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YOUR GO-TO SOURCE FOR ANALYSIS OF ISSUES AFFECTING THE PHARMA & BIOTECH SECTORS

Over the last few months, FDA has continued its efforts to encourage and facilitate the use of the agency’s Expanded Access Program (EAP). This follows other FDA EAP actions, including its announcement of program improvements. Overall, these steps appear to signal that FDA is trying to position the EAP as a desirable option for patients, healthcare providers, and industry following the passage of the Federal Right to Try statute, in which, as noted in FDA’s recent Right to Try Frequently Asked Questions (FAQs), the agency plays a very limited role.

Most recently, FDA held a workshop on Project Facilitate, a pilot program developed under the Oncology Center of Excellence that is intended to provide a “concierge-like” EAP service for providers and patients. Acting FDA Commissioner Ned Sharpless stated that Project Facilitate will assist oncology providers to locate Institutional Review Board (IRB) resources, find EAP contacts at companies, complete the necessary FDA forms, and receive advice on the information needed to complete EAP requests. Project Facilitate will also assist FDA in gathering information on whether drug manufacturers are providing access to investigational drugs through EAPs and, if not, understanding the reasons for denials.

Prior to the workshop, FDA had also issued a statement encouraging the use by sponsors of EAPs following clinical trial completion for both patients who do not qualify for trials and for participating patient follow-on treatment. The agency, however, also acknowledged that there may be impediments to EAP implementation, such as investigational product supply and the cost of manufacturing complex products (e.g., cell and gene therapies).

FDA’s recent encouragement of EAPs may change the way sponsors think about the EAP when deciding between EAP and Right to Try access. For example:

  • While the EAP requires that sponsors and/or healthcare providers comply with requirements that are not applicable to Right to Try (e.g., application submission, FDA reporting, and compliance with most investigational new drug regulations), the use of set plans/protocols, intermediate and large EAPs may alleviate the need for sponsors to address individual patient requests in an ad hoc manner.
  • FDA emphasized that events occurring during the course of an EAP are unlikely to present an impediment to product approval and can add evidence for approval. Specifically, FDA stated, “We want to reassure sponsors that providing a drug under [an EAP] very rarely impacts development timelines.” Only in one case did an EAP impact product labeling, resulting in the inclusion of a labeled drug interaction.
  • However, Right to Try appears to provide some advantages over the EAP in the area of liability protection. As we have previously written, Right to Try provides sponsors and manufacturers of investigational products that are supplied pursuant to the law, as well as prescribers, dispensers, and other entities, some protection from certain liabilities for alleged acts or omissions with respect to the investigational drug and from decisions not to provide access to an investigational drug. Analogous protections are not available under the EAP.

Overall, whether doctors, patients, and sponsors will prefer EAP or Right to try access will depend on many factors that are specific to the individuals, companies, studies, and investigational products. Nonetheless, FDA continues to try to persuade sponsors to use EAP rather than Right to Try.