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FDA Updating Expanded Access to Investigational Drugs, Devices

Scott Gottlieb

Scott Gottlieb, FDA Commissioner, in November 2017 (A. Kotok)

9 Nov. 2018. The Food and Drug Administration says it’s streamlining policies for making drugs and medical devices in clinical trials or under review available for people in the U.S. with life-threatening conditions. The changes to FDA’s Expanded Access program were announced yesterday in a statement by FDA Commissioner Scott Gottlieb.

FDA’s Expanded Access — also known as compassionate use — program makes available drugs, biologics, or medical devices being tested in clinical studies or under review by the agency for people with immediately life-threatening or serious medical conditions. In addition, to use Expanded Access, a patient’s needs cannot be met with comparable alternatives to diagnose or treat the disorder. Moreover, enrollment in a clinical trial of that therapy is not possible, and benefits from the proposed treatment outweigh the risks. And all stakeholders in the process, including developers of the proposed treatment, must concur.

Gottlieb says FDA received more than 9,000 requests for early access to treatments under review through the program, and authorized virtually all (99%) of them. Nonetheless, the agency conducted an independent evaluation of Expanded Access that highlighted confusion encountered in understanding the requirements, such as administrative burdens, and navigating the program’s web pages. Gottlieb says in addition to updating the web pages, the agency’s Patient Affairs Staff that reports to the Commissioner is now the first point of contact for Expanded Access for patients and physicians.

The Commissioner says FDA made other changes in the program, separately from the formal evaluation, as a result of feedback from individuals and Congress. The agency, says Gottlieb, reduced administrative requirements to request use of a drug or device being tested or under review that now take about 45 minutes for physicians to complete. FDA also simplified Institutional Review Board processes, where only the chair of the board — the group looking out for patient welfare in clinical trials — or a designee can approve a patient’s request, rather than the entire board. And the agency clarified reporting of safety data under Early Access to meet concerns of developers that the reports may affect subsequent FDA review.

In the statement, Gottlieb also said FDA is implementing its Right to Try program, a parallel pathway created by legislation signed in May 2018. Under Right to Try, patients with life-threatening conditions can request access to drugs or biologics not yet approved by FDA, but have at least completed early-stage clinical trials that usually test for safety. Gottlieb says FDA established a working group to consider steps needed to implement the law, but Expanded Access is still the main avenue for patients to gain access to unapproved therapies.

Gottlieb notes that these programs have more than a professional interest to him. “As a cancer survivor,” he says, “I understand, on a very personal level, that patients who are fighting serious or life-threatening diseases want the flexibility to try new therapeutic approaches, including investigational medical products. This is especially relevant when there’s no other FDA-approved treatment option available to a patient.”

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