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Better Health Through Better Information

Comparative Effectiveness Research Will Help Deliver Better Medical Care

SOURCE: AP/Denis Poroy

A health researcher conducts tests in San Diego, California.

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Background

How comparative effectiveness will improve health

Point-counterpoint

In the news

The last word

When consumers shop for most products in America, we have the power of information—whether it’s on the Internet or in publications such as Consumer Reports. When making important purchases as informed consumers, we typically try to collect all available data then make the best decision for ourselves and our families. Tools such as Consumer Reports help us make better decisions by gathering the data for us. This well-known journal and website, for example, looks at products in a variety of areas and presents the findings in an understandable and comparable way that helps consumers decide which product is best for them. But isn’t it ironic that good, up-to-date information is readily available to consumers in so many sectors, but not in health care?

An interesting team of authors wrote about this recently in a New York Times op-ed. The unlikely trio of Billy Beane (a former professional baseball player), Newt Gringrich (former Republican Speaker of the House), and John Kerry (senator and former Democratic presidential candidate) used compiling fantasy baseball teams to make their point. They argue that today there is more information available to choose a player for a fantasy baseball team than on the effectiveness of life-and-death medical procedures. Further, they describe how baseball has been improved by better use of data and statistics and suggest that a health care system driven by robust comparative clinical evidence will save lives and money.

But in stark contrast, the quality and quantity of information about medical treatments and services is scarce. Not only does this hamper a consumer’s ability to choose the best product, but it could result in less-than-optimal health outcomes. Comparing the effectiveness of a range of treatments for a condition and assessing how appropriate a treatment is for a person of a certain gender or background is a logical way of closing the information gap that exists in health care today.

Comparative effectiveness research does just that. It evaluates different drugs, medical devices, and clinical procedures for the same illness against each other. In contrast, the bulk of research done today examines whether a specific treatment works compared to doing nothing, but comparative effectiveness research evaluates which therapy works best among a range of possibilities for the same illness or condition.

This research could compare similar treatments, such as competing drugs or a brand new drug compared with a tried-and-true generic. Or it could match up very different therapies for the same condition, such as major surgery compared to taking a medication. It sometimes even evaluates how well a medical procedure works (such as a taking a drug for diabetes or cholesterol) compared to a more behavioral approach (like changing diet).

With the volume of new medical devices, drugs, and other treatments coming into the U.S. health care system, sorting through the details of each product can be a cumbersome task. Health care providers obviously want to choose the best remedy for their patients based on evidence, not anecdotes or (potentially worse) solely on information received from companies trying to promote their product. Thus, comparative effectiveness research will fill a massive information gap that has left health professionals and patients without the proper evidence to assess which treatments work best for a given condition.

The upshot: Equipping patients and the entire health community with the most accurate medical research available will enable them to make the best possible clinical decisions, improve care quality, and avoid ineffective and harmful health outcomes.

What’s more, a better understanding of which medical treatments work and which don’t could save money. It’s estimated that one-third of procedures and treatments administered in the United States have no proven benefit and account for up to $700 billion annually in current spending. Moreover, some of these treatments can have harmful side effects, produce worse health outcomes, and then, as a result, add to the soaring costs of medical care.

Congress and the Obama administration have recognized the importance of this research by investing $1.1 billion in comparative effectiveness research as part of the $787 billion American Recovery and Reinvestment Act. Despite broad support for this kind of research among the American people, health care providers, and much of the health care industry, there has been vocal opposition to comparative effectiveness research based on fears that the government will intrude on the patient-provider relationship and coverage policies.

Some of these concerns can more accurately be labeled fear mongering by extreme right-wing conservatives determined to derail health reform using any means available. But more thoughtful critics worry that the new research may not take the specific needs of patients into consideration.

Supporters argue comparative effectiveness research will produce findings that will improve health outcomes and enhance provider-patient decision-making power.

This brief will provide an overview of the history of comparative effectiveness research and some of its recent successes, how it will be and should be expanded to improve health care delivery, and how this research can be carried out to address critics’ concerns.

Background

Comparative effectiveness research is not a new concept. For decades the medical community has tried to systematically evaluate how different remedies work for the same condition. This kind of research is expensive but when conducted it has proven very effective.

Consider the case of heart stents—small mesh tubes that are surgically placed in a coronary artery to keep the vessel open allowing blood to flow freely to the heart. Heart stents are a revolutionary advancement that have saved countless lives since their introduction in 1986, but they are not always the best treatment for many patients with heart disease. After years of use researchers started comparing the use of stents (where a catheter is routed up a large blood vessel in the groin and the wire stent is inserted to expand an artery) to other less invasive and less expensive treatments such as medication regimes.

One study published in the New England Journal of Medicine found it was just as effective to treat many heart attack patients with drugs instead of stents—and it was far less expensive. Another NEJM study showed that unnecessary stenting was leading to thousands of heart attacks and deaths each year. These misjudgments do not come without a cost. The 100,000 heart attack patients in the United States that do not need stents could translate to annual savings of $700 million.

How comparative effectiveness will improve health

The greatest potential for comparative effectiveness research lies in how it helps the entire health care profession make the best evidence-based decisions to help people get better and stay healthy. While our health professionals are well trained and rigorously credentialed to provide high-quality health care, they often do not have the most up-to-date information on what works best for certain patients and conditions.

Because of the volume of new products on the market and the dynamic nature of health care treatments, the dissemination of information and the adoption of best practices by health professionals is challenging. In addition, scientific peer-reviewed information is competing with promotional advertisements of new products funded by their manufacturers. Without information that accurately compares treatments, health professionals cannot provide the best evidence-based remedies for many kinds of patients.

Today when a new product is released, such as Ambien, the sleeping pill advertised widely on late-night TV, the information the public and the medical community receives comes directly from the manufacturers of that product. So in addition to being shared aggressively with health care providers, messages about the product are also broadcast widely on the airwaves in direct-to-consumer TV advertising paid for by pharmaceutical and medical device companies that manufacture the product.

In contrast, objective research is more difficult to find. Nonindustry funded research is released in more academic forums such as at professional conferences and in peer-reviewed journals—oftentimes months or years after the release of a new medical product. Not surprisingly, these findings are far more difficult to find and consequently less quickly incorporated into medical practices. Health care providers do their best to keep up with the latest research but the quantity of new information makes this a challenge and is much less immediate. Compare this to the TV drug ads where pharmaceutical companies spent about $4.7 billion in magazine and television advertising in 2008 to let the public know about their new products.

More troubling still, there is simply not enough of this kind of objective comparative research done in the United States today. Without a greater volume of rigorous research that compares the range of treatments available for a given condition that is readily accessible, patients and providers will not have the accurate, unbiased, evidence-based information they need to make informed treatment decisions.

It is important to note that this type of research is not about finding the single best option for everyone. Instead, it is about helping health care providers and their patients find the best treatment for each individual. Comparative effectiveness research is similar to the research done by Consumer Reports, the popular magazine and website. Consumer Reports works to promote a fair and safe marketplace for consumers by empowering individuals with the information they need to make the best decisions. In fact, the organization just launched a new project, Best Drugs for Less, which identifies the best drugs for over 20 common conditions including diabetes, depression, insomnia, high blood pressure, and high cholesterol. The drug ratings are based on comprehensive reviews of the scientific evidence by teams of medical experts. Best Drugs for Less is a great resource, but is reliant on scientific data generated by independent researchers. Thus, even services such as this need more and better research to draw upon.

Comparative effectiveness research will help us decipher the effects of different treatments for specific individual needs and enable us to work with the medical team to choose what’s best. Again, the goal is not to find a single best treatment for everyone, but to weed out the therapies that don’t work and use treatments that meet the needs of individual patients.

Federal role

Comparing many different medical treatments is expensive and obtaining the funds to conduct the research is often difficult since most research on effectiveness is paid for by the developers of new treatments. There is no incentive for the companies to fund research that compares their treatment to another since it is not in their best interest to determine if another treatment works as well or better than theirs. Because of this the federal government must invest in this research.

Carolyn Clancy, M.D., the director of the Department of Health and Human Service’s Agency for Healthcare Research and Quality since 2003 (and in leadership at the agency since 1990), has made the same point, and argues that the federal government should help to fund comparative research since drug and device manufacturers do not have an interest in paying for a trial that may prove their drug is not as good as a competitor’s.

If the government does not facilitate an expansion of comparative effectiveness research, it will only be produced in limited quantities and patients and providers will continue to have inadequate information when making many treatment decisions. This investment has the potential to create a more seamless information exchange, where clinical information on medical services could be readily accessible to providers and patients.

To help address this funding inadequacy, the federal government is now helping to bridge the gap and pay for it. In the Medicare Modernization Act of 2003, Congress authorized $15 million to conduct comparative effectiveness research through the Agency for Healthcare Research and Quality. Priority was given to research that would help to determine the effectiveness of various health services, including prescription drugs. The Obama administration continued this commitment by including $1.1 billion in the American Recovery and Reinvestment Act for comparative research earlier this year.

The Recovery Act funds both the development and the dissemination of comparative effectiveness research. It will support new studies or synthesize findings from existing research that compares the clinical outcomes, effectiveness, and appropriateness of treatments for various diseases and health conditions. It will also make sure the findings from this research can be readily accessed by providers and patients through the development and improved use of clinical electronic data. It is important to note that this legislation does not include any guidance for payment, coverage, or treatment.

This legislation also directed the Institute of Medicine, part of the country’s National Academies of Science, to recommend national priorities for this new research. The report, as required by the legislation, was issued on June 30. In order to maximize the use of the findings from new comparative research studies, many of the research priorities the IOM recommended relate to conditions that are most common among Americans such as heart disease, diabetes, and cancer. As directed, the IOM identified the 100 highest-priority research topics on which comparative effectiveness research should focus.

The IOM report prioritized conditions that disproportionately affect certain subgroups of patients who may have been left out of previous research studies. For instance, the IOM recommends comparing how well different types of interventions work for different conditions, such as what might work best to reduce incidences of infant mortality, preterm births, and low birth rates—especially among African-American women who have higher rates of these conditions than other women. The authors note that the best treatment may not even be a medical intervention; in addition to better prenatal care they also suggest examining the effect of behavioral interventions such as nutritional counseling, smoking cessation, and substance abuse treatment. Prioritizing in this way ensures that comparative effectiveness research will include groups of patients that may have been left out of other research studies while addressing a broad range of conditions and treatments.

The debate

Comparative effectiveness research is now under fire despite its critical importance as objective research. Critics worry about the government’s role in determining best practices. They assert that the government will use the findings to intrude on patient-provider relationships. They worry that the research will not account for variations in patient health statuses and backgrounds. And they are concerned that the findings will be used to make coverage determinations.

But most stakeholders, ranging from insurance companies to consumer advocates and health care provider groups, generally support the need for more and better comparative effectiveness research and applaud the recent federal investment. The following are the most common concerns voiced by the opponents of comparative effectiveness research.

First, opponents fear that the government will use the research findings to dominate the doctor-patient relationship by influencing medical decision making. They speculate that comparative research will dictate the plan of care for health care providers, who will then lose decision-making authority.

In fact, the opposite is true. Far from constraining the way medicine is practiced, comparative studies will enhance the decision-making power of patients with their providers by giving them both the tools to make the best clinical decisions. Further, based on how comparative effectiveness studies are designed, they will help to clarify which information is most meaningful in making clinical decisions for specific patient populations.

Second, opponents worry that the new research will not account for variations in patient health status and background. They claim it will lead to “one-size-fits-all” treatments that apply to an “average” patient, neglecting the specific needs of others. The results of existing comparative studies and the proposed recommendations from the Institute of Medicine should help to quell these fears. The IOM has specifically recommended the nation prioritize funding research that includes many different patient populations, in many different settings, and includes many different types of treatments, including behavioral, nonmedical approaches.

Indeed, expanded comparative effectiveness research will create a much more extensive information base, enabling health care providers across the medical spectrum to more accurately tailor treatment strategies for individual patients. This is precisely the kind of research information not provided by pharmaceutical companies or medical device companies when they test their products and then submit the results to the Food and Drug Administration for approval.

Initial studies that prove a new product is effective compared to doing nothing usually only include patients who fit the stringent criteria necessary to win approval for the new product from the FDA. Research participants, for example, might be of a certain age and not have any other disease than the one the researchers are studying. In the real world, of course, many patients have multiple chronic diseases simultaneously. They is really nothing like the controlled group of patients included in an original study.

Depending on the way comparative effectives studies are designed, they could be comprised of many different types of patients, including those that do not typically participate in many clinical trials. For example, one recent study showed that the best treatment for patients with coronary artery disease depended on their age. Heart bypass surgery was a much better choice for older patients (over 65 years of age) and the less invasive angioplasty was a much better treatment decision for patients who were under 55 years old.

This type of differentiation is important not only for patients with varying risk characteristics and coexisting conditions but also for women, members of minority groups, and others who have historically been underrepresented in clinical trials. Current efforts to compare treatments aim to ensure that much more useful data will be collected and that better methods will be developed for understanding differences in effectiveness among different patient groups.

Comparative effectiveness research can therefore accelerate the discovery of approaches to individualized medicine and help providers cater to the specific needs of patients. This is exactly the approach the Institute of Medicine is taking. In its report on national priorities for comparative effectiveness research, IOM recommends research involving very precise subgroups of patients, with the studies done in nontraditional health care settings such as the effect of community practice-based screening for breast can­cer in high-risk women of different ages, risk factors, and races or ethnicities.

Finally, critics are concerned that the findings from comparative effectiveness research will be used to deny coverage of certain more expensive health care treatments. They speculate that cost considerations will take precedence over what is deemed the best mode of treatment for a given patient.

None of the legislation passed or being discussed contains language that would allow the findings from comparative research to be used to mandate coverage, reimbursement, or other policies for any public or private payer. Rather, the purpose of the new research is to enhance the way medical care is delivered, not impede it. Giving patients and providers the tools with which they can better access treatment options and tailoring clinical strategies to individual patient needs will only improve the quality of options available to patients.

Conclusion

The most important goal of comparative effectiveness research is to help doctors and patients work together to make the most appropriate, evidence-based decisions when approaching treatment options. Today there is just not enough information available to know what works and what doesn’t, leaving the public and the medical community ill equipped to make many important clinical decisions.

We need scientifically sound, evidence-based research on a full range of treatment options for medical conditions that can be easily understood by doctors and patients alike. Health care experts, including health professionals, policymakers, and researchers believe that real-life findings from comparative effectiveness research will enhance the decision-making power of health care providers and their patients by improving the quality and quantity of information available on various treatment options.

Further reading

"Research on the Comparative Effectiveness of Medical Treatments." Congressional Budget Office, 2007.

Alliance for Health Reform "Comparative Effectiveness: Better Value for the Money?" August 2008

Alan M. Garber and Sean R. Tunis, "Does Comparative-Effectiveness Research Threaten Personalized Medicine?" New England Journal of Medicine 360 (19) (2009): 1925-27.

Jerry Avorn, "Debate About Funding Comparative-Effectiveness Research." New England Journal of Medicine 360 (19) (2009): 1927-29.

Committee on Comparative Effectiveness Research Prioritization and Institute of Medicine, Initial National Priorities for Comparative Effectiveness Research (Washington: National Academies Press, 2009)

Point-counterpoint

Point Counterpoint The Bottom Line
The government will use comparative effectiveness research to intrude on the patient-provider relationship. Comparative effectiveness research will provide additional information and help you and your doctor decide together which treatments work and which do not—based on the evidence, not anecdote. Today much of the information about new treatments comes from the manufacturers of the products. Comparative effectiveness research will enhance the decision-making power of patients and providers by giving them the tools to make the best clinical decisions.
Comparative effectiveness research will lead to one-size-fits-all medical guidelines that are based on broad population averages, ignoring the differences between patients. Most research performed today examines very specific patient populations that fit strict criteria for entry in the clinical trials necessary to test a drug or medical device before approval by the Food and Drug Administration. These clinical trials do not compare the treatments in different subgroups in ways comparative effectiveness research can accomplish. Comparative effectiveness research can create a more expansive information base, enabling providers to tailor treatment strategies more precisely for individual patients.
Findings from comparative effectiveness research will be used by the federal government to deny individuals coverage of necessary health services. Comparative effectiveness research examines the science to make sure the choices you have to treat your illness are the best for you. Giving patients and providers the tools with which they can better access treatment options and tailor clinical strategies to the individual needs of patients will improve the quality of options available to patients.

In the news

Ruth Faden and Jonathan D. Moreno, "Power for Patients: Comparative Effectiveness Research Will Help People Make Better Health Choices." Baltimore Sun, 2009.

Robert Pear, "U.S. To Compare Medical Treatments," The New York Times, 2009.

Avram Goldstein, "Medical Scans Waste $30 Billion a Year, Insurers Say," Bloomberg, 2008.

Janet Rae-Dupree, "Disruptive Innovation, Applied to Health Care," The New York Times, 2009.

Uwe E. Reinhardt, "How Appropriate Is Your Medical Care?" The New York Times, 2009.

The last word

From a New York Times op-ed by Billy Beane, Newt Gingrich, and John Kerry about comparative effectiveness:

“A doctor today can get more data on the starting third baseman on his fantasy baseball team than on the effectiveness of life-and-death medical procedures. Studies have shown that most health care is not based on clinical studies of what works best and what does not—be it a test, treatment, drug, or technology. Instead, most care is based on informed opinion, personal observation, or tradition… Similarly, a health care system that is driven by robust comparative clinical evidence will save lives and money.”

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