Patients and Caregivers:

Clinical Trials

The process that brings a new treatment from the research laboratory to the patient is a long and rigorous process. One of the most important steps in developing treatments is clinical trials, where people volunteer to receive an experimental therapy and be observed for its effects. These studies determine whether the treatment can benefit humans.

People like you must volunteer and participate in these trials, which may benefit not only the individual but many others as well. Whether a clinical trial is right for you depends on many things, including your medical condition. Your physician will discuss these factors with you and provide information to you on the trial.

Participation in a clinical trial can be a large undertaking, with no guarantees that the treatment will work. Below is information that will help you understand the benefits and risks, and what to expect if you decide to take part.

What is a Clinical Trial?

A clinical trial is a research study that takes place in a hospital, clinic or other healthcare facility in which a doctor and other health care professionals observe a patient. The treatment is studied for its effects on a group of people.  Before a treatment enters clinical trials, it is extensively tested in preclinical studies in the laboratory. These studies provide information on the treatment's possible benefits and limits before it is used in humans.

Both preclinical and clinical studies are carefully supervised, monitored and documented. New treatments must demonstrate their safety and efficacy to government regulators (FDA, or Food and Drug Administration, in the United States) before they can be approved to be sold to all patients. Clinical trial volunteers play an important role in this process.

Only you can decide whether or not a clinical trial is right for you, and you should take the time to think about the trial and ask questions, and talk to friends and family members to help you in your decision. Keep in mind that there are risks associated with clinical trials. Have all the facts before you decide.

What are the Risks Involved in Clinical Trials?

Treatments are tested in clinical trials to learn more about them. Therefore we may learn some drawbacks to a treatment which will not be uncovered until after treatment begins. Your physician will discuss these potential risks with you. You must carefully weigh them against the possible benefits, and ask as many questions as you like. Keep the following in mind when evaluating risks.

• Sometimes the side effects may be no worse than those of your current treatment options. Other times, the side effects may seem greater.
• You may react differently from other people when given the same treatment. Your physician will ask you questions to learn as much as possible about your medical history in order to reduce the potential risks.
• The risks are carefully looked at by your physician, the study physician, the FDA, and an Institutional Review Board (IRB), which is a group of doctors, scientists, patient advocates, legal representatives and clergy which reviews a clinical study before the study begins.
• If something shows up that is a threat to patients in the clinical trial, the patients will be notified by the investigator and the trial may be stopped or placed on hold until more information is known.

What is a Placebo?

A placebo is an inactive product used to compare the active treatment. You and your health care professional will not know which individual has received the placebo and which has not. Therefore, observations in the clinical trial can be conducted more fairly.

What are the Benefits of Participating in a Clinical Trial?

There are a number of potential benefits from participating in a clinical trial:
• To become involved in the testing of a new treatment that would not otherwise be available.
• The possibility of free study medication and medical care during the clinical trial.
• Contributing to medical science which may help others in the future.

Where are Clinical Trials Performed?

Clinical trials can occur in a variety of places. Some are performed in a hospital and others right in the doctor's office. Still others are done in special research centers.
If you decide to participate in a clinical trial, you will receive information regarding the treatment location. In some cases, provisions may even be made for family members if the trial is to be conducted away from home.

Are Volunteers Required to Pay for Participation in Clinical Trials?

No. In most cases, study medication is provided free of charge. Also, the costs of tests associated with the trial will not be charged to you. Your health care professional can tell you exactly what will be covered as part of the clinical trial and what will be considered part of your normal health care routine.

How Do People Become Involved in a Clinical Trial?

People interested in clinical trial participation should discuss this option with their physician. It's the best way to learn whether or not an appropriate trial is available, and where it is being conducted. Patients are screened to determine if they are right for a given trial. Sometimes screening involves just a series of questions, or it may require laboratory tests. In any case, these tests are performed only with the patient's consent (agreement to participate).

Informed consent means that your healthcare professional will explain the clinical trial to you in detail and provide a package of information on the trial along with an informed consent form. The form provides information on the length of the study, potential side effects (if known) from the treatment being tested, how the study protocol is designed, whether or not a placebo is to be used, what kind of tests will be required, your rights to withdraw from the study, and when the study is considered completed. If you don't understand any of the document, it is important that you ask your physician or nurse.

Once you are satisfied that you have a full understanding of the trial and decide to proceed, you may sign the informed consent form. However, remember that you always have the right to say no. If you are uncomfortable with what you are being asked to do in the study, or if you simply don't want to participate any longer, you do not have to participate.

What are the Participant's Responsibilities?

Your responsibilities will be explained to you by your healthcare professional; in general, you must see your physician as scheduled and take your medication as prescribed. You may also be asked to keep a diary of your experience. You should also contact your physician as soon as possible if you are hospitalized for any reason.

It is important to let all of your physicians know that you are participating in a clinical trial. They will need the name and phone number of the study physician in order to let him or her know about any pertinent medical information which may affect your treatment in the trial. You should keep the study physician's name and number with you at all times in case of emergency.