Health care expenditures in the United States have been increasing much more rapidly than the rest of the economy over the past 30 years. The average gap, 2.8% per annum, results in health care's share of the economy doubling every 26 years.1 Why does this matter? Would it matter if expenditures for personal computers were increasing 2.8% per annum more rapidly than the rest of the economy? The appropriate response would be, "So what?" Concern about health care expenditures is often attributed to the large role of these expenditures in the federal budget and the effect on the deficit.2 But that is not the whole story. A dollar spent on health care is not a priori more fiscally toxic than a dollar spent on transportation or education or any other item in the government budget. Moreover, health expenditures in the private sector have also been increasing.

This report evaluates the level of evidence currently available to support the effectiveness and safety of using recombinant activated coagulation factor VII (rFVIIa) for clinical indications not approved by the U. S. Food and Drug Administration (FDA). rFVIIa is approved for a variety of uses in hemophilia patients who have developed antibody inhibitors that compromise the use of standard factor replacement. Use of this costly biologic product has expanded beyond these hemophilia-related indications to encompass a range of off-label uses, most of which are in-hospital uses. These uses differ substantially from the drug’s FDA approved label. The purpose of this report is two-fold: (1) To document the full range of clinical indications for which rFVIIa is being used and the types of studies available to evaluate these uses and (2) To provide a comparative effectiveness review of rFVIIa vs. usual care for several in-hospital clinical indications: intracranial hemorrhage, massive bleeding secondary to trauma, and the selected surgical procedures of cardiac surgery, liver transplantation, and prostatectomy.

Off-label drug use refers to any use of a medication that deviates from the product labeling approved and required by the FDA. The FDA drug approval process mandates randomized clinical trials that demonstrate efficacy and safety for specific indications prior to marketing. Once approval is given, however, the FDA does not regulate whether drugs are prescribed for off-label indications. In most instances, the data supporting off-label drug use falls short of the rigor that accompanies FDA review. This uncertainty may be acceptable, as when a drug’s use is infrequent. Nevertheless, concerns increase when off-label use is clinically distinct from approved indications, when off-label use becomes frequent, when a drug is costly, or when a drug is used in different clinical settings (e.g., shifts from outpatient to in-hospital use).

rFVIIa is a form of human factor VII produced by recombinant technology. This intravenously delivered product works as a potent procoagulant by effectively bypassing parts of the clotting process normally required for clotting. It can facilitate control of bleeding in situations where standard human blood product transfusions have failed. Novoseven® is the only form of rFVIIa available commercially. Developed in the late 1980s, rFVIIa was approved by the FDA in 1999 for use in patients with Hemophilia A and Hemophilia B with antibody inhibitors that lead to unresponsiveness to factor VIII or factor IX, respectively. Both of these X-linked genetic conditions are rare, and most hemophilia patients never require rFVIIa for treatment of bleeding episodes. While the hemophilia population has remained stable over the past decade, in-hospital, off-label use of rFVIIa has increased. 

OBJECTIVE: The aim of this study was to investigate training in developmental and behavioral pediatrics (DBP) for graduating residents, their competencies in diagnosing and treating child mental health (MH) problems, and whether the amount of DBP training and/or perceived competencies are associated with perceived responsibility for treating 3 MH problems.

METHODS: Data were collected from 636 residents who completed the American Academy of Pediatrics's 2007 Graduating Residents Survey. The survey included questions on training and self-rated competencies in multiple MH skill areas and perceived responsibility for identifying and treating/managing children's MH problems. Weighted multivariable logistic regression analyses examined associations between training, competencies, and perceived responsibility for treating/managing attention-deficit/hyperactivity disorder (ADHD), anxiety, and depression.

RESULTS: Ninety percent of respondents completed a DBP rotation, with 86% reporting >3 to 4 weeks of training. Duration of DBP rotation was related to training and perceived competencies in MH skill areas, and nearly all residents who reported high competencies were trained in those skill areas. However, <50% reported their competencies as "very good" or "excellent." Residents with training and high competency in dosing with medications were most likely to agree that pediatricians should be responsible for treating/managing ADHD, anxiety, and depression.

CONCLUSIONS: DBP training is highly associated with self-rated MH competencies, and highly assessed competencies are related to perceived responsibility for treating/managing common MH problems; yet 14% of graduating residents have <3 to 4 weeks of DBP training. These results argue for providing more high-quality educational experience with proven effectiveness to produce confident pediatricians who will be more responsive to identifying and treating MH problems of their patients.

Background: Sodium consumption raises blood pressure, increasing the risk for heart attack and stroke. Several countries, including the United States, are considering strategies to decrease population sodium intake.

Objective: To assess the cost-effectiveness of 2 population strategies to reduce sodium intake: government collaboration with food manufacturers to voluntarily cut sodium in processed foods, modeled on the United Kingdom experience, and a sodium tax.

Design: A Markov model was constructed with 4 health states: well, acute myocardial infarction (MI), acute stroke, and history of MI or stroke.

Data Sources: Medical Panel Expenditure Survey (2006), Framingham Heart Study (1980 to 2003), Dietary Approaches to Stop Hypertension trial, and other published data.

Target Population: U.S. adults aged 40 to 85 years.

Time Horizon: Lifetime.

Perspective: Societal.

Outcome Measures: Incremental costs (2008 U.S. dollars), quality-adjusted life-years (QALYs), and MIs and strokes averted.

Results of Base-case Analysis: Collaboration with industry that decreases mean population sodium intake by 9.5% averts 513 885 strokes and 480 358 MIs over the lifetime of adults aged 40 to 85 years who are alive today compared with the status quo, increasing QALYs by 2.1 million and saving $32.1 billion in medical costs. A tax on sodium that decreases population sodium intake by 6% increases QALYs by 1.3 million and saves $22.4 billion over the same period.

Results of Sensitivity Analysis: Results are sensitive to the assumption that consumers have no disutility with modest reductions in sodium intake.

Limitation: Efforts to reduce population sodium intake could result in other dietary changes that are difficult to predict.

Conclusion: Strategies to reduce sodium intake on a population level in the United States are likely to substantially reduce stroke and MI incidence, which would save billions of dollars in medical expenses.

Primary Funding Source: Department of Veterans Affairs, Stanford University, and the National Science Foundation.

Abstract Objective. To develop and evaluate a clinical decision support system (CDSS) named Assessment and Treatment in Healthcare: Evidenced-Based Automation (ATHENA)-Opioid Therapy, which encourages safe and effective use of opioid therapy for chronic, noncancer pain. Design. CDSS development and iterative evaluation using the analysis, design, development, implementation, and evaluation process including simulation-based and in-clinic assessments of usability for providers followed by targeted system revisions. Results. Volunteers provided detailed feedback to guide improvements in the graphical user interface, and content and design changes to increase clinical usefulness, understandability, clinical workflow fit, and ease of completing guideline recommended practices. Revisions based on feedback increased CDSS usability ratings over time. Practice concerns outside the scope of the CDSS were also identified. Conclusions. Usability testing optimized the CDSS to better address barriers such as lack of provider education, confusion in dosing calculations and titration schedules, access to relevant patient information, provider discontinuity, documentation, and access to validated assessment tools. It also highlighted barriers to good clinical practice that are difficult to address with CDSS technology in its current conceptualization. For example, clinicians indicated that constraints on time and competing priorities in primary care, discomfort in patient-provider communications, and lack of evidence to guide opioid prescribing decisions impeded their ability to provide effective, guideline-adherent pain management. Iterative testing was essential for designing a highly usable and acceptable CDSS; however, identified barriers may limit the impact of the ATHENA-Opioid Therapy system and other CDSS on clinical practices and outcomes unless CDSS are paired with parallel initiatives to address these issues.

Background: Sodium consumption raises blood pressure, increasing the risk for heart attack and stroke. Several countries, including the United States, are considering strategies to decrease population sodium intake.

Objective: To assess the cost-effectiveness of 2 population strategies to reduce sodium intake: government collaboration with food manufacturers to voluntarily cut sodium in processed foods, modeled on the United Kingdom experience, and a sodium tax.

Design: A Markov model was constructed with 4 health states: well, acute myocardial infarction (MI), acute stroke, and history of MI or stroke.

Data Sources: Medical Panel Expenditure Survey (2006), Framingham Heart Study (1980 to 2003), Dietary Approaches to Stop Hypertension trial, and other published data.

Target Population: U.S. adults aged 40 to 85 years.

Time Horizon: Lifetime.

Perspective: Societal.

Outcome Measures: Incremental costs (2008 U.S. dollars), quality-adjusted life-years (QALYs), and MIs and strokes averted.

Results of Base-case Analysis: Collaboration with industry that decreases mean population sodium intake by 9.5% averts 513 885 strokes and 480 358 MIs over the lifetime of adults aged 40 to 85 years who are alive today compared with the status quo, increasing QALYs by 2.1 million and saving $32.1 billion in medical costs. A tax on sodium that decreases population sodium intake by 6% increases QALYs by 1.3 million and saves $22.4 billion over the same period.

Results of Sensitivity Analysis: Results are sensitive to the assumption that consumers have no disutility with modest reductions in sodium intake.

Limitation: Efforts to reduce population sodium intake could result in other dietary changes that are difficult to predict.

Conclusion: Strategies to reduce sodium intake on a population level in the United States are likely to substantially reduce stroke and MI incidence, which would save billions of dollars in medical expenses.

Primary Funding Source: Department of Veterans Affairs, Stanford University, and the National Science Foundation.

BACKGROUND AND OBJECTIVES: Rates of dialysis withdrawal are higher among the elderly and lower among Blacks, yet it is unknown whether preferences for withdrawal and engagement in advance care planning also vary by age and race or ethnicity. DESIGN, SETTING, PARTICIPANTS AND METHODS: We recruited 61 participants from two dialysis clinics to complete questionnaires regarding dialysis withdrawal preferences in five different health states. Engagement in advance care planning (end-of-life discussions), completion of advance directives and 'do not resuscitate' or 'do not intubate' (DNR/DNI) orders were ascertained by a questionnaire and from dialysis unit records. RESULTS: The mean age was 62 +/- 15 years; 38% were Black, 11% were Latino, 34% were White and 16% of participants were Asian. Blacks were less likely to prefer dialysis withdrawal as compared with Whites (odds ratio 0.16, 95% confidence interval 0.03-0.88) and other race/ethnicity groups, and this difference was not explained by age, education, comorbidity and other confounders. In contrast, older age was not associated with preferences for withdrawal. Rates of engagement in end-of-life discussions were higher than for documentation of advance care planning for all age and most race/ethnicity groups. Although younger participants and minorities were generally less likely to document treatment preferences as compared with older patients and Whites, they were not less likely to engage in end-of-life discussions. CONCLUSIONS: Preferences for withdrawal vary by race/ ethnicity, whereas the pattern of engagement in advance care planning varies by age and race/ethnicity. Knowledge of these differences may be useful for improving communication about end-of-life preferences and in implementing effective advance care planning strategies among diverse haemodialysis patients.

A collection of core faculty Victor Fuchs' articles on actions needed for meaningful health care reform in the United States.

• Eliminating "Waste" in Health Care
• Four Health Care Reforms for 2009
• Cost Shifting Does Not Reduce the Cost of Health Care.
• The Proposed Government Health Insurance Company - No Substitute for Real Reform
• Reforming US Health Care - Key Considerations for the New Administration.
• Health Reform: Getting The Essentials Right
• Health Care Reform - Why So Much Talk and So Little Action?
• Three "Inconvenient Truths" about Health Care
• The Perfect Storm of Overutilization
• Who Really Pays for Health Care? The Myth of "Shared Responsibility".
• What Are The Prospects For Enduring Comprehensive Health Care Reform?
• Essential Elements of a Technology and Outcomes Assessment Initiative

Although policymakers have increasingly turned to provider report cards as a tool to improve health care quality, existing studies provide mixed evidence that they influence consumer choices. We examine the effects of providing consumers with quality information in the context of fertility clinics providing Assisted Reproductive Therapies (ART). We report three main findings. First, clinics with higher birthrates had larger market shares after relative to before the adoption of report cards. Second, clinics with a disproportionate share of young, relatively easy-to-treat patients had lower market shares after adoption versus before. This suggests that consumers take into account information on patient mix when evaluating clinic outcomes. Third, report cards had larger effects on consumers and clinics from states with ART insurance coverage mandates. We conclude that quality report cards have potential to influence provider behavior in this setting.