Expanded Access Programs (EAPs), also known as managed access, early access, compassionate use, named patient, etc. allow patients to be treated with investigational new drugs (INDs), which have not yet been approved, or drugs that have been approved but have limited availability. These patients are usually seriously ill and have run out of all other viable treatment options, including participation in a clinical trial. The intent of expanded access is to give these individuals and their families hope when all other avenues fail.

FDA regulations have been in place for years to help govern this process and make sure that the right drugs are made available to the right patients. In June of 2016, however, the FDA released three new guidance documents pertaining to the rules and regulations currently governing expanded access. These documents do not change current regulations but, rather, attempt to streamline the existing processes.

This article will summarize the three documents.

Individual Patient Expanded Access Applications: Form FDA 3926

This document was created to aid physicians requesting the use of an IND for an individual patient. Such documentation was considered necessary by the FDA after numerous physicians complained that theexisting application process was arduous and difficult to complete. This is due to the fact that the existing forms were used for all types of IND requests, including those for large clinical trials.

To address these concerns, the new Form 3926 was created specifically for requests made for individual patients. This new form is now tailored exactly to that one expanded access submission process, and is expected to take physicians no longer than 45 minutes to complete.

The document also helps physicians by providing clarification of the existing requirements. The processes that must be followed when a physician is seeking access to an IND, or an approved drug with limited access, for a single patient, are as follows;

  • IND and LOA àA physician is required to contact the commercial sponsor or drug manufacturer of the IND and get a Letter of Authorization (LOA). Once the FDA has the LOA, it can refer to the information submitted by the sponsor/manufacturer and review the physician’s expanded access request.
  • IND and no LOA àIf a LOA cannot be obtained, the physician should contact the FDA at the relevant review division and find out what information will be required to follow through with the expanded access submission.
  • Approved drug with limited access à A physician should contact the relevant FDA review division if the approved drug is limited by a REMS.

Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers

This documentwas created to allow the FDA to address some frequently asked questions regarding the expanded access program as a whole. Some of the main questions addressed by this document include:

  • What role does the patient, the physician, the FDA, and the IND sponsor each individually play in the expanded access program?
  • What individuals/organizations have decision making power in the process and how much?
  • What is the difference between the new Form 3926 and the existing form?
  • What is IRB approval and why is it needed?
  • How does the timing of the initial treatment vary depending on the various types of approval?
  • Can patients apply for more than one early access IND? If so, what is the process?

Charging for Investigational Drugs under an IND – Questions and Answers

Just like the second document, this documentwas created to address frequently asked questions. Itrefers to questions around the FDA regulations governing the payment for investigational drugs. The questions are not limited to the topic of expanded access, and there are queries about clinical trial payments also included. The document was created to make the charging process for investigational drugs easier to follow for those considering expanded access in particular.

There is a focus on simplifying the revisions made to the regulations in 1987, which govern the conditions under which a sponsor can charge for an investigational drug. This document provides additional clarification on these regulations, specifically the criteria put in place for charging for a drug, the process which should be followed if seeking FDA approval for the charging of a drug, and the cost recovery calculations that are allowable under FDA regulations.