Clinical Trials

Frequently Asked Questions

Are Clinical Trials Safe?

Before an experimental treatment can be applied to people, it is carefully studied in the laboratory to determine its effectiveness and safety. Clinical trials are reviewed at both the national level by the Food and Drug Administration (FDA) and at the local level by an institutional review board (IRB). Each hospital or clinic where a clinical trial is to take place has an IRB made up of healthcare professionals, patient advocates, and community leaders who review the trial for safety and fairness.

Possible participants are also carefully screened - by thorough analysis of a patient's medical history, physical examinations, and possibly other tests - to ensure that they are the best possible candidates for the experimental treatment. During the trial, patients are carefully monitored to track how the treatment is affecting their condition. Since participation in a clinical trial is voluntary, a patient can stop at any time for any reason.

What Should I Consider Before Participating in a Clinical Trial?

Before participating, you must be provided with an "informed consent" document explaining the risks and potential benefits of the trial. Be sure to read over this information carefully. It is important to fully understand the purpose of the trial and what to expect.

You will want to find out if the treatment will interact with any of your current medications or affect any other medical conditions you may have. You should be informed about what tests or procedures, such as biopsies or blood draws, will be performed, and you should consider your comfort level with what will be done. Also, think about whether you are prepared for any anticipated side effects, pain, or discomfort that may be involved.

Another consideration is how the trial will affect your daily life. Think about how long the study lasts, if it will fit in your work schedule and personal life, and if you can commit to it. Will the hours at the clinic require that you take time off work? How many visits will be involved?

If you do decide to participate, you will be required to sign an informed consent document, and a copy will be given to you. The document will include the contact information for someone you can call should any questions or concerns arise during the trial.

Are There Costs Associated With Participating in a Clinical Trial?

This is an important question you should discuss with your doctor. There may be extra costs for participating in a study that may or may not be covered by your insurance carrier. Some examples of extra costs include additional doctor visits, special blood tests, or the cost of administering experimental treatments.

Types of Clinical Trials

There are four phases that describe treatment trials. Additionally, there are many other types of trials in which patients may be asked to participate, such as prevention, diagnostic, screening, and quality-of-life trials. Clinical trials are classified into phases as described below:

Phase 1

These trials constitute the initial testing in humans and usually involve a small number of patients. The primary goal of Phase 1 trials is to demonstrate the safety of the drug or drugs being given. Sometimes Phase 1 trials test the safety of a new drug under development as well as dosage and frequency of administration; others test the safety and efficacy of a particular dosage when two or more drugs are given in combination.

Phase 2

Once a drug or a combination of drugs is shown to be safe in initial Phase 1 trials, it has to be shown to be effective - the primary goal of Phase 2 trials. These trials are generally longer, typically lasting several months and involve more people, up to several hundred, to prove that the drug or combination of drugs is effective in a larger patient group.

Phase 3

This type of trial involves large-scale testing in several hundred to several thousand people for a thorough understanding of a treatment's benefits and side effects in a wide range of patients. Phase 3 studies are usually "randomized" studies in which a patient is randomly assigned to either the standard arm (treatment that has been approved as the current standard therapy by the FDA) or to the experimental arm (patients are given a new drug or new combination of drugs). The assignment is made by a computer or other coinflip mechanism. If the standard treatment with a new drug is being compared to the standard treatment alone, patients in the standard arm may be given a placebo (an inactive substance that has no medicinal value) in conjunction with the standard therapy while patients in the experimental arm will receive the standard therapy in conjunction with the new therapy. The FDA usually requires a successful Phase 3 study showing that a new treatment is better than the standard of care before they will consider the treatment for FDA approval.

Phase 4

Further analysis of a treatment is considered in these types of trials, exploring such issues as cost-effectiveness, long-term effectiveness, or how a drug affects a patient's quality of life.