Gottlieb Outlines Efforts the FDA Is Making to Improve Its Expanded Access Program

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Scott Gottlieb, commissioner of the U.S, Food and Drug Administration (FDA), provided an update on the agency’s Expanded Access program. 

Sometimes called “compassionate use,” the program offers a possible route for a patient with an “immediately life-threatening condition or serious disease or condition” to get access to an investigational medical product, in other words, drug, biologic, or medical device. 

In late May, Congress passed the “Right to Try Act,” which amended the Federal Food, Drug, and Cosmetic Act. The intent of the law was to provide terminally ill patients more access to drugs the FDA hasn’t approved yet. The patients can request the drugs from the drugmakers if they have been part of a small-scale Phase I clinical trial and are still being considered by the FDA.

Shortly after the law was passed, its author, Sen. Ron Johnson (R-Wis.) pushed back on some comments Gottlieb made saying the agency might need to write guidance or regulation to achieve the goals of the law. Johnson wrote a letter to Gottlieb that said, in part, “Given your comments, and as the law’s primary author, I would like to make this legislation’s intent absolutely clear. It is not meant to grant FDA more power or enable the FDA to write new guidance, rules, or regulations that would limit the ability of an individual facing a life-threatening disease from accessing treatments.”

Yesterday’s statement by Gottlieb is an update of what the FDA has been doing, regardless of Johnson’s concerns. “Despite the success of the EA program,” he wrote, “we recognize that there are opportunities for improvement.”

Gottlieb cited the agency streamlining the required supporting documentation for expanded access requests, which cut doctor administrative burden. It also simplified the process for Institutional Review Board (IRB) review. The agency also wrote guidance on how it uses safety data created from the use of drugs administered through the EA pathway. This, he wrote, was in recognition of “companies’ concerns that EA-related adverse event data could be used in ways that complicate the review process.”

Gottlieb also noted the agency had commissioned an independent assessment of the program in 2017. He described some of the findings of that review, including “overall support for FDA’s program, but as we hoped in pursuing this analysis, also identified new steps we can take to improve upon our efforts.”

First, the perception of the program is positive, as well as physicians, patients and patient advocates.

Secondly, manufacturers, patient advocates, IRB representatives and doctors said the FDA had “taken steps to reduce the administrative burden.” They also noted some challenges that needed to be addressed. Gottlieb says the agency has already begun taking steps to address the criticisms.

For example, the assessment described confusion over program navigation, multi-stakeholder coordination and, again, the administrative burden. The assessment recommended improving the agency’s public website and investing in patient/physician support resources. The FDA is working on those issues.

The FDA announced yesterday, for example, that its EA webpages would be updated to improve usability, streamline content and make everything more user-friendly.

The agency also intends to reorganize the Office of the Commissioner and establish an agency-wide Patient Affairs Staff and Health Care Provider Affairs Program under the oversight of the Office of Clinical Policy and Programs. It has also created an agency-wide Expanded Access Coordinating Committee.

“FDA is deeply committed to our Expanded Access program,” Gottlieb stated, “and facilitating access to medical products outside of clinical trials when no alternative therapy options are available to patients. And we are deeply committed to continuing to enhance this program going forward.”