Direction of Comparative Effectiveness Research in the United States and the Role of AHRQ

Keynote Address by Carolyn M. Clancy, M.D., Director, Agency for Healthcare Research and Quality (AHRQ)

China Outcomes Research & Evidence-Based Medicine Summit
Shanghai, China (Via Videoconference), March 29, 2012

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Good morning, everybody! Greetings from the United States. It's 10 o'clock at night here in Rockville, MD, about 20 miles from the Nation's capital. And I am very happy to have the opportunity to speak with you during the opening of the China Outcomes Research & Evidence-Based Medicine Summit in Shanghai.

I would have loved nothing better than to join you there at the Shanghai International Convention Center—to hear your thoughts and ideas about how we can better apply the science to patient care. Unfortunately, that was not possible, and I appreciate your willingness to let me "beam in," so to speak, to talk about the direction of comparative effectiveness research—or what we're calling patient-centered outcomes research—in the United States, as well as my Agency's role in putting patients at the center of care.

But before I begin, I want to say something about this Summit.  As director of the first Federal Agency in the United States with a legislative mandate to have a center for patient-centered outcomes research, I must tell you, I think this is fantastic.

It's very gratifying to see that you're looking to make the most of the growing interest in patient-centered outcomes research in China. We're doing the same thing here, and doing this is key to building the type of health care system that uses all of the available tools to put patients first.

In reviewing the literature for this event, we came across a list of trends in health care that you have identified in China. According to your observations—

• Clinical experts are demanding more data on comparative effectiveness studies and patient-reported outcomes studies.
• National and commercial health insurance organizations are increasingly demanding evidence of medical pathways in the process of reimbursement.
• The China Ministry of Health and State Food and Drug Administration in China are strengthening postmarket safety monitoring of medical products.
• Most health professionals in China have accepted the concept of evidence-based medicine, and are demanding more data on the effectiveness and safety of medical products used by Chinese patients.

These trends are the same or similar to those we are seeing in the United States. For example—

• There is not enough credible, empirically based, patient-centered outcomes research available to help patients, providers, payers, and policymakers make informed treatment decisions.
• The Federal Government has provided us with the resources to initiate the process of building a health care system in which the lag between data acquisition and exploration can be measured in weeks or days, and not months or years, as is currently often the case.
• And health professionals want more data that can help them make better decisions.

I'm the oldest of seven children. When we were growing up, and my brothers would come up with a clever plan for something, they would call it "Management Thinking." Actually they would say "MT" because that was their short-hand for management thinking.

It's the same way we think today about systems intervention. We've acted as if it's something that's really easy. We put out all of the science, and we expect that it's magically going to trickle down to the point of care perfectly and seamlessly. Of course, we have a lot of evidence that it doesn't quite happen that way.

Also contributing to this phenomenon is the fact that health care traditionally has been disease-centered, with physicians making almost all treatment decisions based largely on clinical experience and data from various medical tests.

In the patient-centered model, patients are more active participants in their own care, and interactions between diseases are important. They receive services designed to focus on their individual needs and preferences, in addition to advice and counsel from health professionals.

On the practice side, successful uptake and integration of evidence into practice requires an understanding of not only whether something works (clinical efficacy), but also of what works where, when, and how (effectiveness), and for which patients.

At the Agency for Healthcare Research and Quality, or AHRQ, we have been studying this second set of issues for many years, particularly through our practice-based research mechanisms, which enable clinicians, provider organizations, and health services researchers to collaborate on research involving the translation of evidence into practice in diverse settings—from doctors' offices and clinics to large, integrated health care systems.

My predecessor, the late John Eisenberg, used to say that health services research answers the enduring questions, such as—

• What works?
• Under what circumstances?
• For which condition?
• At what cost?

Patient-centered research adds "for which patients?"

Evidence-based medicine can't fix all of the problems. For example, it can't fix controversies due to values or cost; overcome barriers due to misaligned incentives, patient factors and system failures; and it can't ensure or guarantee appropriate application to policy or practice. But it can reduce the chance of getting something wrong; it can help make decisions more consistent, transparent, and rational and reduce the extent of variations in health care that we see over time. Much of what we see in practice variations is a clear indication of where we don't have good evidence.

Variation in care is a topic that is explored in the National Healthcare Quality and National Healthcare Disparities Reports that my Agency publishes every year. Overall, what we routinely see are modest improvements in quality. In last year's report it was 2.3 percent overall. And the quality of care varies, not only by type of care, but also across parts of the country—and with different types of preventive services.

These variations were first revealed most dramatically in a study by Dr. Jack Wennberg that was published about 45 years ago. Using a $350,000 grant awarded by the Lyndon Johnson administration, Dr. Wennberg and his colleague, Alan Gittelsohn, developed a method called Small Area Analysis. They analyzed Medicare data to determine how well hospitals were doing. People generally anticipated that they would find service lacking mainly in rural hospitals.

What the study found instead was tremendous variation in virtually every aspect of health care delivery—including in communities served by academic medical centers. The findings were completely contradictory to the basic premise that medicine was driven by science and by physicians who make clinical decisions based on well-established fact and theory.

Jack's work pretty much provided the blue print for creating AHRQ. And you can also draw a very direct line from this work to patient-centered outcomes research.

Our mission at the Agency is to improve the quality, safety, effectiveness, and efficiency of health care for all Americans. We do this by providing support for independent, user-driven research that is designed to help people and organizations at the national, state, and local levels make a wide range of informed choices.

Patient-centered outcomes research is a key element in my Agency's safety and quality improvement efforts. And in many ways, AHRQ has been at the forefront of much of the activity that has been generated around the research.

Our mandate for conducting patient-centered outcomes research was put forward in the form of our Effective Health Care Program that was created as part of the Medicare Prescription Drug, Improvement, and Modernization Act of 2003, which expanded medication insurance coverage for the elderly and disabled. The program became operational in 2005 and from then until 2009, it received $129 million from the Congress. We have since received unprecedented investments in the research, through passage of the American Recovery and Reinvestment Act of 2009 and the Patient Protection and Affordable Care Act in 2010.

To provide some context: From 2005 to 2009, we developed about 50 products. With increased resources from the two recent laws, we have completed about 50 more products. And, over the next 2 years, we will be adding about 75 more.

Products the Effective Health Care Program develops include—

• Research reviews, such as comparative effectiveness and effectiveness reviews that outline the benefits and harms of treatment options, and technical briefs that explain what is known—and what is not known—about new and emerging health care tests or treatments.
• Original research reports based on clinical research and studies that explore the practical questions about the effectiveness of treatments.
• Short plain-language summaries that are tailored for use by clinicians, consumers, or policymakers.

Many of the summaries for patients are currently available in English and Spanish. And we would be happy to engage in collaborations to make them available in Chinese. Several years ago (1997), we worked with the National Asian Pacific Center on the Aging on two products. One was a smoking cessation pamphlet titled, You Can Quit Smoking, which was published in Chinese, Tagalog, Korean, Laotian, Vietnamese, and Cambodian. The other was the consumer summary, Prescription Medicines and You. It was available in Chinese, Korean, Vietnamese, and Cambodian.

Collaboration like this reminds me of my favorite African proverb: If you want to travel fast, you travel alone. If you want to travel far, travel with others.

Patient-centered outcomes research is about getting beyond our own experiences. It expands our horizons beyond each transaction and opens avenues to learning much, much more about what happens to patients over time. And it is all about science that improves patient care. And while decisions and policies must be made locally, evidence should be shared globally. Collaboration must also be global.

When we share, we become energized. We come up with lots of new ideas about how we can help our patients, and together, we help to turn local successes into strategies that can improve care everywhere. So it's incumbent on all of us to make sure we build in the type of transparency that breeds trust. Trust in how the research is done as well as in the results.

With the Effective Health Care Program, we engage stakeholders—including patients—from the beginning. We partner with networks of researchers and clinical teams across North America. We use input from stakeholders throughout the process of patient-centered outcomes research, translation, dissemination, and implementation of research findings. We have supported a Community Forum to improve and expand public and stakeholder engagement in the research.  This helps ensure that the Program addresses issues that are relevant and important. It also improves the products, making them accessible, user-friendly, and responsive to stakeholder information needs.

Stakeholders can also contribute by—

• Nominating topics for research.
• Commenting on draft research questions and reports.
• Advising on the distribution of patient-centered outcomes research products and findings.

We also have a Recovery Act-funded Stakeholder Group. Its activities include—

• Providing guidance on program implementation.
• Providing input on critical information gaps for practice and policy, along with research methods to address them.
• Championing objectivity, accountability, and transparency.

Health information technology, or health IT, is a very important component in all of this. Another benefit of technology is that it has also changed our mental model of how we're disseminating research. It used to be that practice and development of evidence were very separate. Now, the boundaries between the conduct of research and the applications of that research to patient care are becoming increasingly blurred.

Health IT is providing a means to make this easier to do. It's not the silver or magic bullet—the answer to our dreams—but it does give patients access to more information from around the world. It also means doctors and hospitals and policymakers have access to data that they couldn't see before. And these data are a resource from which we all can benefit. This has been taking place in other countries, including China, for years. A number of countries have done more to engage full-time doctors in collecting data for research.

Health IT makes it easier, in much the same way that technology is allowing me to join you today from across the world (6,500 miles away). Imagine the impact of this kind of regular, widespread, real-time access to information in health care. Think about what the health care system's capabilities would be if technology was as ubiquitous as it is in most other industries.

Our bodies—our lives—are our most precious possessions. How can it be that it's easier for us to buy gas, or do our banking, than it can be to have our medical records transferred to a hospital in an emergency?

The Affordable Care Act authorized the creation of the Patient-Centered Outcomes Research Institute. The nonprofit Institute—

• Sets priorities and coordinates with existing agencies that support patient-centered outcomes research.
• Prohibits findings to be construed as mandates on practice guidelines or coverage decision, and contains patient safeguards.
• Provides funding for AHRQ through its Trust Fund to disseminate research findings, train researchers, and build capacity for patient-centered outcomes research.

In late January, as is required by law, the first draft of the Institute's National Priorities for Research and Research Agenda were released for public comment. The final versions of these documents will be ready in May. They will be used to guide funding announcements for comparative clinical effectiveness research. The comment period ended March 15, 2012. Once the feedback is reviewed and the draft is revised accordingly, it will be brought to the Board for consideration. The first funding announcements will be issued in May of this year.

Applying the science to patient care. That's what it's all about. That's where we start. And that's where we end. That's the goal.

To that end, another area of particular interest to AHRQ—and one in which questions of effectiveness are especially complex—is in research on promising delivery system interventions. And we have issued grants to explore delivery system changes in areas including the patient-centered medical home, public reporting on provider performance, and alternative approaches of coordinating care across care settings.

We've also developed several projects that are designed to expand the awareness and use of AHRQ's evidence-based products, tools, and information. For example—

• The AHRQ Publicity Center is building a national patient-centered outcomes research partnership network to make decision aids and tools available in patient and professional communities.
• Our Academic Detailing program is working to complete face-to-face educational visits to 1,300 primary care providers and 200 health system directors across the country.
• We have also established five Regional Offices (Atlanta, Chicago, Dallas, Denver, New York, and headquarters in Washington, DC) to develop dissemination partnerships with local and regional health care organizations, businesses, unions, supermarkets, consumers, and other organizations.

We'll stop at nothing. Good information has to be impossible to avoid.

We also continue to examine how best to spread evidence-based practices as well as to undertake a wide variety of broad-based, targeted dissemination approaches. One of our grant initiatives supports the spread of patient-centered outcomes research findings through multistakeholder or multisite networks that already exist. One such program is under way right now at Dartmouth College, where a teen mental health screening, diagnosis, and support system is being implemented in 15 rural primary care practices in Vermont and New Hampshire. Among the practices that were in place by June of last year, about 90 teenagers had screened negative. Another 19 were entered into the registry with a positive finding.

Obviously, we are beyond excited about all of this activity taking place in our own backyard, so to speak. But that is not why I'm telling you about it.  My goal in doing this has been to give you a sense of the scale of this endeavor.

At the AHRQ Annual Meeting last September, someone asked me how much the new health reform legislation and investments that followed are changing our agenda. The serious and very real answer has two parts—not at all—and phenomenally.

We're not doing anything terribly different. We've been building these paths step-by-step for a while. What's different is the scale. This is no longer a cottage industry. It cuts across virtually every aspect of health care. And it is a once-in-a-lifetime chance to make the kinds of transformational change in the system that all of us have been talking and writing about for years. And a lot of it has to take place at the front lines of care. We're not talking only about building a better library. This is all about leveraging evidence-based medicine at the bedside more effectively, and making sure that patients understand what their treatment options are so they can work with their health care teams to make the best possible decision for them. But when it comes to figuring out what works, the only thing that we know for sure, is that there is often no real way to know for sure.

With a lot of the patients that we treat, the preeminent metric for whether we're doing more harm than good, especially for patients with chronic illnesses, is how that patient is feeling. There are a growing number of short patient-reported outcomes measures that can be used to see how patients are doing, but they haven't become part of practice to the extent that they need to be. And in many instances, there is nothing complex or magical about the steps that need to be taken to improve care. It could be something as simple as implementing a hand-washing policy.

Lastly, I would again emphasize the need for improving quality to be a team sport—even a global team sport. In a speech announcing the Department of Health and Human Services' new Global Health Strategy in early January, Secretary Sebelius said, "Health is an issue which aligns the interests of the countries around the world."

Health leaders everywhere are trying to solve the same problems: including obesity, chronic disease, rising health care costs, a shortage of primary care providers, and so forth.

Of course, each country has its own unique characteristics, so what works in one may not work in another. But we can—and we must—learn from each other. And right now, we have an enormous opportunity to pull all of this together, so we can achieve the same precision that we're looking to achieve in conducting the research, in actually applying the research to the benefit of individual patients.

Thank you very much.

Current as of April 2012

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Internet Citation:

Direction of Comparative Effectiveness Research in the United States and the Role of AHRQ. Keynote Address: China Outcomes Research & Evidence-Based Medicine Summit by Carolyn Clancy, March 29, 2010. Agency for Healthcare Research and Quality, Rockville, MD. http://www.ahrq.gov/news/sp032912.htm

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