Statistical methods for cost-effectiveness analyses

The Journal of Mental Health Policy and Economics, 1998

Background: Increasing attention is being focused on the costs of healthcare and the need for cost-effective treatments. Drugs for schizophrenia have not escaped this scrutiny, especially now that several new agents are available, with acquisition costs substantially higher than for established therapies. However, most of the existing evaluations of new drugs for schizophrenia have weak designs, either comparing health care costs before and after introduction of the new drug, or being based on modelling approaches incorporating numerous assumptions. Aim of the Study: The aim of the study was to discuss and resolve the key design issues in the planning of a prospective randomized trial to assess the socioeconomic impact of a new atypical antipsychotic (quetiapine). Methods: Key methodological issues were identified and discussed in the context of the economic evaluation being planned. These were patient recruitment and entry criteria, selection of comparator drug, blinding of doctor and patient, range of socioeconomic outcomes, length of follow-up and sample size. Results: The resulting economic evaluation, the ESTO study, was an international multi-centre randomized controlled trial, with concurrent data collection for a wide range of clinical, economic and quality of life outcomes. The trial had a pragmatic design, enrolling patients experiencing an acute exacerbation on existing therapy. In addition to the presenting exacerbation, patients must have had at least one hospitalization or documented evidence of exacerbation within the previous three years. On admission to the study, existing psychotic medication was withdrawn prior to randomization to quetiapine or haloperidol. Doses of both drugs were titrated up to an optional dose, with flexibility for additional increases if required. Both patients and doctors were blinded to treatment allocations, on the grounds that, since quetiapine was still in development, unblinded assessments of efficacy would not be credible. Patients were followed for 1 year, irrespective of whether they withdrew from study medication. A wide range of socioeconomic outcomes was assessed, including costs falling on the healthcare sector, other agencies and the family. In addition data were collected on patients' earnings and quality of life, measured by the Short-Form 36 health profile. Data were also collected on a range of clinical measures, such as

Revista Brasileira de Psiquiatria. 2013;35:186–192, 2013

Objective: Technological advances in medicine have given rise to a dilemma concerning the use of new health technologies in a context of limited financial resources. In the field of psychiatry, health economic evaluation is a recent method that can assist in choosing interventions with different cost and/or effectiveness for specific populations or conditions. This article introduces clinicians to the fundamental concepts required for critical assessment of health economic evaluations. Methods: The authors conducted a review with systematic methods to assess the essential theoretical framework of health economic evaluation and mental health in Brazil through textbooks and studies indexed in the PubMed, Cochrane Central, LILACS, NHS CRD, and REBRATS databases. A total of 334 studies were found using the specified terms (MeSH - Mental Health AND Economic, Medical) and filters (Brazil AND Humans); however, only five Brazilian economic evaluations were found. Results and conclusions: Ec...

Value in Health, 2005

Objectives: A growing number of prospective clinical trials include economic end points. Recognizing the variation in methodology and reporting of these studies, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) chartered the Task Force on Good Research Practices: Randomized Clinical Trials-Cost-Effectiveness Analysis. Its goal was to develop a guidance document for designing, conducting, and reporting cost-effectiveness analyses conducted as a part of clinical trials. Methods: Task force cochairs were selected by the ISPOR Board of Directors. Cochairs invited panel members to participate. Panel members included representatives from academia, the pharmaceutical industry, and health insurance plans. An outline and a draft report developed by the panel were presented at the 2004 International and European ISPOR meetings, respectively. The manuscript was then submitted to a reference group for review and comment. Results: The report addresses issues related to trial design, selecting data elements, database design and man-agement, analysis, and reporting of results. Task force members agreed that trials should be designed to evaluate effectiveness (rather than efficacy), should include clinical outcome measures, and should obtain health resource use and health state utilities directly from study subjects. Collection of economic data should be fully integrated into the study. Analyses should be guided by an analysis plan and hypotheses. An incremental analysis should be conducted with an intention-to-treat approach. Uncertainty should be characterized. Manuscripts should adhere to established standards for reporting results of costeffectiveness analyses. Conclusions: Trial-based cost-effectiveness studies have appeal because of their high internal validity and timeliness. Improving the quality and uniformity of these studies will increase their value to decision makers who consider evidence of economic value along with clinical efficacy when making resource allocation decisions.

Australian and New Zealand Journal of Psychiatry, 2005

Objective: To assess from a health sector perspective the incremental cost-effectiveness of eight drug treatment scenarios for established schizophrenia. Method: Using a standardized methodology, costs and outcomes are modelled over the lifetime of prevalent cases of schizophrenia in Australia in 2000. A two-stage approach to assessment of health benefit is used. The first stage involves a quantitative analysis based on disability-adjusted life years (DALYs) averted, using best available evidence. The robustness of results is tested using probabilistic uncertainty analysis. The second stage involves application of 'second filter' criteria (equity, strength of evidence, feasibility and acceptability) to allow broader concepts of benefit to be considered. Results: Replacing oral typicals with risperidone or olanzapine has an incremental costeffectiveness ratio (ICER) of A$48 000 and A$92 000/DALY respectively. Switching from low-dose typicals to risperidone has an ICER of A$80 000. Giving risperidone to people experiencing side-effects on typicals is more cost-effective at A$20 000. Giving clozapine to people taking typicals, with the worst course of the disorder and either little or clear deterioration, is cost-effective at A$42 000 or A$23 000/DALY respectively. The least costeffective intervention is to replace risperidone with olanzapine at A$160 000/DALY. Conclusions: Based on an A$50 000/DALY threshold, low-dose typical neuroleptics are indicated as the treatment of choice for established schizophrenia, with risperidone being reserved for those experiencing moderate to severe side-effects on typicals. The more expensive olanzapine should only be prescribed when risperidone is not clinically indicated. The high cost of risperidone and olanzapine relative to modest health gains underlie this conclusion. Earlier introduction of clozapine however, would be cost-effective. This work is limited by weaknesses in trials (lack of long-term efficacy data, quality of life and consumer satisfaction evidence) and the translation of effect size into a DALY change. Some stakeholders, including SANE Australia, argue the modest health gains reported in the literature do not adequately reflect perceptions by patients, clinicians and carers, of improved quality of life with these atypicals.

The European Journal of Health Economics, 2017

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NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics, 2004

Economic evaluations are a set of outcomes and health services research methods to inform the debate about the rising cost of health care and include cost-of-illness studies and cost-effectiveness research. Cost-effectiveness research is the comparative analysis of two or more alternative interventions in terms of their health and economic consequences, whose results are expressed as an incremental cost-effectiveness ratio, the ratio of differences in cost between a pair of medical interventions to the differences in the corresponding health effects. These research methods are particularly important to neurological diseases with debilitating natural histories, long-term courses, and a growing number of exciting, yet costly, treatment options available. The results of economic evaluations of neurological conditions influence resource allocation decisions, help set reimbursement rates, estimate future healthcare expenses, and improve the quality and efficiency of delivering neurologic...

2003

Background Background This paper is part of a This paper is part of a projectto identify the proportion of the projectto identify the proportion of the burden of each mental disorder averted by burden of each mental disorder averted by current and optimalinterventions, and the current and optimalinterventions, and the cost-effectiveness of both. cost-effectiveness of both. Aims Aims To use epidemiological data on To use epidemiological data on schizophrenia to model the cost- schizophrenia to model the cost- effectiveness of current and optimal effectiveness of current and optimal treatment. treatment. Method Method Calculate the burden of Calculate the burden of schizophrenia in the years lived with schizophrenia in the years lived with disability (YLD) component of disability- disability (YLD) component of disability- adjusted life-years lost, the proportion adjusted life-years lost, the proportion averted by current interventions, the averted by current interventions, the proport...

Journal of Health Economics, 1997

To address controversies in the application of cost-effectiveness analysis, we investigate the principles underlying the technique and discuss the implications for the evaluation of medical interventions. Using a standard yon Neumann-Morgenstem utility framework, we show how a cost-effectiveness criterion can be derived to guide resource allocation decisions, and how it varies with age. gender, income level, and risk aversion. Although cost-effectiveness analysis can be a useful and powerful tool for resource allocatiou decisions, a uniform cost-effectiveness criterion that is applied to a heterogeneous population level is unlikely to yield Pareto-optimal resource allocations. JEL classification: i I 8:D61

Journal of Affective Disorders, 2012

Background: Knowledge regarding the relative cost-effectiveness of different antidepressants is crucial for the planning of depression treatment. However, there have been only a small number of reviews of such evidence and synthesizing economic evidence across studies is methodologically challenging. In particular, there have been few reviews of the methods employed in database analyses (studies that use data from real-world practice). Methods: Published economic evaluations based on database analyses were systematically reviewed to compare antidepressant treatments in depression. Prospective studies of costeffectiveness were also reviewed to highlight unanswered questions through comparisons between these two different study designs. Results: Forty papers met the criteria and were included. A relatively large number of industrysponsored evaluations of escitalopram were identified and these found escitalopram to be potentially cost-effective in depression treatment. Evidence of cost-effectiveness differences between other individual SSRIs was not unequivocally established. Inconsistent findings further emerged concerning the cost-effectiveness of SSRIs versus TCAs between retrospective database analyses and prospective studies. Limitations: Different outcome measures and cost perspectives make it difficult to make comparisons across studies. Conclusions: Evidence regarding the cost-effectiveness of different antidepressants in depression continues to accumulate. Beyond the efficacy or tolerability data found for newer antidepressants in controlled trials, further research from real-world settings is needed to examine the relative cost-effectiveness of different antidepressant agents.

Results Results Conventional antipsychotics Conventional antipsychotics had lower costs and higher quality-had lower costs and higher qualityadjusted life-years (QALYs) than atypical adjusted life-years (QALYs) than atypical antipsychotics and were more than 50% antipsychotics and were more than 50% likely to be cost-effective. likely to be cost-effective. Conclusions Conclusions The primary and The primary and sensitivity analyses indicated that sensitivity analyses indicated that conventional antipsychotics may be cost-conventional antipsychotics may be costsaving and associated with a gain in QALYs saving and associated with a gain in QALYs compared with atypical antipsychotics. compared with atypical antipsychotics.