Lower Incremental Cost-effectiveness Ratio Research Articles

Inter-facility transfers for emergency obstetrical and neonatal care in rural Madagascar: a cost-effectiveness analysis

ContextThere is a substantial lack of inter-facility referral systems for emergency obstetrical and neonatal care in rural areas of sub-Saharan Africa. Data on the costs and cost-effectiveness of such systems...

Early lowering of LDL-C is a more cost-effective strategy than lowering LDL-C later in life: a cost-effectiveness analysis using Mendelian Randomisation

Abstract Background Early control of low-density lipoprotein-cholesterol (LDL-C) is an effective way to reduce the risk of coronary heart disease (CHD). However, a comprehensive assessment of cost-effectiveness of different lipid-lowering strategies (LLS) for primary prevention of CHD across different ages is lacking. Objective To assess the cost-effectiveness of four different LLS initiated at ages 30, 40, 50, and 60 years on the primary prevention of CHD from the UK National Health Service perspective. Design, setting and participants: We developed a microsimulation model, based on the causal effect of LDL-C on myocardial infarction (MI) or coronary death derived from Mendelian randomisation, comparing initiation of an LLS to current standard of care (control). Data were primarily drawn from 458,727 participants of the UK Biobank study with available data on date of birth, LDL-C, and who were free of MI at first assessment (between 2006 and 2010), with follow-up until 2021. Interventions: The four LLS were: 1) low/moderate intensity statins; 2) high intensity statins; 3) low/moderate intensity statins and ezetimibe; and 4) inclisiran. Main outcomes measures: The incremental cost-effectiveness ratio (ICER), defined as the incremental healthcare costs divided by the incremental quality-adjusted life years (QALYs) for each LLS compared to control, with 3.5% annual discounting. Results The most effective intervention, low/moderate intensity statins and ezetimibe, was projected to lead to a gain in QALYs of 0.068 per person at age 30 and 0.026 at age 60 compared to control (Figure). The age of intervention with the lowest ICER was age 40, with ICERs of £2,524 (95% uncertainty interval: 1,383, 3,796), £4,466 (3,163, 6,134) £11,118 (8,632, 14,177), and £1,405,123 (1,117,1113, 1,789,012) per QALY gained for strategies 1-4 from age 40 years, respectively (Table). Conclusions LDL-C lowering from early ages is a more cost-effective strategy than late intervention. The approach to primary prevention of CHD may improve with a shift to early and sustained lowering of LDL-C. Figure – Results of 1,000 probabilistic sensitivity analyses presented in a common cost-effectiveness plane, by age of intervention. Inclisiran excluded due to very high costs. Solid line: £20,000 per quality-adjusted life year (QALY) willingness-to-pay threshold; dashed line: £30,000 per QALY willingness-to-pay threshold. Any dot to the right of these lines is considered cost-effective. Table – Summary of LLS interventions by age of intervention. Abbreviations: MI – Myocardial infarction; YLL – Years of life lived; QALYs – Quality-adjusted life-years; ICER – Incremental cost-effectiveness ratio.FigureTable

Cost-effectiveness analysis of atezolizumab as adjuvant treatment of patients with stage II-IIIA non-small cell lung cancer, PD-L1+≥50% of tumor cells in France: A modeling study

IntroductionThe objective of this study was to assess the cost-effectiveness of atezolizumab versus best supportive care (BSC) as adjuvant treatment following resection and platinum-based chemotherapy for patients with stage II-IIIA non-small cell lung cancer (NSCLC) whose tumours have a programmed death-ligand 1 (PD-L1) expression ≥ 50% of tumour cells and excluding those with ALK/EGFR mutations, from a French collective perspective. Material and MethodsA five state Markov model over a 20-year time horizon was considered, including disease-free survival (DFS11Abbreviations: AE: adverse event; ALK: anaplastic lymphoma kinase; BSC: best supportive care; DFS: disease-free survival; DSA: deterministic sensitivity analyses; EFGR: epidermal growth factor receptor; EQ-5D: EuroQol-5 dimension; HAS: Haute Autorité de Santé; ICs: immune cells; ICER: incremental cost-effectiveness ratio; IgG1: immunoglobulin G1; IV: intravenous; KM: Kaplan-Meier; LY: life-year; NSCLC: non-small cell lung cancer; OS: overall survival; PD-L1: programmed death-ligand 1; PSA: probabilistic sensitivity analysis; QALY: quality-adjusted life-year; QoL: quality of life; TCs: tumor cells.) from IMpower010 trial, three progression states (locoregional recurrence, first and second-line metastatic recurrence) and death. Utilities, quality-adjusted life year (QALY) decrements associated to adverse events, costs, resource use, and transition probabilities were considered in the model. These inputs were sourced from IMpower010 trial, literature, and clinical experts’ opinion. Model uncertainty was assessed through deterministic, probabilistic sensitivity analyses and scenario analyses. ResultsAtezolizumab was associated with a QALY gain of 1.662, mainly driven by additional time spent in the DFS state, and a life-year gain of 2.112 years. The incremental cost-effectiveness ratio (ICER) for atezolizumab versus BSC was €21,348/QALY gained. The sensitivity analyses highlighted that uncertainty within the model had limited impact on results. Changing the DFS survival curves to other plausible distributions produced ICERs below €20,000/QALY. Introducing an increasing proportion of cured patients (91.5%) from year two to year five reduced the ICER to €13,083/QALY, while including a loss of efficacy at year two in the atezolizumab treatment arm increased the ICER to €33,755/QALY. DiscussionAtezolizumab as adjuvant treatment in stage II-IIIA NSCLC resected patients with PDL1 ≥ 50% and without ALK/EGFR mutations has a lower ICER than other oncology drugs in France and a similar ICER to other adjuvant treatment in oncology.

Effectiveness and cost-effectiveness of human papillomavirus vaccination strategies among men who have sex with men in China: a modeling study.

The health and economic benefits of human papillomavirus (HPV) vaccination targeted at men who have sex with men (MSM) in developing settings have been rarely assessed. This study aimed to evaluate the effectiveness and cost-effectiveness of different HPV vaccination strategies among MSM in China. A Markov model was developed to simulate HPV transmission dynamics among a total of 30.73 million MSM in China. The corresponding natural history included 6 states: susceptible, infected with low-risk subtypes, high-risk subtypes, anogenital warts and anal cancer, and deaths from anal cancer. MSM were divided into three age groups with cut-off points of 27 and 45 years. Alternative vaccination strategies were built by allocating bivalent, quadrivalent, nine-valent, or no vaccine to each of the groups. We generated the prevented infections and deaths by vaccination compared with baseline (no vaccination) and calculated incremental cost-effectiveness ratios (ICERs) to determine the optimal strategy. The model showed that in 10 years, at baseline, the existing cases of anogenital warts would reach 5,464,225 (IQR, 4,685,708-6,174,175); that of anal cancer would reach 1,922.95 (1,716.56-2,119.93), resulting in 940.55 (732.27-1,141.87) deaths. Under 50% vaccination coverage among one age group, the prevented cases of anogenital warts were maximized with quadrivalent vaccines allocated to MSM aged 27-45 years; that of anal cancer were maximized when offering nine-valent vaccines to the same group. Under 50% vaccination coverage among all groups, the lowest ICER (34,098.09 USD/QALY, 31,146.54-37,062.88) was reached when only quadrivalent vaccines were provided. Based on this strategy, when the annual vaccination rate increased by 30%, the ICER (33,521.75 USD/QALY, 31,040.73-36,013.92) would fall below three times China's per capita GDP. When the vaccine price decreased by 60%, the ICER was reduced to 7,344.44 USD/QALY (4,392.89-10,309.23), indicating good cost-effectiveness taking China's per capita GDP as a threshold. HPV vaccination can effectively reduce the prevalence and mortality of related diseases among MSM in China, especially quadrivalent vaccines for anogenital warts and nine-valent vaccines for anal cancer. MSM aged 27-45 years were the optimal group for vaccination. Annual vaccination and appropriate adjustment of vaccine price are necessary to further improve the cost-effectiveness.

Cost-effectiveness of screening for chronic kidney disease in the general adult population: a systematic review.

Chronic kidney disease (CKD) is a significant public health problem, with rising incidence and prevalence worldwide, and is associated with increased morbidity and mortality. Early identification and treatment of CKD can slow its progression and prevent complications, but it is not clear whether CKD screening is cost-effective. The aim of this study is to conduct a systematic review of the cost-effectiveness of CKD screening strategies in general adult populations worldwide, and to identify factors, settings and drivers of cost-effectiveness in CKD screening. Studies examining the cost-effectiveness of CKD screening in the general adult population were identified by systematic literature search on electronic databases (MEDLINE OVID, Embase, Cochrane Library and Web of Science) for peer-reviewed publications, hand-searched reference lists and grey literature of relevant sites, focusing on the following themes: (i) CKD, (ii) screening and (iii) cost-effectiveness. Studies comprising health economic evaluations performed for CKD screening strategies, compared with no CKD screening or usual-care strategy in adult individuals, were included. Study characteristics, model assumptions and CKD screening strategies of selected studies were identified. The primary outcome of interest is the incremental cost-effectiveness ratio (ICER) of CKD screening, in cost per quality-adjusted life year (QALY) and life-year gained (LYG), expressed in 2022 US dollars equivalent. Twenty-one studies were identified, examining CKD screening in general and targeted populations. The cost-effectiveness of screening for CKD was found to vary widely across different studies, with ICERs ranging from $113 to $430 595, with a median of $26 662 per QALY and from $6516 to $38 372, with a median of $29 112 per LYG. Based on the pre-defined cost-effectiveness threshold of $50 000 per QALY, the majority of the studies found CKD screening to be cost-effective. CKD screening was especially cost-effective in those with diabetes ($113 to $42 359, with a median of $27 471 per QALY) and ethnic groups identified to be higher risk of CKD development or progression ($23 902 per QALY in African American adults and $21 285 per QALY in Canadian indigenous adults), as indicated by a lower ICER. Additionally, the cost-effectiveness of CKD screening improved if it was performed in older adults, populations with higher CKD risk scores, or when setting a higher albuminuria detection threshold or increasing the interval between screening. In contrast, CKD screening was not cost-effective in populations without diabetes and hypertension (ICERs range from $117 769 to $1792 142, with a median of $202 761 per QALY). Treatment effectiveness, prevalence of CKD, cost of CKD treatment and discount rate were identified to be the most common influential drivers of the ICERs. Screening for CKD is especially cost-effective in patients with diabetes and high-risk ethnic groups, but not in populations without diabetes and hypertension. Increasing the age of screening, screening interval or albuminuria detection threshold, or selection of population based on CKD risk scores, may increase cost-effectiveness of CKD screening, while treatment effectiveness, prevalence of CKD, cost of CKD treatment and discount rate were influential drivers of the cost-effectiveness.

Economic evaluation of supplemental breast cancer screening modalities to mammography or digital breast tomosynthesis in women with heterogeneously and extremely dense breasts and average or intermediate breast cancer risk in US healthcare

ObjectiveTo evaluate the cost-effectiveness of supplemental breast imaging modalities for women with heterogeneously and extremely dense breasts and average or intermediate risk of breast cancer (BC) in the USA, and analyze capacity requirements for supplemental magnetic resonance imaging (MRI) and contrast-enhanced mammography (CEM).MethodsClinical and economic outcomes for supplemental imaging modalities including full- and abbreviated-protocol MRI (Fp-MRI, Ab-MRI), CEM, and ultrasound (U/S) as add-on to x-ray mammography (XM) or digital breast tomosynthesis (DBT), were compared to XM or DBT alone, in a decision tree linked to a Markov chain validated by comparison with a microsimulation analysis. A Delphi panel supplemented model input parameters from the literature. A capacity model evaluated the number of additional daily scans and scanners required for Fp-MRI and CEM.ResultsCompared to XM or DBT alone, all supplemental imaging protocols were cost-effective. Both Fp- and Ab-MRI, and to a lesser extent CEM and U/S, yielded superior clinical outcomes to XM or DBT. Compared to XM alone, U/S and Ab-MRI had the lowest incremental cost-effectiveness ratios (ICER). For U/S, the ICER was $23,394 for the average-risk population and $13,241 for the intermediate-risk population. For CEM, the ICER was $38,423 and $23,772, respectively. For the extremely dense subpopulation with intermediate risk, supplemental screening requirements could be accommodated by conducting one Fp-MRI scan per day per existing general scanner.ConclusionsWhile ultrasound had the lowest ICER, MRI and CEM demonstrated the best clinical outcomes, compared to XM or DBT alone for women with dense breasts and intermediate and high risk. Existing MRI scanner capacity has the potential to meet most of the supplemental screening needs of this population.

Pegcetacoplan for Treating Paroxysmal Nocturnal Haemoglobinuria: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.

As part of the Single Technology Appraisal (STA) process, the UK National Institute for Health and Care Excellence (NICE) invited Apellis Pharmaceuticals/Sobi to submit evidence for the clinical and cost effectiveness of pegcetacoplan versus eculizumab and pegcetacoplan versus ravulizumab for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults whose anaemia is uncontrolled after treatment with a C5 inhibitor. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned as the Evidence Review Group (ERG). The company pursued a low incremental cost-effectiveness ratio (ICER) Fast Track Appraisal (FTA). This was a form of STA processed in a shorter time frame and designed for technologies with company base-case ICER < £10,000 per quality-adjusted life-year (QALY) gained and most plausible ICER < £20,000 per QALY gained. This article summarises the ERG's review of the company's evidence submission, and the NICE Appraisal Committee's (AC's) final decision. The company presented clinical evidence from the PEGASUS trial that assessed the efficacy of pegcetacoplan versus eculizumab. At Week 16, patients in the pegcetacoplan arm had statistically significantly greater change from baseline in haemoglobin levels and a higher rate of transfusion avoidance than patients in the eculizumab arm. Using the PEGASUS trial and Study 302 data (a non-inferiority trial that assessed ravulizumab versus eculizumab), the company conducted an anchored matching-adjusted indirect comparison (MAIC) to indirectly estimate the efficacy of pegcetacoplan versus ravulizumab. The company identified key differences between trial designs and populations that could not be adjusted for using anchored MAIC methods. The company and ERG agreed that the anchored MAIC results were not robust and should not inform decision making. In the absence of robust indirect estimates, the company assumed that ravulizumab had equivalent efficacy to eculizumab in the PEGASUS trial population. Results from the company base-case cost-effectiveness analysis showed that treatment with pegcetacoplan dominated eculizumab and ravulizumab. The ERG considered that the long-term effectiveness of pegcetacoplan was uncertain and ran a scenario assuming that after 1 year the efficacy of pegcetacoplan would be the same as eculizumab; treatment with pegcetacoplan continued to dominate eculizumab and ravulizumab. The AC noted that treatment with pegcetacoplan had lower total costs than treatment with eculizumab or ravulizumab because it is self-administered and reduces the need for blood transfusions. If the assumption that ravulizumab has equivalent efficacy to eculizumab does not hold, then this will affect the estimate of the cost effectiveness of pegcetacoplan versus ravulizumab; however, the AC was satisfied that the assumption was reasonable. The AC recommended pegcetacoplan as an option for the treatment of PNH in adults who have uncontrolled anaemia despite treatment with a stable dose of a C5 inhibitor for ≥ 3 months. Pegcetacoplan was the first technology recommended by NICE via the low ICER FTA process.

First-line systemic treatment strategies for unresectable hepatocellular carcinoma: A cost-effectiveness analysis.

Oral multikinase inhibitors and immune checkpoint inhibitors (ICIs) are effective for treating advanced hepatocellular carcinoma (aHCC) but may increase cost. This study compared the cost-effectiveness of oral multikinase inhibitors and ICIs in the first-line treatment of patients with aHCC. A three-state Markov model was established to study the cost-effectiveness of drug treatment from the perspective of Chinese payers. The key outcomes in this study were total cost, quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER). The total costs and QALYs of sorafenib, sunitinib, donafenib, lenvatinib, sorafenib plus erlotinib, linifanib, brivanib, sintilimab plus IBI305, and atezolizumab plus bevacizumab were $9070 and 0.25, $9362 and 0.78, $33,814 and 0.45, $49,120 and 0.83, $63,064 and 0.81, $74,814 and 0.82, $81,995 and 0.82, $74083 and 0.85, and $104,188 and 0.84, respectively. The drug regimen with the lowest ICER was sunitinib ($551 per QALY), followed by lenvatinib ($68,869 per QALY). For oral multikinase inhibitors, the ICER of lenvatinib, sorafenib plus erlotinib, linifanib and brivanib compared with sunitinib was $779576, $1534,347, $1768,971, and $1963,064, respectively. For ICIs, sintilimab plus IBI305 is more cost effective than atezolizumab plus bevacizumab. The model was most sensitive to the price of sorafenib, the utility of PD, and the price of second-line drugs. For oral multikinase inhibitors, the order of possible treatment options is sunitinib > lenvatinib > sorafenib plus erlotinib > linifanib > brivanib > donafenib. For ICIs, the order of possible treatment options is sintilimab plus IBI305 > atezolizumab plus bevacizumab.

Systematic Review of Cost-Effectiveness Analysis for Surgical and Neurostimulation Treatments for Drug-Resistant Epilepsy in Adults

Surgical and neurostimulator treatments are effective for reducing seizure burden in selected individuals living with drug-resistant epilepsy (DRE). We aimed to determine the presence and key model determinants for cost-effectiveness of these interventions, compared with medical management alone, to assist with decisions about resource allocation. A systematic literature search was conducted on June 1, 2022, using MEDLINE, EMBASE, the NHS Economic Evaluation Database, and the Cost-Effectiveness Analysis database. Included studies were economic evaluations in adult DRE cohorts, comparing surgical and neurostimulator treatments (vagus nerve stimulation [VNS], responsive neurostimulation [RNS], and deep brain stimulation [DBS]) vs medical management alone and reporting cost-benefit analysis, cost-utility, or cost-effectiveness. Exclusion criteria were studies with pediatric cohorts and those published in a language other than English. Three independent reviewers screened, extracted, and assessed data against the Consolidated Health Economic Evaluation Reporting Standards checklist, and a fourth reviewer adjudicated discrepancies. Ten studies met inclusion criteria. Seven studies evaluated epilepsy surgery, and 3 evaluated neurostimulation treatments. All relevant studies established that epilepsy surgery is a cost-effective intervention compared with medical management alone, for quality-adjusted life-years and seizure freedom at 2 and 5 years. All relevant studies found neurostimulator treatments to be potentially cost-effective. The incremental cost-effectiveness ratio (ICER), with lower ICER indicating greater cost-effectiveness, was reported for 9 studies and varied between GBP £3,013 and US $61,333. Cost adaptation revealed ICERs from US $170 to US $121,726. Key model determinants included, but were not limited to, improved surgical outcomes and quality of life, reduced surgical and presurgical evaluation costs, higher rates of surgical eligibility after referral and evaluation, epilepsy subtype, less expensive neurostimulator devices with improved longevity, and cost analysis strategy used in the analysis. There is consistent evidence that epilepsy surgery is a cost-effective treatment of eligible candidates with DRE. Limited evidence suggests that VNS, RNS, and DBS may be cost-effective therapies for DRE, although more health economic evaluations alongside prospective clinical trials are needed to validate these findings. PROSPERO CRD42021278436.

Cost-Effectiveness of Sacubitril/Valsartan Compared with Enalapril in Patients with Heart Failure with Reduced Ejection Fraction: A Systematic Review.

To assess the cost-effectiveness of sacubitril/valsartan compared with enalapril in patients with heart failure with reduced ejection (HFrEF). A systematic literature search was conducted searching in major electronic databases from inception to January 1, 2021. All relevant full economic evaluation studies of sacubitril/valsartan versus enalapril for the treatment of patients with HFrEF were identified using ad hoc search strategies. Mortality, hospital admissions, quality-adjusted life years (QALYs), life-years (LYQs), annual drug costs, total lifetime costs, and incremental cost-effectiveness ratio (ICER) were considered as the outcomes. The quality of the included studies was assessed using the CHEERS checklist. This study was conducted and reported in accordance with the "Preferred Reporting Items for Systematic Reviews and Meta-Analyses" (PRISMA) guidelines. The initial search yielded a pool of 1026 articles, of which 703 unique articles were screened, 65 full-text articles were assessed for eligibility and 15 studies finally included in the qualitative synthesis. Studies show that sacubitril/valsartan reduces mortality and hospitalization rate. The mean of death risk ratio and hospitalization were computed at 0.843 and 0.844, respectively. Sacubitril/valsartan produced higher annual and total lifetime costs. The lowest and highest lifetime costs for sacubitril/valsartan were found in Thailand ($4,756) and Germany ($118,815), respectively. The lowest ICER was reported in Thailand ($4857/QALY) and the highest in the USA ($143,891/QALY). Sacubitril/valsartan is associated with better outcomes and may be cost-effective compared to enalapril for the management of HFrEF. However, in developing countries such as Thailand, sacubitril-valsartan costs must be reduced to yield an ICER below the threshold.

A Systematic Review to Compare Costs and Outcomes of Pharmacoeconomic Evaluations of Conventional and Biosimilars G-CSFs

Introduction: The presence of biosimilars in the field of oncology is regarded as a key approach to attain sustainable healthcare. Granulocyte colony-stimulating factor (G-CSF) is a drug mostly prescribed after chemotherapy to avoid neutropenia. There are several G-CSF biosimilars approved to help reduce the significant economic burden on healthcare stakeholders through cost saving and to increase patient access. To date, no systematic assessment of the pharmacoeconomic evaluation of G-CSF biosimilars has been performed. The aim of this study is to synthesize evidence from economic evaluations (EEs) of G-CSF biosimilars published articles to provide essential data for involved stakeholders and policy makers. Materials and methods: PRISMA-guided systematic searches of PubMed, Scopus and EMBASE databases were conducted. Search was done up to April 2023 using predefined keywords. Articles were screened for relevant publications about EEs of G-CSF biosimilars that were used as prophylactic and or as a treatment for chemotherapy-induced neutropenia (CIN). We included articles for cost-effectiveness analysis (CEA) and budget impact analysis (BIA) Exclusion criteria were case reports, abstracts, letters to the editor, conference presentations, editorials, and studies written in languages other than English and articles of other types of pharmacoeconomic analysis. Risk of bias assessments were undertaken to assess data strength and validity. Results: We identified a total of six EEs studies (one cost-effectiveness analysis, two studies reported both cost-effectiveness and cost-utility analysis and three budget impact analyses). Three studies were from United State (US), two from France and one from Singapore. The six studies met &gt; 80% of the JBI quality assessment criteria. The primary prophylaxis with filgrastim biosimilar in breast cancer, non-small cell lung cancer, and non-Hodgkin lymphoma provided an additional 0.102-0.118 FN event avoided, 0.065-0.144 Lys, 0.057-0.13 QALYs at an incremental cost of 651 US$-2463 US$. The incremental cost effectiveness ratio (ICER) ranged from 5660 US$-20806 US$ per FN event avoided, 5123 US$ - 31077 US$ per LY gained, and 7213 US$ - 35563 US$ per QALY gained. The NSCLC has the lowest ICERs. Conclusions: Evaluated studies showed that G-CSF biosimilars are cost effective compared to the references as primary and secondary prophylaxis for chemotherapy induced neutropenia among oncology patients

Cost-effectiveness analysis of serplulimab in combination with cisplatin plus 5-fluorouracil chemotherapy compared to cisplatin plus 5-fluorouracil chemotherapy as first-line treatment for advanced or metastatic esophageal squamous cell carcinoma in China.

This study evaluated the cost-effectiveness of serplulimab plus chemotherapy versus chemotherapy alone in treating advanced/metastatic esophageal squamous cell carcinoma (ESCC) within the Chinese health care system. A partitioned survival model based on ASTRUM-007 trial patient characteristics was developed. Efficacy, safety, and medical/economic data were obtained from the trial and real-world clinical practice. Costs, quality-adjusted life years (QALY), and incremental cost-effectiveness ratios (ICERs) were calculated for both treatment strategies. Sensitivity, subgroup, and scenario analyses were performed to assess the uncertainty impact. Serplulimab combined with chemotherapy yielded an ICER of US$ 53,538.27/QALY. Deterministic sensitivity analysis identified patient survival and serplulimab price as influential parameters. Probabilistic sensitivity analysis showed a 47.33% probability of cost-effectiveness at a willingness-to-pay (WTP) threshold of US$ 53,541/QALY and 0.05% at three times China's GDP per capita. Subgroup analysis revealed that patients with a programmed death-ligand 1 (PD-L1) expression combined positive score (CPS) ⩾10 had a lower hazard ratio (0.59) and ICER (US$ 29,935.23/QALY), with a 95.36% probability of cost-effectiveness. Scenario analysis demonstrated that the drug donation discount policy significantly increased the likelihood of cost-effective serplulimab-chemotherapy combinations in Jiangsu, Fujian, and Guangdong at 99.99%, 99.90%, and 94.16%, respectively. Compared to chemotherapy alone, serplulimab combined with chemotherapy is currently not a cost-effective first-line treatment for advanced/metastatic ESCC in China. However, as serplulimab plus chemotherapy regimens evolve and price competition among programmed death 1 (PD-1) inhibitors intensifies, this combination may become a cost-effective treatment option.

Cost-effectiveness analysis of sintilimab plus bevacizumab biosimilar compared with lenvatinib as the first-line treatment of unresectable or metastatic hepatocellular carcinoma

BackgroundIn recent years, programmed cell death protein-1 inhibitors, including sintilimab, have significantly prolonged the overall survival time of patients with unresectable or metastatic hepatocellular carcinoma (HCC); however, the cost-effectiveness of sintilimab is unclear. The aim of this study was to assess the cost-effectiveness of sintilimab plus bevacizumab biosimilar compared with lenvatinib as first-line treatment in patients with unresectable or metastatic HCC.MethodsA lifetime partitioned survival model was developed to conduct a cost-effectiveness analysis of sintilimab plus bevacizumab biosimilar vs. lenvatinib for advanced HCC from a Chinese healthcare system perspective. The clinical and safety data were derived from two recent randomized clinical trials, the ORIENT-32 and REFLECT studies. Utility data were obtained from previous studies. Long-term direct medical costs and quality-adjusted life-years (QALYs) were predicted. Deterministic and probabilistic sensitivity analyses were performed to verify the robustness of the model.ResultsCompared with lenvatinib, combination therapy with sintilimab and bevacizumab biosimilar yielded an additional 0.493 QALYs at a higher cost ($33,102 vs. $21,037) (2021 US dollars). This resulted in a deterministic incremental cost-effectiveness ratio (ICER) of $24,462 per QALY in the base-case analysis. The ICERs were sensitive to the utility of post-progression and the cost of bevacizumab biosimilar. A lower ICER was estimated when the dose of bevacizumab biosimilar decreased from 15 mg to 7.5 mg per kilogram in the scenario analysis. In the probabilistic sensitivity analysis, the probability of being cost-effective for sintilimab treatment at willingness-to-pay (WTP) thresholds of one ($12,516) and three times the gross domestic product per capita in China ($37,547) were 11.6% and 88.6%, respectively.ConclusionSintilimab plus bevacizumab biosimilar is likely to be a cost-effective treatment option as a first-line treatment for unresectable or metastatic HCC in China when WTP threshold is over $23,650.

Cost-effectiveness of single, high-dose, liposomal amphotericin regimen for HIV-associated cryptococcal meningitis in five countries in sub-Saharan Africa: an economic analysis of the AMBITION-cm trial

HIV-associated cryptococcal meningitis is a leading cause of AIDS-related mortality. The AMBITION-cm trial showed that a regimen based on a single high dose of liposomal amphotericin B deoxycholate (AmBisome group) was non-inferior to the WHO-recommended treatment of seven daily doses of amphotericin B deoxycholate (control group) and was associated with fewer adverse events. We present a five-country cost-effectiveness analysis. The AMBITION-cm trial enrolled patients with HIV-associated cryptococcal meningitis from eight hospitals in Botswana, Malawi, South Africa, Uganda, and Zimbabwe. Taking a health service perspective, we collected country-specific unit costs and individual resource-use data per participant over the 10-week trial period, calculating mean cost per participant by group, mean cost-difference between groups, and incremental cost-effectiveness ratio per life-year saved. Non-parametric bootstrapping and scenarios analyses were performed including hypothetical real-world resource use. The trial registration number is ISRCTN72509687, and the trial has been completed. The AMBITION-cm trial enrolled 844 participants, and 814 were included in the intention-to-treat analysis (327 from Uganda, 225 from Malawi, 107 from South Africa, 84 from Botswana, and 71 from Zimbabwe) with 407 in each group, between Jan 31, 2018, and Feb 17, 2021. Using Malawi as a representative example, mean total costs per participant were US$1369 (95% CI 1314-1424) in the AmBisome group and $1237 (1181-1293) in the control group. The incremental cost-effectiveness ratio was $128 (59-257) per life-year saved. Excluding study protocol-driven cost, using a real-world toxicity monitoring schedule, the cost per life-year saved reduced to $80 (15-275). Changes in the duration of the hospital stay and antifungal medication cost showed the greatest effect in sensitivity analyses. Results were similar across countries, with the cost per life-year saved in the real-world scenario ranging from $71 in Botswana to $121 in Uganda. The AmBisome regimen was cost-effective at a low incremental cost-effectiveness ratio. The regimen might be even less costly and potentially cost-saving in real-world implementation given the lower drug-related toxicity and the potential for shorter hospital stays. European Developing Countries Clinical Trials Partnership, Swedish International Development Cooperation Agency, Wellcome Trust and Medical Research Council, UKAID Joint Global Health Trials, and the National Institute for Health Research. For the Chichewa, Isixhosa, Luganda, Setswana and Shona translations of the abstract see Supplementary Materials section.

Achieving malaria testing and treatment targets for children under five in Mozambique: a cost-effectiveness analysis

BackgroundThe entire population of Mozambique is at risk for malaria, which remains one of the leading causes of death. The 2017–2022 National Malaria Strategic Plan focuses on reducing malaria morbidity and mortality in high- and low-transmission areas. This study aimed to estimate the costs and health benefits of six variations of the World Health Organization’s “test-and-treat” strategy among children under five.MethodsA decision tree model was developed that estimates the costs and health outcomes for children under five. Data on probabilities, costs, weights for disability-adjusted life years (DALYs), and quality-adjusted life years (QALYs) were based on peer-reviewed, grey literature, and primary data analysis of the 2018 Malaria Indicator Survey. Six scenarios were compared to the status quo and calculated the incremental cost-effectiveness ratio (ICER) in terms of cost per QALY gained, DALY averted, and life saved. Deterministic and probabilistic sensitivity analyses were conducted to understand the effect of parameter uncertainty on the findings.ResultsIn the base case, reaching the target of 100% testing with rapid diagnostic tests (RDTs; Scenario 1) is more cost-effective than improving the testing rate alone by 10% (Scenario 2). Achieving a 100% (Scenario 3) or a 10% increase in treatment rate (Scenario 4) have ICERs that are lower than Scenarios 1 and 2. Both Scenarios 5 and 6, which represent combinations of Scenarios 1–4, have lower ICERs than their constituent strategies on their own, which suggests that improvements in treatment are more cost-effective than improvements in testing alone. These results held when DALYs averted or lives saved were used as health outcomes. Deterministic and probabilistic sensitivity analyses revealed that the cost-effectiveness of Scenarios 1–6 are subject sensitive to parameter uncertainty, though Scenarios 4 and 5 are the optimal choice when DALYs averted or QALYs gained were used as the measure of health outcomes across all cost-effectiveness thresholds.ConclusionsImproving testing rates alone among children at risk for malaria has the potential to improve health but may not be the most efficient use of limited resources. Instead, small or large improvements in treatment, whether alone or in conjunction with improvements in testing, are the most cost-effective strategies for children under five in Mozambique.

Economic evaluation of new blood pressure target for hypertensive patients in Taiwan according to the 2022 hypertension clinical practice guidelines of the Taiwan society of cardiology: a simulation modeling study.

With the promising cardiovascular benefits in the STEP and SPRINT trials, the2022 Taiwan's hypertension guidelines redefined the hypertension threshold as 130/80 mmHg and a universal blood-pressure target of <130/80 mmHg. This study's objective was to examine the cost-effectiveness of the intensive blood-pressure target for hypertensive patients using estimated lifetime medical costs and quality-adjusted life years (QALY) from the Taiwan national payer's perspective. We developed a lifetime Markov model comparing the intensive and conservative blood-pressure targets. Incremental cost-effectiveness ratio (ICER) against the willing-to-pay thresholds at the one-time [US$34,000(NT$1,020,000)] and three-time [US$100,000(NT$3,000,000)] gross domestic product per capita were defined as very cost-effect and only cost-effective. The cost-effectiveness in different age stratifications and cardiovascular risks treated with a more intensive target (120 mmHg) were examined in the subgroup analyses. The new blood-pressure treatment target produced more lifetime medical costs [US$31,589(NT$947,670) versus US$26,788(NT$803,640)] and QALYs (12.54 versus 12.25), and the ICER was US$16,589(NT$497,670), which was 99.1% and 100% probability of a very cost-effective and cost-effective strategy. The ICERs in all age stratifications had more than a 90% probability of being very cost-effective, and ICERs decreased with age. More intensive control in patients with high cardiovascular risks produced a lower ICER [US$14,547(NT$436,410)]. In conclusion, Taiwan's new blood-pressure treatment target can prevent more cardiovascular events with acceptable costs per QALY below the willing-to-pay thresholds. The cost-effectiveness of intensive control is consistent across different ages and more pronounced with the increase in age and cardiovascular risk.

The cost-effectiveness of rivaroxaban with or without aspirin in the COMPASS trial.

The Cardiovascular Outcomes for People Using Anticoagulation Strategies (COMPASS) trial demonstrated that rivaroxaban 2.5mg BID with aspirin 100mg was more effective than aspirin 100mg daily alone for the prevention of cardiovascular (CV) death, stroke, or myocardial infarction in patients with stable coronary artery disease (CAD) or peripheral artery disease (PAD). We aimed to examine the cost-effectiveness of rivaroxaban using patient-level data from the COMPASS trial. We performed an in-trial analysis and extrapolated our results for 33 years using a two-state Markov model with a 1-year cycle length. Hospitalization events, procedures, and study drugs were documented for patients. We applied country-specific (Canada, France, and Germany) direct healthcare system costs (in USD) to healthcare resources consumed by patients. Average cost per patient during the trial (mean follow-up of 23 months), quality-adjusted life years (QALYs), and lifetime cost-effectiveness were calculated. Costs of events and procedures were reduced with rivaroxaban 2.5mg BID with aspirin. The addition of rivaroxaban 2.5mg BID increased total costs for the combination group. Over a lifetime horizon (in trial +33 years), rivaroxaban plus aspirin was associated with 1.17 QALYs gained, yielding an incremental cost-effectiveness ratio (ICER) of $3946/QALY, $9962/QALY, and $10264/QALY in Canada, France, and Germany, respectively. PAD and polyvascular disease subgroups had lower ICERs. Rivaroxaban 2.5mg twice daily plus aspirin compared with aspirin alone reduces direct healthcare costs. After acquisition costs of rivaroxaban, the lifetime cost-effectiveness of 2.5mg twice daily plus aspirin is highly cost-effective in Canada, France, and Germany.(COMPASS ClinicalTrials.gov identifier: NCT01776424).

Biomarker testing to guide first-line PARP inhibitor maintenance for patients with advanced ovarian cancer after response to first- line platinum chemotherapy: A cost-effectiveness study (382)

Objectives: Multiple poly-ADP ribose polymerase inhibitor (PARPi) maintenance treatment (mTx) options are available for patients with advanced ovarian cancer after response to first-line platinum chemotherapy (PSaOC). Olaparib (O) is an option as monotherapy for patients with BRCA1/2 mutations or in combination with bevacizumab (O+B) for Homologous Recombination Deficiency (HRD+) patients. Meanwhile, niraparib (N) is approved irrespective of biomarker status. This study aimed to evaluate the cost-effectiveness of biomarker testing and treatment with PARP inhibitor mTx in PSaOC patients from the perspective of a US payer. Methods: Ten testing and treatment strategies (Table 1) were evaluated, representing common biomarker testing (none, BRCA or HRD) and mTx (O, O+B, N, B) in approved populations. PAOLA-1 trial data were used to build a lifetime horizon partitioned survival model with four health states, including progression-free (PFS), post 1st progression (PP1), post 2nd progression (PP2), and death. PFS was modeled through parametric mixture survival modeling to a landmark survival point at five years. Patients who remain PFS at year five are assumed to be long-term survivors. Time to PP2 and OS was modeled by fitting standard parametric models, and time on treatment data was informed by respective trials (up to 24m for O, 15m for B, treat to progression for N). The hazard ratio (HR) of PFS for O+B versus N (0.57 for HRD+/ITT; 0.46 for BRCA+) and O+B versus O (0.71 for BRCA+) were obtained from matching adjusted indirect comparison (MAIC) between PAOLA1 and PRIMA, and PAOLA1 and SOLO1. The HRs for OS are informed by PFS benefits and published OS data. The incremental cost-effectiveness ratios (ICERs) were calculated. Results: Strategy 2 (S2, no testing, B) had the lowest cost while S10 (HRD testing, O+B for HRD+ and B for HRD-) had the greatest life- years (LYs) and quality-adjusted life-years (QALYs). All of the niraparib strategies were dominated (i.e., had higher cost, lower LY/ QALYs), compared to at least one other strategy (Table 1). When compared to S2, S8 had lower ICERs than S4 ($41,204 per LY and $48,211 per QALY for S8 vs S2). S10 had an ICER of $66,588 per LY and $82,321 per QALY compared to S8. Conclusions: HRD testing followed by O+B for BRCA-/HRD+, O for BRCA+ and B for HRD- (S8) is highly cost-effective, dominating six strategies (all except 2, 4 &10) and having ICERs < $50,000/QALY compared to the least costly strategy (S2). Replacing O with O+B in BRCA+ patients (S10) improves outcomes, with ICER < 100,000/QALY compared to S8. All strategies involving niraparib were dominated by other approaches. An HRD biomarker-guided approach followed with O±B treatment in HRD+ patients provides the greatest LY and QALYs with good economic value. Objectives: Multiple poly-ADP ribose polymerase inhibitor (PARPi) maintenance treatment (mTx) options are available for patients with advanced ovarian cancer after response to first-line platinum chemotherapy (PSaOC). Olaparib (O) is an option as monotherapy for patients with BRCA1/2 mutations or in combination with bevacizumab (O+B) for Homologous Recombination Deficiency (HRD+) patients. Meanwhile, niraparib (N) is approved irrespective of biomarker status. This study aimed to evaluate the cost-effectiveness of biomarker testing and treatment with PARP inhibitor mTx in PSaOC patients from the perspective of a US payer. Methods: Ten testing and treatment strategies (Table 1) were evaluated, representing common biomarker testing (none, BRCA or HRD) and mTx (O, O+B, N, B) in approved populations. PAOLA-1 trial data were used to build a lifetime horizon partitioned survival model with four health states, including progression-free (PFS), post 1st progression (PP1), post 2nd progression (PP2), and death. PFS was modeled through parametric mixture survival modeling to a landmark survival point at five years. Patients who remain PFS at year five are assumed to be long-term survivors. Time to PP2 and OS was modeled by fitting standard parametric models, and time on treatment data was informed by respective trials (up to 24m for O, 15m for B, treat to progression for N). The hazard ratio (HR) of PFS for O+B versus N (0.57 for HRD+/ITT; 0.46 for BRCA+) and O+B versus O (0.71 for BRCA+) were obtained from matching adjusted indirect comparison (MAIC) between PAOLA1 and PRIMA, and PAOLA1 and SOLO1. The HRs for OS are informed by PFS benefits and published OS data. The incremental cost-effectiveness ratios (ICERs) were calculated. Results: Strategy 2 (S2, no testing, B) had the lowest cost while S10 (HRD testing, O+B for HRD+ and B for HRD-) had the greatest life- years (LYs) and quality-adjusted life-years (QALYs). All of the niraparib strategies were dominated (i.e., had higher cost, lower LY/ QALYs), compared to at least one other strategy (Table 1). When compared to S2, S8 had lower ICERs than S4 ($41,204 per LY and $48,211 per QALY for S8 vs S2). S10 had an ICER of $66,588 per LY and $82,321 per QALY compared to S8. Conclusions: HRD testing followed by O+B for BRCA-/HRD+, O for BRCA+ and B for HRD- (S8) is highly cost-effective, dominating six strategies (all except 2, 4 &10) and having ICERs < $50,000/QALY compared to the least costly strategy (S2). Replacing O with O+B in BRCA+ patients (S10) improves outcomes, with ICER < 100,000/QALY compared to S8. All strategies involving niraparib were dominated by other approaches. An HRD biomarker-guided approach followed with O±B treatment in HRD+ patients provides the greatest LY and QALYs with good economic value.

Cost effectiveness of aortic valve therapies: a systematic review of the literature

&#x0D; Background: we performed a systematic review on the cost effectiveness of transcatheter aortic valve implantation (TAVI) to standard aortic valve replacement and medical management in high-risk elderly patients with severe aortic stenosis.&#x0D; Methods: in accordance with Preferred Reporting Items for Systematic reviews and Meta-Analyses, a systematic review on current literature for cost-effectiveness of TAVI, standard aortic valve replacement, and medical management for elderly patients with high-risk severe aortic stenosis was performed. Incremental cost effectiveness ratio is used to measure effectiveness through life years gained or quality adjusted life years. Drummond checklist was used to further assess the quality of the included studies.&#x0D; Results: the systematic literature search identified 4 primary publications (derived from 52 citations) that fulfilled the inclusion criteria. Tremendous discrepancy in incremental cost effectiveness ratio is demonstrated with operable patients similar to Cohort A of the PARTNER trial (€ 749 416 and € 39 577). Inoperable patients similar to Cohort B of the PARTNER trial suggest notable differences in favour for transcatheter aortic valve implantation with an increase in quality adjusted life years (0.06 versus 1.6, respectively). With lifetime horizon to transcatheter aortic valve implantation there is a more comparable incremental cost effectiveness ratio in the literature (€ 38 260 and € 37 432). Lowest incremental cost effectiveness ratio witnessed in the technical inoperable group at € 26 482. Lifetime horizon of 10 years with transcatheter aortic valve implantation differ (€ 39 388 versus € 19 947). Overall, a review of the literature suggests TAVI usage in patients for severe aortic stenosis whom are not eligible for surgery. All the studies were overall judged of medium-high quality.&#x0D; Conclusions: transcatheter aortic valve replacement is more cost effective with a lifetime horizon for the treatment of patients with high-risk aortic stenosis compared with medical management considering those ineligible for standard aortic valve replacement. Further cost effectiveness research is needed in the stratifications of patient risk and patient co-morbidities for those candidates eligible for surgery.  &#x0D;

Cost-effectiveness of lung cancer screening combined with nurse-led smoking cessation intervention: A population-based microsimulation study

BackgroundThe appropriate screening inclusion criteria of low-dose computed tomography screening for lung cancer in Chinese population remains unclear and the effect of combining screening with nurse-led smoking cessation intervention is poorly understood as well. ObjectiveWe compared the benefits and costs of lung cancer screening with and without nurse-led smoking cessation intervention in different inclusion criteria to help select optimal screening strategies. MethodsDifferent screening strategies were set based on diverse starting ages, smoking pack-year and whether nurse-led smoking cessation intervention was applied. We use nationally representative data published by the China Health and Retirement Longitudinal Survey, based on a microsimulation model, to predict incremental cost-effectiveness ratio and net health benefits under different strategies. ResultsThe incremental cost-effectiveness ratios for all lung cancer screening strategies were less than three times GDP per capita, and screening combined with smoking cessation intervention had lower incremental cost-effectiveness ratios. The largest net health benefits and probability of cost-effectiveness were both obtained in the strategy which conducted screening and nurse-led smoking cessation intervention for people over 45 years with at least 20 pack-year smoking history. In strategies screening alone, it was obtained in screening for people over 50 years and with at 20 pack-year smoking history. ConclusionsNurse-led smoking cessation intervention is recommended provided in conjunction with lung cancer screening. The optimal strategy is conducted screening with cessation intervention for current smokers or smoking quitters in the past 15 years who are over 45 years with at least 20 pack-year smoking history. For strategies screening alone, the target population should be over 50 years old with at least 20 pack year smoking history, when willingness to pay less than three times GPD per capita.