Yesterday, the Electronic Data Methods (EDM) Forum staff from AcademyHealth attended JAMA’s briefing on their new theme issue on comparative effectiveness research (CER). Researchers presented findings from three reports included in this special issue, and representatives from the Patient-Centered Outcomes Research Institute (PCORI) shared the organization’s national priorities for research and initial research agenda. Dr. Phil Fontanarosa, JAMA’s executive editor, opened the briefing by explaining that the special issue aims to illustrate the potential value of CER to inform, advance, and improve health care decisions, and also to communicate the advantages, limitations, and complexities of CER. Collectively, the featured research reports demonstrate the value of conducting “real-world” (i.e., observational) studies with large datasets where traditional control trials may not be practical, timely, or even ethical. The reports address health questions that are common (lung cancer drug regimens), serious (emergency helicopter vs. ground transport for trauma victims), and have alternative treatment options (radiation therapy for prostate cancer). All of these health questions have important clinical and sometimes financial ramifications. A special emphasis is placed on developing rigorous methodology standards to address issues inherent in all observational studies, especially when using large datasets for CER. The research reports used multiple statistical techniques (including propensity score analysis) to adjust data and ensure that patients included in the study are comparable. A theme emerged from all of the presentations: the importance of dissemination of research to help both patients and providers get information to make better health care decisions. Dr. Anne Beal discussed PCORI’s emphasis on dissemination—all new projects funded by PCORI are required to have a dissemination component—as well as the organization’s priorities for making outcomes research patient-centered, focusing on patient engagement and patient-driven research. Those areas of focus emphasize the need to engage patients, caregivers, clinicians, and other stakeholders as CER evolves to improve transparency and benefit health and health care.  

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