Clinical Trials

People are sometimes confused about the purpose of clinical trials, which are scientific investigations performed in humans to determine if a treatment is safe, how much and how often a treatment should be used, whether a treatment works or not, and, sometimes, whether it’s better than currently available therapies. The best reason to participate in a clinical trial is to advance medical knowledge, which improves the lives of others with diseases and medical conditions.

These trials have brought great benefits to people suffering from diseases. Most of today’s standard treatments for cancers are based on the results of clinical trials. These investigations are responsible for increased survival rates and treatment regimens that produce less toxic side effects and improve people's quality of life.

A clinical trial is sometimes thought of as a way to get access to a treatment that is not yet offered to the general public, but this is not their purpose. While clinical trial participants may be getting a treatment that is not available outside of research settings, there is no guarantee that treatment is going to work or work as well or better than the care your physician can offer. Even if the treatment proves effective for some people in the trial, it may not work for everyone, and you may personally derive no therapeutic benefit from it.

When investigators conduct a clinical trial, they are trying to find out whether a new drug (or drugs already in use for other purposes), drug combinations, surgical procedure, device, or other type of intervention works as well as or better than existing therapies while producing fewer unwanted side effects. If a treatment is already proven to be safer and equally or more effective than what’s available, there would be no reason for the trial.

Many investigations are randomized, double-blind, placebo-controlled trials. This sort of trial is set up so that some portion of the participating receive a placebo (a sugar pill), which means they will not be getting the active agent being tested. Until the trial is over neither participants nor investigators know which patients are receiving active treatments and which are getting placebo until the trial. This ensures that any differences between the group of people getting the active agent and the group taking placebo is due to the agent and not other factors.

If you’re considering participating in clinical trial, is it important to understand that every effort is made to avoid serious side effects, but because the safety of experimental therapies can only be adequately determined after trials are over, you could suffer from an unexpected serious side effect. Elaborate safeguards are in place to ensure the safety of clinical trial participants, but these studies are experiments, and serious unforeseen consequences can and do happen.

Individual trials are designed to answer very specific questions, and this requires that people being studied fall into certain categories of health, gender, age, or other factors, which are defined by “inclusion” and “exclusion” criteria. Once investigators determine that you are the correct fit for the trial you’re considering, you will go through a process called “informed consent.”

During informed consent, an investigator or trial coordinator will explain all the key facts of the trial, including its purpose, how long it will last, what the required treatments are, and all the potential risks and benefits known to investigators. This process will continue throughout the trial as investigators get more information about the treatment under study.

During the informed consent process, you need to make sure you fully understand what investigators are saying. Although investigators make an effort to explain facts in simple language, the complicated information can still be confusing. You should ask as many questions as you need to understand all aspects of the trial, including exactly what is required of you (you might, for example, be asked for periodic blood samples or to undergo other procedures you could find uncomfortable), how much of your time it might take, and how it will affect your life in other ways.

After the initial informed consent process, you will be asked to sign an informed consent document, which is not a contract (you can withdraw from a trial at anytime), but a written record indicating that you have been informed of the key facts about the trial.

The National Institutes of Health (NIH) suggests asking these questions when considering clinical trial participation:

Ø What is the purpose of the study?

Ø Who is going to be in the study?

Ø Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?

Ø What kinds of tests and experimental treatments are involved?

Ø How do the possible risks, side effects, and benefits in the study compare with my current treatment?

Ø How might this trial affect my daily life?

Ø How long will the trial last?

Ø Will hospitalization be required?

Ø Who will pay for the experimental treatment?

Ø Will I be reimbursed for other expenses?

Ø What type of long-term follow up care or time commitment is part of this study?

Ø How will I know that the experimental treatment is working? Will results of the trials be provided to me?

Ø Who will be in charge of my care?  

Clinical trials can offer no guarantees about improving your health and could even make your condition worse, but there are some reasons to consider participating that may go beyond altruism.

If you have a serious or chronic medical condition for which there are few or no effective treatments, taking part in a trial is an opportunity to take a more active role in your health care. During a trial, you may also receive closer, more careful observation of your illness than you might get with standard care. If you suffer from an inherited condition, participating in a trial may not directly benefit you, but could help your children or grandchildren.

In addition, investigators often point out that women and minorities are less likely to participate in clinical trials than other individuals. This means that scientists often know more about disease processes in men and whites that they do in women and minorities. Investigators would like to improve the health of as many people as possible, and women and less-studied ethnic groups may also consider participating to help fill important gaps in medical knowledge.

Visit the NIH clinical trial site for more information and to search for condition-specific trials in your area.