Cost-effectiveness analysis of an intranasal influenza vaccine for the prevention of influenza in healthy children.

OBJECTIVE: Intranasal influenza vaccine has proven clinical efficacy and may be better tolerated by young children and their families than an injectable vaccine. This study determined the potential cost-effectiveness (CE) of an intranasal influenza vaccine among healthy children. METHODS: We conducted a CE analysis of data collected between 1996 and 1998 during a prospective 2-year efficacy trial of intranasal influenza vaccine, supplemented with data from the literature. The CE analysis included both direct and indirect costs. We enrolled 1602 healthy children aged 15 to 71 months in year 1, 1358 of whom were enrolled in year 2. One or 2 doses of intranasal influenza vaccine or placebo were administered to measure the cost per febrile influenza-like illness (ILI) day avoided. RESULTS: During the 2-year study period, vaccinated children had an average of 1.2 fewer ILI fever days/child than unvaccinated children. In an individual-based vaccine delivery scenario with vaccine given twice in the first year and once each year thereafter at an assumed base case total cost of $20 for the vaccine and its administration (ie, per dose), CE was approximately $30/febrile ILI day avoided. CE ranged from $10 to $69/febrile ILI day avoided at $10 to $40/dose, respectively. In a group-based delivery scenario, vaccination was cost saving compared with placebo and remained so if vaccine cost was <$28 (the break-even price per dose). In the individual-based scenario, vaccination was cost saving if vaccine cost was <$5. In this scenario, nearly half of lost productivity in the vaccine group was attributable to vaccine visits, which overshadowed the relatively modest savings in ILI-associated costs averted. CONCLUSIONS: Routine use of intranasal influenza vaccine among healthy children may be cost-effective and may be maximized by using group-based vaccination approaches. cost-effectiveness, influenza, vaccine, children.

The limited state of technology assessment for medical devices: facing the issues.

Medical devices are an integral part of clinical practice and account for a substantial proportion of the national health budget. Clinical testing and regulation of medical devices, however, is vastly different from and inferior to the testing and regulation of drugs. As managed care organizations begin to exert controls on device use, providers are being caught between the policies of their organizations and the demands of device manufacturers and patients, who want wider access to devices. We outline several reasons for the poor state of medical device evaluations and the dangers of using devices without adequate information, and include the recently developed device assessment and reporting guidelines created by the Task Force on Technology Assessment of Medical Devices.

Health care CBA and CEA from 1991 to 1996: an updated bibliography.

OBJECTIVES: This article updates, through 1996, a previously published bibliography of health-care cost-benefit and cost-effectiveness analysis, which described the literature from 1979 to 1990. METHODS: A systematic search of MEDLARS databases was conducted for all articles falling under the medical subject headings "cost-benefit analysis" (which includes cost-effectiveness analysis) and "costs and cost analysis," as well as any article with the term "cost" in the title or abstract. All titles and abstracts were scanned to determine whether articles pertained to personal health services and whether both costs and consequences were assessed. If both criteria were met, the article was included in the bibliography. RESULTS: This search resulted in 3,539 eligible cost-benefit/cost-effectiveness analysis publications from 1991 through 1996. Publications were subdivided into two major categories: reports of studies and "other" publications, including reviews, descriptions of methodology, letters, and editorials. Reports of studies and "other" publications were classified into approximately 250 different topic areas. Studies were further classified by parameters such as study type, publication vehicle, and medical function. This article describes the results of this classification and describes trends during 1991 to 1996 as compared with 1979 to 1990. OVERVIEW OF CONTENTS: The entire bibliography is reproduced in Appendix A. The classification of study reports and "other" publications into topic areas is presented in Appendix B, with numbered references to all bibliography entries. Detailed tables of findings are presented in Appendix C, and the results are illustrated graphically in Appendix D.

The economic and clinical efficiency of point-of-care testing for critically ill patients: a decision-analysis model.

Our study objective was to assess economic and clinical outcomes of use of a point-of-care (POC) blood analysis device for postoperative coronary artery bypass graft (CABG) patients. A decision analytic model was developed for patients with high expected use of blood analysis, high potential benefit from rapid turn around time of results, a large annual volume of patients, and substantial expense associated with surgery. Published literature and clinical experts provided incidence, outcome, and cost estimates associated with four clinical scenarios potentially influenced by POC testing (ventricular arrhythmias, cardiac arrest, severe postoperative bleeding, and iatrogenic anemia). We found that changes in clinical outcomes were predominantly dependent on comparative turn around time or CABG patient volume. The positive clinical impact of using POC testing was consistently associated with a positive economic impact. POC blood gas analysis may be associated with decreased incidence of adverse clinical events or earlier detection of such events, resulting in significant cost savings. This study also supports previous findings that the costs of STAT blood analysis are more personnel-related than equipment-related.

Health and economic outcomes modeling practices: a suggested framework.

In the field of outcomes research, modeling is increasingly being used to assess costs and outcomes associated with healthcare interventions. However, there is little standardization with regard to modeling practices, and the quality and usefulness of economic and health outcomes models may vary. We propose the following set of recommendations for good modeling practices to be used in both the development and review of models. These recommendations are divided into three sections: criteria to be addressed before model development or initial review; criteria used during model development or evaluation; and criteria used following model development. These recommendations also include examples regarding different modeling techniques and practices as well as a checklist (see appendix) to assess model correspondence with the recommendations. We hope that the use of good practice recommendations for modeling will not only improve the development and review of models but also lead to greater acceptance of this methodology.

When is a cost-effectiveness claim valid? How much should the FDA care?

Federal law requires the Food and Drug Administration (FDA) to regulate the promotional claims of prescription drugs and certain devices. Standards of evidence for claims of safety and therapeutic efficacy are rigorous because inappropriate product use may place human life at risk. However, equally demanding criteria for claims of cost-effectiveness of marketed technologies seem to be unnecessary because the consequence of error is principally a bad buy rather than patient harm. Concern exists about the validity of cost-effectiveness studies, the potential for bias, standards for the conduct of cost-effectiveness research, and the needs of managed care. The FDA should moderate its role in regulating cost-effectiveness claims of drugs and devices. This would foster information flow to healthcare providers and insurers and protect the FDA concern regarding false or misleading claims of effectiveness. Although the issues are applicable to both devices and drugs, we draw mainly from the field of pharmacoeconomics because this is where most of the policy has developed.

Managed care pharmacy, socioeconomic assessments and drug adoption decisions.

A telephone survey of a representative national sample of 51 large managed care organizations in the U.S. (> 50,000 enrollees) was undertaken (1) to understand the role of socioeconomic assessments on drug adoption decisions; (2) to determine the sources of these assessments and the reliance of managed care pharmacy on each; and (3) to determine the resources for internally versus externally performed drug assessments. Socioeconomic assessments (clinical effectiveness, safety, cost of treatment, cost-effectiveness, and quality of life) are often tied to formulary decisions. Plans differ in their use of externally available socioeconomic assessments and in their ratings of the importance to decision making of drug assessments from the various sources. Those using a specific source of drug assessment information rated them in the following order of importance: PBM assessments, other HMOs, peer reviewed literature, evaluations performed by industry, articles in non-peer reviewed publications and, lastly, government reports. Timeliness and comprehensiveness are important components of the overall utility of information. A high percentage of plans reported using some of the various types of assessments, with clinical effectiveness most common, and cost-effectiveness second. The percentage of new drugs that undergo assessments in each of the plans covers a broad range, with 57% of the plans evaluating at least half of all new drugs. All but one surveyed managed care plan reported having either implemented or plans to implement a disease management program. Eighty percent of those surveyed are more concerned about drug assessments than in the past and 88% anticipate greater future use. Although 38 plans (75%) have a person in the organization responsible for drug assessments, this is the primary job in only 14 plans (37%). With greater reliance on drug assessments in the future, there are substantial opportunities for integrating drug assessments, formularies and disease management programs.

Guidelines for pharmacoeconomic studies. Recommendations from the panel on cost effectiveness in health and medicine. Panel on cost Effectiveness in Health and Medicine.

This article reports the recommendations of the Panel on Cost Effectiveness in Health and Medicine, sponsored by the US Public Health Service, on standardised methods for conducting cost-effectiveness analyses. Although not expressly directed at analyses of pharmaceutical agents, the Panel's recommendations are relevant to pharmacoeconomic studies. The Panel outlines a 'Reference Case' set of methodological practices to improve quality and comparability of analyses. Designed for studies that inform resource-allocation decisions, the Reference Case includes recommendations for study framing and scope, components of the numerator and denominator of cost-effectiveness ratios, discounting, handling uncertainty and reporting. The Reference Case analysis is conducted from the societal perspective, and includes all effects of interventions on resource use and health. Resource use includes 'time' resources, such as for caregiving or undergoing an intervention. The quality-adjusted life-year (QALY) is the common measure of health effect across Reference Case studies. Although the Panel does not endorse a measure for obtaining quality-of-life weights, several recommendations address the QALY. The Panel recommends a 3% discount rate for costs and health effects. Pharmacoeconomic studies have burgeoned in recent years. The Reference Case analysis will improve study quality and usability, and permit comparison of pharmaceuticals with other health interventions.

National Asthma Education and Prevention Program working group report on the cost effectiveness of asthma care.

The cost effectiveness of asthma care is the first topic of the National Asthma Education and Prevention Program Task Force Report on the Cost Effectiveness, Quality of Care, and Financing of Asthma Care. This working group characterized the role of health economics in understanding optimal asthma management strategies. The report first reviews methods for economic evaluation of medical technologies, with a particular focus on cost-effectiveness analysis. Next, the report explores the nature and usefulness of several key asthma outcome measures, including clinical and symptom measures, measures of lung function, measures of functional status, and measures of health services utilization and cost. The working group also conducted a review of the literature on cost effectiveness of asthma patient education programs, pharmaceutical therapy, and a variety of alternative and adjunct interventions. The report concludes with recommendations for a standardized approach for economic evaluation study designs (e.g., common asthma outcomes, long-term follow-up, and studies with patients of different ages, socioeconomic statuses, and severity levels) and for an expansion in the number of such standardized asthma studies and cost-effectiveness analyses.

The view from managed care pharmacy.

This stratified, national telephone survey of fifty-one managed care organizations concerns the perspectives of managed care pharmacy directors on pharmacoeconomics, disease management, and the roles of the pharmaceutical industry and the Food and Drug Administration (FDA). Respondents rated clinical effectiveness assessments as most useful, cost-effectiveness assessments second, and quality-of-life assessments as least useful. Peer-reviewed and industry literature were rated as equally important for decision making. Most plans would consider establishing a partnership with a drug company for disease management, if they have not already done so. Most plans (76 percent) support some form of FDA regulation of pharmacoeconomic claims. Conversely, 69 percent favor either no regulation (24 percent) or less stringent regulation (45 percent) than exists today.

The use of technology assessment by hospitals, health maintenance organizations, and third-party payers in the United States.

A case study design was used to determine the reliance on technology assessment of decisionmakers in hospitals, health maintenance organizations (HMOs), and third-party payers. Thirty different organizations were contacted and semistructured interviews conducted. The study found that hospitals, HMOs, and insurers are conducting technology assessments, but the form and sophistication of these analyses range widely. Hospitals are particularly focused on traditional financial analyses ("prudent purchasing") with the exception of pharmacy committees, which generally conduct more sophisticated socio-economic analyses. HMOs and insurers conduct outcome assessments for coverage of expensive or controversial technologies but exclude economics. Technology assessment will become increasingly important in resource allocation decision making and it is in the interest of technology providers to foster better information, a more comprehensive assessment process, and a more efficient assessment system.

Methods of pharmacoeconomic evaluation of new medical treatments in psychiatry.

There is concern in developed countries about the increasing cost of health care. Developers of new psychopharmacologic agents are asked to demonstrate cost-effectiveness as well as safety and efficacy before widespread use is encouraged. Pharmacoeconomic studies are designed to evaluate the health outcomes and cost-effectiveness of new medical treatments relative to existing treatments. Retrospective and prospective research designs have been used in pharmacoeconomic studies. New approaches such as medical effectiveness studies and modeling studies based on clinical decision analysis are increasingly used to evaluate cost-effectiveness. This article introduces important concepts of pharmacoeconomic evaluation and discusses the methods and their strengths and weaknesses. Safety and efficacy studies and pharmacoeconomic studies provide useful and complementary evidence on how a new treatment affects clinical, health-related quality-of-life, and economic outcomes.

Methods of cost-effectiveness analysis: areas of consensus and debate.

Methods of evaluating socioeconomic relationships have evolved over many years, and a number of specific approaches have been developed. Among the techniques available, cost-effectiveness analysis (CEA) has emerged as the most widely used and accepted method. Yet, despite considerable effort by the analytical community to refine this technique into one more useful for making health policy decisions, much debate and confusion still persist among analysts, readers, and policy-makers concerning methods standards and the overall usefulness of CEA in resource allocation decision making. Thus the purpose of this paper is to summarize, critically examine, and comment on existing recommended methods for socioeconomic evaluation of health care interventions. In particular, we examine an exhaustive set of component methods within the general area of cost-effectiveness and comment on areas of apparent consensus and debate. Our review reveals many areas of agreement and many yet to be resolved. Analysts generally agree on the components of the overall framework for an analysis; basic methodologic principles; the general treatment of costs; the principle of marginal analysis; the need for and general approach to discounting; the use of sensitivity analysis; the extent to which ethical issues can be incorporated; and the importance of choosing appropriate alternatives for comparison. The principal areas in which disagreement still persists are choice of study design, measurement and valuation of health outcomes including conversion of health outcomes to economic values, transformation of efficacy results into effectiveness outcomes, and the empirical measurement of costs.

Working toward a common currency: is standardization of cost-effectiveness analysis possible?

The field of cost-effectiveness has been quietly evolving for several decades. However, concerns and controversies have recently surfaced regarding the lack of standardization with which cost-effectiveness analyses are performed. This attention is due to the increasing use of the technique in rationing health care, setting price and/or reimbursement levels, adopting new technologies, and marketing pharmaceutical products. Is standardization possible while the field is still developing? This article explores possible answers to this question and reviews worldwide efforts aimed at the development of standardized guidelines for cost-effectiveness analysis.