Medical Experts Skeptical of Many Costly Treatments : Health: ‘Outcomes research’ expands rapidly as analysts try to determine what works best for patients.

WASHINGTON — In Vermont, a child’s chances of undergoing a tonsillectomy range from 8% to 70%, depending on where he or she lives.

In Maine, more than half of the men in some towns have their prostates removed by age 80, while in a neighboring village the rate may be as low as 15%.

Residents of New Haven, Conn., are twice as likely as those in Boston to have heart bypass surgery--but only half as likely to have their carotid arteries cleansed by a high-tech “Roto-Rooter”-like device.

But even more astonishing than these stark differences in treatment patterns are the strikingly similar outcomes: Those who receive the less radical therapies do no worse--and often better--than those treated more aggressively. Sometimes, in fact, the best strategy turns out to be “watchful waiting.”

Such sharp contrasts in practice patterns underscore a growing realization that much of what doctors do is based largely on intuition and guesswork rather than on scientifically valid test results.

But that may change soon. Because of surging health care costs, medical analysts and experts are casting an increasingly skeptical eye on the treatment decisions that doctors make. Many are turning to the rapidly expanding field of “outcomes research” in an effort to determine what works best--and what doesn’t.

The goal of outcomes research is to reduce markedly, if not eliminate, unnecessary and inappropriate medical care, which may account for a third of all such services, experts say. If that can be achieved, despite continued resistance from some doctors, the nation’s health care system might achieve substantial savings.

“Outcomes research may not be the answer to the ills of our health care system. But it sure is an important step. It’s a prerequisite,” says Dr. David Eddy, a nationally known analyst. “The practice of medicine is not based firmly on reality.”

When researchers say that only about 10% of what doctors do is based on scientific test results, “it doesn’t mean that the rest is wrong--it’s just that it isn’t necessarily the best,” says Dr. Brent James, a Salt Lake City medical administrator and researcher.

Even so, notes Dr. Arnold Relman, former editor of the New England Journal of Medicine, “we can no longer afford to provide health care without knowing more about its successes and failures.”

Already, preliminary results assessing the array of treatments for prostate enlargement, a common male affliction after age 65, have led to a sharp drop in the number of surgeries.

Outcomes research, by generating practice guidelines for all physicians to follow, also could help curb the proliferation of malpractice suits. That, in turn, could lead to lower insurance premiums for doctors--and the fees they charge.

“Physicians will be happier when they know they are practicing according to well-founded guidelines,” predicts Eddy, a Duke University professor of health policy management and a senior adviser for Kaiser Permanente in Southern California. “The uncertainty and anxiety of having to make very important decisions with very poor information should decrease considerably.”

Outcomes research is not to be confused with clinical trials. The latter typically involve comparisons of a limited number of technologies, such as two surgical alternatives, or one promising drug against a placebo.

Outcomes research seeks to systematically assess the entire spectrum of treatment alternatives by looking not only at death rates and morbidity but also at patterns of complications, symptoms reduction and quality of life--as reported by patients themselves.

Teams of physicians, statisticians, ethicists and economists are now busily studying the way doctors treat such high-cost and high-risk conditions as back pain, cataracts, ulcers, diabetes, heart attacks, knee replacement, pneumonia and prostate disease--problems that also have highly variable treatment patterns and uncertain outcomes.

Their findings, due in the mid-1990s, “are likely to have a major influence on clinicians’ practice patterns and on third-party payers’ reimbursement policies,” according to a blue-ribbon Institute of Medicine committee.

Outcomes research represents “the coming of age of health services research,” says Dr. David Blumenthal, a member of that panel and senior vice president of Brigham and Women’s Hospital in Boston.

There has been little in the way of outcomes research until now, mostly because researchers simply were unable to keep up with the explosion of advances in medical science and were overwhelmed by the advent of one new technological breakthrough after another.

“There are over 10,000 disease entities, and each has groups and subsets of symptoms. This gets into billions of possibilities,” says Dr. John Williamson, regional director of medical education for the Department of Veterans Affairs in Salt Lake City.

Eddy agrees: “It’s not that physicians are stupid. Medical decisions are complicated, and they are getting more complicated by the day.”

This complexity is compounded by the increasing fragmentation of the health care delivery system, adds Lynn M. Etheridge, a private consultant and longtime Office of Management and Budget analyst. “Seldom is there one person solely responsible for a patient,” he notes.

“Take the issue of breast implants. The surgeons think it’s wonderful. But they’re rarely the ones who see the complications,” Etheridge says.

Three key factors are fueling the rising interest in outcomes research:

--Growing evidence of wide discrepancies in treatment patterns--medicine’s “dirty laundry,” in Eddy’s words.

--The realization that up to a third of therapies are inappropriate, unnecessary--and perhaps harmful. “Clearly, there’s no good scientific studies for a lot of what we do,” says Dr. Robert H. Brook, a RAND Corp. researcher.

--Increasing concern over the runaway cost of health care, which accounted for more than $666 billion last year, or 12.2% of the gross national product. “The issue of waste has moved to the fore because people all of sudden are saying that we don’t have the resources and therefore we ought to be sure the money that we are spending is well spent,” says Rashi Fein, a Harvard health economist.

The primary catalyst in the sudden rush to do outcomes research is Dr. John Wennberg of the Dartmouth Medical School, who first noticed the eye-popping variations in practice patterns as a young federal physician in the 1960s in New England.

In one city in Maine, Wennberg found, the rate of hysterectomies was so high that 70% of the women had their uteri removed by age 75. But in a city less than 20 miles away, the rate was only 25%. Yet there was no perceptible difference in the relative health of the two groups--before or after surgery.

Similar disparities turned up almost everywhere Wennberg looked. In Morrisville, Vt., the explanation for a 65% tonsillectomy rate turned out to be the presence of five aggressive surgeons and family practitioners.

When Wennberg showed them statistics from neighboring communities, where tonsillectomy rates were as low as 7%, they were “astounded,” and then vowed to cut down, he recalls.

“One said he was glad that all this had happened--because there were so many gallbladders that needed tending to,” Wennberg adds.

Other researchers began turning up equally striking regional discrepancies: a sixfold difference in the rate of knee replacements, a threefold difference in hip replacements and, in Washington state, an eighteenfold difference in hospitalization rates among children with intestinal inflammation.

RAND’s Brook zeroed in on carotid endartectomies, a procedure for cleaning out clogged neck arteries that is used on 80,000 Americans each year at a cost of about $10,000 each. An advisory panel concluded that barely a third of more than 1,300 such procedures was appropriate; 32% were borderline; and another 32% should not have been performed.

In a separate study, Brook found that barely half of the 230,000 coronary bypass operations performed each year were clearly appropriate.

And in a comparison of six top hospitals in Massachusetts and California, Harvard Medical School professor Paul Cleary found huge differences in the lengths of stay for nearly 3,000 patients who underwent such procedures as bypass surgery, hip replacement, gallbladder removal and prostatectomy. The different approaches did not affect the outcome of their cases.

More recently, Wennberg, now 57, began focusing on prostate treatment, hoping to eliminate the wide variations in practice patterns among urologists in Maine. He was particularly interested in the split between advocates of surgery for benign prostatic hypertrophy, or BPH, which affects most older men, and proponents of “watchful waiting.”

Amid the raging debate, an international team of experts assembled by Wennberg found that the preventive theory--calling for removal of all or parts of the prostate--was in error.

“We could find no evidence that people would live longer if patients with BPH were operated upon to prevent subsequent development of bladder or kidney obstruction,” Wennberg says. Yet the operation carried with it an inherent risk of complications, including serious infections, diminished cardiac capacity and impotence.

The most volatile finding was the team’s discovery that the most popular type of prostate removal, called transurethral prostatectomy, had a seemingly high mortality rate of 1.3%. A large-scale study is under way in search for an explanation. “It’s still a mystery,” Wennberg says. In recent years, meanwhile, a plethora of new BPH treatments have emerged--drugs, balloon dilation, lasers--that also must be included in the outcomes research.

In the meantime, Wennberg has produced a video that details all the alternatives available to BPH patients. It not only points out the advantages of prostatectomy, such as relieving painful urination; it also informs patients that within three months of an operation, 8% are hospitalized because of complications and 5% become impotent.

After the video was shown recently to several hundred prostate patients in Denver and Seattle, 44% and 60%, respectively, chose “watchful waiting” instead. “Patients seem more risk-averse than surgeons,” Wennberg concludes.

“What works best--that’s the central question we ask,” says Dr. J. Jarrett Clinton, director of the two-year-old Agency for Health Care Policy and Research, which now has a budget of $116 million and funds most of the outcomes research teams around the country.

The preliminary findings of outcomes research performed by other groups also suggest that the effort could significantly reduce unnecessary and inappropriate treatments--and perhaps cut overall health care costs.

A team at the University of Washington has determined that oft-used diagnostic imaging procedures for low-back pain yield very limited information but are costly and occasionally harmful because of radiation exposure. In fact, 90% of patients experiencing low-back pain improve with little or no intervention--and the problem often disappears within six weeks, says Dr. Richard A. Deyo, study director.

Developing practice guidelines is all the more urgent, experts say, because most proposals to reform the U.S. health care system involve some form of uniform package of health insurance benefits. “In order to identify what ought to go into everybody’s benefits package, we need to know what works and what doesn’t,” says Dr. Paul Ellwood, a longtime Minnesota health care reform activist.

The big unknown is the extent to which outcomes research and practice guidelines will help cut overall spending.

“The myth is that if only we could get rid of inappropriate care, we would have enough resources to pay for all effective care. But I’d be surprised if guidelines result in major savings,” says Judith Lave, a professor of health economics at the University of Pittsburgh’s graduate school of public health.

“Some say 30% of all medical care is inappropriate. But it’s also clear that where there’s over-utilization of services, there’s also under-utilization of services,” she adds.

But outcomes research should not be viewed simply as a cost containment tactic, says Wennberg. “It’s a strategy for improving the scientific basis of medicine and the ethical status of the doctor-patient relationship--so decisions can be based on what patients want. If that turns out to save money, it will be all to the advantage of our effort.”

In the final analysis, says James, executive director of the Intermountain Health Care Institute in Salt Lake City, outcomes research is only a tool.

“It’ll only enable us to make wiser decisions regarding the allocation of resources. And that’s not a question the medical profession alone can answer. All we can do is frame the questions. Eventually society will have to answer the question.”