What does it mean “Expanded Access” also called “Compassionate Use?

When a patient does not respond to current approved treatments for a variety of reasons, other options still may be available.

Expanded access (Compassionate use) is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Most human use of investigational new drugs takes place in controlled clinical trials conducted to assess the safety and efficacy of new drugs. Data from these trials are used to determine whether a drug is safe and effective, and serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these controlled trials because of other health problems, age, or other factors, or are otherwise unable to enroll in such trials (e.g., a patient may not live sufficiently close to a clinical trial site).

For patients who cannot participate in a clinical trial of an investigational drug, but have a serious disease or condition that may benefit from treatment with the drug, FDA regulations enable manufacturers of such drugs to provide those patients access to the drug under certain situations, known as “expanded access.” For example, the drug cannot expose patients to unreasonable risks given the severity of the disease to be treated and the patient does not have any other satisfactory therapeutic options (e.g., an approved drug that could be used to treat the patient’s disease or condition). The manufacturer must be willing to make the drug available for expanded access use. The primary intent of expanded access is to provide treatment for a patient’s disease or condition, rather than to collect data about the study drug.

Some investigational drugs are available for treatment use from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. If you or a loved one is interested in treatment with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the protocol eligibility criteria and inquire at the Contact Information number. If there is not an expanded access protocol listed in ClinicalTrials.gov, you or your health care provider may contact a manufacturer of an investigational drug directly to ask about expanded access programs.

Under the Federal Food, Drug, and Cosmetic Act, a patient may seek individual patient expanded access (sometimes called single patient access) to investigational products for the diagnosis, monitoring, or treatment of a serious disease or condition if the following conditions are met.
The person’s physician determines that there is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the person’s disease or condition, and that the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition;

FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;

FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; and

The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with FDA’s statute and applicable regulations for INDs or investigational device exemption applications (IDEs), describing the use of the investigational product.

Also under FDA’s statute, a sponsor or a physician may submit a protocol intended to provide widespread access to an investigational product. In this scenario, FDA will permit the investigational product to be made available under a treatment IND or treatment IDE if certain criteria are met.

Read more:
http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm20080392.htm