Journal of Medical Economics最新文献

Objective: This study aimed to evaluate the cost-effectiveness of deucravacitinib vs. apremilast as a treatment for moderate-to-severe psoriasis patients from a Japan healthcare system perspective.

Methods: A Markov sequence model was developed, consisting of an induction phase, maintenance phase, best supportive care and death. Clinical inputs were predominantly derived from the POETYK-PSO-1 and -2 trials (NCT03624127 and NCT03611751), and cost and resource use inputs were derived from several Japanese sources, including Ministry of Health and Welfare (MHLW) data and the outputs of a Delphi survey with Japanese clinical experts. Health-related quality of life inputs were based on the change in utility associated with different levels of Psoriasis Area and Severity Index (PASI) response. Deterministic and probabilistic sensitivity analyses were conducted to account for uncertainty around the base case and several scenario analyses were performed to explore structural uncertainty related to assumptions and methodological choices.

Results: In the base case, treatment with deucravacitinib results in a discounted QALY gain of 0.30 and discounted incremental costs of ¥459,771 compared to apremilast, resulting in an ICUR of ¥1,546,713 per QALY which is below the Japanese willingness to pay threshold of ¥5,000,000 per QALY. Deterministic and probabilistic sensitivity analyses support the results of the base case. The latter shows that deucravacitinib has a 97.8% probability of being cost-effective compared to apremilast at the ¥5,000,000 per QALY threshold. The outcomes of all scenarios confirmed the cost-effectiveness of deucravacitinib compared to apremilast, with deucravacitinib being dominant in one scenario.

Conclusions: Deucravacitinib is cost-effective compared to apremilast in patients with moderate-to-severe plaque psoriasis in Japan, primarily driven by improvements in health-related quality of life associated with a more favorable PASI response. This conclusion is supported by extensive sensitivity and scenario analyses.

Aim: To estimate the cost-effectiveness of Nirmatrelvir/ritonavir (NMV/r) versus best supportive care (BSC) in patients at high-risk for progression to severe COVID-19 from a German health payer perspective.

Methods: A closed cohort static model of 1,000 COVID-19 patients capturing the short-term (<1 year) via decision-tree and long-term (lifetime) outcomes via Markov model was used to assess the cost-effectiveness of NMV/r versus BSC. Model inputs were derived from the EPIC-HR clinical trial and published contemporary real-world data. Probabilistic and deterministic sensitivity analyses (PSA, DSA) were conducted to test the robustness of model results.

Results: In the base case, treatment with NMV/r versus BSC reduced COVID-19 related hospitalisations (-0.042), intensive care unit admissions (-0.006) and inpatient deaths (-0.003), while increasing life-years (LY) (0.047) per patient, which results in an incremental cost-effectiveness ratio of 10,845 € per hospitalisation avoided and 9,773 € per LY gained. Sensitivity analysis suggests the magnitude of the benefits increased with increasing hospitalisation risk. NMV/r emerged as the dominant strategy in a population with a hospitalisation risk equivalent to 60 years and older. Outcomes were similar with real world effectiveness data. DSA showed the model was most sensitive to hospitalisation and inpatient mortality risk, NMV/r medication cost and efficacy/effectiveness of NMV/r in reducing hospitalisation. PSA confirmed the robustness of the model results.

Limitations: As COVID-19 is a dynamic disease, caution should be taken in generalizing these results. Contemporary data is essential to ensure the model inputs and the outcomes remain relevant as there may be changes in natural disease course or effectiveness of NMV/r.

Conclusions: This cost-effectiveness analysis of NMV/r treatment from a German healthcare payer perspective demonstrates how by preventing progression to severe COVID-19, NMV/r reduces healthcare resource use, associated costs and preserves LY of patients. This analysis provides crucial economic rationale for decision making by policy makers.

Background and objectives: Chronic Kidney Disease-associated Pruritus (CKD-aP) is a disabling condition that affects around 60% of patients with end-stage kidney disease undergoing dialysis. Current off-label treatment options are neither effective nor appropriate for all dialysis patients, leaving a clear unmet need. This study aimed to evaluate the cost-effectiveness of difelikefalin - the only drug approved in Europe for the treatment of moderate to severe CKD-aP adult patients in haemodialysis - compared to the best supportive care (BSC) from the Spanish NHS perspective.

Methods: A Markov model was developed with seven health states: five health states representing levels of pruritus intensity over time (No, Mild, Moderate, Severe and Very severe CKD-aP), kidney transplant and death as the absorbing state. The model included patients with moderate to severe CKD-aP at baseline, in line with difelikefalin approved indication and clinical trials. Local costs, utilities, mortality rates and kidney transplant probabilities were obtained from published literature. Costs and quality-adjusted life-years (QALYs) were discounted at a 3% annual rate with a lifetime horizon (36 years).

Results: Difelikefalin was associated with an increased in QALYs (+0.49) and higher costs (+12,300€) compared to the BSC over a lifetime horizon. At a provisional cost estimate of 270.6€per 28-days for difelikefalin (based on a tentative list price for Spain), the incremental cost-utility ratio was 25,000€/QALY. The sensitivity analysis (DSA) confirmed the robustness of the results. The probabilistic sensitivity analysis (PSA), undertaken with 1000 iterations, indicated a 50% and 83% probability of difelikefalin being cost-effective at a willingness-to-pay (WTP) thresholds of 25,000 €/QALY and 30,000 €/QALY, respectively.

Conclusions: Difelikefalin could be a cost-effective option compared to BSC for the management of CKD-aP in adult haemodialysis patients within the Spanish NHS setting. Considering the unmet needs, these results support the convenience of incorporating difelikefalin in routine clinical practice in Spain.

Objective/aim: In 2009, dronedarone was approved by the United States Food and Drug Administration based on results from the ATHENA trial (NCT00174785), which showed significant reduction of cardiovascular (CV) hospitalization and death in patients with atrial fibrillation (AF) randomized to dronedarone versus placebo. In 2020, a retrospective study by Goehring et al. showed CV hospitalizations and deaths were lower in clinical practice following initiation of dronedarone compared to other antiarrhythmic drugs (AADs) in patients with AF and atrial flutter. However, the economic impact associated with dronedarone use has not been fully assessed. The objective of this study was to estimate the cost associated with CV outcomes reported by Goehring et al. (2020).

Methods: National average Medicare payments in the Centers for Medicare and Medicaid Services (CMS) database (www.data.CMS.gov) were used to assign cost estimates to CV outcomes evaluated in Goehring et al. (2020) by diagnosis-related grouping. When costs were unavailable in the CMS database, a literature search was performed to identify publications reporting hospitalization costs.

Results: The weighted average cost for CV hospitalization was calculated to be $20,508. When multiplied by the event rate reported in Goehring et al. (2020), cost per person year for CV hospitalization was 14% lower with dronedarone versus other AADs ($3,679 vs $4,272, respectively). For hospitalizations due to heart failure, cost was 31% lower with dronedarone compared with other AADs ($324 vs $472, respectively).

Limitations: Costs have been calculated based on national averages reported by CMS (Medicare perspective) and are estimates. Regional differences may be present.

Conclusions: Patients with AF taking dronedarone had lower costs associated with CV hospitalization compared with patients taking other AADs.

Background/aims: KEYNOTE-048 (KN-048), a phase III clinical trial was conducted in first-line patients with recurrent or metastatic (R/M) head and neck squamous cell carcinoma (HNSCC). It demonstrated that pembrolizumab, when combined with platinum-based therapies (cisplatin or carboplatin) plus 5-Fluorouracil (5-FU) in the overall population, and in the combined positive score (CPS) ≥ 1 population, improves overall survival (OS) compared to the combination of cetuximab + platinum + 5-FU (EXTREME regime). The aim was to evaluate the cost-effectiveness of pembrolizumab as a combination therapy in the 1 L HNSCC CPS ≥ 1 sub-population compared to the EXTREME regime from a healthcare system perspective in Colombia.

Methods: We built a three-state partitioned survival model to project the costs and outcomes over 40 years assuming a 3% annual discount. We used data from KEYNOTE-048 to model fits for progression-free survival (PFS), OS and Time-on-treatment curves for 1 L. Parametric extrapolations were then employed for the second part of the fit. The time-point selection was based on a series of statistical criteria including the chow test and log-hazard functions as well as an examination of remaining event within the tail of the curves. The parametric curve fits were guided by a comparison of real-world data, AIC/BIC criteria as well as visual inspection. Cost data for both first-line and subsequent treatments were derived from national public drug and procedures lists, namely SISMED and ISS Tariff Manual. Utilities were derived from KEYNOTE-048 Euro-QoL five dimension, using an Argentina-specific algorithm.

Results: An additional 2.05 life-years (LY) and 1.62 quality-adjusted life-years (QALYs) were the result versus comparator. The incremental cost-effectiveness ratios (ICERs) were COP $48,330,146/LY gained and COP $61,078,685/QALY gained, which were lower than the 2023 Colombian willingness-to-pay (WTP) threshold (COP $69,150,201).

Conclusions: Pembrolizumab combination therapy offers substantial survival and QALY gains for R/M HNSCC patients with an ICER lower than the Colombian willingness to pay making it a cost-effective treatment in Colombia.

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 de-novo 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 post-hoc 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.

Background: Ulcerative colitis (UC) imposes persistent clinical and economic burden on patient and healthcare management in Japan.

Purpose: To evaluate impact of prolonged oral corticosteroid (OC) use on healthcare resource utilization (HCRU) and treatment-related costs, and to assess discontinuation patterns of 5-amino salicylic acid (5-ASA), immunomodulators (IMs), and OCs in Japanese UC patients after biologic initiation.

Methods: Data were extracted from the Japan Medical Data Centre for patients diagnosed with UC with ≥1 prescription of OC with 5-ASA and/or IM, prior or at the index date (first biologic initiation) between 2016 and 2022, grouped by

Results: For all identified patients (N = 1,494; mean ± SD age: 38.6 ± 13.7 years; male: 65.3%), HCRU (inpatient and outpatient visits, length of stay, and procedures) per patient-year (PPY) declined after biologics initiation. Direct inpatient medical costs decreased throughout the study; outpatient costs increased from pre-index to the 1-year post-index period, followed by slight decrease in the 2-year and 3-year post-index. PPY costs of non-biologic UC-related drugs (OCs, 5-ASA, IMs) increased slightly during the post-index period. Overall, HCRU and costs dynamics were similar in patients with <180 days and those with ≥180 days of OC use. Patients with <180 days of OC use had shorter median time to OC discontinuation after biologic initiation compared with ≥180 days group (3.1 months vs 9.5 months).

Conclusions: Biologic initiation was associated with reduced HCRU and inpatient costs, with similar trends observed regardless of prolonged or shorter OC use duration.