Josephine Mauskopf

Consolidated Health Economic Evaluation Reporting Standards (CHEERS)—Explanation and Elaboration: A Report of the ISPOR Health Economic Evaluation Publication Guidelines Good Reporting Practices Task Force

BACKGROUND Economic evaluations of health interventions pose a particular challenge for reporting because substantial information must be conveyed to allow scrutiny of study findings. Despite a growth in published reports, existing reporting guidelines are not widely adopted. There is also a need to consolidate and update existing guidelines and promote their use in a user-friendly manner. A checklist is one way to help authors, editors, and peer reviewers use guidelines to improve reporting. OBJECTIVE The task forces overall goal was to provide recommendations to optimize the reporting of health economic evaluations. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement is an attempt to consolidate and update previous health economic evaluation guidelines into one current, useful reporting guidance. The CHEERS Elaboration and Explanation Report of the ISPOR Health Economic Evaluation Publication Guidelines Good Reporting Practices Task Force facilitates the use of the CHEERS statement by providing examples and explanations for each recommendation. The primary audiences for the CHEERS statement are researchers reporting economic evaluations and the editors and peer reviewers assessing them for publication. METHODS The need for new reporting guidance was identified by a survey of medical editors. Previously published checklists or guidance documents related to reporting economic evaluations were identified from a systematic review and subsequent survey of task force members. A list of possible items from these efforts was created. A two-round, modified Delphi Panel with representatives from academia, clinical practice, industry, and government, as well as the editorial community, was used to identify a minimum set of items important for reporting from the larger list. RESULTS Out of 44 candidate items, 24 items and accompanying recommendations were developed, with some specific recommendations for single study-based and model-based economic evaluations. The final recommendations are subdivided into six main categories: 1) title and abstract, 2) introduction, 3) methods, 4) results, 5) discussion, and 6) other. The recommendations are contained in the CHEERS statement, a user-friendly 24-item checklist. The task force report provides explanation and elaboration, as well as an example for each recommendation. The ISPOR CHEERS statement is available online via Value in Health or the ISPOR Health Economic Evaluation Publication Guidelines Good Reporting Practices - CHEERS Task Force webpage (http://www.ispor.org/TaskForces/EconomicPubGuidelines.asp). CONCLUSIONS We hope that the ISPOR CHEERS statement and the accompanying task force report guidance will lead to more consistent and transparent reporting, and ultimately, better health decisions. To facilitate wider dissemination and uptake of this guidance, we are copublishing the CHEERS statement across 10 health economics and medical journals. We encourage other journals and groups to consider endorsing the CHEERS statement. The author team plans to review the checklist for an update in 5 years.

Conjoint analysis applications in health - A checklist: A report of the ISPOR Good Research Practices for Conjoint Analysis Task Force

BACKGROUND The application of conjoint analysis (including discrete-choice experiments and other multiattribute stated-preference methods) in health has increased rapidly over the past decade. A wider acceptance of these methods is limited by an absence of consensus-based methodological standards. OBJECTIVE The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Good Research Practices for Conjoint Analysis Task Force was established to identify good research practices for conjoint-analysis applications in health. METHODS The task force met regularly to identify the important steps in a conjoint analysis, to discuss good research practices for conjoint analysis, and to develop and refine the key criteria for identifying good research practices. ISPOR members contributed to this process through an extensive consultation process. A final consensus meeting was held to revise the article using these comments, and those of a number of international reviewers. RESULTS Task force findings are presented as a 10-item checklist covering: 1) research question; 2) attributes and levels; 3) construction of tasks; 4) experimental design; 5) preference elicitation; 6) instrument design; 7) data-collection plan; 8) statistical analyses; 9) results and conclusions; and 10) study presentation. A primary question relating to each of the 10 items is posed, and three sub-questions examine finer issues within items. CONCLUSIONS Although the checklist should not be interpreted as endorsing any specific methodological approach to conjoint analysis, it can facilitate future training activities and discussions of good research practices for the application of conjoint-analysis methods in health care studies.

Budget impact analysis-principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force.

BACKGROUND Budget impact analyses (BIAs) are an essential part of a comprehensive economic assessment of a health care intervention and are increasingly required by reimbursement authorities as part of a listing or reimbursement submission. OBJECTIVES The objective of this report was to present updated guidance on methods for those undertaking such analyses or for those reviewing the results of such analyses. This update was needed, in part, because of developments in BIA methods as well as a growing interest, particularly in emerging markets, in matters related to affordability and population health impacts of health care interventions. METHODS The Task Force was approved by the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council and appointed by its Board of Directors. Members were experienced developers or users of BIAs; worked in academia and industry and as advisors to governments; and came from several countries in North America and South America, Oceania, Asia, and Europe. The Task Force solicited comments on the drafts from a core group of external reviewers and, more broadly, from the membership of the International Society for Pharmacoeconomics and Outcomes Research. RESULTS The Task Force recommends that the design of a BIA for a new health care intervention should take into account relevant features of the health care system, possible access restrictions, the anticipated uptake of the new intervention, and the use and effects of the current and new interventions. The key elements of a BIA include estimating the size of the eligible population, the current mix of treatments and the expected mix after the introduction of the new intervention, the cost of the treatment mixes, and any changes expected in condition-related costs. Where possible, the BIA calculations should be performed by using a simple cost calculator approach because of its ease of use for budget holders. In instances, however, in which the changes in eligible population size, disease severity mix, or treatment patterns cannot be credibly captured by using the cost calculator approach, a cohort or patient-level condition-specific model may be used to estimate the budget impact of the new intervention, accounting appropriately for those entering and leaving the eligible population over time. In either case, the BIA should use data that reflect values specific to a particular decision makers population. Sensitivity analysis should be of alternative scenarios chosen from the perspective of the decision maker. The validation of the model should include at least face validity with decision makers and verification of the calculations. Data sources for the BIA should include published clinical trial estimates and comparator studies for the efficacy and safety of the current and new interventions as well as the decision makers own population for the other parameter estimates, where possible. Other data sources include the use of published data, well-recognized local or national statistical information, and, in special circumstances, expert opinion. Reporting of the BIA should provide detailed information about the input parameter values and calculations at a level of detail that would allow another modeler to replicate the analysis. The outcomes of the BIA should be presented in the format of interest to health care decision makers. In a computer program, options should be provided for different categories of costs to be included or excluded from the analysis. CONCLUSIONS We recommend a framework for the BIA, provide guidance on the acquisition and use of data, and offer a common reporting format that will promote standardization and transparency. Adherence to these good research practice principles would not necessarily supersede jurisdiction-specific BIA guidelines but may support and enhance local recommendations or serve as a starting point for payers wishing to promulgate methodology guidelines.

The Role of Cost—Consequence Analysis in Healthcare Decision—Making

SummaryA greater understanding of value associated with new pharmaceutical products should lead to better decision—making. Most commonly cost—effectiveness ratios (CERs) are used to indicate value; however, researchers have recently shown that CER estimates are rarely used by decision—makers in making formulary decisions. In this article, a cost—consequence approach to estimating the value for money of a new treatment for a specific disease is described. Using a cost—consequence approach, the impact of the new treatment on lifetime resource use and costs (including specific healthcare service use and costs, and productivity losses) and health outcomes (including disease symptoms, life expectancy and quality of life) for an individual or group of individuals is estimated and presented in a tabular format. The cost—consequence format is more likely to be approachable, readily understandable and applied by healthcare decision—makers than a simple CER. The decision—maker may use selected items from the costconsequence analysis to compute composite measures of drug value, such as cost per life—year gained or cost per quality—adjusted life—year (QALY) gained. In general, the cost—consequence approach, by making the impact of the new treatment as comprehensive and transparent as possible, will enable decision—makers to select the components most relevant to their perspective and will also give them confidence that the data are credible to use as the basis for resource allocation decisions.

Protease inhibitor exposure and increased risk of cardiovascular disease in HIV-infected patients.

To study the relationship between exposure to protease inhibitor (PI) therapy and increased risk of cardiovascular events in HIV‐infected patients.

Health policy issues and applications for evidence-based medicine and clinical practice guidelines.

Evidence-based medicine and clinical practice guidelines have become increasingly salient to the international health care community in the 1990s. Key issues in health policy in this period can be categorised as costs and access to care, quality of and satisfaction with care, accountability for value in health care, and public health and education. This paper presents a brief overview of evidence-based medicine and clinical practice guidelines and describes how they are likely to influence health policy. Evidence-based medicine focuses on the use of the best available clinical (efficacy) evidence to inform decisions about patient care; guidelines are statements systematically developed from efficacy and effectiveness research and clinical consensus for practitioners and patients to use in making decisions about appropriate care under different clinical circumstances. Both fields have developed methods for evaluating and synthesising available evidence about the outcomes of alternative health care interventions. They have clear implications for health policy analysts: greater reliance should be placed on scientific evidence, policy decisions should be derived systematically, and health care decisionmaking must allow for the active participation of health care providers, policy makers, and patients or their advocates. The methods and information generated from evidence-based guidelines efforts are critical inputs into health policy analysis and decision-making.

Value in Health OnlineConsolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement

Economic evaluations of health interventions pose a particular challenge for reporting. There is also a need to consolidate and update existing guidelines and promote their use in a user friendly manner. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement is an attempt to consolidate and update previous health economic evaluation guidelines efforts into one current, useful reporting guidance. The primary audiences for the CHEERS statement are researchers reporting economic evaluations and the editors and peer reviewers assessing them for publication. The need for new reporting guidance was identified by a survey of medical editors. A list of possible items based on a systematic review was created. A two round, modified Delphi panel consisting of representatives from academia, clinical practice, industry, government, and the editorial community was conducted. Out of 44 candidate items, 24 items and accompanying recommendations were developed. The recommendations are contained in a user friendly, 24 item checklist. A copy of the statement, accompanying checklist, and this report can be found on the ISPOR Health Economic Evaluations Publication Guidelines Task Force website: (www.ispor.org/TaskForces/EconomicPubGuidelines.asp). We hope CHEERS will lead to better reporting, and ultimately, better health decisions. To facilitate dissemination and uptake, the CHEERS statement is being co-published across 10 health economics and medical journals. We encourage other journals and groups, to endorse CHEERS. The author team plans to review the checklist for an update in five years.

Willingness to Pay as a Measure of Health Benefits

In this paper, we discuss the use of cost-benefit analysis (CBA) for evaluating new healthcare interventions, present the theoretical basis for the use of willingness to pay as a method for valuing benefits in a CBA and describe how to obtain willingness-to-pay (WTP) measures of health benefits and how to use these values in a CBA.We review selected economic studies on consumer demand and consumer surplus and studies presenting WTP estimates for healthcare interventions. The theoretical foundations of willingness to pay as a measure of commodity value are rooted in consumer demand theory. The area under the fixed income consumer demand curve represents the consumer’s maximum willingness to pay for the commodity. We identify 3 types of potential benefits from a new healthcare intervention, namely patient benefits, option value and altruistic value, and suggest WTP questions for valuing different combinations of these benefits. We demonstrate how responses to these questions can be adjusted for income effects and incorporated into economic evaluations.We suggest that the lack of popularity of CBAs in the health area is related to the perceived difficulty in valuing health benefits as well as concern over how CBA incorporates the distribution of income. We show that health benefits can be valued using simple survey techniques and that these values can be adjusted to any desired income distribution.

CONSOLIDATED HEALTH ECONOMIC EVALUATION REPORTING STANDARDS (CHEERS) STATEMENT

Economic evaluations of health interventions pose a particular challenge for reporting. There is also a need to consolidate and update existing guidelines and promote their use in a user friendly manner. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement is an attempt to consolidate and update previous health economic evaluation guidelines efforts into one current, useful reporting guidance. The primary audiences for the CHEERS statement are researchers reporting economic evaluations and the editors and peer reviewers assessing them for publication. The need for new reporting guidance was identified by a survey of medical editors. A list of possible items based on a systematic review was created. A two round, modified Delphi panel consisting of representatives from academia, clinical practice, industry, government, and the editorial community was conducted. Out of 44 candidate items, 24 items and accompanying recommendations were developed. The recommendations are contained in a user friendly, 24 item checklist. A copy of the statement, accompanying checklist, and this report can be found on the ISPOR Health Economic Evaluations Publication Guidelines Task Force website (www.ispor.org/TaskForces/EconomicPubGuidelines.asp). We hope CHEERS will lead to better reporting, and ultimately, better health decisions. To facilitate dissemination and uptake, the CHEERS statement is being co-published across 10 health economics and medical journals. We encourage other journals and groups, to endorse CHEERS. The author team plans to review the checklist for an update in five years.

Potential Savings in the Cost of Caring for Alzheimer’s Disease

AbstractObjective: To estimate savings in the cost of caring for patientswith Alzheimer’s disease (AD) during 6 months, 1 year and 2 years of treatment with rivastigmine. An intermediate objective was to estimate the relationship between disease progression and institutionalisation.Design and setting: We assessed the relationship between Mini-Mental State Examination (MMSE) score and institutionalisation using a piecewise Cox proportional hazard model. To estimate cost savings from treatments lasting 6 months, 1 year and 2 years, estimates of the probability of institutionalisation were integrated with data from two 6-month phase III clinical trials of rivastigmine and a hazard model of disease progression.Main outcome measures and results: Our data suggest that savings in the overall cost of caring for patients with mild and moderate AD can be as high as