5 Questions About Clinical Trials

1. What is a clinical trial?

In a nutshell, clinical trials are ways that we answer questions about drugs and other treatments. The following are simplified, but you get the idea.

• Does this new medication work the way we hoped it would when it was developed?

• What's the right dose?

• What are the common side effects?

• Will eating more or less of a certain food help with diabetes, heart disease or any number of conditions?

• What happens when we combine two or more medications?

2. What are the types of clinical studies?

There are two types of clinical studies: clinical trials and observational studies.

• In a clinical trial, people who qualify by meeting certain inclusion criteria are placed into groups by an investigator who is running the trial. One group will receive the intervention that is being studied. This might come in the form of a medication or other new treatment or a change in behavior (like a change in diet). Because investigators are trying to figure out if the new treatment works, they must compare it to either an existing treatment, no treatment or a placebo, which is used as the control in the study.

• In observational studies, investigators look at large numbers of people and try to find common threads to explain why they experienced certain things. One example is the Nurse's Health Study (I happen to be a subject and fill out their questionnaires regularly).

By tracking behaviors, medical conditions, diet, exercise and much more on more than 180,000 nurses for over 20 years, the Nurse's Health Study has found, among other things, that women who exercise for at least 30 minutes each day have a lowered risk of developing breast cancer.

Observational studies can look back at data, which is known as a retrospective study. Observational studies can also track people in real time, by looking at the variables or things that seem to be related to certain outcomes. For example, a hospital may be tracking infection rates and determine that installing handwashing stations outside of every room reduces infections.

3. What is a placebo?

These are substances that do not contain any active ingredient or therapeutic effect (think sugar pills).We use placebos as controls when testing new medications. Many medications studied are only slightly more effective than placebos. This could mean that the medications aren't working or that just the idea of taking a medication or other treatment helps people feel better. In every study I've ever read, even people on placebos have side effects! More evidence of the power of the mind to transform how we feel.

4. Are there different phases to a clinical trial?

This is a great question. Obviously, when a new medication is being developed, safety is the number one concern, followed by whether it works in healthy people and what is the best dose.

As more is learned from each phase of the trial, researchers can fine tune what kinds of effects they are looking for and design the trial to find out more about how the medication works, the side effects and what people are experiencing.

Often a medication or treatment that was designed for one condition will end up working for a different one. An example is finasteride (Proscar, Propecia), which was developed to treat prostate symptoms and was found in clinical trials to also work on helping men regrow their hair.

The phase of clinical trial tells you what type of study it is.

The Food and Drug Administration (FDA) created these categories to describe the types of trials available:

5. How can I (or someone I know) participate in a clinical trial?

Clinicaltrials.gov is a service provided by the U.S. National Institutes of Health that allows you to search lists of current clinical trials that are open to enrollment. To participate in a trial, you must meet the trial's eligibility criteria. This can include things like age, disease stage and other factors that make you either eligible to join or mean that you're not a candidate for participation in that specific clinical trial.

If you or someone you care about is looking into clinical trials for a condition, go to Clinicaltrials.gov and start searching. Many times the information can seem overwhelming, but there are contacts available to answer questions. You can also speak with your own health care provider to help you determine if you'd be considered for inclusion in a trial. They may even help you contact the study coordinators to demonstrate your interest.