I talked a little bit earlier this week about comparative effectiveness research (CER) and the role of cost effectiveness in this research. At ACC's Medical Directors' Institute, a variety of views on the topic were given. Tell me what you think below (and check out what others have said in previous posts on the topic).

The ACC annual Medical Directors’ Institute (MDI) was held this week at the Park Hyatt and Heart House in Washington. The topic was comparative effectiveness research and implementation. It was a terrific policy discussion which included dynamite presentations from AHRQ Director Carolyn Clancy, the UK’s CER agency NICE, Canada’s similar entity, the US Veterans’ Administration, the CMS, the National Health Council, and many other stakeholders in addition to ACC senior leaders, governors, and staff. We strongly support CER, and also want to make sure it proceeds along the lines of a patient-centered, socially responsible, and scientifically excellent path through some difficult political minefields.

The timing for this MDI was perfect in that ACC had just published our own CER policies and principles, called “ACC 2009 Advocacy Position Statement: Principles for CER.” The position statement offers nine principles for CER, outlined in brief below:

• The ACC strongly supports CER as a way of having informed decision-making.
• CER priorities should be set by a multi-stakeholder group to ensure that the research agenda reflects the needs of the country. The research agenda should be based the burden of the disease being considered, mainly morbidity and mortality.
• The ACC recognizes that the research on comparative effectiveness is “only the first step in improving the quality, equity and efficiency of medical care,” and stresses that improving quality must be the primary aim of CER.
• CER should be distinct from entities that create coverage and benefit programs, and requires close monitoring to avoid adverse consequences on access, quality or safety.
• The ACC recognizes that CER will require substantial and long-term financial support.

The paper then goes on to outline how the ACC can participate in the CER process, including informing priorities through our clinical documents, like guidelines and appropriate use criteria, as well as our registries (NCDR) and providing standardized data elements and definitions. In addition, the ACC has the ability to disseminate CER findings to patients through our large membership. The policy statement concludes: “The ACC believes CER research, when conducted correctly, is a useful tool that assists physicians and other providers in delivering high-quality, equitable and effective health care to patients.”

Comparative effectiveness is a frequent topic on this blog (see the post from John Brush, M.D., F.A.C.C.), and clearly has the potential for good – improved quality – and, if done poorly, a potential for evil – reduced access to needed treatments. Because of its potential for evil, I strongly believe that comparative effectiveness needs to happen separately from any cost comparison. This is necessary to maintain physician and patient (consumer) trust that CER is untainted scientifically from societal/government pressure to reduce costs.

Those are my thoughts on CER ... but there have certainly been some spirited discussion by our CER experts here at MDI that disagree with me. We’ve had some great speakers, including a senior scholar at the Institute of Medicine Michael McGinnis, M.D., M.P.P., Myrl Weinberg, president of the National Health Council, panels that include representatives from the Canadian Agency for Drugs and Technology in Health and U.K.’s NICE, and Carolyn Clancy, M.D., of AHRQ representing the U.S., and a briefing from Michael Rapp, M.D., of CMS, among many others.

UPDATED 10/28: New CVN video on "Perspectives on CER" with an introduction from SVP of Science & Quality Janet Wright, M.D., F.A.C.C., and thoughts on CER from attendees of MDI.

I'm sure you all saw the headlines early this month related to the Department of Justice settlement with Pfizer over their past off-label promotions of Bextra and other drugs. The settlement will cost the company a record-breaking $2.3 billion ($1 billion in civil settlements and a $1.3 billion criminal penalty related to Bextra).

Bextra was approved for treatment of arthritis and menstrual pain, but Pfizer allegedly promoted it in doses and for uses not approved by the FDA, putting patients at risk of serious cardiovascular complications including heart attack and stroke. Pfizer voluntarily withdrew Bextra from the market in 2005.

Of course, Pfizer isn’t the only company that has been involved in off label promotions. And we physician are also involved in how off label uses occur, and in relationships with industry that are under increased scrutiny in these regards. Off label usage of pharmaceuticals is often how new therapeutic advances occur and it’s not evil. But, if we had more comparative effectiveness research and dollars (and we soon will) available to research off label use, we could more rapidly advance therapeutics of newer agents and protect patient safety as well.

ESC’s Congress is just one meeting of the many international meetings the ACC leadership team and I attend each year that foster relationships with other international societies, although it is certainly the largest. In the five brief days we'll be in Barcelona, we’ve met with 14 international cardiovascular societies, along with Huon Gray, Chair of our new International Council, and that's not counting the informal conversations we've had along the way. This is a reflection of how strongly the ACC feels about working with international societies.

As mentioned at the start of the Congress, cardiovascular disease is not just a problem in the U.S. – it’s a problem across the world. And if all the countries across the world work together to find what works best for treatment CVD, then patients everywhere benefit.

One area in which this is particularly evident is comparative effectiveness research. Comparative effectiveness research has the potential to do so much: from informing the practice of medicine to improving care. Not only do we need to work together with international societies to collect comparative effectiveness data to improve care, we also need to work with other societies to disseminate these best practices. It’s not good enough that the information stay within the U.S. or the originating country; the information needs to be spread as far and wide as possible. By keeping the lines of conversation open at meetings like ESC’s, hopefully we’ll be able to increase our collaborations to benefit patients around the world.

*** Image from Flickr (katmere). ***

Tyler Cowen, professor of economics at George Mason University, put forth a modest suggestion for health care reform in a New York Times editorial earlier this month: Cowen advocates a U.K. NICE-style system that would take advantage of comparative effectiveness research to cut costs for less-effective treatments — essentially, limiting or ending reimbursement for less effective treatments.

NICE has considerable respect, even among British physicians, but most of the debate here now seems to suggest that we must separate the coverage decisions from comparative effectiveness. ACC's John Brush last month gave adifferent take on comparative effectiveness research in his post on this blog.

*** Image from Flickr (SqueakyMarmot). ***

The Brookings Institution recently held a panel discussion of key questions around comparative effectiveness research.  Senate Finance Committee Chair Max Baucus (D-Mont.) and Office of Management and Budget (OMB) Director Peter Orszag offered opening comments. Participants discussed the importance of comparative effectiveness research to overall health care reform for lowering costs, eliminating variation in spending and quality of care, and promoting measurement and benchmarking.

Citing the recent JAMA article underscoring our desire and need to enlarge the evidence base for ACC/AHA guidelines, panelists noted that comparative effectiveness research could help develop more robust guideline evidence in the future.

Meanwhile, ACC’s new policy position on comparative effectiveness and clinical effectiveness research is due to be disseminated and published soon!

*** Image from Flickr (Leo Reynolds). ***

The ACC/AHA Task Force (TF) on Practice Guidelines (GL) convened in Chicago last week to continue its meticulous examination of the process and methodologies for guideline development. This important group, under the leadership of Dr. Alice Jacobs, is committed to rigorous scientific and ethical standards, near-continuous analysis of new studies to keep guidelines current and relevant, and easy accessibility by users. We’ve asked this beleaguered TF to “turbocharge” the GL process to try to keep up with the ever increasing progression of science. (Gird your loins folks, because the National Academy of Sciences says that in the next decade the total body of human science, accumulated from the dawn of humanity to now, will increase by at least 4-7 fold! I know that sounds absurd--unless you’re a Terminator fan who wants to let the machine doctors do their thing for whatever they will be paid then.) 

The point is, how can we keep up? The GLs in the future will be done by benevolent automation I assume. But today, we rely on dedicated volunteers who are already working hard in their day jobs. Asking them to turbocharge is not easy.

Let’s slip back from this transgression from the ‘singularity’ (sentient machine future) to a reality we’re more comfortable with: 54% adherence to GLs and pressure from outsiders (like the IOM) to consider GLs produced by professional societies as perhaps too biased to recognize. We need to be on our toes here. This must remain our AHA-ACC turf.

Our TF rose to the challenge of an uncertain future in my view. We had already decided that those persons with industry conflicts, no matter how dominant in the science of a particular issue, cannot a chair a GL committee, and cannot be panel members without broad disclosure and compelling reason. In the future such individuals may serve only as consultants or testifiers, with no voting privileges, I suspect. We are tightening this up.

In recognition of the critical significance of comparative effectiveness research, members agreed to consider cost and cost-effectiveness whenever relevant and possible in future guidelines. This would not have been considered even 5 years ago. They considered changes to the recommendation language to convey incremental value in comparisons and suggested the addition of health economists and statisticians to the team of contributors. Again, a bold move.  

This month’s post comes to us from past president of ACC’s Virginia Chapter, John Brush, Jr., M.D., F.A.C.C. In addition to serving three years as Chapter president, Dr. Brush practices at Cardiology Consultants, Ltd., in Norfolk, Va., and is an Assistant Professor of Clinical Internal Medicine at Eastern Virginia Medical School. He also has been a leader in quality improvement, assisting ACC efforts with “Door-to-Balloon: An Alliance for Quality” and the IC³ Program, and as a member of ACC’s Clinical Quality Committee.

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In the current health care reform debate, there has been considerable discussion about comparative effectiveness. This method of evaluation could provide valuable information on the relative value of competing drugs, devices and treatment strategies, which in turn could improve outcomes, efficiency and satisfaction. Critics are concerned, however, that comparative effectiveness could be used to deny coverage, squelch innovation and ration care. Because of these concerns, some stakeholders forcefully argue that comparative effectiveness evaluations should be totally devoid of cost considerations.

But how can you compare competing treatments and ignore costs? To use heart failure as an example, could you really compare the relative effectiveness of ACE inhibitors and left ventricular assist devices and ignore the wide difference in costs between the two treatments? And isn’t the public’s desire to gain “more bang for the buck” what’s driving health care reform in the first place?

The Case for Cost Effectiveness
Cost effectiveness research is difficult and has recognized limitations. Yet no method of research is perfect or definitive. Although cost effectiveness research has some limitations, we should not reject the useful information that it provides for comparative effectiveness analysis.

There is a compelling need to contain costs in order to extend health care coverage universally in America.  Comparative effectiveness research will give policy makers important information that will help set priorities for spending.  As with clinical practice guidelines, comparative effectiveness analysis should inform, but not dictate clinical decisions.  Personalized decision-making for individual patients should always trump broad policy recommendations.

Comparative + Cost Effectiveness
Comparative effectiveness research and analysis will require a disciplined approach.  Comparative effectiveness research should be a transparent scientific process, absolutely free of economic influence.  Advisory boards that oversee this research and analyze the results should be shielded from undue political influence.  For years, NIH has distributed billions of dollars in funding, using established methods that are generally respected as fair and non-biased.  Similar independence and discipline can be established for overseeing comparative effectiveness research and analysis. 

Comparative effectiveness research using cost considerations should be a two-stage process.  The first stage should pertain to relative clinical effectiveness and the second stage should deal with costs.  For competing treatments with similar clinical effectiveness, no further cost effectiveness research is needed because direct cost comparisons would be simple.  But in comparisons where one treatment is more effective, careful analysis of costs will be necessary to estimate the monetary value of the increased effectiveness.

Constructing a Firewall against Undue Influence
To maintain the integrity of this process, and to shield the process from political and financial influence, a firewall should be constructed between comparative effectiveness evaluation and insurance coverage decisions.  The funding level for coverage is a political or a business issue, not a scientific issue.  The funding level for Medicare is up to Congress, and, ultimately, to taxpayers.  The funding level for private health plans is up to the purchasers and benefit design managers.

Comparative effectiveness analysis can be separated from coverage decisions by borrowing the method used in the process of grant funding:  

• When judging grants, the judges evaluate the grants based on the scientific merit of the grant, without consideration of whether the grant will actually receive funding.
• Grants are graded on a relative scale.
• Top grants that fall within the funding range receive a grant.

Is that rationing?  Perhaps so, but this explicit method of determining coverage seems more rational than the current method for rationing where we deny care to nearly 50 million Americans because they lack employer-based insurance or don’t meet the criteria for Medicare or Medicaid.

The device and pharmaceutical industry is predictably worried about comparative effectiveness.  Undoubtedly, comparative effectiveness would provide pressure on pricing, which is generally lacking when providers and patients pass on costs to third party payers. Transparent comparative effectiveness would give consumers of health care an opportunity to shop for greater value, which will help contain overall costs. 

We Can’t Have it All
This is the unfortunate truth: the growth in health care spending is unsustainable and is making health care unaffordable for average Americans.  In health care, we can have nearly anything we want – we just can’t have everything we want.  Because of escalating costs and limited funding, we need mechanisms to differentiate medical treatments with high value and those with little incremental value.  Without a method to objectively analyze the relative value of treatments, the costs of medical care will continue to rise to unaffordable levels.

-- John E. Brush, Jr., M.D., F.A.C.C.

* Dr. Brush’s post is part of a monthly series of guest posts by ACC leadership. Check back next month to see which ACC leader is sharing his or her thoughts on health care reform!

*** Image from morgueFile (jdurham). ***

UPDATE: The ACC has released this statement on our committment to comparative effectiveness, in response to a recent Bloomberg article on a new coalition called the Partnership for Improving Patient Care, an organization formed to promote comparative effectiveness and comparative effectiveness research (see yesterday's post on the subject "Clinical and Cost Effectiveness: The Bloomberg Boo Boo). It states:

We must develop much-needed randomized clinical trial data to fill many research gaps in the current environment. CE and CER provide a critical means to address these data and research inadequacies. Without a multi-billion dollar ongoing commitment to comparative effectiveness through federal funding, many unanswered vital clinical questions could persist for decades. This is unacceptable.

The College fully understands that cost effectiveness also is important to society and to health care reform and that it is an important parallel process to clinical effectiveness. If we are to distribute scarce resources fairly to the most important scientific and clinical priorities and to all patients regardless of income, we must determine cost effectiveness of care options, once the science is clear, and recognize that marginal increases in clinical effectiveness with very large price tags will not be affordable in a sustainable health system.

It concludes: "There’s no conspiracy here. ACC’s participation in PIPC is consistent with our principles, policies, commitment to ethics and appropriate relationships with industry."

Bloomberg News this week reported a damning story on a new organization called the Partnership for Improving Patient Care (PIPC). PIPC is a benign and positive new coalition (about 4 months old), which industry folks have decided to participate in.

The Bloomberg story suggests PIPC is nothing more than a front for big PhRMA, and actually opposes comparative effectiveness. Wrong. ACC joined PIPC about two months ago (for a nominal membership fee) to be part of a very broad constituency of consumer groups, professional societies and others who believe that comparative clinical effectiveness is a critical priority in health care reform, as well as a process that must be determined in a scientific, unbiased and pure fashion -- and thus not mixed up with cost effectiveness as the hidden goal -- in order to have the respect and trust of physicians and patients.

Other members of this partnership include the American Association of People with Disabilities, American Academy of Nursing, the American Association of Neurological Surgeons, the Association of Clinical Research Organizations, Easter Seals, the National Alliance for Hispanic Health, the AIDS Institute and many more. Some pharmaceutical companies, device manufacturers and related coalitions have recently also joined to support the aforementioned goals. That shouldn’t be surprising. More...

Going after the $20 billion for health information technology (IT), $1 billion for comparative effectiveness research, $10 billion for NIH clinical research, and $2 – $3 billion for prevention activities in the stimulus bill (ARRA) will be a big-time goal for many organizations. It’s time to get in there and grab some dough. The competition for dollars will be fierce, but we have some very sound ideas about how to spend stimulus funds for quality health care. There is an opportunity for registries to be piloted for quality improvement activities. There is still no reimbursement “business case” proposed, but that too could be piloted. Our academic colleagues need to get ready to go after the NIH dollars as well.

The comparative effectiveness research (CER) piece is probably the most controversial. Frankly we would need $1 B annually for 20 years to really get serious about having the massive amounts of additional evidence we need for turbocharging guidelines, performance measures and appropriate use criteria.

Separating cost effectiveness from clinical effectiveness is key to de-politicizing the CER process. Cost effectiveness is critical, but it needs to be done once an objective clinical effectiveness process is completed around any topic. The ACC strongly supports clinical CER. But, one of the risks here -- as shared this week by Avalere Health is that some observers see CER as a way for the federal government to take complete control over guidelines, performance measures and appropriate use criteria. AHRQ shares openly how they did not succeed well in this, when they did try engaging in guideline development a few years back. It costs them too much to produce what we can do with mostly volunteer FACC and researcher efforts; and they got hung up in dangerous political issues in the Congress when various elements of industry didn’t like their results. Better to have a federal agency like NQF or AHRQ look over the shoulders of the profession to vet our guideline efforts, which are bound to be more trustworthy to physicians, and therefore more widely adopted.

Stay tuned, and be certain ACC will be centrally involved in pushing for the best, most objective, and patient centered evidence. We need more of it -- and it’s a important part of the profession’s accountability -- to lead in making that happen! Importantly, we need to clarify to the confused that guidelines are not rigid cookbooks -- see the post last week from ACC past president Jim Dove.

*** Obama signs ARRA into law. Image from Wikimedia Commons (Pete Souza) ***