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.

FDA recently signaled that it plans to be more involved in facilitating expanded access to investigational new drugs. This follows the agency’s announcement of its efforts to improve and clarify the expanded access program (EAP), as well as state and federal legislation intended to simplify the process to use investigational drugs for treatment purposes.

One item that stakeholders may have missed, given the almost daily FDA developments, was the agency’s announcement that it will continue to improve and clarify its expanded access program (EAP). Specifically, FDA

  • updated its EAP webpage to streamline content and make the page more user friendly;
  • established an agency-wide Patient Affairs Staff and Health Care Provider Affairs Program to increase FDA engagement with stakeholder groups;
  • established an agency-wide Expanded Access Coordinating Committee to facilitate cross-center communications and discussion of cross-cutting issues; and
  • established a work group for the implementation of the Federal Right to Try law.

These follow a May 2018 independent assessment commissioned by the agency, which found that while stakeholders reported positive overall perceptions of the EAP and FDA’s role, there continue to be “pain points.” For instance, there continues to be confusion regarding program navigation, difficulties with multi-stakeholder coordination, and administrative burden.

The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (Federal Right to Try Act), signed into law on May 30, creates a federal framework for patients to access investigational new drugs without enrolling in clinical trials and without FDA expanded access approval. The law, however, leaves a number of unanswered questions that industry, healthcare providers, and patients must currently navigate without the benefit of regulatory guidance. For example, exactly how the Federal Right to Try Act interacts with state right to try legislation and FDA’s existing expanded access program is currently unclear. Implementation of the Federal Right to Try Act will be a developing area that stakeholders should continue to monitor. Moreover, stakeholders considering providing investigational drug access outside of clinical trials will have a number of areas to think about when determining which pathway to use.

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