• FDA Takes Steps to Streamline Individual Patient Expanded Access
  • August 3, 2016 | Authors: Edgar Asebey; Maureen Bennett; Christian B. Fulda; Colleen M. Heisey; Cristiana Spontoni
  • Law Firms: Jones Day - Miami Office ; Jones Day - Boston Office ; Jones Day - Munich Office ; Jones Day - Washington Office ; Jones Day - Brussels Office
  • On June 2, 2016, FDA announced the availability of three final guidance documents aimed at simplifying and clarifying expanded access for investigational drugs and assisting health care professionals, patients, and industry in navigating the expanded access process. In the FDA blog, FDA Voice, Dr. Richard A. Moscicki, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, heralded the documents, writing that "FDA has recently made significant changes to streamline and simplify the process for single patient expanded access requests." The term "expanded access" refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient's disease or condition (as compared to investigational purposes).

    Under current regulations, there are three categories of expanded access—for individual patients, including for emergency use; for intermediate-size patient populations; and for widespread treatment use through an investigational new drug or treatment protocol.

    The three final guidance documents address:
    • "Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers" (FDA-2013-D-0446). The guidance provides industry, researchers, physicians, institutional review boards, and patients with information about the implementation of FDA's regulations on expanded access to investigational drugs for treatment use under an investigational new drug application ("IND"). The document is in question-and-answer format and explains what expanded access is, when and how to request expanded access, and the type of information that should be included in requests, among other frequently asked questions. The guidance finalizes a May 2013 draft.
    • "Charging for Investigational Drugs Under an IND—Questions and Answers" (FDA-2013-D-0447). The guidance informs the same audiences about FDA's implementation of the rules regarding charging for investigational drugs under an IND for the purpose of either clinical trials or expanded access for treatment use. The document is in question-and-answer format and addresses FAQs, including general questions and questions about charging in clinical trials, charging for expanded use, and cost recovery calculations. The guidance finalizes a May 2013 draft.
    • "Individual Patient Expanded Access Applications: Form FDA 3926" (FDA-2015-D-0258). The guidance describes the availability of Form FDA 3926, which may be used by licensed physicians to request expanded access for individual patients. Individual patient expanded access allows for the use of an investigational new drug outside of a clinical investigation, or the use of an approved drug where availability is limited by a risk evaluation and mitigation strategy, for an individual patient who has a serious or immediately life-threatening disease or condition when there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition. The form may be used in lieu of an IND for individual patient expanded access, including for emergency use. The guidance finalizes a February 2015 draft.
    The finalization of these documents is only part of recent FDA efforts on expanded access. In May 2016, FDA held a public workshop with the Reagan-Udall Foundation where interested stakeholders explored viable options to help patients and their physicians understand the request process for access to unapproved drugs. FDA held an educational webinar on the documents on July 12, 2016, which was open to the public but directed toward physicians seeking more information about expanded access to an investigational drug.