What is a clinical trial?

A clinical trial is a research study designed to examine the safety and effectiveness of a specific drug, device, or treatment. Before new medications are given to humans, the drugs must undergo extensive research in the laboratory. The resulting data are presented to the Food and Drug Administration (FDA) for review before permission is granted to conduct research with humans. If this initial research is successful, pharmaceutical companies may then develop a research plan, or protocol, for studies involving humans. In many cases, these studies are testing investigational medications to obtain FDA approval, a necessary step before medications may be prescribed to patients. Other times, the medications used in the studies have already been approved by the FDA, and the trial is studying different dosages or methods of administering the medication or different uses for the medication.

Clinical trials are held at select medical centers nationwide. These centers are chosen because of the expertise and reputation of physicians who conduct the research studies at that center. People who qualify based on certain criteria are invited to participate in these trials, which are supervised by trained clinical research personnel.

What rules govern clinical trials?

All clinical trials are extensively regulated by the FDA and an institutional review board (IRB). Before a medication is approved to be prescribed or sold to patients, the medication must undergo a series of rigorous tests, as described above. Once the FDA allows a medication to be tested in humans, an IRB provides additional regulations and review to ensure that all research participants are protected. The IRB is a board consisting of medical professionals and community members that reviews every proposed clinical trial prior to allowing human participation. The IRB makes sure that all proposed clinical trials have appropriate safeguards for those participating in them and carefully weighs the risks in the study with the anticipated benefits. At all times, the patient's well-being is considered, and certain rules that the IRB sets forth help ensure the patient's health and comfort, such as voluntary informed consent.

Participation in a clinical trial is completely voluntary. If you decide to participate in a clinical trial, you can choose to stop at any time. Before you agree to participate in a clinical trial, the investigators and staff will ask you to read a document called an informed consent form. This form contains detailed information about the research study, including possible risks and benefits, tests and procedures that are involved, and alternative treatments. After reading the document you will have an opportunity to ask questions. If you are interested in participating, you will be asked to sign the informed consent document.

In what areas are clinical trials conducted?

Clinical trials can be found in a variety of health fields. Ranging from asthma to psoriasis, arthritis to cancer, research studies are being conducted in areas that could be important to you. For a listing of study areas, click here.

Should I participate in clinical research?

Deciding to participate in clinical research is an important and personal decision. You and your physician should discuss issues such as the length of the trial, the purpose of the trial, the risks involved, and the alternative treatments that exist.

Patients participate in clinical research for a variety of reasons. In some trials, participants may benefit from access to promising drugs long before these medications or treatments are available to the general public. Patients also may receive certain examinations, laboratory tests, and other medical services and physician care free of charge. Additionally, participants in clinical trials are providing invaluable assistance in helping physicians and other health professionals find new and better treatments for patients worldwide.

It is always possible that in a clinical trial the experimental medication or device may not work or may cause side effects or increased discomfort. For these reasons, participants are monitored closely and may always contact study personnel with questions or problems.

What do these terms mean?

Investigational: Being researched. The FDA will allow certain drugs to be studied for a specific disease or condition but not for general use. These are called investigational medications.

Placebo: A substance containing no active medication. Placebos are often used in clinical trials to compare with investigational medications.

Double-Blind: The procedure in research studies in which neither you nor your doctor will know if the medication you receive is the investigational drug or a placebo. This is done to ensure no bias occurs.

Randomized: The procedure to assign patients to treatment groups so that every person has an equal chance of being in any of the groups (like flipping a coin or rolling dice).

The Four Phases of Clinical Trials

• Phase I – These trials are done to find out about the side effects and safety of a new drug. Side effects are looked at as the drug dose level is increased. These trials also study what happens to a drug in the human body, looking at how it is absorbed, metabolized and excreted.
• Phase II – After a drug has been shown to be safe in Phase I trials, Phase II studies are done to test if the drug works in fighting disease. Often these studies are comparing the new drug to the current standard of care for the disease, to see which works better.
• Phase III – These very large studies are done to give researchers a more complete understanding of how safe and effective the drug is. Another goal of these types of studies is to understand the benefits to patients and range of adverse experiences patients have.
• Phase IV – These trials are done after the drug has been approved by the Food and Drug Administration. These studies can be done for many reasons, but the main goal of these post-marketing trials is to find out as much as possible about long-term risks associated with the use of the drug.