Incremental Net Monetary Benefit Research Articles

Real-world cost-effectiveness of multi-gene panel sequencing to inform therapeutic decisions for advanced non-small cell lung cancer: a population-based study

Multi-gene panel sequencing streamlines treatment selection for advanced non-small cell lung cancer (NSCLC). Implementation continues to be uneven across jurisdictions, partly due to uncertain clinical and economic impacts. In British Columbia (BC), Canada, the public healthcare system reimbursed a multi-gene panel in September 2016. This study determined the population-level cost-effectiveness of publicly reimbursed multi-gene panel sequencing compared to single-gene testing for advanced NSCLC. Our population-based retrospective study design used patient-level linked administrative health databases. We considered adult BC residents with a panel-eligible lung cancer diagnosis between September 2016 and December 2018. Using a machine learning approach, we conducted 1:1 genetic algorithm matching of recipients receiving multi-gene panel sequencing to controls receiving single-gene testing, maximising balance on observed demographic and clinical characteristics. Following matching, we estimated mean three-year survival time and costs (public healthcare payer perspective; 2021 CAD) and calculated the incremental net monetary benefit (INMB) for life-years gained (LYG) at conventional willingness-to-pay thresholds using inverse probability of censoring weighted linear regression and nonparametric bootstrapping. We matched 858 panel-eligible advanced NSCLC patients to controls, achieving balance for the 16 included covariates. Average test turnaround times were 18.6 days for multi-gene panel sequencing and 7.0 days for single-gene testing. After matching, mean incremental costs were $3529 (95% CI:-$4268, $10,942) and mean incremental LYG were 0.08 (95% CI:-0.04, 0.18). Among the 1000 bootstrap samples, 14.5% had lower costs and increased survival and 78.6% had higher costs and increased survival. The INMB was $523 (95% CI:-$6256, $7023) at $50,000/LYG, with a 57.5% probability of being cost-effective, and $4575 (95% CI:-$5468, $14,064) at $100,000/LYG, with an 84.0% probability of being cost-effective. Using population-based real-world data, we found a moderate to high probability that panel-based testing to inform targeted treatment for NSCLC would be cost-effective at higher thresholds. This research was supported by Genome British Columbia/Genome Canada (G05CHS) and the Terry Fox Research Institute.

Economic evaluation of nine intersectoral strategies to improve youth mental health and alleviate financial burden in Colombia using system dynamics modelling

ObjectivesLimited evidence exists for strategic actions in improving the youth population-level mental health, particularly in low- and middle-income countries. This study investigates the health and economic impacts of inter-sectoral mental health programs to inform the optimal suite of interventions for young people in Bogotá, Colombia, where many are at risk of mental disorders and lack access to appropriate health services. MethodsThis study examined nine interventions compared to business-as-usual over a projected 10-year time horizon using a system dynamics model developed through a participatory process involving diverse local stakeholders. Sensitivity analyses were performed to account for uncertainties from a healthcare payer perspective. ResultsAnti-bullying, community-based rehabilitation, and online mental health programs were dominant. GP mental health training and gatekeeper suicide prevention training could significantly improve mental health outcomes at an incremental cost-effectiveness ratio (ICER) below the threshold of 4,890 USD/QALY. However, simultaneously implementing all nine strategies could produce a synergistic effect, yielding the greatest benefit relative to business-as-usual with an incremental net monetary benefit of USD 465 million and an ICER of USD 586 per QALY gained. ConclusionsCombined intersectoral mental health strategies were synergistic and resulted in optimum health outcomes relative to business-as-usual. System dynamics approach offers additional attributes to economic evaluation in mental health settings by considering capacity constraints, unintended consequences, and interactions between multiple interventions. It is a valuable approach for decision-makers to effectively and efficiently prioritise and allocate scarce resources in complex public health systems.

Cost-Effectiveness of Corneal Collagen Crosslinking for Progressive Keratoconus: A Brazilian Unified Health System Perspective

Keratoconus is a burden to health systems and patients worldwide. Corneal collagen crosslinking (CXL) treatment has been shown abroad to be cost-effective for treating progressive keratoconus. However, no cost-effectiveness studies have been performed in Brazil. The aim of this study was to assess the cost-effectiveness of corneal collagen crosslinking (CXL) compared with the conventional treatment for progressive keratoconus from the Brazilian Unified Health System (SUS) payer’s perspective. A lifetime microsimulation model was utilized to compare the lifetime costs and quality-adjusted life years for patients undergoing corneal collagen CXL or conventional treatment. Two groups of 5000 18-year-old patients were simulated, with one group receiving corneal CXL at the outset and the control group remaining untreated. The TreeAge Pro Healthcare 2024 software was used for modeling and analysis. Corneal collagen CXL demonstrated superior cost-effectiveness compared to the conventional treatment, with an incremental cost-effectiveness ratio of 58.26 USD/quality-adjusted life years (QALY) gained (95% CI: 58.17–58.36) and a positive incremental net monetary benefit of USD 11,613.82 (95% CI: 11,605.66–11,621.99). CXL significantly reduced the number of required corneal transplants, with a mean of 968.8 (95% CI: 959–978.58) fewer transplants per 10,000 eyes treated. The variable with the most significant impact on the incremental net monetary benefit was the duration of the CXL effect. This study concluded that corneal CXL is a highly cost-effective intervention for progressive keratoconus within the Brazilian SUS. These findings advocate for broader accessibility to this vision-saving treatment within the SUS.

Cost-effectiveness analysis of tislelizumab plus chemotherapy versus placebo plus chemotherapy as first-line treatment for advanced gastric or gastroesophageal junction adenocarcinoma: perspectives from the United States and China.

The efficacy of tislelizumab plus chemotherapy in improving progression-free survival (PFS) and overall survival (OS) in unresectable gastric or gastroesophageal junction cancer (GC/GEJC) has recently been emphasized. This study compared the cost-effectiveness of tislelizumab plus chemotherapy versus placebo plus chemotherapy for the United States (US) and Chinese populations. Using data from the RATIONALE-305 phase 3 trial, a Markov model was developed to analyze quality-adjusted life years (QALYs), incremental cost-effectiveness ratios (ICERs), incremental net health benefits (INHBs), and incremental net monetary benefits (INMBs). The health state utilities and direct medical costs were obtained from the relevant literature and local cost databases. The model uncertainty was evaluated using sensitivity analyses. In the base-case analysis, the addition of tislelizumab to chemotherapy yielded an ICER of $37,768.48 per QALY in China, slightly below the willingness-to-pay (WTP) threshold of $38,042.49 per QALY, showing marginal cost-effectiveness with an INHB of 0.05 QALYs and an INMB of $1,852.49. Subgroup analyses revealed ICERs of $23,853.52 for patients with a PD-L1 TAP score ≥ 5% (TAP ≥ 5%). In the US, the ICER was $502,786.22 per QALY in the intent-to-treat (ITT) and $321,395.28 per QALY in the TAP ≥ 5% subgroup, exceeding the US WTP threshold of $150,000.00. In China, tislelizumab plus chemotherapy is a cost-effective first-line therapy for unresectable GC/GEJC in both ITT and TAP ≥ 5% subgroups. In the US, tislelizumab plus chemotherapy is not cost-effective.

Health Economic Evaluation of Antimicrobial Stewardship, Procalcitonin Testing, and Rapid Blood Culture Identification in Sepsis Care: A 90-Day Model-Based, Cost-Utility Analysis.

We evaluated the cost-effectiveness of a bundled intervention including an antimicrobial stewardship program (ASP), procalcitonin (PCT) testing, and rapid blood culture identification (BCID), compared with pre-implementation standard care in critically ill adult patients with sepsis. We conducted a decision tree model-based cost-effectiveness analysis alongside a previously published pre- and post-implementation quality improvement study. We adopted a public Canadian healthcare payer's perspective. Two intensive care units in Alberta with 727 adult critically ill patients were included. Our bundled intervention was compared with pre-implementation standard care. We collected healthcare resource use and estimated unit costs in 2022 Canadian dollars (CAD) over a time horizon from study entry to hospital discharge or death. We calculated the incremental net monetary benefit (iNMB) of the intervention group compared with the pre-intervention group. The primary outcome was cost per sepsis case. Secondary outcomes included readmission rates, Clostridioides difficile infections, mortality, and lengths of stay. Uncertainty was investigated using cost-effectiveness acceptability curves, cost-effectiveness plane scatterplots, and sensitivity analyses. Mean (standard deviation [SD]) cost per index hospital admission was CAD $83,251 ($107,926) for patients in the intervention group and CAD $87,044 ($104,406) for the pre-intervention group, though the difference ($3,793 [$7,897]) was not statistically significant. Costs were higher in the pre-intervention group for antibiotics, readmissions, and C. difficile infections. The intervention group had a lower mean expected cost; $110,580 ($108,917) compared with pre-intervention ($125,745 [$113,210]), with a difference of $15,165 ($8278). There were no statistically significant differences in quality adjusted life years (QALYs) between groups. The iNMB of the intervention group compared with pre-intervention was greater than $15,000 for willingness-to-pay (WTP) per QALY values of between $0 and $100,000. In our sensitivity analysis, the intervention was most likely to be cost-effective in roughly 56% of simulations at all WTP thresholds. Our bundled intervention of ASP, PCT, and BCID among adult critically ill patients with sepsis was potentially cost-effective, but with substantial decision uncertainty.

Cost-effectiveness of a short-course antibiotic treatment strategy for the treatment of ventilator-associated pneumonia: an economic analysis of the REGARD-VAP trial

The REGARD-VAP trial showed that individualised shortened antibiotic therapy was non-inferior to usual care for mortality and pneumonia recurrence in patients with ventilator-associated pneumonia (VAP). We aimed to assess the cost-effectiveness of an individualised shortened antibiotic therapy approach in this planned economic analysis. REGARD-VAP was a phase 4, multicentre, open-label, randomised trial to assess a short-course antibiotic treatment strategy for treatment of VAP. In this planned economic analysis, we fitted a decision tree with data from the REGARD-VAP trial to estimate the cost-effectiveness of individualised short-course therapy for VAP, compared to usual care from the health system perspective, in Nepal, Singapore, and Thailand. Incremental cost-effectiveness ratios (ICERs) and incremental net monetary benefits with 95% uncertainty intervals (UIs) were reported against relevant willingness-to-pay thresholds. Parameter uncertainties were evaluated using scenario analyses. A value of information analysis was conducted. Adopting individualised short-course therapy was cost-effective for Nepal (ICER=US$1086; percentage cost-effectiveness=50·3%), Singapore (ICER=-$6069; percentage cost-effectiveness=55·2%), and Thailand (ICER=$263; percentage cost-effectiveness=60·5%). The associated incremental net monetary benefits were $41 (95% UI -2308 to 2390) in Nepal, $5156 (-45 805 to 56 117) in Singapore, and $804 (-6245 to 7852) in Thailand. Value of information analysis showed that reducing uncertainties for mortality probabilities, bed-day costs, and variable costs were valuable for decision making. We found that an individualised short-course antibiotics strategy in patients with VAP is likely to be cost-effective in high-income, middle-income, and low-income settings, although with evident uncertainty. Considered alongside the positive externalities of reduced antimicrobial use, our findings foster confidence in policy makers contemplating adoption of short-course antibiotics. UK Medical Research Council, Singapore National Medical Research Council, and Wellcome Trust.

Cost-effectiveness of strengthening blood pressure classification in South Korea: comparing the 2017 ACC/AHA and KSH guidelines

BackgroundHypertension is a significant risk factor for cardiovascular disease (CVD), with hypertension-related deaths increasing annually. While South Korea uses the Korean Society of Hypertension (KSH) guideline of 140/90 mmHg, the American College of Cardiology (ACC) and American Heart Association (AHA) updated their guidelines in 2017 to 130/80 mmHg. This study evaluates the cost-effectiveness of transitioning to the 2017 ACC/AHA guidelines by estimating early treatment impacts and potential CVD risk reduction.MethodsA Markov state-transition simulation model with a 10-year horizon was used to estimate cost-effectiveness, focusing on strengthening target blood pressure. Quality-adjusted life years (QALYs) served as the measure of effectiveness. Cohorts of 10,000 individuals representing South Koreans in their 20s through 80s were compared in scenario analyses from the healthcare system perspective. A 4.5% annual discount rate was applied to costs and effectiveness. Primary outcomes were incremental cost-effectiveness ratio (ICER) and net monetary benefit (NMB). The willingness-to-pay (WTP) threshold was < KRW 30,000,000/QALY gained. Probabilistic sensitivity analyses (PSAs) addressed model input parameter uncertainties.ResultsThe base-case analysis showed an ICER value of KRW 1,328,395/QALY gained across all populations. ICER values increased with age, from − KRW 3,138,071/QALY for 20-year-olds to KRW 16,613,013/QALY for individuals over 80. The 60s age group showed the greatest benefit with an incremental QALY gain of 0.46. All scenarios had ICERs below the WTP threshold, with a PSA probability of 98.0% that strengthening blood pressure classification could be cost-effective.ConclusionsThis economic evaluation found that adopting the 2017 ACC/AHA guidelines may result in early treatment, reduce the potential incidence of CVD events, and be cost-effective across all age groups. The study findings have implications for policymakers deciding whether and when to revise official guidelines regarding target blood pressure levels, considering the impacts on public health and budgetary concerns.

Health impact and cost-effectiveness of vaccination using potential next-generation influenza vaccines in Thailand: a modelling study

IntroductionThailand was one of the first low- and middle-income countries to publicly fund seasonal influenza vaccines, but the lack of predictability in the timing of epidemics and difficulty in predicting the dominant influenza subtypes present a challenge for existing vaccines. Next-generation influenza vaccines (NGIVs) are being developed with the dual aims of broadening the strain coverage and conferring longer-lasting immunity. However, there are no economic evaluations of NGIVs in Thailand.MethodsWe estimated the health impact and cost-effectiveness of NGIVs in Thailand between 2005 and 2009 using a combined epidemiological and economic model. We fitted the model to data on laboratory-confirmed influenza cases and then simulated the number of influenza infections, symptomatic cases, hospitalisations and deaths under different vaccination scenarios based on WHO-preferred product characteristics for NGIVs. We used previous estimates of costs and disability adjusted life years (DALYs) for influenza health outcomes to estimate incremental net monetary benefit, vaccine threshold prices and budget impact.ResultsWith the current vaccine programme, there were an estimated 61 million influenza infections. Increasing coverage to 50% using improved vaccines reduced infections to between 23 and 57 million, and with universal vaccines to between 21 and 49 million, depending on the age groups targeted. Depending on the comparator, threshold prices for NGIVs ranged from US$2.80 to US$12.90 per dose for minimally improved vaccines and US$24.60 to US$69.90 for universal vaccines.ConclusionInfluenza immunisation programmes using NGIVs are anticipated to provide considerable health benefits and be cost-effective in Thailand. However, although NGIVs might even be cost-saving in the long run, there could be significant budget implications for the Thai government even if the vaccines can be procured at a substantial discount to the maximum threshold price.

Cost-effectiveness of oral versus injectable disease modifying therapies in relapsing multiple sclerosis: a systematic review analysis

BackgroundMultiple sclerosis (MS) is a chronic and progressive neurological autoimmune disease that affects the central nervous system. There are two types of drugs used to treat this disease: injectable and oral drugs. The present study aimed at systematically reviewing the cost effectiveness of oral versus injectable drugs.MethodsThe researchers searched the PubMed, Scopus, and Web of Science databases to find relevant studies. After removing the duplicates, two authors independently assessed the records. The studies that had conducted full economic evaluations of oral versus injectable drugs in MS patients were included. The Quality of Health Economic Studies (QHES) tool was also used to assess the quality of the studies.ResultsThirty studies that had conducted the economic analysis of oral versus injectable therapies in MS patients were included in this review. The QHES scores for all records were generally high (≥ 77) and they were of good quality. The lowest and highest levels of incremental net monetary benefit were respectively obtained through the comparison of Fingolimod and Alemtuzumab (-1,419,333) and the comparison of Teriflunomide and Interferon β-1a (1,792,810). The amount of INMB (incremental net monetary benefit) in the comparisons between oral and injectable drugs showed that the highest and lowest amount of INMB calculated between) Fingolimod and injectable drugs, respectively, compared to (interferon β-1a) 98,253 and (Ocrelizumab) -212,417, the highest amount in dimethyl fumarate is also against (peginterferon β-1a) 191,470 and the lowest against (alemtuzumab) -124,333, Teriflunomide against injectable drugs is the highest against (peginterferon β-1a) 89,956 and the lowest (Ocrelizumab) − 194,169, as well as Cladribine compared to injectable drugs, the highest was compared to (interferon β-1a) 236,430 and the lowest (Ocrelizumab) was 23,965.ConclusionA large number of health economic evaluations of disease-modifying therapies (DMTs) in MS were available at the international level, the comparison of which was difficult and sometimes contradictory. However, despite the difference in the results, Cladribine tablets were cost-effective in all studies compared with injectable drugs. In addition, the present study could be of great importance for policymakers and other beneficiaries regarding the cost-effectiveness of the aforementioned drugs.

Cost-Effectiveness of an Ultrasound-First Strategy in the Diagnostic Evaluation of Noncalcified Lesions Recalled From Screening Digital Breast Tomosynthesis.

BACKGROUND. Ultrasound may be sufficient in the diagnostic evaluation of many noncalcified lesions recalled from screening digital breast tomosynthesis (DBT). In some scenarios, omission of diagnostic mammography can save health care costs. OBJECTIVE. The purpose of this study was to evaluate the cost-effectiveness of a strategy of performing ultrasound first versus diagnostic mammography first in the diagnostic evaluation of noncalcified lesions recalled from screening DBT. METHODS. Decision tree analysis was performed to compare ultrasound first versus diagnostic mammography first in the diagnostic evaluation of DBT-recalled noncalcified lesions from a U.S. health care system perspective with a 40-year horizon. The analysis used probabilities and prevalence information from published single-institution prospective data, additional literature-derived estimates of diagnostic test performance, and Medicare-allowable reimbursement rates. Health states were represented in a Markov chain model. For each strategy, the total cost and effectiveness (expressed in quality-adjusted life-years [QALYs]) were estimated. Cost-effectiveness was assessed through incremental cost-effectiveness ratios (ICERs) and incremental net monetary benefit, with use of a willingness-to-pay (WTP) threshold of US$100,000 per QALY gained. Deterministic sensitivity analyses were performed to estimate the impact of different input parameters, and probabilistic sensitivity analysis with Monte Carlo simulations was conducted to estimate the impact of combined uncertainty across parameters. RESULTS. In the base-case scenario, for diagnostic evaluation of DBT-recalled noncalcified lesions, a strategy of performing ultrasound first versus diagnostic mammography first resulted in more cost savings (total cost, US$17,672 vs US$18,323) and greater cost-effectiveness (QALYs, 23.1309 vs 23.1306) over the 40-year horizon. The ultrasound-first strategy resulted in an ICER of -2,170,000 (expressed as U.S. dollars per QALY) and an incremental net monetary benefit of US$681 versus the diagnostic mammography-first strategy. Therefore, performing ultrasound first was deemed the more cost-effective strategy at the WTP threshold. In deterministic sensitivity analyses, the most important driver of cost-effectiveness was lost utility from delayed diagnosis, followed by the relative sensitivities of ultrasound and diagnostic mammography. In probabilistic sensitivity analysis, ultrasound first was the better strategy in 93.0% of iterations. CONCLUSION. A strategy of performing ultrasound first, with or without diagnostic mammography, is more cost-effective than a traditional strategy of conducting diagnostic mammography first. CLINICAL IMPACT. This cost-effectiveness analysis supports the growing prioritization of ultrasound as the primary method for evaluating DBT-recalled noncalcified lesions.

Cost-effectiveness of iptacopan for paroxysmal nocturnal hemoglobinuria

AbstractIptacopan, a novel oral factor B inhibitor, recently obtained US Food and Drug Administration approval for treating paroxysmal nocturnal hemoglobinuria, a rare blood disorder characterized by persistent complement-mediated hemolytic anemia. The standard-of-care (SOC) has traditionally relied on complement C5 inhibitors eculizumab and ravulizumab, which are limited by persistent anemia from extravascular hemolysis and requirement for intravenous infusion. Recent publication of phase 3 studies in this arena reinforces iptacopan as an effective anticomplement monotherapy compared with SOC. Given ongoing price negotiations and limited literature showing its cost-ineffectiveness in the anti-C5–treated population, we conducted a comprehensive cost-effectiveness analysis of iptacopan monotherapy in anti-C5–treated patients from the societal perspective, as compared with C5 inhibition. The primary outcomes were the incremental net monetary benefit across a lifetime horizon and the cost-effective maximum monthly threshold price of iptacopan monotherapy compared with the SOC. The secondary outcome was time saved for patients and nurses with the use of oral iptacopan therapy. Iptacopan monotherapy and SOC accrued 12.6 and 10.8 quality-adjusted life-years at costs of $9.52 million and $13.5 million, respectively. Iptacopan remained cost saving across extensive sensitivity and all scenario analyses, including alternative parameterization for anemia resolution and aggregated individual-level utilities and transition probability matrix. Across all probabilistic sensitivity analyses, iptacopan therapy was favored over SOC in 100% of 10 000 Monte Carlo iterations. Cost-saving thresholds for iptacopan vs anti-C5 in are ∼1.1, 1.4, and 1.4 in Brazil, Japan, and the United States, respectively. Iptacopan monotherapy can improve quality-adjusted life expectancy for patients while saving health care costs across jurisdictions.

Cost-utility and cost consequence of a telehealth intervention targeting improvement in addictive eating for Australian adults (the TRACE program).

The TRACE (Targeted Research for Addictive and Compulsive Eating) intervention was evaluated in a 3-month randomized controlled trial which demonstrated significant improvement in Yale Food Addiction Scale scores favoring dietitian-led telehealth (active intervention) compared with passive and control groups. This study aimed to determine intervention costs and cost-utility. Costs of each intervention (2021$AUD) and incremental net monetary benefit (iNMB; incremental benefit, defined as Quality-Adjusted Life Years (QALY) gained, multiplied by willingness to pay threshold minus incremental cost) were calculated to estimate differences between groups. The active intervention (n=38) cost $294 (95% UI: $266, $316) per person compared to $47 (95% UI: $40, $54) in the passive intervention (n=24), and $26 in the control group (n=37). At a cost-effectiveness threshold of $50 000 per QALY score gained, the active intervention iNMB was -$186 (95% UI: -$1137, $834) and the passive group $127 (95% UI: -$1137, $834). Compared to the control group, estimates indicate a 30% chance of the active intervention, and a 60% chance of the passive intervention being cost effective. Although the overall cost of the active intervention was low, this was not considered cost-effective in comparison to the passive intervention, given small QALY score gains. Australia New Zealand Clinical Trial Registry ACTRN12621001079831.

Lifetime Cost-Effectiveness of Structured Education and Exercise Therapy for Knee Osteoarthritis in Australia.

Structured education and exercise therapy programs have been proposed to reduce reliance on total knee replacement (TKR) surgery and improve health care sustainability. The long-term cost-effectiveness of these programs is unclear. To estimate the lifetime cost-effectiveness of implementing a national structured education and exercise therapy program for individuals with knee osteoarthritis with the option for future TKR compared with usual care (TKR for all). This economic evaluation used a life table model in combination with a Markov model to compare costs and health outcomes of a national education and exercise therapy program vs usual care in the Australian health care system. Subgroup, deterministic, and probabilistic sensitivity analyses were completed. A hypothetical cohort of adults aged 45 to 84 years who would undergo TKR was created. Structured education and exercise therapy intervention provided by physiotherapists. The comparator was usual care where all people undergo TKR without accessing the program in the first year. Incremental net monetary benefit (INMB), with an incremental cost-effectiveness ratio threshold of 28 033 Australian dollars (A$) per quality-adjusted life-year (QALY) gained, was calculated from a health care perspective. Transition probabilities, costs, and utilities were estimated from national registries and a randomized clinical trial. The hypothetical cohort included 61 394 individuals (53.9% female; 93.6% aged ≥55 years). Implementation of an education and exercise therapy program resulted in a lifetime cost savings of A$498 307 942 (US $339 922 227), or A$7970 (US $5537) per individual, and resulted in fewer QALYs (0.43 per individual) compared with usual care. At a population level, education and exercise therapy was not cost-effective at the lifetime horizon (INMB, -A$4090 [-US $2841]). Subgroup analysis revealed that the intervention was cost-effective only for the first 9 years and over a lifetime only in individuals with no or mild pain at baseline (INMB, A$11 [US $8]). Results were robust to uncertainty around model inputs. In this economic evaluation of structured education and exercise therapy compared with usual care, the intervention was not cost-effective over the lifetime for all patients but was for the first 9 years and for those with minimal pain. These findings point to opportunities to invest early cost savings in additional care or prevention, including targeted implementation to specific subgroups.

Cost-Effectiveness of an Extended-Role General Practitioner Clinic for Persistent Physical Symptoms: Results From the Multiple Symptoms Study 3 Pragmatic Randomized Controlled Trial

ObjectivesThis study aimed to evaluate the cost-effectiveness of an extended-role general practitioner symptoms clinic (SC), added to usual care (UC) for patients with multiple persistent physical symptoms (sometimes known as medically unexplained symptoms). MethodsThis was a 52-week within-trial cost-utility analysis of a pragmatic multicenter randomized controlled trial comparing SC + UC (n = 178) with UC alone (n = 176), conducted from the primary perspective of the UK National Health Service and personal and social services (PSS). Base-case quality-adjusted life-years (QALYs) were measured using EQ-5D-5L. Missing data were imputed using multiple imputation. Cost-effectiveness results were presented as incremental cost-effectiveness ratios and incremental net monetary benefits. Uncertainty was explored using cost-effectiveness acceptability curves (using 1000 nonparametric bootstrapped samples) and sensitivity analysis (including societal costs, using SF-6D and ICECAP-A capability measure for adults outcomes to estimate QALYs and years of full capability, respectively, varying intervention costs, missing data mechanism assumptions). ResultsMultiple imputation analysis showed that compared with UC alone, SC + UC was more expensive (adjusted mean cost difference: 704; 95% CI £605-£807) and more effective (adjusted mean QALY difference: 0.0447; 95% CI 0.0067-0.0826), yielding an incremental cost-effectiveness ratio of £15 765/QALY, incremental net monetary benefit of £189.22 (95% CI −£573.62 to £948.28) and a 69% probability of the SC + UC intervention arm being cost-effective at a threshold of £20 000 per QALY. Results were robust to most sensitivity analyses but sensitive to missing data assumptions (2 of the 8 scenarios investigated), SF-6D, and ICECAP_A capability measure for adults quality-of-life outcomes. ConclusionsA symptoms clinic is likely to be a potentially cost-effective treatment for patients with persistent physical symptoms.

Cost-effectiveness analysis of tislelizumab plus chemotherapy versus standard chemotherapy in first-line treatment for extensive-stage small cell lung cancer: perspectives from the United States and China.

Tislelizumab combined with chemotherapy has shown significant clinical benefits in improving overall survival compared to chemotherapy alone for patients with extensive-stage small-cell lung cancer (ES-SCLC). This study aimed to evaluate the cost-effectiveness of tislelizumab plus chemotherapy versus standard chemotherapy as a first-line treatment for ES-SCLC from the US payer perspective and the perspective of the Chinese healthcare system. We conducted an economic evaluation using a Markov state-transition model, reflecting the US payer perspective and the perspective of the Chinese healthcare system. Baseline patient characteristics and essential clinical data were obtained from the RATIONALE-312 trial. The costs and utilities were derived from open-access databases and published literature. The primary outcomes measured included quality-adjusted life years (QALYs), incremental cost-effectiveness ratio (ICER), incremental net health benefit (INHB), and incremental net monetary benefit (INMB). Uncertainties in the model were addressed by probabilistic sensitivity analysis (PSA) and one-way sensitivity analysis (OWSA). In the base-case analysis, the addition of tislelizumab to chemotherapy provided an incremental gain of 0.16 QALYs at an additional cost of $7430.73, resulting in an ICER of $46,132.33 per QALY. Although above the willingness-to-pay (WTP) threshold of China of $38,042.49 per QALY, the cost-effectiveness was marginal, with an INHB of - 0.03 QALYs and an INMB of $- 1303.06. In the US, despite a slightly higher effectiveness gain of 0.28 QALYs, the increased cost of $45,157.35 resulted in an unfavorable ICER of $163,885.06 per QALY, exceeding the US WTP threshold of $150,000.00. PSA showed probabilities of cost-effectiveness of tislelizumab plus chemotherapy at 17.18% in China and 40.41% in the US. Tislelizumab combined with chemotherapy was not a cost-effective first-line treatment option for ES-SCLC in China or the US; however, the margin of cost-effectiveness was narrow.

Cost-effectiveness of adding a smartphone app (BlueIce) to the mental health care of adolescents who repeatedly self-harm

Digital interventions can offer crises support although their cost-effectiveness is unknown. We undertook an economic evaluation alongside a two-arm, single blind, randomised controlled trial. 170 adolescents aged 12–17, receiving child and adolescent mental health care who had self-harmed ≥2 in the past 12 months were randomised to usual care with or without an app (BlueIce). The Risk-Taking and Self-Harm Inventory for Adolescents (RTSHIA), and Child Health Utility 9-Dimensions (CHU-9D) were completed at baseline, 12-weeks, and 6-months. Mental healthcare use was extracted from clinical records. CHU-9D responses were converted to preference-based utility values to estimate quality-adjusted life-years (QALYs). Generalised linear models examined the effect of BlueIce from the NHS and Personal Social Services perspective on costs and QALYs. The cost of BlueIce was £32.26 with the mean cost of mental healthcare over 6 months ranging between £1750 - £2472 per participant. The 6-month difference in mean costs [-£722.09 (95 % CI:1998.84, 334.65)] and the utility score [0.009 (95 %CI:0.033, 0.052)] both favoured BlueIce. Youth derived QALYs showed an incremental net monetary benefit (NMB) at 6-months of £782.09 with an almost 70 % probability of being cost-effective. Given the low intervention cost, the addition of an app could be considered a good investment.

Chemoradiotherapy plus immunotherapy for locoregionally advanced nasopharyngeal carcinoma: A cost-effectiveness analysis.

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. 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]). 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. Adding sintilimab to chemoradiotherapeutic regimens represents an innovative and cost-effective means for patients with locoregionally advanced NPC management in China.

Exploring Heterogeneity in the Cost-Effectiveness of High-Flow Nasal Cannula Therapy in Acutely Ill Children—Insights From the Step-Up First-line Support for Assistance in Breathing in Children Trial Using a Machine Learning Method

ObjectivesTo investigate heterogeneity in the cost-effectiveness of high-flow nasal cannula (HFNC) therapy compared with continuous positive airway pressure (CPAP) for acutely ill children requiring noninvasive respiratory support. MethodsUsing data from the First-line Support for Assistance in Breathing in Children trial, we explore heterogeneity at the patient and subgroup levels using 2 causal forest approaches and a seemingly unrelated regression approach for comparison. First-line Support for Assistance in Breathing in Children is a noninferiority randomized controlled trial (ISRCTN60048867) involving 24 UK pediatric intensive care units. The Step-up trial focuses on acutely ill children aged 0 to 15 years, requiring noninvasive respiratory support. A total of 600 children were randomly assigned to HFNC and CPAP groups in a 1:1 allocation ratio, with 94 patients excluded because of data unavailability. ResultsThe primary outcome is the incremental net monetary benefit (INB) of HFNC compared with CPAP, using a willingness-to-pay threshold of £20 000 per quality-adjusted life year gain. INB is derived from total costs and quality-adjusted life years at 6 months. Subgroup analysis showed that some subgroups, such as male children, those aged less than 12 months, and those without severe respiratory distress at randomization, had more favorable INB results. Patient-level analysis revealed heterogeneity in INB estimates, particularly driven by the cost component, with greater uncertainty for those with higher INBs. ConclusionsThe estimated overall INB of HFNC is significantly larger for specific patient subgroups, suggesting that the cost-effectiveness of HFNC can be heterogeneous, which highlights the importance of considering patient characteristics in evaluating the cost-effectiveness of HFNC.

Arthroscopic Partial Meniscectomy for a Degenerative Meniscus Tear Is Not Cost Effective Compared With Placebo Surgery: An Economic Evaluation Based on the FIDELITY Trial Data.

In patients with a degenerative tear of the medial meniscus, recent meta-analyses and systematic reviews have shown no treatment benefit of arthroscopic partial meniscectomy (APM) over conservative treatment or placebo surgery. Yet, advocates of APM still argue that APM is cost effective. Giving advocates of APM their due, we note that there is evidence from the treatment of other musculoskeletal complaints to suggest that a treatment may prove cost effective even in the absence of improvements in efficacy outcomes, as it may lead to other benefits, such as diminished productivity loss and reduced costs, and so the question of cost effectiveness needs to be answered for APM. (1) Does APM result in lower postoperative costs compared with placebo surgery? (2) Is APM cost-effective compared with placebo surgery? One hundred forty-six adults aged 35 to 65 years with knee symptoms consistent with a degenerative medial meniscus tear and no knee osteoarthritis according to the American College of Rheumatology clinical criteria were randomized to APM (n = 70) or placebo surgery (n = 76). In the APM and placebo surgery groups, mean age was 52 ± 7 years and 52 ± 7 years, and 60% (42 of 70) and 62% (47 of 76) of participants were men, respectively. There were no between-group differences in baseline characteristics. In both groups, a standard diagnostic arthroscopy was first performed. Thereafter, in the APM group, the torn meniscus was trimmed to solid meniscus tissue, whereas in the placebo surgery group, APM was carefully mimicked but no resection of meniscal tissue was performed; as such, surgical costs were the same in both arms and were not included in the analyses. All patients received identical postoperative care including a graduated home-based exercise program. At the 2-year follow-up, two patients were lost to follow-up, both in the placebo surgery group. Cost effectiveness over the 2-year trial period was computed as incremental net monetary benefit (INMB) for improvements in quality-adjusted life years (QALY), using both the societal (primary) and healthcare system (secondary) perspectives. To be able to consider APM cost effective, the CEA analysis should yield a positive INMB value. Nonparametric bootstrapping was used to assess uncertainty. Several one-way sensitivity analyses were also performed. APM did not deliver lower postoperative costs, nor did it convincingly improve quality of life scores when compared with placebo surgery. From a societal perspective, APM was associated with € 971 (95% CI -2013 to 4017) higher costs and 0.015 (95% CI -0.011 to 0.041) improved QALYs over 2-year follow-up compared with placebo surgery. Both differences were statistically inconclusive (a wide 95% CI that crossed the line of no difference). Using the conventional willingness to pay (WTP) threshold of € 35,000 per QALY, APM resulted in a negative INMB of € -460 (95% CI -3757 to 2698). In our analysis, APM would result in a positive INMB only when the WTP threshold rises to about € 65,000 per QALY. The wide 95% CIs suggests uncertain cost effectiveness irrespective of chosen WTP threshold. The results of this study lend further support to clinical practice guidelines recommending against the use of APM in patients with a degenerative meniscus tear. Given the robustness of existing evidence demonstrating no benefit or cost effectiveness of APM over nonsurgical treatment or placebo surgery, future research is unlikely to alter this conclusion.Level of Evidence Level III, economic analysis.

Estimating the value of new antibiotic treatment strategies in Zhejiang province, China: cost-effectiveness analysis based on a validated dynamic model

ObjectivesThis analysis aims to better reflect the value of new antibiotic treatment strategies, thereby informing clinical antibiotic use, antimicrobial reimbursement and/or hospital formulary decision-making in China.DesignWe adapted a published and...