Single-Patient Expanded Access

Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition, to gain access to an investigational medical product (drug, biologic, or medical device) to treat their condition outside of a clinical trial. The term “compassionate use” is sometimes used to describe this type of use. Whenever possible, an investigational medical product should be used in the context of a clinical trial. However, if patient enrollment in a clinical trial is not possible (for example, the patient does not meet the eligibility criteria for current trials, or there are no trials open), then expanded access offers a possible route for the physician to gain access to the product for their patient. This process is also sometimes used to continue to provide a medical product to clinical trial participants who are deriving benefit from the product, after a clinical trial ends.

Expanded access requests, while not clinical trials, still require that the physician submit an IND or IDE to the FDA to allow the use of the product. These applications are typically much shorter than those required for a clinical trial. FDA will review the application and notify the physician if the use is approved, within 30 days of receipt and often sooner. Expanded access of an investigational product also requires IRB approval.

There is a subset of single-patient expanded access called emergency use expanded access. This is for cases where the patient is in a life-threatening situation and needs treatment immediately, and there is not time to use existing procedures. In these cases the initial communication with FDA is done by phone and the FDA gives a verbal authorization. The paperwork is then submitted in the next few days. For emergency use expanded access, IRB approval can take the form of concurrence by an IRB chair, or notification to the IRB up to 5 days following the emergency use.

Please contact the IND/IDE Management Office for assistance regarding the appropriate mechanism for submission, which varies depending on therapeutic area and product type.

For Emergency Expanded Access IND requests you may call FDA’s Emergency Call Center at 866-300-4374 to request the use, and follow up with the IND/IDE Management Office for the formal submission of the documents.

Helpful Forms and Links

FDA Expanded Access webpage

FDA Guidance Q&A

For Drug or Biologics Single-Patient Expanded Access IND you will need to collect:

3926 form (and instructions)
Letter of Authorization from the company
Copy of treating physician (IND holder) CV
Informed consent form to be used with the patient

For Medical Devices, you will need to collect:

A description of the patient’s condition and circumstances necessitating treatment, discussion of why alternative therapies are not suitable, and why the probable risk of using the investigational device is not greater than that from the patient’s disease or condition
Treatment plan for the use of the medical device and any patient safety monitoring measures to be implemented
Authorization from the device manufacturer for the use of the device
Informed consent form to be used with the patient
Clearance from the institution as required by internal processes
An independent assessment form an uninvolved physician
Concurrence of the IRB chairperson

If there is an IDE for the device, the IDE Holder should submit an IDE Supplement requesting approval for the compassionate use; the treating physician is not required to submit a separate expanded access IDE. However if there is not an IDE for the device, the physician will need to submit an IDE for the expanded access use.

Single-Patient Expanded Access

Helpful Forms and Links

IND/IDE Office Contact