A clinical trial is a research study that tests new ways to prevent, detect, diagnose or treat diseases. Clinical trials help determine whether investigational vaccines, medicines or new uses for existing medicines are safe and effective. Clinical trials are necessary to find potential medicines and vaccines to treat disease and improve health. Clinical trials are also known as intervention studies, clinical studies and clinical research.
Clinical trial participation involves risk, so it is important that you speak to your physician as well as the trial physician before you decide if participation is right for you. There may be unpleasant, serious or even life-threatening side effects to treatment, treatment may or may not be effective for the participant and participation in the trial may require more time and attention than would be required outside of a clinical trial. This may include travel to trial sites, more treatments, hospital stays or complex dosage requirements.
Before considering whether to participate in a clinical trial, you should know as much as possible about the trial and feel comfortable asking questions about the trial, the expected care during the trial and the costs involved. We invite you to spend time exploring the site to learn more about our clinical trials.
Phase 1 clinical trials test the medicine or vaccine in a small group of 20 to 100 volunteers who are usually healthy, but not always. The trial often takes place in a hospital. The goals are to determine:
Phase 2 clinical trials test the vaccine or medicine in approximately 100 to 500 volunteers. In the case of medicines, volunteers usually have the disease or condition the investigational medicine is designed to treat. In vaccine studies, the volunteers are usually healthy. The goals of this phase are to determine:
Phase 3 clinical trials can test the medicine or vaccine in 1,000 to 5,000 patient volunteers. For medicines, volunteers have the disease or condition the medicine is designed to treat. In vaccine studies, the volunteers may be healthy or have diseases or conditions. Phase 3 trials take place in hospitals, clinics or physician offices. Researchers closely monitor patients at regular intervals to:
Phase 4 clinical trials are conducted after the medicine or vaccine has been approved by the appropriate government and regulatory agencies and is being marketed. Researchers continue to gather information about the medicine or vaccine and its safety, side effects and effectiveness.
Marketed products also are studied for new indications. Thousands of people usually participate in ongoing trials.
Choosing to participate in a clinical trial is a personal decision, and it is important to gather as much information as possible to make an informed decision. The following frequently asked questions provide information that you may find helpful as you consider participation.
Ideas for clinical trials usually come from researchers. Researchers first test new therapies or procedures in the laboratory and in animal studies. The potential treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about a new possible treatment, its risks and how well it may work.
Clinical trials are sponsored (i.e. funded) by organizations or individuals: physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, as well as federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, including hospitals, universities, doctors' offices or community clinics.
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or investigational treatment, while the control group is given either a standard treatment for the illness or a placebo.
Side effects are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.
The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built-in safeguards to protect you.
Each trial follows a carefully controlled protocol – a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.
Every clinical study in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is a committee of physicians, statisticians, community advocates, and others that helps ensure that a clinical study is conducted in an ethical manner and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
Informed consent is the process in which researchers communicate with potential and enrolled participants about a clinical study. It provides the potential benefits as well as the risks to the patient. It informs the patients that participation in the trial is voluntary, and they may discontinue participation at any time.
The goal of the informed consent process is to protect participants. It begins when a potential participant first asks for information about a study and continues until the study ends.
Yes. Most clinical trials provide short-term treatments related to a designated illness or condition but do not provide extended or complete primary healthcare. In addition, by having the healthcare provider work with the research team, you can ensure that other medications or treatments will not conflict with the protocol.
Yes. You can leave a clinical trial at any time. When withdrawing from the trial, you should let the research team know about it, and the reasons for leaving the study.
Most human use of investigational new drugs takes place in controlled clinical trials conducted to assess safety and efficacy of new drugs. Data from the trials can serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these carefully controlled trials because of other health problems, age, or other factors. For patients who may benefit from the drug use but don't qualify for the trials, FDA regulations enable manufacturers of investigational new drugs to provide for "expanded access" use of the drug. For example, a treatment IND (Investigational New Drug application) or treatment protocol is a relatively unrestricted study. The primary intent of a treatment IND/protocol is to provide for access to the new drug for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose for a treatment IND/protocol is to generate additional information about the drug, especially its safety. Expanded access protocols can be undertaken only if clinical investigators are actively studying the new treatment in well-controlled studies, or all studies have been completed. There must be evidence that the drug may be an effective treatment in patients like those to be treated under the protocol. The drug cannot expose patients to unreasonable risks given the severity of the disease to be treated.
Some investigational drugs are available from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. Expanded access protocols are generally managed by the manufacturer, with the investigational treatment administered by researchers or doctors in office-based practice.
There is a lot to consider when deciding whether to participate in a clinical study. Any clinical study includes risks, which the study doctor will review with you. Make sure you understand the risks before participating.