Comparative Effectiveness Research as Patient Education Tool

In this era of 12-figure federal bailouts, a government proposal to spend a billion dollars on something seems pretty ho-hum. But the relatively paltry $1.1 billion that President Obama's budget would put toward comparative effectiveness research has created a firestorm of controversy, with conservative commentators and some providers raising the specter of regulation-happy government bureaucrats intruding into medical decisions that should be left to patients and their doctors.

It's hard to understand how reasonable people could oppose conducting the sort of research that aims to collect data so that we can actually know if one drug or medical device works better than another at fixing the same medical problem. This is good stuff, not only for the medical establishment but also for patients, who are increasingly expected to play an active role in managing and paying for their healthcare. And while I understand the concern that data could eventually be used to deny care if treatment is deemed not effective enough or too pricey, that hardly justifies not doing the research in the first place.

Last week, the news offered a compelling example of how this type of research could be beneficial. Two long-term studies have been evaluating whether the PSA test—the blood test that screens for prostate cancer—actually saves lives. Until now, it has been assumed that it does, and millions of men have been screened and have subsequently undergone surgery and other procedures on the basis of their test results. The side effects of prostate cancer treatment, including impotence and incontinence, are hardly trivial, but many men have been willing to risk them in the hope that they'd avoid an even scarier consequence—death. But what if it turns out that getting the PSA test doesn't necessarily prolong men's lives after all, as one of the studies suggests? This information could be factored into men's decision-making process, and millions might be spared the risk and expense of procedures with unpleasant side effects, while saving our healthcare system millions annually. (My colleague Bernadine Healy offers some analysis of the PSA screening studies.)

At this time, however, comparative effectiveness research enters very little into most of our healthcare experiences. Clinicians still generally make decisions based on often limited research, professional guidelines, and their own experience. They don't have any other options: Comparative effectiveness research exists for only about 30 percent of medical treatments, experts say. "We know macro answers, like whether surgery helps reduce heart attacks," says Jed Weissberg, associate executive director for quality and performance improvement for the Permanente Federation, the umbrella organization representing physician groups at Kaiser Permanente, which has been a pioneer in this area. "But we don't know answers to precise questions, like for different groups of patients, which drugs or which surgeries work best."

Most of the comparative effectiveness research at Kaiser to date has focused on drug therapy. Research Kaiser 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.

But now Kaiser is moving in another direction. It has developed a joint registry database with details about replacement hips, knees, and the like. 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. "Before we had the registry, clinicians were getting the bulk of their information from vendors and manufacturers' representatives," says Weissberg.

Although cost is one of the elements that are often factored into comparative effectiveness calculations, it's not necessarily the overriding one. Last week, Consumers Union, the publisher of Consumer Reports, released a list of "Best Buy Drugs" based on data on the relative effectiveness, cost, and side effects of drugs in more than two dozen drug classes. In many cases, the group recommended brand-name drugs as the best buy, says John Santa, director of the consumer reports health rating center for Consumers Union. Drugs to treat overactive bladder are one example, he says; in the consumer group's judgment, the generic drug's side effects tipped the scales in favor of the brand-name drug.

Seattle-based Group Health Cooperative is another longtime leader in the area of comparative effectiveness research. One way that Group Health uses the information is to involve patients in the clinical decision-making process. The healthcare system recently received a $400,000 grant from the Commonwealth Fund to evaluate how educational materials such as DVDs, booklets, and Web-based videos that discuss medical treatments like low-back surgery, knee replacement, and lumpectomy vs. mastectomy for breast cancer surgery affect patient decision making, among other things. "We have found that if people have information about the comparative effectiveness of testing for prostate cancer, for example, or a back operation, they make better decisions, they're more satisfied, and in general their cost is less," says Eric Larson, executive director of the Group Health Center for Health Studies.