Expanded Access

Non-emergent Expanded Access to Investigational Drugs

IMPORTANT: A single patient expanded access may take up to 30-days for FDA to perform a safety review.  If the intervention must occur within days to weeks, follow the procedures for an emergency expanded access request. Emergent requests are not required to be submitted to the IIS team. Work directly with the manufacturer, FDA, and IRB.  The IIS team is available for guidance on an emergency request.

What is expanded access?

Expanded access, sometimes referred to as compassionate use or a single patient IND, refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient rather than to obtain the kind of information about the drug that is generally derived from clinical trials.

Who is eligible for expanded access?

• Patients with an immediately life-threatening disease or condition defined as a stage of disease in which there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment; OR
   
• Patients with a serious disease or condition associated with morbidity that has substantial impact on day-to-day functioning.

Who can make an expanded access request?

• The sponsor of an existing IND (manufacturer or sponsor-investigator)
   
• The patient’s physician

What is an intermediate-sized population expanded access IND?

Access to an investigational drug (including a biologic) for use by more than one patient, but generally fewer patients than are treated under a typical treatment IND or protocol, submitted as a protocol under a new IND. The investigational product may or may not be under development for marketing. Unless FDA notifies the sponsor that treatment may begin earlier, there is a 30-day waiting period before treatment may begin.