When President Barack Obama signed the 21st Century Cures Act into law in December 2016, he said “We are bringing to reality the possibility of new breakthroughs to some of the greatest health-care challenges of our time.” One year later, where does the law stand?
Food and Drug Administration Commissioner Scott Gottlieb testified before the House Committee on Energy and Commerce nearly a year later to provide an update on the law’s implementation. In prepared remarks, Gottlieb said this task is “one of our highest priorities.”
He told the committee that if his testimony had taken place five years earlier, it would have been “unrealistic” for him to say cures for diseases such as sickle cell, hemophilia, and certain cancers were coming in five years, but that now “we should expect these opportunities”
He shared more details on their efforts with regard to cancer. In cases where a targeted drug has an “outsized effect on overall survival” among a small trial of patients with rare or deadly forms of cancer, they may be able to approve the product earlier to accelerate its use in a larger clinical setting. At that point, a post-market study could be used to confirm the product’s effectiveness.
He noted that congress had granted the agency authority to permit accelerated approvals based on “intermediate clinical endpoints,” but that he wants to better define the meaning of the term to “ensure that product developers with promising drugs take full advantage of this provision.”
A draft guidance on expedited programs for regenerative medicine therapies has been issued, and additional guidance will be coming soon, along with a document he promised would take “full measure” of how the agency is expanding on the provisions in the act.
Regardless of the specifics, as Gottlieb noted in his testimony, they will invariably come with some risks. In the event that those risks lead to a recall, companies developing new drugs and devices under the law’s provisions should ensure their plans account for this risk. It will take many more years for Cures to be fully implemented. But as it progresses, plans should be regularly updated and tested to keep up with the developments.
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Reporting Adverse Events: FDA Seeks Comments on Revisions to Submission Forms https://t.co/919JeroD4O by @RAPSorg https://t.co/L87wZe8ITD
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