Comparative effectiveness research plan seeks input from all sides

Washington -- An independent medical effectiveness research organization has outlined a broad agenda that does not name specific diseases but instead seeks input from patients and other stakeholders on what specific research questions and areas it should pursue.

The Patient-Centered Outcomes Research Institute was created by the health system reform law to advance public knowledge of the comparative effectiveness of different types of medical care. Such research often compares the relative impact of drugs, medical procedures and medical devices on patients with the same conditions.

The institute's draft research priorities agenda, released on Jan. 23, includes studying the effectiveness of health systems, reducing health disparities, spreading awareness of research results and determining the validity of medical effectiveness research methodology. The institute also is funding studies and drafting guidelines for research.

PCORI leaders want feedback from patients, caregivers, clinicians and others on its research agenda. They want to know if the tentative agenda covers key subjects for which more evidence-based information is needed, said Eugene Washington, MD, MSc, PCORI's board chair and dean of the David Geffen School of Medicine at the University of California Los Angeles.

The leaders also want to involve patients in a significant way in the research process, according to PCORI Executive Director Joe Selby, MD, MPH. "This initial agenda does not limit which conditions or treatments will be studied. It is a starting point," he said, adding that 2012 "will be a year of engagement."

The importance of patient input

Setting such a broad, nonspecific agenda is a somewhat passive, yet valid, way to proceed, said Gail Wilensky, PhD, a former Medicare administrator and current senior fellow at Project Hope, an international health advocacy organization.

Dr. Selby said he expects PCORI will refine the draft agenda and identify specific areas and questions that could lead to important advances in knowledge. The institute's leaders did not want to exclude potentially valid research by naming specific diseases or conditions that they would study.

Wilensky said studying variations in care for similar patients could help reduce unnecessary health care spending. One of the entity's priority categories -- improving health care systems -- also identifies an area that needs attention, she said.

PCORI is doing a good job ensuring that its work is transparent, said Dr. Lisa Simpson, MPH, a pediatrician and president and CEO of Academy Health, a health services research organization. Getting patients -- the ultimate beneficiaries of medical effectiveness research -- involved in setting guidelines and laying out questions for such research activities is important, she said.

Research that the institute will sponsor should produce more information about caring better for specific patient populations, such as children, Dr. Simpson said. Many prescription drugs, for instance, are tested only on adults.

PCORI is accepting comments on its draft priorities through March 15. The institute will hold a national forum on Feb. 27 featuring a public webcast.

The research entity received startup funding through the health reform law, but it will be funded mostly by an annual tax on certain health insurance plans. The fee is expected to generate a total of $2.6 billion through 2019, according to the Congressional Budget Office.

Big fish, small research pond

PCORI has the potential to increase significantly the pool of existing medical effectiveness research. As little as 1.5% of medical research funding in 2002 was devoted to outcomes-related studies, including medical effectiveness research. This is according to a review of medical research spending between 1994 and 2004 in the Sept. 28, 2005, issue of The Journal of the American Medical Association.

Drug and medical device companies fund much of existing medical effectiveness research, but these businesses are under no obligation to publish findings that could hurt their bottom lines. Also, pharmaceutical research typically compares a given drug's effectiveness to that of a placebo, not an alternative drug, treatment or device.

Other medical effectiveness research has been conducted by the National Institutes of Health, Dept. of Health and Human Services, and Agency for Healthcare Research and Quality. Each received a share of $1.1 billion in medical effectiveness research funding in the 2009 federal stimulus package.

PCORI also is not the first organization to draft priorities for medical effectiveness research. An Institute of Medicine committee in June 2009 identified 100 research topics that it said were the most deserving of attention after receiving nearly 1,300 suggestions from the public. Dr. Selby also served on this committee, which advised federal agencies on prioritizing their use of stimulus package funding for medical effectiveness research.

Some lawmakers and health policy experts, most of them conservatives, have warned that medical effectiveness research will be used to justify future government health care rationing. However, the language authorizing PCORI prohibits the institute from using research results to create practice guidelines or coverage recommendations.

Ignorance is not bliss when it comes to health care outcomes, Dr. Selby said. "To say we shouldn't have the information is flawed."