Why Clinical Trials Are Conducted

People want and expect their doctors to use treatments that work well and to stop using those that do not. Long ago, trial and error was the primary way that physicians and medical care providers learned how to recognize treatment alternatives. Later, through rigorous approaches that use clinical trials, physicians and researchers were able to gather far more meaningful information about diseases and how best to treat them.

For thousands of years, healers, shamans, and medical care providers have been administering treatments and remedies. One of the earliest known medical treatments dates back more than 3,500 years to ancient Egypt. Some ancient remedies, such as those used for simple fractures and minor injuries, are effective even today. However, many ancient medical treatments did not work and were actually harmful and even fatal. Two hundred years ago, cutting open a vein to drain a pint or more of blood and giving toxic substances to force vomiting or diarrhea were common remedies. And only a century ago, along with mention of some useful drugs such as aspirin and digitalis, the Merck Manual— one of the most respected sources for information on medical treatments then as well as now mentioned cocaine as a treatment for alcoholism; arsenic and tobacco smoke as treatments for asthma; and sulfuric acid nasal spray as a treatment for the common cold. Today these approaches are known to be very dangerous.

There are many reasons that doctors recommended ineffective and harmful treatments and that people accepted them. In many cases there were no alternatives. Doctors and patients usually prefer doing something to doing nothing. Patients also find comfort in sharing their problems and ailments with an authority figure. And doctors feel compelled to provide attention, support, and reassurance.

The primary reason doctors recommended ineffective and harmful treatments is that doctors couldn’t tell what worked from what didn’t. Doctors relied on cause-and-effect to identify potential treatments. For example, if an ill person’s fever broke after the doctor drained a pint of blood or after the shaman chanted a certain spell, then people naturally assumed those actions must have been what caused the fever to break. To the person desperately seeking relief, getting better was all the proof necessary. Unfortunately, these apparent causal relationships observed in early medicine were rarely correct. Still, they were enough to promulgate centuries of ineffective remedies. Of course, people had to be getting better in order to reassure doctors that a given treatment was working. Indeed, this is exactly what often happens. People do get better spontaneously. Sick people often get well on their own—and despite their doctor’s care—when the body heals itself or the disease runs its course. Colds are gone in a week; stomach flu passes within hours; migraine headaches typically last a day or two; and food poisoning symptoms may end in 12 hours. Many people even recover from life-threatening disorders, such as a heart attack or pneumonia, without treatment. Symptoms of chronic diseases (such as asthma or sickle-cell disease) come and go. Many treatments may seem to be effective if given enough time. And any treatment given near the time of spontaneous recovery may seem dramatically effective.

Belief in the power of a treatment or remedy is often enough to make people feel better. Belief cannot cause an underlying disorder—such as a broken bone, heart disease, or diabetes—to disappear. But people who believe they are receiving a strong, effective treatment very often feel better. Pain, nausea, fatigue, and many other symptoms can diminish. This happens even when the drug contains no active ingredients and can be of no possible benefit, such as a sugar pill or an inactive substance called a placebo. An ineffective (or even harmful) treatment prescribed by a confident doctor to a trusting, hopeful person often results in remarkable improvement of symptoms. This improvement is termed the placebo effect. People may see an actual (not simply misperceived) benefit from a treatment that has no real effect on the disease itself.

Some people argue that the only matter of importance is whether a treatment or remedy makes people feel better. Whether it works or not is of little consequence. This argument may be reasonable when the symptom is the problem, such as in many day-to-day aches and pains, or in illnesses such as colds, which always go away on their own. In such cases, doctors do sometimes prescribe treatments for their placebo effect. However, in any dangerous or potentially serious disorder, or when the treatment itself may cause side effects, it is critically important for doctors not to miss an opportunity to prescribe a treatment that really does work.

For more information on clinical trials and making informed decisions about volunteering for clinical research, read “The Gift of Participation” by Ken Getz, Founder and Board Chair, CISCRP.