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Alerts and Updates

Study Sponsors Take Heed: FDA Plans Changes to Expanded Access Program

February 7, 2019

Study Sponsors Take Heed: FDA Plans Changes to Expanded Access Program

February 7, 2019

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With an eye towards further improvement and expansion of the EA program, FDA commissioned an independent assessment and published a report on its findings and recommendations in 2018. 

Companies developing investigational drugs, biologics and devices are likely to face new obligations under FDA-proposed changes to the Expanded Access (EA) program, communicated in a recently issued Expanded Access Program Report and FDA Commissioner Scott Gottlieb’s related remarks in an FDA Statement. This Alert reviews the changes being considered and the principles guiding the changes. 

What Is the Expanded Access Program?

Expanded Access is an FDA program that makes drugs, biologics and devices that are still being tested available to patients with serious or life-threatening conditions who would not otherwise meet the testing criteria. The program traces its origins to the 1970s and continues to undergo formalization and refinement.

Why the Changes?

With an eye towards further improvement and expansion of the EA program, FDA commissioned an independent assessment and published a report on its findings and recommendations in 2018. On November 8, 2018, FDA Commissioner Gottlieb published an FDA Statement as a companion piece to this report, highlighting recent and anticipated changes to the EA program.

The report reinforces FDA’s commitment to the EA program and points to the high percentage of FDA approvals—nearly 99% across all application types—as an indicator of the program’s strength. The agency considered stakeholder feedback in its analysis, noting that the challenges identified by stakeholders and the program’s inherently multi-stakeholder environment guided the study as well as the recommendations.

Guiding Principles

The high-level guiding principles articulated by FDA to steer specific future developments are:

  1. Ensuring continued timely and medically appropriate access to the investigational treatments through the program.
  2. Reducing administrative burdens on stakeholders.
  3. Making the program easier for stakeholders to understand and navigate.
  4. Integrating data from the EA program with medical product review activities to meet the goals of transparency and quality decision-making.

What Guidance Has FDA Provided?

Taking each of these high-level principles in turn, the agency has made specific recommendations for actions to occur in both the near- and long-term, as follows:

First Principle – Access

The first principle focuses on FDA’s goal of maintaining and growing participation in the EA program. The agency proposes to address this principle by more consistently incorporating the topic of EA into its interactions with clinical testing sponsors. The agency envisions these interactions spurring the development and publication by companies of product-level policies for granting EA requests; allowing for better planning for EA by manufacturers in their production of clinical supply; and providing prospective clarifications for companies about how data from EA might be used in the review of applications.

Second Principle – Reduced Administrative Burden

The second principle focuses on streamlining internal processes to decrease the administrative burden to stakeholders who participate in the program. One recommendation is to use product-specific packets for frequently requested products that provide step-by-step instructions as well as pre-filled forms that are easier for physicians to complete and submit.

Third Principle – Easier Integration

The third principle looks to improve the points of contact between stakeholders and FDA. Recommendations include clarifying the language used to describe the program to create consistent messaging; enhancing communication through a campaign of continuing medical education courses and other forms of stakeholder outreach; a redesign of web materials with tools such as step-by-step guides; and, perhaps most significantly for patients and providers, the creation of a central triage resource. This triage program would create one point of entry for FDA that could help to direct communication more efficiently to subject matter experts.

Fourth Principle – Data Integration

The final principle addresses a matter of great concern to manufacturers—the use of data from the EA program in FDA medical product review. The agency wants to address what it has termed as a “misconception” that data from the EA program might harm product development. One of the ways FDA may fix this issue is by releasing guidance for manufacturers on how to create awareness of their own EA programs without violating prohibitions on marketing unapproved products. In recent comments to BioCentury, Commissioner Gottlieb stated that he wanted “to provide every possible incentive for sponsors to offer expanded access.” Richard Pazdur, the director of FDA’s Oncology Center for Excellence who proposed these changes, told BioCentury that drug manufacturers would continue to have discretion to approve an EA request, but that they would now have to provide a reason for their decision not to grant a request. Commissioner Gottlieb echoed these sentiments by noting that drug companies have an “obligation” to consider an EA request.


The FDA plans to introduce these changes through public meetings and the launch of a pilot program for cancer drugs in 2019. To get ahead of the curve, we recommend that companies develop or modify EA policies to reflect FDA’s positions and recommendations.

For Further Information

If you have any questions about this Alert, please contact Frederick R. Ball, Sandra G. Stoneman, Ryan Wesley Brown, any of the attorneys in our Life Sciences and Biotechnology Industry Group or the attorney in the firm with whom you are regularly in contact.

Disclaimer: This Alert has been prepared and published for informational purposes only and is not offered, nor should be construed, as legal advice. For more information, please see the firm's full disclaimer.