Incremental Net Monetary Benefit Research Articles

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 (MSS3) pragmatic randomised controlled trial

To evaluate the cost-effectiveness of an extended-role general practitioner (GP) symptoms clinic (SC), added to usual care (UC) for patients with multiple persistent physical symptoms (sometimes known as "medically unexplained symptoms"). A 52-week within-trial cost-utility analysis of a pragmatic multicentre randomised controlled trial comparing SC+UC (n=178) against UC alone (n=176), conducted from the primary perspective of the UK National Health Service (NHS) 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 (MI). Cost-effectiveness results were presented as incremental cost-effectiveness ratios (ICERs) and incremental net monetary benefits (INMBs). Uncertainty was explored using cost-effectiveness acceptability curves (using 1000 non-parametric bootstrapped samples) and sensitivity analysis (including societal costs, using SF-6D and capability ICECAP-A outcomes to estimate QALYs and years of full capability (YFC) respectively, varying intervention costs, missing data mechanism assumptions). Multiple imputation analysis showed that, compared to 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 ICER of £15,765/QALY, INMB of £189.22 (95% CI:-£573.62, £948.28) and a 69% probability of the SC+UC intervention arm being cost-effective at a threshold of £20000 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 quality of life outcomes. A 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...

Cost‐Effectiveness of Prehospital Ambulance Helicopter Transportation of Patients With Presumed Stroke in the Era of Mechanical Thrombectomy

Background Mechanical thrombectomy has emerged as standard of care in treatment of patients with acute ischemic stroke attributable to large‐vessel occlusion. Efforts to reduce the time from symptom onset to treatment initiation remain critically important to improve functional outcomes in patients treated with either intravenous thrombolysis, mechanical thrombectomy, or both. The use of ambulance helicopters in prehospital acute stroke care could potentially reduce symptom onset to treatment time to mechanical thrombectomy. This study aims to optimize the use of ambulance helicopters in prehospital transportation of patients with presumed acute stroke attributable to large‐vessel occlusion given economic constraints in health care budget. Methods By means of an economic model within a decision‐analytical framework, this study evaluates the cost‐effectiveness of complementary ambulance helicopters compared with road ambulances only for prehospital acute transportation of patients with presumed stroke and potential eligibility for mechanical thrombectomy, in regard to the number and locations of ambulance helicopters in Sweden. Results A single, optimally located complementary ambulance helicopter was cost‐effective compared with road ambulances only. The most cost‐effective solution in the base‐case scenario with the current 8 thrombectomy centers comprised 13 optimally located helicopters. It reduced mean onset to treatment time with intravenous thrombolysis and onset to treatment time with mechanical thrombectomy by 7.9 and 32.1 minutes, respectively, and produced health gains equal to 91 quality‐adjusted life‐years per year. When the willingness to pay per quality‐adjusted life‐years gained was set at Euro (EUR) 80 000, the incremental net monetary benefit per patient reached EUR 2240. The solution with 13 optimally located helicopters prevailed as the most cost‐effective in the extended scenario analysis with 11 thrombectomy centers, reaching an estimated incremental net monetary benefit per patient at EUR 1754. Furthermore, mean incremental net monetary benefit per patient reached global maximum (EUR 6046) at the geodesic distance of 165.2 km between patient location and the nearest thrombectomy center in the base‐case scenario, and at 238.5 km in the extended scenario with an estimated incremental net monetary benefit per patient at EUR 7994. Conclusion This study demonstrates the cost‐effectiveness of ambulance helicopters as a complementary mode of transportation to road ambulances. The most cost‐effective solution of complementary ambulance helicopters comprises 13 optimally located heliports across Sweden.

Cost-effectiveness of liquid biopsy for molecular profiling of newly diagnosed advanced non-squamous NSCLC in an Asian population.

178 Background: Liquid biopsy is complementary to tissue biopsy for lung cancer profiling, yet evidence of the cost-effectiveness is limited. This could retard implementation and reimbursement in clinical practice. The aim of this study is to estimate the cost-effectiveness of profiling strategies that include liquid biopsy and to identify the optimal profiling approach for newly diagnosed advanced non-squamous non-small cell lung cancer (NSCLC) in an Asian population using Singapore as an example. Methods: A decision tree and partitioned-survival model was developed from the Singapore healthcare system’s perspective to evaluate the cost-effectiveness of five molecular profiling strategies: either tissue or plasma next-generation sequencing (NGS) alone, a concurrent, and two sequential approaches. Model inputs were informed by local data or published literature. Sensitivity analyses and scenario analyses were undertaken to understand the robustness of the conclusions for decision making. The optimal strategy at different willingness-to-pay (WTP) thresholds was presented by cost-effectiveness acceptability frontier and the expected loss curve. Results: The sequential tissue-plasma NGS approach revealed an additional 0.0981 quality adjusted life years (QALYs) for an extra cost of S$3,074 over a 20-year time horizon compared to tissue NGS alone, resulting in an incremental cost-effectiveness ratio (ICER) of S$31,318/QALY and an incremental net monetary benefit of S$1,343 per patient. The findings were sensitive to the costs of pembrolizumab and osimertinib and the probabilities of re-biopsy after tissue NGS. Sequential plasma-tissue NGS and plasma NGS alone were more costly and less effective than alternatives. Conclusions: The sequential tissue-plasma NGS approach generated the highest net monetary benefit and was the optimal testing strategy when WTP was S$45,000/QALY. It retained superiority but understandably with a higher ICER when expensive, non-first line treatments were included. Overall, its routine clinical practice should be proactively considered for newly diagnosed advanced non-squamous NSCLC in an Asian population.

Cost-effectiveness analysis of osimertinib plus chemotherapy for patients with EGFR-mutated advanced non-small cell lung cancer.

First-line osimertinib plus chemotherapy significantly prolonged progression-free survival of patients with EGFR-mutated advanced non-small cell lung cancer (NSCLC) compared to osimertinib, according to the FLAURA2 trial. We established a Markov model to compare the cost-effectiveness of osimertinib plus chemotherapy with that of osimertinib alone. Clinical data were obtained from the FLAURA and FLAURA2 trials, and additional data were extracted from online resources and publications. Sensitivity analyses were conducted to evaluate the robustness of the findings. We used A willingness-to-pay threshold of $150,000 per quality-adjusted life-years (QALYs) gained. The main outcomes were QALYs, overall costs, incremental cost-effectiveness ratio (ICER), incremental net monetary benefit, and incremental net health benefit. Subgroup analyses were conducted according to patients' mutation type and central nervous system (CNS) metastatic status. In a 20-year time horizon, the ICER of osimertinib plus chemotherapy versus osimertinib alone was $223,727.1 per QALY gained. The sensitivity analyses identified the cost of osimertinib and the hazard ratio for overall survival as the top 2 influential factors and a 1.9% probability of osimertinib plus chemotherapy to be cost-effective. The subgroup analyses revealed ICERs of $132,614.1, $224,449.8, $201,464.1, and $130,159.7 per QALY gained for L858R mutations, exon 19 deletions, CNS metastases, and no CNS metastases subgroups, respectively. From the perspective of the United States health care system, osimertinib plus chemotherapy is not cost-effective compared to osimertinib alone for treatment-naïve patients with EGFR-mutated advanced NSCLC, but more favorable cost-effectiveness occurs in patients with L858R mutations and patients without baseline CNS metastases.

Cost-Effectiveness Analysis of Bevacizumab Biosimilars Versus Originator Bevacizumab for Metastatic Colorectal Cancer: A Comparative Study Using Real-World Data

ObjectivesMVASI (Amgen) and Zirabev (Pfizer) are 2 of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). We aimed to confirm and quantify the real-world cost savings and cost-effectiveness of MVASI and Zirabev relative to originator bevacizumab for patients with mCRC. MethodsWe conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. All mCRC patients who received originator bevacizumab between January 2008 and August 2019 or biosimilar bevacizumab between August 2019 and March 2021 were propensity score matched (1:4) to adjust for baseline differences. Total 1-year patient-level costs (CAD) and effects (life years [LY] and quality-adjusted LYs) were calculated from the public health payer’s perspective. Primary outcomes included incremental net monetary benefit and incremental net health benefit (INHB). Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and a 2-year analysis. ResultsThe matched cohort included 747 biosimilar cases and 2945 comparators. Bevacizumab biosimilars were associated with an incremental cost of −$6379 (95%CI: −9417, −3537) (ie, cost saving) and incremental effect of 0.0 (95% CI: −0.02, 0.02) LY and −0.01 (95% CI: −0.03, 0) quality-adjusted LYs gained. Incremental net monetary benefit and INHB estimates were $6331 (95% CI: 6245, 6417) and 0.127 LY (95% CI: 0.125, 0.128), respectively, at a willingness-to-pay threshold of $50 000/life year gained, with all estimates indicating the cost-effectiveness of biosimilar bevacizumab. Cost-effectiveness remained consistent across biosimilar brand subgroups and 2-year sensitivity analyses. ConclusionBevacizumab biosimilars demonstrated real-world cost savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation and supporting health system sustainability.

Cost‑effectiveness analysis of tislelizumab plus chemotherapy in Chinese patients with advanced or metastatic oesophageal squamous cell carcinoma

The RATIONALE-306 study revealed that patients with advanced or metastatic oesophageal squamous cell carcinoma (OSCC) could benefit from treatment with tislelizumab plus chemotherapy. This study aimed to evaluate the cost-effectiveness of tislelizumab plus chemotherapy for treating OSCC from the perspective of the Chinese healthcare system. Partitioned survival model estimated the cost-effectiveness of tislelizumab plus chemotherapy compared with chemotherapy alone for treating OSCC using RATIONALE-306 data. Costs and utilities were obtained from local databases and published studies. Costs, quality-adjusted life-years (QALYs), life-years, incremental cost-effectiveness ratios (ICER), incremental net health benefits (INHB), and incremental net monetary benefits (INMB) were outcomes. Price simulation were conducted at the willingness-to-pay (WTP) threshold. Sensitivity and subgroup analyses were performed to assess model robustness. Compared with chemotherapy alone, tislelizumab plus chemotherapy yielded an ICER of USD 27,896/QALY, gained an additional 0.414 QALYs and 0.751 life-years, and increased the cost by USD 11,560. Probabilistic sensitivity analysis revealed that tislelizumab plus chemotherapy was cost-effective at the WTP of USD 38,258/QALY with probability of 94.43%. When the price in China was less than USD 3.714 per mg, the price simulation results indicated that tislelizumab plus chemotherapy was cost-effective at a WTP threshold of USD 38,258. Tislelizumab plus chemotherapy yielded an INHB of 0.112 QALYs and an INMB of USD 4,279 compared with chemotherapy alone at a WTP threshold of USD 38,258. Based on the sensitivity analyses, the above results were stable. A general trend was observed for subgroups with better survival benefits related to a higher probability of cost-effectiveness. From the Chinese healthcare perspective, tislelizumab plus chemotherapy is more cost-effective than chemotherapy alone as a first-line therapy for OSCC. These findings can help clinicians make optimal clinical decisions and assist decision-makers in evaluating the cost-effectiveness of tislelizumab in clinical practice.

Cost–utility analysis of a palliative care program in Colombia

BackgroundThe economic assessment of health care models in palliative care promotes their global development. The purpose of the study is to assess the cost-effectiveness of a palliative care program (named Contigo) with that of conventional care from the perspective of a health benefit plan administrator company, Sanitas, in Colombia.MethodsThe incremental cost-utility ratio (ICUR) and the incremental net monetary benefit (INMB) were estimated using micro-costing in a retrospective, analytical cross-sectional study on the care of terminally ill patients enrolled in a palliative care program. A 6-month time horizon prior to death was used. The EQ-5D-3 L questionnaire (EQ-5D-3 L) and the McGill Quality of Life Questionnaire (MQOL) were used to measure the quality of life.ResultsThe study included 43 patients managed within the program and 16 patients who received conventional medical management. The program was less expensive than the conventional practice (difference of 1,924.35 US dollars (USD), P = 0.18). When compared to the last 15 days, there is a higher perception of quality of life, which yielded 0.25 in the EQ-5D-3 L (p < 0.01) and 1.55 in the MQOL (P < 0.01). The ICUR was negative and the INMB was positive.ConclusionBecause the Contigo program reduces costs while improving quality of life, it is considered to be net cost-saving and a model with value in health care. Greater availability of palliative care programs, such as Contigo, in Colombia can help reduce existing gaps in access to universal palliative care health coverage, resulting in more cost-effective care.

Exploring Heterogeneity in Cost-Effectiveness Using Machine Learning Methods: A Case Study Using the FIRST-ABC Trial.

The aim of this study was to explore heterogeneity in the cost-effectiveness of high-flow nasal cannula (HFNC) therapy compared with continuous positive airway pressure (CPAP) in children following extubation. Using data from the FIRST-line support for Assistance in Breathing in Children (FIRST-ABC) trial, we explore heterogeneity at the individual and subgroup levels using a causal forest approach, alongside a seemingly unrelated regression (SUR) approach for comparison. FIRST-ABC is a noninferiority randomized controlled trial (ISRCTN60048867) including children in UK paediatric intensive care units, which compared HFNC with CPAP as the first-line mode of noninvasive respiratory support. In the step-down FIRST-ABC, 600 children clinically assessed to require noninvasive respiratory support were randomly assigned to HFNC and CPAP groups with 1:1 treatment allocation ratio. In this analysis, 118 patients were excluded because they did not consent to accessing their medical records, did not consent to follow-up questionnaire or did not receive respiratory support. The primary outcome of this study is the incremental net monetary benefit (INB) of HFNC compared with CPAP using a willingness-to-pay threshold of £20,000 per QALY gain. INB is calculated based on total costs and quality adjusted life years (QALYs) at 6 months. The findings suggest modest heterogeneity in cost-effectiveness of HFNC compared with CPAP at the subgroup level, while greater heterogeneity is detected at the individual level. The estimated overall INB of HFNC is smaller than the INB for patients with better baseline status suggesting that HFNC can be more cost-effective among less severely ill patients.

Health Economic Evaluation of Lung Cancer Screening Using a Diagnostic Blood Test: The Early Detection of Cancer of the Lung Scotland (ECLS).

Diagnostic blood tests have the potential to identify lung cancer in people at high risk. We assessed the cost-effectiveness of a lung cancer screening intervention, using the EarlyCDT®-Lung Test (ECLS) with subsequent X-ray and low-dose chest CT scans (LDCT) for patients with a positive test result, compared to both usual care and LDCT screening for the target population. We conducted a model-based lifetime analysis from a UK NHS and personal social services perspective. We estimated incremental net monetary benefit (NMB) for the ECLS intervention compared to no screening and to LDCT screening. The incremental NMB of ECLS intervention compared to no screening was GBP 33,179 (95% CI: -GBP 81,396, GBP 147,180) and GBP 140,609 (95% CI: -GBP 36,255, GBP 316,612), respectively, for a cost-effectiveness threshold of GBP 20,000 and GBP 30,000 per quality-adjusted life year. The same figures compared with LDCT screening were GBP 162,095 (95% CI: GBP 52,698, GBP 271,735) and GBP 52,185 (95% CI: -GBP 115,152, GBP 219,711). The ECLS intervention is the most cost-effective screening alternative, with the highest probability of being cost-effective, when compared to no screening or LDCT screening. This result may change with modifications of the parameters, suggesting that the three alternatives considered in the main analysis are potentially cost-effective.