Expanded Access Submissions

Use the following format for a proposed Expanded Access, Compassionate or Emergency Use submission of an FDA-regulated drug or device and email it as instructed below. Guidance documents and templates are available in CATS IRB, including:

• HRP-023 – SOP All Emergency Use, Expanded Access for an Individual Patient (Drug Only), and Compassionate Use (Device Only) Review
• HRP-027 –SOP -All Emergency Use, Expanded Access for an Individual Patient (Drug Only) and Compassionate Use (Device Only) Post Review
• HRP-322 - Worksheet – Emergency Use
• HRP-325 -- Worksheet – Compassionate Use of an Unapproved Medical Device
• HRP-582 – HRPP Consent Form for Emergency Use/Individual Patient Expanded Access/Compassionate Use
• HRP-593 – Protocol for Humanitarian Use Device

Expanded Access/Compassionate Use/Humanitarian Use Device: 

Non-emergency individual patient expanded access/compassionate use of an investigational drug or device when a patient has a life-threatening or serious disease or condition. 

• Individual Patient Expanded Access Applications: Form FDA 3926 Guidance for Industry; https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm432717.pdf
• Asked Questions About Medical Devices: http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM127067.pdf 
• Humanitarian Device Exemption (HDE) Program:  Guidance for Industry and Food and Drug Administration Staff:  https://www.fda.gov/media/74307/download 

Complete a submission in CATS-IRB (irb.psu.edu), See “Expanded Access/Compassionate Use/HUD instructions”.  
 

Emergency Use (Rare situation) 

To use a drug, biologic, or device under the emergency use category it must be a true emergency, e.g., a life threatening or severely debilitating situation. FDA (Food and Drug Administration) (Food and Drug Administration) guidance where these terms are clearly defined:  https://www.fda.gov/regulatory-information/search-fda-guidance-documents/emergency-use-investigational-drug-or-biologic.

1. Confirm in the email that the circumstances meet the following criteria for emergency use:
   • The patient is confronted by a disease or condition that is life-threatening or severely debilitating, or the patient is confronted by a life-threatening disease or a serious condition requiring immediate use of an unapproved device.
   • The situation necessitates the use of the test article (drug, biologic, device).
   • No generally acceptable alternative for treating the patient is available.
   • There is insufficient time to obtain IRB approval. (Or if there is time for review at the next IRB meeting, indicate this and the information will be added to the agenda.)
2. Attach the following materials to the email as supporting documentation:
   • Document or email that describes the patient’s medical condition and the reason for the use of the test article. (Do not include any identifiers in documents.)
   • Correspondence to and from the FDA with the investigational new drug (IND) number or investigational device exemption (IDE) number, if available. (For a single-patient IND or compassionate IDE, submit the number when it is available).
   • Correspondence to and from the sponsor regarding use of the test article.
   • Protocol or detailed treatment plan for this patient.
   • Investigator’s brochure or drug package insert, or device information.
   • Emergency use consent form to be used. (Use “HRP-582 – HSPO Consent Form” in CATS IRB.)
3. Send the email and all subsequent communications about the emergency use to irb-orp@psu.edu or to staff member contacted; also copy other persons involved in coordinating the use.
4. Keep the HRPP informed about the status of a proposed use to proceed or if circumstances change.
5. By close of business within five days after the use of the device or first administration of the drug/biologic, submit to the IRB written notification of the emergency use of a test article in a life-threatening situation, and include a copy of the signed consent document used (or provide certification by the treating physician and an uninvolved physician that the criteria for the exception from informed consent were met).