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Background: Deep inspiration breath hold (DIBH) is an effective technique for reducing heart exposure during radiotherapy for left-sided breast cancer. Despite its benefits, cost considerations and its impact on workflow remain significant barriers to widespread adoption. Objectives: This study aimed to assess the cost-effectiveness of DIBH and compare its operational, financial, and clinical outcomes with free breathing (FB) in breast cancer treatment. Methods: Treatment plans for 100 patients with left-sided breast cancer were generated using both DIBH and FB techniques. Dosimetric data, including the average mean heart dose, were calculated for each technique and used to estimate the cardiotoxicity of radiotherapy. A state-transition microsimulation model based on SCORE2 (Systematic Coronary Risk Evaluation) algorithms projected the effects of DIBH on cardiovascular outcomes and quality-adjusted life-years (QALYs). Costs were calculated from a provider perspective using time-driven activity-based costing, applying a willingness-to-pay threshold of 40,000 for cost-effectiveness assessment. A discrete event simulation model assessed the impacts of DIBH vs FB on throughput and waiting times in the radiotherapy workflow. Results: In the base case scenario, DIBH was associated with an absolute risk reduction of 1.72% (95% CI: 1.67%-1.76%) in total cardiovascular events and 0.69% (95% CI: 0.67%-0.72%) in fatal cardiovascular events over 20 years. Additionally, DIBH was estimated to provide an incremental 0.04 QALYs (95% CI: 0.05-0.05) per left-sided breast cancer patient over the same time period. However, DIBH increased treatment times, reducing maximum achievable throughput by 12.48% (95% CI: 12.36%-12.75%) and increasing costs by 617 per left-sided breast cancer patient (95% CI: 615-619). The incremental cost-effectiveness ratio was 14,023 per QALY. Conclusions: Despite time investments, DIBH is cost-effective in the Belgian population. The growing adoption of DIBH may benefit long-term cardiovascular health in breast cancer survivors.
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Purpose: This study assesses the cost-effectiveness of rimegepant for the on-demand acute treatment of migraine in the Chinese population, focusing on headache relief within a 2 h timeframe. Utilizing data from Phase III clinical trials on rimegepant involving Asian populations, this analysis aims to provide essential insights for healthcare decision-making in the context of migraine management in China. Patients and methods: Employing a decision tree model, this research evaluates the cost-effectiveness of rimegepant over a concise 2 h period, exclusively considering its direct market price of 219.00 CNY per dose for on-demand, single-use treatment upon approval in China. This model is based on pain relief outcomes from a clinical trial, categorizing health outcomes by the achievement of pain freedom and alleviation from the most bothersome symptom within two hours post-administration. Results: The study unveils that rimegepant adds 0.0018 quality-adjusted life days (QALD) with an incremental cost-effectiveness ratio (ICER) of 122,166.07 CNY/QALD. Against a daily cost-effectiveness threshold derived from the 2023 per capita GDP of China (734.45 CNY/day), rimegepant falls short of proving its cost-effectiveness. A significant price reduction to approximately 1.32 CNY per dose is required for rimegepant to be considered cost-effective within this framework. Furthermore, a series of sensitivity analyses were conducted to validate the robustness of these results. Conclusion: While rimegepant shows clinical efficacy in providing rapid relief from migraine symptoms, its current pricing exceeds the threshold for cost-effectiveness in the Chinese healthcare setting. This study underscores the need for price adjustments to enhance the accessibility and economic viability of new migraine treatments.
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PURPOSE: The present study aimed to systematically collect and synthesize available cost-effectiveness studies of semaglutide in patients with obesity or overweight in comparison with other interventions. METHODS: We comprehensively searched multiple electronic databases to identify relevant literature. Studies were selected based on inclusion and exclusion criteria. The quality of studies was appraised using the "Consolidated Health Economic Evaluation Reporting Standards" (CHEERS) tool. This study is conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement. RESULTS: Out of a total of 252 items, after review, 32 articles were fully reviewed, and, finally, 7 studies met inclusion and exclusion criteria. The discount rate was in the range of 1.5-3.5%. Studies included showed semaglutide offered more QALYs than anti-obesity drugs but because of higher cost, in some cases, ICER exceeds the willingness to pay threshold. Results show that semaglutide creates higher total cost compared to conventional interventions in patients with class I, II, and III obesities. Results show that in patients with class I obesity (BMI 33) lifestyle intervention (LI), endoscopic sleeve gastroplasty (ESG), Sleeve gastrectomy (SG), and semaglutide create $124,195; $126,732; $139,971; and $370,776, respectively. CONCLUSION: The current systematic review showed that semaglutide provides more QALYs and creates more costs in comparison with phentermine-topiramate, phentermine, and naltrexone-bupropion. Semaglutide may be cost-effective with substantial cost reduction. Semaglutide appears to be cost-effective versus diet and exercise (D&E) and liraglutide but it was not cost-effective versus sleeve gastrectomy, endoscopic sleeve gastroplasty, and gastric bypass.
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Background: We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis. Methods: We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence. Results: The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal cancer but may be cost-effective for the remaining comparisons. Limitations: We were unable to obtain individual participant data as planned. The limited number of randomised controlled trials for each comparison and the paucity of data on health-related quality of life mean that the recommendations may change as new evidence (from trials with a low risk of bias) emerges. Conclusions: In people with peritoneal metastases from colorectal cancer with limited peritoneal metastases and who are likely to withstand major surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should not be used in routine clinical practice (strong recommendation). There is considerable uncertainty as to whether hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy or cytoreductive surgery + systemic chemotherapy should be offered to patients with gastric cancer and peritoneal metastases (no recommendation). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered routinely to women with stage III or greater epithelial ovarian cancer and metastases confined to the abdomen requiring and likely to withstand interval cytoreductive surgery after chemotherapy (strong recommendation). Future work: More randomised controlled trials are necessary. Study registration: This study is registered as PROSPERO CRD42019130504. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.
Cancers of the bowel, ovary or stomach can spread to the lining of the abdomen ('peritoneal metastases'). Chemotherapy (the use of drugs that aim to kill cancer cells) given by injection or tablets ('systemic chemotherapy') is one of the main treatment options. There is uncertainty about whether adding cytoreductive surgery (cytoreductive surgery; an operation to remove the cancer) and 'hyperthermic intraoperative peritoneal chemotherapy' (warm chemotherapy delivered into the lining of the abdomen during cytoreductive surgery) are beneficial. We reviewed all the information from medical literature published until 14 April 2022, to answer the above uncertainty. We found the following from eight trials, including about 1000 participants. In people with peritoneal metastases from bowel cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably does not provide any benefits and increases harm compared to cytoreductive surgery + systemic chemotherapy, while cytoreductive surgery + systemic chemotherapy appears to increase survival compared to systemic chemotherapy alone. There is uncertainty about the best treatment for people with peritoneal metastases from stomach cancer. In women with peritoneal metastases from ovarian cancer who require systemic chemotherapy before cytoreductive surgery to shrink the cancer to allow surgery ('advanced ovarian cancer'), hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably increases survival compared to cytoreductive surgery + systemic chemotherapy. In people who can withstand a major operation and in whom cancer can be removed, cytoreductive surgery + systemic chemotherapy should be offered to people with peritoneal metastases from bowel cancer, while hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered to women with peritoneal metastases from 'advanced ovarian cancer'. Uncertainty in treatment continues for gastric cancer. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.
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Análisis Costo-Beneficio , Procedimientos Quirúrgicos de Citorreducción , Quimioterapia Intraperitoneal Hipertérmica , Neoplasias Peritoneales , Humanos , Neoplasias Peritoneales/secundario , Neoplasias Peritoneales/terapia , Neoplasias Peritoneales/tratamiento farmacológico , Procedimientos Quirúrgicos de Citorreducción/economía , Evaluación de la Tecnología Biomédica , Ensayos Clínicos Controlados Aleatorios como Asunto , Femenino , Años de Vida Ajustados por Calidad de Vida , Neoplasias Ováricas/patología , Neoplasias Ováricas/tratamiento farmacológico , Neoplasias Ováricas/cirugía , Neoplasias Ováricas/terapia , Hipertermia Inducida/economía , Análisis de Costo-EfectividadRESUMEN
OBJECTIVE: In the Brazilian public national healthcare system, botulinum toxin type A has traditionally been the sole treatment option for patients with dystonia. However, as of October 2022, Deep Brain Stimulation (DBS) garnered positive recommendations for the condition. This study aims to assess the cost-effectiveness of DBS in treating adults with generalized and cervical dystonia within the Brazilian healthcare context, considering its recent inclusion. METHODS: A systematic review identified randomized controlled trials (RCTs) assessing DBS efficacy in treating adults with generalized and cervical dystonia. Two cost-utility analyses compared the cost-effectiveness of DBS plus the Best Clinical Practice (BCP) to BCP alone. Markov models, which included three health states (no clinical improvement, clinical improvement, and death), employed a one-year cycle and a lifetime horizon. The study utilized both one-way and probabilistic sensitivity analyses. RESULTS: Two RCTs, one for each condition, revealed superior clinical improvement with DBS when compared to sham simulation. The Incremental Cost-Utility Ratio (ICUR) was $ 1,121.66 for generalized dystonia and $4,556.50 for cervical dystonia. Effectiveness discount rates and age at surgery were identified as influential parameters. In 1,000 Monte Carlo simulations, 99.9% of the ICUR values for generalized dystonia and 74.2 % for cervical dystonia fell below the cost-effectiveness threshold in Brazil ($8,146.64 per QALY). CONCLUSIONS: From the perspective of the Brazilian public health system, the combination of DBS and BCP appears to be cost-effective for the treatment of both generalized and cervical dystonia when compared to BCP alone.
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BACKGROUND: Research focused on the addition of immune checkpoint inhibitors (ICIs) to radiotherapeutic regimens in patients with cancer has become increasingly common, revealing promising improvements in efficacy outcomes. In patients with locoregionally advanced nasopharyngeal carcinoma (NPC), combining immunotherapy with chemoradiotherapy can facilitate the significant prolongation of survival, emphasizing the need for pharmacoeconomic studies focused on the clinical uptake of these innovative treatment regimens. METHODS: A three-state Markov model was developed based on clinical data from the randomized phase 3 CONTINUUM trial and used to compare the cost-effectiveness of chemoradiotherapy plus sintilimab (sintilimab group) to chemoradiotherapy alone (standard group), analyzing outcomes including incremental cost-effectiveness ratio (ICER), incremental net monetary benefit (INMB), and incremental net-health benefit (INHB) values at a willingness-to-pay (WTP) threshold corresponding to three times the Chinese GDP per capita ($37 035 per quality-adjusted life year [QALY]). RESULTS: The total costs for patients in the sintilimab and standard groups (QALYs [LYs]) were $92 116 (6.68 [10.03]) and $53 255 (3.75 [5.55]), respectively, for an ICER of $13 230/QALY ($8672/LY), an INMB of $70 021 with INHB of 1.89 QALYs. Using the selected WTP threshold. On the standard WTP threshold, the prevalence of sintilimab group as the primary treatment was 90.55% in China. The establishment of the model is stable. CONCLUSIONS: Adding sintilimab to chemoradiotherapeutic regimens represents an innovative and cost-effective means for patients with locoregionally advanced NPC management in China.
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AIMS: This study aims to evaluate the cost effectiveness of genetic and genomic testing strategies for the diagnosis of rare developmental disorders in NHS Scotland. METHODS: Six genetic and genomic testing strategies were evaluated using a decision tree model. First-line, second-line and last-resort trio genome sequencing (GS), and second-line and last-resort trio exome sequencing (ES) were compared with standard genetic testing. The cost effectiveness of each strategy was expressed in terms of incremental cost per additional diagnosis. The impact of uncertainty on cost-effectiveness results was explored using deterministic and probabilistic sensitivity analysis. RESULTS: 2nd-line ES was a cost-saving option, increasing diagnostic yield by 13.9% and decreasing cost by £1027 per trio compared to standard genetic testing. Compared to ES, strategies involving GS increased costs significantly, with only a moderate or zero improvement in diagnostic yield. Sensitivity analysis indicated that significant reductions in cost or improvements in diagnostic yield are required before 1st-line GS becomes cost effective. CONCLUSION: 2nd-line ES (after chromosomal microarray; replacing gene panel testing) for the diagnosis of developmental disorders is a cost-saving option for the Scottish NHS. Ongoing economic evaluation is required to monitor the evolving cost and diagnostic yield of GS and ES over time.
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Introduction: Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor T-cell therapy approved for patients with relapsed/refractory multiple myeloma (RRMM). In the phase 3 trial, CARTITUDE-4 (NCT04181827), cilta-cel demonstrated improved efficacy vs. standard of care (SOC; daratumumab plus pomalidomide and dexamethasone [DPd] or pomalidomide plus bortezomib and dexamethasone [PVd]) with a ≥ complete response (≥CR) rate of 73.1% vs. 21.8%. Methods: A cost-per-responder model was developed to assess the value of cilta-cel and SOC (87% DPd and 13% PVd) based on the CARTITUDE-4 trial data from a US mixed payer perspective (76.7% commercial, 23.3% Medicare). The model was developed using progression-free survival (PFS), overall survival (OS), and ≥CR endpoints from CARTITUDE-4 over a period of 25.4 months. Inpatient stays, outpatient visits, drug acquisition, administration, and monitoring costs were included. The base-case model assumed an inpatient setting for each cilta-cel infusion; another scenario included 30% outpatient and 70% inpatient infusions. Costs of managing grade 3-4 adverse events (AEs) and grade 1-4 cytokine release syndrome and neurotoxicity were included. Subsequent therapy costs were incurred after disease progression; terminal care costs were considered upon death events. Outcomes included total cost per treated patient, total cost per complete responder, and cost per month in PFS between cilta-cel and SOC. Costs were adjusted to 2024 US dollars. Results: Total cost per treated patient, total cost per complete responder, and total cost per month in PFS were estimated at $704,641, $963,941, and $30,978 for cilta-cel, respectively, and $840,730, $3,856,559, and $42,520 for SOC over the 25.4-month period. Cost drivers included treatment acquisition costs before progression and subsequent treatment costs ($451,318 and $111,637 for cilta-cel; $529,795 and $265,167 for SOC). A scenario analysis in which 30% of patients received an outpatient infusion (assuming the same payer mix) showed a lower cost per complete responder for cilta-cel ($956,523) than those with an infusion in the inpatient setting exclusively. Discussion: This analysis estimated that cost per treated patient, cost per complete responder, and cost per month in PFS for cilta-cel were remarkably lower than for DPd or PVd, highlighting the substantial clinical and economic benefit of cilta-cel for patients with RRMM.
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Análisis Costo-Beneficio , Inmunoterapia Adoptiva , Lenalidomida , Mieloma Múltiple , Talidomida , Humanos , Mieloma Múltiple/terapia , Mieloma Múltiple/mortalidad , Mieloma Múltiple/tratamiento farmacológico , Lenalidomida/uso terapéutico , Lenalidomida/administración & dosificación , Inmunoterapia Adoptiva/economía , Inmunoterapia Adoptiva/efectos adversos , Inmunoterapia Adoptiva/métodos , Talidomida/análogos & derivados , Talidomida/uso terapéutico , Talidomida/economía , Talidomida/administración & dosificación , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Dexametasona/uso terapéutico , Dexametasona/administración & dosificación , Receptores Quiméricos de Antígenos/uso terapéutico , Receptores Quiméricos de Antígenos/inmunología , Masculino , Femenino , Bortezomib/uso terapéutico , Bortezomib/administración & dosificación , Persona de Mediana Edad , Supervivencia sin Progresión , Resultado del Tratamiento , Anciano , Resistencia a Antineoplásicos , Anticuerpos Monoclonales/uso terapéutico , Anticuerpos Monoclonales/economíaRESUMEN
AIMS: The present analysis estimated the cost-effectiveness of treatment with the Tandem t: slim X2 insulin pump with Control IQ technology (CIQ) in children with type 1 diabetes in Sweden. METHODS: A four-state Markov model and probabilistic sensitivity analyses (PSA) were used to assess the cost-effectiveness of CIQ use compared with treatment with multiple daily insulin injections (MDI) or continuous subcutaneous insulin infusion (CSII) in conjunction with CGM. Data sources included clinical input data from a recent retrospective, observational study, cost data from local diabetes supply companies and government agencies, and published literature. Outcomes measures were quality adjusted life years (QALYs) at 10, 20 and 30-year time horizons based on cost per QALY and incremental cost-effectiveness ratio (ICER). RESULTS: A total of 84 type 1 diabetes children were included (CIQ, n = 37; MDI, n = 19; CSII, n = 28). For all time horizons, the use of CIQ was a dominant strategy (e.g. more effective and less costly) compared with MDI or CSII use: 10-year ICER, SEK -88,010.37 and SEK -91,723.92; 20-year ICER, SEK -72,095.33 and SEK -87,707.79; and 30-year ICER, SEK -65,573.01 and SEK -85,495.68, respectively. PSA confirmed that CIQ use was less costly compared with MDI and CSII. CONCLUSIONS: Initiation of CIQ use in children with type 1 diabetes is cost-saving, besides previously shown improved glycaemic control, and increased quality of life. Further investigations are needed to more fully elucidate the cost-effectiveness of these technologies in different countries with existing differences in payment models.
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BACKGROUND: Duchenne muscular dystrophy (DMD) is a genetic disease resulting in progressive muscle weakness, loss of ambulation, and cardiorespiratory complications. Direct estimation of health-related quality of life for patients with DMD is challenging, highlighting the need for proxy measures. This study aims to catalog and compare existing published health state utility estimates for DMD and related conditions. METHODS: Using two search strategies, relevant utilities were extracted from the Tufts Cost-Effectiveness Analysis Registry, including health states, utility estimates, and study and patient characteristics. Analysis One identified health states with comparable utility estimates to a set of published US patient population utility estimates for DMD. A minimal clinically important difference of ± 0.03 was applied to each DMD utility estimate to establish a range, and the registry was searched to identify other health states with associated utilities that fell within each range. Analysis Two used pre-defined search terms to identify health states clinically similar to DMD. Mapping was based on the degree of clinical similarity. RESULTS: Analysis One identified 4,308 unique utilities across 2,322 cost-effectiveness publications. The health states captured a wide range of acute and chronic conditions; 34% of utility records were extrapolated for US populations (n = 1,451); 1% were related to pediatric populations (n = 61). Analysis Two identified 153 utilities with health states clinically similar to DMD. The median utility estimates varied among identified health states. Health states similar to the early non-ambulatory DMD phase exhibited the greatest difference between the median estimate of the sample (0.39) and the existing estimate from published literature (0.21). CONCLUSIONS: When available estimates are limited, using novel search strategies to identify utilities of clinically similar conditions could be an approach for overcoming the information gap. However, it requires careful evaluation of the utility instruments, tariffs, and raters (proxy or self).
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Distrofia Muscular de Duchenne , Calidad de Vida , Humanos , Estado de Salud , Masculino , Sistema de Registros , Análisis Costo-Beneficio , Niño , Años de Vida Ajustados por Calidad de VidaRESUMEN
OBJECTIVE: Despite the beneficial effects of DOACs, suboptimal adherence is widely documented, and real-world adherence is lower than in clinical trials. The objective of this study is to compare the cost-effectiveness of apixaban versus rivaroxaban for stroke prevention by incorporating real-world adherence from the US payer's perspective. METHODS: We developed a Markov model with three health states to evaluate the total costs and quality-adjusted life years (QALY) at a willingness to pay threshold of $100,000. The population was a hypothetical cohort of 65-year-old patients with moderate to high stroke risk. The transition probabilities of healthy adherent, nonadherent, and stroke were obtained from a Medicare Advantage Plan. The utilities and costs were obtained from prior clinical studies. Deterministic and probabilistic sensitivity analyses were conducted. RESULTS: Apixaban was cost-effective than rivaroxaban at a willingness to pay threshold of $100,000. Apixaban yielded an additional 0.12 QALYs at a cost of $1904.39, resulting in an incremental cost-effectiveness ratio (ICER) of $16,279.25 per QALY gained. The Monte Carlo simulations indicated that apixaban was cost-effective at 89.67% of simulations. The ICER results were impacted by the medical costs among nonadherent patients. CONCLUSION: After incorporating adherence, apixaban 5 mg was a cost-effective alternative to rivaroxaban 20 mg for stroke prevention among elderly atrial fibrillation (AF) patients.
Due to the improved safety and efficacy profile, DOACs are preferred over warfarin for stroke prevention in AF patients. Suboptimal adherence to DOACs is commonly reported in real-world settings, leading to poorer patient outcomes. Prior pharmacoeconomic analyses have not incorporated real-world adherence data. The findings suggest that apixaban 5 mg is a cost-effective alternative to rivaroxaban 20 mg for stroke prevention. Apixaban was identified as the cost-effective option compared with rivaroxaban for stroke prevention among elderly AF patients after incorporating adherence data in the cost-effectiveness analysis. Healthcare decision-makers can utilize the findings of this study to inform formulary decisions and treatment guidelines.
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BACKGROUND: Active-duty service women rely on the civilian sector for most abortion care due to limits on federal funding for abortion. Abortion is now banned in many states with large military presences. The Department of Defense has implemented policies to assist active-duty service women in accessing abortion, but there is debate to reverse this support. OBJECTIVE: Our goal was to compare the cost-effectiveness and incidence of adverse maternal and neonatal outcomes of a hypothetical cohort of active-duty service women living in abortion-restricted states comparing restricted abortion access (abortion not available cohort) to abortion available with Department of Defense travel support (abortion available cohort). STUDY DESIGN: We developed a decision tree model to compare abortion not available and abortion available cohorts for active-duty service women living in abortion-restricted states. Our cohorts were subdivided into normal pregnancies and those with a major fetal anomaly. Cost estimates, probabilities, and disability weights of various health conditions associated with abortion and pregnancy were obtained and derived from the literature. Effectiveness was expressed in disability-adjusted life years and the willingness to pay threshold was set to $100,000 per disability-adjusted life year gained or averted. We completed probabilistic sensitivity analyses with 10,000 simulations to test the robustness of our results. Secondary outcomes included numbers of stillbirths, neonatal deaths, neonatal intensive care unit admissions, maternal deaths, severe maternal morbidities, and first and second trimester abortions. RESULTS: The abortion not available cohort had a higher annual cost to the military ($299.1 million, 95% CI 239.2 - 386.6, vs. $226.0 million, 95% CI 181.9 - 288.5) and was associated with 203 more disability-adjusted life years compared to the abortion available cohort. The incremental cost effectiveness ratio was dominant for abortion available. Abortion not available resulted in an annual additional 7 stillbirths, 1 neonatal death, 112 neonatal intensive care unit admissions, 0.016 maternal deaths, 24 severe maternal morbidities, 27 less second trimester abortions, and 602 less first trimester abortions. Probabilistic sensitivity analysis revealed that the chance of the abortion available cohort being the more cost-effective strategy was greater than 95%. CONCLUSION: Limiting active-duty service women's access to abortion care increases costs to the military, even with costs of travel support, and increases adverse maternal and neonatal outcomes. This analysis provides important information for policymakers about economic and health burdens associated with barriers to abortion care in the military.
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Background: Evidence on the long-term benefits and cost-effectiveness of colorectal cancer (CRC) screening strategies in China remains limited. This modelling study aims to address this issue for various CRC screening strategies in China between 2020 and 2060. Methods: Using a previously developed microsimulation model (MIMIC-CRC) with Chinese epidemiological data, we evaluated four CRC screening strategies targeting population aged 45-74 years: no screening, colonoscopy every 10 years, biennial faecal immunochemical testing (FIT), and a roll-out FIT screening strategy. Screening coverage (invitation) rates from 5% to 100% were analysed. Single-cohort analysis of 100,000 individuals was conducted to estimate the relative cost-effectiveness of each strategy. A multiple-cohort analysis of 100,000 people aged 40+ over 2020-2060 was conducted to project nation-wide long-term benefits and cost-effectiveness. Findings: In single-cohort analysis, all strategies yielded reductions in CRC incidence and mortality compared to no screening, with colonoscopy outperforming FIT-based strategies at the same invitation rates. In multiple-cohort analysis, among people over 40 years of age in China over 2020-2060, compared to no screening, at invitation rate of 5%, screening by colonoscopy, biennial FIT and roll-out FIT-based approach were estimated to avert 1.2, 0.4, and 0.3 million incident CRCs and 0.2, 0.1, and 0.1 million CRC-related deaths, respectively, compared to no screening (25.4 million incident CRCs and 4.4 million CRC-related deaths), and this preventive effect enlarged as the screening coverage rate increased. At full coverage, colonoscopy achieved the largest reductions (38.2% lower incidence and 43.2% lower mortality) but required the most resources. Biennial FIT and roll-out FIT-based approach screening was slightly less effective but had significant reduced colonoscopy needs (reduction of 83.8% and 85.2%, respectively) and overall cost (reduction of 23.4% and 37.8%, respectively) compared to colonoscopy screening. Interpretation: Nation-wide implementation of screening would be effective in reducing the burden of CRC in China. Biennial FIT and roll-out FIT-based screening strategies could prevent incident CRC cases and CRC-related deaths with considerably fewer resources than colonoscopy screening. Efforts should be made to increase the screening coverage in China. Funding: Chinese Academy of Medical Science Innovation Fund for Medical Science (2022-I2M-1-0031); National Natural Science Foundation of China (82173606; 82273726); Beijing Nova Program of Science and Technology (20230484397).
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BACKGROUND: Efficiency, equity and financial risk protection are key health systems objectives. Equitable distribution of health care is among the priority strategic initiative of the government of Ethiopia. However, data on the distribution of interventions benefits or on disease burden disaggregated by subpopulations to guide health care priority setting is not available in Ethiopia. METHODS: Aligned with policy documents, we identified the following groups to be the worse off in the Ethiopian context: under-five children, women of reproductive age, the poor, and rural residents. We used the Delphi technique by a panel of 28 experts to assign a score for 253 diseases/conditions over a period of two days, in phases. The expert panel represented different institutes and professional mix. Experts assigned a score 1 to 4; where 4 indicates disease/condition predominantly affecting the poor and rural residents and 1 indicates a condition more prevalent among the wealthy and urban residents. Subsequently, the average equity score was computed for each disease/condition. RESULTS: The average scores ranged from 1.11 (for vitiligo) to 3.79 (for obstetric fistula). We standardized the scores to be bounded between 1 and 2; 1 the lowest equity score and 2 the highest equity score. The scores for each disease/condition were then assigned to their corresponding interventions. We used these equity scores to adjust the CEA values for each of the interventions. To adjust the CEA values for equity, we multiplied the health benefits (the denominator of the cost-effectiveness value) of each intervention by the corresponding equity scores, resulting in equity adjusted CEA values. The equity adjusted CEA was then used to rank the interventions using a league table. CONCLUSIONS: The Delphi method can be useful in generating equity scores for prioritizing health interventions where disaggregated data on the distribution of diseases or access to interventions by subpopulation groups are not available.
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Técnica Delphi , Seguro de Salud , Humanos , Etiopía , Femenino , Seguro de Salud/economía , Población Rural , Equidad en Salud , Pobreza , Beneficios del Seguro , MasculinoRESUMEN
BACKGROUNDS: Biologics and JAK inhibitors were the most effective innovative systemic treatments for moderate-to-severe atopic dermatitis (AD). However, their cost-effectiveness in China remains unclear. This study aims to compare both the short- and long-term cost-effectiveness of abrocitinib and dupilumab in adults with moderate-to-severe AD from the perspective of the Chinese healthcare system. METHODS: A hybrid decision tree and Markov model were developed to simulate the costs and health outcomes of interventions on both short-term and long-term horizons. Short- and long-term horizons were employed to reflect the 26-week induction treatment and model the extended 10-year maintenance treatment period, respectively. The cost-effectiveness of strategies was measured by incremental cost-effectiveness ratios (ICERs), which were then compared with the willingness-to-pay threshold (WTP) that was equivalent to the gross domestic product (GDP) per capita of China in 2023 ($12,681 [11679.26]). One-way and probabilistic sensitivity analyses were conducted to validate the robustness of the model. RESULTS: Over the short-term horizon, the QALYs (quality-adjusted life years) gained were 0.43 for the abrocitinib group and 0.42 for the dupilumab group, with the costs being $2,716.01 (2501.46) and $3,940.33 (3629.06), respectively. Over the long-time horizon, abrocitinib therapy yields higher QALYs (6.60 versus 6.53) and incurs a lower cost ($22,765.15 [20966.81] versus $30,683.38 [28259.54]) compared to dupilumab. The probability of abrocitinib being cost-effective was nearly 100% under the current WTP. Both short- and long-term results showed that abrocitinib was more effective and less costly than dupilumab, making abrocitinib the dominant option. CONCLUSIONS: Abrocitinib was dominant compared to dupilumab both over the short- and long-term horizon for moderate-to-severe AD in China. Future research incorporating real-world evidence and long-term efficacy outcomes could further refine these economic evaluations.
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BACKGROUND: This study assessed the cost-effectiveness of proton beam therapy (PBT) compared to conventional radiotherapy (CRT) for treating patients with brain tumors in Sweden. METHODS: Data from a longitudinal non-randomized study performed between 2015 and 2020 was used, and included adult patients with brain tumors, followed during treatment and through a one-year follow-up. Clinical and demographic data were sourced from the longitudinal study and linked to Swedish national registers to get information on healthcare resource use. A cost-utility framework was used to evaluate the cost-effectiveness of PBT vs. CRT. Patients in PBT group (n = 310) were matched with patients in CRT group (n = 40) on relevant observables using propensity score matching with replacement. Costs were estimated from a healthcare perspective and included costs related to inpatient and specialized outpatient care, and prescribed medications. The health outcome was quality-adjusted life-years (QALYs), derived from the EORTC-QLQ-C30. Generalized linear models (GLM) and two-part models were used to estimate differences in costs and QALYs. RESULTS: PBT yielded higher total costs, 14,639 US$, than CRT, 13,308 US$, with a difference of 1,372 US$ (95% CI, -4,914-7,659) over a 58 weeks' time horizon. Further, PBT resulted in non-significantly lower QALYs, 0.746 compared to CRT, 0.774, with a difference of -0.049 (95% CI, -0.195-0.097). The probability of PBT being cost-effective was < 30% at any willingness to pay. CONCLUSIONS: These results suggest that PBT cannot be considered a cost-effective treatment for brain tumours, compared to CRT. TRIAL REGISTRATION: Not applicable.
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We aimed to directly compare the cost-effectiveness of Hungarian (following the NELSON trial) and NLST screening protocols, two trials influencing lung-cancer-screening implementation internationally. A decision-analytic model analyzing the cost-effectiveness of Hungarian protocols was manipulated to reflect the protocols of the NLST, while maintaining features specific to the Hungarian healthcare setting. In the Hungarian protocol, there are three possible outcomes to the initial round of screening, positive, negative, and indeterminate, indicating an uncertain degree of suspicion for lung cancer. This protocol differs from the NLST, in which the only possible screening outcomes are positive or negative, with no indeterminate option. The NLST pathway for smokers aged 55-74 resulted in a EUR 43 increase in the total average lifetime costs compared to the Hungarian screening pathway and resulted in a lifetime gain of 0.006 QALYs. The incremental costs and QALYs yielded an ICER of 7875 EUR/QALY. Our results demonstrate that assigning any suspicious LDCT screen as a positive result (NLST protocol) rather than indeterminate (Hungarian protocol) can reduce patient uncertainty and yield a slight QALY gain that is worth the additional use of resources according to Hungary's willingness-to-pay threshold. A stratified analysis by age was also conducted, revealing decreasing cost-effectiveness when screening older cohorts. Our study provides insight into the cost-effectiveness, advantages, and disadvantages of various LDCT screening protocols for lung cancer and can assist other countries as they implement their screening programs.
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BACKGROUND: US News and World Report (USNWR) hospital rankings influence patient choice of hospital, but their association with surgical outcomes remains ill-defined. We sought to characterize clinical outcomes and costs of surgery for colon cancer among USNWR top ranked and unranked hospitals. METHODS: Using Medicare Standard Analytic Files, patients aged ≥65 years undergoing surgery for colon cancer were identified. Hospitals were categorized as 'ranked' or 'unranked' based on USNWR cancer hospital rankings. One-to-one matching was performed between patients treated at ranked and unranked hospitals, and clinical outcomes and costs of surgery were compared. RESULTS: Among 50 ranked and 2522 unranked hospitals, 13,650 patient pairs were compared. Overall, 30-day mortality was 2.13% in ranked hospitals versus 3.68% in unranked hospitals (p < 0.0001), and the overall paired cost difference was $8159 (p < 0.0001). As patient risk increased, 30-day mortality differences became larger, with the ranked hospitals having 30-day mortality of 7.59% versus 11.84% for unranked hospitals among the highest-risk patients (p < 0.0001). Overall paired cost differences also increased with increasing patient risk, with cost of care being $72,229 for ranked hospitals versus $56,512 for unranked hospitals among the highest-risk patients (difference = $14,394; p = 0.02). The difference in cost per 1% reduction in 30-day mortality was $9009 (95% confidence interval [CI] $6422-$11,597) for lowest-risk patients, which dropped to $3387 (95% CI $2656-$4119) for highest-risk patients (p < 0.0001). CONCLUSION: Treatment at USNWR-ranked hospitals, particularly for higher-risk patients, was associated with better outcomes but higher-cost care. The benefit of being treated at highly ranked USNWR hospitals was most pronounced among high-risk patients.
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Background: Lynch syndrome is an inherited condition which leads to an increased risk of colorectal, endometrial and ovarian cancer. Risk-reducing surgery is generally recommended to manage the risk of gynaecological cancer once childbearing is completed. The value of gynaecological colonoscopic surveillance as an interim measure or instead of risk-reducing surgery is uncertain. We aimed to determine whether gynaecological surveillance was effective and cost-effective in Lynch syndrome. Methods: We conducted systematic reviews of the effectiveness and cost-effectiveness of gynaecological cancer surveillance in Lynch syndrome, as well as a systematic review of health utility values relating to cancer and gynaecological risk reduction. Study identification included bibliographic database searching and citation chasing (searches updated 3 August 2021). Screening and assessment of eligibility for inclusion were conducted by independent researchers. Outcomes were prespecified and were informed by clinical experts and patient involvement. Data extraction and quality appraisal were conducted and results were synthesised narratively. We also developed a whole-disease economic model for Lynch syndrome using discrete event simulation methodology, including natural history components for colorectal, endometrial and ovarian cancer, and we used this model to conduct a cost-utility analysis of gynaecological risk management strategies, including surveillance, risk-reducing surgery and doing nothing. Results: We found 30 studies in the review of clinical effectiveness, of which 20 were non-comparative (single-arm) studies. There were no high-quality studies providing precise outcome estimates at low risk of bias. There is some evidence that mortality rate is higher for surveillance than for risk-reducing surgery but mortality is also higher for no surveillance than for surveillance. Some asymptomatic cancers were detected through surveillance but some cancers were also missed. There was a wide range of pain experiences, including some individuals feeling no pain and some feeling severe pain. The use of pain relief (e.g. ibuprofen) was common, and some women underwent general anaesthetic for surveillance. Existing economic evaluations clearly found that risk-reducing surgery leads to the best lifetime health (measured using quality-adjusted life-years) and is cost-effective, while surveillance is not cost-effective in comparison. Our economic evaluation found that a strategy of surveillance alone or offering surveillance and risk-reducing surgery was cost-effective, except for path_PMS2 Lynch syndrome. Offering only risk-reducing surgery was less effective than offering surveillance with or without surgery. Limitations: Firm conclusions about clinical effectiveness could not be reached because of the lack of high-quality research. We did not assume that women would immediately take up risk-reducing surgery if offered, and it is possible that risk-reducing surgery would be more effective and cost-effective if it was taken up when offered. Conclusions: There is insufficient evidence to recommend for or against gynaecological cancer surveillance in Lynch syndrome on clinical grounds, but modelling suggests that surveillance could be cost-effective. Further research is needed but it must be rigorously designed and well reported to be of benefit. Study registration: This study is registered as PROSPERO CRD42020171098. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: NIHR129713) and is published in full in Health Technology Assessment; Vol. 28, No. 41. See the NIHR Funding and Awards website for further award information.
Lynch syndrome is an inherited condition which puts people at a higher risk of getting bowel cancer, womb cancer and ovarian cancer. Although people with Lynch syndrome are more likely to get these cancers, they are more likely to survive cancer if they get it. People diagnosed with Lynch syndrome get regular testing (surveillance) using a camera to check for bowel cancer or polyps. For womb and ovarian cancer, surveillance may also be an option, but it is less well studied in these cancers. This means that many women are not offered surveillance. Women with Lynch syndrome are recommended to have risk-reducing surgery when their risk starts rising, if they do not want any more children. We wanted to find out whether surveillance for womb and ovarian cancer would work and would be good value for money. Doctors and patients have said that these are important research questions. We searched for published research on this subject and found a lot of studies, but these studies were often small or not well designed, so they could only tell us a limited amount. Studies did not always measure the things that patients want to know. There was some evidence that people having surveillance might live longer than people not having surveillance, but there was also some evidence that risk-reducing surgery is better than surveillance. Surveillance has detected some cancers which had no symptoms, but there are also cancers diagnosed soon after a surveillance visit where nothing was found. People often find surveillance painful, but experiences vary. Our work shows that surveillance and surgery could be good value for money for many women with Lynch syndrome. We need better research to help patients and doctors decide whether surveillance is right for them.