Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Sometimes this is referred to as “compassionate use”.

There are three categories of expanded access:

1. Expanded access for individual patients, including for emergency use

• May be either a new, single patient IND or a single patient protocol added to an existing IND
• IRB review and approval is required prior to treatment, except in emergency uses where it must then be reported to an IRB within five working days

2. Expanded access for intermediate-size patient groups

• May be either a new IND, or a protocol added to an existing IND, depending on population size
• IRB review and approval is required prior to treatment

3. Expanded access for widespread treatment use

• May be either a new IND or a protocol added to an existing IND, for large/widespread populations
• IRB review and approval is required prior to treatment
  
  

Expanded Access Process

The physician’s role in the FDA Expanded Access Program is to:

• Determine there are no available clinical trials for the patient.
• Confirm the patient’s current disease or condition is either serious or immediately life-threatening with no available comparable or satisfactory alternative available.
• Identify the appropriate expanded access request type.
  • For assistance, contact the FDA directly via the FDA Expanded Access Contact Information webpage.
• Confirm the manufacturer will provide the product.
  • Federal law (21st Century Cures Act) requires that a company developing investigational drugs (including biologics) shall make its policy, regarding evaluating and responding to requests for expanded access, public and readily available.
  • Use the Reagan-Udall Foundation’s Expanded Access Navigator or the particular company’s website for more information.
• Facilitate the process:
  • Obtain the investigational medical product from the manufacturer
  • Submit an application to the FDA for expanded access on the patient’s behalf
  • Submit an application to OSF Research Administration and receive OSF permission to proceed
    • Send copies of the FDA and IRB submission documents via email
      
      
  • Submit for review and approval from an Institutional Review Board (IRB) prior to treatment*
    • For assistance with Peoria IRB procedures for expanded access submissions, contact the Peoria IRB directly.
    • *Emergency uses are to be reported to, and receive concurrence from, an IRB within five working days
  • Obtain informed consent from the patient for the use of the investigational medical product.
    • OSF provides a template Informed Consent & HIPAA Authorization for Expanded Access .
• Manage the use of the product and the patient's medical care.
• Provide appropriate reporting to the FDA, OSF Research Administration, and the IRB of record.

Emergency Use

If a patient needs access to an investigational medical product on an emergency basis, physicians should contact the specific FDA Office (if known) or FDA Patient Affairs at 301-796- 8460 or patientaffairs@fda.hhs.gov . For emergency requests after hours and on weekends, physicians should contact FDA's Emergency Call Center at 866-300-4374.

Additional Resources

Detailed information on the process for requesting expanded access can be found on the FDA Expanded Access: How to Submit a Request webpage.

For more information, visit the FDA Expanded Access webpage.

For questions, contact the FDA directly via the FDA Expanded Access Contact Information webpage.