Payer Perspective Research Articles

INEAS's Cost-Effectiveness Analysis of Vemurafenib: Paving the Way for Value-Based Pricing in Tunisia.

The Tunisian Health Technology Assessment (HTA) body, INEAS, conducted a cost-effectiveness analysis (CEA) of vemurafenib in the treatment of locally advanced or metastatic BRAF V600-mutated melanoma. The objective of this analysis was to enable the use of value-based pricing as a new approach to price negotiation. This study was part of a broader HTA report that was prepared in response to a joint request from the regulatory authorities and the CNAM, Tunisia's compulsory insurance scheme. Our analysis was based on a probabilistic Markov cohort model that calculated the costs and quality-adjusted life years (QALY) associated with vemurafenib compared to the standard of care from a public payer perspective. The CEA indicated that vemurafenib provides a gain of 0.38 life years (1.78 vs. 1.4) for an incremental cost of USD 101,106.62 from the perspective of the main public payer (CNAM). This study revealed an incremental cost-effectiveness ratio (ICER) of 163,311.40 USD/QALY and 163,911.46 USD/QALY, respectively, from the CNAM and public health facilities' perspectives. Vemurafenib cannot be considered cost-effective in terms of what has normally been considered a reasonable willingness to pay (WTP) in Tunisia. A significant price reduction would be necessary to bring the incremental cost-effectiveness ratio to an acceptable level.

Evaluating cost-effectiveness of PFO management strategies: closure with Cardioform vs. Amplatzer, and treatment with medical therapy alone, for secondary stroke prevention

Aim The aim of this study was to evaluate the cost-effectiveness of patent foramen ovale (PFO) closure using CARDIOFORM Septal Occluders versus AMPLATZER Septal Occluders, as well as compared to Medical Therapy Alone, from a payor perspective in the United States. Methods An economic evaluation compared the value of CARDIOFORM, AMPLATZER, and Medical Therapy Alone. A Markov model simulated a cohort of 1,000 individuals with PFO and a history of cryptogenic stroke, with baseline demographic and clinical characteristics reflecting individuals enrolled in the REDUCE and RESPECT trials over a five-year time horizon. The costs and health consequences associated with complications and adverse events, including recurrent stroke, were compared over a time horizon of 5 years. Results PFO closure using CARDIOFORM was economically dominant, providing both cost-savings and improved effectiveness compared to closure with AMPLATZER. It resulted in an estimated savings of over $1.3 million, an additional 24.8 quality-adjusted life-years (QALYs) gained, and 28 strokes avoided in a cohort of 1,000 patients. When compared to Medical Therapy Alone, closure with CARDIOFORM was found to be cost-effective, with an incremental cost-effectiveness ratio (ICER) of $36,697 per QALY gained. Sensitivity and scenario analysis showed the model findings to be highly robust across reasonable changes to baseline input values and assumptions. Conclusions The results of this analysis suggest that PFO closure using the CARDIOFORM Septal Occluder is the most cost-effective treatment strategy for patients with a PFO-associated stroke, particularly compared to AMPLATZER where it resulted in both cost-saving and improved patient outcomes.

Inclusion of phase III clinical trial costs in health economic evaluations

IntroductionProtocol-driven trial activities contribute to the utility gain demonstrated in the phase III clinical trial of a new drug. If this utility gain cannot be distinguished from the effects of the new drug itself, protocol-driven trial costs cannot be easily dismissed for consistency reasons. This study aims to estimate the impact of including per-patient costs of phase III clinical trials on the incremental cost-effectiveness ratio (ICER).MethodsThe analysis utilized a modeling approach with secondary data from an ad-hoc literature review, considering both societal and payer perspectives. While the costs of phase III clinical trials may cancel out during the period of “normal” life-years due to the incremental cost calculation, they do not cancel out when differential early treatment termination occurs (e.g., due to differential mortality). Assuming the presence of differential mortality, per-patient phase III trial costs were calculated for the period of added life-years. These costs were then included in the ICER of a new drug, under the assumption that direct patient-related costs constitute 30–70% of the total trial costs. Capital costs were also incorporated from a societal perspective.ResultsBased on assumptions of $40,000 out-of-pocket expenses per patient enrolled in a phase III trial and a life expectancy gain of three months, incremental costs increased by $27,000 from a societal perspective. From a payer perspective, the estimate was $12,000.ConclusionsThe costs of phase III trials are a relevant component of the ICER, and excluding it is generally not appropriate for consistency reasons. Properly considering these trial costs is essential for a comprehensive evaluation of a new drug’s cost-effectiveness.

Multidisciplinary perspectives in Demodex blepharitis: A new view of treatment from clinical, payer, and patient perspectives.

Demodex infestation is the cause of more than two-thirds of all cases of blepharitis in the United States. Although symptoms may include crustiness, redness, or itching of the eyelids, diagnosis can be accomplished through a simple examination of the eyelashes. The presence of a waste product of the Demodex mite, known as collarettes, on the base of the eyelashes is a pathognomonic sign of Demodex blepharitis. Demodex infestation that results in blepharitis may cause blockage and ultimately atrophy of the meibomian glands, worsening dry eye disease. Until recently, management of Demodex blepharitis has been limited by a lack of approved therapy options. Lotilaner ophthalmic solution 0.25%, the first approved therapy for treatment of Demodex blepharitis, has not only been shown to eradicate Demodex mites in one-half to two-thirds of patients following short-term treatment but also demonstrated continued benefits through 1 year of follow-up. In addition to managing Demodex blepharitis, treatment with lotilaner ophthalmic solution 0.25% may aid in the management of dry eye disease and other forms of ocular surface disease caused by complications of Demodex infestation. As a result, it is possible that successful management of Demodex blepharitis may reduce chronic use of health care resources dedicated to managing other chronic ocular conditions. As eye care professionals recognize Demodex infestation as a key mediator of ocular surface disease, increasing diagnostic awareness and addressing this underlying cause of Demodex blepharitis may reduce the need for specialist follow-up care, decrease the need for chronic therapy, and improve patient outcomes. Through routine screening for Demodex infestation and Demodex blepharitis, eye care professionals can now address an underlying factor in ocular surface disease to improve use of health care resources in the community.

Cost-Effectiveness of 9-Valent HPV Vaccination for Patients Treated for High-Grade Cervical Intraepithelial Neoplasia in the UK

Patients who have been treated for high-grade cervical intraepithelial neoplasia (CIN grade ≥2) are at a high risk for subsequent CIN and other cancers and diseases related to human papillomavirus (HPV). HPV vaccination can reduce the risk of subsequent disease in patients surgically treated for grade 2 or greater CIN; however, there is no formal recommendation for prophylactic HPV vaccination in this high-risk population, and the cost-effectiveness is unknown. To assess the incremental lifetime outcomes, costs, and cost-effectiveness of integrating peritreatment 9-valent HPV (9vHPV) vaccination in combination with posttreatment surveillance for the prevention of cervical cancer and other HPV-attributable diseases in patients surgically treated for grade 2 or greater CIN vs posttreatment surveillance alone from a UK payer perspective. This economic evaluation used 3 independent Markov model structures. Model inputs for vaccine efficacy, utilities, and costs were obtained from published sources, and cervical cancer screening data were obtained from the National Health Service Cervical Screening Program. Costs were adjusted to 2022 to 2023 reference years. Data were analyzed from October 2022 to September 2023. Peritreatment vaccination with 9vHPV in combination with posttreatment surveillance compared with posttreatment surveillance alone. Clinical outcomes included grade 1, 2, or 3 CIN; cervical cancer; vaginal cancer; vulvar cancer; anal cancer; head and neck cancer; genital warts; and recurrent respiratory papillomatosis. Incremental cost-effectiveness ratios (ICERs) using a willingness-to-pay threshold (WTP) of £20 000 (US $26 200) per quality-adjusted life-year (QALY) were estimated. Deterministic sensitivity analysis and probabilistic sensitivity analysis were performed. Vaccination with 9vHPV in conjunction with posttreatment surveillance was cost-effective, with a favorable ICER of £13 789.07 (US $18 064.68) per QALY gained (ie, below the WTP of £20 000 per QALY) vs posttreatment surveillance alone. The resulting ICER was £52 358.01 (US $68 588.99) per HPV-related cancer averted and £64 090 (US $83 958.18) per HPV-related cancer death averted. The ICER was most sensitive to discount rate, incidence of HPV infection, vaccine price, and age at initial treatment for grade 2 or greater CIN. Results of the probabilistic sensitivity analysis showed peritreatment 9vHPV vaccination was cost-effective at the WTP recommended by the UK's Joint Committee on Vaccination and Immunisation (90% of iterations <£30 000 [US $39 300] per QALY) in 100% of iterations. These findings suggest that peritreatment prophylactic 9vHPV vaccination is a cost-effective option for preventing subsequent HPV-attributable diseases in patients surgically treated for grade 2 or greater CIN.

Virtual/Mobile Overdose Response in Canada: A Social Return on Investment Analysis

The overdose epidemic continues to be one of the leading causes of death in North America and continues to contribute to high healthcare costs. While harm reduction initiatives have significantly reduced the aforementioned costs, there is a dearth of evidence regarding overdose response hotlines and applications. We aim to evaluate the social return on investment (SROI) from a payer perspective of one such overdose response hotline, Canada's National Overdose Response Service, and its implications for service users, service operators, the Canadian healthcare system, and program funders. Outcome variables determined from theory of change models were developed in consultation with the aforementioned vested interest groups. Proxy values were attributed to each variable identified through values present within existing literature and databases. These values were then compared to operational costs accounting for deadweight, attribution, and displacement to determine a final SROI ratio. A discount rate was then applied based on the influence of risk on the outcome achieved. The ratio illustrating the value created for all stakeholders, resulting from the $1,592,00 investment made over two years, is $15.84 per single dollar invested. The value generated stems primarily from overdose prevention, mental health support, staff employment, reductions in emergency service utilization, service referrals, and volunteer well-being, which outweigh costs including operational funding, work-related stressors, compassion fatigue, and false calls. The results of our study demonstrate that the National Overdose Response Service provides a social value that far outweighs the costs attributed to the program's operation.

Cost-Effectiveness Analysis of Current Treatment Options for Eosinophilic Esophagitis.

The management strategies for eosinophilic esophagitis (EoE) include proton pump inhibitors (PPI), swallowed topical steroids (tCS), elimination diets, and the biologic agent dupilumab, although there remains little guidance on the selection of initial treament. We performed cost-effectiveness analyses to compare these approaches of first-line therapy. A Markov model was constructed from a payer perspective to evaluate the cost-effectiveness of first-line therapies for EoE, including PPI, tCS, and six-food elimination diet (SFED), with.crossover in treatments for primary and secondary non-response. The primary outcome was incremental cost-effectiveness ratio (ICER) at two- and five-year time horizons. Secondary analyses included modeling from a societal perspective that also accounted for patient-specific costs, as well as a separate simplified model comparing dupilumab to tCS and PPI. In the base-case scenario (five-year time horizon), the average costs were SFED:$15,296.81, PPI:$16,153.77, and tCS:$20,975.33 as initial therapy, with SFED being the dominant strategy (more effective/less costly), while PPI offered the lowest cost on a two-year time horizon. From a societal perspective, PPI was the dominant initial strategy on both two- and five-year time horizons. Amongst pharmacologic therapies, PPI was the most cost-effective first-line option. Dupilumab was not cost-effective relative to tCS, unless the quarterly cost is reduced from $7,311 to $2,038.50 per price threshold analysis under permissive modeling conditions. SFED was the most effective/least costly first-line therapy from payer perspective, while PPI was more cost-effective from societal perspective. PPI is also the most cost-effective pharmacologic strategy. Dupilumab requires substantial cost reductions to be considered cost-effective first-line pharmacotherapy.

Transarterial radioembolization vs transarterial chemoembolization with drug-eluting beads for treating hepatocellular carcinoma: a cost-effectiveness analysis in Japanese healthcare system.

Transarterial radioembolization (TARE) is effective for unresectable hepatocellular carcinoma; however, it awaits approval in Japan. This study aimed to simulate the cost-effectiveness of TARE over chemoembolization when TARE is approved in Japan and identify the requirements for cost-effectiveness. A Markov model was constructed to analyze the costs and effectiveness associated with TARE and transarterial chemoembolization with drug-eluting beads (DEB-TACE) for 2-month cycles over 5years. In the primary analysis, the intention-to-treat survival data were used to calculate transition probabilities, whereas the ancillary analysis assessed the per-protocol survival data. DEB-TACE costs were calculated using the Japanese nationwide claims Diagnosis Procedure Combination database between April 2018 and March 2022, whereas TARE costs were estimated using database and international sources. The incremental cost-effectiveness ratio (ICER) was determined based on the payer's perspective and compared with the Japanese willingness-to-pay threshold of 5 million Japanese yen (JPY) (31,250 USD) per quality-adjusted life years (QALY). From the claims database, 6,986 patients with hepatocellular carcinoma who received DEB-TACE were identified. In the primary analysis, the ICER was 5,173,591 JPY (32,334 USD)/QALY, surpassing the Japanese willingness-to-pay threshold. However, the ancillary analysis showed a lower ICER of 4,156,533 JPY (25,978 USD)/QALY, falling below the threshold. The one-way deterministic sensitivity analysis identified progression-free survival associated with TARE and DEB-TACE, DEB-TACE costs, and radioactive microsphere reimbursement price as key ICER influencers. The primary analysis suggested that setting the reimbursement price of radioactive microspheres below 1.399 million JPY (8,744 USD), approximately 2.8% lower than the price in the United Kingdom, would place the ICER below the Japanese willingness-to-pay threshold. Under specific conditions, TARE can be a more cost-effective treatment than DEB-TACE. If the reimbursement price of radioactive microspheres is set approximately 2.8% lower than that in the United Kingdom, TARE could be cost-effective compared with DEB-TACE.

Genicular Artery Embolization, Radiofrequency Ablation, and Corticosteroid Therapy for Knee Osteoarthritis: A Cost-Effectiveness Analysis Using Randomized Clinical Trial Data.

Background: Many patients with symptomatic knee osteoarthritis (KOA) are refractory to traditional nonsurgical treatments such as intraarticular corticosteroid (CS) injection but are not yet eligible for or decline surgery. Genicular artery embolization (GAE) and radiofrequency ablation (RFA) are emerging adjunctive or alternative minimally invasive treatments. Objective: To perform a cost-effectiveness analysis (CEA) comparing CS, GAE, and RFA, for treatment of symptomatic KOA using a Markov model based on a de novo network meta-analysis (NMA) of randomized control trials. Methods: CEA was conducted to compare GAE and RFA to CS using a Markov cohort state-transition model from a U.S. Medicare payer's perspective over a 4-year time horizon. The model incorporated each treatment's success and attrition rates, costs, and utility benefit. Utility benefit values were derived at short-term (0.5-3 months) and long-term (6-12 months) posttreatment follow-up from NMA of published RCTs using an outcome of improved knee pain and/or function. Analyses were conducted at a willingness-to-pay threshold of $100,000 per quality-adjusted life year (QALY). Sensitivity analyses were performed, including when simulating various cost setting scenarios (i.e., office vs hospital outpatient treatment). Results: RFA demonstrated larger treatment effect than GAE, more pronounced at short-term [standardized mean difference (SMD), -1.6688, 95% CI [-2.7806; -0.5571], p=.003] than long-term (SMD -0.3822, 95% CI [-1.9743; 1.2100], p=.64) follow-up. Across cost setting scenarios, incremental cost-effectiveness ratios relative to CS were $561-1563/QALY for GAE versus $76-429/QALY for RFA (not counting scenarios in which RFA was dominated by CS). GAE demonstrated higher cost-effectiveness probability compared to RFA (41.6-54.8% vs. 18.4-29.2%, respectively). GAE was more cost-effective than RFA when the GAE clinical success rate and post-GAE utility value exceeded 32.1-51.0% and 0.562-0.617, respectively, and when the GAE quarterly attrition rate was less than 8.8-17.4%. RFA was more cost-effective when baseline pre-treatment utility values exceeded 0.695-0.713. Neither GAE costs nor RFA costs were sensitive parameters. Conclusion: Across scenarios, GAE was consistently the most likely cost-effective treatment option compared to RFA and CS, although clinical success rates, attrition rates, and utility values impact its cost-effectiveness. Clinical Impact: GAE is likely to be more cost-effective than RFA or CS for treatment of symptomatic KOA.

End-of-Life Costs in Cancer Patients: A Systematic Review.

Identify the costs of an oncology patient at the end of life. A systematic literature review was conducted by screening Embase, PubMed and Lilacs databases, including all studies evaluating end-of-life care costs for cancer patients up to March 2024. The review writing followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. The quality of the included studies was assessed using the Drummond checklist. The protocol is available at PROSPERO CRD42023403186. A total of 733 studies were retrieved, and 43 were considered eligible. Among the studies analyzed, 41,86% included all types of neoplasms, 18.60% of lung neoplasm, All articles performed direct cost analysis, and 9.30% also performed indirect cost analysis. No study evaluated intangible costs, and most presented the macrocosting methodology from the payer's perspective. The articles included in this review presented significant heterogeneity related to populations, diagnoses, periods considered for evaluation of end-of-life care, and cost analyses. Most of the studies were from a payer perspective (74,41%) and based on macrocosting methodologies (81,39%), which limit the use of the information to evaluate variabilities in the consumption of resources. Considering the complexity of end-of-life care and the need for consistent data on costs in this period, new studies, mainly in low- and middle-income countries with approaches to indirect and intangible costs, with a societal perspective, are important for public policies of health in accordance with the trend of transforming value-based care, allowing the health care system to create more value for patients and their families.

Cost-effectiveness of an interactive voice response system for improving retention in care and adherence to antiretroviral therapy among young adults in Uganda

BackgroundNew interventions aimed at increasing access to and adherence to antiretroviral therapy among young people living with the human immunodeficiency virus (YPLHIV) are needed. This study assessed the cost-effectiveness of the call-for-life interaction voice response tool compared to that of the standard of care (SOC) for promoting treatment adherence and retention in care among YPLHIV in Western Uganda.This cost-effectiveness study used data from a randomized controlled trial and a decision-analytic Markov model to estimate the long-term outcomes and costs of the Call for Life-Interactive Voice Response (CFL-IVR) tool and the usual care from the Ugandan public payer perspective. The model was parameterized using primary data and the literature and adopted a 1-year Markov cycle. The main outcomes were mean annual costs, disability-adjusted life-years (DALYs), and the incremental cost-effectiveness ratio (ICER) in form of cost per DALY averted. The CLF-IVR was deemed cost-effective if the ICER was between 1% and 51% of Uganda’s gross domestic product. We conducted deterministic and probabilistic sensitivity analyses to assess the effect of adjusting parameter values on cost-effectiveness estimates. All costs were reported in 2021 US dollars, and a discount rate of 3% was applied to both costs and outcomes.ResultsThe base case analysis showed that, from the Ugandan public payer perspective, the CLF-IVR led to more mean annual costs ($359 vs. $280) and averted more mean annual DALYs (15.78 vs. 11.09) than the SOC, leading to an ICER of $17 per DALY averted. The base-case results did not change significantly in the deterministic and probabilistic sensitivity analyses. The cost-effectiveness estimates were more responsive to uncertainties surrounding ART duration, viral load suppression rates, and discount rates.ConclusionThe CLF-IVR may be a cost-effective intervention for promoting treatment adherence and retention in care among YPLHIV in Uganda and other low-income settings. Once implemented, similar interventions may lead to greater returns in encouraging adherence to ART and retention in care among YPLHIV and other vulnerable groups, and eventually favorable clinical outcomes.Trial registrationNCT04718974 Registry: clinical Trials.gov https://ichgcp.net/nl/clinical-trials-registry/NCT04718974 (20 Jan 2021).

Cost-effectiveness of multigene sequencing test and treatment for metastatic non-small cell lung cancer: A unique setting in the initial adoption phase in Japan allowing testing only after standard treatment

ObjectiveTo clarify the cost-effectiveness of comprehensive diagnosis and treatment of metastatic non-small cell lung cancer in Japan, from initial diagnosis to post-standard treatment, using three different strategies. MethodsA decision tree was created using three diagnostic and treatment strategies, assuming that Foundation One CDx (F1CDx), a comprehensive genome panel, was introduced in Japan in June 2019. This comprehensive decision tree includes Markov models, cost-effectiveness analyses (CEA), and cost-utility analyses (CUA) of the three strategies from the perspective of Japanese payers. Specifically, Strategy1 involves single-gene testing at the initial diagnosis and F1CDx after standard treatment; Strategy2 involves only single-gene testing at the initial diagnosis; Strategy3 involves F1CDx at the initial diagnosis. The incremental cost-effectiveness ratios (ICERs) of the three strategies are estimated. Sensitivity analyses were performed to assess the uncertainty of the parameter settings. ResultsStrategy3 was dominated for both CUA and CEA. The ICER/quality-adjusted life year (QALY) for Strategy2 versus Strategy1 was USD 13,734 (JPY 2,080,923, USD 1 = JPY 151.39 on April 1st, 2024), which is less than the willingness to pay of USD 45,900 (JPY 7,500,000), and Strategy2 was more cost-effective than Strategy1. F1CDx was not cost-effective compared to multiple simultaneous single tests at the initial diagnosis, either after standard treatment or at the initial diagnosis. Sensitivity analysis also showed that the most influential factor on the ICER for both CEA and CUA was treatment cost. ConclusionsFrom both patient benefit and health economic perspectives, introducing F1CDx after standard treatment in June 2019 was not as cost-effective as multiple simultaneous single tests at the initial diagnosis but was more realistic from a health economic perspective than introducing F1CDx at the time of initial diagnosis. Therefore, the policy at the time of F1CDx introduction in Japan was appropriate from a short-term health-economic perspective.

Economic Evaluation of a Novel Lung Cancer Diagnostic in a Population of Patients with a Positive Low-Dose Computed Tomography Result

Background: Early detection of lung cancer is crucial for improving patient outcomes. Although advances in diagnostic technologies have significantly enhanced the ability to identify lung cancer in earlier stages, there are still limitations. The alarming rate of false positives has resulted in unnecessary utilization of medical resources and increased risk of adverse events from invasive procedures. Consequently, there is a critical need for advanced diagnostics after an initial low-dose computed tomography (LDCT) scan. Objectives: This study evaluated the potential cost savings for US payers of CyPath® Lung, a novel diagnostic tool utilizing flow cytometry and machine learning for the early detection of lung cancer, in patients with positive LDCT scans with indeterminate pulmonary nodules (IPNs) ranging from 6 to 29 mm. Methods: A cost offset model was developed to evaluate the net expected savings associated with the use of CyPath® Lung relative to the current standard of care for individuals whose IPNs range from 6 to 29 mm. Perspectives from both Medicare and private payers in a US setting are included, with a 1-year time horizon. Cost calculations included procedure expenses, complication costs, and diagnostic assessment costs per patient. Primary outcomes of this analysis include cost savings per cohort and cost savings per patient. Results: Our analysis showed positive cost savings from a private payer’s perspective, with expected savings of $895 202 311 per cohort and $6460 per patient, across all patients. Scenario analysis resulted in cost savings of $890 829 889 per cohort, and $6429 per patient. Similarly, savings of $378 689 020 per cohort or $2733 per patient were yielded for Medicare payers, across all patients. In addition, scenario analysis accounting for false negative patients from a Medicare payer perspective yielded savings of $376 902 203 per cohort and $2720 per patient. Discussion: The results suggest substantial cost savings, primarily due to reductions in follow-up diagnostic assessments and procedures, and highlight the importance of accurate diagnostic tools in reducing unnecessary healthcare expenditures. Conclusion: CyPath® Lung utilization yields savings for private and Medicare payers relative to the current standard of care in a US setting for individuals with 6 to 20 mm IPNs.

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.

Real-time continuous glucose monitoring vs. self-monitoring of blood glucose: cost-utility in South Korean type 2 diabetes patients on intensive insulin

Aims This study investigated the cost-utility of real-time continuous glucose monitoring (rt-CGM) versus self-monitoring of blood glucose (SMBG) in people with type 2 diabetes (T2D) receiving intensive insulin therapy in South Korea. Methods The IQVIA Core Diabetes Model (CDM v9.5) was used, with clinical effectiveness data obtained from a large-scale real world study. Costs were obtained from South Korean sources and inflated to 2022 South Korean Won (KRW). A South Korean payer perspective was adopted over a lifetime horizon, with future costs and effects discounted at 4.5% per annum. Baseline characteristics included a mean baseline HbA1c level of 8.6% (71 mmol/mol), and a mean age of 64.4 years. A willingness-to-pay (WTP) threshold of KRW 46.0 million was used. Results Rt-CGM led to an increase of 0.683 quality-adjusted life years (QALYs) versus SMBG (7.526 QALYs for rt-CGM versus 6.843 QALYs for SMBG). An increase in costs of KRW 16.4 million (from KRW 90.4 million to KRW 106.8 million) was associated with rt-CGM. The incremental cost-utility ratio was KRW 24.0 million per QALY gained, significantly lower than the KRW 46 million threshold. Conclusions For individuals with T2D managed by intensive insulin therapy in South Korea, rt-CGM is cost-effective relative to SMBG.

Budget impact of dostarlimab plus carboplatin-paclitaxel for primary advanced or recurrent endometrial cancer from a third-party US payer perspective

Aim Dostarlimab plus carboplatin-paclitaxel (CP) significantly increased progression-free survival in patients with primary advanced or recurrent endometrial cancer (pA/rEC) vs CP alone in the RUBY trial (NCT03981796). This analysis estimated the per-member-per-month (PMPM) costs of introducing dostarlimab + CP as a treatment alternative from a third-party US payer perspective. Materials and methods A budget impact model was developed to estimate the costs of introducing dostarlimab + CP into commercial and Medicare health plans over a 3-year time horizon (2023–2025). Costs were sourced from relevant literature and US-specific databases and were calculated using epidemiology data, clinical inputs, treatment costs, and market share estimates. Clinical inputs were sourced from primary clinical trials for each respective treatment (i.e. dostarlimab + CP, CP, pembrolizumab, pembrolizumab plus lenvatinib, bevacizumab + CP, and pembrolizumab + CP). Current and future market shares assumed dostarlimab + CP reduced the market share of CP only. Analyses were performed in mismatch repair–deficient/microsatellite instability–high (dMMR/MSI-H) and overall populations using a US 2023 cost year. Results For a commercial plan, the model estimated (dMMR/MSI-H and overall populations) that 7 and 26 patients would be treated with dostarlimab + CP, respectively; average annual budget impacts per patient treated were $118,257 and $116,094; average budget impacts per patient treated per month (PPPM) were $9,855 and $9,675; average budget impacts PMPM were $0.02 and $0.06. For a Medicare plan, the model estimated that 28 and 93 patients, respectively, would be treated with dostarlimab + CP. Average annual budget impacts per patient treated and PPPM were the same as those for the commercial plan in both populations; average budget impacts PMPM were $0.07 and $0.22, respectively. Conclusions Introducing dostarlimab + CP as a first-line treatment for patients with pA/rEC results in minimal budget impact PMPM from a US third-party payer’s perspective. Together with the efficacy and safety results from RUBY, these results support the use of dostarlimab + CP as a treatment option.

Cost-effectiveness of first-line enfortumab vedotin in addition to pembrolizumab for metastatic urothelial carcinoma in the United States.

The EV-302 trial found that the combination of enfortumab vedotin (EV) with pembrolizumab significantly improved survival for patients with metastatic urothelial carcinoma (mUC). However, given the high cost of the drugs, there is a need to assess its value by considering both efficacy and cost. This study assessed the cost-effectiveness of EV plus pembrolizumab as a first-line treatment for patients with mUC from the perspective of U.S. payers. A Markov model was developed to compare the lifetime costs and effectiveness of EV in combination with pembrolizumab with chemotherapy in the treatment of mUC patients from U.S. payer perspective. Life-years (LYs), quality-adjusted LYs (QALYs), and lifetime costs were estimated. One-way, two-way and probabilistic sensitivity analyses were conducted to evaluate model uncertainty. Additionally, subgroup analyses were performed. Compared to chemotherapy, the combination of EV and pembrolizumab provided an additional 2.10 LYs and 1.72 QALYs, at an incremental cost of $962,240.8 per patient. The incremental cost-effectiveness ratio (ICER) is $558,973 per QALY. Subgroup analysis indicated that patients ineligible for cisplatin treatment had a lower ICER compared to those who were eligible for cisplatin. From the perspective of US payers, at a willingness-to-pay threshold of $150,000 per QALY, the combination of EV and pembrolizumab is estimated to not be cost-effective compared to traditional chemotherapy in the first-line treatment of mUC patients.

Cost-utility evaluation of a health and social care integration programme for frail older adults in Argentina

PurposeOur objective was to evaluate the cost-utility of a health and social care integration programme for frail older adults in Buenos Aires, Argentina.Design/methodology/approachBased on a study of the programme’s effectiveness, a Markov model was conducted to assess its cost-utility. The active intervention was the health and social care integration programme, and the control was the best standard of care so far. The setting was the patients' home of residence. A third-party payer perspective and a lifelong time horizon were adopted. All transition probabilities, quality-adjusted life years (QALYs) and costs were estimated from the effectiveness study. A discount rate of 3.5% was applied to costs and benefits. Costs are expressed in international dollars (Int$), calculated according to the International Monetary Fund’s purchasing power parity rate. Different sensitivity analyses were performed. The model was built in Excel 365. Construct validity, verification during model construction and internal consistency of the results were assessed.FindingsThe programme had an average cost of Int$18,768.22/QALY, and the control Int$42,609.68/QALY. In the incremental analysis, the programme saved Int$26,436.10 and gained 0.81 QALYs over the control. In the sensitivity analyses, in 99.96% of cases, the programme was less costly and more effective.Practical implicationsThe cost savings can facilitate the scalability.Originality/valueThe health and social care integration programme for frail older adults was more effective and less costly than the best standard of care to date. This study contributes to the scarce evidence on the efficiency of integrated care strategies for frail older persons.

Modelling the potential clinical and economic impact of universal immunisation with nirsevimab versus standard of practice for protecting all neonates and infants in their first respiratory syncytial virus season in Spain

BackgroundRespiratory syncytial virus (RSV) is associated with substantial morbidity among infants. This study modelled the potential public health and economic impact of nirsevimab, a long-acting monoclonal antibody, as an immunoprophylactic strategy for all infants in Spain in their first RSV season.MethodsA static decision-analytic model of the Spanish birth cohort during its first RSV season was developed to estimate the impact of nirsevimab on RSV-related health events and costs versus the standard of practice (SoP). Spain-specific costs and epidemiological data were used as model inputs. Modelled outcomes included RSV-related outpatient visits, emerging room (ER) visits, hospitalisations – including pediatric intensive care unit (PICU) admission, mechanical ventilation, and inpatient mortality.ResultsUnder the current SoP, RSV caused 151,741 primary care visits, 38,798 ER visits, 12,889 hospitalisations, 1,412 PICU admissions, and 16 deaths over a single season, representing a cost of €71.8 million from a healthcare payer perspective. Universal immunisation of all infants with nirsevimab was expected to prevent 97,157 primary care visits (64.0% reduction), 24,789 ER visits (63.9%), 8,185 hospitalisations (63.5%), 869 PICU admissions (61.5%), and 9 inpatient deaths (52.6%), saving €47.8 million (62.4%) in healthcare costs.ConclusionsThese results suggest that immunisation with nirsevimab of all infants experiencing their first RSV season in Spain is likely to prevent thousands of RSV-related health events and save considerable costs versus the current SoP.

Economic Evaluation of Population-Based BRCA1 and BRCA2 Testing in Canada

Population-based BRCA testing can identify many more BRCA carriers who will be missed by the current practice of BRCA testing based on family history (FH) and clinical criteria. These carriers can benefit from screening and prevention, potentially preventing many more breast and ovarian cancers and deaths than the current practice. To estimate the incremental lifetime health outcomes, costs, and cost-effectiveness associated with population-based BRCA testing compared with FH-based testing in Canada. For this economic evaluation, a Markov model was developed to compare the lifetime costs and outcomes of BRCA1/BRCA2 testing for all general population women aged 30 years compared with FH-based testing. BRCA carriers are offered risk-reducing salpingo-oophorectomy to reduce their ovarian cancer risk and magnetic resonance imaging (MRI) and mammography screening, medical prevention, and risk-reducing mastectomy to reduce their breast cancer risk. The analyses were conducted from both payer and societal perspectives. This study was conducted from October 1, 2022, to February 20, 2024. Outcomes of interest were ovarian cancer, breast cancer, additional heart disease deaths, and incremental cost-effectiveness ratio ICER per quality-adjusted life-year (QALY). One-way and probabilistic-sensitivity-analyses (PSA) were undertaken to explore the uncertainty. In the simulated cohort of 1 000 000 women aged 30 years in Canada, the base case ICERs of population-based BRCA testing were CAD $32 276 (US $23 402.84) per QALY from the payer perspective or CAD $16 416 (US $11 903.00) per QALY from the societal perspective compared with FH-based testing, well below the established Canadian cost-effectiveness thresholds. Population testing remained cost-effective for ages 40 to 60 years but not at age 70 years. The results were robust for multiple scenarios, 1-way sensitivity, and PSA. More than 99% of simulations from payer and societal perspectives were cost-effective on PSA (5000 simulations) at the CAD $50 000 (US $36 254.25) per QALY willingness-to-pay threshold. Population-based BRCA testing could potentially prevent an additional 2555 breast cancers and 485 ovarian cancers in the Canadian population, corresponding to averting 196 breast cancer deaths and 163 ovarian cancer deaths per 1 000 000 population. In this economic evaluation, population-based BRCA testing was cost-effective compared with FH-based testing in Canada from payer and societal perspectives. These findings suggest that changing the genetic testing paradigm to population-based testing could prevent thousands of breast and ovarian cancers.