ClinicoEconomics and Outcomes Research最新文献

Background: Anti-diabetic drugs help reduce premature diabetes-related mortality. However, their prices constitute a barrier to accessing diabetes treatment. The aim of this study was to analyze the price of antidiabetic drugs sold in private pharmacies in Côte d'Ivoire.

Methods: All anti-diabetic drugs listed in the Côte d'Ivoire drug registration database were selected, and wholesale prices were collected. The median price of insulins was calculated per 1000 units of active ingredient, and that of oral antidiabetics was calculated per 30 days of treatment, based on the maximum daily dose (MDD). Prices were compared with corresponding 2015 International Medical Products Price Guide by calculating the median price ratio.

Results: A total of 138 drugs were identified (123 oral antidiabetics and 15 insulins and analogs). Median prices ranged from US$39.5 for rapid-acting insulins to US$245.6 for analogs (Deglutec). Median prices for oral antidiabetics ranged from US$4.5 to US$11.3 for Metformin 500mg, from US$1 (Glibenclamid 5mg) to US$27.7 (Glimepiride 1mg) for Sulfonylureas, from US$13.9 (Sitagliptin 50mg) to US$26.3 (Vidagliptin 50mg) for DDP-4 inhibitors and from US$24.2 (Empaglifozin 25mg) to US$39.4 (Empaglifozin 10mg) for SGLT-2 inhibitors. The median price ratio ranged from 7.016 (Human regular) to 38.427 (NPH70/30) for insulins group and from 2.375 (Metformin 500mg) to 26.7 (Glimepirid 4mg) for oral antidiabetics.

Conclusion: Prices of antidiabetic drugs were higher than the international references prices. The development of a pricing policy emphasizing added value and price referencing, together with the development of a local generics industry, could guarantee prices that are accessible to the population and bearable by universal health coverage.

Objective: This study evaluated the long-term cost-effectiveness of once-weekly insulin icodec versus daily basal insulins in Chinese adults with type 2 diabetes mellitus (T2DM) across treatment backgrounds (insulin-naïve to basal-bolus users).

Methods: Using the UKPDS-OM2.1 model calibrated to ONWARDS trial data (1-5), we simulated lifetime outcomes over a 40-year horizon. Cost-utility analyses incorporated direct healthcare costs, complication utilities. Uncertainties were addressed using one-way and probabilistic sensitivity analyses. Scenario analyses explored pricing thresholds and adherence assumptions.

Results: Insulin icodec demonstrated cost-effectiveness versus degludec in insulin-naïve populations (ICER=$24974.29, below China's 3 times WTP threshold) but not versus glargine U100 (ICER=$45544.68) and once-daily basal insulin (ICER=$76877.59). For basal and basal-bolus insulin treated patients, insulin icodec does not offer long-term cost-effectiveness advantages over either insulin degludec and insulin glargine U100. One-way sensitivity analyses identified the simulation time horizon and discount rate as the most influential parameters, with probabilistic sensitivity analyses confirming the robustness of these findings. Scenario analyses demonstrated that insulin icodec's would become cost-effective compared to basal insulins when patients were willing to pay an additional $150 annually.

Conclusion: Insulin icodec offers cost-saving potential versus degludec in insulin-naïve T2DM patients. For basal and basal-bolus-treated patients, the clinical use of icodec needs to be critically evaluated for cost burden, and it is recommended that it be used preferentially in patients who are sensitive to the frequency of injections.

Background: The United States (US) HIV/AIDS strategy has targeted a 90% reduction in HIV infections by 2030. Whilst progress has been made in US HIV policy, the persistence of nearly 32,000 new infections annually highlights substantial barriers that still hinder effective treatment and the achievement of national targets. While the humanistic burden of HIV is well documented, there are broader economic effects on employment, disability and retirement. The objective of this study is to evaluate these economic gains when improving various HIV policies.

Methodology: This analysis adapts a published Markov model assessing the role of six key policy parameters related to diagnosis, pre-exposure prophylaxis (PrEP) uptake, treatment initiation, and treatment as prevention (TasP) on the HIV epidemic in the US. Improvements in these parameters were explored to estimate averted HIV infections over 50 years and, subsequently, the feasibility of achieving the 2030 target of a 90% reduction in HIV incidence.A fiscal economic framework was also applied, linking HIV cases and policy targets to productivity, tax revenue, transfer benefits, and healthcare costs incurred by the US government.

Results: Results were calculated for the general US population and for men who have sex with men (MSM), a subgroup experiencing a high HIV burden. For the general US population, policy improvements led to an average of 5,324 averted HIV infections annually over the 50-year horizon, corresponding to a total net annual fiscal gain of $397 million or $74,511 per averted infection. For the MSM subgroup, 911 infections were averted annually resulting in a net fiscal gain of $96 million, or $105,031 per averted infection.

Conclusion: This analysis demonstrates that the benefits of HIV policy are not limited to the healthcare setting and show how initial investments provide long-term benefits by preventing HIV transmission and the associated impact on individuals' labor market outcomes. While relying on assumptions and projections that may not capture all real-word complexities, fiscal analyses can provide a useful tool for policy evaluation that facilitate a holistic assessment of the wider costs and benefits that fall on governments as well as benefits of achieving policy targets.

Background: Interim results from the ESSENCE clinical trials indicate that 72-week treatment with high-dose subcutaneous (SC) semaglutide may result in metabolic dysfunction-associated steatohepatitis (MASH) resolution and improvements in fibrosis. As some patients with MASH have been prescribed SC semaglutide for the treatment of comorbid type 2 diabetes and/or obesity, this study assessed real-world 72-week treatment patterns among patients with MASH.

Methods: In a linked electronic health records (Veradigm Network EHR) and claims (Komodo Health) dataset, adults (≥18 years old) with a MASH diagnosis, who initiated treatment with SC semaglutide between 7/1/2018 and 6/30/2023, were identified. Other causes of liver disease (eg, viral hepatitis) or severe complications (eg, cirrhosis) were excluded. The study period included ≥52 weeks before and ≥72 weeks after the first SC semaglutide claim. A subgroup analysis was conducted among those who received the brand approved for 2.4 mg/week dosage (SC semaglutide 2.4) and among people at risk for MASH. Patient characteristics and treatment patterns are reported.

Results: This study identified 6,537 patients with MASH, who initiated treatment with SC semaglutide, 358 of whom received only the brand approved for 2.4 mg/week dosage. Patients were ~50 years old, and a majority were female. Non-persistence occurred in 68.4% of the overall SC semaglutide cohort and 78.5% of the SC semaglutide 2.4 subgroup. The mean time to non-persistence was 24.8 (19.3) and 20.1 (16.9) weeks in the SC semaglutide and SC semaglutide 2.4 groups, respectively. In the SC semaglutide 2.4 subgroup, 182 patients (50.8%) reached the recommended dosage of 2.4 mg/week, and 28 (7.8%) reached the recommended dosage within the first 16 weeks and sustained that dosage for ≥56 weeks. Trends were similar among patients at risk for MASH.

Conclusion: In a real-world setting, very few patients achieved the treatment regimen associated with MASH resolution and improvements in fibrosis.

Purpose: Treatment options for muscle-invasive bladder cancer (MIBC) are generally limited to radical cystectomy (RC) with or without neoadjuvant chemotherapy (NAC), adjuvant chemotherapy (AC), or immunotherapy. To contextualize emerging data on novel therapies, a systematic literature review (SLR) was conducted to evaluate real-world treatment effectiveness and economic and humanistic burden in patients with MIBC who received RC with or without systemic therapy.

Methods: Literature searches identified studies (published January 2018-June 2023) in adult patients with MIBC who received RC in the US, Germany, France, Italy, Spain, and the UK and reported effectiveness, economic burden, or humanistic burden.

Results: Of 4192 references identified, 61 reported real-world effectiveness, 12 economic burden, and 5 humanistic burden. No studies on immunotherapy were identified. Reported median overall survival (OS) ranged from 0.7 to 8.3 years with RC alone (n=9), 1.8-7.5 years with NAC+RC (n=6), and 1.5-6.1 years with RC+AC (n=7). Hospital stays for RC had median length of 5-10 days (n=5) and cost >$34,000 in the US. Health-related quality of life (HRQOL) was reported during 1 to 2 years post RC (n=5), but approximately 40% of patients continued to experience high psychosocial distress or low HRQOL.

Conclusion: Real-world studies have reported modestly longer survival with NAC or AC over RC alone; however, rates of disease recurrence were high and OS remained poor. Given this and the high economic burden for patients with MIBC, more effective therapies are needed.

Objective: Severe blepharospasm is a disabling neurological condition that significantly affects patients' quality of life. Botulinum toxin type A (BoNT-A) is considered the standard treatment due to its targeted therapeutic effect and fewer systemic side effects compared to oral medications. However, its high cost poses a barrier to access within Thailand's healthcare system. This study aimed to assess the budget impact of introducing BoNT-A treatment (Onabotulinumtoxin A and Abobotulinumtoxin A) compared with current oral medications for severe blepharospasm in Thailand.

Methods: A budget impact model was developed from the perspective of Thailand's healthcare system over a 5-year time horizon. The current scenario (oral medications use) was compared with a new scenario involving BoNT-A treatment. The costs considered included drug acquisition, outpatient visits, and accident-related injuries. The base-case assumed gradual uptake of BoNT-A (30% in year 1, 50% in year 2, and 100% from year 3). Sensitivity analyses explored full uptake from year 1, no dose sharing, and inclusion of injury-related costs.

Results: Excluding injury-related costs, the 5-year net budget impact (NBI) was 7.91 million THB (223,040 USD) for onabotulinumtoxin A and 7.27 million THB (205,064 USD) for abobotulinumtoxin A. Including injury-related costs reduced the NBI to 4.20 million THB (118,564 USD) and 3.57 million THB (100,588 USD), respectively. Without dose sharing, the NBI rose significantly, reaching 40.5 million THB (1.14 million USD) for abobotulinumtoxin A.

Conclusion: BoNT-A treatment increases healthcare costs, primarily due to drug costs. However, reduced injury costs and dose-sharing strategies may enhance affordability and support BoNT-A's inclusion in Thailand's National List of Essential Medicine.

Background: This study evaluated the clinical and economic outcomes associated with disruptive surgical bleeding (ie, hemorrhage/hematoma that complicates a procedure despite the use of hemostatic agents) among patients with bariatric, colorectal, spine, total hip arthroplasty (THA), and total knee arthroplasty (TKA) surgery.

Methods: Premier Healthcare Database patients aged ≥18 with one of the five procedures and hemostatic agent use between January 1-December 31, 2019, were included. Clinical and economic outcomes (ie, operating room time, 90-day all-cause inpatient readmission, in-hospital mortality, intensive care unit [ICU] admission/duration, ventilator use, hospital costs, and length of stay [LOS]) were compared between patients with and without disruptive surgical bleeding. Multivariable analyses adjusted for differences in baseline characteristics.

Results: Among 119,994 patients meeting inclusion criteria, 10.8% had disruptive surgical bleeding despite the use of hemostatic agents (bariatric surgery 5.4%, colorectal surgery 20.0%, spine surgery 11.0%, THA 11.5%, TKA 5.6%). Disruptive bleeding was associated with significantly longer operating room times for bariatric, colorectal, and spine surgery (incremental increases 42.3-62.4 minutes; p≤0.001), increased 90-day all-cause readmission risks for bariatric and spine surgery (incremental absolute risk increases 4.1% bariatric, 0.7% spine; both p=0.011), and increased inpatient mortality risk for all procedures except TKA (incremental absolute risk increases 0.2-55.0%; p≤0.001). ICU admission risks were increased for all procedures except TKA (incremental absolute risk increases 3.0-21.4%; p≤0.05), and ICU days were increased for bariatric, colorectal, and spine surgery (incremental increases 0.8-2.8 days; p≤0.001). Risks for ventilator use were higher for all procedures except THA (incremental absolute risk increases 3.5-25.2%; p≤0.05). Disruptive bleeding increased hospital costs (incremental increases $3,377-$23,346; p≤0.05) and LOS (incremental increases 1.0-4.9 days; p≤0.05) for all five procedures.

Conclusion: The clinical and economic burden of disruptive bleeding despite hemostatic agent use among patients with bariatric, colorectal, spine, THA, and TKA surgery was substantial, highlighting the need for improved surgical bleeding interventions.

Background: Prefilled syringes provide an opportunity to improve clinical safety and operational efficiency in hospital settings, especially amid mounting and ongoing challenges such as staff shortages, escalating drug costs, and increasing importance of safe medication administration. Despite these potential benefits, adoption remains limited. This study develops an economic model to assess the clinical and financial impacts of switching from conventional vial-and-syringe methods to prefilled syringes in United States (US) hospitals' intensive care units (ICU).

Methods: To address the gap between the potential benefits of prefilled syringes and their limited adoption, an economic model was developed to help decision-makers make informed choices based on the clinical and financial impact of switching to prefilled syringes in US ICUs. The model used peer-reviewed literature and hospital practices around the most utilized dosages in a US hospital. To illustrate model utility, three hypothetical ICU cases were developed: administering 30 daily doses of atropine 1mg/10mL, epinephrine 1mg/10mL, and ephedrine 25mg/10mL. Sensitivity analyses were performed to test model robustness.

Results: Switching to prefilled syringes resulted in annual cost savings of $729,912 for atropine, $786,502 for epinephrine, and $709,772 for ephedrine. The model estimated annual savings to be $696,551 due to fewer pADEs, along with savings of $53,411, $89,744 and $50,244 annually, due to unused drug wastage reduction for each drug, respectively. Hospital staff preparation time decreased by 255 hours for atropine, 285 for epinephrine and 227 hours for ephedrine per year. Sensitivity analyses confirmed the robustness of the model by varying drug wastage rates, with potential savings of up to $740,443, $795,894 and $724,757 for each drug, respectively, showing the model's adaptability across different ICU scenarios.

Conclusion: This model suggests prefilled syringes may help hospitals address pharmacy operational challenges by reducing preparation time, drug wastage, and pADEs. They offer a practical approach to support safer and more efficient medication delivery in clinical settings.

Purpose: Evaluate the prevalence and economic burden of adult spinal deformity (ASD) in a large, United States (US) commercial payer population.

Patients and methods: Patients aged 21-64 having an encounter with an ASD diagnosis from the Merative^TM Marketscan^® Commercial Databases 2016-2022 were included to calculate prevalence. The economic burden cohort included those with an outpatient ASD encounter and no spine surgery within the prior year. Continuous health plan enrollment was required for tracking. Expenditures are tabulated from the payer and societal perspectives (2023 US$) and rates of utilization and expenditures are reported overall and by service category.

Results: Annual ASD prevalence was 0.50%. 169,855 patients (46±13 years, 67.7% female) had an outpatient ASD encounter and were included in the economic burden cohort. Total spine-related payer expenditures averaged $7,619 (95% CI; $7,438, $7,800) per patient within 1 year - a payer burden of $3.8 million per 100,000 commercially-insured beneficiaries. Spine-related societal expenditures were $8,759 ($8,570, $8,947) per patient within 1 year - a societal burden of $6.2 billion among the US commercially-insured population. Nonoperative costs comprised 44% of the 1-year payer burden and 48% of the societal burden. While surgical treatment rates were low (3.5% fusions and 2.9% decompressions within 1 year), the associated economic burden was high (55% of payer burden, 51% of societal burden). The 2-year cumulative payer burden totaled $5.4 million per 100,000 commercially-insured beneficiaries, and the US commercially-insured societal burden totaled $8.9 billion.

Conclusion: The burden of both operative and nonoperative care for ASD is large. Considerable opportunity exists for development of improved nonoperative treatment modalities to increase the value of ASD care by reducing the need for continued nonoperative interventions of limited benefit and reducing the use of costly surgical interventions.

Purpose: Rapid molecular assays such as Xpert MTB/RIF and TB-LAMP accelerate pulmonary tuberculosis (TB) diagnosis but are more expensive than smear microscopy. This study provided an updated economic synthesis for presumptive adult pulmonary TB in high-burden settings, broadening the evidence from Xpert MTB/RIF to other WHO endorsed tests compared to conventional strategies.

Methods: Medline, Embase and Scopus were searched through March 2025. The strategy combined search terms related to molecular diagnostic tests, pulmonary tuberculosis, and economic evaluation study designs. Full economic evaluations comparing molecular tests with smear microscopy, culture or passive case-finding were eligible. Two reviewers independently screened articles, extracted data, and adjusted costs to 2025 US dollars (USD) using average exchange rates. Reporting quality was appraised using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist. Due to heterogeneity in evaluation criteria, model structures, time horizons, and outcome measures, meta-analysis were not feasible. Therefore, results were synthesized narratively, and incremental cost-effectiveness ratios (ICERs) were contextualized against country-specific cost-effectiveness thresholds to enable meaningful cross-study interpretation.

Results: Eight studies conducted in low- and middle-income countries with high TB burdens were included. All evaluated Xpert MTB/RIF and the Thai studies also examined TB-LAMP. Five studies reported cost per disability-adjusted life years (DALYs) averted or quality-adjusted life years (QALYs) gained, while three used TB cases detected or years of life saved (YLS). CHEERS reporting quality was high (median is 23/28 items). Reported ICERs for molecular testing were either cost-saving or highly cost-effective compared with country-specific thresholds. Probabilistic sensitivity analyses (five studies) indicated ≥90% probability of cost-effectiveness in four studies and 6% in one.

Conclusion: Recent evidence supports the cost-effectiveness and cost-saving of Xpert MTB/RIF and TB-LAMP for diagnosing adult pulmonary TB. Policymakers should prioritize reducing cartridge costs and implementing models that capture patient-level benefits to maximize economic benefits.