What is an investigational drug?

An investigational drug is one that is under study but does not have permission from the U.S. Food and Drug Administration (FDA) to be legally marketed and sold in the United States.

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FDA approval is the final step in the process of drug development. The first step is for the new drug to be tested in the laboratory. If the results are promising, the drug company or sponsor must apply for FDA approval to test the drug in people. This is called an Investigational New Drug (IND) Application. Once the IND is approved, clinical trials can begin. Clinical trials are research studies to determine the safety and measure the effectiveness of the drug in people. Once clinical trials are completed, the sponsor submits the study results in a New Drug Application or Biologics License Application to the FDA. This application is carefully reviewed and, if the drug is found to be reasonably safe and effective, it is approved.

How do patients get investigational drugs?

By far, the most common way that patients get investigational drugs is by taking part in a clinical trial sponsored under an IND. A patient’s doctor may suggest a clinical trial as one treatment option. Or a patient or family member can ask the doctor about clinical trials or new drugs available for cancer treatment.

Patients and their families can also learn about new drugs being tested in clinical trials by searching NCI’s clinical trials database or by calling NCI’s Cancer Information Service (CIS) at 1–800–4–CANCER (1–800–422–6237). CIS information specialists can search the database and provide a list of trials for individuals to discuss with their doctor.