Expanded Access Programmes

Expanded access or compassionate use is the use of an unauthorized drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by the patient with an immediately life-threatening illness or severely debilitating disease or condition who do not meet the enrollment criteria for the clinical trial in progress. These programs go under various names, including early access, special access, or managed access program, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

When patient participation in clinical trials is not possible due to ineligibility or scarcity of ongoing trials, or if enrollment in clinical trials is not conceivable because the distance to the trial inhibits access, expanded access offers a possible route for gaining access to an investigational medical product. These are investigational drugs, biologics or medical devices that are being tested to see whether they are safe and effective. As investigational drugs have not yet received regulatory approval from FDA, the agency has not found these products to be safe or effective for their specific use. It is very important to remember that their potential risks and benefits are not yet established and their effectiveness to treat the condition is not yet known.