Abstract Objective To assess the cost-effectiveness of various modes of mediastinal staging in non–small cell lung cancer (NSCLC) in a single-payer health care system. Methods We performed a decision ...analysis to compare the health outcomes and costs of 4 mediastinal staging strategies: no invasive staging, endobronchial ultrasound-guided transbronchial needle aspiration (EBUS-TBNA), mediastinoscopy, and EBUS-TBNA followed by mediastinoscopy if EBUS-TBNA is negative. We determined incremental cost effectiveness ratios (ICER) for all strategies and performed comprehensive deterministic sensitivity analyses using a willingness to pay threshold of $80,000/quality adjusted life year (QALY). Results Under the base-case scenario, the no invasive mediastinal staging strategy was least effective (QALY, 5.80) and least expensive ($11,863), followed by mediastinoscopy, EBUS-TBNA, and EBUS-TBNA followed by mediastinoscopy with 5.86, 5.87, and 5.88 QALYs, respectively. The ICER was ∼$26,000/QALY for EBUS-TBNA staging and ∼$1,400,000/QALY for EBUS-TBNA followed by mediastinoscopy. The mediastinoscopy strategy was dominated. Once pN2 exceeds 2.5%, EBUS-TBNA staging is cost-effective (∼$80,000/QALY). Once the pN2 reaches 57%, EBUS-TBNA followed by mediastinoscopy is cost-effective (ICER ∼$79,000/QALY). Once EBUS-TBNA sensitivity exceeds 25%, EBUS-TBNA staging is cost-effective (ICER ∼$79,000/QALY). Once pN2 exceeds 25%, confirmatory mediastinoscopy should be added, in cases of EBUS-TBNA sensitivity ≤ 60%. Conclusions Invasive mediastinal staging in NSCLC is unlikely to be cost-effective in clinical N0 patients if pN2 <2.5%. In patients with probability of mediastinal metastasis between 2.5% and 57% EBUS-TBNA is cost-effective as the only staging modality. Confirmatory mediastinoscopy should be considered in high-risk patients (pN2 > 57%) in case of negative EBUS-TBNA.
The negative effects of loneliness on population health and wellbeing requires interventions that transcend the medical system and leverage social, cultural, and public health system resources. ...Group-based social interventions are a potential method to alleviate loneliness. Moreover, nature, as part of our social and health infrastructure, may be an important part of the solutions that are needed to address loneliness. The RECETAS European project H2020 (Re-imagining Environments for Connection and Engagement: Testing Actions for Social Prescribing in Natural Spaces) is an international research project aiming to develop and test the effectiveness of nature-based social interventions to reduce loneliness and increase health-related quality of life.
This article describes the three related randomized controlled trials (RCTs) that will be implemented: the RECETAS-BCN Trial in Barcelona (Spain) is targeting people 18+ from low socio-economic urban areas; the RECETAS-PRG Trial in Prague (Czech Republic) is addressing community-dwelling older adults over 60 years of age, and the RECETAS-HLSNK trial is reaching older people in assisted living facilities. Each trial will recruit 316 adults suffering from loneliness at least sometimes and randomize them to nature-based social interventions called "Friends in Nature" or to the control group. "Friends in Nature" uses modifications of the "Circle of Friends" methodology based on group processes of peer support and empowerment but including activities in nature. Participants will be assessed at baseline, at post-intervention (3 months), and at 6- and 12-month follow-up after baseline. Primary outcomes are the health-related quality-of-life according to 15D measure and The De Jong Gierveld 11-item loneliness scale. Secondary outcomes are health and psychosocial variables tailored to the specific target population. Nature exposure will be collected throughout the intervention period. Process evaluation will explore context, implementation, and mechanism of impact. Additionally, health economic evaluations will be performed.
The three RECETAS trials will explore the effectiveness of nature-based social interventions among lonely people from various ages, social, economic, and cultural backgrounds. RECETAS meets the growing need of solid evidence for programs addressing loneliness by harnessing the beneficial impact of nature on enhancing wellbeing and social connections.
Barcelona (Spain) trial: ClinicalTrials.gov, ID: NCT05488496. Registered 29 July 2022. Prague (Czech Republic) trial: ClinicalTrials.gov, ID: NCT05522140. Registered August 25, 2022. Helsinki (Finland) trial: ClinicalTrials.gov, ID: NCT05507684. Registered August 12, 2022.
Clinical trials demonstrate improving survival in patients with multiple myeloma (MM) after treatment. However, it is unclear whether increased survival translates to a similar benefit in a real ...world setting.
We analyzed the overall survival of 347 multiple myeloma patients in Austria by means of a national registry (AMR), focused on results from 3rd and later lines of therapy. This benchmark was chosen to define a baseline prior to the broad application of upcoming 2nd generation drugs (carfilzomib, pomalidomide).
Projected 10 years survival for patients with MM in Austria is estimated to be 56% in patients diagnosed in between the years 2011-2014, 21% in patients with a diagnosis made between 2000-2005, and 39% in those with a diagnosis made between 2006-2010). For the same intervals a significant increase in the use of both bortezomib, lenalidomide and thalidomide-so called IMiDs (from 2005 onwards) and their simultaneous use in combination therapies (from 2010 onwards) could be shown. The use of autologous transplantation (ASCT) remained more or less constant at ~ 35% of patients in the 1st line setting over the whole period, comparing well to international practice patterns, while the use of 2nd line ASCT increased from 5.5% to 18.7% of patients. Patients in 3rd or later line treatment (n = 105), showed that even in relapsed and refractory disease median survival was 27 months with a considerable proportion of long-term survivors (~20%).
With the expected emergence of additional active anti-myeloma compounds, we aim to assess survival in patients with relapsed and refractory MM.
To provide an overview of thresholds for incremental cost-effectiveness ratios (ICERs) representing willingness-to-pay (WTP) across multiple countries and insights into exemptions pertaining to the ...ICER (e.g., cancer). To compare ICER thresholds to individual country's estimated ability-to-pay.
We included AHRQ/USA, BIQG-GOEG/Austria, CADTH/Canada, DAHTA@DIMDI/Germany, DECIT-CGATS/Brazil, HAS/France, HITAP/Thailand, IQWiG/Germany, LBI-HTA/Austria, MSAC/Australia, NICE/England/Wales and SBU/Sweden. ICER thresholds were derived from systematic literature/website search/expert surveys. WTP was compared with ATP using Spearman's rank correlation.
Two general and explicitly acknowledged thresholds (England/Wales, Thailand), implicit thresholds in six countries and different ICER thresholds/decision-making rules in oncology were identified. Correlation between WTP and ability-to-pay was moderate.
Our overview supports country-specific discussions on WTP and on how to define value(s) within societies.
Clear evidence on the benefit-harm balance and cost effectiveness of population-based screening for colorectal cancer (CRC) is missing. We aim to systematically evaluate the long-term effectiveness, ...harms and cost effectiveness of different organized CRC screening strategies in Austria.
A decision-analytic cohort simulation model for colorectal adenoma and cancer with a lifelong time horizon was developed, calibrated to the Austrian epidemiological setting and validated against observed data. We compared four strategies: 1) No Screening, 2) FIT: annual immunochemical fecal occult blood test age 40-75 years, 3) gFOBT: annual guaiac-based fecal occult blood test age 40-75 years, and 4) COL: 10-yearly colonoscopy age 50-70 years. Predicted outcomes included: benefits expressed as life-years gained LYG, CRC-related deaths avoided and CRC cases avoided; harms as additional complications due to colonoscopy (physical harm) and positive test results (psychological harm); and lifetime costs. Tradeoffs were expressed as incremental harm-benefit ratios (IHBR, incremental positive test results per LYG) and incremental cost-effectiveness ratios ICER. The perspective of the Austrian public health care system was adopted. Comprehensive sensitivity analyses were performed to assess uncertainty.
The most effective strategies were FIT and COL. gFOBT was less effective and more costly than FIT. Moving from COL to FIT results in an incremental unintended psychological harm of 16 additional positive test results to gain one life-year. COL was cost saving compared to No Screening. Moving from COL to FIT has an ICER of 15,000 EUR per LYG.
Organized CRC-screening with annual FIT or 10-yearly colonoscopy is most effective. The choice between these two options depends on the individual preferences and benefit-harm tradeoffs of screening candidates.
Due to high survival rates and the relatively small benefit of adjuvant therapy, the application of personalized medicine (PM) through risk stratification is particularly beneficial in early breast ...cancer (BC) to avoid unnecessary harms from treatment. The new 21-gene assay (OncotypeDX, ODX) is a promising prognostic score for risk stratification that can be applied in conjunction with Adjuvant!Online (AO) to guide personalized chemotherapy decisions for early BC patients. Our goal was to evaluate risk-group specific cost effectiveness of adjuvant chemotherapy for women with early stage BC in Austria based on AO and ODX risk stratification.
A previously validated discrete event simulation model was applied to a hypothetical cohort of 50-year-old women over a lifetime horizon. We simulated twelve risk groups derived from the joint application of ODX and AO and included respective additional costs. The primary outcomes of interest were life-years gained, quality-adjusted life-years (QALYs), costs and incremental cost-effectiveness (ICER). The robustness of results and decisions derived were tested in sensitivity analyses. A cross-country comparison of results was performed.
Chemotherapy is dominated (i.e., less effective and more costly) for patients with 1) low ODX risk independent of AO classification; and 2) low AO risk and intermediate ODX risk. For patients with an intermediate or high AO risk and an intermediate or high ODX risk, the ICER is below 15,000 EUR/QALY (potentially cost effective depending on the willingness-to-pay). Applying the AO risk classification alone would miss risk groups where chemotherapy is dominated and thus should not be considered. These results are sensitive to changes in the probabilities of distant recurrence but not to changes in the costs of chemotherapy or the ODX test.
Based on our modeling study, chemotherapy is effective and cost effective for Austrian patients with an intermediate or high AO risk and an intermediate or high ODX risk. In other words, low ODX risk suggests chemotherapy should not be considered but low AO risk may benefit from chemotherapy if ODX risk is high. Our analysis suggests that risk-group specific cost-effectiveness analysis, which includes companion prognostic tests are essential in PM.
Introduction
Diabetes mellitus (DM) has become an important and exacerbating health epidemic, with severe consequences for both patients and health systems. The National Diabetes Strategy of Austria ...addresses the lack of high‐quality data on DM in Austria and the need for developing a national data network. The aims of our study are to establish a cohort including all adult diabetes patients in a district in western Austria, describe the demographic and clinical characteristics of this cohort, and provide an estimation of diabetes prevalence.
Methods
We recruited a population‐based cohort of adult patients with a diagnosis of DM in cooperation with a network of all caregivers. Data collection was based on a case report form, including patient characteristics, clinical parameters and long‐term complications.
Results
In total, 1845 patients with DM were recruited and analysed. We observed an overall prevalence of 5.3% 95% CI: 5.0%–5.5%. For the subsequent main analysis, we included 1755 patients with DM after excluding 90 patients with gestational DM. There were significant differences between genders in the distribution of specific clinical parameters, patient characteristics, and the long‐term complications diabetic foot, amputation and cardiovascular disease.
Conclusion
To the best of our knowledge, we established the first diabetes cohort study in Austria. Prevalence and the proportion of specific long‐term complications were lower when compared to the international context. We assume that the Diabetes Landeck Cohort has reached a high degree of completeness; however, we were not able to identify independent data sources for a valid check of completeness.
The aims of this project and study are to establish a cohort including all adult DM patients in a district in western Austria, describe the demographic and clinical characteristics of these patients, and provide an estimation of diabetes prevalence, both at an overall level and stratified by gender and age groups.
Objective More than 30% of the German population suffers from mild to moderate iodine deficiency causing goiter and other iodine deficiency disorders (IDDs). The economic burden of iodine deficiency ...is still unclear. We aimed to assess costs for prevention, monitoring and treatment of IDDs in Germany. Design We performed a comprehensive cost analysis. Methods We assessed direct medical costs and direct non-medical costs for inpatient and outpatient care of IDDs and costs for productivity loss due to the absence of work in 2018. Additionally, we calculated total costs for an IDD prevention program comprising universal salt iodization (USI). We performed threshold analyses projecting how many cases of IDDs or related treatments would need to be avoided for USI to be cost-saving. Results Annual average costs per case in the year of diagnosis were € 211 for goiter/thyroid nodules; € 308 for hyperthyroidism; and € 274 for hypothyroidism. Average one-time costs for thyroidectomy were € 4184 and € 3118 for radioiodine therapy. Average costs for one case of spontaneous abortion were € 916. Annual costs of intellectual disability were € 14,202. In the German population, total annual costs for USI would amount to 8 million Euro. To be cost-saving, USI would need to prevent, for example, 37,900 cases of goiter/thyroid nodules. Conclusion USI potentially saves costs, if a minimum amount of IDDs per year could be avoided. In order to recommend the implementation of USI, a full health-economic evaluation including a comprehensive benefit-harm assessment is needed.
Evidence on long-term effectiveness and cost effectiveness of treatment sequences for multiple myeloma (MM) is sparse. We used published data and country-specific data to assess the cost ...effectiveness of four-line treatment sequences for elderly transplant-ineligible patients with MM in Serbia.
We developed a Markov cohort model to compare long-term effectiveness and cost effectiveness of five sequential MM treatment alternatives from the perspective of the national healthcare provider. Effectiveness parameters on progression, mortality and adverse events were extracted from published clinical trials. Costs were based on price lists of the National Health Insurance Fund. We compared life expectancy, costs, and incremental cost-effectiveness ratios among alternative courses of action. The model was analyzed over a lifelong time horizon applying a 3% annual discount rate for effectiveness outcomes and costs. Robustness of the model was tested in multiple deterministic sensitivity analyses.
The sequences were defined by the frontline treatment: MPT (melphalan-prednisone-thalidomide), MPV (melphalanprednisone-bortezomib), CTD (cyclophosphamide-thalidomide-dexamethasone), VCD (bortezomib-cyclophosphamidedexamethasone) and BP (bendamustine-prednisone). MPV sequence resulted in the highest remaining life expectancy (4.76 life years). Cost-effectiveness analysis resulted in three non-dominated strategies: MPT, VCD, and MPV sequences, with an incremental cost-effectiveness ratio of EUR 35,300 per life-year gained (LYG) for VCD and EUR 47,200/LYG for MPV relative to MPT.
MPV sequence was the most effective in terms of life expectancy for elderly transplant-ineligible MM patients in Serbia. Bortezomib-based strategies would be recommended for the frontline treatment of patients with MM in Serbia if the willingness-to-pay threshold is around EUR 35,000-60,000/LYG.
Background
Bevacizumab is efficacious in delaying ovarian cancer progression and controlling ascites. The ICON7 trial showed a significant benefit in overall survival for bevacizumab, whereas the ...GOG-218 trial did not. GOG-218 allowed control group patients to switch to bevacizumab upon progression, which may have biased the results. Lack of data on switching behavior prevented the application of g-methods to adjust for switching. The objective of this study was to apply decision-analytic modeling to estimate the impact of switching bias on causal treatment-effect estimates.
Methods
We developed a causal decision-analytic Markov model (CDAMM) to emulate the GOG-218 trial and estimate overall survival. CDAMM input parameters were based on data from randomized clinical trials and the published literature. Overall switching proportion was based on GOG-218 trial information, whereas the proportion switching with and without ascites was estimated using calibration. We estimated the counterfactual treatment effect that would have been observed had no switching occurred by denying switching in the CDAMM.
Results
The survival curves generated by the CDAMM matched well with the ones reported in the GOG-218 trial. The survival curve correcting for switching showed an estimated bias such that 79% of the true treatment effect could not be observed in the GOG-218 trial. Results were most sensitive to changes in the proportion progressing with severe ascites and mortality.
Limitations
We used a simplified model structure and based model parameters on published data and assumptions. Robustness of the CDAMM was tested and model assumptions transparently reported.
Conclusions
Medical-decision science methods may be merged with empirical methods of causal inference to integrate data from other sources where empirical data are not sufficient. We recommend collecting sufficient information on switching behavior when switching cannot be avoided.
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