Compassionate Use / Expanded Access

FDA regulations use the term "Expanded Access" although you may also see the terms "Compassionate Use" or "Treatment Use".  Expanded Access regulations provide a mechanism to use or administer an unapproved medical device or investigational drug outside a clinical trial.

Submissions for all categories of Expanded Access require sponsors to include adequate information to demonstrate that such access to the drug or device will not interfere with its clinical investigations or product development.  The FDA must determine that access to the investigational product for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the Expanded Access use, or otherwise compromise its potential development.

Investigational Devices:

Expanded Access for devices includes emergency use and treatment use of an investigational device to treat a patient with a serious or immediately life-threatening disease or condition for whom there are no comparable or satisfactory alternative treatment options.

Investigational Drugs:

Expanded Access for drugs permits the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition for whom there are no comparable or satisfactory alternative treatment options.