Patient Volunteer Information
Why do people participate in a clinical trial?
People choose to participate in a clinical trial for a number of reasons. Volunteers for Phase I and Phase II studies may have access to a medication that might be better than medications that are currently available. Usually the medical care associated with the trial is of no cost to the volunteers. Volunteers in clinical trials receive excellent care from the physicians and coordinators conducting the study. Often the collection of study data leads to new information about the disease and the effects of the disease on patients. The clinical trial may not always help the volunteer directly, but may help the other patients with the same disease. In some clinical trials patients may be paid a small fee.
What is a clinical trial?
A clinical trial is a research study performed by physicians and other healthcare professionals to help determine if a medicine is safe and effective. Before newly developed medications are given to people, they are first studied carefully in a laboratory. If favored results are seen, then a research plan, called a protocol, is designed to study the medication. Physicians conduct clinical trials according to strict rules set by the Food and Drug Administration(FDA). The FDA has set rules and guidelines to assure that volunteers in clinical trials are treated as safely as possible.
What is a protocol?
A protocol is a set of rules on which the clinical trial is based. It describes what type of patients may participate, the schedule of tests and procedures, medications and dosages, and the length of the study. Participants in a clinical trial are seen on a regular basis by the research staff to determine the safety and effectiveness of the medication being studied, monitoring their health and safety.
What are the phases of a clinical trial?
Phase I:
Initial safety study to establish the dosage range tolerated by volunteers for single and multiple doses. approximately 20-100 normal, healthy volunteers are administered the drugs. The tests study the drugs safety profile, including the safe dosage range. The studies also determine how the drug is absorbed, distributed in the body, metabolized and excreted. The duration of its action is also measured. Normally this phase takes nearly one year.
Phase II:
The second phase of clinical research studies includes further safety evaluation and ensures the drug is effective. It involves a larger patient population of Approximately 100-500 patient volunteers with the disease or condition to be treated, diagnosed or prevented. Objectives of these studies may focus on dose response, type of patient, frequency of dosing or other characteristics of safety and efficacy. Most Phase II studies are randomized trials. One group of patients receive the investigational drug, while the second "control" group receive a standard treatment or placebo. These studies are often "blinded" - neither the patients nor investigators know who is getting the investigational drug. This is so the pharmaceutical company and the FDA are provided with comparative information about the relative safety of the investigational drug and its effectiveness. Phase II takes an average of two years.
Phase III:
Approximately 1,000-15,000 patient volunteers with the disease intended for the investigational drug to treat participate in Phase III studies. These large scale studies provide the pharmaceutical company and the FDA with more thorough information concerning the effectiveness of the study drug. In addition, they provide more information as to the the benefits and range of possible adverse reactions. Most Phase III studies are randomized and blinded trials. This stage can take several years to complete. When Phase III studies are completed, the pharmaceutical company can provide this data to the FDA and apply for approval of the drug for marketing.
Phase IV:
This phase may have several objectives. The pharmaceutical company may compare a drug with others already on the market. Phase IV studies may be designed to assess the drugs long-term safety, effectiveness and the impact on the patients quality of life. Often these studies determine the cost-effectiveness of the therapy compared to traditional therapies.
Where can I get more information on clinical trials?
For more information on clinical trials visit the Food and Drug Administration's website or contact the St. Louis Center for Clinical Research.