FAQ on Clinical Trials

What is a clinical trial or clinical study?
The FDA defines "Clinical Trial" as the scientific term for a test or study of a drug or medical device in people. It 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. These tests are done to see if the product is safe and effective for people to use and 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 the clinical trials.

Doctors and other health professionals run the tests according to strict rules set by the Food and Drug Administration (FDA). The FDA sets the rules to make sure that people who agree to be in the studies are treated as safely as possible. New therapies are tested on people only after laboratory and animal studies show promising results.

What are the different types of clinical trials? 

• Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

• Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.

• Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.

• Screening trials test the best way to detect certain diseases or health conditions.

• Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.

Are clinical studies safe?
Before a prospective treatment is tested on humans, it is thoroughly tested through laboratory and model studies to determine if it's safe. The tests are run by health professionals and doctors according to strict rules set by the Food and Drug Administration (FDA). The FDA sets the rules to make sure that people who agree to be in the studies are treated as safely as possible. Before a clinical trial can begin, an Institutional Review Board (IRB) reviews and approves the trial protocol. This includes looking at the informed consent and volunteer selection processes, as well as other benefits and risks of participation.

What is a protocol? 
All clinical trials are based on a set of rules called a protocol. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

What is a control or a control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

What is a placebo?
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.

What is a "Phase"?
Clinical studies are done in a series of four different phases:
In Phase I Studies, researchers test a new drug or treatment in a small group of healthy people (20-80). During this phase, scientists find the best way to give a new treatment, how much they can safely give, and to identify side effects.

In Phase II Studies, the study drug or treatment is given to a larger group of people (100-300) that have the disease that the product is going to treat. In this larger group of people, researchers try to see how well the product works against the disease and to further evaluate the treatment’s safety.

In Phase III Studies, if Phase II is successful, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.

Phase IV Studies may occur after FDA approval and are done after the drug or treatment has been marketed. These studies continue testing the study drug or treatment to collect information about their effect in various populations and any side effects associated with long-term use.

Why does the FDA encourage women and minorities to participate in clinical trials?
Sometimes drugs work differently in men and women, and in people from varying ethnicities, such as African Americans, Hispanics/Latinos, American Indians, Asians, and Pacific-Islanders. The FDA wants people from many different groups included in these studies because in the past, most drug testing had been done on white men.

What protection is there for people who participate in clinical trials?
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.

What do I need to know before I decide to be part of a clinical study?
Before making your decision, the FDA recommends asking the following questions:

• What is the study trying to find out?
• What kinds of test and exams will I have to take while I'm in the study? How much time do these take? What is involved in each test?
• How often does the study require me to go to the doctor or clinic?
• Will I be hospitalized? If so, how often and for how long?
• What are the costs to me? Will my health insurance pay for it?
• What follow-up will there be?
• What will happen at the end of the study?
• What are my other treatment choices? How do they compare with the treatment being studied?
• What side effects can I expect from the treatment being tested? How do they compare with side effects of standard treatment?
• How long will the study last?

What is an "informed consent"?
Informed consent is a FDA requirement that says you must be given complete information about the study before you agree to take part. The FDA requires that you receive and understand the following information:

• 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 secret
• 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
• Make sure you understand all of the information before you agree to be in the study

If you are considering joining a clinical trial, our research team will give you informed consent documents that include the details about the study. If English is not your native language, you can ask for the consent documents in languages other than English. Since joining a clinical trial is an important decision, you should ask our research team any questions you may have about the study and the consent forms before you make a decision.

It is also a good idea to take the consent documents home and discuss them with family members or friends. Talking about your options can help you to feel comfortable with your decision. If you decide to join the clinical trial, be sure to ask for a copy of the informed consent documents so you can review them at any time.

Remember - informed consent is more than signing a form. It is a process that continues through the study. You should feel free to ask the research team questions before, during, and after the study. Informed consent continues as long as you are in the study.

Who can participate in a clinical trial? 
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.

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.

What if I change my mind and no longer want to be in the clinical study?
You can leave the study at any time, for any reason. The informed consent form is NOT a contract. If you plan to stop participating, please let the research team know why you are leaving the study so our office can fill out the proper paperwork for our files.

Who sponsors clinical trials?
Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

Will I be paid for participating in a clinical trial? 
Some clinical trials will pay you for joining the trial, while others will not. In some programs, researchers will reimburse you for expenses associated with participating in the research. Such expenses may include transportation costs, child care, meals, and accommodations.

Who should I contact for more information?
If you would like more information about participating as a volunteer for a clinical study with Pensacola Research Consultants, please contact our office at (850) 477-7900, and a member of our research team will be happy to help you.