Frequently Asked Questions About Clinical Trials

Participants in clinical trials gain access to new research treatments before they are widely available. Often the process of collecting information in the study will allow your doctor to find out more about your condition and the effects it has on you. This may allow you to benefit from better treatment after completing the trial. Many patients also derive satisfaction from knowing they are aiding in an effort to help reduce the suffering of other people suffering from the same ailment.

Benefits: Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

• Gain access to new research treatments before they are widely available.
• Obtain expert medical care during the trial.
• Help others by contributing to medical research.

Risks: There are risks to clinical trials.

• There may be unpleasant, or even serious side effects to treatment.
• The treatment may not be effective for the participant.
• The study may require more of their time and attention, including trips to the study site, more treatments, or complex dosage requirements.

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan that details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various governmental agencies. Individual participants’ names will remain secret and will not be mentioned in these reports.

People should know as much as possible about the clinical trial and feel comfortable asking the members of the healthcare team questions about it. The following questions might be helpful for you to discuss with the healthcare team. Some of the answers to these questions are found in the informed consent document.

• What is the purpose of the study?
• Who is going to be in the study?
• Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
• What kinds of tests and treatments are involved?
• How do the possible risks, side effects, and benefits in the study compare with my current treatment?
• How might this trial affect my daily life?
• How long will the trial last?
• Will I be reimbursed for other expenses?
• What type of long-term follow up care is part of this study?
• How will I know that the treatment is working? Will results of the trials be provided to me?
• Who will be in charge of my care?

• Plan ahead and write down possible questions to ask.
• If you would feel more comfortable, ask a friend or relative to come along for support.
• Bring a notebook to take notes while meeting with the doctor.

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provided extended or complete primary healthcare. Additionally, this will ensure that other medications or treatments provided by your primary doctor not conflict with the study medication.

Yes. You can leave a clinical trial at any time. When withdrawing from the trial, you should let the research team know and briefly explain your reasons for the leaving the study.

A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest way to find new treatments that will improve health problems.

Our clinical trial team includes doctors, psychologists, nurses, and study coordinators. They check your health at the beginning of the trial, give specific instructions for participating in the trial, and monitor your health carefully during the trial.

Side effects are any undesired effects of the study medication. These effects may include headache, nausea, skin irritation, or other physical problems.

All clinical trials have guidelines about who can participate, which are an important aspect of medical research that helps to produce reliable results. "Inclusion criteria" are the factors that allow someone to participate in a clinical trial, while "exclusion criteria" are those that disallow someone from participating. These criteria are based on such factors as age, the type and stage of disease, previous treatment history, and other medical conditions. The criteria are used to identify appropriate participants, keep those participants safe, and ensure that researchers are able to answer the desired questions. Before joining a clinical trial, a participant must qualify for the study. Our standard practice is to have a study coordinator conduct a telephone screen with you to determine whether you meet the criteria for the study and whether the study may be a benefit to you.

Anyone entering a clinical trial in the United States is required to sign an informed consent, a form indicating that they understand what will happen to them during the study. It is a continuing process throughout the study to provide updated information for participants. To help you decide whether or not to participate, the doctor will help to explain the details of the study. Then you will be given the informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. Then you decide whether or not to sign the document. Informed consent is not a contract, but an agreement that you understand the study. You may withdraw from the trial at any time.

The pharmaceutical company, government research institution, or other health organization that funds a clinical trial and designs its protocol.

A protocol is the plan for how to conduct the clinical trials. The plan is carefully designed to safeguard the health of the participants while answering specific research questions. 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.

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.

The Food and Drug Administration (FDA) is a government agency that monitors the manufacture, testing, effectiveness and use of drugs and medical devices. The FDA must initially approve a drug before it can be further tested on humans in our studies. When the results of the studies are completed, the FDA will then determine the effectiveness of the drug to determine whether it should be placed on the market for general public use.

A placebo is an inactive pill that has no treatment value. In clinical trials, experimental treatments are often compared with a placebo to determine the effectiveness of the treatment. In some studies, a group of participants are randomly selected to receive a placebo instead of an active medication.

If you and the study doctor determine your condition is not improving while you are in the study, it could be because you are on the placebo. We will then provide you with other standardized treatment, which you and the study doctor determine, and discuss with you other treatment options.

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists to answer different questions:

• In Phase I trials, researchers test a new drug in a small group of people (20-80 for the first time to evaluate its safety, determine a safe dosage range, and identify any side effects.

• In Phase II trials, the study drug is given to a larger group of people (100-300) to determine the effectiveness and safety.
• In Phase III trials, the study drug is given to a larger group of people (1,000-3,000) to confirm its effectiveness, determine its safety, monitor side effects, and compare it to commonly used treatments.
• In Phase IV trials, post-marketing studies identify additional information including the drug’s risks, benefits, and optimal use.