Journal of Medical Economics最新文献

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: The aim of this paper was to determine a claims-based timeline of unilateral elbow fracture recovery including the effect of comorbidities and post-treatment complications on recovery.

Methods: This study utilized data from the IBM MarketScan database (2015-2018) to assess costs and recovery timelines after unilateral elbow fracture treatment. All costs are reported in U.S. dollars and reflect prices during the 2015-2018 period. Costs examined included: (i) index surgery/treatment, (ii) complication surgery, (iii) revision or salvage surgeries, (iv) non-operative hospitalization, (v) motion restoring surgery and (vi) additional elbow-related outpatient surgery. Costs of related physician visits, physical/occupational therapy and diagnostic radiology were also incorporated. The impact of comorbidities-diabetes, obesity, peripheral vascular disease, and cardiovascular disease-was evaluated. Additionally, data on re-hospitalizations, with or without further surgery, were analyzed to understand complications after the initial treatment. Perioperative complications including joint fibrosis/contracture, infection, and pulmonary embolus were also reported.

Results: Index surgery/treatment median cost and length of post-treatment recovery (from index surgery/treatment to last physical/occupational therapy claim) was $4,494 ($872-$10,444) and 102.5 days (36-480 days), respectively. A total of 59% of patients completed their post-treatment period in 6 months with 41% of patients taking longer. Patients who required a complication surgery had median recovery times and costs that increased three- and seven-fold, respectively, in comparison to those without complication surgeries. Comorbidities added 66-113 days to recovery. Peripheral and cardio-vascular disease coincided with 1.7-3 times higher post-treatment costs.

Conclusion: Comorbidities and complications following elbow fracture treatment lead to substantial increases in both costs and recovery durations. Understanding the typical recovery timeline after elbow fracture treatment, as well as the variations in outlier patients, can assist in optimizing recovery management and guiding appropriate interventions.

Background: Barth syndrome (BTHS) is an ultra-rare, X-linked genetic disorder for which there is limited economic data. Because compiling such data that target rare indications is difficult, we assessed real-world data to increase understanding of the cost of BTHS based on disease burden and health care resource utilization (HCRU).

Methods: A search of the published medical literature identified individual case studies and registry data used to assess the burden of disease and potential costs associated with BTHS, including the potential ability of developing treatments to positively impact those costs. In addition, a claims database analysis was conducted to assess HCRU and associated costs in the United States for patients with BTHS. These real-world data were summarized and compared with registry data.

Results: The diagnostic journey for patients with BTHS is difficult, with the majority of affected individuals not receiving a diagnosis until after the development of cardiomyopathy or until a family member is diagnosed, even in those who are symptomatic. Of the living individuals known to have BTHS, a high proportion experience heart failure, with >15% requiring heart transplantation. Data extrapolated during the claims database analysis demonstrated that patients with diagnostic codes linked to BTHS are complex, with a high percentage of complications, necessitating a high level of HCRU and associated costs of care in the inpatient setting. Specifically, National Inpatient Sample hospital cost per claim was $32,702 and the Kids' Inpatient Database hospital cost per claim was $62,596.

Conclusions: Health economic evaluations for rare diseases are scarce. With a noteworthy absence of pharmacoeconomic evidence, utilization of combined clinical case report data from the medical literature, along with registry and claims datasets, demonstrate that BTHS is a costly disease associated with high disease burden and excessive HCRU. Forthcoming treatments (e.g. elamipretide) have the potential to reduce the high disease burden/HCRU.

Background: In India, influenza presents a significant burden of disease to the population at large. Influenza vaccination is a cost-effective means of preventing the disease, with quadrivalent vaccines providing broader protection against influenza by covering more strains, but generally at a higher list price, than older trivalent vaccines.

Research design and methods: Broadly, the economic burden of influenza can be categorized into treatment and productivity costs. We undertake a cost-benefit analysis to determine the cost-effectiveness of a quadrivalent influenza vaccine versus no vaccination amongst healthy adults and pregnant women from the perspective of both employers and the Employees' State Insurance Company (ESIC) in India.

Results: Administration of the quadrivalent vaccine results in better outcomes in terms of productive days worked and disease infection rates amongst the target populations whilst incurring lower overall health resource utilization costs than when no vaccine is employed. In healthy adults, we estimate a saving of INR 13,730 per case averted from the corporate perspective and a saving of INR 11,211 from the ESIC perspective. Deterministic and probabilistic sensitivity analyses indicate that there is a high probability that these results hold true given the uncertainty in our model's input parameters.

Conclusions: We find that for healthy adults and pregnant women, receiving a quadrivalent influenza vaccine is dominant over no vaccination in terms of cost-effectiveness that is, it results in better health outcomes at a lower overall cost.

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.

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.