You must apply for an Institutional Review Board’s (IRB) approval for compassionate use prior to initiating treatment. You should specify in the application that the primary intent is treatment, not clinical research, because not all IRBs are familiar with expanded access protocols and how to review them.
In October 2017, FDA released a Information Sheet Guidance for sponsors, clinical investigators and IRBs on the Waiver of IRB requirements for Drug and Biological Product Studies. FDA concludes that such a waiver is appropriate for individual patient expanded access INDs when the physician obtains concurrence by the IRB chairperson or another designated IRB member before treatment use begins (no separate IRB approval process or notification to the IRB would be needed). To request a waiver, select an appropriate box on Form 3926.
If your institution does not have an IRB you can:
• Use independent IRBs. This is private IRBs that review research proposals at a cost, such as Western IRB, Quorum Review IRB, Advarra, and Sterling IRB.
• Partner with an IRB from your local hospital/institution. Universities that are affiliated with hospitals will most likely have an IRB.
• Start an IRB at your institution.
An excellent resource for advice on IRBs is Clinical Research Pathways.
Some drug companies may require an IRB approval letter before the drug will be shipped. If treatment cannot be delayed, some IRBs have sent manufacturers a written statement indicating that the IRB is aware of the proposed use and considers it to meet the requirements for emergency compassionate use. Although this is not a formal IRB approval, the acknowledgment letter has been acceptable to drug companies.
In an emergency life-threatening situation in which no standard acceptable treatment is available, and in which there is not sufficient time to obtain IRB approval, by law you may begin treatment and report the emergency use of the investigational drug to an IRB within 5 working days (21 CFR 56.104). Some IRBs have specific procedures for approving emergency requests (see example of emergency use guidance here). However, you should notify the IRB right away and follow your institution’s procedures for such cases.
Requirements of IRBs differ, but as a general rule you should email the following to the IRB:
• description of case and treatment plan, including justification for compassionate use;
• a proposed consent form;
• any available documentation from the FDA regarding the emergency IND.
Obtain informed consent
In general, the consent should satisfy federal requirements under 21 CFR 50. A waiver of an IRB review does not apply to an informed consent.
You can edit this sample informed consent form as needed.
Print all application steps
as one document (PDF)