About Clinical Trials

Expanded access programs (EAPs) make experimental drugs available to those patients with limited or no treatment options before the U.S. Food and Drug Administration (FDA) approval process for the drug is complete. The approval process involves three phases of clinical trials to determine if the experimental drug is safe and effective. In some clinical trials, treatments that have already been approved are tested for use in new formulations, or for different conditions or illnesses.

During the later stages of the clinical trial process, some experimental drugs may be considered for an EAP if they treat conditions that are serious or life-threatening in people with limited or no treatment options. For more in-depth information regarding clinical trials and EAPs, see www.clinicaltrials.gov.

About clinical trial participation

There are benefits and risks associated with participating in clinical trials. If you or a loved one is interested in participating in a clinical trial, it's important that you learn as much as you possibly can about that trial. "Informed consent" is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also an ongoing process during the study to provide information to participants. You or a loved one will be asked to sign an informed consent document prior to any study-related procedures. If the study participant is under the age of 18, a parent or guardian will be asked to provide this written consent.

When you or a loved one consent to participate in a clinical trial, there is the potential benefit of gaining access to a new research treatment with expert medical care. But, there is also the possibility of generally known and unknown risks. These risks could include unpleasant or even life-threatening side effects. The treatment may also end up not being effective for you or your loved one. It's important to talk to your doctor about questions and concerns to make sure you and your loved one are comfortable with your decision. The informed consent document is not a contract, so you or your loved one may withdraw from the trial at any time.

Ultimately, participation in a clinical trial can be a contribution to the health of others with cystic fibrosis, whether the treatment being tested goes on to be widely used or is found to be ineffective and not approved.

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