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. FDA approval is the final step in the process of drug development. 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 Investigational New Drug. 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. Are there other ways to get investigational drugs? Less common ways that patients can receive investigational drugs include mechanisms such as the special or compassionate exception. The sponsor, such as the pharmaceutical company must agree to provide the drug for this use. Investigational drugs given under these mechanisms must meet the following criteria: • Ther
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. 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 (NDA) or Biologics License Application (BLA) 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 com
