Journal of Medical Economics最新文献

Aim: This study aimed to describe treatment patterns and quantify the economic impact of recurrence in early-stage human epidermal growth factor receptor 2 (HER2)-negative breast cancer (BC).

Materials & methods: Medicare beneficiaries with stages I-III HER2-negative BC and lumpectomy or partial/total mastectomy were identified from SEER-Medicare data (2010-2019). Perioperative therapies were reported in the neoadjuvant and adjuvant setting. Locoregional recurrence and distant metastasis were identified using a claims-based algorithm developed with clinical input and consisting of a diagnosis-based and treatment-based indicator. All-cause and BC-related healthcare resource utilization (HRU) per-patient-month and monthly healthcare costs were estimated from the recurrence date for patients with recurrence and from an imputed index date for patients without recurrence using frequency matching. HRU and costs were compared between groups stratified by hormone receptor-positive (HR+) or triple negative BC (TNBC) using multivariable regression models.

Results: Of 28,655 patients, 8.5% experienced recurrence, 90.4% had HR+ disease, and 5.6% received neoadjuvant therapy. Relative to patients without recurrence, patients with recurrence had more advanced disease (stage II/III: 73.7% vs. 34.0%) and higher-grade tumors (Grade 3/4: 40.6% vs. 18.0%) at diagnosis. Recurrence in HR+/HER2-negative BC and TNBC was associated with higher rates of all-cause hospitalizations (incidence rate ratio [IRR]: 2.84 and 3.65), emergency department (ED) visits (IRR: 1.75 and 2.00), and outpatient visits (IRR: 1.46 and 1.55; all p < 0.001). Similarly, recurrence was associated with higher rates of BC-related HRU, particularly for ED visits in HR+/HER2-negative BC (IRR: 4.24; p < 0.001) and hospitalizations in TNBC (IRR: 11.71; p < 0.001). Patients with HR+/HER2-negative BC and TNBC recurrence incurred higher monthly all-cause (cost difference [CD]: $3988 and $4651) and BC-related healthcare costs (CD: $3743 and $5819).

Conclusions: Our findings highlight the considerable economic burden of recurrence in early-stage HER2-negative BC and underscore the unmet need for optimization of therapies that reduce recurrence in this population.

Objective: The cost-effectiveness of neoadjuvant pembrolizumab + chemotherapy followed by adjuvant pembrolizumab compared to neoadjuvant chemotherapy plus placebo followed by adjuvant placebo was assessed in high-risk, early-stage, triple-negative breast cancer patients from an Egyptian societal perspective over a lifetime horizon.

Methods: A 4-state Markov cohort model was developed to compare the cost-effectiveness of pembrolizumab + chemotherapy/pembrolizumab vs chemotherapy alone for the treatment of high-risk, early-stage, triple-negative breast cancer. The model simulated the clinical course of high-risk, early-stage, triple-negative breast cancer across four health states: event-free survival, locoregional recurrence, distant metastasis, and death. Clinical inputs for the simulation were derived from modeling of efficacy and safety data collected in the KEYNOTE-522 trial. Direct medical costs and indirect costs were reported in 2022 Egyptian pounds (EGP) and converted to US dollars ($). Probabilistic and deterministic sensitivity analyses were conducted to assess the robustness of model results.

Results: Compared with chemotherapy alone, pembrolizumab + chemotherapy/pembrolizumab led to expected gains of 2.92 life years and 2.25 quality-adjusted life years, respectively, while increasing overall treatment costs by EGP 491,695 ($102,436). Incremental costs per year gained were EGP 218,285 ($45,476) per quality-adjusted life year and EGP 168,223 ($35,046) per life year, both of which were lower than the 2022 Egyptian cost-effectiveness threshold of EGP 398,439 ($83,008). The findings of sensitivity analyses indicated that the model was robust across a range of inputs and assumptions.

Conclusions: In Egypt, pembrolizumab + chemotherapy/pembrolizumab is a cost-effective treatment for high-risk, early-stage, triple-negative breast cancer when considering health-related quality-of-life and years of life gained.

Objective: The objective of this study was to explore the financial consequences of adopting cenobamate as a treatment alternative in epilepsy patients with drug-resistant focal onset seizures (FOS) from a societal perspective in the Netherlands.

Methods: A previous budget impact model with a 5-year time horizon was adapted to the Dutch setting accounting for the eligible population, real-world market shares, treatment effectiveness and resource use in two scenarios: cenobamate with constant market share versus cenobamate with linearly increased market share up to 20%. Clinical inputs included treatment response, seizure reduction and adverse events. Costs consisted of drugs, medical and non-medical costs. One-way sensitivity analysis and scenario analysis were conducted to test the robustness of our results.

Results: 14,723 patients were eligible for cenobamate in 2022. Although cenobamate adds a gross budget impact of €12,686,30, the displacement of other drugs yields a total impact on the drug budget of €3,722,596 over 5 years. Adopting cenobamate resulted in a medical cost savings of €13,499,498 due to less resource use, and non-medical cost savings of €22,144,054 due to reduced productivity losses. Overall, savings generated at medical and non-medical cost level offset the gross drug budget impact of cenobamate, resulting in a saving of €31,920,955 over 5 years. Results were robust in the sensitivity/scenario analyses.

Conclusion: Treatment with cenobamate is associated with both medical and non-medical cost savings, which offset the increase in drug budget and result in a significant potential budget saving. The higher the market share of cenobamate, the larger the budget savings. We acknowledge several limitations; Complex scenarios such as drug interactions, stopping/switching drugs, and multiple drug use were not taken into account. The long-term efficacy and safety of cenobamate and its comparators remains uncertain. Future real-world data are needed to confirm our findings.

Aim: Inadequate response to antidepressant therapy (ADT) is common in major depressive disorder (MDD); atypical antipsychotic (AA) adjunctive therapy may be effective for these patients. This study aimed to compare healthcare resource utilization (HRU) and costs between patients initiating the AA cariprazine as their first adjunctive therapy vs those initiating cariprazine subsequently.

Methods: The Merative MarketScan Commercial Database (January 1, 2015, to June 30, 2021) was used to identify US adults with MDD and ≥1 pharmacy claim for cariprazine adjunctive to ADT in 2018 or after. Rates of mental health (MH)‑related and all‑cause HRU per patient-year (PPY) and mean healthcare costs per-patient-per-year (PPPY) were assessed after patients first initiated adjunctive therapy. HRU and costs were compared between cohorts using rate ratios (RRs) and mean cost differences, respectively, estimated from multivariable regression models.

Results: Of 838 patients receiving cariprazine, 44.7% initiated cariprazine as their first adjunctive therapy to ADT, and 55.3% initiated it subsequently. Those initiating cariprazine first had significantly lower rates of MH‑related hospitalizations (RR [95% confidence interval] = 0.55 [0.30, 0.90], p = .020) and outpatient (OP) visits (0.67 [0.57, 0.82], p < .001) PPY than those initiating cariprazine subsequently. Moreover, patients initiating cariprazine as their first adjunctive therapy had lower annual total MH‑related healthcare costs (mean cost difference [95% confidence interval] -$2,182 [-$4,206, -$69], p = .040), driven primarily by lower OP visit costs (-$1,511 [-$2,330, -$615], p < .001). Similar trends were observed for all-cause HRU and costs.

Limitations: This was a retrospective analysis of secondary data with limited follow-up. Claims were a proxy for cariprazine use.

Conclusions: Results from this real‑world study of commercially insured US adults suggest that initiating cariprazine as the first adjunctive therapy rather than a subsequent therapy could help mitigate the considerable economic burden of MDD for appropriate patients.

Objective: This study aimed to investigate the willingness to pay (WTP) of the Japanese population for the transmission prevention function of SARS-CoV-2 antiviral treatments and identify the attributes associated with higher WTP.

Methods: A web-based survey (registration number: UMIN000054955) was conducted from May 17 to June 1, 2024, targeting a general population using a survey company panel. We aimed to obtain around 3,000 valid responses. Respondents were randomly divided into two groups: one assuming a COVID-19 infection (infection-assumed group) and the other without this assumption (non-infection-assumed group). WTP was assessed using an open-ended question format, asking how much they would be willing to pay out-of-pocket for a hypothetical antiviral drug that reduces the risk of transmitting COVID-19 to others by half. The survey also collected demographic information, COVID-19 related attributes, empathy levels using the Multidimensional Empathy Scale (MES), and health literacy using the Communicative and Critical Health Literacy scale. The mean WTP for COVID-19 treatment was calculated for all respondents and for the infection-assumed and non-infection-assumed groups. Subgroup analyses examined the effects of respondent attributes on WTP. A linear regression model with stepwise selection identified factors associated with WTP.

Results: Responses were obtained from 3,657 individuals, with 3,131 valid responses analyzed. The mean WTP among all respondents was JPY 3,205 (USD 20.85) (standard error: JPY 84 [USD 0.55]). The infection-assumed group showed a 21% higher WTP than the non-infection-assumed group (p < 0.001). Subgroup analyses indicated that WTP varied based on attributes such as co-residing children, occupation, empathy levels, and health literacy. Higher WTP was significantly associated with being aged 65 years and older, higher household income, absence of co-residing children, being a company employee, executive, or public servant, fear of COVID-19 infection, higher other-oriented emotional reactivity (a factor of MES), and higher health literacy.

Conclusion: We presented the WTP of the Japanese population for the transmission prevention function of COVID-19 treatments as an actual monetary value. Factors such as empathy, health literacy, and some attributes were significantly associated with WTP. These findings might help inform policymakers in developing health policies based on the universal health insurance system in Japan.

AimsThe cardioprotective effects of semaglutide 2.4 mg reported in the SELECT cardiovascular (CV) outcomes trial (ClinicalTrials.gov NCT03574597) provide clinical benefit for subjects with overweight or obesity and established CV disease without type 2 diabetes (T2D). We assessed cost-effectiveness of semaglutide 2.4 mg in this population against the American College of Cardiology/American Heart Association value framework.Materials and methodsA cohort-level Markov-state cost-effectiveness model using trial-derived data with outcomes from a healthcare sector perspective measured over a lifetime horizon was developed. Treatment costs were based on US list prices; scenario analyses used literature-reported estimated rebates. Healthcare costs and benefits were discounted at 3.0%. A simulated cohort of 100,000 subjects was aligned to the SELECT trial population baseline characteristics and time-on-treatment. Subjects received either semaglutide 2.4 mg or placebo in addition to standard of care (SoC). Modelled outcomes included clinical events (CV events, progression to T2D, chronic kidney disease [CKD]) and health economic measures, including direct costs and quality-adjusted life years (QALYs).ResultsMean semaglutide 2.4 mg treatment duration was 2.79 years. Per 100,000 subjects, treatment avoided 2,791 non-fatal myocardial infarctions, 3,000 coronary revascularizations, 487 non-fatal strokes, and 115 CV deaths over the modeled lifetime horizon. Average per-subject lifetime treatment costs were $47,353; savings arose from avoided T2D ($14,431), CKD ($2,074), and CV events ($1,512). Semaglutide 2.4 mg was associated with increased lifetime costs ($29,767), additional QALYs gained (0.218) and an incremental cost-effectiveness ratio of $136,271/QALY at list price; a scenario using an empirically estimated 48% rebate predicted $32,219/QALY.LimitationsThe generalizability of observations from SELECT to a broader US population is unknown. Our model does not capture all outcomes nor costs that may be affected by weight loss. Modeling assumptions may present limitations.ConclusionsSemaglutide 2.4 mg use as in SELECT is cost-effective at list price, using a $150,000/QALY willingness-to-pay threshold.

Aims: Iron deficiency anemia (IDA) is among the most common extraintestinal sequelae of inflammatory bowel disease (IBD). Intravenous iron is often the preferred treatment in patients with active inflammation with or without active bleeding, iron malabsorption, or intolerance to oral iron. The aim of the present study was to evaluate the cost-utility of ferric derisomaltose (FDI) versus ferric carboyxymaltose (FCM) in patients with IBD and IDA in Norway.

Materials and methods: A published patient-level simulation model was used to evaluate the cost-utility of FDI versus FCM in patients with IBD and IDA from a Norwegian national payer perspective. Iron need was modelled based on bivariate distributions of hemoglobin and bodyweight combined with simplified tables of iron need from the FDI and FCM summaries of product characteristics. Patient characteristics and disease-related quality of life data were obtained from the PHOSPHARE-IBD trial. Cost-utility was evaluated in Norwegian Kroner (NOK) over a five-year time horizon.

Results: Patients required 1.64 fewer infusions of FDI than FCM over five years (5.62 versus 7.26), corresponding to 0.41 fewer infusions per treatment course. The reduction in the number of infusions resulted in cost savings of NOK 7,720 (NOK 35,830 with FDI versus NOK 43,550 with FCM). The need for phosphate testing in patients treated with FCM resulted in further cost savings with FDI (no costs with FDI versus NOK 4,470 with FCM). Total cost savings with FDI were therefore NOK 12,190. FDI also increased quality-adjusted life expectancy by 0.071 quality-adjusted life years (QALYs) driven by reduced incidence of hypophosphatemia and fewer interactions with the healthcare system.

Conclusions: FDI resulted in cost savings and improved quality-adjusted life expectancy versus FCM in patients with IDA and IBD in Norway. FDI therefore represents the economically preferable iron formulation in Norwegian patients with IBD and IDA in whom it is indicated.