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1.

Objectives

To provide a practical approach for calculating uncertainty intervals and variance components associated with initial-condition and dynamic-equation parameters in computationally expensive population-based disease microsimulation models.

Methods

In the proposed uncertainty analysis approach, we calculated the required computational time and the number of runs given a user-defined error bound on the variance of the grand mean. The equations for optimal sample sizes were derived by minimizing the variance of the grand mean using initial estimates for variance components. Finally, analysis of variance estimators were used to calculate unbiased variance estimates.

Results

To illustrate the proposed approach, we performed uncertainty analysis to estimate the uncertainty associated with total direct cost of osteoarthritis in Canada from 2010 to 2031 according to a previously published population health microsimulation model of osteoarthritis. We first calculated crude estimates for initial-population sampling and dynamic-equation parameters uncertainty by performing a small number of runs. We then calculated the optimal sample sizes and finally derived 95% uncertainty intervals of the total cost and unbiased estimates for variance components. According to our results, the contribution of dynamic-equation parameter uncertainty to the overall variance was higher than that of initial parameter sampling uncertainty throughout the study period.

Conclusions

The proposed analysis of variance approach provides the uncertainty intervals for the mean outcome in addition to unbiased estimates for each source of uncertainty. The contributions of each source of uncertainty can then be compared with each other for validation purposes so as to improve the model accuracy.  相似文献   

2.

Objectives

To examine associations between four health behaviors (smoking, physical inactivity, heavy alcohol drinking, and obesity) and three health indices (health-related quality of life, life expectancy, and quality-adjusted life expectancy (QALE)) among US adults with depression.

Methods

Data were obtained from the 2006, 2008, and 2010 Behavioral Risk Factor Surveillance System data. The EuroQol five-dimensional questionnaire (EQ-5D) health preference scores were estimated on the basis of extrapolations from the Centers for Disease Control and Prevention’s healthy days measures. Depression scores were estimated using the eight-item Patient Health Questionnaire. Life expectancy estimates were obtained from US life tables, and QALE was estimated from a weighted combination of the EQ-5D scores and the life expectancy estimates. Outcomes were summarized by depression status for the four health behaviors (smoking, physical inactivity, heavy alcohol drinking, and obesity).

Results

For depressed adults, current smokers and the physically inactive had significantly lower EQ-5D scores (0.040 and 0.171, respectively), shorter life expectancy (12.9 and 10.8 years, respectively), and substantially less QALE (8.6 and 10.9 years, respectively). For nondepressed adults, estimated effects were similar but smaller. Heavy alcohol drinking among depressed adults, paradoxically, was associated with higher EQ-5D scores but shorter life expectancy. Obesity was strongly associated with lower EQ-5D scores but only weakly associated with shorter life expectancy.

Conclusions

Among depressed adults, physical inactivity and smoking were strongly associated with lower EQ-5D scores, life expectancy, and QALE, whereas obesity and heavy drinking were only weakly associated with these indices. These results suggest that reducing physical inactivity and smoking would improve health more among depressed adults.  相似文献   

3.

Background

Several utility-based instruments have been applied in cost-utility analysis to assess health state values for people with dementia. Nevertheless, concerns and uncertainty regarding their performance for people with dementia have been raised.

Objectives

To assess the performance of available utility-based instruments for people with dementia by comparing their psychometric properties and to explore factors that cause variations in the reported health state values generated from those instruments by conducting meta-regression analyses.

Methods

A literature search was conducted and psychometric properties were synthesized to demonstrate the overall performance of each instrument. When available, health state values and variables such as the type of instrument and cognitive impairment levels were extracted from each article. A meta-regression analysis was undertaken and available covariates were included in the models.

Results

A total of 64 studies providing preference-based values were identified and included. The EuroQol five-dimension questionnaire demonstrated the best combination of feasibility, reliability, and validity. Meta-regression analyses suggested that significant differences exist between instruments, type of respondents, and mode of administration and the variations in estimated utility values had influences on incremental quality-adjusted life-year calculation.

Conclusions

This review finds that the EuroQol five-dimension questionnaire is the most valid utility-based instrument for people with dementia, but should be replaced by others under certain circumstances. Although no utility estimates were reported in the article, the meta-regression analyses that examined variations in utility estimates produced by different instruments impact on cost-utility analysis, potentially altering the decision-making process in some circumstances.  相似文献   

4.

Background

Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors were approved by the US Food and Drug Administration (FDA) as cholesterol-lowering therapies for patients with familial hypercholesterolemia or atherosclerotic cardiovascular disease.

Objectives

To estimate the long-term health and economic value of PCSK9 inhibitors for Americans (51 years and older).

Methods

We conducted simulations using the Future Elderly Model, an established dynamic microsimulation model to project the lifetime outcomes for the US population aged 51 years and older. Health effects estimates and confidence intervals from published meta-analysis studies were used to project changes in life expectancy, quality-adjusted life-years, and lifetime medical spending resulting from the use of PCSK9 inhibitors. We considered two treatment scenarios: 1) current FDA eligibility and 2) an extended eligibility scenario that includes patients with no pre-existing cardiovascular disease but at high risk. We assumed that the price of PCSK9 inhibitors was discounted by 35% in the first 12 years and by 57% thereafter, with gradual uptake of the drug in eligible populations.

Results

Use of PCSK9 inhibitors by individuals covered by current FDA approval would extend life expectancy at the age of 51 years by an estimated 1.1 years and would yield a lifetime net value of $5800 per person. If use was extended to those at high risk for cardiovascular disease, PCSK9 inhibitors would generate a lifetime net benefit of $14,100 per person.

Conclusions

Expanded access to PCSK9 inhibitors would offer positive long-term net value for patients and the US health care system at the current discounted prices.  相似文献   

5.

Background

Cost-utility analysis prioritizes services using cost, life-years, and the health state utility of the life-years. Nevertheless, a significant body of evidence suggests that the public would prefer more variables to be considered in decision making and at least some sharing of the budget with services for severe conditions that are not cost-effective because of their high cost.

Objectives

To examine whether this preference for sharing persists for less severe conditions when both cost effectiveness and illness severity would indicate that resources should be allocated to other services.

Methods

Survey respondents were asked to divide a budget between two patients facing life-threatening illnesses. The severity of the illnesses differed and the price of treatment was varied.

Results

Sharing occurred in all scenarios including scenarios in which the illness was less severe and services were not cost-effective. Results are consistent with behavior commonly observed in other contexts.

Conclusions

Results suggest that sharing per se is important and that the public would support some funding of cost-ineffective services for less severe health problems.  相似文献   

6.

Objectives

Estimates of future health technology diffusion, or future uptake over time, are a requirement for different analyses performed within health technology assessments. Methods for obtaining such estimates include constant uptake estimates based on expert opinion or analogous technologies and on extrapolation from initial data points using parametric curves—but remain divorced from established diffusion theory and modeling. We propose an approach to obtaining diffusion estimates using experts’ beliefs calibrated to an established diffusion model to address this methodologic gap.

Methods

We performed an elicitation of experts’ beliefs on future diffusion of a new preterm birth screening illustrative case study technology. The elicited quantities were chosen such that they could be calibrated to yield the parameters of the Bass model of new product growth, which was chosen based on a review of the diffusion literature.

Results

With the elicitation of only three quantities per diffusion curve, our approach enabled us to quantify uncertainty about diffusion of the new technology in different scenarios. Pooled results showed that the attainable number of adoptions was predicted to be relatively low compared with what was thought possible. Further research evidence improved the attainable number of adoptions only slightly but resulted in greater speed of diffusion.

Conclusions

The proposed approach of eliciting experts’ beliefs about diffusion and informing the Bass model has the potential to fill the methodologic gap evident in value of implementation and research, as well as budget impact and some cost-effectiveness analyses.  相似文献   

7.

Background

The validation of health economic (HE) model outcomes against empirical data is of key importance. Although statistical testing seems applicable, guidelines for the validation of HE models lack guidance on statistical validation, and actual validation efforts often present subjective judgment of graphs and point estimates.

Objectives

To discuss the applicability of existing validation techniques and to present a new method for quantifying the degrees of validity statistically, which is useful for decision makers.

Methods

A new Bayesian method is proposed to determine how well HE model outcomes compare with empirical data. Validity is based on a pre-established accuracy interval in which the model outcomes should fall. The method uses the outcomes of a probabilistic sensitivity analysis and results in a posterior distribution around the probability that HE model outcomes can be regarded as valid.

Results

We use a published diabetes model (Modelling Integrated Care for Diabetes based on Observational data) to validate the outcome “number of patients who are on dialysis or with end-stage renal disease.” Results indicate that a high probability of a valid outcome is associated with relatively wide accuracy intervals. In particular, 25% deviation from the observed outcome implied approximately 60% expected validity.

Conclusions

Current practice in HE model validation can be improved by using an alternative method based on assessing whether the model outcomes fit to empirical data at a predefined level of accuracy. This method has the advantage of assessing both model bias and parameter uncertainty and resulting in a quantitative measure of the degree of validity that penalizes models predicting the mean of an outcome correctly but with overly wide credible intervals.  相似文献   

8.

Background

The economic evaluation of colorectal cancer screening is challenging because of the need to model the underlying unobservable natural history of the disease.

Objectives

To describe the available Markov models and to critically analyze their main structural assumptions.

Methods

A systematic search was performed in eight relevant databases (MEDLINE, Embase, Econlit, National Health Service Economic Evaluation Database, Health Economic Evaluations Database, Health Technology Assessment database, Cost-Effective Analysis Registry, and European Network of Health Economics Evaluation Databases), identifying 34 models that met the inclusion criteria. A comparative analysis of model structure and parameterization was conducted using two checklists and guidelines for cost-effectiveness screening models.

Results

Two modeling techniques were identified. One strategy used a Markov model to reproduce the natural history of the disease and an overlaying model that reproduced the screening process, whereas the other used a single model to represent a screening program. Most of the studies included only adenoma-carcinoma sequences, a few included de novo cancer, and none included the serrated pathway. Parameterization of adenoma dwell time, sojourn time, and surveillance differed between studies, and there was a lack of validation and statistical calibration against local epidemiological data. Most of the studies analyzed failed to perform an adequate literature review and synthesis of diagnostic accuracy properties of the screening tests modeled.

Conclusions

Several strategies to model colorectal cancer screening have been developed, but many challenges remain to adequately represent the natural history of the disease and the screening process. Structural uncertainty analysis could be a useful strategy for understanding the impact of the assumptions of different models on cost-effectiveness results.  相似文献   

9.

Background

Population-wide screening for melanoma is unlikely to be cost-effective. Nevertheless, targeted surveillance of high-risk individuals may be.

Objectives

To estimate the cost-effectiveness of various surveillance strategies in the UK population, stratified by risk using a simple self-assessment tool scoring between 0 and 67.

Methods

A decision model comparing alternative surveillance policies from the perspective of the UK National Health Service over 30 years was developed. The strategy with the highest expected net benefit for each risk score was identified, resulting in a compound risk-stratified policy describing the most cost-effective population-wide strategy. The overall expected cost and quality-adjusted life-years (QALYs), the incremental cost-effectiveness ratio, and associated uncertainty were reported.

Results

The most cost-effective strategy is for those with a Williams score of 15 to 21 (relative risk [RR] of 0.79–1.60 vs. a mean score of 17 in the United Kingdom) to be offered a one-off full-body skin examination, and for those with a score of 22 or more (RR 1.79+) to be enrolled into a quinquennial monitoring program, rising to annual recall for those with a risk score greater than 43 (RR 20.95+). Expected incremental cost would be £164 million per annum (~0.1% of the National Health Service budget), gaining 15,947 additional QALYs and yielding an incremental cost-effectiveness ratio of £10,199/QALY gained (51.3% probability <£30,000).

Conclusions

The risk-stratified policy would be expensive to implement but cost-effective compared with typical UK thresholds (£20,000–£30,000/QALY gained), although decision uncertainty is high. Phased implementation enrolling only higher risk individuals would be substantially less expensive, but with consequent foregone health gain.  相似文献   

10.

Objective

To assess the logic and consistency of three prominent value frameworks.

Methods

We reviewed the value frameworks from three organizations: the Memorial Sloan Kettering Cancer Center (DrugAbacus), the American Society of Clinical Oncologists, and the Institute for Clinical and Economic Review. For each framework, we developed case studies to explore the degree to which the frameworks have face validity in the sense that they are consistent with four important principles: value should be proportional to a therapy’s benefit; components of value should matter to framework users (patients and payers); attribute weights should reflect user preferences; and value estimates used to inform therapy prices should reflect per-person benefit.

Results

All three frameworks can aid decision making by elucidating factors not explicitly addressed by conventional evaluation techniques (in particular, cost-effectiveness analyses). Our case studies identified four challenges: 1) value is not always proportional to benefit; 2) value reflects factors that may not be relevant to framework users (patients or payers); 3) attribute weights do not necessarily reflect user preferences or relate to value in ways that are transparent; and 4) value does not reflect per-person benefit.

Conclusions

Although the value frameworks we reviewed capture value in a way that is important to various audiences, they are not always logical or consistent. Because these frameworks may have a growing influence on therapy access, it is imperative that analytic challenges be further explored.  相似文献   

11.

Background

Several public cord blood banks are struggling financially, and the question remains as to whether additional allocations of funds to them are justified.

Objectives

To estimate the social benefits of public cord blood bank inventory net of cord blood banks’ operational costs.

Methods

We used publicly available data from the Health Resources and Service Administration on the number of annual cord blood transplants as well as the patient age distribution in 2010, and the survival estimates between 2008 and 2012 for the several diseases treated by cord blood transplantation. Data on aggregate annual costs to the cord blood industry for recruitment, processing, and storage were obtained from published work. We used estimated increases in life expectancy due to treatment using umbilical cord blood and value for life-years gained to estimate the social benefits of the public cord blood inventory annually.

Results

We found that the annual social benefits of between $500 million and $1.5 billion outweigh the current operational annual costs of running cord blood banks of $60 to $70 million by a significant margin.

Conclusions

We estimated that the annual social benefit of having a cord blood system far outweighs its costs, by more than an order of magnitude. Thus, the social benefits of maintaining the US public cord blood banking system at the present time far outweigh the costs of collecting, storing, and distributing cord blood. This suggests that there is a potential justification for government intervention to align social benefits and costs. Nevertheless, simple fixes may produce unintended consequences, and so a careful design for subsidies is needed.  相似文献   

12.

Objectives

To test the hypothesis that the “severity effect”—the preference for more than utility-maximizing expenditure on severe health states—may be the result of, or exacerbated by, the uncertainty associated with the chance of contracting the illness that causes the severe health state.

Methods

Survey respondents were asked to imagine that they will contract one of two illnesses and asked to allocate a budget between two insurance policies, each of which provides services for the treatment of one of the illnesses. A person’s final health state varied with the amount of insurance purchased for the illness that occurred and therefore with the level of treatment. The relative cost of the two policies was altered and the selected levels of insurance compared with the levels that would be provided by a health authority that sought to maximize total utility or quality-adjusted life-years from its own budget.

Results

Respondents selected more than utility-maximizing insurance for protection against severe health states. A number of psychological factors that affect measurement under uncertainty do not affect utility as currently measured. This difference may explain the present results and also explain the “severity paradox” that personal preferences as presently measured imply less expenditure on severe health states than do “social preferences” for the treatment of strangers.

Conclusions

Uncertainty alters preferences. Incorporating these preferences in decision making would result in greater spending on severe health states.  相似文献   

13.

Background

Brief interventions (BIs) delivered in primary care have shown potential to increase physical activity levels and may be cost-effective, at least in the short-term, when compared with usual care. Nevertheless, there is limited evidence on their longer term costs and health benefits.

Objectives

To estimate the cost-effectiveness of BIs to promote physical activity in primary care and to guide future research priorities using value of information analysis.

Methods

A decision model was used to compare the cost-effectiveness of three classes of BIs that have been used, or could be used, to promote physical activity in primary care: 1) pedometer interventions, 2) advice/counseling on physical activity, and (3) action planning interventions. Published risk equations and data from the available literature or routine data sources were used to inform model parameters. Uncertainty was investigated with probabilistic sensitivity analysis, and value of information analysis was conducted to estimate the value of undertaking further research.

Results

In the base-case, pedometer interventions yielded the highest expected net benefit at a willingness to pay of £20,000 per quality-adjusted life-year. There was, however, a great deal of decision uncertainty: the expected value of perfect information surrounding the decision problem for the National Health Service Health Check population was estimated at £1.85 billion.

Conclusions

Our analysis suggests that the use of pedometer BIs is the most cost-effective strategy to promote physical activity in primary care, and that there is potential value in further research into the cost-effectiveness of brief (i.e., <30 minutes) and very brief (i.e., <5 minutes) pedometer interventions in this setting.  相似文献   

14.

Background

The German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen) adapted the efficiency frontier (EF) approach to conform to statutory provisions on cost-effectiveness analysis of health technologies. EF serves as a framework for evaluating cost-effectiveness and indirectly for pricing and reimbursement decisions.

Objectives

To calculate an EF on the basis of single multidimensional benefit by taking patient preferences and uncertainty into account; to evaluate whether EF is useful to inform decision makers about cost-effectiveness of new therapies; and to find whether a treatment is efficient at given prices demonstrated through a case study on chronic hepatitis C.

Methods

A single multidimensional benefit was calculated by linear additive aggregation of multiple patient-relevant end points. End points were identified and weighted by patients in a previous discrete-choice experiment (DCE). Aggregation of overall benefit was ascertained using preferences and clinical data. Monte-Carlo simulation was applied. Uncertainty was addressed by price acceptability curve (PAC) and net monetary benefit (NMB).

Results

The case study illustrates that progress in benefit and efficiency of hepatitis C virus treatments could be depicted very well with the EF. On the basis of cost, effect, and preference data, the latest generations of interferon-free treatments are shown to yield a positive NMB and be efficient at current prices.

Conclusions

EF was implemented taking uncertainty into account. For the first time, a DCE was used with the EF. The study shows how DCEs in combination with EF, PAC, and NMB can contribute important information in the course of reimbursement and pricing decisions.  相似文献   

15.

Background

When proven effective, decision making regarding reimbursement of new health technology typically involves ethical, social, legal, and health economic aspects and constraints. Nevertheless, when applying standard value of information (VOI) analysis, the value of collecting additional evidence is typically estimated assuming that only cost-effectiveness outcomes guide such decisions.

Objectives

To illustrate how decision makers’ constraints can be incorporated into VOI analyses and how these may influence VOI outcomes.

Methods

A simulation study was performed to estimate the cost-effectiveness of a new hypothetical technology compared with usual care. Constraints were defined for the new technology on 1) the maximum acceptable rate of complications and 2) the maximum acceptable additional budget. The expected value of perfect information (EVPI) for the new technology was estimated in various scenarios, both with and without incorporating these constraints.

Results

For a willingness-to-pay threshold of €20,000 per quality-adjusted life-year, the probability that the new technology was cost-effective equaled 57%, with an EVPI of €1868 per patient. Applying the complication rate constraint reduced the EVPI to €1137. Similarly, the EVPI reduced to €770 when applying the budget constraint. Applying both constraints simultaneously further reduced the EVPI to €318.

Conclusions

When decision makers explicitly apply additional constraints, beyond a willingness-to-pay threshold, to reimbursement decisions, these constraints can and should be incorporated into VOI analysis as well, because they may influence VOI outcomes. This requires continuous interaction between VOI analysts and decision makers and is expected to improve both the relevance and the acceptance of VOI outcomes.  相似文献   

16.

Background

The EuroQol five-dimensional questionnaire (EQ-5D) is a generic preference-based measure of health-related quality of life, and several studies have made attempts to estimate the minimally important difference (MID) for the EQ-5D index score.

Objectives

To estimate the MID of the five-level EQ-5D (EQ-5D-5L) index score in a population-based sample of adults with type 2 diabetes and to explore whether the MID estimate varies by baseline index score and the direction of change in health status.

Methods

We used longitudinal survey data of adults with type 2 diabetes in Alberta, Canada. The EQ-5D-5L MID was estimated first by the instrument-defined approach, which used the difference between the baseline index scores and the index scores of simulated single-level transitions, and then by the anchor-based approach, which categorized 1-year changes in depressive symptoms, diabetes-related distress, as well as physical and mental health functioning into no change, small change, and large change groups, wherein the MID was estimated as the average change in index score of the small change group.

Results

Using the instrument-defined approach, MID estimates were 0.043, 0.040, and 0.045, whereas anchor-based MID estimates were 0.042, 0.034, and 0.049 for all change, improvement, and deterioration, respectively. Larger MID estimates were observed for lower baseline index scores and for deterioration in health status.

Conclusions

MID estimates of the EQ-5D-5L index score were consistent between instrument-defined and anchor-based approaches and ranged between 0.03 and 0.05. Estimates varied by baseline index score and the direction of change, with similar results for patient subgroups.  相似文献   

17.

Background

Management of hypertension can lead to significant reductions in blood pressure, thereby reducing the risk of cardiovascular disease. Modeling the course of cardiovascular disease is not without complications, and uncertainty surrounding the structure of a model will almost always arise once a choice of a model structure is defined.

Objectives

To provide a practical illustration of the impact on the results of cost-effectiveness of changing or adapting model structures in a previously published cost-utility analysis of a primary care intervention for the management of hypertension Targets and Self-Management for the Control of Blood Pressure in Stroke and at Risk Groups (TASMIN-SR).

Methods

The case study assessed the structural uncertainty arising from model structure and from the exclusion of secondary events. Four alternative model structures were implemented. Long-term cost-effectiveness was estimated and the results compared with those from the TASMIN-SR model.

Results

The main cost-effectiveness results obtained in the TASMIN-SR study did not change with the implementation of alternative model structures. Choice of model type was limited to a cohort Markov model, and because of the lack of epidemiological data, only model 4 captured structural uncertainty arising from the exclusion of secondary events in the case study model.

Conclusions

The results of this study indicate that the main conclusions drawn from the TASMIN-SR model of cost-effectiveness were robust to changes in model structure and the inclusion of secondary events. Even though one of the models produced results that were different to those of TASMIN-SR, the fact that the main conclusions were identical suggests that a more parsimonious model may have sufficed.  相似文献   

18.

Background

Many decision-analytic models with varying structures have been developed to inform resource allocation in chronic obstructive pulmonary disease (COPD).

Objectives

To review COPD models for their adherence to the best practice modeling recommendations and their assumptions regarding important aspects of the natural history of COPD.

Methods

A systematic search of English articles reporting on the development or application of a decision-analytic model in COPD was performed in MEDLINE, Embase, and citations within reviewed articles. Studies were summarized and evaluated on the basis of their adherence to the Consolidated Health Economic Evaluation Reporting Standards. They were also evaluated for the underlying assumptions about disease progression, heterogeneity, comorbidity, and treatment effects.

Results

Forty-nine models of COPD were included. Decision trees and Markov models were the most popular techniques (43 studies). Quality of reporting and adherence to the guidelines were generally high, especially in more recent publications. Disease progression was modeled through clinical staging in most studies. Although most studies (n = 43) had incorporated some aspects of COPD heterogeneity, only 8 reported the results across subgroups. Only 2 evaluations explicitly considered the impact of comorbidities. Treatment effect had been mostly modeled (20) as both reduction in exacerbation rate and improvement in lung function.

Conclusions

Many COPD models have been developed, generally with similar structural elements. COPD is highly heterogeneous, and comorbid conditions play an important role in its burden. These important aspects, however, have not been adequately addressed in most of the published models.  相似文献   

19.

Background

In 2014, Austrian health authorities implemented an organized breast cancer screening program. Until then, there has been a long-standing tradition of opportunistic screening.

Objectives

To evaluate the cost-effectiveness of organized screening compared with opportunistic screening, as well as to identify factors influencing the clinical and economic outcomes.

Methods

We developed and validated an individual-level state-transition model and assessed the health outcomes and costs of organized and opportunistic screening for 40-year-old asymptomatic women. The base-case analysis compared a scenario involving organized biennial screening with a scenario reflecting opportunistic screening practice for an average-risk woman aged 45 to 69 years. We applied an annual discount rate of 3% and estimated the incremental cost-effectiveness ratio in terms of the cost (2012 euros) per life-year gained (LYG) from a health care perspective. Deterministic and probabilistic sensitivity analyses were performed to assess uncertainty.

Results

Compared with opportunistic screening, an organized program yielded on average additional 0.0118 undiscounted life-years (i.e., 4.3 days) and cost savings of €41 per woman. In the base-case analysis, the incremental cost-effectiveness ratio of organized screening was approximately €20,000 per LYG compared with no screening. Assuming a willingness-to-pay threshold of €50,000 per LYG, there was a 70% probability that organized screening would be considered cost-effective. The attendance rate, but not the test accuracy of mammography, was an influential factor for the cost-effectiveness.

Conclusions

The decision to adopt organized screening is likely an efficient use of limited health care resources in Austria.  相似文献   

20.

Background

Extrapolation of time-to-event data can be a critical component of cost-effectiveness analysis.

Objectives

To contrast the value of external data on treatment effects as a selection aid in model fitting to the clinical data or for the direct extrapolation of survival.

Methods

We assume the existence of external summary data on both treatment and control and consider two scenarios: availability of external individual patient data (IPD) on the control only and an absence of external IPD. We describe how the summary data can be used to extrapolate survival or to assess the plausibility of extrapolations of the clinical data. We assess the merit of either approach using a comparison of cemented and cementless total hip replacement as a case study. Merit is judged by comparing incremental net benefit (INB) obtained in scenarios with incomplete IPD with that derived from modeling external IPD on both treatment and control.

Results

Measures of fit with the external summary data did not identify survival model specifications that best estimated INB. Addition of external IPD for the control only did not improve estimates of INB. Extrapolation of survival using the external summary data comparing treatment and control improved estimates of INB.

Conclusions

Our case study indicates that summary data comparing treatment and control are more valuable than IPD limited to the control when extrapolating event rates for cost-effectiveness analysis. These data are best exploited in direct extrapolation of event rates rather than as an aid to select extrapolations on the basis of the clinical data.  相似文献   

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