Research Studies

What is a Clinical Trial?
A clinical trial is a scientific research study to evaluate new medications, devices, 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 for approving medications is the Food and Drug Administration (FDA) and in Canada it is the Therapeutic Health Directorate (TPD). Clinical trials may be sponsored by pharmaceutical or biotechnology companies, federal agencies like the National Institutes of Health or the Department of Veteran's Affairs, or private medical institutions and foundations. The trials require volunteers, known as subjects, who fit specific guidelines or criteria. Clinical trials can be done in an inpatient or outpatient setting.

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

• What patient group or disease is the treatment meant to improve?
• What are the criteria to include or exclude subjects in the study?
• What general and disease-specific facts are going to be studied?

Benefits of Clinical Trials:
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.

How is the Safety of the Volunteers Protected?
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 protocols, or set of rules and 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 their name in these reports.

Most types of clinical trials in the United States 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 benefits relative to the risks and 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 the informed consent document should include: the trial's purpose, how long it lasts, 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 for any reason.

Phases of Clinical Trials:
Phase I: Phase I studies are usually designed to find out the effects of a new drug in humans. These studies are typically 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 in Phase II to determine if it works. Phase II trials are usually randomized trials, with one group of subjects receiving the experimental drug, while a second "control" group receives a standard treatment or 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 a slightly larger group 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 for the disease. The results of these trials usually provide the information that is included in the package insert and labeling.

Phase IV: After a drug has been approved by the FDA, phase IV studies are conducted to compare the drug to a competitor, explore more patient groups, or to further study any negative 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 8-10 years to complete the first three phases of research for a new drug and typically costs millions of dollars.

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

Check our web site for resources about clinical trials. And coming this fall, our web site will have a new section about clinical trials and current research studies. (www.vdf.org)