Societal Perspective Research Articles

Access in all areas? A round up of developments in market access and HTA: part 5.

In this latest update, we explore the recent announcement by Canada's Drug Agency (CDA-AMC, formerly CADTH) on their pilot to include the societal perspective in the evaluation of certain new medicines; a recent Office of Health Economics (OHE) report on the evaluation of HTA agency methods over time; and publications examining the impact of Project Orbis on patient access to oncology treatments.

A systematic review of cost-effectiveness analyses of gene therapy for hemophilia type A and B.

In 2022-2023, the US Food and Drug Administration approved 2 novel gene therapies, valoctocogene roxaparvovec and etranacogene dezaparavovec, for hemophilia A and B, respectively. These one-time-administered gene therapies have been marketed at prices that create financial challenges for payers and patients. Understanding the magnitude and uncertainties around the long-term value of these therapies and how they can potentially relate to managed care practices is of high interest to the payer and patient community. To conduct a systematic review of cost-effectiveness analysis (CEA) studies to assess (1) the long-term value of valoctocogene roxaparvovec and etranacogene dezaparavovec and (2) the relevance and validity of the underlying data and assumptions used in the CEA models and discuss how they relate to the challenges identified for CEAs of gene therapies. A systematic review of cost-effectiveness studies of novel hemophilia A and B gene therapy was conducted. PubMed and Embase were searched for published studies from inception to January 12, 2024. Original research articles published in English that conducted a CEA on gene therapy treatments for hemophilia A and B, with a comparison of incremental costs and health effects, were considered. Critical appraisal of the quality of reporting and the underlying modeling assumptions were conducted to assess the relevance and validity of the results. Two hundred thirty-eight studies were identified, of which 4 met the inclusion criteria. Three studies were conducted from a US health care perspective and 1 from a Dutch societal perspective. Despite the high upfront costs of the gene therapies, all included studies' (3 hemophilia A and 1 hemophilia B) modeled results showed that gene therapies had lower overall costs and better health outcomes compared with factor concentrate replacement therapies and emicizumab. The results were driven by the assumption that gene therapies will have a durable effect of at least 10 years and offset the high cost of the current standard of care. The modeled health improvements varied substantially across studies, showing that the long-term value is sensitive to varying clinical and economic assumptions. The novel hemophilia gene therapy treatments can potentially be a cost-effective use of treatment resources if the treatment effects are durable over time. To reduce the risk for payers while still facilitating patient access, outcomes-based agreements similar to what has recently been proposed by the Centers for Medicare & Medicaid Services for sickle-cell therapies are well supported.

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.

Cost-effectiveness analysis of COVID-19 booster doses and oral antivirals: Case studies in the Indo-Pacific.

Decision-makers in middle-income countries need evidence on the cost-effectiveness of COVID-19 booster doses and oral antivirals to appropriately prioritise these healthcare interventions. We used a dynamic transmission model to assess the cost-effectiveness of COVID-19 booster doses and oral antivirals in Fiji, Indonesia, Papua New Guinea, and Timor-Leste. We conducted cost-effectiveness analysis from both healthcare and societal perspectives using data collated from publicly available sources. We developed an interactive R Shiny which allows the user to vary key model assumptions, such as the choice of discounting rate, and view how these assumptions affect model results. Booster doses were cost saving and therefore cost-effective in all four middle-income settings from both healthcare and societal perspectives using 3% discounting. Providing oral antivirals was cost-effective from a healthcare perspective if procured at a low generic price (US$25) or middle-income reference price (US$250); however, their cost-effectiveness was strongly influenced by rates of wastage or misuse, and the ongoing costs of care for patients hospitalised with COVID-19. The cost or wastage of rapid antigen tests did not appear strongly influential over the cost-effectiveness of oral antivirals in any of the four study settings. Our results support that COVID-19 booster programs are cost-effective in middle-income settings. Oral antivirals demonstrate the potential to be cost-effective if procured at or below a middle-income reference price of US$250 per schedule. Further research should quantify the rates of wastage or misuse of oral COVID-19 antivirals in middle-income settings.

Treatments with versus without medication for children with behavioural difficulties in clinical practice: an economic evaluation with observational data.

Economic evaluations of treatments for children with behavioural difficulties (i.e., characteristics of attention-deficit/hyperactivity disorder (ADHD) and/or oppositional defiant disorder (ODD)) usually rely on data of randomised controlled trials or are model-based. Findings of such studies may not be representative of cost-effectiveness and cost-utility in clinical practice. The current longitudinal study aimed to perform an economic evaluation of treatments for children with hyperactivity, impulsive behaviours, inattention, and/or behavioural difficulties using observational data that were obtained in clinical practice. Parents of 209 children (aged 5-12) who were referred to 1 of 10 Dutch youth mental healthcare institutions and who received treatment with (n = 108) or without (n = 101) the use of medication, filled out questionnaires at three timepoints (baseline, and ~ 6 and ~12 months later). Propensity score matching was used to make both groups comparable. Outcomes included quality-adjusted life years (QALYs), ADHD and ODD symptom severity, and impairment. Costs were measured from a societal perspective. Incremental cost-effectiveness ratios (ICERs) were estimated, and cost-effectiveness acceptability curves (CEACs) were derived to show uncertainty around the ICER. Results did not show statistically significant differences in costs and effects between children who were treated with medication (alone or in combination with non-medication treatment) and those who were treated without medication. CEAC suggested that medication treatment has a 55% probability of being cost-effective at the €80,000 threshold and 36% at the €20,000 threshold compared with treatment without medication. Using observational data, our study did not provide clear evidence of the cost-effectiveness and cost-utility of treatment with medication compared with treatment without medication in clinical practice.

Budget impact analysis of high-priced orphan medicinal products intended for the treatment of rare diseases in China: evidence from a densely populated metropolis of Chengdu

BackgroundAn increasing number of orphan medicinal products (OMPs) are being included in social health insurance schemes, significantly improving access to medicines for patients with rare diseases. However, high-priced OMPs are still not covered, primarily due to health equity controversies and inadequate data systems required for economic evaluation. The aim of this study was to estimate the burden of drug expenditures and the size of the reimbursement budget required for high-priced OMPs from the perspectives of society and healthcare payers.MethodsThe study performed a budget impact analysis using data from multiple sources to estimate the reimbursement budget for high-priced OMPs in Chengdu, a densely populated metropolis in China. The budget analysis consisted of three main elements: the number of patients, the price of drugs, and the simulated policy scenario. By adjusting the combinations of these elements, the budget fluctuations for payers were estimated. Furthermore, the study predicted the budget trend for the next three years to validate its sustainability.ResultsThe analysis indicated that 98 rare disease patients in Chengdu required high-priced OMPs in 2019. This suggested a projected budget of CNY 179 million for these patients without reimbursement policies, from a societal perspective. Under six assumed policy scenarios, this budget ranged from CNY 32 million to CNY 156 million. Over the next three years, the annual budget was estimated to range from CNY 200 million to CNY 1.303 billion.ConclusionIntegration of multi-source data helps to obtain more scientifically reliable results on budget impacts. The study found that the budgetary impacts of high-priced OMPs on society and payers are relatively limited. Health policymakers can choose appropriate reimbursement strategies based on financial affordability among a diverse mix of elements. The results of related studies provided insights for optimizing the allocation of health resources and improving patient access to medications.

Cost-Effectiveness of Hepatitis E Vaccination Strategies among Patients with Chronic Liver Diseases in China: A Model-Based Evaluation.

Hepatitis E virus (HEV) is a leading cause of acute viral hepatitis worldwide, primarily transmitted through contaminated water and food. In patients with chronic liver disease (CLD), HEV infection might worsen the prognosis. This study aimed to evaluate the cost-effectiveness of hepatitis E vaccination strategies in CLD patients. A decision tree-Markov cohort model was used to assess the cost-effectiveness of universal-vaccination, vaccination-following-screening, and no-vaccination strategies in 100,000 CLD patients over their lifetimes, simulating cohorts aged ≥16 years, ≥40 years, and ≥60 years, based on the licensed vaccination ages and typical ages of CLD onset, from a societal perspective. Model parameters were retrieved and estimated from previous publications and government data. The outcomes included HEV-related cases, costs, and the incremental cost-effectiveness ratio (ICER). Compared to no-vaccination, universal-vaccination reduced HEV-related cases by 32.8% to 39.6%, while vaccination-following-screening reduced them by 38.1% to 49.3%. Furthermore, universal-vaccination showed ICERs of USD 6898.33, USD 6638.91, and USD 6582.69 per quality-adjusted life year (QALY) for cohorts aged ≥16, ≥40, and ≥60 years, respectively. Moreover, the vaccination-following-screening strategy significantly enhanced cost-effectiveness, with ICERs decreasing to USD 6201.55, USD 5199.46, and USD 4919.87 per QALY for the cohorts. Additionally, one-way sensitivity analysis identified the discount rate and utility for CLD patients as the key factors influencing ICER. Probabilistic sensitivity analysis indicated the vaccination-following-screening strategy was cost-effective with probabilities of 92.50%, 95.70%, and 95.90% for each cohort. Hepatitis E vaccination in CLD patients costs less than GDP per capita for each QALY gained in China. The vaccination-following-screening strategy may be the optimal option, especially in those over 60 years.

Weaving the Web to achieve the SDGs: the presence of women in the boardrooms as a key driver

Purpose Due to the importance of mitigating the issues that afflict society and natural habitats on a global scale, the United Nations has established 17 key goals to directly and effectively mitigate problems such as extreme poverty, lack of access to quality educational environments or the loss of life in underwater and terrestrial ecosystems, which need to be addressed from multiple parties, particularly from the business network. In this context, the purpose of this paper is to analyse the potential impact of female participation in boardrooms on the disclosure of corporate information regarding the integration of the Sustainable Development Goals (SDGs) into business strategy. Design/methodology/approach The research hypotheses were developed on the basis of a multi-theoretical perspective, which included agency theory, upper echelon theory, resources dependency theory and critical mass theory. The hypotheses were tested using a regression model for a sample of 8,903 companies for the period 2019–2022. Findings The results highlight the importance of an increase in female boardroom representation as a key driver in the achieving of the SDGs with favourable effects on environmental and social issues, being robust to changes in methodological specifications. Research limitations/implications This research provides valuable evidence for the analysis of gender issues in the business field; however, it has some limitations that can be addressed by academics. In this sense, this research solely examined the impact of chief executive officer (CEO) duality while overlooking the potential for its influence to be amplified by a distinctive configuration of the board, such as the involvement of a former CEO, a greater or lesser degree of participation of executive directors in the boardroom or the fact that the CEO is a woman. These factors could either reinforce or obstruct the benefits of female directors in the accomplishment of the SDGs. Practical implications From a practical standpoint, this research illustrates the advantages of the active involvement of women in the boardroom. Their participation has resulted in notable modifications to corporate strategies, with a focus on aligning business actions with the attainment of the SDGs and related transparency. This facilitates a more comprehensive understanding of the business interest in conserving natural habitats and eradicating social inequalities. Social implications From a societal perspective, the findings illustrate the significance of actively promoting the involvement of women in organisational leadership roles. The inclusion of women's perspectives can enhance the planning of business processes, with far-reaching implications for stakeholders. Therefore, it is vital to guarantee opportunities for women, such as access to higher education and gainful employment, particularly in nations where there are considerable constraints to the intellectual and economic advancement of girls and women. Originality/value The findings contribute to the growing body of knowledge on the optimal configuration of the boardroom, especially the gendered boardroom (i.e. García-Sánchez et al., 2023b; Liao et al., 2015), and its ability to address today's global challenges. Furthermore, the findings indicate the necessity of establishing pathways for women to reach positions of influence within the organisational hierarchy, thereby facilitating beneficial societal changes. These changes can include the mitigation of environmental damage and the rectification of historical social inequalities.

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.

Poor sample size reporting quality and insufficient sample size in economic evaluations conducted alongside pragmatic trials: a cross-sectional survey

ObjectivesEconomic evaluations based on well-designed and -conducted pragmatic randomized controlled trials (pRCTs) can provide valuable evidence on the cost-effectiveness of interventions, enhancing the relevance and applicability of findings to healthcare decision-making. However, economic evaluation outcomes are seldom taken into consideration during the process of sample size calculation in pragmatic trials. The reporting quality of sample size and information on its calculation in economic evaluations that are well-suited to pRCTs remain unknown. This study aims to assess the reporting quality of sample size and estimate the power values of economic evaluations in pRCTs. Study Design and SettingWe conducted a cross-sectional survey using data of pRCTs available from PubMed and OVID from 1 January 2010 to 24 April 2022. Two groups of independent reviewers identified articles; three groups of reviewers each extracted the data. Descriptive statistics presented the general characteristics of included studies. Statistical power analyses were performed on clinical and economic outcomes with sufficient data. ResultsThe electronic search identified 715 studies and 152 met the inclusion criteria. Of these, 26 were available for power analysis. Only 9 out of 152 trials (5.9%) considered economic outcomes when estimating sample size, and only one adjusted the sample size accordingly. Power values for trial-based economic evaluations and clinical trials ranged from 2.56% to 100% and 3.21%–100%, respectively. Regardless of the perspectives, in 14 out of the 26 studies (53.8%), the power values of economic evaluations for quality-adjusted life years (QALYs) were lower than those of clinical trials for primary endpoints (PEs). In 11 out of the 24 (45.8%) and in 8 out of the 13 (61.5%) studies, power values of economic evaluations for QALYs were lower than those of clinical trials for PEs from the healthcare and societal perspectives, respectively. Power values of economic evaluations for non-QALYs from the healthcare and societal perspectives were potentially higher than those of clinical trials in 3 out of the 4 studies (75%). The power values for economic outcomes in Q1 were not higher than those for other journal impact factor quartile categories. ConclusionTheoretically, pragmatic trials with concurrent economic evaluations can provide real-world evidence for healthcare decision makers. However, in pRCT-based economic evaluations, limited consideration, and inadequate reporting of sample-size calculations for economic outcomes could negatively affect the results’ reliability and generalisability. We thus recommend that future pragmatic trials with economic evaluations should report how sample sizes are determined or adjusted based on the economic outcomes in their protocols to enhance their transparency and evidence quality.

Cost-effectiveness analysis of transthoracic echocardiographic assessment in patients with ischemic stroke or TIA of undetermined cause

BackgroundThe multicenter ATTEST study recently assessed 1084 patients with ischemic stroke or transient ischemic attack (TIA) of undetermined cause and found that routine transthoracic echocardiography (TTE) detects abnormalities with treatment implications (i.e., major cardiac sources of embolism) in only 1 % of patients, of whom most (91 %) also had major electrocardiographic (ECG)-abnormalities. In this study, we performed a cost-effectiveness analysis of different TTE strategies. MethodsWe compared the cost-effectiveness of three strategies of TTE assessment: (1) TTE in all patients; (2) TTE only in patients with major ECG-abnormalities; and (3) TTE not performed. Input data were derived from ATTEST and systematic literature reviews. A Markov model was developed that simulated recurrent ischemic stroke or TIA and intracranial and gastro-intestinal bleeding complications in patients with ischemic stroke or TIA of undetermined cause. Primary outcome was the additional costs per additional quality-adjusted life-year (QALY) from a Dutch societal perspective. ResultsPerforming TTE only in patients with major ECG-abnormalities led to 0.0083 additional QALYs and €108 additional costs per patient as compared with not performing TTE (€12,987/QALY). Performing TTE in all patients resulted in 0.0005 additional QALYs and €422 additional costs per patient as compared with performing TTE only in case of major ECG-abnormalities (€805,336/QALY). ConclusionsIn patients with ischemic stroke or TIA of undetermined cause, a strategy of performing TTE only in patients who also had major ECG-abnormalities resulted in the most favorable ratio of additional costs per additional QALY. This supports performing TTE only in patients, who also have major ECG-abnormalities.

The need for a broad perspective when assessing value-for-money for out-of-hours primary care.

Out-of-hours primary care (OOH-PC) has emerged as a promising solution to improve efficiency, accessibility, and quality of care and to reduce the strain on emergency departments. As this modality gains traction in diverse healthcare settings, it is increasingly important to fully assess its societal value-for-money and conduct thorough process evaluations. However, current economic evaluations mostly emphasise direct- and short-term effect measures, thus lacking a broader societal perspective. This study offers a comprehensive overview of current effect measures in OOH-PC evaluations and proposes additional measures from the evaluation of integrated care programmes. First, we systematically identified the effect measures from published cost-effectiveness studies and classified them as process, outcome, and resource use measures. Second, we elaborate on the incorporation of 'productivity gains', 'health promotion and early intervention', and 'continuity of care' as additional effects into economic evaluations of OOH-PC. Seeking care affects personal and employee time, potentially resulting in decreased productivity. Challenges in taking time off work and limited access to convenient care are often cited as barriers to accessing primary care. As such, OOH-PC can potentially reduce opportunity costs for patients. Furthermore, improving access to healthcare is important in determining whether people receive promotional and preventive services. Health promotion involves empowering people to take control of their health and its determinants. Given the unscheduled nature and the fragmented or rotational care in OOH-PC, the degree to which interventions and modalities provide continuity should be monitored, assessed, and included in economic evaluations. Continuity of care in primary care improves patient satisfaction, promotes adherence to medical advice, reduces reliance on hospitals, and reduces mortality. Although it is essential to also address local settings and needs, the integration of broader scope measures into OOH-PC economic evaluations improves the comprehensive evaluation that aligns with welfare gains.

Estimating the Economically Justifiable Price of Limited-Duration Treatment with Donanemab for Early Symptomatic Alzheimer's Disease in the United States.

The goal of this economic model is to estimate an economically justifiable price (EJP) for using donanemab for the treatment of early symptomatic Alzheimer's disease (AD) in the United States based on clinical data from the phase 3 TRAILBLAZER-ALZ 2 trial (NCT04437511). We adapted an AD Markov state-transition model developed by the Institute for Clinical and Economic Review to estimate the EJP for donanemab at different willingness-to-pay (WTP) thresholds from the health care system perspective and the societal perspective as co-base cases. Assuming a WTP threshold of $150,000 per quality-adjusted life-year (QALY) gained, the model estimates a 1-year (13-dose) EJP for donanemab of $80,538 from the health care system perspective and $91,126 from the societal perspective; at a WTP threshold of $100,000 per QALY gained, the model estimates a 1-year (13-dose) EJP for donanemab of $44,691 from the health care system perspective and $55,419 from the societal perspective. Mean total treatment costs per patient at the $150,000 per QALY gained EJP derived from the health care system perspective were estimated at $77,812 based on the average number of doses of donanemab patients received in the co-base case analysis. One-way sensitivity analysis (OWSA) indicated that treatment efficacy, disease severity at the time of treatment initiation, and duration of treatment effect were the main drivers of the potential EJP. Results from this modeling simulation informed by the TRAILBLAZER-ALZ 2 study support an EJP for limited-duration treatment with donanemab that exceeds per-dose list prices for currently available amyloid-targeting therapies, implying potentially lower lifetime costs and better value for money.

Is a 1-year course of methotrexate in patients with arthralgia at-risk for rheumatoid arthritis cost-effective? A cost-effectiveness analysis of the randomised, placebo-controlled TREAT EARLIER trial

ObjectivesRheumatoid arthritis (RA) has a considerable disease burden with life-long physical limitations, reduced work productivity and high societal costs. Trials on arthralgia at-risk for RA are therefore conducted, aiming to...

The costs of implementing anaemia reduction interventions among women fish processors in Ghana

BackgroundAnaemia among women of reproductive age (WRA) remains persistently high in Ghana, affecting 41% in 2022. Women in low-income communities in Ghana engaged in fish processing activities are at increased risk of anaemia due to inadequate diets, exposure to infectious pathogens, and pollutants. The Invisible Fishers (IFs) project was implemented among women fish processors in their reproductive age in Central and Volta regions of Ghana to mitigate anaemia. Despite the efficacy, feasibility and scalability of the intervention, the cost of implementing the intervention is unknown. The objective of this study was to estimate the costs of implementing the IFs project in Ghana.MethodsWe used micro-costing approach to analyse the costs of implementing the IFs project. Data were collected as part of a pilot randomized control trial with three interventions: Behaviour Change Communication (BCC), Strengthening Market Engagement of fish processors plus Behaviour Change Communication (SME + BCC), and Fish Smoking Technology and Practices plus Behaviour Change Communication (FST + BCC). The interventions were delivered to 60 women fish processors in the Central region and 60 in Volta region. The cost of the intervention was estimated from the societal perspective. Economic costs were categorized as direct costs (i.e. personnel, transportation, meetings, training, and monitoring) and indirect cost (i.e. value of productive time lost due to women and community volunteers’ participation in the activities of the IFs project).ResultsThe FST + BCC had the highest average cost per beneficiary (US$11898.62), followed by the SME + BCC (US8962.93). The least expensive was the BCC (US$4651.93) over the intervention period of 18 months. Recurrent costs constituted the largest component of economic costs (98%). Key drivers of direct costs were personnel (58%), administrative expenses (14%), and transportation (7%).ConclusionThere is a high cost for implementing interventions included in the IFs project. Planning and scaling -up of the interventions across larger populations could bring about economies of scale to reduce the average cost of the interventions.

Cooperation in Return-to-work Interventions for Common Mental Disorders: An Ideal Theory Analysis of Actors, Goals, and Ethical Obstacles.

The rise in the number of people on sick leave for common mental disorders is a growing concern, both from a societal and individual perspective. One common suggestion to improve the return-to-work process is increased cooperation between the relevant parties, including at least the employer, the social insurance agency and health care. This suggestion is often made on the presumption that all parties share the common goal of reintegrating the patient-employee back into the workplace. In this paper we investigate this presumption by mapping out the ethical frameworks of these three key actors in any return-to-work process. We show that although the goals of these actors often, and to a large extent, overlap there are potential differences and tensions between their respective goals. Further, we emphasise that there may be other limitations to an actor's participation in the process. In particular the health care system is required to respect patient autonomy and confidentiality. There is also an inherent tension in the dual roles of health care professionals as therapists and expert witnesses in work ability assessment. In conclusion, there are potential tensions between the key actors in the return-to-work process. These tensions need to be addressed in order to enable an increased cooperation between actors and to facilitate the development of a feasible plan of action for all parties, including the employee.

Cost-Utility Analysis of Erenumab Compared to Topiramate for Preventive Therapy of Migraine in Iran

Background: Migraine is a prevalent, chronic neurovascular disorder that incurs significant indirect costs due to productivity loss. Preventive therapy is an effective way to alleviate the societal and healthcare burden of migraine. Approximately 14% of both the global and Iranian populations are affected by migraine, which has substantial economic implications. Objectives: To determine the cost-effectiveness of Erenumab compared to Topiramate for migraine treatment in Iran. Methods: A three-state Markov model was used to evaluate the cost-effectiveness of Erenumab. The model considered both direct and indirect costs from a societal perspective. The incremental cost-effectiveness ratio (ICER) was calculated by determining the cost per quality-adjusted life year (QALY) gained. Costs and QALYs were discounted annually at 5.8% and 5%, respectively. Deterministic and probabilistic sensitivity analysis (PSA) were performed to assess the robustness of the model. Results: The average cost for patients using the Erenumab strategy was 16,836 USD over five years, whereas the average cost for the Topiramate strategy was estimated to be 2,660 USD. Additionally, the average QALYs for the Erenumab and Topiramate strategies were 3.64 and 3.46, respectively. The ICER for the Erenumab strategy was 78,923 USD/QALY. This ICER is significantly higher than the fixed Iranian willingness-to-pay (WTP) threshold of 2,456 USD. Conclusions: The study concludes that preventive treatment of migraine with Erenumab, compared to Topiramate, is not cost-effective in Iran based on current prices. Therefore, for Erenumab to be considered cost-effective, a significant price reduction is necessary for its entry into the Iranian pharmaceutical market.

Cost-effectiveness analysis of a lung cancer screening program in the Netherlands: a simulation based on NELSON and NLST study outcomes

Background In the Netherlands, lung cancer is the leading cause of cancer-related death, accounting for more than 10,000 annual deaths. Lung cancer screening (LCS) studies using low-dose computed tomography (LDCT) have demonstrated that early detection reduces lung cancer mortality. However, no LCS program has been implemented yet in the Netherlands. A national LCS program has the potential to enhance the health outcomes for lung cancer patients in the Netherlands. Objective and Methods This study evaluates the cost-effectiveness of LCS compared to no-screening in the Netherlands, by simulating the screening outcomes based on data from NEderlands–Leuvens Longkanker Screenings ONderzoek (NELSON) and National Lung Screening Trial (NLST). We simulated annual screening up to 74 years of age, using inclusion criteria from the respective studies. A decision tree and Markov model was used to predict the incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICERs) for the screening population. The analysis used a lifetime horizon and a societal perspective. Results Compared to no-screening, LCS resulted in an ICER of €5,169 per QALY for the NELSON simulation, and an ICER of €17,119 per QALY for the NLST simulation. The screening costs were highly impactful for the cost-effectiveness. The most influential parameter was the CT scan cost. Cost reduction for CT from €201 to €101 per scan would reduce the ICER to €2,335 using NELSON criteria. Additionally, LCS could prevent 15,115 and 12,611 premature lung cancer deaths, accompanied by 1.66 and 1.31 QALYs gained per lung cancer case for the NELSON and NLST simulations, respectively. Conclusion LCS was estimated to be cost-effective in the Netherlands for both simulations at a willingness-to-pay threshold of €20,000 per QALY. Using the NELSON criteria, less than €5,500 per QALY had to be spent. Lowering the cost per CT exam would lead to a further reduction of this amount.

Cost-effectiveness analysis of e-cigarettes compared to nicotine replacement therapy for smoking cessation among Medicaid users in California.

Smoking is the leading preventable cause of death and disability in the United States (US). A 2019 randomized controlled trial found e-cigarettes are more effective than nicotine replacement therapy (NRT) in achieving abstinence when both are combined with behavioral counselling. Few cost-effectiveness studies have been performed regarding e-cigarettes as an adjunct to cessation efforts, and none in a US context. This study evaluated the cost-effectiveness of e-cigarettes compared to NRT, both with counselling, as cessation aids for Medicaid smokers in California. A cost-effectiveness analysis from a US societal perspective comparing e-cigarettes with NRT was constructed. Using a lifetime horizon, smokers were followed to different health states to their expected lifespan. Costs were expressed in 2020 US dollars and health outcomes in quality-adjusted life years. All costs and outcomes were discounted at a 3% annual rate and model findings were assessed with a series of sensitivity analyses. Using US willingness-to-pay thresholds, e-cigarettes were more cost-effective than NRT, with an incremental cost-effectiveness ratio of $11,454 per additional QALY gained over smokers' lifetimes. Results were sensitive to smokers' healthcare costs, model start ages, discount rate, and QALY weights. E-cigarettes used as a cessation treatment by smokers are more cost-effective relative to NRT. However, e-cigarettes as a treatment are currently unapproved by the Food and Drug Administration (FDA). Despite lack of FDA approval, e-cigarettes are a popular choice among smokers trying to quit. Policymakers should consider whether providing financial support for e-cigarettes as a harm reduction strategy is worth pursuing. As e-cigarettes grow in popularity and are an increasingly preferred choice of smoking cessation aid among smokers attempting to quit, it is important to examine the inclusion of these products in tobacco policy, research, and practice. Several countries have now adopted or are in the process of adopting e-cigarettes as a medically licensed and regulated smoking cessation aid. Future smoking cessation strategies by US policymakers may consider the adoption of e-cigarettes as a cost-effective harm reduction tool for use by current smokers seeking to quit.

Artificial-Intelligence Cloud-Based Platform to Support Shared Decision-Making in the Locoregional Treatment of Breast Cancer: Protocol for a Multidimensional Evaluation Embedded in the CINDERELLA Clinical Trial.

Shared decision-making (SDM) plays a crucial role in breast cancer care by empowering patients and reducing decision regret. Patient decision aids (PtDAs) are valuable tools for facilitating SDM, now available in digital and artificial intelligence (AI)-powered formats to offer increasingly personalized contents. The ongoing CINDERELLA clinical trial (ClinicalTrials.gov: NCT05196269) evaluates an innovative AI cloud-based approach using a web platform and a mobile application (CINDERELLA APProach) versus the conventional approach to support SDM in breast cancer patients undergoing locoregional treatment. This protocol outlines a trial-based multidimensional evaluation, encompassing economic, financial, implementability, and environmental considerations associated with the CINDERELLA APProach. A within-trial cost-consequence and cost-utility analysis from a societal perspective will be performed using patient-level data on outcomes and resource use. The latter will be valued in monetary terms using country-specific unit costs or patient valuations. A budget impact analysis will be performed over 1 and 5 years from the budget holder perspectives. The CINDERELLA APProach implementability will be assessed through an evaluation of its usability, acceptability, organizational impact, and overall feasibility. The environmental impact will be quantitatively assessed across several dimensions, such as quantity, appropriateness, and emissions, supplemented by qualitative insights. Overall, data for the evaluation will be gathered from patient questionnaires, interviews with patients and managers, focus groups with healthcare professionals, and app electronic data. A thorough understanding of the broad consequences of the CINDERELLA APProach may foster its successful translation into real-world settings, hopefully benefiting breast cancer patients and clinical practice.