FDA Notes Progress Implementing 21st Century Cures Act


FDA plans to further expedite novel treatments under the 21st Century Cures Act.

FDA Commissioner Scott Gottlieb, MD, recently testified before the House Committee on Energy and Commerce on the steps that have been taken to implement the 21st Century Cures Act, according to a press release.

The 21st Century Cures Act was signed into law in December 2016 by former President Barack Obama. The law provides $6.3 billion in funding for multiple initiatives that are expected to lead to novel breakthroughs in cancer, substance misuse disorders, and mental health conditions, among other disease states. When it was signed, former Vice President Joseph Biden said the bill would significantly improve cancer research.

Dr Gottlieb told the committee that the legislation provides the FDA with new resources to change policies and organizational structure to ensure that their efforts are in-line with the innovative and breakthrough medical products being developed, according to the release.

He also said that the FDA is highly committed to implementing the changes under the law that will bring cures and new treatments to many Americans.

The act allows the FDA to realize 2 main opportunities, Dr Gottlieb said.

According to the testimony, the law brings about significant opportunities for new science and technology, including gene therapy, targeted drugs, regenerative medicine, digital health platforms, and new biomaterials, all of which offer the hope of better therapies for countless conditions.

The opportunities provided by Congress are also significant, he said.

Additionally, through the reauthorization of user fees and the Cures Act, the FDA is able to create a new platform to translate scientific research into treatments that will improve patient lives, according to the release.

The Cures Act asks the FDA to think differently to achieve potential breakthroughs, while providing a more efficient and modern approval process, according to the release.

In addition to what Congress requested of the agency, the FDA plans to release a document that details how they are expanding on the Cures provisions, Dr Gottlieb said.

These new efforts include ways to facilitate the development and review of targeted medicines, especially in oncology. The FDA plans to further expedite the review of these drugs by bolstering the agency’s existing programs, such as accelerated approval, according to the release.

Rather than wait for a significant number of clinical trials for a potentially groundbreaking targeted therapy that has outsized benefits, the FDA may consider granting approval earlier and requiring post-market studies to confirm the earlier findings, according to his testimony.

Moving forward, Dr Gottlieb also wants to better define intermediate endpoints under accelerated approval to ensure that drug developers are able to take advantage of the provision and can consider it in a wider scope.

The FDA is considering a similar proposal for oncology drugs seeking a supplemental approval to use a more targeted data set when appropriate. This approach may be suitable when there is an outsized treatment effect and the second indication is for the same disease but in a different setting, according to the release.

“Cures refashioned and modernized FDA’s footprint for enabling new technologies to reach patients more efficiently. It’s given the agency new authorities and resources to invest in our workforce—it shapes the spirit of our mission,” Dr Gottlieb concluded. “We’ll continue to build on its framework.”

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