When a patient has a serious or life-threatening condition that is not addressed by current approved treatments, options may exist to use an investigational drug or device to treat. The use of an investigational product outside of a clinical trial for treatment of a patient is called “expanded access.” Expanded access for use of an investigational drug or device in an individual patient is divided into 2 pathways; "Emergency use" and "Compassionate use". Please see detailed guidance below for submission and approval of each of these pathways.
All requests for Single Patient Expanded Access require an Expanded Access Application - Click Here to Download the Application
For Single Patient Expanded Access requiring a Consent Form - Click Here to Download the IRB Template for Single Patient Expanded Access Consent
Expanded Access applications may be submitted via HSERA or Via E-mail. Please contact Jessica Yoos and Stephanie Lesage
Single Patient Emergency Use Pathway
Emergency Use Pathway Criteria:
- The patient has a life-threatening or serious disease or condition that needs immediate treatment;
- No generally acceptable alternative treatment for the condition exists; and
- Because of the immediate need to use the drug/device, there is no time to use existing procedures to obtain FDA approval for the use.
*"Life-threatening" is defined as:
- The likelihood of death is high unless the course of the disease is interrupted;
- A disease or condition with a potentially fatal outcome, where the end-point is survival.
- The disease or condition causes major irreversible morbidity.
If the above 3 criteria are not met, refer to the Compassionate Use Pathway Criteria
If the above 3 criteria ARE met - please click here to download the submission guidance and steps for obtaining approval for the single patient emergency use.
Single Patient Compassionate Use Pathway
Compassionate Use Pathway Criteria
1.The patient has a life-threatening or serious disease or condition; and
2.No generally acceptable alternative treatment for the condition exists.
This guidance should be used by any physician who wishes to use an investigational drug, device or biologic in a non-emergency situation with a single patient.
It is strongly recommended that submission to the FDA and IRB occur simultaneously to improve the efficiency of the submission process.
Expanded Access for More Than One Patient
If an investigator anticipates more than one patient may benefit from the expanded access of a drug or device, an Intermediate-size patient population expanded access protocol, or a treatment protocol should be submitted through HSERA. These require convened review. Please see below for additional guidance regarding these types of requests.
Drugs Only: Intermediate-Size Patient Populations:
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 protocol.
Treatment Protocol for Drugs or Devices:
Access to an investigational drug or device for treatment use by a large (widespread) population, submitted as a protocol. For more information see: www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/....
Humanitarian Use Device
A Humanitarian Use Device (HUD) is defined as a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year (Orphan Diseases).
IRB Submissions for Humanitarian Use Require:
-A completed HUD facesheet (click here to download form)
-HUD Protocol Summary (click here to download template)
-Cover letter with additional information that may help in the review and document list identifying each attachment (name, version, date)
-The HUD manufacturer’s product labeling, clinical brochure, and/or other pertinent manufacturer informational materials
-The FDA HDE approval letter.
-Humanitarian Use Device Consent Form (click here to download template)
Additional Guidance References
1.FDA Regulations: §812.36, §812.47, §50.24
2.FDA’s Expanded Access Program: www.fda.gov/newsevents/publichealthfocus/expandedaccesscompassionateuse/...
3.FDA Information Sheets for IRBs and Investigators www.fda.gov/oc/ohrt/irbs/default.htm
4.Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guid...
5.IND Applications: https://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelope...
6.Additional FDA guidance for HUD Applications: https://www.fda.gov/forindustry/developingproductsforrarediseasesconditi...