The Patient Protection and Affordable Care Act created a new phrase and concept:patient-centered outcomes research. The author of this commentary, who served as a member of the US House of Representatives from California, is also a patient with epilepsy and a patient advocate. He consulted with lawmakers in the passage of the health reform law. Here he draws on his varied experiences to provide perspective on how the law will provide the necessary policy framework for patient-centered comparative effectiveness research. Elements of that framework include the meaningful engagement of multiple stakeholders, most especially patients; acknowledgment of individual differences among patients and the importance of advancing personalized medicine; transparency and opportunities to participate; a broad, clinically focused research agenda; and the need to communicate results appropriately.
My commitment to patient-centered care springs from my personal journey of developing, being diagnosed with, and—many years later—receiving effective treatment for epilepsy. The same motivation to empower the individual and bring marginalized people into the center of society and of health care is responsible for my decision to champion the Americans with Disabilities Act (ADA), which was signed into law in 1990. I have viewed this work as my ministry. Equally, I see the work of health care as a ministry of care for each patient who enters the health care system.
I’ve lived with epilepsy for more than a half-century, waking up every day not knowing if or when I’m going to have another seizure. Unlike many epileptics, I wasn’t born with the disorder. My epilepsy is a result of my having been in an automobile accident when I was fifteen. And it wasn’t until a year after the accident, while I was out milking cows on my family farm in California, that I had my first epileptic seizure.
Residents of the farming town I grew up in during the 1950s had plenty of misinformation and confusion about many diseases and disorders. My parents reflected the prevailing cultural mind-set about epilepsy and did not accept the diagnosis given to them by doctors. Instead, they chose to view my problem as a spiritual one. So instead of having access to current medical information and getting effective treatment, I was kept in the dark about the initial diagnosis. My parents took me to several other doctors and even to three witch doctors, who poured oil and candle wax on me.
Then at seventeen, I knew something strange was going on, but no one explained to me exactly what it was. I went through high school and college continuing to have spells of passing out and still not knowing why this was happening to me.
When I graduated from college, I decided to become a Catholic priest. President John F. Kennedy had just been assassinated, and I decided that I wanted to devote my life to public service. During a routine physical for seminary, the physician explained to me that my symptoms were caused by something called epilepsy.
I was excited. I finally knew what was causing my seizures! And I was crushed when I heard that, according to Canon Law dating back to 400 CE, my condition disqualified me from the priesthood. During this time everything and everyone close to me seemed to turn against me, often out of ignorance about what it actually meant to have epilepsy. I became severely depressed, even suicidal.
I came through this very difficult time with the absolute conviction that I would live my life overcoming my condition rather than being defined by it. It was at this point that I had the opportunity to live and work with the comedian Bob Hope, who taught me that we can practice true ministry in any vocation and who set me on a path toward politics. Little did he know then that his influence would lead me to become a champion of the Americans with Disabilities Act and an advocate for patients.
My experiences as an advocate for patients and people with disabilities, as a former policy maker and as a patient with epilepsy, have imbued in me the belief that patients’ voices should not only be heard but should also be acted upon. More recently, my work for individual empowerment has focused on supporting individual patients in health care and moving toward patient-centered approaches to care. Making sure that we patients are given ample opportunities to be well informed about our medical conditions and health care options is key to achieving patient-centered health care. My commitment to patient-centeredness is what has spurred my involvement in comparative effectiveness research.
Comparative Effectiveness Research
The concept of comparative effectiveness research is not new. The goal of the research is to provide evidence-based information that helps patients and their physicians make care decisions that meet patients’ individual needs and preferences.1 Until now, unfortunately, decisions on research priorities and the communication and use of results have been undertaken by academic researchers working in conjunction with federal research agencies. As a result, support for the research has been undermined by its perceived affiliation with government, a lack of transparency, and the perception—or even an explicit mandate—that containing costs is the primary goal.
I realized early on that comparative effectiveness research, if done in a patient-centered way, can be a strong force in empowering patients to better understand and take charge of their health care. But if the research is not done in a patient-centered way, it can have the opposite effect, leaving treatment options out of reach for people who aren’t like the “average” patient.
Although the concept of federally supported comparative effectiveness research became controversial during the debate on health care reform, there was remarkable breadth of support for the related provisions that were ultimately signed into law. More than one hundred organizations “representing patients, providers, people with disabilities and others”3 were united in support for these provisions. They educated policy makers and the public about the necessary elements of patient-centered comparative effectiveness research.
The Affordable Care Act now creates a new framework for federally supported comparative effectiveness research, with its establishment of the Patient-Centered Outcomes Research Institute. It gives patients a seat at the table alongside other stakeholders, including doctors, researchers, payers, and government agencies. It recognizes that comparative effectiveness research is too important to be controlled by the elites and that it must be shared with patients and health care professionals.
If the health reform law is implemented correctly, the most important stakeholders in health care—patients—will have a key vote in the effort to fill gaps in evidence and identify the most effective treatments. They will have an equal voice in determining what questions researchers will study, and equal input into how research findings will be communicated to the public.
It will be critical that patient advocates be aware of research results and then help translate them into understandable, useful information that can be acted upon.
Patient-Centeredness In A New Context
Of the forty-one times that the phrase patient-centered appears in the Affordable Care Act, most occur in the law’s establishment of the Patient-Centered Outcomes Research Institute. The institute will be a new, independent organization for comparative effectiveness research. Donald Berwick, now administrator of the Centers for Medicare and Medicaid Services, has definedpatient-centered as “the experience (to the extent the informed, individual patient desires it) of transparency, individualization, recognition, respect, dignity, and choice in all matters, without exception, related to one’s person, circumstances, and relationships in health care.”
When the patient is at the center of care, then care is organized around that person and is responsive to his or her needs, preferences, and experience of care. As someone who has spent more time than I would have liked in doctors’ offices and hospitals over the past forty years, I am personally and painfully aware of how often patients’ needs and preferences are ignored.
Correctly implemented, the comparative effectiveness research provisions in the Affordable Care Act can go a long way toward addressing the inadequacies of health care delivery and answering the research questions that matter most to patients and providers. But the research must be patient centered, focused on the needs of patients and providers. The law recognizes not only the importance of strong clinical evidence to support decision making, but also the need to use the research to support doctors’ and patients’ decisions, not to dictate what those decisions should be.
Although the health reform law does not define patient-centered as the term relates to comparative effectiveness research, requirements and procedures within the law enumerate what I believe are the elements, outlined below. The new Patient-Centered Outcomes Research Institute embodies these elements, and, as a result of a lot of hard work on the part of Congress, the Obama administration, and the broader health care community, we finally have a blueprint for patient-centered comparative effectiveness research written into law. Patients’ voices must now be heard as priorities are established for comparative effectiveness research, research topics are selected, research is conducted, and results are disseminated.
The Patient’s Perspective
Since 2007, well before passage of the Affordable Care Act, patient and provider groups have articulated the elements or principles of patient-centered comparative effectiveness research. These include the meaningful engagement of multiple stakeholders and opportunities to participate; acknowledgment of individual differences and the importance of advancing personalized medicine; a broad, clinically focused research agenda; transparency of decision making and research results; and the need to communicate results appropriately. I believe that these overarching principles should be at the core of any comparative effectiveness research enterprise that claims to be patient centered.
Meaningful Stakeholder Engagement
Bias in health care can come from manufacturers, private insurance companies, government payers, providers, patients—in fact, from any stakeholder. The Affordable Care Act recognizes this and brings all stakeholders together in support of a common bias—in favor of the patient. Until now, patients and providers have rarely, if ever, been given a central role in research programs. Although some external advisory bodies have occasionally had an important part to play in research, too often they have ended up operating at the margins of federal programs, with no authority to ensure that patients’ concerns are recognized and addressed.
Health reform addresses this through a major focus on stakeholder integration in the governance of the new Patient-Centered Outcomes Research Institute. This element may be the most critical for achieving patient-centered comparative effectiveness research and ensuring the objectivity and integrity of the overall research agenda and individual studies. The new institute will be run by a governing board of nineteen stakeholders, in addition to the directors of the Agency for Healthcare Research and Quality (AHRQ) and the National Institutes of Health. The law specifies that the board must include three individuals representing patients and consumers. It also mandates that the board be appointed by the comptroller general by September 23, 2010.
These provisions will give patients a direct, meaningful role in setting research priorities, overseeing the conduct of research, ensuring strong methodological standards, and releasing research findings.
In addition, the resources provided by the new institute to support patient involvement will help ensure that patients play an active, meaningful role in the board’s deliberations. For instance, there are resources for technical support and training so that board members at every level of technical expertise will be able to develop the skills to participate fully in the institute’s work. This will allow the institute’s research to incorporate the vital patient perspective and will help ensure that the patient perspective on the board is not marginalized by other perspectives.
Ensuring that patients have adequate representation on the institute’s board, and can participate fully on the board and advisory panels, will make the final research products more acceptable to the broader patient community and may lead to more rapid adoption and use of findings by patients and providers.
A concern frequently raised about comparative effectiveness research is that its results are based on averages across large populations and may not adequately reflect individual differences among patients.6 Indeed, particular patient subgroups, such as people with multiple chronic conditions, are often excluded from clinical trials.
Moreover, when the research fails to respect differences among patients, the clinical impact of the results may be undermined. Subgroups, such as patients with a particular genetic marker, may respond differently to a given therapy than the average of larger populations.1 If research findings that do not deal with subgroup differences are incorporated into practice guidelines or materials for patients or providers about a particular intervention, there can be harmful consequences for some patients.
The Patient-Centered Outcomes Research Institute alleviates these concerns by incorporating consideration of different patient subgroups—such as minority populations, the elderly, and children—into research design and the reporting of results. The Affordable Care Act also recognizes the growing importance of genomic science in helping explain differences in patients’ responses to treatment. Including genetic and molecular subtypes in research populations will help advance our ability to help individual patients choose the best treatments for them.
Transparency And Public Input
The legislation establishing the institute requires it to solicit comments from the public at several critical stages of the research process, including setting research priorities, establishing methodological standards, and drafting reviews of existing evidence. With very few exceptions, the institute’s governing board meetings, bylaws, and proceedings are to be kept open and accessible to the public. These explicit requirements for transparency and public input will help ensure that the organization remains accountable to patients and that all who wish to participate in the institute’s projects or observe its processes are able to do so.
Broad Research Agenda
It is clear that achieving patient-centeredness in health care will require examining various approaches to care delivery and management. In a June 2009New Yorker article, Atul Gawande reinforced the point that addressing the needs of all patients, including the disabled and those battling chronic illness, requires research on the effectiveness of different systems of care.
The law establishing the institute recognizes that comparative effectiveness research addressing “protocols for treatment, care management, and delivery” as well as medical treatments and procedures will lead to important improvements in health care’s quality and value.10 My own experience convinces me that this is a major opportunity to improve patient care by finding better ways to communicate information throughout the health care system.
Appropriately Communicating Results
It is essential that results from this research do not end up on a shelf somewhere, ignored by patients and providers as too hard to understand. Instead, the results need to be communicated in language that doctors and patients can understand and apply to a set of individual circumstances. Because the institute’s stated purpose is to inform health care decision making, how the research results are communicated is critical.
The Affordable Care Act includes explicit requirements for the release of results by the institute and the dissemination of research results by the Agency for Healthcare Research and Quality. Communications must provide details about the research’s limitations and its consideration of subpopulations. Requiring AHRQ to consult with major medical societies and patient organizations will strengthen patients’ and providers’ confidence in the research.
Dispelling The Myths
Although patient advocacy groups have worked hard to educate policy makers and the public on the elements of patient-centered comparative effectiveness research,11 many Americans still misinterpret the intent of the research.12 In particular, there have been concerns that the Patient-Centered Outcomes Research Institute will come between doctors and patients in decision making, instead of providing information to help them make good choices.
Concerns about the potential misuse of the research are not without merit. The use of an implicit cost-effectiveness threshold by the National Institute for Health and Clinical Excellence in England and Wales has led to denial of coverage for some new treatments and has provoked controversy among patients and providers. One important difference between the US and UK institutes, of course, is that the Affordable Care Act limits the new US institute’s focus to comparative clinical effectiveness, putting assessment of cost-effectiveness by the institute out of bounds.
That restriction together with the law’s incorporation of patient-centered principles into the research reflect the recognition that comparative effectiveness research rarely, if ever, will provide a single answer about what is best for all patients. Indeed, the law explicitly acknowledges that “there is an art to medical practice that extends beyond hard science and numbers and often requires swift, sound decision-making in an environment of incomplete evidence. Sometimes no formula or calculation based on quantified evidence and data can determine the appropriate action.”13
It is time for us to move beyond the heated rhetoric of the health reform debate and engage in an honest, open dialogue about how to achieve patient-centeredness in comparative effectiveness research. When such research is used well, it can be a powerful tool in making medical care better informed, without limiting patients’ and providers’ choices. The Affordable Care Act takes a thoughtful approach to the research, achieving patient-centeredness through objectivity, integrity, respect for patient differences, and an appreciation of the limitations of comparative effectiveness studies.
There is no doubt that the Affordable Care Act sets the gold standard for patient-centeredness in comparative effectiveness research. This effort can bear fruit for years to come by creating a sustained program of important research. It can also form the basis for additional dialogue about how to meet the critically important challenge of controlling health care costs while avoiding oversimplified rationing of patient care.
We have a great deal of work to do in the months ahead. As I write this article, policy makers are already taking the first steps to create the Patient-Centered Outcomes Research Institute, which will guide the comparative research effort. These initial steps are important opportunities to maintain and reinforce patients’ involvement in the institute and their trust that it will be committed to meeting our needs.
What’s more, the health reform law’s processes for involving stakeholders and the public, setting research priorities, ensuring transparency, recognizing patient differences, and disseminating results can also be incorporated into the comparative effectiveness studies already under way within the Department of Health and Human Services under the $1.1 billion allocated in the American Recovery and Reinvestment Act of 2009.
If the provisions for the new institute in the Affordable Care Act are implemented effectively, we can ensure that comparative effectiveness research is patient centered. Anything less would be a disservice to the thousands of patients and providers who supported health reform. Most important, it would mean that we have failed the millions of patients and providers who are relying on the institute to produce strong evidence to help inform their health care decisions in ways that meet their individual, personal needs.
ABOUT THE AUTHOR: TONY COELHO
Tony Coelho represented California’s 15th congressional district for six terms, from 1978 to 1989. Now a patient advocate, he has had epilepsy since he was in a car accident at the age of fifteen. “I’ve been a patient and disability advocate for over forty years,” he says. “Due to my passion to protect patients and give the disability community a voice, I have been involved in the comparative effectiveness research process and believe that, if done correctly, the research has the opportunity to empower patients.”
During Coelho’s tenure in the House of Representatives, he was primary author and sponsor of the 1990 Americans with Disabilities Act. By 1994, the US Census Bureau reported that an estimated 800,000 more people with severe disabilities had found employment than were employed when the bill was enacted fourteen years earlier. President Bill Clinton appointed Coelho as chair of the President’s Committee on Employment of People with Disabilities, a position the former congressman held from 1994 to 2001. Coelho also served as vice chair of the National Task Force on Employment of Adults with Disabilities.
In March 2009, Coelho was named chair of the Partnership to Improve Patient Care, a patient advocacy organization made up of more than forty member groups. In this capacity, Coelho advocates on behalf of the partnership’s diverse members, who include people with disabilities, racial and ethnic communities, and the elderly. He chairs two other advocacy organizations—Life without Limits and the Disability Pride and Power Committee—and he serves on the Epilepsy Foundation’s Board of Directors. He holds endowed chairs in public policy at the University of California, Merced, and in neurology for pediatric surgery at the University of California, Los Angeles.