Medscape One-on-One: Comparative Effectiveness

Eli Y. Adashi, MD; JoAnn E. Manson, MD, DrPH; Milton C. Weinstein, PhD


April 15, 2010

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Eli Y. Adashi, MD: Hello. My name is Eli Adashi, Professor of Medical Science and former Dean of Medicine and Biological Sciences at Brown University [Providence, Rhode Island]. Welcome to Medscape One-on-One.

Today's topic is comparative effectiveness, a concept seemingly catapulted to national consciousness by its inclusion and underwriting in the 2009 American Recovery and Reinvestment Act to the tune of $1.1 billion. Additional funding is likely forthcoming, courtesy of the Healthcare Reform Bill or the President's budget request for the 2011 fiscal year.

What is comparative effectiveness? Why now? Where does it intersect with cost-effectiveness? And how, if and when might it affect the US healthcare scene and its variant constituencies?

To help us sort this out, we are joined today by two authorities, Dr. Milton C. Weinstein, Henry J. Kaiser Professor and Director of the Program on Economic Evaluation of Medical Technology at the Harvard School of Public Health [Boston, Massachusetts]. He's the coauthor of a very recent contribution on the subject to The New England Journal of Medicine. Dr. JoAnn E. Manson, Professor of Medicine with Harvard Medical School and Chief of Preventive Medicine at the Brigham and Women's Hospital [Boston, Massachusetts], served as a member of the Institute of Medicine Committee on Comparative Effectiveness Research Prioritization, whose report was released in mid-2009.

Welcome, Drs. Weinstein and Manson. It's good to have you both.

Dr. Weinstein, if I might begin with you, what is comparative effectiveness, and what do we know about its history in the US and abroad?

Milton Weinstein, PhD: The goal of comparative effectiveness research is to compare the benefits and the risks of alternative clinical approaches to a health problem. Unlike what the FDA does to evaluate new drugs, in comparative effectiveness research, alternative treatments or diagnostic tests are compared head-to-head. Examples might be comparisons of alternative treatments for depression, alternative surgical or nonsurgical approaches to low back pain, or alternative diagnostic workup strategies for chest pain.

An important feature of comparative effectiveness research is that it is quantitative. So, in addition to assessing whether one approach is better than another, one tries to ask the question, "How much better is one approach over another?"

In the United States, comparative effectiveness research has been going on for at least half a century, but under different names. Clinical trials have been funded by the National Institutes of Health for a long, long time. During the 1980s and 1990s, a new approach to comparative effectiveness was developed, thanks largely to the discovery by Dr. Jack Wennberg and others that the Medicare claims database could be used as a research tool. So, it became possible to use this database to study how alternative patterns of utilization of healthcare services related to the outcomes derived from those services. However, using these observational databases required the development of new methodologic tools, such as causal inference methods and decision analysis.

Dr. Adashi: So it's about the outcome, essentially, of 2 comparable diagnostic, therapeutic, or preventive measures, and the like, compared head-to-head.

Dr. Weinstein: That's right. And we've been calling it "outcomes research" for a long time, and now the name "comparative effectiveness research" has taken hold.

Dr. Adashi: Dr. Manson, why the current surge of interest in comparative effectiveness, particularly since it's not necessarily a novel concept? There must be other overriding reasons why the government chose to invest so heavily in this form of research going forward?

JoAnn Manson, MD, DrPH: With the expanding array of options, it's become increasingly important to understand what really works in healthcare and what works in a real-world setting, because as Milton points out, many of the studies that are done are not necessarily in the kinds of patient populations that clinicians are dealing with day in and day out, and they're not directly comparing the options that are available. There's also an increasing awareness that just having more health care, more tests, more procedures, and more treatments doesn't necessarily translate into better health, and we really have to think about what is actually working to improve health and to improve outcomes.

We know that the United States is lagging behind many other developed countries in terms of some of the important indicators of health -- life expectancy, infant mortality, and rates of preterm birth and low birth weight. We need to do more to try to improve these outcomes and also to implement and translate, in disseminating the evidence base, the knowledge that we're getting.

Dr. Adashi: Does this also mean that therapeutic, diagnostic, and other approaches that may turn out to be ineffective or less effective could be weeded out?

Dr. Manson: Exactly. I think that clinicians really need more information, a greater evidence base to work with in terms of understanding what is working and what isn't. And in fact, it may be determined that many of the things that we're doing in clinical practice are just not effective, and you're utilizing resources that could be better spent on activities that actually improve healthcare. That information, that evidence base has not really been available to clinicians. And also we know that much of the information that is available is not being used. It's just not being implemented in clinical practice. For example, we know from several types of studies that about 80% of heart attacks could be prevented by lifestyle modifications, good treatment of blood pressure, lowering cholesterol, and modifying risk factors, yet a very large percentage of Americans have risk factors that are not well controlled and are not modifying lifestyle factors that can be tremendously important to improving health. We need more information about how to effect behavior change and how to translate and disseminate this information in terms of clinical practice.

Dr. Adashi: But we can never perhaps sufficiently emphasize how important evidence is as opposed to gut feeling, and even experience, which tend to be anecdotal and individual as opposed to population-based.

Dr. Manson: Having the rigorous evidence comparing different interventions, different options, and alternatives for diagnosis and treatment will be tremendously valuable to clinicians and to the public in terms of the decision-making that takes place in families and among caregivers, to understand which options are really going to be of interest to pursue.

Dr. Adashi: So, the consumers of that information tend to be multiple. They are not just physicians. The patients themselves would be interested, of course, in knowing what they're into, and presumably the payors would be interested in understanding what works and what does not work.

Dr. Manson: Very much so, and in fact a major goal of the comparative effectiveness research program is to involve consumers and policy-makers and payers in some of these decisions, so that it's really a collaborative process.

Dr. Adashi: Thus far, we have spoken about the utility of comparative effectiveness as a tool to establish outcomes, relative outcomes, and effectiveness of outcomes. Now, Dr. Weinstein, if I may ask, where does the notion of comparative effectiveness intersect with the concept of cost-effectiveness, which we haven't touched upon? And what do we know about the history of this element of the equation in the US and abroad?

Dr. Weinstein: Cost-effectiveness can be thought of as a type of comparative effectiveness research that goes a step further. In addition to evaluating the comparative health outcomes of alternative approaches to a condition, it also looks at the comparative costs, with the idea that if there are interventions that are underutilized but have very good value and cost relatively little and produce large improvements in outcome, resources could be channeled to them and possibly taken away from other interventions that are either totally ineffective or may be effective, but only very slightly so, and at a very high price.

In the United States, cost-effectiveness has a history somewhat shorter than that of comparative effectiveness. But as early as the 1970s and 1980s, there were research institutions beginning to develop the techniques and applications of cost-effectiveness analysis. In that period, in the government, the Office of Technology Assessment, which at the time was an agency of the US Congress, began to do cost-effectiveness studies of medical technologies, with the idea of informing Congress about decisions that they might make about things like Medicare coverage of new medical procedures.

In the 1980s, an agency was created called the National Center for Health Care Technology, part of whose mission was to evaluate medical technologies in terms of their cost-effectiveness. This agency had a very short life. There was a lot of political backlash, largely from industry but also from elsewhere in the healthcare sector. And so the agency was disbanded.

Subsequently, though, the government got back into the act. The Centers for Disease Control and Prevention has been doing cost-effectiveness analyses of preventive services for several decades. And in the 1990s, the Secretary of Health and Human Services convened a panel, an expert panel, of which I was a cochairman, to make recommendations to the government and to the community at large about how to do cost-effectiveness analysis and appropriate methodologies, to try to develop some kind of standards for the field.

Ironically, the very industries that objected to the National Center for Health Care Technology began to use cost-effectiveness analysis for their purposes, largely at first as a marketing tool. They wanted to be able to convince their payors that what they were offering to them at sometimes higher prices than what they had been paying was good value for money and would benefit their members. And today, even the NIH [National Institutes of Health] has been involved in cost-effectiveness research. They support cost-effectiveness studies linked to clinical trials that they support but also separately, using modeling and other methodologies to evaluate cost-effectiveness.

Now, has cost-effectiveness actually influenced policy in the United States? It's hard to tell. Guideline panels of the medical professional societies do cite cost-effectiveness studies from time to time in making their recommendations, but there's been very little explicit use of cost-effectiveness analysis in the formulation of government policy. There's evidence that this is changing, and we will see going forward to what extent cost-effectiveness analysis will begin to be used more explicitly.

In the rest of the industrialized world, cost-effectiveness analysis has been embraced. Australia was the first country to adopt procedures -- formal, regulatory procedures through its pharmaceutical benefits plan -- to make decisions about coverage of drugs based in part, not exclusively, but in part on cost-effectiveness. Canada and its provinces followed behind very closely, and the United Kingdom, particularly England and Wales, has created an agency to make recommendations to the National Health Service that are based in part on cost-effectiveness analysis.

Dr. Adashi: So, while we cannot say perhaps with certainty what the popularity or applicability of comparative effectiveness and cost-effectiveness studies in the United States will be, the sense is that it is likely going to penetrate the fabric increasingly, because we all recognize that we need to bend the curve or contain the costs of healthcare.

Dr. Weinstein: I think what we have to do is get away from the idea that looking at cost-effectiveness, the value for money, is pulling the plug on Granny. I think we have to get away from that equation and recognize that the way –

Dr. Adashi: As that paralyzes the discussion.

Dr. Weinstein: Right. The way to better health and, at the same time, cost containment – I think the first step, as Dr. Manson points out is to identify those interventions that are worthless, that are doing nothing. But I think we have to go even farther than that. We have to identify those interventions that are underutilized, and Dr. Manson mentioned some in the area of prevention that are underutilized, channel resources towards them and perhaps pull back a little bit on those interventions that produce very, very little benefit at a very high cost.

Dr. Adashi: Thank you. Dr. Manson, going back to the Institute of Medicine committee that you participated in, what was the charge of that committee, and what conclusions were arrived at and how?

Dr. Manson: The charge of the Institute of Medicine Committee on Comparative Effectiveness Research was to make recommendations to the government to the Department of Health and Human Services Secretary about the use of $400 million of the $1.1 billion that you mentioned at the start that would be used on cost-effectiveness research at the beginning of this program. So, we were charged with putting together the list of priorities. But we actually went far beyond coming up with a list and went into many issues related to developing an infrastructure and a workforce, and expanding the workforce to be able to do effective CER.

Dr. Adashi: CER. is...?

Dr. Manson: Comparative effectiveness research. The process for coming up with the initial list of priorities involved 3 strategies. One was a Web-based questionnaire that was open to the entire public to submit questions that would be evaluated by the committee. And there were over 2600 questions submitted by healthcare professionals, professional societies, the public, patients, caregivers, and payors -- all of the stakeholders submitted questions that we then reviewed. There was also a public forum where many stakeholders could present their recommendations and their views about this process. In addition, the committee members weighed in on what they thought was important as questions to be considered and different studies to be done.

We gradually narrowed down the list to 100 top priorities that were then submitted. But as I mentioned, we went far beyond, because there's a pretty lengthy book that came out of this process, to also try to improve the research structure for doing comparative effectiveness research. But the initial list of priorities was very well balanced in terms of applying to all of the demographic groups. There were research questions that relate to children, maternal and child healthcare, middle adulthood, later adulthood, and older years, and also across all disease groups and a very wide range of health outcomes.

Dr. Adashi: For the benefit of our listeners, could you maybe list one example of a topic that seemed to be in great need of comparative effectiveness research?

Dr. Manson: For example, the very first topic was to look at atrial fibrillation, where there are many treatment options available. There are surgical options, medical therapy, ablation therapy. And clinicians often don't know which option to be offering to patients. And also, many of the studies have been done in patients who tend to be healthier than those we see in clinical practice, patients without a lot of comorbidity; that's important to consider.

Dr. Adashi: So in fact, the number 1 priority was the various therapeutic options for atrial fibrillation?

Dr. Manson: That was one example. And we divided them up into quartiles, the top 25. They weren't necessarily ranked within each of the quartiles. But it was very important, for example, to have research done to understand how do you best implement the decisions that are made with clinical effectiveness, with comparative effectiveness research, what actually works in terms of disseminating and translating that information into clinical practice? That is one of the research questions that we recommended addressing.

Dr. Adashi: But I do think that the example you cite really exemplifies the importance of the process. I believe there are 2 or 3 million individuals in the United States living with atrial fibrillation. And for them, and for their physicians and for their payors, it's a very critical issue to understand what works best, and this is a wonderful example, I think, illustrating the importance of the technique.

Dr. Manson: I also want to emphasize that it isn't a one-size-fits-all kind of approach. I think it's more to understand what's effective in the real-world setting and what patient characteristics and clinical characteristics might actually modify the response to different treatment options. So, it by no means says that all patients should be treated in one way, but it will help clinicians and patients understand what might work best for them.

Dr. Weinstein: If I may add, there has been a lot of discussion about how the new pharmacogenomics might affect comparative effectiveness research. Some critics of comparative effectiveness go so far as to say it makes it useless because when you get to the level of individualized medicine, you can't do the kinds of studies that comparative effectiveness research entails. So, I think it remains to be seen. I think it's a challenge. I think like all these kinds of challenges, it will be overcome, but it is a challenge.

Dr. Adashi: I'd like for us to close, perhaps, with a brief discussion of the implications of comparative effectiveness research on the various stakeholders of the US healthcare system. And if I may begin with you, Dr. Manson, what is your sense of how receptive US physicians who are critical, obviously, will be to the lessons learned, the conclusions reached by rigorous comparative effectiveness research?

Dr. Manson: I actually think that clinicians will be receptive to it. I think that physicians and other healthcare providers really want to make the best decisions for their patients, and they want the information and the evidence base to work with. It won't be well received if it is a one-size-fits-all, cookie-cutter kind of approach to medicine. But that won't happen, because the decision-making latitude of the healthcare provider working with individual patients will always take precedence, and there will always be room for clinical judgment and the opportunity to make what might be in fact a very different decision for an individual patient.

But I think that the comparative effectiveness research program provides an opportunity to get very useful information to clinicians and also to focus on how can you best translate that information into improved healthcare and improved clinical care.

Dr. Adashi: So, disseminate newly generated information, but also existing information in the best interest of all involved.

You mentioned, Dr. Weinstein, that the Europeans in particular have embraced both comparative effectiveness research and cost-effectiveness research. Is there something for us to learn from our European colleagues in that regard? Can you see lessons that are ripe for application across the ocean?

Dr. Weinstein: I'm not a political scientist or a sociologist, but my political science and sociology colleagues tell me that there are vast cultural differences between Europeans and Americans. Europeans, in general, and the British in particular, since they've been in the lead in using cost-effectiveness, have a much greater tolerance for authority. Americans demand their independence. And we are unlikely to have the kind of regulatory structure that they have in Britain. We are likely to continue to rely on markets, free markets, with individual freedom of choice within our system.

That said, we do tend, at times, to be willing to accept the imposition of carrots and sticks -- incentives. So I think what we may see more of are financial and other incentives to use cost-effective medical practices more and to use cost-ineffective practices less. One way that might happen is through the payments to providers. They might get, perhaps, a premium on top of their cost for delivering cost-effective services, and they might just barely make ends meet on cost-ineffective services.

For the patient, we now have copayments that are in tiers. Those tiered copayments might be linked to cost-effectiveness or comparative effectiveness so that individual patients will have that incentive. To make that work, there has to be more information. I totally agree with Dr. Manson that one of the most important products of comparative effectiveness research will be the information for providers, for physicians, but also information for consumers and patients so that they, too, can collaborate with their physicians in making more informed and cost-effective decisions.

Dr. Adashi: So, perhaps another way of recapping this is that healthcare is local and so we, on this continent, are going to have to find an American solution to interpreting and capitalizing on comparative effectiveness and cost-effectiveness research.

Well, we've come to the end of our brief conversation. And I want to thank you both for a really wonderful treatise on an important issue. I also want to thank our viewers for listening to Medscape One-on-One. Until next time, I am Eli Adashi.


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