Perspective
The Patient-Centered Outcomes Research Institute — Promoting Better Information, Decisions, and Health
N Engl J Med 2011; 365:e31October 13, 2011
Comments
open through October 19, 2011
- Article
Within the 2000 pages of the 2010 Patient Protection and Affordable Care Act (ACA) is a short section authorizing the creation of the Patient-Centered Outcomes Research Institute (PCORI) — a research organization dedicated to the support and promotion of comparative clinical effectiveness research. The establishment of PCORI represents the culmination of long-standing interest in comparative effectiveness research, a lengthy legislative gestation, and compromise among varied congressional perspectives and priorities.1 PCORI responds to a widespread concern that, in many cases, patients and their health care providers, families, and caregivers do not have the information they need to make choices aligned with their desired health outcomes.
PCORI funding is set at a total of $210 million for the first 3 years and increases to approximately $350 million in 2013 and $500 million annually from 2014 through 2019. With more than $3 billion to spend between now and the end of the decade, PCORI will support many studies encompassing a broad range of study designs and outcomes that are relevant to patients, aiming to assist people in making choices that are consistent with their values, preferences, and goals.
By statute, PCORI is an independent, not-for-profit, private entity. Its independence and governance structure are meant to ensure that it represents the perspectives of the broader health care community, operates with predictable funding, and establishes itself as a credible, trusted source of information for patients, caregivers, and providers. To facilitate efficient use of the new funding, Congress directed PCORI to collaborate with existing federal agencies that engage in outcomes research, particularly the Agency for Healthcare Research and Quality (AHRQ) and the National Institutes of Health (NIH).
PCORI's board of governors, appointed last September by the Government Accountability Office (GAO), includes patients and health care consumers, nurses, physicians, and health services researchers, as well as representatives of integrative health care; hospitals and health systems; health plans; self-insured employers; the pharmaceutical, device, and diagnostic industries; the Veterans Health Administration; and state government. The board also includes the heads of the AHRQ and the NIH. In addition, PCORI has a methodology committee, whose 17 members were appointed by the GAO last January. This committee — which has methodologic expertise in fields including epidemiology, health services research, clinical research, biostatistics, genomics, and research methods — is charged with defining rigorous, high-integrity standards and methods to strengthen the science underpinning patient-centered outcomes research. The board has appointed as executive director Dr. Joe Selby, a family physician with expertise in clinical epidemiology and health services research and a history of leading and conducting research informed by the needs of patients and clinicians.
Since being impaneled, the board has worked to ensure transparency, credibility, and access, holding open board meetings every other month in Washington, D.C., Los Angeles, St. Louis, New York, and Seattle (meeting webcasts and summaries are available at www.pcori.org). These meetings have focused on strengthening the institute's governance, establishing an initial organizational framework, developing a consensus on PCORI's mission, and delineating processes for proposing national research priorities, establishing a research agenda, and producing a methodology report. Ample time is allotted for public comment at each meeting.
The board has adopted the following mission statement: “PCORI helps people make informed healthcare decisions, and improves healthcare delivery and outcomes, by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader healthcare community.” These sources of guidance and the involvement of stakeholders in all phases of research are not only distinguishing characteristics of PCORI's mission — they are also essential to broad acceptance of the evidence-based information the research is intended to produce. As one example of how we are pursuing our mission, PCORI board meetings include roundtable discussions with myriad stakeholders, including patients and patient advocates, clinicians, researchers, and industry representatives, to explore their perceptions of the meaning of “patient-centered outcomes research,” develop a deeper understanding of outcomes valued by patients, and establish an inventory of the sources to which patients turn for health information. The forums in St. Louis and New York each drew about 100 participants. In Washington, board and methodology committee members met with patients and caregivers and fanned out in small teams across the District of Columbia to meet with 42 organizations representing nursing disciplines, medical specialties, medical technology and research fields, and patient-advocacy groups. In Seattle, the board spent an evening with representatives from health service organizations, health care providers, and comparative effectiveness researchers engaged in collaborations across the Pacific Northwest.
The institute has also launched a speakers' bureau, making governors available to participate in national and local meetings and professional conferences with patients, clinicians, caregivers, researchers, industry representatives, health care purchasers, and policymakers. The goal of these communication and outreach activities is to promote the inclusion of all voices in the conversation about providing better information to patients and the people who care for them.
One of the principle duties of PCORI is to identify national priorities for research. In addition to engaging patients and other stakeholders, the priority-setting process will include a set of landscape reviews, pilot projects, and conference grants. The board's work is guided by four questions asked from a patient's perspective:
“Given my personal characteristics, conditions, and preferences, what should I expect will happen to me?”
“What are my options, and what are the benefits and harms of those options?”
“What can I do to improve the outcomes that are most important to me?”
“How can the health care system improve my chances of achieving the outcomes that I prefer?”
To provide answers, the institute will conduct outcomes research assessing the benefits and harms of preventive, diagnostic, therapeutic, or delivery-system interventions, highlighting comparisons and outcomes that people care about and experience, such as survival, function, symptoms, and health-related quality of life. People's life circumstances vary, as do their biology, lifestyles, behaviors, and other important factors affecting health status. PCORI-sponsored research will produce information tailored to diverse settings and individuals to capture the attributes of a broad range of patients.
Our landscape reviews will assess the country's current capacity for conducting patient-centered outcomes research and what is currently known about engaging patients and other stakeholders in the research process, including the dissemination of findings. PCORI's pilot projects (announced on September 28, 2011) will focus on developing, testing, and evaluating novel approaches that inform the national priorities; bring together various stakeholders; translate research into practice; identify gaps in evidence that most affect vulnerable populations; examine the prognostic information available to patients and providers; and elucidate behaviors, lifestyles, and choices within patients' control that may affect their outcomes. The PCORI conference grants are aimed at bringing together diverse perspectives to address discrete problems related to engaging patients in research and health care decisions, creating a patient-centered research agenda, and disseminating results effectively.
Concurrently, PCORI's methodology committee will review and summarize state-of-the-art methods and standards for the conduct of patient-centered outcomes research. This work includes a detailed assessment of methods for patient engagement; establishment of research priorities; use of clinical data systems, including those covering usual practice settings; study design; and statistical analysis. Once national priorities have been drafted, vetted with the public, and approved by the board, a research agenda will be developed. We expect this agenda to deploy a full research arsenal, including randomized trials, observational outcomes studies based in registries or databases derived primarily from clinical care, and data syntheses.
By statute, 20% of the institute's funding is provided to the secretary of health and human services — most of it for the AHRQ, to support dissemination of relevant research findings to patients, providers, and other stakeholders, as well as for training and career development in comparative clinical effectiveness research. The importance of PCORI's work in the communication, dissemination, and uptake of its research findings cannot be overstated.
Most important, as we mark PCORI's first anniversary this month, the institute is poised to lead a national movement that meaningfully involves patients and incorporates their voices in producing trusted, evidence-based information, promoting better decisions and ultimately better health for all.
Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.
This article (10.1056/NEJMp1109407) was published on September 28, 2011, at NEJM.org.
Source Information
Dr. Washington is the chair, and Mr. Lipstein the vice-chair, of the board of governors of the Patient-Centered Outcomes Research Institute. Other members of the board include Debra Barksdale, Ph.D., R.N., Kerry Barnett, J.D., Lawrence Becker, Carolyn Clancy, M.D., Francis Collins, M.D., Ph.D., Leah Hole-Curry, J.D., Allen Douma, M.D., Arnold Epstein, M.D., Christine Goertz, D.C., Ph.D., Gail Hunt, Robert Jesse, M.D., Ph.D., Harlan Krumholz, M.D., Richard E. Kuntz, M.D., Sharon Levine, M.D., Freda Lewis-Hall, M.D., Grayson Norquist, M.D., M.S.P.H., Ellen Sigal, Ph.D., Harlan Weisman, M.D., and Robert Zwolak, M.D., Ph.D. Methodology Committee members include Sherine Gabriel, M.D. (chair), Sharon-Lise Normand, Ph.D. (vice-chair), Naomi Aronson, Ph.D., Ethan Basch, M.D., Alfred O. Berg, M.D., M.P.H., David Flum, M.D., M.P.H., Steven Goodman, M.D., Ph.D., Mark Helfand, M.D., M.P.H., John Ioannidis, M.D., D.Sc., Michael S. Lauer, M.D., David O. Meltzer M.D., Ph.D., Brian S. Mittman, Ph.D., Robin Newhouse, Ph.D., R.N., Sebastian Schneeweiss, M.D., Sc.D., Jean R. Slutsky, P.A., M.S.P.H., Mary Tinetti, M.D., and Clyde Yancy, M.D.
- References
References
1
Wilensky GR . The policies and politics of creating a comparative clinical effectiveness research center. Health Aff (Millwood) 2009;28:w719-w729
CrossRef | Web of Science | Medline
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Muin J. Khoury, Marta L. Gwinn, Russell E. Glasgow, Barnett S. Kramer. (2012) A Population Approach to Precision Medicine. American Journal of Preventive Medicine 42:6, 639-645
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Valerie G. Press, Vineet M. Arora, Lisa M. Shah, Stephanie L. Lewis, Jeffery Charbeneau, Edward T. Naureckas, Jerry A. Krishnan. (2012) Teaching the Use of Respiratory Inhalers to Hospitalized Patients with Asthma or COPD: a Randomized Trial. Journal of General Internal Medicine
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Mary Norine Walsh, Alfred A. Bove, Russell R. Cross, Keith C. Ferdinand, Daniel E. Forman, Andrew M. Freeman, Suzanne Hughes, Elizabeth Klodas, Michelle Koplan, William R. Lewis, Brian MacDonnell, David C. May, Joseph V. Messer, Susan J. Pressler, Mark L. Sanz, John A. Spertus, Sarah A. Spinler, Louis Evan Teichholz, John B. Wong, Katherine Doermann Byrd. (2012) ACCF 2012 Health Policy Statement on Patient-Centered Care in Cardiovascular Medicine. Journal of the American College of Cardiology
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Moshe Vardi, Andrew P. Levy. (2012) Is It Time to Screen for the Haptoglobin Genotype to Assess the Cardiovascular Risk Profile and Vitamin E Therapy Responsiveness in Patients with Diabetes?. Current Diabetes Reports
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Tom Walley. (2012) Translating Comparative Effectiveness Research into Clinical Practice. Drugs 72:2, 163-170
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- Comments (9)
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9 Reader's Comments
JAMES RICKERT, MD | Physician | Disclosure: NoneBLOOMINGTON INOctober 13, 2011Patient Centered Care
Can be a great thing. Lots of data show patients don't view their care in the same way docs think they do.
BRIAN KOFFMAN, MD | Physician | Disclosure: NoneDIAMOND BAR CAOctober 12, 2011Brian Koffman / Physician
PCORI has noble ambitions and is clearly making efforts to understand how to make research meaningful at the patient level. I hope the unique demands of each individual patient don't get lost in the need to produce a broad evidence based consensus. I hope the therapeutic role of the relationship between healthcare provider and patient remains part of the equation. I hope that PCORI sees fit to have representation at the board level of all community based primary care providers that are most often the patient's first and defining chance to ask the questions that PCORI says it wants to help answer.
DR LINDA BERGTHOLD | Other | Disclosure: NoneSANTA CRUZ CAOctober 12, 2011PCORI needs our support
Those of us who are health professionals need to support PCORI now more than ever. The comments to this article reveal a discouraging ignorance about what PCORI is, does, and can do for patients and physicians. The latest controversy over prostate cancer screening shows that the public (and the politicians who say they speak for the public) does not understand how science can help patients and their doctors make better decisions. Patients are at the core of the work of PCORI and if those of us who support PCORI do not begin to stand up for this work, we will not have the kind of research we need to help each other make the important treatment decisions. The politicization of PCORI's work and the outright lies about its mission by some politicians is disgraceful. The rest of the world looks at us in stunned disbelief. The best thing we can do is let our representatives know of our support and get our colleagues to do the same.
Richard Longland | Other | Disclosure: NoneOctober 03, 2011Faulty Assumptions, Redundancy, Inefficiency
Sadly, this $3 billion represents “business as usual” in terms of how outdated policies are perpetuated. The first two comments speak to obvious redundancies and missed opportunities.
I am concerned with fundamental flaws in the guiding questions of this program: why are studies assuming that patients are already sick and in need of treatment? Why not determine why millions of Americans are chronically ill and getting worse each day? Our preconceptions of degenerative disease lead to palliation or surgery rather than to a true cure. Truly healing therapies are based on finding root causes of medical conditions and many diagnostics and treatments are already available.
Future studies under the auspices of “patient-guided” research will be tightly controlled, regulated and disseminated. In reality, thousands of formal and informal studies have been executed via non-profits in the Americas. I implemented a SF-36 survey to spine patients (with additional questions) and was shocked with the quality of anecdotal data I collected.
In short, patients’ degenerative diseases are getting worse. Insurance companies and government are only contributing to the problem – not the solution.Fauzia Khan | Physician | Disclosure: NoneOctober 01, 2011An Equitable Health Care System
The patient centered outcomes research is a great initiative, and its mission to evaluate the impact of different therapies is right on. As long as it pays attention to factors such as socioeconomic status, gender, age, and ethnicity, it will help us move toward a more equitable and just health care system.
Louis Zrebiec, Ph.D. | Other | Disclosure: NoneSeptember 30, 2011Outcomes Within Context
Outcomes in PCORI must appreciate the whole patient (i.e., physiology) and his/her cultural context (e.g., socio-economic level). Checklists and protocols must be presented. Outcomes must be rooted in medical integrity.
Outcomes must present general observations. Such observations may facilitate diagnosis.
Patient reported problems and complications are critical.
Patient contribution to care is critical.JAMES GAULT | Student | Disclosure: NoneCHICAGO ILSeptember 29, 2011Does PCORI overlap AHRQ?
There seems to be substantial overlap between PCORI and AHRQ. If so, taxpayers should ask why they're paying twice for the same research mission.
DR COLIN TAYLOR, MD | Physician | Disclosure: NoneNEW YORK NYSeptember 28, 2011Patient-Centered Internet Questionnaires
This PCOR Perspective discusses "engaging patients … in the research process.” A collaborative study with UCLA and the NIH Center for Computational Biology has recruited 278,854 knee pain people to date; logistic regression analysis identified strong diagnostic predictors; patients receive an immediate on-line screening knee pain report to take to their physician with diagnostic estimates and estimated responses to various treatments. Patient-centered Internet surveys have limitations but can also generate large, clinically useful, databases very quickly.
waiting missed opportunities forever
We have shared with our patients,since 1985 at the Metabolic Care Center, an extensive patient centered research for years.We used computational real life user friendly applications which could be applied for managing most chronic diseases like Diabetes,Obesity,Hypertension,and the Metabolic Syndrome spectrum, but were not able to convince the big stick holders of responding in meaningful way. Not the government,executive, legislative or any branch. Not the big or small insurance industry executives. Not even professional societies,medical organisations,or academic centers.
Unfortunately decisions in health are often based on politics not evidence. We hope this time the interest is genuine ,the support is real and the goal is clear .