FDA Expanded Access Pathway
The Food and Drug Administration’s (FDA’s) expanded access program provides a pathway for critically ill patients to try investigational (unapproved) drugs, biologics, and medical devices as a last resort. The program includes special provisions for emergency treatment access.
Deciding to seek treatment via the FDA expanded access pathway is a serious decision. It’s important to realize that there are many unknowns about medical products still in development. And the earlier in development, the less is usually known about the safety of the drug, biologic, or medical device, or how effective it will be in treating a particular disease or condition.
Who is eligible?
FDA expanded access is available only to patients who have a serious or life-threatening condition—and have exhausted all other treatment options. The terms “serious” and “life-threatening” are defined as follows:
- A serious disease or condition is one that has substantial impact on day-to-day functioning. Short-lived and self-limiting illness will usually not be sufficient. However, the illness need not be irreversible, provided it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on clinical outcomes such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.
- An immediately life-threatening disease or condition is a stage of disease in which there is reasonable likelihood that death will occur within months or in which premature death is likely without early treatment.
A patient who is eligible to enter a clinical trial is not eligible for FDA’s expanded access program. Only patients who do not meet the clinical trial entry criteria, or are otherwise unable to participate in an ongoing trial for the disease or condition in question, can be considered for expanded access.
If possible, choose a clinical trial
Clinical trials are preferable because they usually provide more intensive oversight and monitoring of the patient. This may add a level of safety to receiving an investigational drug, biologic, or medical device. And, importantly, participants in clinical trials help move our knowledge about a medical product and disease or condition forward, contributing to the data necessary to determine whether a proposed treatment really works, how well, and the risk of side effects that may result from the treatment. The data gathered from clinical trials are used by FDA to determine whether to approve new therapies. If approved, these new therapies become available to all patients who might benefit from them.
It is important to talk to your physician about all the options that are available to you, including other approved medical products and clinical trials. If you are interested in a particular drug, biologic, or medical device, remember that other companies might be studying similar products for the same illness or condition. Keep in mind that investigational products might be the riskiest option with no known benefit.
Emergency Treatment Use
There are situations that require immediate action and access to an investigational product, for example, time-sensitive treatments that may prevent death or permanent disability—such as might occur as a result of stroke or heart attack.
FDA has emergency procedures in place for situations where there is not sufficient time to prepare and submit the paperwork. In such situations, FDA can receive and agree to treatment use via telephone, email, or fax. Usually this can be accomplished within hours. The physician, however, must submit the required paperwork to FDA within 15 working days.
In the case of an emergency, FDA will waive the requirement for prospective IRB approval, although the IRB must be informed within five (5) working days of initiating the emergency treatment use.
Emergency contact information
- For emergency use during regular business hours: Contact the appropriate review division (See List).
- For emergency use after 4:30 p.m. ET weekdays and on weekends: Contact FDA’s Emergency Call Center at 866-300-4374.
From Clinical Research Pathways
- Intermediate-size Patient Population Expanded Access Use
- Responsibilities of a Sponsor-Investigator (Treating Physician)
- How to Request Treatment Use via FDA’s Expanded Access Process (Non-Emergency)
- Points for Patients to Consider, PDF version, printed brochure, printing instructions
- The Right-to-Try Pathway
- Streamlining IRB Procedures
- FDA Guidance: Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers (October 2017)
- FDA Guidance on IDE Policies and Procedures, (January 20, 1998)
- FDA website
- FDA Form 3926 *Right-click and select “save link” to save the file to your computer)
- For drug-related expanded access: FDA Division of Drug Information, 301-796-3400 or email@example.com
- For biologics-related expanded access: FDA Biologics Office of Communication, Outreach, and Development, 800-835-4709 or firstname.lastname@example.org
- For medical device-related expanded access: FDA Device Division of Industry and Consumer Education, 301-796-7100 or DICE@fda.hhs.gov