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Regulatory Update, July 2015

Wednesday, July 1, 2015

FDA draft guidance on individual patient expanded access application form

In the February 10 Federal Register, FDA announced the availability of a draft guid­ance for industry titled Individual Patient Expanded Access Applications: Form FDA 3926. The draft guidance seeks public com­ment and describes draft Form FDA 3926 (Individual Patient Expanded Access—In­vestigational New Drug Application [IND)], which, when made final, FDA intends to make available for licensed physicians to use for expanded access requests for in­dividual patient INDs. Individual patient expanded access allows for the use of an in­vestigational drug outside of a clinical trial for an individual patient who has a serious or immediately life-threatening disease or condition and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition. When made final, draft Form 3926 should provide a streamlined alternative for sub­mitting an Investigational New Drug Ap­plication (IND; Form FDA 1571) for use in cases of individual patient expanded access.

On August 13, 2009, FDA published a fi­nal rule amending its IND regulations by removing certain sections of 21 CFR part 312 on treatment use of investigational drugs and adding new subpart I to part 312 on expanded access (part 312, subpart I).

Among other things, the final rule was intended to increase awareness and knowl­edge of expanded access programs and the procedures for obtaining investigational drugs for treatment use for patients who have serious or immediately life-threaten­ing diseases or conditions who lack thera­peutic alternatives. The final rule also was intended to facilitate the availability, when appropriate, of INDs for treatment use, while protecting patient safety and avoid­ing interference in the development of in­vestigational drugs for marketing under approved applications. The draft guidance addresses the submission of Form FDA 3926 for a new individual patient expanded access IND by a licensed physician.

FDA may permit expanded access to an IND for an individual patient when the ap­plicable criteria in 21 CFR 312.305(a), which pertains to all types of expanded access, and the criteria in 21 CFR 312.310(a), which pertains to individual patient expanded ac­cess, including in an emergency, are met. A physician may satisfy some of the submis­sion requirements by referring to informa­tion in an existing IND, ordinarily the one held by the manufacturer, if the physician obtains permission from that IND holder. With permission, the physician should then provide FDA a letter of authorization (LOA) from the existing IND holder that permits FDA to reference that IND.

Section 312.305(b) sets forth the submis­sion requirements for all expanded access uses. One of the requirements is that a “cover sheet” must be included “meeting the requirements of § 312.23(a).” FDA is concerned that its goal of facilitating ac­cess to drugs for individual patient treat­ment use may have been complicated by difficulties experienced by physicians in submitting current Form FDA 1571, which is not tailored to requests for individual patient expanded access.

In an effort to streamline the submis­sion process for individual patient ex­panded access INDs, FDA intends to make Form FDA 3926 available (when final) for licensed physicians to request expanded access to an IND outside of a clinical trial for an individual patient who has a serious or immediately life-threatening disease or condition and for whom there is no com­parable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.

Draft Form FDA 3926 is shorter than Form FDA 1571 and requests the following information: (1) Patient’s initials and date of submission; (2) clinical information; (3) treatment information; (4) LOA from the investigational drug’s manufacturer, if ap­plicable; (5) physician’s qualifications; (6) physician’s contact information and the physician’s IND number, which is not the same as the manufacturer’s IND number, (if the number is known); (7) request for authorization to use Form FDA 3926 for individual patient expanded access ap­plications instead of Form FDA 1571; and (8) certification statement and physician’s signature. FDA provided draft Form FDA 3926 in the draft guidance for public com­ment (Appendix 1).

FDA intends to accept submission of Form FDA 3926 and to the extent that in­formation required under part 312 is not contained in Form FDA 3926, FDA intends to consider that form with the box in item 7 checked and the form signed by the phy­sician, to constitute a request to waive any other standard application requirements, including additional information included in Form FDA 1571 and Form FDA 1572.

FDA requested that comments on this draft be submitted by April 13, 2015. However, interested persons can com­ment on draft guidance at any time, and FDA will accept those comments, al­though it may not consider them until a future date when this document is being revised. Submit electronic comments to www.regulations.gov or written comments to the Division of Dockets Management (HFA-305), FDA, 5630 Fishers Lane, Room 1061, Rockville, MD 20852. It is only neces­sary to send one set of written comments. Identify comments with Docket No. FDA-2015-D-0268.


The Regulatory Update is excerpted from Research Practitioner, Volume 16, Number 3, May-June 2015.

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