Medical care and cost outcomes after pentoxifylline treatment for peripheral arterial disease.

We assessed the medical outcomes and costs associated with the pharmacologic treatment of patients with peripheral arterial disease (PAD) in a population-based historical cohort study of patients enrolled in a health maintenance organization. For up to 2 years, we compared 58 patients who used therapeutic amounts of pentoxifylline with a comparison group of 112 patients who received a minimal subefficacious trial of pentoxifylline. Medical records data were used to assess and control for the severity of PAD and other potentially confounding factors. Continuous use of a therapeutic amount of pentoxifylline during an initial 120-day period significantly reduced the incidence of PAD-related invasive therapeutic and diagnostic procedures in the first year of follow-up (adjusted relative risk, 0.35; 95% confidence interval, 0.12 to 0.99). However, there were no significant differences in the risk of a PAD-related hospitalization or cost of PAD-related care between continuous pentoxifylline users and the comparison group. Pentoxifylline therapy may reduce the risk of vascular surgery while not increasing the total cost of PAD care.

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.

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 pharmaceutical industry and health reform: lessons from Europe.

Many countries in Europe, which have significant pharmaceutical industries, recently have embarked on reforms in the organization of their health care systems and methods of regulating the pharmaceutical market. This paper describes the different systems in major European countries and the changes taking place. Although it is too early to make final judgment on the impact of these changes, it is possible to predict some of the more likely outcomes. The organizational models and regulatory approaches in Europe offer a useful store of experience for those assessing the potential impact of reform proposals in the United States.

Cost analysis of imipenem-cilastatin versus clindamycin with tobramycin in the treatment of acute intra-abdominal infection.

Clinical effectiveness of imipenem/cilastatin (I/C) versus tobramycin with clindamycin (T + C) in treatment of patients presenting with suspected acute intra-abdominal infection was assessed in a multicentre randomised clinical trial conducted during 1985 to 1986. The principal finding was a lower incidence of treatment failure among patients in the I/C arm (p = 0.043). We now report results of retrospective analysis of hospital treatment costs during an episode of infection incurred by patients enrolled in the trial. Treatment costs (in 1989 US dollars) were calculated from a hospital perspective, using an intention-to-treat analysis. Among 161 patients with low illness severity (APACHE II less than or equal to 14) the mean cost for the episode of care was $US7038 in the I/C arm versus $US8404 for the T + C regimen; the difference was not statistically significant (p = 0.40). For 93 more severely ill patients (APACHE II score greater than 14) the mean cost for the I/C arm was $US19 985 versus $US16 582 for the T + C regimen; the difference was not statistically significant (p = 0.36). Multiple regression analysis, controlling for patient demographics and study site, showed that the cost of the episode was positively associated with the severity of illness (p less than 0.01) and presence of malnutrition (p < 0.01), but that the total cost of the episode of infection was not statistically different for the 2 drug regimens (p = 0.45).

Recombinant human granulocyte-macrophage colony-stimulating factor after autologous bone marrow transplantation for lymphoid cancer: an economic analysis of a randomised, double-blind, placebo-controlled trial.

In a blinded retrospective economic evaluation of a double-blind, randomised, placebo-controlled clinical trial, total utilisation and charges for lymphoid cancer patients who received recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) or placebo were compared following autologous bone marrow transplantation. The 40 patients enrolled (22 rhGM-CSF, 18 placebo) could have acute lymphoblastic leukaemia, non-Hodgkins lymphoma or Hodgkin's disease, be of any age, and were undergoing autologous bone marrow transplantation in a metropolitan cancer research centre. Main outcome measures consisted of initial hospital lengths of stay (LOS), total and department charges, rehospitalisation rates and charges, and outpatient charges, all inclusive of the first 100 days following bone marrow infusion. The perspective of the study is that of the third party payer. Initial hospitalisation charges were $US54 100 for patients who received rhGM-CSF and $US68 600 for patients who received placebo (p = 0.05). The difference of $US14 500 was 21% less in patients who received rhGM-CSF, mainly due to lower average LOS with rhGM-CSF (24.2 days) compared with placebo (30.8 days). Outpatient charges were $US9500 (rhGM-CSF) and $US6800 (placebo) {p = 0.18}. Total charges, including readmission (10 per group) were $US12 200 lower in the rhGM-CSF group ($US70 300 vs $US82 500, p = 0.19). The use of rhGM-CSF after autologous bone marrow transplantation was shown to result in substantial cost savings during the initial hospitalisation. When comparing total inpatient and outpatient medical charges within the first 100 days following bone marrow infusion, we found no evidence that these savings were negated.

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.

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.

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.

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.

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.

Making better technology purchasing decisions.

Many health care technologies are being used without a clear idea of their worth, a problem that Congress has addressed through the legislative process. This article describes an approach hospital trustees can use to make more cost-effective decisions about whether to acquire a given technology and when to use it.

Evaluating technology under changing financing arrangements.

Changes in health care financing, such as Medicare prospective pricing and Medicaid capitated payment programs, have encouraged medical product industries to provide evidence of new technologies' cost-effectiveness. Health care providers have an equal responsibility to determine the validity of that research.

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.

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.

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.

Estimating costs in the economic evaluation of medical technologies.

The complexities and nuances of evaluating the costs associated with providing medical technologies are often underestimated by analysts engaged in economic evaluations. This article describes the theoretical underpinnings of cost estimation, emphasizing the importance of accounting for opportunity costs and marginal costs. The various types of costs that should be considered in an analysis are described; a listing of specific cost elements may provide a helpful guide to analysis. The process of identifying and estimating costs is detailed, and practical recommendations for handling the challenges of cost estimation are provided. The roles of sensitivity analysis and discounting are characterized, as are determinants of the types of costs to include in an analysis. Finally, common problems facing the analyst are enumerated with suggestions for managing these problems.