SCIENCE / MEDICINE : Doubt Grows Over Reliance On Clinical Trials
For decades, medical progress has been determined by the results of clinical trials, in which a new treatment such as an AIDS drug or a clot-dissolving drug for heart attack patients is carefully compared with existing treatments under ideal experimental conditions. Once a new therapy is shown to work under such circumstances, it is often accepted into medical practice.
But in recent years, some of the nation’s leading physicians have become convinced that too much emphasis is being given to the results of such trials. This is because treatments that work in ideal circumstances for carefully selected patients may not be effective in average circumstances for unselected patients.
Two examples illustrate this point. They were cited at a recent conference organized by the Institute of Medicine and sponsored by the U.S. Health Care Financing Administration.
First, almost all studies of new heart attack treatments, such as clot-dissolving drugs, exclude patients over age 65. As a result, it is impossible to apply the results of such studies to older patients. Physicians do not know whether the elderly are just as likely to benefit from clot-dissolving drugs as younger patients or more likely to suffer life-threatening complications, such as bleeding into the brain.
Second, there are large variations in the frequency of many operations, such as heart-bypass surgeries and hysterectomies, in different areas of the country and different parts of large cities. Physicians do not know whether patients in some areas are being over-treated or under-treated in others.
“For the first time, there is a consensus in the medical community . . . that one has to know what actually happens in the practice of medicine rather than to look at the results of clinical trials in major academic centers,” said Dr. Kenneth I. Shine, the dean of the UCLA School of Medicine and the chairman of the Institute of Medicine committee that has been investigating the problem.
Shine and others advocate a two-step approach--clinical trials of new treatments under ideal conditions followed by broadly based “effectiveness” studies to determine how the treatments perform in daily practice.
Speakers at the conference also said that physicians know relatively little about the non-medical outcomes of different treatments, such as which treatments are most likely to help heart attack patients return to work or to help hip fracture patients resume independent lives. They urged physicians to incorporate assessments of the social and psychological outcomes of medical care into research studies as well as their daily practices.
At the recommendation of the Institute of Medicine committee, the Health Care Financing Administration has targeted three common conditions in the elderly--heart attack, breast cancer and hip fracture--for intensive study to determine the most effective treatments. Other conditions that are likely to be scrutinized include prostate surgery, congestive heart failure, angina, depression and cataracts.
The results of these studies will not be available for at least several years. Eventually, however, the studies may have a major impact on patient care.
A key uncertainty, however, is whether practicing physicians will be as enthusiastic about effectiveness research as the medical, business and government leaders who attended the Institute of Medicine meeting. Some see the initiative as a thinly veiled attempt by the government to dictate how physicians practice medicine and to indiscriminately cut costs.
“Individuals in a position to alter the behavior . . . (of other physicians are) skeptical,” said Dr. John D. Stobo, director of the department of medicine at Johns Hopkins Hospital in Baltimore. Practicing physicians, Stobo said, “have to to be involved early on” and “buy into” the concept of effectiveness research or it is unlikely to succeed.
