Transcript: Expanded Access Part 1: Introduction (May 2019)
Hi, my name is Monica Hughes, and I am a Chief Project Manager at FDA. This video is the first in a series of four that describe how physicians can submit expanded access requests for investigational drugs, including biologics, to the FDA, on behalf of a patient.
Expanded access is a program regulated by FDA through which manufacturers can provide investigational medical products to patients for treatment use. Expanded access can provide seriously ill patients with access to investigational treatments when they have no other options and they are willing to accept greater risk.
For a patient to qualify for expanded access to an investigational drug:
- the patient must have a serious or immediately life-threatening disease or condition for which there is no satisfactory alternative therapy,
- the patient must have exhausted approved therapies and is unable to participate in available clinical trials, and
- the potential risks from the drug does not exceed that from the disease.
Once these criteria are met, you can request expanded access by submitting, depending on the situation, either an Emergency or a Non-emergency Single Patient Investigational New Drug Application to FDA. Investigational New Drug Application is commonly abbreviated as IND.
We recognize that time is critical for patients who have a serious illness or condition. Applications are authorized quickly, often in a matter of days. In emergency cases, FDA will typically provide authorization over the phone within hours.
You should be aware of 2 criteria that must be met before FDA can authorize your application.
- The benefit to the patient must justify the potential risks of the treatment.
- The use of the investigational drug for the requested treatment must not interfere with the product’s development.
In addition, the manufacturer must be willing to provide the drug. FDA cannot require a manufacturer to provide a product.
FDA has a long history of supporting patient access to investigational new treatments. We recognize that this may be your first time exploring expanded access, and we’re here to help if you have questions.
If you’re already working with the appropriate Review Division, give them a call. If you’re just getting started, contact the Division of Drug Information.