ClinicoEconomics and Outcomes Research最新文献

Purpose: Three prenatal screening protocols are currently used in Thailand to prevent severe thalassemia in newborns; however, economic evaluation data to guide the selection of the most cost-effective strategy remain limited. This study aimed to conduct the cost-effectiveness of these screening strategies to support policy and clinical decision-making.

Patients and methods: A decision-tree model was employed to evaluate the costs and outcomes associated with three prenatal screening algorithms: 1) a protocol starting with mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), and the dichlorophenol indophenol precipitation (DCIP) test for pregnancies; 2) a protocol starting with MCV/MCH and hemoglobin (Hb) typing for pregnancies; and 3) a protocol starting with MCV/MCH and DCIP testing for couples based on a societal perspective. Results were presented as incremental cost-effectiveness ratios (ICERs) to compare the three prenatal screening strategies. Both one-way and probabilistic sensitivity analyses were performed to account for uncertainties in the parameters used.

Results: From a societal perspective, Strategies 2 and 3 were found to be cost-effective compared to Strategy 1. Screening 10,000 couples under Strategies 2 and 3 could prevent 60 and 49 additional cases of severe thalassemia, respectively, compared to Strategy 1. The ICERs for Strategies 2 and 3 were approximately 141,863 Baht (4,023 United States Dollar, USD) and 97,996 Baht (2,779 USD) per severe thalassemia case prevented, respectively. The most sensitive parameter for comparing Strategy 2 with Strategy 1 was the specificity of prenatal diagnosis for α-thalassemia.

Conclusion: Strategy 3 is the most cost-effective approach in Thailand. However, Strategy 2 may be implemented in hospitals with the capacity to perform Hb typing. These results offer significant value to policymakers by presenting strong evidence that could inform adjustments to the reimbursement framework in Thailand's Universal Health Coverage benefit package, ultimately enhancing thalassemia management and prenatal care.

Purpose: The study investigated the potential association between commercial blenderized tube feeding formulas (CBTF) or plant-based standard tube feeding formulas (STD-TF), and healthcare resource utilization (HCRU) and costs, in children and adults receiving enteral nutrition (EN) in a post-acute care setting.

Patients and methods: United States insurance claims were retrospectively analyzed to evaluate HCRU and costs for visits to emergency department (ED), inpatient, outpatient, urgent care, and other services at 12 weeks post hospital discharge. Between-cohort comparisons were performed for proportions of patients with claims for each place of service, mean number of visits, and unadjusted and adjusted costs.

Results: Significantly fewer children receiving CBTF vs plant-based STD-TF required inpatient services (16% vs 35%), urgent care (3% vs 10%), ED (15% vs 28%), and other facilities (10% vs 22%) (p < 0.001 for each comparison), with a lower mean number of visits to any healthcare service reported (28 vs 96; p < 0.001). In adults, significantly fewer patients in the CBTF cohort vs the plant-based STD-TF cohort required inpatient (23% vs 38%; p = 0.003) and other services (13% vs 27%; p < 0.001), with a corresponding lower mean number of visits to any healthcare service (24 vs 79; p < 0.001). Total unadjusted costs were significantly lower in both children and adults receiving CBTF compared with plant-based STD-TF (children: $140,962 vs $1,060,345; adults: $166,591 vs $820,905; each p < 0.001). Cost savings associated with CBTF were maintained when costs were adjusted for age, sex, and Charlson's comorbidity index. Children and adults receiving CBTF had significantly lower total adjusted costs compared with those receiving plant-based STD-TF (children: $222,735 vs $965,451; adults: $258,460 vs $919,060 each p ≤ 0.001).

Conclusion: The use of CBTF was associated with lower HCRU and costs both in children and adults receiving EN in post-acute care settings. To our knowledge, this is the first large real-world study comparing these formulas in post-acute care across both populations. Although the retrospective design and lack of adjustment for all potential confounders preclude conclusions about causation, these findings may guide formula selection based on potential economic impact.

Introduction: The World Health Organization (WHO) has advocated for multiple micronutrient supplementation (MMS) for pregnant women since 2016 to mitigate the risk of various complications. As a country considering investment in MMS, Indonesia requires an assessment of the cost-effectiveness analysis and priority setting in the transition from IFA to MMS, which is essential in the decision-making process.

Methods: An open-access online modelling tool was used to estimate the cost-effectiveness of MMS (ICERs per DALY averted) at national and sub-national levels (38 provinces) in Indonesia. The results were then prioritized via a cost-effectiveness league table, with a deterministic sensitivity analysis testing the robustness of the results.

Results: Implementing MMS under 44% and 100% coverage scenarios has the potential to avert 54,897 and 124,766 DALYs in Indonesia, respectively, and produced an equivalent incremental cost-effectiveness ratio (ICER) value of USD 10 per DALY averted, which is regarded as highly cost-effective. Since costs and benefits scale linearly with coverage, ICER per DALY averted stays unchanged. The MMS program is recommended to be prioritized in 18 provinces having favorable ICERs, which are Southwest Papua, Highlands Papua, Bali, West Java, South Kalimantan, North Maluku, West Papua, Aceh, North Sumatra, Central Java, Central Kalimantan, West Sulawesi, Maluku, Yogyakarta, East Java, North Kalimantan, South Sulawesi, and South Papua. Related to the sensitivity analysis, the cost of MMS and IFA are the most significant variables influencing the ICER value.

Conclusion: The findings show that the implementation of MMS, in comparison to IFA, produces a highly cost-effective outcome in 44% and 100% scenarios. The MMS implementation strategy may initiate with a 44% coverage scenario across 18 prioritized provinces, subsequently expanding to achieve 100% coverage for all pregnant women in Indonesia.

Purpose: The EuroQoL-five dimensions-three levels (EQ-5D-3L) utility values vary across regions, hence become non-interchangeable. This study details a methodology to adjust EQ-5D-3L utility values from the published United States (US) scores for use in the United Kingdom (UK) when individual-level patient data (IPD) are inaccessible.

Methods: Linear and polynomial regression models were fitted to map the US-derived EQ-5D-3L health state utility weights (HSUWs) into UK values. Model fit was evaluated via visual inspection and coefficient of determination (R ²). The best-fit model was used to develop a utility-adjustment algorithm. Data for external validation of the algorithm were collected through a targeted literature review, which identified studies reporting mean EQ-5D-3L utilities from both the US and UK. The coefficients of the best-fit model were validated against the published mean US and UK utilities, and the algorithm was validated by comparing published and predicted mean UK utilities.

Results: The linear model confirmed linearity between US and UK HSUWs, and was the best-fit over the polynomial model, both visually and with R ² (0.9574 vs 0.9576). Eleven publications (N = 93,625 participants) reporting the mean US and UK EQ-5D-3L utilities based on the valuation of EQ-5D-3L responses were included for validation. The linearity between the published mean US and UK EQ-5D-3L utilities was deemed valid (R ² = 0.9955) with similar slopes (1.45 vs 1.39) and intercepts (-0.43 vs -0.38) between the linear model and the published utility-based line. The algorithm (Utility^UK = [-0.3813 + 1.3904 × Utility^US]) predicted the mean UK utilities, which marginally differed from the reported mean UK utilities, confirming its validity.

Conclusion: A robust method was developed to adjust US-derived EQ-5D-3L utilities and reflect UK preferences. This method helps predict and transpose utilities from the literature across countries as a supporting tool when IPD are unavailable, which is necessary when adapting cost-effectiveness models to different settings.

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.