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How Clinical Trials Are Conducted
A clinical trial is a carefully controlled study designed to test and evaluate new drugs, treatment plans, and devices (such as the pacemakers used in cardiac surgery). In addition to determining safety and effectiveness in humans, clinical trials help researchers determine such features as the proper dosage, the best way to administer the treatment, and what the potential side effects—if any—may be.
A new treatment or medication often takes a long and difficult journey before it becomes available to you. Therapies often undergo years of laboratory and animal studies before they are tested in humans. In the U.S., the Food and Drug Administration (FDA) is the government agency that oversees and regulates how new therapies come to market. The FDA has a stringent review process that determines whether or not a therapy is ready for testing in humans.
Usually when a sponsor—a pharmaceutical or biotechnology company, research institution, or other organization that takes responsibility for developing a new therapy—has done enough studies in the lab, it files an Investigational New Drug Application (IND) with the FDA. The sponsor must show the FDA the results of its lab and animal studies, and what it proposes to do for human testing. At this stage, the FDA decides whether or not it is safe for the company to move forward with testing the drug in humans.
If the FDA approves, then the study will be conducted at one or more medical centers, usually large teaching hospitals. Every medical center has an institutional review board (IRB)—a panel of scientists and non-scientists that oversees clinical research and is responsible for approving the studies conducted at that institution. IRBs approve the clinical trial protocols, detailed guidelines that describe the type of people who may participate in the study, the schedule of tests and procedures, the medications and dosages to be studied, the length of the study, the study's objectives, and so on. IRBs make sure that the study meets the institution’s standards, that participants have given consent and are fully informed of their risks, and that researchers take appropriate steps to protect patients from harm. If the study is a multi-center trial—one that involves several medical centers—each institution’s IRB must give its approval.
Once this structure is in place, a clinical trial can begin.
The Four Phases of Clinical Trials
There are four phases of clinical trials, each designed to provide more information about the therapy being tested. A sponsor may not request FDA approval to market a therapy until Phase III trials are completed.
Phase I studies assess the initial safety of a drug or device. This phase, which can take several months to complete, is a small study done in healthy volunteers (20 to 100). Phase I studies determine the effects of the therapy on humans, including any side effects (also known as adverse reactions).
Phase II studies test the efficacy (how well it works) of a new therapy. This phase can last from several months to two years. It also involves a greater number of volunteers, up to several hundred patients. Phase II trials are often randomized, double-blind, placebo-controlled studies. What, exactly, does this long string of words mean?
Most Phase II studies are randomized—meaning that one group of patients receives the therapy being tested, while a second "control" group receives a currently existing standard treatment or a placebo (a harmless substance that looks like the study drug). The two groups are chosen randomly—like the flip of a coin. Often these studies are double-blinded—neither the patients nor the researchers know who has received the experimental drug while the trial is going on. This allows investigators to provide the FDA with unbiased, comparative information about the relative safety and effectiveness of the new treatment.
Phase III studies are even larger than Phase II studies, and can involve several hundred to several thousand patients. These are also randomized, blinded trials. This large-scale testing, which can last several years, provides the FDA with a more thorough understanding of the effectiveness of the therapy or device, the benefits, and the range of possible adverse reactions (side effects). Once Phase III is complete, a pharmaceutical company or other maker of the treatment can request FDA approval to market the product.
Phase IV studies, often called Post-Marketing Surveillance Trials, are conducted after a drug or device has been approved for sale and made available to physicians and their patients. The manufacturer of the therapy is looking for long-term information that will help compare a drug or treatment with other treatments already in the market. The information will also help monitor a treatment’s long-term effectiveness and impact on a patient's quality of life.
What Happens During a Clinical Trial?
When you participate in a clinical trial, you’ll work with a team that includes doctors and nurses, as well as social workers and other health care professionals. Team members check the health of participants at the beginning of the trial, give specific instructions for participation, monitor participants carefully throughout the trial, and stay in touch after it is completed.
For a trial to be successful, the study design must be carefully followed and there should be frequent contact with the research staff. Some trials, therefore, involve more tests and doctor visits than you would normally have for an illness or condition.
Participating in a clinical trial does not take the place of ongoing medical care by your primary physician and health care team. If you are thinking of participating in a clinical trial, you should speak with your doctor first. While you are enrolled in the trial, your doctor should be informed of the study’s progress.
Learn more about deciding to participate in a clinical trial.