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ABSTRACT Background: The extent to which patient-based outcomes can be used to evaluate and communicate the effect of new drugs and devices is a subject of much debate. Criteria for evaluating the scientific quality of data to support health-related quality of life (HRQL) and other patient-based labeling and promotional claims in the United States and Europe have been proposed by various scientists and organizations. Since March 2000, a working group composed of members of the International Society for Quality of Life Research (ISOQOL), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Pharmaceutical Manufacturer's Association Health Outcomes Committee (PhRMA-HOC), and the European Regulatory Issues on Quality of Life Assessment (ERIQA) met to discuss and coordinate the various recommendations by their respective groups and address the need to harmonize outcomes review criteria within and across United States and European regulatory agencies. Over time, the discussion expanded from HRQL outcomes to include any outcome based on data provided by the patient or patient proxy, that is, patient-reported outcomes (PROs). The working group therefore became known as the PRO Harmonization Group. Methods: Working with a member of the US Food and Drug Administration (FDA), four key issues requiring clarification were identified: how PROs are defined and put into operation for research purposes; the added value of PROs in the drug review and evaluation process; selected questions related to the PRO measurement and research methodology; and the interest and demand for PRO information by decision makers. On February 15, 2001, all members of the PRO Harmonization Group attended a meeting in Rockville, Maryland, to discuss these four issues further, and on February 16, 2001, a formal presentation was made to representatives from various departments and reviewing divisions of the FDA. These presentations are summarized in this report. Results: All participants agreed that PROs are important for understanding the impact of treatment on patient functioning and well-being. They also stressed the need to communicate PRO information to key decision makers, including regulatory agencies, clinicians, patients and their families, and payers. Finally, the meeting resulted in plans for continuing the dialogue on PRO measurement and interpretation. Summary: The February 16, 2001, meeting represented an important step in harmonizing efforts across various organizations and in opening a dialogue with the FDA around major issues related to methodologic standards for measuring and interpreting PROs in the drug evaluation process. [ABSTRACT FROM AUTHOR]

Findings from health services research highlight continuing health care disparities in the United States, especially in the areas of access to health care and quality of care. Although attention to health care disparities has increased, considerable knowledge gaps still exist. A better understanding of how cultural, behavioral, and health system factors converge and contribute to unequal access and differential care is needed. Research-informed approaches for reducing health care disparities that are feasible and capable of sustained implementation are needed to inform policymakers. More important, for health equity to be achieved, it is essential to create a health care system that provides access, removes barriers to care, and provides equally effective treatment to all persons living in the United States. [ABSTRACT FROM AUTHOR]

The US President's Emergency Plan for AIDS Relief (PEPFAR) has supported the extension of HIV care and treatment to 2.4 million individuals as of September 2009. With increasing resources targeted toward rapid scale-up, it is important to understand the characteristics of current PEPFAR-supported HIV care and treatment sites.Forty-five sites in Botswana, Ethiopia, Nigeria, Uganda, and Vietnam were sampled. Data were collected retrospectively from successive 6-month periods through reviews of facility records and interviews with site personnel between April 2006 and March 2007. Facility size and scale-up rate, patient characteristics, staffing models, clinical and laboratory monitoring, and intervention mix were compared.Sites added a median of 293 patients per quarter. By the evaluation's end, sites supported a median of 1649 HIV patients, 922 of them receiving antiretroviral therapy. Patients were predominantly adult (97.4%), and the majority (96.5%) were receiving regimens based on nonnucleoside reverse transcriptase inhibitors. The ratios of physicians to patients dropped substantially as sites matured. Antiretroviral therapy patients were commonly seen monthly or quarterly for clinical and laboratory monitoring, with CD4 counts being taken at 6-month intervals. One-third of sites provided viral load testing. Cotrimoxazole prophylaxis was the most prevalent supportive service.HIV treatment sites scaled up rapidly with the influx of resources and technical support through PEPFAR, providing complex health services to progressively expanding patient cohorts. Human resources are stretched thin, and delivery models and intervention mix differ widely between sites. Ongoing research is needed to identify best-practice service delivery models. [ABSTRACT FROM AUTHOR]