Exenatide BID Observational Study (ExOS): results for primary and secondary endpoints of a prospective research study to evaluate the clinical effectiveness of exenatide BID use in patients with type 2 diabetes in a real-world setting.

Objective: The Exenatide BID Observational Study (ExOS) was designed to evaluate the clinical effectiveness of exenatide BID use in patients with type 2 diabetes (T2D) in a real-world clinical practice setting in the United States.

Methods: Patients were enrolled from 74 practice sites from 9/2007 through 1/2009 and followed for 12 months. The primary effectiveness endpoint was achieving or maintaining hemoglobin A1C of ≤7.

The effects of exenatide bid on metabolic control, medication use and hospitalization in patients with type 2 diabetes mellitus in clinical practice: a systematic review.

The objective of this systematic review was to assess the published literature on the effectiveness of exenatide twice daily (exenatide) in clinical practice, specifically its effects on haemoglobin A1c (A1C), fasting glucose (FG), weight, systolic blood pressure (SBP), medication use, hospitalization and cardiovascular disease (CVD) outcomes. A systematic literature search using the MEDLINE database of English language literature published between January 2005 and May 2011 was performed. The review included retrospective or prospective observational studies that included 100 or more patients per treatment group.

The use, safety and cost of bariatric surgery before and after Medicare's national coverage decision.

Objective: To determine the impact of the Centers for Medicare and Medicaid Services' (CMS) bariatric surgery national coverage decision (NCD) on the use, safety, and cost of care CMS beneficiaries.

Background: In February 2006, the CMS issued a NCD restricting reimbursement for bariatric surgery to accredited centers and including coverage for laparoscopic adjustable gastric band (LAGB).

Methods: A pre/postinterrupted time-series cohort study using nationwide Medicare data (2004-2008) evaluating rates of bariatric procedures/100,000 enrollees, 90-day mortality, readmission rate and payments.

Surveillance for waterborne disease outbreaks and other health events associated with recreational water --- United States, 2007--2008.

Problem/condition: Since 1978, CDC, the U.S. Environmental Protection Agency, and the Council of State and Territorial Epidemiologists have collaborated on the Waterborne Disease and Outbreak Surveillance System (WBDOSS) for collecting and reporting data on waterborne disease outbreaks associated with recreational water.

Economic evaluation of everolimus versus sorafenib for the treatment of metastatic renal cell carcinoma after failure of first-line sunitinib.

Background: A recent indirect comparison study showed that sunitinib-refractory metastatic renal cell carcinoma (mRCC) patients treated with everolimus are expected to have improved overall survival outcomes compared to patients treated with sorafenib. This analysis examines the likely cost-effectiveness of everolimus versus sorafenib in this setting from a US payer perspective.

Methods: A Markov model was developed to simulate a cohort of sunitinib-refractory mRCC patients and to estimate the cost per incremental life-years gained (LYG) and quality-adjusted life-years (QALYs) gained.

Cost-effectiveness of risk stratification for preventing type 2 diabetes using a multi-marker diabetes risk score.

Background: Personalized medicine requires diagnostic tests that stratify patients into distinct groups that may differentially benefit from targeted treatment approaches. This study compared the costs and benefits of two approaches for identifying those at high risk of developing type 2 diabetes for entry into a diabetes prevention program. The first approach identified high risk patients using impaired fasting glucose (IFG).

Innovation in diagnostic imaging services: assessing the potential for value-based reimbursement.

Innovation in the field of diagnostic imaging is based primarily on the availability of new and improved equipment that opens the door for new clinical applications. Payments for these imaging procedures are subject to complex Medicare price control schemes, affecting incentives for appropriate use and innovation. Achieving a "dynamically efficient" health care system-one that elicits a socially optimal amount of innovation-requires that innovators be rewarded in relation to the value they add and can demonstrate with evidence.

Global eradication of measles: an epidemiologic and economic evaluation.

Background: Measles remains an important cause of morbidity and mortality in children in developing countries. Due to the success of the measles mortality reduction and elimination efforts thus far, the WHO has raised the question of whether global eradication of measles is economically feasible.

Methods: The cost-effectiveness of various measles mortality reduction and eradication scenarios was evaluated vis-à-vis the current mortality reduction goal in six countries and globally.

Using cost-effectiveness analysis to support research and development portfolio prioritization for product innovations in measles vaccination.

Background: Several potential measles vaccine innovations are in development to address the shortcomings of the current vaccine. Funders need to prioritize their scarce research and development resources. This article demonstrates the usefulness of cost-effectiveness analysis to support these decisions.

Assessing the cost-effectiveness of measles elimination in Uganda: local impact of a global eradication program.

Background: Measles control has succeeded worldwide, and many countries have substantially reduced incidence and mortality. This has led to consideration of the feasibility of measles elimination in Uganda within the context of global eradication. Before an elimination program is initiated, it is important to consider its potential economic impact, including its cost-effectiveness.

Cost effectiveness of a pharmacy-only refill program in a large urban HIV/AIDS clinic in Uganda.

Background: HIV/AIDS clinics in Uganda and other low-income countries face increasing numbers of patients and workforce shortages. We performed a cost-effectiveness analysis comparing a Pharmacy-only Refill Program (PRP), a form of task-shifting, to the Standard of Care (SOC) at a large HIV/AIDS clinic in Uganda, the Infectious Diseases Institute (IDI). The PRP was started to reduce workforce shortages and optimize patient care by substituting pharmacy visits for SOC involving monthly physician visits for accessing antiretroviral medicines.

Can modeling of health outcomes facilitate regulatory decision making? The benefit-risk tradeoff for rosiglitazone in 1999 vs. 2007.

Rosiglitazone was initially approved for type 2 diabetes monotherapy. We tested health-outcomes modeling as an aid to regulatory decision making by quantifying the incremental net benefit (INB) value of rosiglitazone (relative to a comparator), both at the time of first approval (1999) and at the FDA advisory committee review (2007). Using 1999 data, rosiglitazone was projected to provide an additional 0.

Weight-related quality of life, health utility, psychological well-being, and satisfaction with exenatide once weekly compared with sitagliptin or pioglitazone after 26 weeks of treatment.

Objective: To assess change in patient-reported outcomes in subjects with type 2 diabetes treated with exenatide once weekly compared with those treated with sitagliptin or pioglitazone.

Research Design And Methods: In this 26-week randomized, multicenter, double-dummy study, 491 subjects received 2 mg of exenatide once weekly or maximum daily doses of sitagliptin (100 mg) or pioglitazone (45 mg) on a background of metformin. Weight-related quality of life, health utility, psychological well-being, and diabetes treatment satisfaction were assessed at baseline and week 26.

Exenatide bid observational study (ExOS): baseline population characteristics of a prospective research study to evaluate the clinical effectiveness of exenatide bid use in patients with type 2 diabetes in a real-world setting.

Objectives: To describe the Exenatide Observational Study (ExOS) and patients initiating exenatide therapy in a real-world clinical practice setting.

Methods: ExOS is a prospective, single-arm, multicenter, observational study to assess the effectiveness of up to 24 months of exenatide therapy in patients with type 2 diabetes (T2D). Patients with T2D ≥18 years of age, who initiated exenatide therapy, were eligible.

Patient characteristics and medical care costs associated with hypertriglyceridemia.

Hypertriglyceridemia is a lipid abnormality prevalent in 1/3 of the United States adult population. Our objective was to describe the independent contribution of hypertriglyceridemia to medical care costs. Using an observational cohort of 108,324 members of Kaiser Permanente Northwest, we analyzed the electronic medical records of those patients aged ≥18 years who had triglyceride (TG) measurements in 2008 and had been members of Kaiser Permanente Northwest for the entire year.

Pharmaceutical interventions for mitigating an influenza pandemic: modeling the risks and health-economic impacts.

Model-based analyses built on burden-of-disease and cost-effectiveness theory predict that pharmaceutical interventions may efficiently mitigate both the epidemiologic and economic impact of an influenza pandemic. Pharmaceutical interventions typically encompass the application of (pre)pandemic influenza vaccines, other vaccines (notably pneumococcal), antiviral treatments and other drug treatment (e.g.

A flexible approach to evidentiary standards for comparative effectiveness research.

The Patient Protection and Affordable Care Act established a new Patient-Centered Outcomes Research Institute to identify and address research priorities for comparative effectiveness research. Among its many responsibilities, the institute has been charged with setting priorities, developing methodological standards, and communicating research results to decision makers. In this paper we consider how the institute can support the different standards for acceptable evidence used by various government agencies, providers, patients, and other decision makers.

Regulatory benefit-risk assessment and comparative effectiveness research: strangers, bedfellows or strange bedfellows?

Over the past 5 years, we have witnessed growing interest in both comparative effectiveness research (CER) and regulatory benefit-risk assessment (BRA). Both deal with benefits and harms, although at different stages of the product lifecycle. There are growing pressures for a more systematic and quantitative approach to regulatory BRA.

A formal risk-benefit framework for genomic tests: facilitating the appropriate translation of genomics into clinical practice.

Purpose: Evaluation of genomic tests is often challenging because of the lack of direct evidence of clinical benefit compared with usual care and unclear evidence requirements. To address these issues, this study presents a risk-benefit framework for assessing the health-related utility of genomic tests.

Methods: We incorporated approaches from a variety of established fields including decision science, outcomes research, and health technology assessment to develop the framework.

Stakeholder perspectives on a risk-benefit framework for genetic testing.

A key to accelerating the appropriate integration of genomic applications into healthcare in the coming decades will be the ability to assess the tradeoffs between clinical benefits and clinical risks of genetic tests in a timely manner. Several factors limit the ability of stakeholders to achieve this objective, including the lack of direct evidence, the lack of a framework to quantitatively assess risk and benefit, and the lack of a formal analytic approach to assess uncertainty. We propose that a formal, quantitative risk-benefit framework may be particularly useful for assessing genetic tests intended to influence health outcomes, and communicating the potential clinical benefits, harms, and uncertainty to stakeholders.

Economic evaluation of capecitabine as adjuvant or metastatic therapy in colorectal cancer.

Capecitabine, an oral prodrug of 5-fluorouracil, is indicated for adjuvant treatment in patients with Dukes' C colon cancer and for subsequent lines in metastatic colorectal cancer. The aim of this article is to review the literature on the economics of capecitabine for the treatment of colon cancer. A systematic review was conducted to search for articles published from January 2003 to December 2009 that met the inclusion criteria.

The economic value of reducing medication dosing frequency with drug delivery technologies: an evidence assessment.

Objective: To critically evaluate published cost-effectiveness studies of novel drug products requiring less-frequent medication dosing compared to conventional formulations of the same drug substance.

Methods: A search was conducted in the Medline and Embase databases for cost-effectiveness studies published before May 2009 that compared two or more drug delivery technologies formulated with the same active drug substance. The Quality of Health Economic Studies (QHES) grading criteria for cost-effectiveness studies was applied to the selected publications.