FDA

FDA looks to strengthen expanded access program

The FDA on Thursday unveiled plans to further strengthen its expanded access program, as the controversial Right to Try legislation has yet to help any patients.

The expanded access program allows patients with serious or immediately life-threatening diseases who are unable to participate in clinical trials to gain access to promising investigational treatments.

Over the last five years, the FDA has authorized more than 9,000 applications across drugs, biologics and devices through the agency’s expanded access program, and the agency expects about 800 more individual requests next year. About 99% of all the requests the FDA has received, across all application types, have been granted, though those requests are granted after the company offering the treatment also signs off on the request.

Following an independent assessment of the expanded access program last year, the agency says that it has now agreed to update the FDA’s Expanded Access webpages to improve usability through the streamlining of content and a more user-friendly organization. “This includes a reduction in duplication, as well as the addition of new pages with commonly requested information, such as forms and keywords,” the FDA said.

And as part of the recently announced proposed reorganization of the Office of the Commissioner, the FDA also intends to formally establish an agency-wide Patient Affairs Staff and Health Care Provider Affairs Program, under the oversight of the Office of Clinical Policy and Programs.

“This will enhance our engagement with these important external stakeholder groups. The Patient Affairs Staff is already in place and charged with serving as the ‘home base’ and primary point of entry for patients and physicians starting the EA process and navigating them through the steps,” the FDA said.

Meanwhile, the FDA has established an agency-wide Expanded Access Coordinating Committee, which facilitates cross-center communication and promotes discussion to address cross-cutting issues related to expanded access to promote consistency and best practices.

Right to Try

Meanwhile, the FDA has released a new website to help patients understand Right to Try, and the agency has established a work group to consider what steps may be required to implement this legislation in a way that advances Congress’ intent to promote access and protect patients.

But such implementation efforts from the FDA have already proven controversial, as Sen. Ron Johnson (R-WI) made clear that the law “intends to diminish the FDA’s power over people’s lives.

Focus reached out to two companies advertising the use of Right to Try – StemVacs and Therapeutic Solutions International – though neither responded.

A recent article in Health Affairs on Right to Try noted, “In a way, patients may have benefited from RTT after all: not because it created a new pathway that cut the FDA out of the picture, but because it raised awareness that non-trial access was possible, thus galvanizing patients and their doctors to request it.”


First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email news@raps.org for more information.


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