Using Investigational Medicines

Investigational medicines may be used if you are seriously ill but you're not enrolled in a clinical trial. These medicines are being studied but have not yet been approved as safe and effective by the U.S. Food and Drug Administration (FDA). This is sometimes called compassionate medicine use.

Normally, investigational medicines have strict testing through many stages of clinical trials before they are available to the public. They are tested for safety and side effects. They are also tested to see how well they work and at what doses and with what conditions or diseases they are to be used. The process may take many years. In certain cases, these medicines can be used outside of a clinical trial.

Investigational medicine use is legal. The FDA has been approving investigational medicines to be used in this way for critically ill people since 1987.

The most common way people access unapproved medicines is by enrolling in a clinical trial. This is the safest route. The FDA closely regulates clinical trials, and people who are taking the new medicines are carefully monitored. Not everyone will qualify for a clinical trial. Because the use of new, unapproved medicines is risky, only certain people who are not enrolled in clinical trials can get investigational medicines for compassionate use. According to guidelines from the National Cancer Institute (NCI), you are eligible if:

• You have used standard treatments and they didn't work.
• You have an illness that is life-threatening.
• You are not eligible for any current clinical trials.
• You have no acceptable treatment options.
• You have a cancer diagnosis for which an investigational medicine has shown some benefit and is currently being studied in clinical trials.
• You are likely to have benefits that outweigh the risks involved.

To use investigational medicines, your doctor has to apply for them through a treatment investigational new drug application (treatment IND). The application process includes information on your condition and past treatments.

The most common way to get approval for an investigational medicine outside of clinical trials is through expanded access or a special exception or single-patient exception.

• Expanded access uses medicines that have been well-tested and are nearing FDA approval. The medicine must show in studies that it is effective at treating a specific cancer. The program is offered by a medicine company, and the medicine is given out to a group of people.
• Special exceptions are individual requests (often from a doctor) that are made to medicine companies or whoever is overseeing a program or clinical trial. The decision to give out the medicine is weighed on a case-by-case basis. There must be some proof that the medicine would either improve a person's quality of life or lengthen their life.

There are downsides to compassionate medicine use for you and for the medicine approval process. One is that this process may interfere with clinical trials if more people seek compassionate use rather than being part of clinical trials. Although the NCI gathers some safety information on the use of investigational medicines, it is not as helpful in getting FDA approval of these medicines. You are not monitored as closely as you would be if you were part of a clinical trial.

Other problems include:

• The chance that you'll get no lasting benefit from the medicine.
• Harmful side effects that may not have been discovered yet.
• Extra costs that are linked to using the medicine may not be covered by health insurance.

To learn more about investigational medicines and clinical trials, talk with your doctor about your options, the risks and side effects of the medicine you are considering, and what the process is to apply to use the medicine. Clear communication with the doctor who treats you is important. Together, you can determine whether a clinical trial or compassionate use is right for you.

Call NCI's Cancer Information Service at 800-4-CANCER (800-422-6237) or visit www.clinicaltrials.gov to search for clinical trials for your condition.

If you don't qualify for a clinical trial, your doctor can ask the trial's sponsor about requesting a single-patient exception. Your doctor may also contact the NCI or the company making the medicine to find out if expanded-access programs exist.