Comparative Effectiveness Research: the Horizon

Last month, the Cambridge, Mass.-based New England Healthcare Institute (NEHI) released an issue brief, “From Evidence to Practice: Making CER Findings Work for Providers and Patients,” which detailed the hurdles and policy choices facing leaders in comparative effectiveness research, in their efforts to widely disseminate comparative effectiveness research (CER) findings. Given the provisions in the federal healthcare reform legislation, called the Patient Protection and Affordable Care Act (PPACA), passed by Congress and signed by President Obama in March, that will establish the creation of a federal Patient-Centered Outcomes Research Institute to identify research priorities and conduct research comparing the clinical effectiveness of medical treatments, NEHI’s issue brief certainly provides a timely look at the challenges ahead in this dynamic area.

HCI Editor-in-Chief Mark Hagland spoke recently with Tom Hubbard, senior program director at NEHI, on the healthcare reform context of comparative effectiveness research, as well as on the major industry-wide challenges to CER results dissemination related to the state of healthcare IT evolution.

Healthcare Informatics: Can you explain the mechanism for comparative effectiveness research that was established on the federal level via the federal healthcare reform legislation passed into law in March?

Tom Hubbard: Stepping back a few steps, comparative research is certainly nothing new; doctors have been comparing research results for as long as there have been doctors. But there’s no question that the field of comparative effectiveness research has been growing in the last few years. And in the couple of years leading up to the passage of the healthcare reform legislation, despite the fight that broke out, a number of folks on Capitol Hill realized that it would be a good thing to expend funding on CER. The most contentious issue that came up was doing comparisons based on cost-effectiveness. You can do research on clinical effectiveness purely from the patient outcomes standpoint; or you can mix clinical effectiveness and cost-effectiveness. And cost-effectiveness clearly was the most contentious thing. And I think members of Congress had decided to downplay the cost-effectiveness issue even before the healthcare reform debate broke out over this. And clearly, the cost-effectiveness issue became the most contentious, with images of shoving Granny off the ventilator and such.

But many European countries have been applying that mixed model to healthcare decision-making for a long time. And this debate got off to an early start, because the federal stimulus goal provided the incoming Obama administration with the opportunity to fund CER. So $1.1 billion was appropriated both to commission new research, but also to provide money for CER infrastructure, meaning the research networks and tool-building to support CER, and quite a bit of that involved information technology. The research infrastructure includes building the health IT networks to do the data mining to start doing more rapid and real-time research on what’s effective. And that’s the kind of thing that some major hospitals are doing already, diving into their patient data and determining what works best for their patients.

So the stimulus bill turbocharged CER in the same way that it turbocharged IT automation, and that began in January or February of last year. In so doing, it sparked this sort of firestorm that led to Sarah Palin talking about death panels and all that, and that led into the debate over the healthcare reform bill itself. What came out of the healthcare reform bill was pretty much exactly what the leadership in Congress had sketched out before the healthcare reform debate. That is to say that they went with the strategy for CER that had been sketched out by the Senate, specifically Sen. Max Baucus [D-MT], the chairman of the finance committee, and that is, moving CER into this independent, albeit federally funded organization, PCORI—the Patient-Centered Outcomes Research Institute. And that is an organization that is roughly similar in organization and mission to the Institute of Medicine (IOM). It will be governed by a board of governors that will be appointed by the Comptroller General, the head of the U.S. Government Accountability Office (GAO), which is itself an independent agency. So the task of actually appointing the board of governors was handed off to a non-political appointee.

The bill does specify that the governors must be diverse, so there will be academic experts, government leaders, clinicians, representatives of healthcare industries including pharmaceutical and device manufacturers. Dr. Clancy from AHRQ [Carolyn Clancy, M.D., director of the Agency for Healthcare Research and Quality] will be on it, and the Director of NIH will be on it, Dr. Collins [Francis S. Collins, M.D., Ph.D., director of the National Institutes of Health]. Those two, from government, will be on it; otherwise, it will be a group of non-governmental people. And PCORI will have a budget, and will commission research.

HCI: Will your organization do research for them?

Hubbard: Probably not, actually. We’re set up as a nonprofit health policy organization, so our focus will be more on the policy aspects of this.

HCI: Tell us about your study and its overall goals.

Hubbard: It’s an overview of the hurdles that have to be overcome in order to get to an effective dissemination of CER findings. We’ve mostly been talking to various experts and to people from key stakeholder groups with some role in the dissemination of information.

HCI: So what do you see as the greatest hurdles in the dissemination of comparative effectiveness researching findings?

Hubbard: Just for starters, and this is a set of findings that probably a lot of hospital CIOs are familiar with already, but in general, what has been demonstrated over and over from the IOM and other organizations, is that all kinds of evidence coming from solid peer-review research, virtually all kinds of evidence have a difficult time getting fully disseminated and picked up and used in the healthcare system. In general, evidence in all fields of medicine faces a very protracted and uneven and erratic path, to get to the point where it actually gets seen by doctors and patients and gets utilized, and influences decision-making. That is something that is surprising to many of us who are laypeople or patients, that good research findings don’t get instantaneously absorbed by doctors across the country. So in comes this new investment in comparative effectiveness research. And one of the reasons to push new comparative findings out there is that comparative findings should be more useful to physicians and patients from the beginning.

What is it that doctors and patients are most interested in at the point of care? It’s what will work; and comparative findings naturally lend themselves to that, more so than do non-comparative findings. But comparative findings will face the same challenge as all other clinical information in getting into use. So there will have to be a very thoughtful, national, dissemination strategy for CER findings. And that’s where our strategy comes in. And one of the insights is that the health IT investment is critical. And the health IT investment, fundamentally, is the means of communication of literally piping findings to clinicians. But the capabilities that we’re now seeing in healthcare IT are such that it can significantly improve the odds of turning this research into something more immediate at the point of care for clinicians and patients to use.

HCI: So what should be on the radar screens of CIOs and healthcare IT professionals, as PCORI gets set up and developed?

Hubbard: First, I think that there are at least two pretty straightforward things that CIOs would probably want to look at and bear in mind as far as CER. One is, to the extent that clinical decision support is further built out, will be a means of disseminating CER findings. And it may be a comment on the obvious, but to the extent that CPOE and CDS get expedited into use, that clearly becomes an important path of dissemination for CER findings, as for all forms of medical evidence. And all the pieces of healthcare reform have to interlock in order for the big objectives to be realized, including covering more people, getting them treated, and treating them optimally. So it’s not just that HIT all by itself will have the total effect, it involves payment reform, chronic care management, CER, and everything. The other big thing, though, for CIOs to keep watch on, and as I was saying, a number around the country are already involved, is in the use of their existing data to look at that in conjunction with CER findings that they might be getting through medical journals, to do real-time search on what works in their systems.

This is one of the things we called out in the issue brief, is that when you look at what the major integrated health systems are doing, such as Intermountain Healthcare, or Kaiser Permanente, or the Mayo Clinic, they’re continuously looking at their own research and new research in the literature, to develop new protocols for the way they practice medicine. So what we’re seeing in the integrated system is a contextualization of evidence. So it’s not just that they’re ripping open the cover of the New England Journal of Medicine every week; they’re saying, what does that data say compared to our own data? And that speaks to one of the controversies around CER, that it would lean towards one-size-fits-all, ‘cookbook’ medicine that would be stuffed down the throats of clinicians. Instead, you can convert it to your own purposes, and clearly, a key element in that is HIT.

HCI: As they look for more CER findings to be disseminated through PCORI and other venues, what should CIOs be thinking or planning?

Hubbard: It’s difficult right now to predict how the larger CER effort is going to play out, in the sense that the PCORI board is not up and running yet. I think a lot of people I talk to in the CER field are waiting to see who gets on that board of governors and what the dynamic will be. They’ll have to pick an executive director soon. From the standpoint of dissemination, there are a couple of issues for them. One, how aggressive will they be? The legislation says they’ll commission research, but they’ll utilize AHRQ as the dissemination agency for findings. So there’s an issue of how expansively PCORI will define its mission beyond that, particularly to the extent that it can leverage its role as a non-governmental organization, to potentially partner with groups outside government, such as medical specialty societies, and such.

Of course, there’s a question at AHRQ regarding how broadly they might define their expanded role. And the other big question is how healthcare reform will be received more broadly over time. CER certainly attracted the earliest controversy; so there’s a question as to how well CER will be able to work through what had been an initial crisis of legitimacy and such… And there’s an ongoing pipeline of CER findings that predated the healthcare reform legislation and stimulus legislation. But all new research takes time. So I think it’s an open question as to whether there will be a dramatic acceleration of findings that provider organizations will need to deal with. On the other hand, we do have these leading-edge organizations out there that are doing it on their own, and that are in that way, perhaps setting a standard for other providers to catch up to.