Frequently Asked Questions: Clinical Research | OSF Saint Francis Medical Center

"Clinical trial" is the scientific term for a test or study of a drug or medical device in people. These tests are done to see if the product is safe and effective for people to use. Doctors and other health professionals run the tests according to strict rules set by the Food and Drug Administration (FDA). FDA sets the rules to make sure that people who agree to be in the studies are treated as safely as possible. Clinical trials are an important step in the process of making a newly discovered treatment available to the general public. Many of today's most effective treatments are the direct result of knowledge gained through clinical trials.

Why do people participate in clinical trials?

People may be interested in clinical trials for a variety of reasons. Some people participate as a way to contribute to medical science and to help doctors and researchers find better ways to help others. Others participate in order to receive investigational treatments because their illness is not responding to standard treatment. Their hope is that the study treatment - possibly an investigational drug, combination of drugs or device - will work better for then than standard therapy. This will not always be the case because some investigational treatments may not work as well as standard therapies or may have unexpected side effects. For this reason, all patients enrolled in clinical trials are carefully monitored. Frequent study-related medical examinations and tests are performed to ensure safety.

What Happens in a Clinical Trial?

Clinical trials are done to test whether new products are safe and work against disease. Study products are tested to see how they compare to standard treatments or to no treatment if there is not presently one. Many studies require that neither the patient nor the doctor know 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.

What questions should I ask?

Here are some questions to ask the study team which may help you decide if you want to take part in a clinical trial:

What is the reason for doing this trial?

Is this trial going to help me?

What medical tests and/or treatment will be done during the trial and for how long?

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 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?

Have there been other trials using this treatment?

What are the costs to me? Will my health insurance pay for it?

Will I be paid for mileage/hotel costs?

What will happen at the end of the study?

Can I quit before the end of the trial?

What are the Phases of Clinical Trials?

Most discoveries of new treatments are the result of laboratory experiments. When researchers are ready to evaluate a new treatment in people, they carefully design clinical trials to test the treatment's effectiveness, its safety, and its side effects. Clinical trials follow an orderly progression of steps or phases to take the medication or other type of treatment from discovery to approval by the Food and Drug Administration (FDA).

Phase I Trials

The very first trials to evaluate a new treatment may involve people without the illness or condition (healthy volunteers), or they may involve those who have the condition. Phase I clinical trials evaluate how a new treatment should be given (for example, orally or by injection), how often, and in what dose.

Phase II Trials

A phase II trial, which involves people with a particular illness or condition, provides preliminary information about the benefit and safety of the new treatment.

Phase III Trials

These trials are designed to compare the effectiveness and safety of the new medication or treatment with treatments already available for general use. Patients participating in Phase III trials are likely to be assigned by chance (randomized) to receive one of the following:

the new medication/treatment

the standard medication/treatment

an inactive substance called a placebo

Most of these trials are designed so that neither the participants nor the researchers know which treatment is being given to a particular individual. This is done so that the effects of the treatment are reported fairly.

Phase III trials involve hundreds or thousands of patients nationwide or even worldwide. If the results of a Phase III trial are favorable for the new treatment, then FDA approval will be sought. Once approved by the FDA, a treatment will be available to the general public.

Phase IV Trials

At times, even after FDA approval, additional information is needed on the effectiveness or the safety of a new treatment. If so, a phase IV clinical trial is conducted to provide such information.

Other Trials

Other supportive care, prevention, and screening trials are not done in phases, but instead compare groups of people using another strategy (counseling, behavior changes, detection method) with those who do not.

What are the risks/benefits?

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 don't always know what the side effects will be. Many side effects are temporary and go 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.

In weighing the risks of research you should consider two important factors:

the degree of harm that could result from participating in the study, and

the chances of any harm occurring.

The specific risks associated with a research protocol are described in detail in the informed consent document, which you will be asked to sign before participating in research. Also, a member of the research team will explain the risks of participating in a study and will answer any questions you have. Before deciding to participate, you should carefully consider the possible risks and benefits.

Potential benefits Well-designed and well-executed clinical trials provide the best approach for participants to:

Play an active role in their health care.

Gain access to new research treatments before they are widely available.

Receive regular and careful medical attention from a research team that includes doctors and other health professionals.

Help others by contributing to medical research.

Potential risks: Risks to participating in clinical trials include the following:

There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.

The study may require more time and attention than standard treatment would, including visits to the study site, more blood tests, more treatments, hospital stays, or complex dosage requirements.

How are people in clinical trials protected?

Researchers follow strict ethical and scientific principles to make sure that patients are protected and valid results are produced. A plan of how the study will be conducted, which is called a "protocol" is written to ensure that every researcher involved in the study follows the same ethical and scientific principles.

To ensure patient safety, the protocol must be reviewed and approved by the Food and Drug Administration (FDA) and a local Institutional Review Board (IRB). IRBs are comprised of Scientists, doctors and other people from the local community who review and monitor their hospital's or research institution's medical research involving people. They monitor studies to help make sure that there is the least possible risk to volunteers and that the risks are reasonable in relation to the expected benefits. IRBs make sure volunteer selection is fair and that informed consent is done correctly.

Other Ways Volunteers Are Protected

Data Monitoring Committees

These committees are used mainly when one treatment is being compared with another. These committees are particularly important in tests of treatments for serious or life threatening disease. These experts review information from studies to make sure they are being done in a way that is safest for the volunteers. During a study, if the committee finds that the treatment is harmful or of no benefit, it will stop the study. If a study shows that one treatment works better than another, the committee stops the study and all volunteers are offered the better treatment.

FDA Inspections

FDA inspects records and various scientists, clinics, and other research sites involved in a study. The agency does this to make sure volunteers are being protected and studies are being done correctly. From time to time such inspections are done in response to complaints.

What are my rights?

As a potential study participant you have the right to:

Refuse to participate or withdraw from participation at any time.

Ask questions and have your questions answered to the best of the investigators ability.

Obtain a copy of the informed consent document.

Be informed of any new findings related to the study that may alter your willingness to participate.

What is 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

that 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.

Informed consents must be written so you can understand it. If you don't, be sure to ask the doctor or other medical person to explain it. Make sure you understand all of it before you 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 understand it. The informed consent form is NOT a contract and you can leave the study at any time, for any reason.

What are eligibility criteria (inclusion/exclusion criteria)?

Participants in clinical trials must eligibility criteria (inclusion/exclusion criteria) that are outlined in the protocol. These criteria, which differ from trial to trial, include such characteristics as gender, age, health problems, and previous or current treatments. These criteria help to prevent those who may be harmed by the study drug or device (such as pregnant women) involved in the research. The criteria also help to produce scientifically reliable and valid results by ensuring that the same types of patients are enrolled across the country. This allows researchers to more accurately determine the benefits and risks of the study drug or device.

Can a participant leave a clinical trial?

Yes, trial participants may leave a clinical trial at any time for any reason. If you chose to leave the trial, you will have the opportunity to discuss other treatments with either your own doctor or a doctor from the trial.

Do all clinical trials involve a placebo?

No, not all clinical trials involve the use of placebo (an inactive substance that looks like the drug being tested). Some Phase III trials may compare investigational therapies to standard therapy used to treat the disease in question. However, in the case of some illnesses, no standard treatment exists therefore, in order to insure that the investigational drug is truly safe and effective the FDA may require that it be compared to placebo. When a placebo is used, trial participants are informed before they agree to take part in the trial that there is a chance that they will not receive the investigational drug, but may receive placebo instead.

What is a "blind" or "masked" trial?

Most clinical trials have a "treatment group" (those who receive study drug) and a "control group" (which may receive the current standard of care treatment or placebo). In a "blinded" or "masked" trial, participants are not told which group they are in. The reason for this is to avoid bias. If participants expect to improve on the investigational treatment they may report hopeful signs to researchers simply because they want to believe they are improving. Similarly, physicians and their staff may also be "blinded" to the treatment a trial participant is receiving so that their opinion of how the participant is doing is not affected by any preconceived opinions of the investigational treatment.

What does it mean to be "Randomized" in a clinical trial?

In trials where there are both treatment and control groups, participants are assigned to either group completely by chance (like the flip of a coin). This process is called "randomization." Randomization is used to help ensure that the trial results are influenced only by the treatment being tested.

Who pays for the study treatment in a clinical trial?

In most cases, the investigational drug or device used in clinical trials are provided at no cost to the participants. However, there may be other costs associated with the trial which should be discussed prior to enrollment. In some cases blood work, x-rays, and travel expenses are provided at no charge however, there are trials in which the trial related activities are considered "standard of care" and as such are billed to the participant's insurance provider.

Is a referral from my physician required for participation in a clinical trial?

No, you do not need a referral. While participants are encouraged to discuss participation with their own physician and family members, ultimately it is up to the individual to decide if they wish to participate in a clinical trial.

Can clinical trial participants continue to see their regular doctor?

Yes. Participation in a clinical trial does not replace normal health care. Trial participants are encouraged to continue their routine course of care with their current physicians.

What happens to the information gathered in a clinical trial?

The information gathered during the course of a clinical trial is carefully evaluated to determine whether the investigational drug or device is safe and effective enough to move into the next phase of study. In some cases the results are extremely encouraging but in some cases the investigational drug or device is determined to be unsafe or ineffective.

If a treatment is proven to be safe and effective after the completion of the phase III trial, the clinical trial results are presented to the FDA for review. If approved by the FDA, the new treatment then becomes available to the general public offering a new treatment option for physicians and their patients. In this way, clinical trials help to bring new therapies to patients with a wide range of medical conditions.

How Can I Find Out More About Clinical Trials?

One good way to find out if there are any treatments in clinical trials that might help you is to ask your doctor. Other sources of information include:

The Belmont Report, Ethical Principles and Guidelines for the Protection of Human Subjects of Research, April 18, 1979
Web site: http://www.hhs.gov/ohrp/humansubjects/guidance/belmont.htm

For cancer, call 1-800-4-CANCER (1-800-422-6237) or
visit this World Wide Website: http://www.cancer.gov/clinicaltrials

For AIDS and HIV, call 1-800-TRIALS-A (1-800-874-2572) or
visit this World Wide Website: http://www.actis.org/

For general information about clinical trials, call FDA's Office of Special Health Issues at 301-827-4460 or
visit this World Wide Website: http://www.fda.gov/

For other clinical trials of other diseases,
visit this World Wide Website:http://www.clinicaltrials.gov/