Single Patient Expanded Access

There are two types of single patient expanded access:

• Non-emergency use: When there is enough time to obtain FDA authorization and IRB review before treatment
• Emergency use: When immediate treatment is needed and there is not enough time to complete the standard submission process

In both cases, the FDA must determine that the investigational product is needed for a serious or life-threatening condition and that no satisfactory alternatives are available.

Although emergency use may occur before full approvals are in place, both emergency and non-emergency uses follow the same submission requirements for NIH IRB review.

Single patient expanded access is considered a clinical investigation under FDA regulations, even when only one patient is treated.

In most cases:

• The NIH IRB has time to review the request before treatment
• The IRB Chair may provide concurrence instead of requiring full board review (when FDA authorization allows this pathway)

To use this pathway, the sponsor-investigator must request “Authorization to Use Alternative IRB Review Procedures” from the FDA. You can do this by filling out section 10.b. on FDA form 3926, or if you’re using FDA form 1571, you can include a separate attachment with your request.

Please note, this pathway is only available when you, the physician sponsor-investigator, will hold the IND. It is not available if your Institute or a pharmaceutical sponsor will hold the IND.

To obtain IRB Chair's concurrence, submit a new study in PROTECT. Attach the following documents to your submission:

• A completed "single patient use expanded access form," which you can find in the IRB Library in PROTECT under the “templates” tab.
• The documentation you submitted to the FDA, such as FDA form 3926.
• The FDA communication that includes the assigned IND/IDE number and approval to proceed with the treatment.
• The investigator brochure (if available)
• Any other supplemental documents.
• A patient-specific consent document

Note that you do not need to obtain any ancillary review approvals for single patient expanded access studies.

These requirements apply to both emergency and non-emergency cases.

A consent document must be developed specifically for the individual patient and treatment.

• Use the NIH expanded access consent template below as a starting point
• Tailor it to the specific patient, treatment plan, pregnancy prevention and follow-up
• Refer to the use of the product as treatment, not research, when appropriate