Why Volunteer  |  What Are the Risks  |  What Questions You Should Ask

Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans.

These studies also may show which medical approaches work best for certain illnesses or groups of people.

Clinical trials produce the best data available for healthcare decision-making.

The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results. Clinical trials are one of the final stages of a long and careful research process. The process often begins in a laboratory (lab), where scientists first develop and test new ideas. If an approach seems promising, the next step may involve animal testing. This shows how the approach affects a living body and whether it is harmful. However, an approach that works well in the laboratory with animals does not always work well in people. Thus, research in humans is needed. For safety purposes, clinical trials start with small groups of patients to find out whether a new approach causes any harm. In later phases of clinical trials, researchers learn more about the new approach's risks and benefits. A clinical trial may find that a new strategy, treatment, or device improves patient outcomes; offers no benefit, or causes unexpected harm. All these results are important because they advance medical knowledge and help improve patient care.

Why Volunteer?

Each clinical trial defines who is eligible to take part in the study. Each trial must include only people who fit the patient traits for that study (the eligibility criteria) and will enroll people who have a specific disease or condition. Others enroll healthy people to test new approaches to prevention, diagnosis, or screening. So, the FDA wants people from many different groups included in these studies. 

Why Should Minorities and Women Participate in Clinical Trials?

In the past, most drug testing had been done on white men. This means that some groups, such as African Americans, Hispanics/Latinos, American Indians, Asians, Pacific Islanders, and women, had not always been included in the tests done on drugs. But sometimes drugs work differently in these people than on white men. The FDA wants people from many different groups included in these studies.

What Happens in a Clinical Trial?

During a clinical trial, doctors, and other healthcare providers might be part of your treatment team. They will monitor your health closely. You may have more tests and medical exams done than you would if you were not taking part in a clinical trial.  Many studies require that neither the patient nor the doctor knows whether the patient is receiving the study treatment, the standard treatment, or a placebo (an inactive substance that looks like the drug being tested). In other words, some people may be getting no treatment at all.

Studies are done in phases to find different kinds of information. For example, Phase I clinical trials test new treatments in small groups of people for safety and side effects. Phase II clinical trials look at how well treatments work and further review these treatments for safety. Phase III clinical trials use larger groups of people to confirm how well treatments work, further examine side effects, and compare new treatments with other available treatments.

Phase II studies include more people than Phase I studies, and the people have a disease that the product is going to treat. Now scientists try to see how well the product works against the disease. If the product works, the study moves into Phase III. In Phase III large numbers of patients with the disease are included to see if the new treatment works as well as the standard treatment.

A Phase IV clinical research study occurs after a drug has been approved by the FDA. The drug may be compared with similar medications, it may be evaluated for additional patient populations, or it may be studied for any specific, unusual, or unexpected side effects.  

What Are the Risks? 

Some treatments that are being tested have side effects that can be unpleasant, serious, or even life-threatening. Because the treatments being studied are new, doctors do not always know what the side effects will be. Many side effects are temporary and then it goes away when the treatment is stopped. But others can be permanent. Some side effects appear during treatment, while others may not show up until after the treatment is over. The risks depend on the treatment being studied and all known risks should be fully explained to you by the researchers.

How Am I Protected?

Informed Consent 

To help you decide if you want to be in a study, FDA requires that you be given complete information about the study before you agree to take part. This is known as informed consent. FDA requires that people be told:

• that the study involves research of an unproven drug or device

• the purpose of the research

• how long the study will take

• what will happen in the study and which parts of the study are experimental

• possible risks or discomforts

• possible benefits

• other procedures or treatments that you might want to consider instead of the treatment being studied

• The FDA may look at study records, but the records will be kept confidential.

• whether any medical treatments are available if you are hurt, what those treatments are, where they can be found, and who will pay for the treatment

• the person to contact with questions about the study, your rights, or if you get hurt

• you can quit at any time.

Informed consents must be in writing and in the language of your preference, so you can understand it. If you do not, be sure to ask the doctor or the medical research staff in charge to explain it to you. Make sure you understand everything before you sign and agree to be in the study. Before you can be in the study, you must sign the informed consent form, showing that you have been given this information and that you understand it completely. The informed consent form is NOT a contract and you can leave the study at any time, for any reason.