FDA News about Expanded Access Program

"The National Organization for Rare Disorders (NORD) applauds the Food and Drug Administration (FDA) for finalizing its efforts to streamline the process used by patients and their physicians seeking expanded access to investigational new drugs and biologics.

Their release of the final Individual Patient Expanded Access Investigational New Drug Application - Form FDA 3926 - can be used by physicians to request expanded access, often referred to as “compassionate use,” for patients suffering from serious or immediately life-threatening diseases and for whom no comparable or satisfactory alternative therapy is available. The shortened form reduces the time to completion from hours to only 45 minutes; a drastic improvement in alleviating the burden on patients and their physicians. To provide further clarity, step-by-step instructions have also been provided for completing the new form.

In addition, the FDA also released two additional final guidance documents outlining what expanded access is, when and how to request it, and the type of information required to do so. The other provides regulations on how patients may be charged for investigational new drugs.

NORD submitted comments to the FDA on the Individual Patient Expanded Access Application: Form FDA 3926 in April 2015 outlining our enthusiastic support for the creation of a unique form tailored specifically to individual patient expanded access requests."

- From NORD Press Release.