Comparative Effectiveness:  a  comparison of the impact of different options that are available for treating a given  medical condition for a particular set of patients. Such studies may compare  similar treatments, such as competing drugs, or they may analyze very different  approaches, such as surgery and drug therapy. The analysis may focus only on the relative medical benefits and risks of each option, or it may go on to weigh both the costs and the benefits of those options.

I am afraid that by focusing so much on HITECH and on the definition of Meaningful Use and Certification on this blog we have been missing on the most important part of the “ARRA” stimulus package , the initial $1.1 bn. funding of Comparative Effectiveness Research (CER) to be spent by Sep. 2010. Specifically some of the $400 millions to be used at the discretion of  the DHHS Secretary that must be allocated to encourage the development and use of clinical registries, clinical data networks, and other forms of electronic health data that can be used to generate or obtain outcomes data. It certainly looks like the national  implementation of EHRs may become fully intertwined with CER.

The minimum definition of “Meaningful Use” as defined in ARRA includes e-prescribing, electronic exchange of medical information and interoperability. These are also the necessary elements to start implementing a national CER strategy.  There is just too much at stake this time to think it won’t happen.

e-Patients should become informed about the potential impact of CER for future care. Please read the summary of the “Listening Session of the Federal Coordinating Council for Comparative Effectiveness Research” from April 14, 2009 to get a better understanding why we, the patients & patient groups, must get directly involved in helping to develop the CER private/public infrastructure & activities.

The Administration’s Real Focus: CER

Peter Orszag, White House Budget Director, produced a study on CER (“Research on the Comparative Effectiveness of Medical Treatments“)  when he was director of the Congressional Budget Office (CBO).  He held that position during 2007 and 2008, where he emerged as a forceful advocate of controlling entitlement spending and improving the effectiveness of the health system. His current position puts him in the center of the Obama administration’s health policy circle.  Twice this week he has spoken about CER. First in an NPR interview on May 12:

Some of the cost-saving measures discussed by the coalition representatives involve computerizing medical records and coordinating care, but other initiatives are more controversial — including the use of comparative effectiveness research in patient care.

A few months ago, as part of ARRA there was controversy over the use of CER and yesterday at the meeting with the President the incoming head of the AMA and the representative of the pharmaceutical industry both embraced CER.”

This was followed by an opinion piece in the Wall Street Journal:

How can we move toward a high-quality, lower-cost system? There are four key steps:

1. health information technology, because we can’t improve what we don’t measure;
2. more research into what works and what doesn’t, so doctors don’t recommend treatments that don’t improve health;…

Why is this so significant? What follows are a few quotes from the 2007 CBO document produced under his supervision:

[..]The rate at which health care costs grow relative to income is the most important determinant of the country’s long-term fiscal balance; it exerts a significantly larger influence on the budget over the long term than other commonly cited factors, such as the aging of the population or the coming retirement of the baby-boom generation. Rising health care costs represent a challenge not only for the federal government but also for private payers.

In addition, because federal health insurance programs play such a large role in financing  medical care and account for such a large share of the budget, the federal government itself has an interest in generating evaluations of the effectiveness of different approaches to health care.

[..]Generating evidence that compares treatments is what research on “comparative effectiveness” does.

Orszag may have been the CER main proponent until now but there is clearly a concerted effort by the administration to ramp up public mentions of CER. During the Senate hearings on her confirmation, HHS Secretary nominee, Kansas Gov. Kathleen Sebelius, spoke of her support for the use of electronic medical records as a way of data mining patient information:

EHR data is crucial to conduct “comparative effectiveness research [CER] to provide information on the relative strengths and weaknesses of alternative medical interventions to health providers and consumers.”

AHRQ (Agency for Healthcare Research and Quality) is the lead agency of the federal government doing CER since 2005 and has been allocated $300 million for CER. Carolyn M. Clancy, M.D., Director of AHRQ has been speaking about CER for a long while. In a 2007 congressional testimony she said:

I would like to mention briefly the role of health IT, which will make it easier for researchers to gather information for their research and for users of research findings to get information in real time when they need it.  The health care system’s growing investments in health IT provide us with an unprecedented opportunity for redefining the possibilities of observational studies, accelerating and targeting the uptake of relevant information, and providing feedback to the biomedical enterprise itself.

Health IT will make it possible for research to answer the pressing questions facing the health care system more quickly and efficiently.  In the future, health IT will provide us with the vehicle for transforming our health services research enterprise so that we can evaluate the effectiveness of interventions and treatments in real time as a byproduct of providing care.

Why The push To Use CER?

There are so many more clinical options now than ever before, with new products and services flowing into the market, that questions about evidence of effectiveness, quality, and value have reached unparalleled intensity.

Until now most medical evidence has been derived from randomized controlled trials (RCT). However, there is growing recognition that most questions relevant to everyday clinical practice cannot be addressed by RCTs. There is a real need to devise other means of creating evidence in a timely manner. There is clearly a major interest by many to extract evidence from electronic health records (EHR) data and use that evidence to conduct CER studies. Integrated systems like Geisinger or Kaiser Permanente have not been waiting for Federal funding to start conducting such studies.

For Kaiser, “the goal is to figure out under what circumstances is a given therapy best for certain patients“. To achieve that, a team of pharmacists, doctors and other researchers have been conducting CER, “scouring the globe” for data on how well medications, devices and procedures work. To date, most of the comparative effectiveness research has focused on drug therapy. Research it has conducted on various cholesterol-lowering drugs, for example, has led Kaiser to recommend that its clinicians prescribe generic simvastatin as a first-line treatment for patients with heart disease or diabetes. If that doesn’t work, the dose is doubled. If that doesn’t get the patient’s cholesterol under control, the guidelines suggest adding other drugs.

By its own estimates Kaiser, using data from the CER, has saved so far:

• $70 million on treatments for high blood pressure,
• $80 million on antidepressants and
• about $100 million on cholesterol-lowering medicines.

And now it is expanding the CE program, using an in-house joint registry database with details about replacement hips, knees, and other similar surgical procedures. Clinicians and their patients can consult the database to help them decide which device is most appropriate, given each patient’s age and level of activity. A risk calculator tells patients what they can expect in terms of likelihood of infection and replacement joint failure based on Kaiser’s experience with a particular device. There is at least one clear advantage for this database. Before the registry, clinicians were getting the bulk of their information from vendors and manufacturers’ representatives.

The Public View On EHR/CER

A recent NPR/Kaiser Family Foundation poll shows that the American public is surprisingly more positive about the potentials of EHRs than most professionals. People already are familiar with computerized information and accept its risks.

People do their banking. They certainly do shopping these days by electronic means, and so in any of those cases you find people concerned about their privacy, but people are still doing it. They see enough of an upside for the convenience.

Mollyann Brodie, director of Kaiser Family Foundation’s Public Opinion and Survey Research program (NPR.org, 4/22).

It may be that the public is unaware of the kind of data mining that is already one with their financial information. It puts to shame any potential use of EHR data for CER! Read the NY Times article “What Does Your Credit-Card Company Know About You?”

When it comes to CER this poll also found that

• a majority of U.S. adults (55%) would trust a panel of experts from an independent scientific organization to make recommendations about which treatments should be covered by insurers, but
• fewer people (41%) would trust if the panel’s experts are described as being “appointed by the federal government.”

Mollyann Brodie said that people are concerned that their physicians would not have control over their health decisions.

People are really trusting of their individual doctors, and a lot of experts might say that that’s, you know, misplaced trust, that the doctors don’t have all the information they need. But it is the individual doctors that the public most trusts to make these decisions.

That corresponds to the data from the Pew Internet Project.

So, one thing is clear: the way comparative effectiveness research is framed is critical to how it is likely to be received. If it is seen as simply another attempt by payers to control costs, doctors, patients and the public at large are likely to resist the concept.

Although the final text of ARRA removed earlier drafts references to comparative cost-effectiveness we can be quite certain that this will come back. Comparative cost-effectiveness analysis, brings economics into the equation and seeks to establish which of several therapeutic strategies is capable of achieving a given therapeutic goal at the least-cost strategy. No wonder it has had such forceful opposition from most professional medical societies and the pharmaceutical industry!

You can be sure that we haven’t seen yet the big fight over healthcare reform. And you can be sure it will be directly related with the implementation of CER and it’s use to help us decide what procedures should be used to treat patients, individually, locally or nationally.