Now that you are the sponsor of a Single Patient IND, do you understand the expectations and requirements for treating a patient under expanded access?
After the expanded access application is allowed to proceed, a letter is issued. This letter will provide the details of your obligations for safety reporting and annual reports. Follow-up submissions use the same Form FDA 3926 as the initial submission; however, you will only need to complete certain parts of the form. You can think of the form as a cover page.
Complete fields 1 through 3 every time Form FDA 3926 is submitted to FDA. Check the box in field 3.b. to indicate it is a follow-up submission. Include the investigational drug or biologic name and the IND number provided by FDA.
Remember, fields 4 through 7 are only for initial submissions and are not needed for follow-up submissions. For follow-up submissions to an existing expanded access IND, we will focus on fields 8 and 9.
In field 8, enter your contact information. This field should be completed each time Form FDA 3926 is submitted.
In field 9, check one or more of the following boxes if they apply. There are eight options.
Check box 1, Initial Written IND Safety Report, if you are submitting initial safety information. You are required to report certain suspected adverse reactions within a certain number of calendar days of learning of the reaction. Within 7 days, report unexpected fatal or life-threatening suspected adverse reactions. Within 15 days, report serious and unexpected suspected adverse reactions. The regulations for mandatory safety reporting are found in Title 21 of the Code of Federal Regulations, 312.32. FDA’s regulations are available online at ecfr.gov. Review FDA’s Guidance for Industry and Investigators Safety Reporting Requirements for INDs and BA/BE Studies for more information.
Check box 2, Follow-up to a Written IND Safety Report, if you are submitting follow-up information to an initial written safety report. This usually includes more details about the adverse event. Follow-up information must be submitted to FDA within 15 calendar days of receiving the information.
Check box 3, Annual Report, if you are submitting a brief annual report for a treatment provided for longer than one year. This brief report is a summary of the treatment experience throughout the year and is due within 60 days of the anniversary of the date that the IND went into effect. The annual report is not required if treatment was completed and FDA was notified before one year passed.
Check box 4, Summary of Expanded Access Use, if the treatment is completed and you are submitting a summary of the treatment experience, including adverse effects. Include this summary when requesting withdrawal of the IND.
Check box 5, Change in Treatment Plan, if you are revising the protocol or providing additional information. This includes any changes to the initial submission, such as protocol or information amendments and changes of investigators.
Check box 6, General Correspondence, if your submission covers any other communication between you and FDA. Typically, this includes administrative changes, such as withdrawing the expanded access IND after treatment is complete.
Check box 7, Response to FDA Request for information, if you are providing information we requested.
And, finally, check box 8, Response to Clinical Hold, if you are providing a response to a Clinical Hold letter. Your response is expected to address the concerns that FDA identified in the Clinical Hold letter you received. Include sufficient detail for FDA to review your request. If your response satisfies FDA’s concerns, FDA will authorize the expanded access request.
For a more detailed overview of expanded access, a Physician’s Guide to Non-Emergency Single Patient Expanded Access Submissions is available on FDA’s website. It is divided into two sections. The top section covers initiating expanded access, and the bottom section covers follow-up submissions.
Still have questions? Contact FDA’s Division of Drug Information. If you’re already working with the Review Division, give them a call.