The following are the most frequently asked questions about clinical trials.The information presented has been compiled with information from clinicaltrials.gov, a service of the National Institutes of Health.

• What is a clinical trial?

• Are clinical trials proven treatment?

• Why participate in a clinical trial?

• What is a protocol?

• What are the different types of clinical trials?

• What are the phases of clinical trials?

• What is a control or control group?

• What is a placebo?

• What is randomization?

• Is the government involved in all clinical trials?

• How is the government involved in clinical trials at UF and Shands?

• How do UF and Shands protect research subjects?

• What is the Institutional Review Board, and how does it protect research subjects?

• What is informed consent, and how does it work?

• What trial-specific information should participants know?

• What is the significance of "risks v. benefits"?

• What should participants know about conflict of interest?

• Are there alternatives to the clinical trial?

What is a clinical trial?

According to clinicaltrials.gov, a clinical trial is a "research study to answer specific questions about vaccines, 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 in people."

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Are clinical trials proven treatment?

No, clinical trials are not proven treatment. They are research, and the purpose of all clinical trials is to learn more about what is being studied in the trial.

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Why participate in a clinical trial?

According to clinicaltrials.gov, "participants in clinical trials can play a more active role in their healthcare, gain access to new research treatments before they are widely available, and help others by contributing to medical research."

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What is a protocol?

A protocol is the study plan on which the clinical trial is based. The plan is designed to protect the participants and to answer specific research questions. The protocol details who may participate in the trial, what will be done to participants, how long the study will be conducted and everything that is required for the study.

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What are the different types of clinical trials?

Five of the typical types of clinical trials are listed below. Some trials may incorporate more than one type of trial.

• 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 the disease from returning. Approaches may include medicines, vitamins, vaccines, minerals or lifestyle changes.

• Screening trials try to find a better way to detect certain diseases or health conditions.

• Quality of life trials explore ways to improve comfort and quality of life for individuals with chronic illness.

• Nontherapeutic trials hope to answer specific research questions without using any type of treatment on participants. This may be accomplished by reviewing medical records, doing certain types of medical tests or asking participants questions in a survey.

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What are the phases of clinical trials?

There are four phases of clinical trials, each of which has a different purpose and answers different questions for scientists.

• Phase I trials test a new drug or treatment in a small group of people (20-80) for the first time in order to evaluate the treatment's safety, determine how much can be given safely and identify possible side effects.

• Phase II trials typically test the treatment in a larger group of people (100-300) to see if the treatment is effective and to further evaluate the safety of the treatment.

  • Pilot studies are usually Phase II studies but use a much smaller group of people (10-30) to determine if the study should be conducted on a much larger scale.

• Phase III trials typically test the treatment in a large group of people (1,000-3,000) to confirm that the treatment is effective, monitor side effects, compare the treatment to other commonly used treatments, and collect additional information that should enable safe use of the treatment. Patients usually are randomized between different treatments.

• Phase IV trials are usually post-marketing studies that establish additional information about the treatment's risks, benefits and best use.

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What is a control or control group?

According to clinicaltrials.gov, "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 of the illness or a placebo."

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What is a placebo?

A placebo can by any form of a medication, or it may look like the experimental drug or device being studied. The placebo provides no treatment value and should not affect patients in any way. Subjects assigned to receive a placebo will not be exposed to any of the risks of the experimental treatment and will not receive any of the benefits of the experiemental treatment.

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What is randomization?

Randomization is a process whereby researchers can easily compare results between individuals receiving different treaments in trials in which more than one type of treatment is available. The treatment a study subject receives is determined by chance, similar to flipping a coin or rolling dice. Neither the study participant nor the researcher may select to which group the participant will be assigned.

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Is the government involved in all clinical trials?

No, the government is not involved in all clinical trials. However, according to our agreement with the government (called an assurance), all clinical trials conducted at UF and Shands will comply with federal regulations. These regulations include the Office of Human Research Protections Code of Federal Regulations 45 CFR 46 (the "Common Rule"), the FDA's rules and regulations applying to the use of investigational drugs or devices, and the FDA's Good Clinical Practice guidelines.

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How is the government involved in clinical trials at UF and Shands?

UF has an agreement with the government called an assurance that requires all clinical trials conducted in a Shands facility or by UF faculty and staff to comply with federal regulations as stipulated in 45 CFR 46, 21 CFR, etc.

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How do UF and Shands protect reserach subjects?

All clinical trials must be reviewed and approved by an Institutional Review Board. IRBs are required by the government to review research to ensure the protection of research participants.

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What is the Institutional Review Board, and how does it protect research participants?

The IRB consists of a committee of physicians, statisticians, researchers, community advocates and others. The U.S. government requires all research involving human subjects to be reviewed and approved by an IRB prior to any subject particpation and periodically thereafter in order to protect the rights of human participants.

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What is informed consent, and how does it work?

Informed consent is the process of educating people about the key facts of a clinical trial and allowing people to voluntarily decide whether to paricipate in the trial. There are typically three steps taken to inform potential participants about the study:

• Someone who is involved in and familiar with the trial explains the details of the study.

• Potential participants are provided with two copies of the IRB-approved informed consent form. The potential participant should keep one of these forms for their records. The other form should be signed and returned to the investigator if the subject chooses to take part in the trial.

• Potential participants should have an opportunity to ask questions about the trial, their condition/illness, and alternatives to participating in the trial.

Informed consent does not end once someone agrees to participate in a trial. According to federal regulations, informed consent is an ongoing process that continues throughout the trial to ensure that participants remain fully informed about the trial. Participants should be informed about any new information discovered during the trial whether it is about the trial itself, the condition/illness being studied, or possible new alternatives to the trial.

The informed consent document is reviewed and approved by the IRB. The form outlines the right of study participants and details about the study such as its purpose, duration, required procedures, risks, benefits, alternatives and key contacts. You are not required to sign the informed consent document unless you want to participate in the trial. This form is not a contract -- participants may choose to withdraw from the trial at any time after agreeing to participate.

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What trial-specific information should patients know?

Participants should be provided with the following information:

• Title: What is the official title of the trial?

• Phase: In what phase is the trial?

• Purpose: What is the trial about? What do researchers hope to learn?

• Eligibility criteria: Who may or may not participate in the trial?

• Trial duration: How long will the research be conducted?

• Time commitment for subject: How much time does each research participant have to spend in order to be part of the trial?

• Description: What will be done to the participants in the trial?

  • What kind of tests and treatments are involved?

  • How might the trial affect daily life?

  • Will participants have to be admitted to the hopsital during the trial?

  • Who will be in charge of participants' care?

  • How will participants know if the treatment is working?

  • Who will pay for the treatment?

  • Will participants be reimbursed for other expenses?

  • What will be donce once the treatment is finished?

  • Will results of the trial be provided to participants?

• Balanced description of risks v. benefits: What is a realistic explanation of what may happen (both good and bad) to participants in the trial? Click here to learn more about risks v. benefits.

• Compensation for injury: How will participants be compensated if they are injured as a result of participating in this trial?

• Trial sponsors: Who is funding the trial?

• Names of clinical investigators: Who is conducting the trial locally?

• Contact information: Who can participants contact with questions about the trial?

• Alternatives to the trial: What other types of treatments are available if a person chooses not to participate in the trial?

• Conflict of interest: Do any of the researchers, the hospital or the university stand to benefit personally or privately from specific outcomes?

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What is the significance of "risks v. benefits"?

Participating in a clinical trial may expose people to known and/or unknown risks. Risks can consist of physical side effects from the study such as headache, nausea, hair loss or skin irritation or a wide variety of other physical problems. Other risks could be financial or psychological.

Some risks may occur immediately while participating in the trial while others may not occur until much later, sometimes years, after participating in the trial. The risks should be detailed in the informed consent form and should be described by a knowledgeable researcher affiliated with the trial. During a trial, participants should be notified of any changes in the risks associated with the study.

The primary benefit of participating in a trial is that the knowledge obtained from the study may help other people in the future. Previous studies or other information may indicate that study subjects might benefit directly by participating in a research trial, but this can never be guaranteed.

Participants should compare the risks and benefits of participating. Participants may decide that the potential benefits are not worth the risks.

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What should participants know about conflict of interest?

Sometimes people or institutions might benefit if the results of a clinical trial are positive. For example, a physician or the university might make money if they own the patents for an effective drug or device studied in the trial. UF and the IRB strive to ensure that conflicts of interest do not affect the clinical trial process. The informed consent form received by potential subjects contains information about possible conflicts to help them decide if they want to participate in the clinical trial.

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Are there alternatives to the clinical trial?

First and foremost, no one is ever required to participate in a clinical trial. Participation in a clinical trial is voluntary, and potential subjects may always decide not to particpate without it affecting their relationship with the researcher, other medical staff, the hospital and/or the university.

Most times there are alternative treatments that people can receive rather than participating in the clinical trial. Sometimes you can receive the same therapy without enrolling in the clinical trial. Other times the only way to receive a certain drug or device is to participate in the clinical trial.