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- Form updated 6/15/2020

If a consent form is not available from the manufacturer, please utilize the Single Patient Expanded Access Consent Form - Click Here to Download the IRB Template for Single Patient Expanded Access Consent. -Template updated 4/2/2021

DOCUMENTS REQUIRED FOR APPROVAL: Please ensure the Expanded Access Application is accompanied by the following supporting documents: 

NOTE: PLEASE DO NOT SEND IDENTIFIABLE PHI TO THE IRB

  • Unsigned consent form
  • Regulatory documentation from FDA for the IND or IDE, OR notice that documentation is pending receipt from FDA and will be provided to the IRB upon receipt. 
  • Drugs/Biologics Only: Drug Investigator’s Brochure / Package Insert AND FDA form 3926 (If expedited IRB review is requested, box 10b must be checked on form 3926)
  • Devices Only: Device Brochure / Manual AND Documented independent assessment from an uninvolved physician. The name of the physician should be clearly printed. 

The following are also required with your submission when applicable:

  • Clinical Protocol (i.e., if available from and provided by manufacturer)
  • Manufacturer authorization for the treating physician to utilize the device / drug (when required)
  • If appropriate: Reference a published protocol or journal article to support proposed treatment. 
  • If you require a letter: A complete list of documents being submitted for review as they should appear in your determination letter (document name, version #, date)

Expanded Access submissions 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:

  1. The patient has a life-threatening or serious disease or condition that needs immediate treatment;
  2. No generally acceptable alternative treatment for the condition exists; and
  3. 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. This means that there is NOT an immediate need to use the drug/device, AND there is time to use existing procedures to obtain FDA approval for the use.

It is strongly recommended that submission to the FDA and IRB occur simultaneously to improve the efficiency of the submission process.

Click here to download submission guidance and steps for obtaining approval for single patient compassionate use.

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 the following to be provided in HSERA: 

  • 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)
  • HIPAA Authorization (if you are collecting data to study the safety and effectiveness of the HUD)

Training Requirements

  • Physicians may take the full CITI Human Subjects Protections course if they are otherwise engaged in human subjects research. 
  • Physicians may take the standalone Humanitarian Use Devices (HUDs) module if they are NOT otherwise engaged in human subjects' research. Within the CITI Course Enrollment Questions, check the box next to Humanitarian Use Devices (HUDs).

See the CITI Guidance page for more information. 

HUD protocols require full convened review. If a compassionate use or emergency use of an HUD is necessary, please follow the guidance above under the Single Patient Pathways.

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...