On June 21, 2016, Richard A. Moscicki, M.D., the FDA’s Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, wrote about gaining access to investigational drugs on the FDA Voice blog. The blog is stated below.
The FDA is only too aware that there are many patients who have a serious or life-threatening medical condition for which there is no available FDA-approved therapy. For such patients, one option may be to obtain access to an investigational drug that has not yet been approved by the FDA. To do this, a physician submits an application to the FDA requesting authorization to use the investigational drug in the treatment of their patient. This is called expanded access to investigational drugs.
While the FDA has been helping physicians navigate the system for many years, there have been physician and patient concerns about this process, which can be time consuming and difficult to understand. Consequently, the FDA has recently made significant changes to streamline and simplify the process for single patient expanded access requests.
To make the expanded access process more efficient, the agency just introduced a much simpler application form called the Form FDA 3926, which will be the form doctors now will typically fill out when they want to provide an investigational drug for a patient through expanded access. While the Form 1571 had 26 information fields and seven attachments, the new Form 3926 has fewer fields (11) and only one attachment. With this streamlined format, it is estimated that physicians will be able to complete the form in just 45 minutes, as compared to the more difficult and time consuming effort required previously.
Also, on May 16, 2016, the FDA and the Reagan-Udall Foundation held a meeting with interested stakeholders to explore additional options that might help patients and their physicians understand the process to request access to unapproved drugs. A common theme of the meeting was that navigating the expanded access process really does take a village. The physician, the drug company, the FDA, and the institutional review board (IRB) all have important roles and must work together for the expanded access process to succeed.
The FDA and Reagan-Udall Foundation convened this forum to listen to the public express their needs about expanded access and to discuss ideas with stakeholders on ways that the complex process can be made more efficient and effective. Much work on the details remains, but in general there was agreement on the need for a central repository or clearinghouse where useful and relevant information could be stored in one place — a sort of “one-stop-shop” for physicians and patients to seek information about the expanded access process. As the thinking about this resource develops, the public will be kept informed.
For physicians seeking more information about expanded access to an investigational drug, the FDA has developed an educational webinar to help them become familiar with the new application form. This live webinar will occur on July 12, 2016 at 1:00 PM EDT and will offer one hour of Continuing Education (CE) credit. The webinar will be recorded for viewing without CE credit. The agency also has released a guidance regarding Form FDA 3926, a guidance with Questions and Answers on expanded access, as well as a guidance directed at industry addressing questions regarding charging for investigational drugs.
Expanded access is designed for seriously ill patients who have exhausted other options. The last thing a patient suffering from a serious or life-threatening condition needs is red tape. For many years, the FDA has dedicated staff to assist physicians and patients in navigating the system. The FDA expects these important steps will help it continue its efforts to serve patients in need and to advance public health.
See the FDA Voice Blog Post
See also Medical Law Perspectives, May 2013 Report: Drugs, Dosage, and Damage: Physician Liability for Prescribing or Administering Medication