Evidence-based research is a large focus of the recently enacted health care reform legislation. As part of this focus, the Patient Protection and Affordable Care Act calls for creation of a Patient-Centered Outcomes Research Institute, or PCORI, to help patients, payers, physicians and other stakeholders make medical decisions by providing them with evidence-based research.
During the next few years, this 19-member institute could establish itself as the nation's leading source for evidence-based research. This could put the PCORI in a position to influence coverage, and possibly even treatment, decisions, according to analysts interviewed by AAFP News Now.
The PCORI will operate as an independent nongovernmental body, encompassing both the public and private sectors. It will focus on evidenced-based research, which is defined as research that evaluates and compares health outcomes and the clinical effectiveness, risks and benefits of two or more medical treatments or services. This also is known as comparative effectiveness research. In time, the institute should be able to help identify what treatments work best for which populations and under what conditions, say experts in this area.
"This is going to be a staged process and not something that produces immediate effects," said Gail Wilensky, Ph.D., an economist and former administrator of the Health Care Financing Administration -- now known as CMS -- under former President George H.W. Bush. "I would like to see the institute better disseminate what is known about alternative ways to treat various medical conditions as well as make significant investments in looking at different ways to treat medical conditions that are not funded adequately."
The institute will carry out a variety of functions, including
- identifying national priorities for research by taking into account factors of disease incidence, prevalence and burden in the United States;
- establishing a 15-member methodology committee to develop and periodically update scientifically based methodological standards for research conducted through the institute;
- funding and conducting research with federal agencies, academic centers and institutions, and other entities;
- disseminating findings to all stakeholders in an understandable manner; and
- ensuring transparency by requiring public input.
The PCORI will be governed by a board of directors, including the director of the Agency for Healthcare Research and Quality, the director of the NIH, and 17 additional members representing a broad section of public and private interests. These 17 members will include physicians, patients, health care consumers, private payers and pharmaceutical representatives, as well as state and federal officials. The Government Accountability Office is set to appoint the 17 members by Oct. 1.
The AAFP is a long-time supporter of evidence-based research and was one of the founding members of the Alliance for Better Health Care(www.chsr.org), a broad-based coalition formed to advocate on behalf of sound evidence to improve the medical decision-making process.
As part of the Academy's evidence-based research activities, Ted Epperly, M.D., of Boise, Idaho, who was AAFP president at the time, testified before the Institute of Medicine, or IOM, in 2009 on the importance of evidence-based research in improving quality, reducing costs and enhancing access to care in the United States.
Epperly stressed how important comparative effectiveness research was to AAFP members because family physicians see patients with common problems, and evidence-based research on these problems often is lacking. At that time, the IOM was gathering information from the AAFP and others for a report on research-based spending priorities. The IOM report, which was issued in June 2009, is expected to influence much of the Patient-Centered Outcomes Research Institute's work.
In addition, the AAFP has nominated several physicians for the institute's governing board.
Although the Patient Protection and Affordable Care Act authorizes $10 million in funding for the institute in 2010 and $50 million in 2011, with more increases slated for future years, Congress still must appropriate money so the institute can begin its work.
The institute will be required to operate as an informational entity, and it will be prohibited by law from mandating coverage or payment requirements for public and private plans. However, the institute's recommendations are expected to carry a great deal of weight, influencing insurance coverage decisions and ultimately filtering down to the patient-physician relationship. One of the major roles of the institute is to act as a central resource for gathering and disseminating information in a way that can be understood by clinicians as well as the general public.
"The institute is meant to be a kind of coordinating function for how the federal government, as well as the private sector, prioritizes (comparative effectiveness research)," said Kavita Patel, M.D., director of the health policy program for the New America Foundation in Washington. "What we have right now is a lot of disparate entities -- including the federal government -- doing (comparative effectiveness research) in a lot of different places. This is designed to try and have a focused agenda."
The institute also should bring more uniformity to the way public and private entities conduct and evaluate evidence-based research, which would strengthen and add credence to recommendations issued by the institute. This should help reduce unwarranted diagnostic and practice variations and make physicians' jobs easier, said Wilensky.
"I think (the institute) has the potential of helping physicians and patients in learning how to treat and be treated in a smarter way and to help us as a country learn how to spend smarter, as well," she said.
For example, according to Wilensky, health care spending has grown approximately twice as fast as the economy for the past 40 years, a pace that jeopardizes the nation's fiscal health. "It does not make any sense," said Wilensky. "We do not have the kind of health outcomes to suggest that (spending) has been an appropriate differential."
Establishing the PCORI fits in with attempts to make care and treatment more patient-focused and evidenced-based, especially within the context of the recently enacted Patient Protection and Affordable Care Act, according to Len Nichols, Ph.D., director of the Center for Health Policy Research and Ethics and professor of health policy for the College of Health and Human Services at George Mason University in Fairfax, Va.
"I would not want to go forward in a serious effort to improve our health care system without a significant investment in best practice research and in the dissemination of that research," said Nichols.
The institute will attempt to build on comparative effectiveness research investments made by the American Recovery and Reinvestment Act of 2009, which directed the Institute of Medicine to issue a list of 100 priority topics for comparative effectiveness research. That list, which was issued in June 2009, could influence much of the PCORI's work.
Wilensky, meanwhile, said the institute must accomplish a few key tasks to be successful. It must look at alternative ways to treat major medical conditions so the United States can stop spending a lot of money on a variety of treatments, not all of which lead to better-quality health care.
In addition, the institute must disseminate existing information on health care treatments and generate new information about what works best under what circumstances, said Wilensky.
She is aware that some stakeholders may oppose establishing the institute because they believe it will generate information that could lead to health care rationing. However, Wilensky said the institute could help disseminate better information and better incentives. "Could we misuse the information? Sure," she said. "We could misuse any information, but we can make bad decisions even without information."