Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.
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ABSTRACT:
SUBMITTER: Spieker AJ
PROVIDER: S-EPMC8211369 | biostudies-literature |
REPOSITORIES: biostudies-literature
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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.
| S-EPMC3400570 | biostudies-literature
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.
| S-EPMC4949771 | biostudies-literature
Project description:This systematic review aimed to comprehensively synthesize cost-effectiveness evidences of bariatric surgery by pooling incremental net monetary benefits (INB). Twenty-eight full economic evaluation studies comparing bariatric surgery with usual care were identified from five databases. In high-income countries (HICs), bariatric surgery was cost-effective among mixed obesity group (i.e., obesity with/without diabetes) over a 10-year time horizon (pooled INB = $53,063.69; 95% CI $42,647.96, $63,479.43) and lifetime horizon (pooled INB = $101,897.96; 95% CI $79,390.93, $124,404.99). All studies conducted among obese with diabetes reported that bariatric surgery was cost-effective. Also, the pooled INB for obesity with diabetes group over lifetime horizon in HICs was $80,826.28 (95% CI $32,500.75, $129,151.81). Nevertheless, no evidence is available in low- and middle-income countries.
| S-EPMC8175295 | biostudies-literature
Project description:BackgroundThis study evaluates the cost-effectiveness of the DAZT program for scaling up treatment of acute child diarrhea in Gujarat India using a net-benefit regression framework.MethodsCosts were calculated from societal and caregivers' perspectives and effectiveness was assessed in terms of coverage of zinc and both zinc and Oral Rehydration Salt. Regression models were tested in simple linear regression, with a specified set of covariates, and with a specified set of covariates and interaction terms using linear regression with endogenous treatment effects was used as the reference case.ResultsThe DAZT program was cost-effective with over 95% certainty above $5.50 and $7.50 per appropriately treated child in the unadjusted and adjusted models respectively, with specifications including interaction terms being cost-effective with 85-97% certainty.DiscussionFindings from this study should be combined with other evidence when considering decisions to scale up programs such as the DAZT program to promote the use of ORS and zinc to treat child diarrhea.
| S-EPMC5465559 | biostudies-literature
Project description:BackgroundNeonatal hypoglycaemia is a common but treatable metabolic disorder that affects newborn infants and which, if not identified and treated adequately, may result in neurological sequelae that persist for the lifetime of the patient. The long-term financial and quality-of-life burden of neonatal hypoglycaemia has not been previously examined.MethodsWe assessed the postnatal hospital and long-term costs associated with neonatal hypoglycaemia over 80 year and 18 year time horizons, using a health-system perspective and assessing impact on quality of life using quality-adjusted life year (QALYs). A decision analytic model was used to represent key outcomes in the presence and absence of neonatal hypoglycaemia.ResultsThe chance of developing one of the outcomes of neonatal hypoglycaemia in our model (cerebral palsy, learning disabilities, seizures, vision disorders) was 24.03% in subjects who experienced neonatal hypoglycaemia and 3.56% in those who do did not. Over an 80 year time horizon a subject who experienced neonatal hypoglycaemia had a combined hospital and post-discharge cost of NZ$72,000 due to the outcomes modelled, which is NZ$66,000 greater than a subject without neonatal hypoglycaemia. The net monetary benefit lost due to neonatal hypoglycaemia, using a value per QALY of NZ$43,000, is NZ$180,000 over an 80 year time horizon.ConclusionsEven under the most conservative of estimates, neonatal hypoglycaemia contributes a significant financial burden to the health system both during childhood and over a lifetime. The combination of direct costs and loss of quality of life due to neonatal hypoglycaemia means that this condition warrants further research to focus on prevention and effective treatment.
| S-EPMC7863541 | biostudies-literature
Project description:Background:Whether routine urinary analysis has a net benefit for urinary tract infection (UTI) screening is unclear. Methods:Using the laboratory information system (LIS), we retrospectively extracted the data of urine culture and routine analysis between January 2017 and April 2017. Receiver operating characteristic (ROC) curve, logistic regression model, net reclassification improvement (NRI) and integrated discrimination improvement (IDI) were used to estimate the screening performance of routine urinary analysis. Decision curve analysis (DCA) was used to estimate the net benefit of routine urinary analysis. Results:A total of 927 specimens with 156 UTIs were included in the present study. The area under ROC curves (AUCs) of white blood cells (WBCs) and bacteria were 0.729 and 0.836, respectively. The logistic regression model incorporating WBCs, bacteria and nitrite together had an AUC of 0.851, which is significantly higher than that of WBCs. NRI and IDI analyses also indicated that WBCs, bacteria and nitrite, when used together, had better a screening performance than each single test alone. DCA revealed that 0.08 net benefit can be obtained for bacteria and the model, while the net benefit of WBCs is limited. Conclusions:WBCs, bacteria and nitrite, when used together, can significantly improve the efficiency for UTI screening. Bacteria and the model incorporating WBCs, bacteria and nitrite have a net benefit in UTI screening, while the net benefit of WBCs, when used alone, is limited.
| S-EPMC7290540 | biostudies-literature
Project description:BackgroundClear 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.MethodsA 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.ResultsThe 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.ConclusionsOrganized 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.
| S-EPMC6896501 | biostudies-literature
Project description:BACKGROUND:Invasive (IPD, defined as detection of pneumococci in sterile body fluids like meningitis or bacteremic pneumonia) and non-invasive Streptococcus pneumoniae infections (i.e. non-bacteremic pneumonia, otitis media) in adults are associated with substantial morbidity, mortality and costs. In Germany, Pneumococcal polysaccharide vaccination (PPV23) is recommended for all persons >60?years and for defined risk groups (age 5-59). The aim of this model was to estimate the potential cost-effectiveness and benefit-cost ratios of the adult vaccination program (18?years and older), considering the launch of the pneumococcal conjugate vaccine for adults (PCV13). METHODS:A cross-sectional steady state Markov model was developed to estimate the outcomes of PCV13, PPV23 vaccination schemes and 'no vaccination'. Conservative assumptions were made if no data were available for PCV13 and PPV23 respectively. The effectiveness of individual pneumococcal vaccination in adults was adjusted for expected indirect effects due to the vaccination in infants. Data on incidences, effectiveness and costs were derived from scientific literature and publicly available databases. All resources used are indicated. Benefit-cost ratios and cost-effectiveness were evaluated from the perspective of the German Statutory Health Insurance as well as from social perspective. RESULTS:Under the assumption that PCV13 has a comparable effectiveness to PCV7, a vaccination program with PCV13 revealed the potential to avoid a greater number of yearly cases and deaths in IPD and pneumonia in Germany compared to PPV23. For PCV13, the costs were shown to be overcompensated by monetary savings resulting from reduction in the use of health care services. These results would render the switch from PPV23 to PCV13 as a dominant strategy compared to PPV23 and 'no vaccination'. Given the correctness of the underlying assumptions every Euro spent on the PCV13 vaccination scheme yields savings of 2.09 € (social perspective: 2.16 €) compared to PPV23 and 1.27 € (social perspective: 1.32 €) compared to 'no vaccination', respectively. CONCLUSIONS:Results of the model indicate that the health economic benefit of immunizing adults with PCV13 can be expected to outperform the sole use of PPV23, if the effectiveness of PCV13 is comparable to the effectiveness of PCV7.
| S-EPMC3463422 | biostudies-literature
Project description:BackgroundTypically, a two-phase (double) sampling strategy is employed when classifications are subject to error and there is a gold standard (perfect) classifier available. Two-phase sampling involves classifying the entire sample with an imperfect classifier, and a subset of the sample with the gold-standard.Methodology/principal findingsIn this paper we consider an alternative strategy termed reclassification sampling, which involves classifying individuals using the imperfect classifier more than one time. Estimates of sensitivity, specificity and prevalence are provided for reclassification sampling, when either one or two binary classifications of each individual using the imperfect classifier are available. Robustness of estimates and design decisions to model assumptions are considered. Software is provided to compute estimates and provide advice on the optimal sampling strategy.Conclusions/significanceReclassification sampling is shown to be cost-effective (lower standard error of estimates for the same cost) for estimating prevalence as compared to two-phase sampling in many practical situations.
| S-EPMC3278465 | biostudies-literature
Project description:Background Treatment with ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine) is a well-established therapy for advanced Hodgkin’s lymphoma (HL). However, the recently completed ECHELON-1 trial showed potential net clinical benefit for brentuximab vedotin (BREN+AVD) compared to ABVD as frontline therapy in patients with advanced Hodgkin’s lymphoma. The objective of this analysis is to determine whether, on current evidence, BREN+AVD is cost-effective relative to ABVD as frontline therapy in patients with advanced HL. Methods We constructed a probabilistic Markov model with two arms and six mutually exclusive health states, using six-month?cycle lengths, and a 15-year time horizon. Time-dependent transition probabilities were calculated from ‘real-world’ data collected by the BC Cancer’s Centre for Lymphoid Cancer database or from the literature for ABVD. Time-dependent transition probabilities for BREN+AVD were taken from the ECHELON-1 trial. We estimated the incremental cost and effects per patient of each therapy and calculated the incremental cost-effectiveness ratio (ICER). Costs were measured in 2018 Canadian dollars and effects measured in quality-adjusted life years (QALYs). A probabilistic analysis was used to generate a cost-effectiveness acceptability curve (CEAC). Results The incremental cost between standard therapy with ABVD and therapy with BREN+AVD was estimated to be $192,336. The regimen of BREN+AVD resulted in a small benefit in terms of QALYs (0.46 QALYs). The estimated ICER was $418,122 per QALY gained. The probabilistic analysis suggests very few (8%) simulations fall below $100,000 per QALY. Even at a threshold of $200,000 per QALY gained, there was only a 24% chance that BREN+AVD would be considered cost-effective. Sensitivity analyses evaluating price reductions for brentuximab showed that these reductions needed to be in excess of 70% for this regimen to be cost-effective at a threshold of $100,000 per QALY. Conclusions There may be a clinical benefit associated with BREN+AVD, but on current evidence the benefit is not adequately substantive compared to ABVD therapy given the cost of brentuximab vedotin. Agencies responsible for making decisions about BREN+AVD as frontline therapy for patients with advanced HL should consider whether they are willing to implement this treatment given the current uncertainty and cost-benefit profile, or negotiate substantial price-reductions from the manufacturer should they choose to reimburse.
| S-EPMC7557030 | biostudies-literature