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Understanding the Approval Process for New Cancer Treatments
    Posted: 12/30/1999    Updated: 01/06/2004

Understanding the Approval Process for New Cancer

FDA's Role

Clinical Trials

Special Needs

Q&A: Off-Label Drugs


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Understanding the Approval Process for New Cancer Treatments

Every year, medical researchers develop new cancer treatments or new uses for treatments already on the market. These treatments are most often drugs, chemically produced substances used to treat or prevent disease. But they may also be biologics, treatments that are made from living organisms such as vaccines or recombinant proteins.

The U.S. Food and Drug Administration (FDA) is the division of the U.S. Department of Health and Human Services charged with making sure that drugs and biologics are safe and effective before they go on the market. The FDA maintains a list of drugs and biologics approved for use with cancer.

FDA regulators face two main challenges during the approval process. First, they must make sure the treatment is safe and effective. For this, regulators rely on the results of clinical trials - research studies that test how well medical treatments or other interventions work in people. (For more information about trials, see What Is a Clinical Trial?)

The second challenge is to make promising treatments available as quickly as possible to the people most in need of them. Ordinarily this occurs through clinical trials. The FDA may also allow access to an unapproved investigational treatment outside of a clinical trial, if no approved therapy for the disease exists.

This guide will acquaint you with the main parts of the FDA approval process and point you to other resources for learning more about it.

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