONS: The inclusion of lonapegsomatropin in the formulary of a US health plan can result in a significant reduction in total costs for the plan.
Comparison of utilization and total medicare fee-for-service expenditures for subcutaneous versus intravenous versions of a select group of therapies.
Kardel P, Sheetz C, Maynard J … +1 more , Sarocco P
J Med Econ · 2025 Dec · PMID 40938305 · Publisher ↗
INTRODUCTION: Comparisons of subcutaneous (SC) and intravenous (IV) delivery of biotherapeutics in Europe have revealed differences in cost, convenience, and preference. In the US, claims-based studies comparing SC and I...
INTRODUCTION: Comparisons of subcutaneous (SC) and intravenous (IV) delivery of biotherapeutics in Europe have revealed differences in cost, convenience, and preference. In the US, claims-based studies comparing SC and IV delivery have been limited. This study compared resource utilization and expenditures between cohorts of patients receiving the same drug by the SC or IV route of administration (ROA) across several indications. METHODS: Medicare Fee-For-Service claims were compared between cohorts of patients who received SC- or IV-delivered abatacept, belimumab, daratumumab, infliximab, tocilizumab, trastuzumab/pertuzumab, or vedolizumab. For each drug, utilization rates and healthcare expenditures per service site were compared between ROAs. Observed differences in baseline characteristics were accounted for using Inverse Probability Treatment Weighting models that balanced pre-index differences between ROAs per drug. Each ROA comparison per drug and service site was weighted and conducted independently. RESULTS: Across seven drugs, a total sample size of 158,632 (72,820 IV; 85,812 SC) was analyzed. For most comparisons, high-spend sites were utilized at a higher rate for IV administration. In particular, all comparisons revealed more frequent hospital outpatient department utilization for the IV ROA. For five of the seven drugs, SC treatment was associated with lower mean total Medicare expenditures, with savings of up to $56,000 per patient annually. Although three SC treatments had higher medication index spend, the total spend for these drugs across sites was significantly lower for SC delivery. LIMITATIONS: Limitations of this study include differences in billing between the SC and IV ROAs, potential treatment selection bias, and the assumption of equivalent efficacy. CONCLUSIONS: To our knowledge, this study was the first and largest US Medicare claims analysis, comparing patients receiving SC or IV versions of the same therapeutic across multiple drugs and indications. Findings demonstrated that SC delivery may facilitate reduced resource utilization and expenditures across drugs and disease indications.
Net present value cost analysis for treating retinitis pigmentosa.
Hellems S, Puri S, Yip S … +2 more , Fehr J, Milentijevic D
J Med Econ · 2025 Dec · PMID 40923851 · Publisher ↗
OBJECTIVES: To provide insights into the financial burden and opportunity cost of vision loss from retinitis pigmentosa (RP) in the US by using net present value (NPV) of direct medical and nonmedical costs. METHODS: Ass...
OBJECTIVES: To provide insights into the financial burden and opportunity cost of vision loss from retinitis pigmentosa (RP) in the US by using net present value (NPV) of direct medical and nonmedical costs. METHODS: Assumptions, including economic (discount rate, median income, cost-of-living, Social Security and Medicare taxes, public insurance/supplemental benefits, nutrition assistance, and prescription drug assistance), medical (federal National Health Expenditure tables, a recent retrospective claims analysis, and Optum Health claims database) and demographic (mortality rate, increase in mortality due to visual impairment, progression of blindness, probability of survival, retirement rate, rate of disability, and RP diagnosis probability) were made to develop a NPV model. Scenario analyses were performed on benefits and costs arising from patients with RP, if vision could be preserved novel gene therapies. RESULTS: Based on modeled assumptions, for a patient with RP treated with gene therapy at age 15, the NPV opportunity cost of not treating was $3,973,909 ($1,027,358 in lost accrual of government benefits throughout employment and $2,946,551 paid by government programs due to visual impairment). For a patient treated at age 35, the NPV opportunity cost of not treating was $2,866,964 ($719,926 in lost accrual of benefits and $2,147,038 paid by government programs). For a patient treated at age 55, the NPV opportunity cost of not treating was $2,091,032 ($758,445 in lost accrual of government benefits and $1,332,587 paid by government programs). LIMITATIONS: As it was not possible to include all potential factors, the model may under- or overestimate true NPV. Potential treatment costs were included as hypothetical estimates. CONCLUSIONS: Novel gene therapies for RP likely offset significant government costs toward disability payments; these savings may be even greater when administered during adolescence.
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