This is the thirteenth in a series of interviews between CFAH President and Founder Jessie Gruman and patient and consumer group leaders about their experiences with and attitudes toward comparative effectiveness research (CER).
Gruman: Tell me about yourself and your organization.
Richard Birkel: The National Council on Aging (NCOA) is a service and advocacy organization. Our goal is to improve the wellbeing of older adults, particularly those who are disadvantaged and most vulnerable'those over 60 at the edge of poverty. There are 22 million people in our target population and they are growing rapidly. My role as Senior Vice President for Healthy Aging is to oversee our portfolio of healthy aging, consumer education, self-management, fall prevention and behavioral health programs.
Gruman: Where do you see comparative effectiveness research (CER) fitting in the effort to improve the effectiveness of health care?
Richard Birkel: We believe that CER can be a valuable strategy to improve health care. The basic idea is to figure out from head to head trials or systematic reviews of the scientific literature which treatments are most effective overall and which treatments have the best profile for different populations.
We are concerned, however, that older adults have often been excluded from clinical trials of drugs, medical devices and procedures. Many trials exclude people based on age alone, and many others exclude people with cognitive impairment, other illnesses, physical disabilities or for living in a nursing home. As a result, the people enrolled in a trial or study may be quite different from those who will actually use the drug or undergo the procedure in the real world.
Gruman: Tell me how your organization views the relationship between CER and patient-centered outcomes research (PCOR).
Richard Birkel: We view them as closely related activities. In PCOR, the emphasis is on identifying outcomes valued by the population being studied. CER, on the other hand, doesn't necessarily take into account the patients' perspective, even though it may produce important implications for treatment. In PCOR, the idea is to work with the affected population to better understand what outcomes they value.
Take, for example, a question like, 'Should we do surgery or medically manage this condition?' The answer depends on which outcomes we look at. How long does it take to recuperate? What are the risks? What are the side effects? What is the impact on quality of life and ability to live independently, or without pain? Such outcomes are best identified by asking patients and caregivers what is most important to them. The value of a particular procedure is in the eye of the beholder.
Gruman: Can you give me an example of how your constituents have been affected by CER?
Richard Birkel: Here's an example of the value of CER. A recent CER study compared bypass to percutaneous coronary intervention, that is, doing a procedure through the skin using an insert, versus opening the chest. You'd assume that the less invasive intervention would be safer for everyone. The study found something quite interesting, however. For people over 65, it was safer to do by-pass surgery. The opposite was true for people under 50. This is an example of the kind of CER study that will lead to better decisions. This result was counterintuitive: you couldn't know this for sure based on experience alone. Systematically gathered data on patients followed over time was necessary.
More CER studies will result in better guidelines. Those guidelines will provide doctors with the opportunity to talk with their patients about their choices and options by discussing the evidence and bringing to bear the best science-based advice they can about alternative treatments and their potential outcomes.
Gruman: How do you see the increased attention to CER affecting the care your constituents will receive in the future?
Richard Birkel: There are so many important questions related to the care of an aging society. Is community or home care better than nursing home or assisted living care? For whom? What is the best way to prevent falls? What treatments are effective for hearing loss? What is the relative effectiveness of pharmacologic and non-pharmacologic treatments for treating behavioral disorders in people with Alzheimer's disease? What are the best ways to help people learn skills to manage multiple chronic conditions? Many of these questions are new ones in health and long-term care and are important to rapidly growing segments of the population. Unfortunately, right now, we don't always have the data we need to answer these questions. Increasing our commitment to CER will provide the science base we need to help people age well with dignity.
Gruman: Some professionals believe that patients are opposed to comparative effectiveness research. Do you think this is accurate?
Richard Birkel: I think the American public has conflicting ideas and beliefs about what they want in healthcare. On the one hand, we have a strong belief in personalized medicine. We believe that decisions about our health care are highly individualized and best left to us and the providers who know us best. We see anything that smacks of generalizing or categorizing, anything that minimizes our individual relationship with our health care provider, as an infringement on our health care 'rights'. On the other hand, many of us believe in science and its power to improve care. So, there is a tension here. We want care tailored to our needs and we believe that our personal clinicians can do that best. We also want our clinicians to be well informed and steeped in research.
At the same time, we are suspicious that research will be used to develop guidelines that don't take into account our unique needs. Many people don't realize that their health care provider, if he or she is doing their job properly, is consulting the most current research, including CER, to determine what might work best for each of us.
So I think we have a false tension between patient-centered medicine versus a big machine cranking out guidelines that are blind to individual differences. There IS tremendous difference among individuals in their response to medicine and procedures. I served as Executive Director of the National Alliance for Mental Illness from 2001- 2004, and we were faced with CER that showed that at, a population level, there was no difference in outcomes between new and old antipsychotics.
Nonetheless, we all knew that, for someone we knew or loved, a particular new and more expensive medicine had been lifesaving and had worked when nothing else did. What position do you take here?
If that research had been used to prevent access to whole categories of drugs or treatment, it would have ignored the fact that there is a percentage of people who will benefit; perhaps even a group of people for whom this is the ONLY medicine that works. And this is a source of the fear about CER: That scientists or payers will make grand comparisons and come up with the answer: 'This is medicine or procedure for the common person,' when there is no common person. Everyone is a unique organism.
We hoped that genomics would have already gotten us to the point where we understand the genetic basis for why one drug works for one person and not for another. But we aren't there yet except in a few isolated cases. So in the meantime, how do you personalize medicine? There may be strong study finding that say this treatment is better than that ' but doctors need discretion, and they need to apply the best available evidence from CER to make decisions about individual patients. That's why you see in the guidelines for PCORI that these results are not to be used to establish payment policy ' that is not their primary purpose.
This will only get more complicated in coming years. Some new drugs being developed are very expensive but are highly effective with a particular portion of the population, say 5%. How do we make decisions about payment for those medicines? Do we cover the costs of all medicines that are effective with some portion of the population, regardless of the cost? From a population level it won't be highly used and it seems extravagant. But, from an individual level, if you don't have it, you may die.
Gruman: What are your fears and hopes for CER?
Richard Birkel: I hope that we are moving toward better guidelines for older adults. As a population, they have been neglected in CER. Increasingly, studies must include them as older people become a larger percentage of our overall population. CER is necessary ' and it will come of age when it looks at what works for people who are 70, 80 and 90 years old.