Dataset Information

Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.

ABSTRACT: Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy and promote adequate resource allocation. The net monetary benefit aggregates information on differences in mean cost and clinical outcomes; the cost-effectiveness acceptability curve was developed to characterize the extent to which the strength of evidence regarding net monetary benefit changes with fluctuations in the willingness-to-pay threshold. Methods to derive insights from characteristics of the cost/clinical outcomes besides mean differences remain undeveloped but may also be informative. We propose a novel probabilistic measure of cost-effectiveness based on the stochastic ordering of the individual net benefit distribution under each treatment. Our approach is able to accommodate features frequently encountered in observational data including confounding and censoring, and complements the net monetary benefit in the insights it provides. We conduct a range of simulations to evaluate finite-sample performance and illustrate our proposed approach using simulated data based on a study of endometrial cancer patients.

SUBMITTER: Spieker AJ

PROVIDER: S-EPMC8211369 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.

Spieker Andrew J AJ Illenberger Nicholas N Roy Jason A JA Mitra Nandita N

Statistical methods in medical research 20210407 5

Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy and promote adequate resource allocation. The net monetary benefit aggregates information on differences in mean cost and clinical outcomes; the cost-effectiveness acceptability curve was developed to characterize the extent to which the strength of evidence re ...[more]

PMID: 33826460

Similar Datasets

Project description:BackgroundCoverage of maternal and newborn health (MNH) interventions is often influenced by important determinants and decision makers are often concerned with equity issues. The net-benefit framework developed and applied alongside clinical trials and in pharmacoeconomics offers the potential for exploring how cost-effectiveness of MNH interventions varies at the margin by important covariates as well as for handling uncertainties around the ICER estimate.AimWe applied the net-benefit framework to analyze cost-effectiveness of the Skilled Care Initiative and assessed relative advantages over a standard computation of incremental cost effectiveness ratios.MethodsHousehold and facility surveys were carried out from January to July 2006 in Ouargaye district (where the Skilled Care Initiative was implemented) and Diapaga (comparison site) district in Burkina Faso. Pregnancy-related and perinatal mortality were retrospectively assessed and data were collected on place of delivery, education, asset ownership, place, and distance to health facilities, costs borne by households for institutional delivery, and cost of standard provision of maternal care. Descriptive and regression analyses were performed.ResultsThere was a 30% increase in institutional births in the intervention district compared to 10% increase in comparison district, and a significant reduction of perinatal mortality rates (OR 0.75, CI 0.70-0.80) in intervention district. The incremental cost for achieving one additional institutional delivery in Ouargaye district compared to Diapaga district was estimated to be 170 international dollars and varied significantly by covariates. However, the joint probability distribution (net-benefit framework) of the effectiveness measure (institutional delivery), the cost data and covariates indicated distance to health facilities as the single most important determinant of the cost-effectiveness analysis with implications for policy making.ConclusionThe net-benefit framework, the application of which requires household-level effects and cost data, has proven more insightful (than traditional ICER) in presenting and interpreting cost-effectiveness results of the Skilled Care Initiative.

Project description:BackgroundRisk prediction models have been proposed for various diseases and are being improved as new predictors are identified. A major challenge is to determine whether the newly discovered predictors improve risk prediction. Decision curve analysis has been proposed as an alternative to the area under the curve and net reclassification index to evaluate the performance of prediction models in clinical scenarios. The decision curve computed using the net benefit can evaluate the predictive performance of risk models at a given or range of threshold probabilities. However, when the decision curves for 2 competing models cross in the range of interest, it is difficult to identify the best model as there is no readily available summary measure for evaluating the predictive performance. The key deterrent for using simple measures such as the area under the net benefit curve is the assumption that the threshold probabilities are uniformly distributed among patients.MethodsWe propose a novel measure for performing decision curve analysis. The approach estimates the distribution of threshold probabilities without the need of additional data. Using the estimated distribution of threshold probabilities, the weighted area under the net benefit curve serves as the summary measure to compare risk prediction models in a range of interest.ResultsWe compared 3 different approaches, the standard method, the area under the net benefit curve, and the weighted area under the net benefit curve. Type 1 error and power comparisons demonstrate that the weighted area under the net benefit curve has higher power compared to the other methods. Several simulation studies are presented to demonstrate the improvement in model comparison using the weighted area under the net benefit curve compared to the standard method.ConclusionsThe proposed measure improves decision curve analysis by using the weighted area under the curve and thereby improves the power of the decision curve analysis to compare risk prediction models in a clinical scenario.

Project description:BackgroundAccess to evidence-based psychological treatment is a concern in many parts of the globe due to government-level financial constraints and patient-level barriers. Transdiagnostic cognitive behavioural therapy (tCBT) is an effective treatment approach that uses a single protocol for anxiety disorders which could enhance the dissemination of evidence-based psychotherapy. In a context of limited resources, the study of treatment moderators can allow to identify subgroups for which the cost-effectiveness of an intervention differs, information that could impact decision-making. So far, there has been no economic evaluation of tCBT for different subpopulations. The objectives of this study, using the net-benefit regression framework, were to explore clinical and sociodemographic factors as potential moderators of the cost-effectiveness of tCBT compared to treatment-as-usual (TAU).MethodsThis is a secondary data analysis of a pragmatic randomized controlled trial opposing tCBT added to TAU (n = 117) to TAU only (n = 114). Data on costs from the health system and the limited societal perspectives, as well as anxiety-free days, an effectiveness measure based on the Beck Anxiety Inventory, were collected over an 8-month time horizon and used to derive individual net-benefits. The net-benefit regression framework was used to assess moderators of the cost-effectiveness of tCBT + TAU as opposed to TAU alone. Variables of sociodemographic and clinical nature were assessed.ResultsResults showed that the number of comorbid anxiety disorders significantly moderated the cost-effectiveness of tCBT + TAU compared to TAU from the limited societal perspective.ConclusionsThe number of comorbid anxiety disorders was identified as a moderator affecting the cost-effectiveness of tCBT + TAU compared to TAU from the limited societal perspective. More research is needed to strengthen the case of tCBT from an economic standpoint for large-scale dissemination.Trial registrationClinicalTrials.gov: NCT02811458, 23/06/2016.

Project description:BackgroundUniversal home fortification of complementary foods with iron-containing multiple micronutrient powders (MNPs) is a key intervention to prevent anaemia in young children in low-income and middle-income countries. However, evidence that MNPs might promote infection raises uncertainty about whether MNPs give net health benefits and are cost-effective. We aimed to determined country-specific net benefit or harm and cost-effectiveness of universal provision of MNPs to children aged 6 months.MethodsWe developed a microsimulation model to estimate net country-specific disability-adjusted life-years (DALYs), years lived with disability (YLDs), and years of life lost (YLLs) due to anaemia, malaria, and diarrhoea averted (or increased) by provision of a 6-month course of MNPs to children aged 6 months, compared with no intervention, who would be followed up for an additional 6 months (ie, to age 18 months). Anaemia prevalence was derived from Demographic and Health Surveys or similar national surveys, and malaria and diarrhoea incidence were sourced from the Global Burden of Disease Study. Programme and health-care costs were modelled to determine cost per DALY averted (US$). Additionally, we explored the effects of reduced MNP coverage in a sensitivity analysis.Findings78 countries (46 countries in Africa, 20 in Asia or the Middle East, and 12 in Latin America) were included in the analysis, and we simulated 5 million children per country. 6 months of universal distribution of daily MNPs, assuming 100% coverage, produced a net benefit (DALYs averted) in 54 countries (24 in Africa, 19 in Asia and the Middle East, 11 in Latin America) and net harm in 24 countries (22 in Africa, one in Asia, and one in Latin America). MNP intervention provided a benefit on YLDs associated with anaemia, but these gains were attenuated and sometimes reversed by increases in YLLs associated with malaria and diarrhoea, reducing the benefits seen for DALYs. In the 54 countries where MNP provision was beneficial, the median benefit was 28·1 DALYs averted per 10 000 children receiving MNPs (IQR 20·6-40·4), and median cost per DALY averted was $3576 (IQR 2474-4918). DALY effects positively correlated with moderate and severe anaemia prevalence in Asia, the Middle East, and Latin America, but correlated inversely in Africa. Suboptimal coverage markedly reduced DALYs averted and cost-effectiveness.InterpretationNet health benefits of MNPs vary between countries, are highest where prevalence of moderate and severe anaemia is greatest but infection prevalence is smallest, and are ameliorated when coverage of the intervention is poor. Our data provide country-specific guidance to national policy makers.FundingInternational Union of Nutrition Sciences.

Dataset Information

Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.

Publications

Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets