Access to Investigational Drugs

Investigational or experimental drugs are new drugs that have not yet been approved by the FDA or approved drugs that have not yet been approved for a new use, and are in the process of being tested for safety and effectiveness.

Patients may decide to seek access to investigational drugs for different reasons. Some patients with serious or life-threatening illnesses seek treatment with investigational drugs if FDA-approved therapies are not working or if their side effects are too severe. Others may have heard about promising early study results for a specific investigational drug, and they might want to learn more.

Investigational drugs are available through two pathways designed to protect patients, because an investigational drug may pose unknown risks to patients and we do not know if it is effective. Patients may be eligible to receive an investigational drug as a participant in a clinical trial or as part of an expanded access program  (also known as compassionate use). If you are interested in seeking access to an investigational drug, the information provided here can help guide your decision and your next steps.

• Deciding Whether to Seek Access to an Investigational Drug
• Clinical Trials and Investigational Drugs
• Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access)