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

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.

These efforts also come in the wake of the federal and state Right to Try legislation, which have the potential to drive patients, doctors, and drug sponsors away from FDA’s established program. Whether these steps will be sufficient for the EAP to remain an attractive pathway for access to investigational products is yet to be seen. FDA previously attempted to improve the EAP process, but, as found in FDA’s assessment, physicians still reported significant administrative burden. According to the assessment, even with the streamlined form, an EAP application can average nearly 30 hours of non-reimbursable time. As access under the Right to Try laws requires little administrative effort, this may be an appealing option for healthcare providers, patients, and industry.

Administrative burden, however, is not the only factor that stakeholders should consider when contemplating treatment access to investigational drugs. For example:

  • Although Right to Try access may be less burdensome, EAPs may provide healthcare providers and industry with a level of comfort, as EAPs are approved by FDA.  Additionally, as reported by BioCentury, in 2019, FDA plans to take steps to streamline the single-patient application process for physicians and patients (more on this in a coming post).  
  • Moreover, while the Right to Try laws include liability protections that are not included in the EAP, these liability protections are not absolute and will vary from state to state
  • While on paper, the federal Right to Try law generally prohibits FDA from using clinical outcomes to delay or adversely impact product review or approval, this may not substantially differ from how FDA uses EAP information. In guidance, FDA previously stated that it views EAP data in context. While there have been some limited instances in which EAP data informed product labeling, according to FDA, EAP data has not prevented the approval of a drug candidate
  • Finally, there is still significant uncertainty concerning the federal Right to Try law, which passed less than a year ago, and, despite the establishment of an FDA Right to Try website, little public guidance is currently available.

As the EAP and Right to Try continue to evolve, stakeholders should continue to track this developing area. By example, as discussed by FDA Commissioner Scott Gottlieb and Richard Pazdur, the director of FDA’s Oncology Center of Excellence, in a BioCentury article, which we will analyze in a coming post, FDA aims to become significantly more involved in requests for expanded access in 2019 (including contacting drug company sponsors concerning providing investigational drug product for expanded access use).  In the meantime, when considering treatment use of investigational drugs, sponsors, healthcare providers, and patients should consider the pros and cons of both programs to determine which may best suit everyone’s needs.