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Clinical Trials : FAQs

Q. What is a clinical trial?

A. A clinical trial is a scientific research study to evaluate new medications or other treatments like diet and exercise in human volunteers.  Clinical trials must follow guidelines established by the government in each country. In the United States, the governing body is the Food & Drug Administration (FDA) and in Canada, it is the Therapeutic Health Directorate (TPD).  Pharmaceutical or biotechnology companies, federal agencies such as the National Institutes of Health or the Department of Veteran's Affairs, or private medical institutions and foundations, may sponsor clinical trials.  Each trial requires volunteers, known as subjects, who fit specific guidelines or criteria. Clinical trials can be done in an inpatient or outpatient setting.

Q. What purpose is served by clinical trials?

A. The purpose of a clinical trial is to find out whether a medication or particular treatment is safe and effective to use in a specific disease or medical condition and that it works as intended. Before the trial, researchers must answer several questions:

Q. What are the benefits of participating in a clinical trial?

A. People who take part in clinical trials are playing an active role in their own health care.  They can find out about and have access to new treatments before they are offered to the general public, and they help others by taking part in medical research.

Q. How is the safety of volunteers protected?

A. The ethical and legal codes that apply to general medical practice also apply to clinical trials.  Most clinical research is regulated by the government, which requires built-in safeguards to protect volunteers.  Trials follow carefully controlled protocol, or sets of rules (study plans), which detail what researchers will do in each study.  Results of clinical trials are reported by researchers at meetings, in medical journals, and to government agencies. People who take part in the study are not mentioned by name in these reports.

Most types of clinical trials in the U.S. and Canada must be approved and monitored by an Institutional Review Board (IRB) to make sure the trial is safe.  An IRB is an independent committee of physicians, scientists, mathematicians, community advocates, and others that assure the trial will produce the benefits relative to the risks and that the rights of volunteers are protected.  All institutions that conduct or support biomedical research involving humans that is funded by the federal government and most private and public organizations have an IRB that initially approves the protocol and reviews the research from time to time.

You must sign a consent form before you take part in a clinical trial.  This document should answer all of your questions about the trial in plain language.  Explanations in this "informed consent" document should include: the trial's purpose, how long it will last,  required procedures or things you must do, if you will be paid, people to contact if there is a problem, risks and possible benefits.  If you have questions that are not addressed in the written consent, you should ask the study staff for more information.  An informed consent document is not a contract.  Volunteers may quit the trial at any time.

Q. What is meant by the different phases of clinical trials?

A. Phase I: Phase I studies are usually designed to find out the effects of a new drug in humans. These studies are usually conducted on a small number of healthy people to determine if a drug is safe. Occasionally, Phase I trials are offered to patients with a life-threatening disease.

Phase II:  After a drug is proven safe in Phase I, it is tested for efficacy (to determine if it works) in Phase II.  Phase II trials are usually "randomized" trials in which one group of subjects receives the experimental drug, while a second "control" group receives a standard treatment or a placebo.  Often these studies are "blinded" and neither the subjects nor the researchers know who is getting the experimental drug.  Phase II trials are usually conducted in slightly larger groups of people who suffer from the disease or condition for which the drug was developed.

Phase III:  The third and last pre-approval round of testing of a drug is conducted on large groups of afflicted patients.  Phase III studies usually test the new drug in comparison with medications currently being used to treat the disease.  The results of these trials usually provide the information that is included in the medication's package insert and labeling.

Phase IV:  After the FDA approves a drug, Phase IV studies are conducted to compare the drug to a competitor, explore more patient groups, or further study any bad outcomes and collect safety data.

Each phase of a clinical trial must be evaluated by the FDA for safety and effectiveness before approval is given to move to the next phase. It takes approximately 8 to 10 years to complete all three phases of research for a new drug and typically costs millions of dollars.

Q. Should I participate in a clinical trial?

A. Choosing to volunteer for a clinical trial is an important personal decision.  While study medications provide benefit, there is often a "control group" in each study that receives a "placebo" which is an inactive substance with no treatment value.  There are no guarantees that participation will improve your health in any way. Carefully consider the risks and benefits, and discuss your questions with your physician and the research scientists.

Source: Spilker, B., Guide to Clinical Trials. Lippincott, Williams & Wilkins, 2000


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