Milton C. Weinstein

Foundations of Cost-Effectiveness Analysis for Health and Medical Practices

Limits on health-care resources mandate that resource-allocation decisions be guided by considerations of cost in relation to expected benefits. In cost-effectiveness analysis, the ratio of net health-care costs to net health benefits provides an index by which priorities may be set. Quality-of-life concerns, including both adverse and beneficial effects of therapy, may be incorporated in the calculation of health benefits as adjustments to life expectancy. The timing of future benefits and costs may be accounted for by the appropriate use of discounting. Current decisions must inevitably be based on imperfect information, but sensitivity analysis can increase the level of confidence in some decisions while suggesting areas where further research may be valuable in guiding others. Analyses should be adaptable to the needs of various health-care decision makers, including planners, administrators and providers.

Performance of a five-item mental health screening test

We compared the screening accuracy of a short, five-item version of the Mental Health Inventory (MHI-5) with that of the 18-item MHI, the 30-item version of the General Health Questionnaire (GHQ-30), and a 28-item Somatic Symptom Inventory (SSI-28). Subjects were newly enrolled members of a health maintenance organization (HMO), and the criterion diagnoses were those found through use of the Diagnostic Interview Schedule (DIS) in a stratified sample of respondents to an initial, mailed GHQ. To compare questionnaires, we used receiver operating characteristic analysis, comparing areas under curves through the method of Hanley and McNeil. The MHI-5 was as good as the MHI-18 and the GHQ-30, and better than the SSI-28, for detecting most significant DIS disorders, including major depression, affective disorders generally, and anxiety disorders. Areas under curve for the MHI-5 ranged from 0.739 (for anxiety disorders) to 0.892 (for major depression). Single items from the MHI also performed well. In this population, short screening questionnaires, and even single items, may detect the majority of people with DIS disorders while incurring acceptably low false-positive rates. Perhaps such extremely short questionnaires could more commonly reach use in actual practice than the longer versions have so far, permitting earlier assessment and more appropriate treatment of psychiatrically troubled patients in primary care settings.

Updating Cost-Effectiveness — The Curious Resilience of the

The ratio of

50,000-per-QALY Threshold

50,000 per quality-adjusted life-year (QALY) gained by using a given health care intervention has long served as a benchmark for the value of U.S. health care. But evidence suggests that it is too low and might best be thought of as an implied lower boundary.

von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA)†

Summary.  von Willebrand disease (VWD) is a commonly encountered inherited bleeding disorder affecting both males and females, causing mucous membrane and skin bleeding symptoms, and bleeding with surgical or other haemostatic challenges. VWD may be disproportionately symptomatic in women of child‐bearing age. It may also occur less frequently as an acquired disorder (acquired von Willebrand syndrome). VWD is caused by deficiency or dysfunction of von Willebrand factor (VWF), a plasma protein that mediates platelet haemostatic function and stabilizes blood coagulation factor VIII. The pathophysiology, classification, diagnosis and management of VWD are relatively complex, but understanding them is important for proper diagnosis and management of patients with VWD. These evidence‐based guidelines for diagnosis and management of VWD from the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel (USA) review relevant publications, summarize current understanding of VWD pathophysiology and classification, and present consensus diagnostic and management recommendations based on analysis of the literature and expert opinion. They also suggest an approach for clinical and laboratory evaluation of individuals with bleeding symptoms, history of bleeding or conditions associated with increased bleeding risk. This document summarizes needs for further research in VWF, VWD and bleeding disorders, including clinical research to obtain more objective information about bleeding symptoms, advancements in diagnostic and therapeutic tools, and enhancement in the education and training of clinicians and scientists in bleeding and thrombotic disorders. The NHLBI Web site (http://www.nhlbi.nih.gov/guidelines/vwd) has a more detailed document, a synopsis of these recommendations, and patient education information.

The Survival Benefits of AIDS Treatment in the United States

BACKGROUND As widespread adoption of potent combination antiretroviral therapy (ART) reaches its tenth year, our objective was to quantify the cumulative survival benefits of acquired immunodeficiency syndrome (AIDS) care in the United States. METHODS We defined eras corresponding to advances in standards of human immunodeficiency virus (HIV) disease care, including opportunistic infection prophylaxis, treatment with ART, and the prevention of mother-to-child transmission (pMTCT) of HIV. Per-person survival benefits for each era were determined using a mathematical simulation model. Published estimates provided the number of adult patients with new diagnoses of AIDS who were receiving care in the United States from 1989 to 2003. RESULTS Compared with survival associated with untreated HIV disease, per-person survival increased 0.26 years with Pneumocystis jiroveci pneumonia prophylaxis alone. Four eras of increasingly effective ART in addition to prophylaxis resulted in per-person survival increases of 7.81, 11.05, 11.57, and 13.33 years, compared with the absence of treatment. Treatment for patients with AIDS in care in the United States since 1989 yielded a total survival benefit of 2.8 million years. pMTCT averted nearly 2900 infant infections, equivalent to 137,000 additional years of survival benefit. CONCLUSIONS At least 3.0 million years of life have been saved in the United States as a direct result of care of patients with AIDS, highlighting the significant advances made in HIV disease treatment.

The Cost Effectiveness of Combination Antiretroviral Therapy for HIV Disease

BACKGROUND Combination antiretroviral therapy with a combination of three or more drugs has become the standard of care for patients with human immunodeficiency virus (HIV) infection in the United States. We estimated the clinical benefits and cost effectiveness of three-drug antiretroviral regimens. METHODS We developed a mathematical simulation model of HIV disease, using the CD4 cell count and HIV RNA level as predictors of the progression of disease. Outcome measures included life expectancy, life expectancy adjusted for the quality of life, lifetime direct medical costs, and cost effectiveness in dollars per quality-adjusted year of life gained. Clinical data were derived from major clinical trials, including the AIDS Clinical Trials Group 320 Study. Data on costs were based on the national AIDS Cost and Services Utilization Survey, with drug costs obtained from the Red Book. RESULTS For patients similar to those in the AIDS Clinical Trials Group 320 Study (mean CD4 cell count, 87 per cubic millimeter), life expectancy adjusted for the quality of life increased from 1.53 to 2.91 years, and per-person lifetime costs increased from

The lifetime cost of current human immunodeficiency virus care in the United States.

45,460 to

Use and Misuse of the Term “Cost Effective” in Medicine

77,300 with three-drug therapy as compared with no therapy. The incremental cost per quality-adjusted year of life gained, as compared with no therapy, was

Preferences for Health Outcomes: Comparison of Assessment Methods

23,000. On the basis of additional data from other major studies, the cost-effectiveness ratio for three-drug therapy ranged from