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Q & A: Participating in a Clinical Trial
Choosing to participate in a clinical trial is an important personal decision. The following questions and answers provide information about the clinical trial questions most frequently asked by patients.
Q: What is a clinical trial?
A: A clinical trial is a research study to answer specific questions about vaccines, new therapies, or new ways of using known treatments. Clinical trials are used to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people.
Q: Why participate in a clinical trial?
A: Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research
Q: Where do the ideas for trials come from?
A: Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the treatments with the most promising laboratory results are moved into clinical trials.
Q: Who sponsors clinical trials?
A: Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health, the Department of Defense, and the Department of Veterans Affairs. Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics.
Q: What is a protocol?
A: 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.
Q: What is a placebo?
A: A placebo is an inactive pill, liquid, powder, or procedure that has no treatment value. In clinical trials, experimental treatments often are 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.
Q: What is a control or control group?
A: 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 treatment, while the control group is given either a standard treatment for the illness, a placebo, or no treatment.
Q: What is an "expanded access" protocol?
A: 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 allow manufacturers to provide expanded access to the investigational drug. Expanded access protocols generally are managed by the manufacturer, with the investigational treatment administered by researchers or doctors in office-based practice.
Q: Who can participate in a clinical trial?
A: All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps produce reliable results. The factors that allow someone to participate in a clinical trial are called inclusion criteria and those that disallow someone from participating are called exclusion criteria. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. The criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.
Q: What happens during a clinical trial?
A: The clinical trial process depends on the kind of trial being conducted. The clinical trial team checks the health of the participant at the beginning of the trial, gives specific instructions for participating in the trial, monitors the participant carefully during the trial, and stays in touch after the trial is completed.
Q: What is informed consent?
A: Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.
Q: What should people consider before participating?
A: People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it. The following questions might be helpful:
Q: Does a participant continue to work with a primary health care provider while in a trial?
A: Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.
Q: What are side effects and adverse reactions?
A: 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 immediate and long-term side effects.
Q: How is the safety of the participant protected?
A: 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 the participants. Every clinical trial in the United States 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 an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected.
Source: National Institutes of Health. For more information, visit www.clinicaltrials.gov.