Expanded Access

Expanded Access is also called “compassionate use,” which provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions. Investigational drugs and devices have not yet been approved by the FDA and they have not been proven to be safe and effective. Therefore, they may be effective in the treatment of a condition, or they may not. It is important to remember that the drug/biologic/medical device may have unexpected serious side effects and that patients need to consider all the possible risks when seeking access to an investigational medical product. To gain access to an investigational medical product outside of a clinical trial, the sponsors must decide whether to make their experimental medical product available to patients via expanded access. FDA regulations specify two groups of people eligible for expanded access:

(a) those with life-threatening diseases or conditions for which “there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment”

(b) those with serious diseases or conditions that have a “substantial impact on day-to-day functioning”
In most cases, patients who seek compassionate use must have exhausted all approved therapies for their condition, and be unable to enrol in a clinical trial.