Evidence Based Medicine

Are Doctors Just Playing Hunches?

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Nobody pretends medicine is easy, but if there's one thing we ought to be able to rely on, it's that the doctors looking out for us are doing more than playing hunches. We take certain medicines because they work, right? We go into the operating room for certain procedures because they'll make us well, don't we?

Well, maybe. More and more, however, doctors are making the unnerving case that no matter how reliable a drug or other treatment appears to be, too often there's simply little hard evidence that it would make a long-term difference in a person's quality of life or prolonged survival. Obviously, drugs are tested rigorously to show that they are safe and effective before they are approved by the U.S. and other developed countries. But a clinical study is not the real world, and just because a drug leads to a statistically significant improvement in, say, cholesterol levels doesn't guarantee that the desired effect--a healthier heart and a longer life--will follow. Often your doctor is left to make prescription decisions based at least in part on faith, bias or even an educated guess. That ought to be enough to spook even the least jumpy patient, but the fact is, recognizing just what a roll of the dice medicine can be may be a good thing.

Increasingly, doctors seeking to provide their patients with the best possible care are exploring what is known as evidence-based medicine--a hard, cold, empirical look at what works, what doesn't and how to distinguish between the two. It's not enough to prove that a particular blood test or CT scan really spots cancer, for example. You also need to know whether early detection of that cancer would make a difference in your ability to respond to treatment or it merely means that you would die at the same point but learn about your illness earlier than you would have without the test.

Evidence-based medicine, which uses volumes of studies and show-me skepticism to answer such questions, is now being taught--with varying degrees of success--at every medical school in North America. It has been extraordinarily successful in shooting down some of the most cherished beliefs in health care, like the idea that long-term hormone-replacement therapy would help prevent heart disease in women. And it has clearly saved lives. Many doctors used to give anti-arrhythmia drugs to everyone who experienced irregular heartbeats after a heart attack because severely irregular beats could rapidly prove fatal. But then came the results of a randomized trial showing that patients with only mildly irregular heartbeats were more likely to die if given the anti-arrhythmia medication than their untreated counterparts were. Doctors now prescribe more judiciously, though treatment still saves lives in the case of severe arrhythmias.

Advocates believe that evidence-based medicine can go much further, reducing the reliance on expert opinion and overturning the flawed assumptions and even financial incentives that underlie many decisions. "This is a whole way of looking at the world," says Dr. Gordon Guyatt of McMaster University in Hamilton, Ont., who coined the term and is a pioneer of the evidence-based movement.

But is such certainty possible--or even desirable? Medicine, after all, is a personalized service, one built around the uniqueness of each patient and the skilled physician's ability to design care accordingly. "I'm worried about training a generation of physicians who don't have the other skills they need for the optimal practice of medicine," says Dr. Mark Tonelli, a pulmonary-care specialist at the University of Washington in Seattle. "They can read the scientific literature, understand the statistics, but they don't understand how that should influence their treatment of the individual in front of them." What's more, some insurance companies have been very aggressive in using evidence-based arguments to deny payment for untested treatments--a circular problem, because how do you create the evidence the insurers demand unless you test the untested?

Whatever the merits of evidence-based medicine, it got off to a rocky start. When Guyatt began championing it back in the 1990s, he called it "scientific medicine," but he learned quickly that if you want to start a revolution, it helps to pick the right slogan. Many of his colleagues were outraged by the implied insult to their expertise. So he quickly went with "evidence-based," and tempers cooled.

Guyatt's ideas complemented the work of the Cochrane Collaboration, an international network of researchers, physicians and others that was founded in 1993 to systematically gather and evaluate the knowledge found in medical research. The organization aggregates all published scientific studies on a particular treatment question to get a sense of the field. Then reviewers carefully consider the design of the research to determine just how strong the evidence is. One of their most famous reports was a 2005 finding based on 139 studies showing that there was "no credible evidence" that the vaccine against measles, mumps and rubella was involved in the development of either autism or Crohn's disease.

Guyatt and another doctor, David Sackett, wanted to go a step further by making sure doctors used the evidence that was collected and ranked. Many physicians began doing just that, but there have been a few nasty surprises.

Consider the case of Dr. Daniel Merenstein, a family-medicine physician trained in evidence-based practice. In 1999 Merenstein examined a healthy 53-year-old man who showed no signs of prostate cancer. As he had been taught, Merenstein explained to his patient that there are advantages and disadvantages to having a blood test for prostate-specific antigen (PSA). The test can lead to early detection of prostate cancer but also to unnecessary biopsies and even treatment--with all its attendant risks of impotence and incontinence--for a cancer that might have grown so slowly that it didn't need immediate attention. And for aggressive prostate cancers, there is little evidence that early detection makes a difference in whether treatment could save your life. As a result, the patient did not get a PSA test.

Even champions of evidence-based practice acknowledge that the approach has limits. "Some things can't be tested in randomized trials, and some things are so obvious, they don't need it," says Dr. Paul Glasziou, director of the Center for Evidence-Based Medicine in Oxford, England. There have never been randomized trials to show that giving electrical shocks to a heart that has stopped beating saves more lives than doing nothing, for example. Similarly, giving antibiotics to treat pneumonia has never been rigorously tested from a scientific point of view. It's clear to everyone, however, that if you want to survive a bout of bacterial pneumonia, antibiotics are your best bet, and nobody would want to go into cardiac arrest without a crash cart handy. "Where randomized trials are most important is where you're trying to affect a long-term condition, like stroke or cancer," Glasziou says.

Finally, the very definition of evidence-based medicine is something of a moving target. Physicians who encouraged their female patients to take hormone-replacement therapy to prevent heart problems later on were practicing a kind of evidence-based medicine, since the best available evidence at the time--observational studies and the like--suggested a benefit. Of course, when a randomized controlled trial showed otherwise, the advice changed. Even at that, the case is not entirely closed. Some researchers now believe there may be a window of opportunity right around the years of menopause during which hormone-replacement therapy could help the heart. Proving that would, naturally, require another study.

All the same, few people deny that the trend in medicine is increasingly to be guided, if not governed, by the data--an idea that is spreading to other fields as well. Evidence-based practice is now being taught in nursing, general education and even philanthropy, thanks to the influence of the Bill and Melinda Gates Foundation, a results-based group if ever there was one. You could see even the political fights over global warming as the birth pangs of the new practice of evidence-based policy.

But it is in medicine that the practice will have the most emotional impact. All patients would probably benefit if their doctors were abreast of the latest data, but none would benefit from being reduced to one of those statistical points. "You have to be able to take a good history and do a physical examination," Guyatt says. "And you have to have an understanding of patients' values and preferences." As much as some physicians might wish it otherwise, there is still as much art to medicine as there is science.

Shame:
A Major Reason Why Most Medical Doctors
Don't Change Their Views

In the 1960s the results of a large randomized controlled study by the University Group Diabetes Program showed that tolbutamide, virtually the only blood sugar lowering agent available at the time in pill form, was associated with a significant increase in mortality in patients who developed myocardial infarction. The obvious response from the medical profession should have been gratitude: here was an important way to improve the safety of clinical practice. But in fact the response was doubt, outrage, even legal proceedings against the investigators; the controversy went on for years.

Why?

An important clue surfaced at the annual meeting of the American Diabetes Association soon after the study was published. During the discussion a practitioner stood up and said he simply could not, and would not, accept the findings, because admitting to his patients that he had been using an unsafe treatment would shame him in their eyes. Other examples of such reactions to improvement efforts are not hard to find.

Indeed, it is arguable that shame is the universal dark side of improvement.

After all, improvement means that, however good your performance has been, it is not as good as it could be. As such, the experience of shame helps to explain why improvement, which ought to be a "no brainer", is generally such a slow and difficult process.

What is it about shame that makes it so hard to deal with? Along with embarrassment and guilt, shame is one of the emotions that motivate moral behavior. Current thinking suggests that shame is so devastating because it goes right to the core of a person's identity, making them feel exposed, inferior, degraded; it leads to avoidance, to silence.

The enormous power of shame is apparent in the adoption of shaming by many human rights organizations as their principal lever for social change; on the flip side lies the obvious social corrosiveness of "shameless" behavior.

Despite its potential importance in medical life, shame has received little attention in the medical literature: a search on the term shame in Medline in November 2001 yielded only 947 references out of the millions indexed. In a sense, shame is the "elephant in the room": something so big and disturbing that we don't even see it, despite the fact that we keep bumping into it. An important exception to this blindness to medical shame is a paper published in 1987 by the psychiatrist Aaron Lazare which reminded us that patients commonly see their diseases as defects, inadequacies, or shortcomings, and that visits to doctors' surgeries and hospitals involve potentially humiliating physical and psychological exposure.

Patients respond by avoiding the healthcare system, withholding information, complaining, and suing. Doctors too can feel shamed in medical encounters, which Lazare suggests contributes to dissatisfaction with clinical practice. Indeed, much of the extreme distress of doctors who are sued for malpractice appears to be attributable to the shame rather than to the financial losses. Also, who can doubt that a major concern underlying the controversy currently raging over mandatory reporting of medical errors is the fear of being shamed? Doctors may, in fact, be particularly vulnerable to shame, since they are self-selected for perfectionism when they choose to enter the profession. Moreover, the use of shaming as punishment for shortcomings and "moral errors" committed by medical students and trainees such as lack of sufficient dedication, hard work, and a proper reverence for role obligations probably contributes further to the extreme sensitivity of doctors to shaming.

What are the lessons here for those working to improve the quality and safety of medical care?

Firstly, we should recognize that shame is a powerful force in slowing or preventing improvement and that unless it is confronted and dealt with progress in improvement will be slow. Secondly, we should also recognize that shame is a fundamental human emotion and not about to go away. Once these ideas are understood, the work of mitigating and managing shame can flourish.

This work has, of course, been under way for some time. The move away from "cutting off the tail of the performance curve" that is, getting rid of bad apples towards "shifting the whole curve" as the basic strategy in quality improvement and the recognition that medical error results as much from malfunctioning systems as from incompetent practitioners are important developments in this regard.

They have helped to minimize challenges to the integrity of healthcare workers and support the transformation of medicine from a culture of blame to a culture of safety.

But quality improvement has another powerful tool for managing shame. Bringing issues of quality and safety out of the shadows can, by itself, remove some of the sting associated with improvement. After all, how shameful can these issues be if they are being widely shared and openly discussed?

Here is where reports by public bodies and journals like Quality and Safety in Health Care come in. More specifically, such a journal supports three major elements autonomy, mastery, and connectedness that motivate people to learn and improve, bolstering their competence and their sense of self worth, and thus serving as antidotes to shame.