FDA Commissioner Scott Gottlieb and NIH Director Francis Collins testified on Thursday before the House Energy & Commerce Committee on how their agencies have worked to implement provisions from the 21st Century Cures Act passed and signed by President Barack Obama a month before he left office.
The hearing centered on the mammoth new law that includes major tweaks to how FDA regulates drugs, devices and biologics, and provides the agency with $500 million over 10 years to implement it.
In opening comments, Gottlieb told the committee that the agency is working on a proposal whereby cancer treatments already approved for one indication might be able to win approval for a second indication based on a supplemental application that relies on “a more targeted data set, like a single-arm study. We intend to issue guidance further clarifying the circumstances in which this is appropriate.
“This may be suitable, for example, when there’s a clear and outsized treatment effect, and the second indication concerns the same disease as the first one, but for a new setting. For example, a targeted drug approved for a third-line use that shows benefit in a second-line indication,” Gottlieb added.
In the question and answer portion of the hearing, Gottlieb noted another new guidance to be released “very soon” on targeted therapies, outlining how to win approval for drugs targeted at biomarkers, “such as a cancer that might appear in multiple organs.”
He also noted that since the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA), the agency has been able to grant accelerated approval based on intermediate clinical endpoints.
“We want to better define what’s meant by intermediate endpoints to ensure that product developers with promising drugs take full advantage of this provision and can consider it in a broader range of such settings,” he said.
In praise of FDA’s Oncology Center of Excellence, Gottlieb added that it was instrumental in the recent approvals of two new CAR-T cell therapies, which he said could be transformative: Novartis’ Kymriah (tisagenlecleucel) and Kite Pharma’s Yescarta (axicabtagene ciloleucel).
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 email@example.com for more information.
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