Center For Outcomes Research Research Articles

Open Science and Data Sharing in Clinical Research

A patient tentatively enters the examination room. A decision looms, and the patient waits with some trepidation. A doctor soon appears and pulls up a chair, prepared for the discussion by having conscientiously reviewed the medical literature, examined the evidence-based systematic reviews, and highlighted the relevant information. The ensuing conversation reflects on the patient's treatment goals and the published information about the benefits and harms of the available options. The tension breaks with a decision, and plans for the next steps are put in place. Every day, patients and their caregivers are faced with difficult decisions about treatment. They turn to physicians and other healthcare professionals to interpret the medical evidence and assist them in making individualized decisions. Unfortunately, we are learning that what is published in the medical literature represents only a portion of the evidence that is relevant to the risks and benefits of available treatments. In a profession that seeks to rely on evidence, it is ironic that we tolerate a system that enables evidence to be outside of public view. Those who own data, usually scientists or industry, have the choice of what, where, and when to publish. As a result, our medical literature portrays only a partial picture of the evidence about clinical strategies, including drugs and devices. Experts have recently drawn attention to this issue, including contributions in this issue of our journal, but there is resistance to change.1–5 Studies document that a remarkable percentage of trials are not published within a reasonable period after they are completed. Ross and colleagues reported that fewer than half of trials were published within 2 years of completion.4 Although industry-sponsored studies tended to have the …

The quality of three decision-analytic diabetes models: a systematic health economic assessment

Aims: Diabetes mellitus has important economic impacts worldwide. Interventions to prevent diabetes-related complications are often analyzed using model-based cost–effectiveness analyses. As model results are usually influenced by structural assumptions and by the data used, decision-makers should be able to assess the quality of diabetes models. The aim of this study was to assess the quality of selected diabetes models and to determine if modeling recommendations by the American Diabetes Association are considered. Methods: The quality of three selected diabetes models (Archimedes Model, CDC Model and Center for Outcomes Research [CORE] Diabetes Model) was assessed using systematic methods. Results: This systematic approach to assess model quality proved to be feasible and highlighted two areas for improvement: the rationale for model structure and methods to identify parameter values, which should be presented more transparently. Conclusions: Overall, the need for a quality assessment of diabetes models is emphasized.

Commentary on “Patient-reported long-term outcomes after conventional and high-dose combined proton and photon radiation for early prostate cancer”. Talcott JA, Rossi C, Shipley WU, Clark JA, Slater JD, Niemierko A, Zietman AL, Center for Outcomes Research, MGH Cancer Center, Massachusetts General Hospital, Boston, MA: JAMA 2010;303:1046–53

Commentary on “Patient-reported long-term outcomes after conventional and high-dose combined proton and photon radiation for early prostate cancer”. Talcott JA, Rossi C, Shipley WU, Clark JA, Slater JD, Niemierko A, Zietman AL, Center for Outcomes Research, MGH Cancer Center, Massachusetts General Hospital, Boston, MA: JAMA 2010;303:1046–53

Point of View

From the Clinical Outcomes Research Center, University of Minnesota, Minneapolis, MN. The manuscript submitted does not contain information about medical device(s)/drug(s). No funds were received in support of this work. One or more of the author(s) has/have received benefits for personal or professional use from a commercial party related directly or indirectly to the subject of this manuscript: e.g., honoraria, gifts, consultancies, royalties, stocks, stock options, decision-making position. Address correspondence to Terry P. Corbin, University of Minnesota CORC, 14528 64th Avenue North, Maple Grove, MN 55311. E-mail: [email protected]

Quality-Adjusted Life-Year as a Surgical Outcome Measure: A Primer for Plastic Surgeons

Historically, surgical outcomes have been measured by rates of mortality and morbidity. Fortunately, in plastic surgery, mortality is rarely an issue unless one is practicing in high-risk settings such as burn units or war zones. The improvement of presurgical morbidity, however, is very relevant to our specialty, and great effort should be made to measure it as precisely as possible. The success of plastic surgery interventions has traditionally been measured by beforeand-after photographs in cosmetic surgery, pinch and grip strength and range of motion in hand surgery, percentage of successful flaps or replanted digits in microsurgery, and so forth. Although such outcomes are important, they serve only as proxies for the ultimate goal of optimal physical, mental, and social well-being of the patient. Would one seriously consider a face-lift result satisfactory if the patient required psychiatric support after her operation despite a much improved postoperative photograph? Likewise, would an improvement of 0.5 kg in grip strength after a novel hand procedure be considered successful if the patient was still unable to return to work? What about a meticulously executed flap to reconstruct a through-and-through defect of the mandible in a head and neck cancer where the patient died 6 months later from cancer recurrence?1 Plastic surgeons have recognized for some time now that there can be a discrepancy between their interpretation of a successful surgical outcome and the patient’s expectation. A rapid expansion of outcomes research has occurred over the past two decades in many medical specialties. This has been fueled, at least in part, by the practice of evidence-based medicine. Evidence-based medicine integrates individual clinical expertise with the best available clinical evidence from systematic research.2,3 Outcomes research sets out to measure a patient’s quality of life. The broader term “quality of life” can be defined as “the adequacy of people’s material circumstances and their feelings about these circumstances.”4,5 This encompasses indicators of life satisfaction; personal wealth and possessions; level of safety; level of freedom; spirituality; health perceptions; and physical, psychological, social, and cognitive well-being.4,5 Health-related quality of life, a subcomponent of quality of life, includes all areas specific to health, that is, physical, emotional, psychological, social, cognitive, and role functioning, in addition to abilities, relationships, perceptions, life satisfaction, and well-being. It refers to patients’ appraisals of their current level of functioning and satisfaction with it, compared with what they perceive to be ideal.6,7 There are several different methods available to measure a patient’s health-related quality of life. In the past two decades, a number of validated and reliable scales have been introduced to measure improvement in quality of life of plastic surgical interventions. One particular health-related quality-of-life outcome measure, although infrequently used in plastic surgery, is the quality-adjusted life-year. The quality-adjusted life-year was originally introduced as a measure of health effectiveness for cost-effectiveness analysis to assist decision makers in allocating scarce resources across competing health care programs.8–10 Health care spending in the United States is the highest in the world, averaging $7026 per person per year.11 However, the U.S. health care system ranks 37th among the 191 countries.12 Although nationwide efforts to decrease this unsustainable growth have had limited success so far, this issue will be dealt with sooner From the Department of Clinical Epidemiology and Biostatistics and the Surgical Outcomes Research Center, McMaster University, and the Department of Surgery, Division of Plastic and Reconstructive Surgery, St. Joseph’s Healthcare. Received for publication July 23, 2009; accepted October 29, 2009. Copyright ©2010 by the American Society of Plastic Surgeons

Clinical effectiveness and cost-effectiveness of continuous subcutaneous insulin infusion for diabetes: systematic review and economic evaluation

The National Institute for Health and Clinical Excellence (NICE) was reviewing its previous guidance on continuous subcutaneous insulin infusion (CSII). The review provided an assessment of evidence which had been published since the previous NICE appraisal (TA 151) in 2007. To examine the clinical effectiveness and cost-effectiveness of using CSII to treat diabetes. To update the previous assessment report by reviewing evidence that has emerged since the last appraisal, and to take account of developments in alternative therapies, in particular the long-acting analogue insulins, which cause fewer problems with hypoglycaemia. A systematic review of the literature and an economic evaluation were carried out. The bibliographic databases used were MEDLINE and EMBASE, 2002 to June 2007. The Cochrane Library (all sections), the Science Citation Index (for meeting abstracts only) and the website of the 2007 American Diabetes Association were also searched. The primary focus for type 1 diabetes mellitus (T1DM) was the comparison of CSII with multiple daily injection (MDI), based on the newer insulin analogues, but trials of neutral protamine Hagedorn (NPH)-based MDI that had been published since the last assessment were identified and described in brief. For type 2 diabetes mellitus (T2DM), all trials of MDI versus CSII were included, whether the long-acting insulin was analogue or not, because there was no evidence that analogue-based MDI was better than NPH-based MDI. Trials that were shorter than 12 weeks were excluded. Information on the patients' perspectives was obtained from four sources: the submission from the pump users group--Insulin Pump Therapy (INPUT); interviews with parents of young children who were members of INPUT; some recent studies; and from a summary of findings from the previous assessment report. Economic modelling used the Center for Outcomes Research (CORE) model, through an arrangement with the NICE and the pump manufacturers, whose submission also used the CORE model. The 74 studies used for analysis included eight randomised controlled trials (RCTs) of CSII versus analogue-based MDI in either T1DM or T2DM, eight new (since the last NICE appraisal) RCTs of CSII versus NPH-based MDI in T1DM, 48 observational studies of CSII, six studies of CSII in pregnancy, and four systematic reviews. The following benefits of CSII were highlighted: better control of blood glucose levels, as reflected by glycated haemoglobin (HbA1c) levels, with the size of improvement depending on the level before starting CSII; reduction in swings in blood glucose levels, and in problems due to the dawn phenomenon; fewer problems with hypoglycaemic episodes; reduction in insulin dose per day, thereby partly off-setting the cost of CSII; improved quality of life, including a reduction in the chronic fear of severe hypoglycaemia; more flexibility of lifestyle--no need to eat at fixed intervals, more freedom of lifestyle and easier participation in social and physical activity; and benefits for the patients' family. The submission from INPUT emphasised the quality of life gains from CSII, as well as improved control and fewer hypoglycaemic episodes. Also, there was a marked discrepancy between the improvement in social quality of life reported by successful pump users, and the lack of convincing health-related quality of life gains reported in the trials. With regard to economic evaluation, the main cost of CSII is for consumables, such as tubing and cannulas, and is about 1800-2000 pounds per year. The cost of the pump, assuming 4-year life, adds another 430-720 pounds per annum. The extra cost compared with analogue-based MDI averages 1700 pounds. Most studies, assuming a reduction in HbA1c level of 1.2%, found CSII to be cost-effective. The most important weakness of the evidence was the very small number of randomised trials of CSII against the most modern forms of MDI, using analogue insulins. Based on the totality of evidence, using observational studies to supplement the limited data from randomised trials against best MDI, CSII provides some advantages over MDI in T1DM for both children and adults. However, there was no evidence that CSII is better than analogue-based MDI in T2DM or in pregnancy. Further trials with larger numbers and longer durations comparing CSII and optimised MDI in adults, adolescents and children are needed. In addition, there should be a trial of CSII versus MDI with similar provision of structured education in both arms. A trial is also needed for pregnant women with pre-existing diabetes, to investigate using CSII to the best effect.

Reply: Liver transplantation costs in the era of a health care crisis

We would like to respond to Dr. Haque's recent letter1 regarding our article on health care charges incurred by liver transplant recipients beyond the transplant admission.2 In his letter, he addressed the need for health care cost curtailment across medical specialties. He stated that analyzing the real cost in terms of the paid amount would have been more helpful for guiding specific interventions for cost containment in this population. Although we agree that actual payments would be interesting, we were limited to the available data. Unlike kidney transplantation, there is no national registry of payments following liver transplants. Thus, access to a national, private payer database offered a unique and valuable opportunity for comparing relative costs and the factors that influence transplant payments across many providers. There is ample evidence that charge data are highly correlated with actual costs and can be used to accurately describe relative differences in health care expenditures following liver transplantation as a function of the Model for End-Stage Liver Disease score. Liver transplantation remains the only life-saving therapy for patients with end-stage liver failure and must be considered in the context of other, expensive life-saving treatments such as implantable cardiac assist devices and multi-agent chemotherapy, both of which are associated with significantly less survival benefit. In addition, the cost of liver transplantation is only partially within the control of providers. The cost of liver transplantation is directly linked to the interplay of the recipient's disease severity, the local allograft supply, and the donor quality.2, 3 Therefore, centers in geographic areas with a very high demand for organs with respect to the supply are able to provide transplants to substantial proportions of their candidates only at advanced stages of disease severity, and they use higher risk organs in order to reduce the risk of certain death from untreated liver failure. As shown in our articles, these factors, more than the process of care, drive the cost of this treatment. We certainly agree that appropriate cost containment measures such as avoiding unnecessary investigation and minimizing the practice of defensive medicine should be pursued where possible. We disagree with the assertion that liver transplant costs are significantly inflated by clinical practice. Transplant specialists are limited in what they can do to minimize costs under an organ allocation system that requires the sickest transplant candidates to undergo transplantation first and is characterized by marked regional differences in the organ supply. We believe that resolving geographic disparities in the organ supply to allow earlier transplantation is vital to the economic sustainability of transplantation in an era of limited health care resources. We thank Dr. Haque for his comments and encourage exploration of other data sources to advance our knowledge of modifiable factors that may help us to manage transplant-related costs without worsening disparities in underserved populations. It is equally important to consider the fact that liver transplantation routinely produces 5-year survival rates in excess of 70% for patients destined to die without treatment. Paula M. Buchanan*, Nino Dzebisashvili*, Krista L. Lentine*, David A. Axelrod* , Mark A. Schnitzler*, Paolo R. Salvalaggio* , * Center for Outcomes Research, Saint Louis University School of Medicine, Saint Louis, MO, Department of Surgery, Dartmouth-Hitchcock Medical Center, Hanover, New Hampshire, Department of Surgery, University of Washington, Seattle, WA.

A Simulation of the Comparative Long‐term Effectiveness of Liraglutide and Glimepiride Monotherapies in Patients with Type 2 Diabetes Mellitus

To project and compare long-term outcomes of morbidity and mortality, and costs of complications of type 2 diabetes mellitus from a randomized controlled trial of patients receiving liraglutide versus glimepiride monotherapy. Mathematic simulation using the validated Center for Outcomes Research (CORE) Diabetes Model, calibrated to baseline patient characteristics from a short-term, randomized, controlled trial of liraglutide and glimepiride monotherapies (Liraglutide Effect and Action in Diabetes [LEAD]-3 trial) and using data from long-term outcomes studies. Simulated routine clinical practice. Seven hundred forty-six patients with type 2 diabetes who participated in the LEAD-3 trial, and three hypothetical cohorts of 5000 patients each that were based on the baseline characteristics of the patients in the LEAD-3 trial. The patients in the LEAD-3 trial were randomly assigned to monotherapy with liraglutide 1.2 mg/day (251 patients), liraglutide 1.8 mg/day (247 patients), or glimepiride 8 mg/day (248 patients). The impact of the three treatments for type 2 diabetes on survival and cumulative incidence of cardiovascular, ocular, or renal events and costs were estimated at three time periods: 10, 20, and 30 years. Simulations predicted improved survival for liraglutide 1.8 and 1.2 mg at all three time points compared with glimepiride. Survival benefits were greatest after 30 years of follow-up: 16.5%, 13.6%, and 7.3%, respectively. The frequency of nonfatal renal and ocular events was lower for both liraglutide doses than for glimepiride. The rate of neuropathies leading to first or recurrent amputation was higher for glimepiride compared with both liraglutide doses. The average cumulative cost/patient was higher for glimepiride compared with liraglutide 1.2 mg and liraglutide 1.8 mg. With use of the CORE Diabetes Model and data from the LEAD-3 trial, long-term projected survival, diabetes complications, and costs favored liraglutide 1.2- and 1.8-mg monotherapies compared with glimepiride in the treatment of type 2 diabetes.

Registries and Selection Bias

Outcomes researchers share a strong interest in determining what is being achieved in practice. In the quest to reveal what is actually being done in medicine, we are often in a position to use existing data about clinical practice and patient experience to answer relevant questions, rather than having to generate data de novo. Patient registries, with their size, scope, and focus on patients who represent those seen in clinical practice, have become an increasingly common source of data for outcomes researchers. There is much to like about patient registries. These organized collections of data about clinical practice are a vehicle by which we can assemble massive amounts of information about patients and use it to assess quality of care, identify patterns of care, facilitate real-world research, and improve performance. In cardiovascular medicine, registries capture information about patients with acute coronary syndromes and heart failure as well as those who undergo procedures related to cardiac catheterization, percutaneous coronary intervention, carotid stents, and internal cardioverter defibrillators. Federal agencies are turning to registries to provide information about quality and produce insights about how to best practice medicine through an emphasis on comparative effectiveness research. Outcomes research articles based on registry data are numerous, including those from NRMI, CRUSADE, GWTG-HF, …

Review of the ICD Registry's Third Year, Expansion to include Lead Data and Pediatric ICD Procedures, and Role for Measuring Performance

ntroduction he National ICD Registry became the sole repository of CD implantation data for Medicare beneficiaries on April , 2006, and as of June 2009 had collected data from ,432 hospitals in the United States totaling over 380,000 mplants. The registry was developed through a partnership f the Heart Rhythm Society (HRS [www.HRSonline.org]) nd the American College of Cardiology Foundation ACCF [www.acc.org]) utilizing the expertise of the ational Cardiovascular Data Registry (NCDR [www. ccncdr.com]) and has made substantial progress toward everal predefined goals:

Medicine Should Be More Like Missouri

Missouri is the “Show Me” State. Although the origins of the term are uncertain, it seems that it might have been etched into the national consciousness during a speech by U.S. Congressman Willard Duncan Vandiver. In an 1899 speech he stated, “I come from a state that raises corn and cotton and cockleburs and Democrats, and frothy eloquence neither convinces nor satisfies me. I am from Missouri. You have got to show me.”1 The “Show Me” spirit is sometimes lacking in the field of Medicine, where we seem intent on integrating new tests and procedures before they are thoroughly vetted. Although we bemoan the slow adoption of effective treatments, we often prematurely adopt unproven strategies. Our quality measures are focused on underuse—the missed opportunity to provide a strongly indicated test or treatment. National registries, often industry-sponsored, also are commonly oriented toward addressing undertreatment, further fueling a national preoccupation on having not done enough rather than on having done too much. It is true that this emphasis on undertreatment has improved quality of care. In the mid-1990s, for example, only about half of the patients nationally, and in several states only about one-third, who were ideal candidates for β-blocker therapy after having survived an acute myocardial infarction were prescribed the medication. Ideal candidates were identified by careful chart reviews that determined a group with no documented absolute or relative contraindications.2 This pattern of care was described almost 15 years after clinical trials provided evidence of the benefit. Moreover, those who were treated had better survival, concordant with what would be expected based on the trials. Studies that revealed similarly stark patterns for the prescription of aspirin and angiotensin converting-enzyme inhibitors2–5 led to quality measures that tracked undertreatment, …

Questioning Conventional Wisdom

In science, what seems obvious may not be true, and what is accepted as conventional wisdom, may sometimes be based on flawed assumptions. What is more obvious than the fact that the sun revolves around the earth? Each day the sun rises in the eastern horizon, and we observe it moving across the sky until it retires in the west. And yet, it is not true that the earth is the center of the universe. Galileo de’ Galilei, the scientist born in Pisa, was persecuted for questioning such conventional wisdom and supporting the theory proposed by Nicolaus Copernicus of a heliocentric universe. Ultimately, the truth could not be suppressed. We have many examples of progress that resulted from the questioning of strongly held beliefs that once seemed so obvious. Who anticipated that a gastric ulcer could be considered an infectious disease?1 Who expected that drugs that effectively suppressed premature ventricular beats in patients with ischemic heart disease would cause harm?2 Who believed that treatment strategies associated with substantial risk reduction might be poorly adopted into practice even 10 years after the publication of the trials establishing their efficacy?3 Who could …

Public Reporting of 30-Day Mortality for Patients Hospitalized With Acute Myocardial Infarction and Heart Failure

For cardiac care, the Centers for Medicare M how often smokers are counseled to quit; how rapidly hospitals provide reperfusion therapy for patients with an ST-segment elevation myocardial infarction; and how often the ejection fraction is measured for patients with heart failure. All of these measures assess care that is strongly recommended by guidelines. The measures include only those patients who should receive this care and are reported at the hospital level simply as percentages. The performance on these measures should ideally be 100%, and the rates have improved markedly over time. ; View this table: Table. Acute Myocardial Infarction and Heart Failure Process-of-Care Measures On June 21, 2007, CMS expanded the measures to include outcomes and posted information on its “Hospital Compare” website1 regarding hospital-specific 30-day mortality rates for patients with acute myocardial infarction and heart failure. These measures used administrative claims data and were validated against measures using medical record data; the measures were approved by the National Quality Forum.2,3 In 2007, the publicly reported information was limited to describing hospitals as having rates that were higher, lower, or no different than the national average. Hospitals received information about their rates, their patients, and comparisons with other hospitals in their state and with the nation. This year, CMS is expanding the publicly available information. For acute myocardial infarction, heart failure, and now pneumonia, CMS will post the calculated risk-standardized 30-day mortality rates (RSMRs) and the 95% interval estimates for each hospital that treated Medicare patients with these conditions; hospital volume for each condition also will be posted. As …

Seeking Better Outcomes in Coronary Artery Bypass Grafting

Are we delivering the safest and most effective care? How best can we learn from our experience? For healthcare professionals who ask these questions, the article by Guru and colleagues in this issue of Circulation is essential reading.1 These Canadian investigators performed an implicit review and retrospective analysis of 347 randomly selected in-hospital deaths after coronary artery bypass grafting (CABG) surgery and determined that approximately one third might have been prevented by better care. The study offers many important lessons with implications far beyond cardiac surgery. Article p 2969 Guru and colleagues uncovered many opportunities for important improvements in a hospital system that could have been content with its successes. Their investigation was conducted in a system with no low-volume hospitals and low risk-adjusted mortality rates and involved the participation of experienced staff surgeons and division chiefs. They found remarkably high percentages of preventable deaths, defined as deaths that could have been avoided had optimal care been delivered. Optimal care was considered to be the best possible care that could be delivered if current resources were operating at peak performance in accordance with the best available evidence at the time of the hospital admission. The investigators found that as many as 107 preventable CABG-related deaths occurred in Ontario in fiscal year 2000 to 2001. These deaths occurred in every institution. In particular, the highest percentages of preventable deaths were not clustered at the institutions with the highest risk-adjusted mortality rates, indicating that even a low mortality rate should not beget complacency. Even in the hospital with the lowest risk-adjusted mortality rate, about 20% of deaths were deemed to be preventable. Guru and colleagues focused specifically on contributing causes of deaths that may have been prevented. In this way, many deaths had more than 1 potential contributing cause. In each …

Application of economic analyses in U.S. managed care formulary decisions: a private payer's experience.

Promoting use of pharmaco-economic models by formulary reviewers is a goal of the Academy of Managed Care Pharmacy (AMCP) Format for Formulary Submissions, but relatively few decision makers use such models, and many doubt that they provide meaningful input. To demonstrate how sophisticated disease-based pharmaco-economic models can aid formulary decision makers when long-term outcomes data are lacking. The Center for Outcomes Research (CORE) Diabetes Model (CDM), a published, validated Markov pharmaco-economic model that projects clinical and economic endpoints, was used to model the cost-effectiveness of exenatide, a new injectable antidiabetic agent that enhances glucose-dependent insulin secretion, in a standard cohort of type 2 diabetes patients (mean body mass index [BMI] = 27.5 3 kg/m2), compared with a modified obese cohort (mean BMI = 35 3 kg/m2) that was otherwise demographically identical at baseline to the standard cohort. The standard cohort was assumed to maintain baseline weight during treatment, and the modified obese cohort was assumed to experience weight loss of approximately 9% (mean = 3 kg/m2), with corresponding improvements in blood pressure, low density lipoprotein cholesterol, and triglycerides. We selected a 30-year time horizon because it was the time interval during which the CDM predicted most of the subjects would have died, and the costs obtained thus reasonably projected lifetime total direct medical costs for these cohorts. While treatment options certainly will change over a 30-year period, our goal was to estimate the incremental effect of exenatide over other available therapies. The model predicted reduced long-term treatment costs in obese patients, driven by an 11% decrease in cardiovascular disease burden and derived from the presumed weight loss. The incremental cost-effectiveness ratio (ICER) for adding exenatide over 3 years was 35,000 dollars/quality-adjusted life-year (QALY). Using a 30-year horizon, ICER values were 13,000 dollars/QALY versus insulin, 32,000 dollars versus generic glyburide, and 16,000 dollars versus no additional treatment. Exenatide dominated pioglitazone. By comparison, the 30-year ICER for exenatide versus insulin in the nonobese cohort was 33,000 dollars. These results were presented to the pharmacy and therapeutics (P&T) committee and influenced its decision to add exenatide to the drug formulary. While our modeling assumed certain patient characteristics (e.g., obesity, need of further A1c reduction at baseline, motivation to lose weight), the P&T committee imposed only a step-therapy requirement to try either metformin or a sulfonylurea before trying exenatide and did adopt a nonspecific requirement for physician reauthorization of refills before the fourth pharmacy claim for exenatide. Disease-based pharmaco-economic models may help third party payers project costs and be particularly useful when only data from short-term clinical trials are available. In the present case, the pharmacy staff of a health plan used a pharmaco-economic model for drug treatment of type 2 diabetes provided by the manufacturer as part of the AMCP Format dossier process to project cost outcomes for exenatide, adjunct injectable therapy for patients taking metformin and/or sulfonylurea. The P&T committee approved the drug for inclusion in the drug formulary based in part on the results of the pharmaco-economic model produced from the cost inputs entered into the model by the health plan pharmacists.

The contribution of medical co-morbidity to the risk of low birth weight and prematurity in blacks

WEIGHT AND PREMATURITY IN BLACKS DEBORAH EHRENTHAL, CLAUDINE JURKOVITZ, MATTHEW HOFFMAN, JAMES BOWEN, BARBARA TEMPLE, MILTON GOTTSCHALL, WILLIAM WEINTRAUB, Christiana Care Health System, Obstetrics and Gynecology, Newark, Delaware, Christiana Care Health System, Center for Outcomes Research, Newark, Delaware, Christiana Care Health System, Information Technology, Newark, Delaware OBJECTIVE: To test the hypothesis that the higher prevalence of medical comorbidities among black women accounts for the increased risk of low birth weight and prematurity in that racial group. STUDY DESIGN: Clinical data from a population-based regional cohort of women receiving obstetrical care at a community hospital between 2003 and 2006 were analyzed using univariate and multivariate analysis. RESULTS: Complete data were available for 18,850 singleton pregnancies: 62% of the mothers were white, 22% black, 10% Hispanic and 4.6% Asian. Low birth weight (LBW, !2500g) was identified in 7.9% of all births: 13% for black, 6.5% for white, 6.3% for Hispanic and 6.0% for Asian populations. The unadjusted odds ratio (OR) for LBW among blacks was 2.2 (1.9-2.4). Prevalence of BMI!18.5, obesity, chronic hypertension (HTN), pregnancy induced hypertension (PIH), diabetes (DM), gestational diabetes (GDM), asthma (AST) and smoking (SMK) was determined and stratified by race (Table). PIH (OR= 3.7 (3.2-4.2)), CHTN (OR= 2.4 (1.9-3.1)), black race (OR=2.2 (1.9-2.5)), DM (OR=2.1 (1.3-3.4)), smoking (OR=1.8 (1.6-2.0)), age !20 (OR=1.3 (1.1-1.5)) and BMI !18.5 (OR=1.6 (1.3-2.1)) were identified as independent risk factors for LBW in a logistic regression model. No statistically significant increase in risk was associated with asthma, obesity, or GDM. Controlling for the presence of these medical co-morbidities did not attenuate the elevated risk associated with black race. This held true for other adverse outcomes including the risk of very low birthweight (!1500g) and prematurity. CONCLUSION: These findings suggest that though chronic medical problems are more common among blacks, they do not explain the increased risk of low birth weight and prematurity seen in this group.

Bringing Prostate Cancer Quality of Life Research Back to the Bedside: Translating Numbers Into a Format That Patients can Understand

Although measuring quality of life of patients with prostate cancer serves important research goals, its primary clinical purpose is informing patients. Sophisticated quality of life measures produce purely numerical results that patients have difficulty understanding. We present an approach that preserves the methodological strengths of validated multi-item measures but provides more accessible information for clinical use. Using validated indexes measuring urinary, bowel and sexual function we surveyed patients with clinically localized prostate cancer before treatment and at intervals thereafter. Based on patient responses to parallel distress measures we defined 3 levels of function, including normal-no abnormal symptom, intermediate-any abnormal symptom but none severely abnormal and poor-any severely abnormal symptom. We then translated patient survey results into these levels. To assess measurement properties we compared average symptom distress scores in patients at each symptom level. Levels of function and patient distress scores correlated strongly. Large and approximately equal differences in distress scores separated patients at successive levels in all symptom indexes (effect size greater than 1.2, p < 0.0001). Using these categories we created tables showing 24-month outcomes in 417 previously reported patients by pretreatment symptom level and treatment, providing a tool for patients to determine posttreatment outcomes in similar patients. Using symptom indexes to define levels of function produces a quality of life metric that is valid, defines quantitative intervals, is transparent and may be more useful to patients. This approach provides methodologically sound outcome information to patients attempting to choose a prostate cancer treatment.

Cost-effectiveness of basal insulin from a US health system perspective: Comparative analyses of detemir, glargine, and NPH

The purpose of this study was to compare in clinical and economic terms the long-acting insulin analogue detemir with intermediate-acting Neutral Protamine Hagedorn (NPH) insulin and with long-acting insulin glargine. Investigators used the validated Center for Outcomes Research (CORE) Diabetes Model to project clinical and cost outcomes over a 35-year base case time horizon; outcome data were extracted directly from randomized, controlled trials designed to compare detemir with NPH and with insulin glargine. Modeled patient characteristics were derived from corresponding trials, and simulations incorporated published quality-of-life utilities with cost data obtained from a Medicare perspective. Detemir, when compared with NPH, increased quality-adjusted life expectancy by 0.698 quality-adjusted life-years (QALYs). Lifetime direct medical costs were increased by 10,451 dollars per patient, although indirect costs were reduced by 4688 dollars. On the basis of direct costs, the cost per QALY gained with detemir was 14,974 dollars. In comparison with glargine, detemir increased quality-adjusted life expectancy by 0.063 QALYs, reduced direct medical costs by 2072 dollars per patient, and decreased indirect costs by 3103 dollars (dominant). Reductions in diabetes-related comorbidities were also associated with detemir in both instances, most notably in the complications of retinopathy and nephropathy. Relative reductions in rates of complications were greatest in the comparison of detemir with NPH. Results were most sensitive to variation in hemoglobin A1c (HbA1c) levels. However, variation among any of the key assumptions, including HbA1c, did not alter the relative results. Detemir represents an attractive clinical and economic intervention in the US health care setting compared with both NPH insulin and insulin glargine.

Development of an integrated stroke outcomes database within Veterans Health Administration

A fundamental goal of the Rehabilitation Outcomes Research Center of Excellence is to improve care and outcomes for veterans with rehabilitation needs. To achieve this goal, the Center's primary objective is increasing research capacity. The Integrated Stroke Outcomes Database is a collection of Veterans Health Administration (VHA) clinical and administrative data containing patient information on a cohort of stroke patients found in the Functional Status Outcomes Database (FSOD), National Patient Care Database (NPCD), and other VHA sources. Clinical and administrative data were abstracted from several VHA data sources and linked to form an integrated outcomes database. A primary cohort of stroke patients treated during fiscal year (FY) 2001 was identified from the FSOD. Matching data from the NPCD, Decision Support System, Health Economics Resource Center, and the National Veterans Survey were obtained, merged, and reported in brief. This integrated database structure will provide valuable support to enhance the VHA capacity to perform stroke rehabilitation research.

The cost‐effectiveness of continuous subcutaneous insulin infusion compared with multiple daily injections for the management of diabetes: response to Schuffman and Carr

Diabetic MedicineVolume 21, Issue 12 p. 1372-1372 The cost-effectiveness of continuous subcutaneous insulin infusion compared with multiple daily injections for the management of diabetes: response to Schuffman and Carr A. J. Palmer, A. J. Palmer CORE—Center for Outcomes Research, BaselSearch for more papers by this authorS. Roze, S. Roze CORE—Center for Outcomes Research, BaselSearch for more papers by this authorW. J. Valentine, W. J. Valentine CORE—Center for Outcomes Research, BaselSearch for more papers by this authorG. Spinas, G. Spinas Zürich Universtiy Hospital, Zürich, SwitzerlandSearch for more papers by this author A. J. Palmer, A. J. Palmer CORE—Center for Outcomes Research, BaselSearch for more papers by this authorS. Roze, S. Roze CORE—Center for Outcomes Research, BaselSearch for more papers by this authorW. J. Valentine, W. J. Valentine CORE—Center for Outcomes Research, BaselSearch for more papers by this authorG. Spinas, G. Spinas Zürich Universtiy Hospital, Zürich, SwitzerlandSearch for more papers by this author First published: 24 November 2004 https://doi.org/10.1111/j.1464-5491.2004.1349a.xCitations: 3Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onFacebookTwitterLinked InRedditWechat No abstract is available for this article.Citing Literature Volume21, Issue12December 2004Pages 1372-1372 RelatedInformation