Clinical trials are only a small part of the research that goes into developing a new treatment. Drugs of the future, for example, first have to be discovered, purified, described, and tested in labs (in cell and animal studies) before ever reaching human clinical trials. About 1,000 potential drugs are tested before just one reaches the point of being tested in a clinical trial. On average, a new drug has at least six years of research behind it before it even makes it to clinical trials. But the major holdup in making new drugs available is the time it takes to complete clinical trials themselves. It takes an average of eight years from the time a drug enters clinical trials until it is approved.

Why so long? To be sure it is safe and effective, researchers look at each new treatment in several different studies. Only certain people are eligible to take part in each clinical trial. Clinical trials take years to complete, and it takes months, if not years, to see if a given therapy is effective. Thus, the rate of clinical trials participation determines how fast we advance the science.

Furthermore, in the United States, the FDA must approve new drugs and medical devices (but not dietary supplements) before they can be advertised or sold to the general public. The FDA began overseeing the safety of new treatments in the late 1930s, but didn’t require proof of effectiveness until the early 1960s. Today, new drugs and medical devices must go through several phases of clinical trials before being approved for use.

The biggest barrier to completing clinical trials is that not enough people take part in them. Fewer than 5% of adults (less than 1 in 20) with a given disease will take part in a clinical trial. For example, only 3% to 5% of adult cancer patients actually participate in cancer clinical trials while approximately 20% may be eligible. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), thousands of medicines were being tested in clinical trials in 2006. Not all of them will prove to be useful, but those that are may be delayed in getting approved because the number of adult volunteers for clinical trials is so low.

The main reason people give for not taking part in a clinical trial is that they didn’t know the studies were an option for them. But there are many other reasons. Some people may want to take part but aren’t eligible. Some people are uncomfortable with the idea of being a “subject” in a study. Others worry that they won’t be treated fairly or could be harmed by an unproven treatment.

What To Ask Before Proceeding With A Clinical Trial
One of the most important points in deciding if a clinical trial can be done is whether or not it would be ethical to ask patients to volunteer for the experimental treatment. Has the study been designed, as much as possible, to make sure the people involved will be safe? Would the participants receive a treatment that is at least as good as, and maybe even better than, what they would get if they did not volunteer for the study? Scientific panels are set up to review and approve all clinical trials to make sure questions like these are answered before the researchers are allowed to enroll patients.

Two other important questions clinical trials must answer are:

1. Does the new treatment work? Is it better than what’s now available to treat a certain disease? If it’s not better, is it at least as good, perhaps while causing fewer side effects? Or does it work in some people who don’t benefit from current treatments? In other words, is it a step forward? A treatment that doesn’t offer anything new probably isn’t valuable to healthcare.

2. Is the new treatment safe? This must be answered while realizing that no treatment or procedure — even one already in common use — is entirely without risk. But do the benefits of the new treatment outweigh the possible risks?

Remember, clinical trials are conducted to answer specific scientific questions. They are not the same as treatment and are conducted to assist healthcare professionals assess what therapies are best for patients.

Furthermore, not all clinical trials study treatments. Many clinical trials study new ways to detect, diagnose, or learn the extent of disease. Some even look at ways to prevent the disease from happening in the first place. Even among clinical trials that do study treatments, not all of them study drugs. Many clinical trials test other forms of treatment, such as new surgery or radiation therapy techniques, or even complementary or alternative medicines. When clinical trials do focus on drugs, not all of them study new ones. Even after a drug has been approved for use against a type of cancer, doctors sometimes find it works better when given a certain way or when combined with other treatments. It may even work on a different kind of cancer. Clinical trials are needed to study these possibilities as well.

New Treatments, Much Sooner
Some doctors and scientists conducted what would now be considered clinical trials as far back as the late 1700s, but clinical trials were not used widely until the middle of the 20th century. Up until that time, doctors relied on their own experience in particular cases and on the teachings of those who came before them. Progress was slow, and there were very few medicines that could even be tested.

With the discovery of the first antibiotics and other drugs, doctors needed a reliable way to tell what worked and what didn’t. They also needed ways to find out which of the countless remedies available at that time were safe for people to use. So, they developed studies that tested and compared treatments in certain groups of people. The results of these early clinical trials proved to be more useful than relying on whether or not something worked in one person or a few people.

Based on what we have learned in recent years, researchers now can develop new treatments in a more logical way and much faster than in the past, which is important since it is estimated that approximately 100,000 people die annually due to drug side effects. Millions more die too early in life because current treatments are not good enough. Clinical trials serve a vital role in helping those people find new treatments.

About the Author
William McGinnis is the CEO of Centurion Clinical Research, LLC. Since retiring from 27 years of service at Eli Lilly, he has served as president and CEO of Diabetogen Biosciences and Acera Biosciences as well as COO of Bioenergy Pharmaceuticals and Cell Technology and managing director of Heartland Biosciences Inc. In addition to his role at Centurion, he is also currently the president and CEO of NephRx Corp.