IntroductionDespite a recent meta-analysis including 31 randomised controlled trials comparing methadone and buprenorphine for the treatment of opioid use disorder, important knowledge gaps remain ...regarding the long-term effectiveness of different treatment modalities across individuals, including rigorously collected data on retention rates and other treatment outcomes. Evidence from real-world data represents a valuable opportunity to improve personalised treatment and patient-centred guidelines for vulnerable populations and inform strategies to reduce opioid-related mortality. Our objective is to determine the comparative effectiveness of methadone versus buprenorphine/naloxone, both overall and within key populations, in a setting where both medications are simultaneously available in office-based practices and specialised clinics.Methods and analysisWe propose a retrospective cohort study of all adults living in British Columbia receiving opioid agonist treatment (OAT) with methadone or buprenorphine/naloxone between 1 January 2008 and 30 September 2018. The study will draw on seven linked population-level administrative databases. The primary outcomes include retention in OAT and all-cause mortality. We will determine the effectiveness of buprenorphine/naloxone vs methadone using intention-to-treat and per-protocol analyses—the former emulating flexible-dose trials and the latter focusing on the comparison of the two medication regimens offered at the optimal dose. Sensitivity analyses will be used to assess the robustness of results to heterogeneity in the patient population and threats to internal validity.Ethics and disseminationThe protocol, cohort creation and analysis plan have been approved and classified as a quality improvement initiative exempt from ethical review (Providence Health Care Research Institute and the Simon Fraser University Office of Research Ethics). Dissemination is planned via conferences and publications, and through direct engagement and collaboration with entities that issue clinical guidelines, such as professional medical societies and public health organisations.
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.
Objectives: The purpose of this study was to assess the accuracy of contrast-enhanced magnetic resonance imaging and diffusion-weighted imaging in distinguishing benign from malignant non-mass-like ...breast lesions. Methods: 103 lesions showing non-mass-like enhancement in 100 consecutive patients were analyzed. Distribution, internal enhancement patterns, and contrast kinetic curve patterns were classified according to the BI-RADS lexicon. Apparent diffusion coefficient (ADC) values were obtained from manually placed regions of interest (ROIs) on diffusion-weighted images. The optimal ADC value threshold for the distinction between benign and malignant lesions was determined by ROC analysis. Univariate and multivariate analyses were performed to identify independent predictors of malignancy, and the probability of malignancy was calculated for various combinations of findings. Histological diagnosis obtained by means of core needle biopsy was used as gold standard. Results: According to the univariate and multivariate analysis, odds ratios for malignancy were significantly elevated for clumped or clustered ring internal enhancement and low ADC values (p < 0.001), whereas distribution patterns and contrast kinetic patterns were not significantly correlated with benignity or malignancy. In non-mass lesions with homogeneous or heterogeneous internal enhancement and ADC values greater than 1.26×10-3mm2/s, no malignancy was detected, while all other combinations of findings had a probability of malignancy ranging from 22.2 to 76.6%. Conclusions: A combination of BI-RADS descriptors of internal enhancement and ADC values is useful for the differential diagnosis of lesions showing non-mass enhancement. Lesions with homogeneous or heterogeneous enhancement and high ADC can be followed up, while all other lesions should be biopsied. Doi: 10.28991/SciMedJ-2021-0302-1 Full Text: PDF
On the role of data, statistics and decisions in a pandemic Jahn, Beate; Friedrich, Sarah; Behnke, Joachim ...
Advances in statistical analysis : AStA : a journal of the German Statistical Society,
2022/9, Letnik:
106, Številka:
3
Journal Article
Recenzirano
Odprti dostop
A pandemic poses particular challenges to decision-making because of the need to continuously adapt decisions to rapidly changing evidence and available data. For example, which countermeasures are ...appropriate at a particular stage of the pandemic? How can the severity of the pandemic be measured? What is the effect of vaccination in the population and which groups should be vaccinated first? The process of decision-making starts with data collection and modeling and continues to the dissemination of results and the subsequent decisions taken. The goal of this paper is to give an overview of this process and to provide recommendations for the different steps from a statistical perspective. In particular, we discuss a range of modeling techniques including mathematical, statistical and decision-analytic models along with their applications in the COVID-19 context. With this overview, we aim to foster the understanding of the goals of these modeling approaches and the specific data requirements that are essential for the interpretation of results and for successful interdisciplinary collaborations. A special focus is on the role played by data in these different models, and we incorporate into the discussion the importance of statistical literacy and of effective dissemination and communication of findings.
Tumor profiling is increasingly used in advanced cancer patients to define treatment options, especially in refractory cases where no standard treatment is available. Caris Molecular Intelligence ...(CMI) is a multiplatform tumor profiling service that is comprehensive of next-generation sequencing (NGS) of DNA and RNA, immunohistochemistry (IHC) and in situ hybridisation (FISH). The aim of this study is to compare costs of CMI-guided treatment with prior or planned treatment options in correlation with outcome results.
Retrospective data from five clinical trials were collected to define the treatment decision prior to the receipt of the CMI report (n = 137 patients). A systematic review of treatment data from 11 clinical studies of CMI (n = 385 patients) allowed a comparison of planned vs actual (n = 137) and prior vs actual (n = 229) treatment costs.
Treatment plan was changed in 88% of CMI-profiled cases. The actual CMI guided treatment cost per cycle was £995 in 385 treated patients. Planned treatment costs were comparable to actual treatment costs (£979 vs £945; p = 0.7123) and prior treatment costs were not significantly different to profiling-guided treatments (£892 vs £850; p = 0.631).
Caris Molecular Intelligence guided treatment cost per cycle was in the range of prior or planned treatment cost/cycle. Due to beneficial overall survival the additional cost of performing CMI's multiplatform testing to the treatment costs seems to be cost-effective.
Background
Clinical decision‐making for patients with myelodysplastic syndromes (MDS) is challenging, and both disease and treatment effects heavily impact health‐related quality of life (HRQoL) of ...these patients. Therefore, disease‐specific HRQoL measures can be critical to harness the patient voice in MDS research.
Methods
We report a prospective international validation study of the Quality of Life in Myelodysplasia Scale (QUALMS) with a main focus on providing information on the psychometric characteristics of its three subscales: physical burden (QUALMS‐P), emotional burden (QUALMS‐E), and benefit finding (QUALMS‐BF). The analysis is based on patients enrolled from three European countries and Israel, participating to the MDS‐RIGHT Project. The scale structure and psychometric properties of the QUALMS were assessed.
Results
Overall, 270 patients with a median age of 74 years were analyzed and the majority of them (60.3%) had a low MDS‐Comorbidity Index score. Results of the confirmatory factor analysis supported the underlying scale structure of the QUALMS, which, in addition to a total score, includes three subscales: QUALMS‐P, QUALMS‐E, and the QUALMS‐BF. The QUALMS‐P exhibited the highest Cronbach's alpha coefficients. Discriminant validity analysis indicated good results with the QUALMS‐P and QUALMS‐E distinguishing between patients with different performance status, comorbidity, anemia, and transfusion dependency status. No floor and ceiling effects were observed. Responsiveness to change analysis supported the validity of the measure. Patients with a hemoglobin (Hb) level of <11 g/dL at study entry, who subsequently showed an improvement in their Hb levels, also reported a mean score change of 9 and 8 points (scales ranging between 0 and 100) in the expected direction of the QUALMS‐E and QUALMS‐P, respectively.
Conclusions
Our study provides additional validation data on the QUALMS from the international MDS‐RIGHT Project. The use of this disease‐specific HRQoL measure may contribute to raise quality standards of patient‐centered outcomes research in MDS.
The QUALMS is a Patient‐Reported Outcome measure to assess health‐related quality of life in patients with myelodysplastic syndromes. This study, conducted across three European countries and Israel, supports the validity of the QUALMS and provides novel information on the psychometric performance of its three subscales (QUALMS‐P; QUALMS‐E; QUALMS‐BF).
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.
(1) Background: The Austrian supply of COVID-19 vaccine is limited for now. We aim to provide evidence-based guidance to the authorities in order to minimize COVID-19-related hospitalizations and ...deaths in Austria. (2) Methods: We used a dynamic agent-based population model to compare different vaccination strategies targeted to the elderly (65 ≥ years), middle aged (45-64 years), younger (15-44 years), vulnerable (risk of severe disease due to comorbidities), and healthcare workers (HCW). First, outcomes were optimized for an initially available vaccine batch for 200,000 individuals. Second, stepwise optimization was performed deriving a prioritization sequence for 2.45 million individuals, maximizing the reduction in total hospitalizations and deaths compared to no vaccination. We considered sterilizing and non-sterilizing immunity, assuming a 70% effectiveness. (3) Results: Maximum reduction of hospitalizations and deaths was achieved by starting vaccination with the elderly and vulnerable followed by middle-aged, HCW, and younger individuals. Optimizations for vaccinating 2.45 million individuals yielded the same prioritization and avoided approximately one third of deaths and hospitalizations. Starting vaccination with HCW leads to slightly smaller reductions but maximizes occupational safety. (4) Conclusion: To minimize COVID-19-related hospitalizations and deaths, our study shows that elderly and vulnerable persons should be prioritized for vaccination until further vaccines are available.
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.
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