Health care policy leaders discuss "comparative effectiveness" research

By Kristen Bole

National experts in health care research and policy will convene on Friday, January 29, to discuss how to take some of the guesswork out of doctors’ treatment decisions, with the goal of improving health care.

The afternoon conference will be hosted by the UCSF Clinical and Translational Science Institute (CTSI) and the San Francisco Coordinating Center, which coordinates multicenter clinical studies. The event will take place from 2:00 p.m. to 5:00 p.m. in the William J. Rutter Center on UCSF Mission Bay Campus, at 1675 Owens Street.

The conference will address the topic of comparative-effectiveness research, a key component of current health-care reform efforts. The research is meant to address the lack of solid evidence comparing current treatments, how that type of information could be generated and compiled, and the obstacles inherent in research that compares two treatments or practices.

“Currently, health care providers often choose among several treatments that have been shown to have some benefit,” said Deborah Grady, MD, MPH, a UCSF professor of medicine and co-director of CTSI. “But there is very little research that compares two or more treatments or systems of medical care to allow us to choose the best.”

While individual medications and devices undergo intense scrutiny for safety and effectiveness, how those different treatments perform in everyday practice is largely unstudied on a national or global basis, Grady said.

As a result, it is difficult to assess whether a given diagnostic test or treatment is either the most appropriate or cost-effective. Without that information, she said, physicians are left to assess for themselves which therapy seems right for a given patient, based on their own observations or those of colleagues, rather than upon scientific data that could show them which treatments work best and for whom.

While this type of information is available for frequent treatments of common diseases, it is often missing in the routine care that affects the majority of patients.

“One important aspect of comparative effectiveness research is that it should be conducted in ‘real world’ settings, with a full range of patients, not just those selected for clinical trials, and involve the typical resources available to physicians and patients,” said Joe Selby, MD, MPH, director of the Kaiser-Permanente Division of Research, which has pioneered much of the comparative-effectiveness research and has improved patient care based on that research. “An important asset for conducting this research is the large clinical databases generated from the delivery of real world care and captured in electronic health records.”

Advances in information technology and electronic health records have provided a vast amount of data that could be used to answer some of these questions. However, using this type of clinical and administrative data can result in errors, such as in comparing treatments between two patient populations in which one set is much sicker before treatment.

Charles McCulloch, PhD, chief of the UCSF Division of Biostatistics, and a leader in the development of new analytical techniques necessary to tackle these problems, will discuss new statistical tools to overcome these challenges.

“Until we develop the tools to make sure that we get the correct answers to those questions, we will never be able to create a health care system that delivers either truly effective care or eliminates therapies that don’t work,” McCulloch said.

The day will include presentations and a panel discussion with key thought-leaders in the field, including Selby and McCullough. Also presenting will be Robert Temple, MD, director of the Office of Medical Policy in the U.S. Food and Drug Administration, who will make recommendations about how this work should be considered by FDA in its decisions.

The conference agenda is available at:

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