Research & Clinical Trials

A clinical trial is a research study to answer specific questions about vaccines or new therapies or new ways of using known treatments. Clinical trials (also called medical research and research studies) 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.

Ideas for clinical trials usually come from researchers. Once researchers test new therapies or procedures in the laboratory and get promising results, they begin planning Phase I clinical trials. New therapies are tested on people only after laboratory and animal studies show promising results.

All clinical trials have guidelines about who can get into the program. Guidelines are based on such factors as age, type of disease, medical history, and current medical condition. Before you join a clinical trial, you must qualify for the study. Some research studies seek volunteers with illnesses or conditions to be studied in the clinical trial, while others need healthy volunteers. Healthy volunteers participate in Phase I trials, vaccine studies, and trials on research on preventive care for children or adults.

The factors that allow you to participate in a clinical trial are called inclusion criteria and the factors that keep you from participating are called exclusion criteria.

It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, 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.

There are both benefits and risks associated with clinical trials. By participating in a clinical trial, you can:

• Take an active role in your own health care.
• Gain access to new treatments that are not available to the public.
• Obtain expert medical care at leading health care facilities during the trial.
• Help others by contributing to medical research.

Clinical trials have risks:

• There may be side effects or adverse reactions to medications or treatments.
• The treatment may not be effective for you.
• The protocol may require a lot of your time for trips to the study site, treatments, hospital stays, or complex dosage requirements.

You should know as much as possible about the research study. It is important for you to feel very comfortable asking questions and the staff should answer them in a way you can understand. A list of sample questions appears below:

Some questions you might ask about the research include:

• Why is this research being done?
• What is the purpose of the study?
• Who is sponsoring the study?
• Who has reviewed and approved this study?
• Why does the research team think the treatment, drug, or medical device will work?

Some questions about your participation in the study include:

• Where is the study site?
• What kinds of therapies, procedures, and/or tests will I have during the trial?
• Will they hurt? If so, for how long?
• How will the tests in the study compare to tests I would have outside the study?
• How long will the study last?
• How often will I have to go to the study site?
• Who will provide my medical care after the study ends?
• Will I be able to take my regular medications during the trial?
• What medications, procedures, or treatments must I avoid while in the study?
• What are my responsibilities during the study?
• Will I have to be in the hospital during the study?
• Will the study researchers work with my doctor while I am in the study?
• Can anyone find out that I am participating in a study?
• Can I talk to other people in the study?
• Will I be able to find out the results of the trial?

Questions about risks and benefits include:

• How do the possible risks and benefits of the study compare with approved treatments for me?
• What are the possible immediate and long-term side effects?

Other questions include:

• What other treatment options do I have?
• Will I have to pay anything to participate in the study?
• What are the charges likely to be?
• Is my insurance likely to cover those expenses?

The government has strict guidelines and safeguards to protect people who choose to participate in clinical trials. Every clinical trial 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 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. 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.