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FDA Details How it Will Implement 21st Century Cures Act


In published documents and a detailed blog post from its Commissioner, the FDA made public its plans for using data modelling to improve regulatory trials.

In a blog post Friday, FDA Commissioner Scott Gottlieb, MD, doubled down on his consistent calls for a streamlined regulatory body that relies more heavily on new technology and takes a risk-based approach to drug and device approval. This time, he brought his most concrete details to the table by focusing on the ways in which the Food and Drug Administration would implement the 21st Century Cures Act.

Saying that the Act gave the FDA “many new authorities and resources” to work towards that risk-based mission, he emphasized a hope that the actions would eventually lead to benefits for consumers, in terms of cost, access, and safety of drugs and devices. Section 1002 of the Act allocates $500 million in new funding to implement new provisions of the law.

Corresponding with the post, the FDA made public the work plan that it submitted to Congress last month about how the agency would implement the law, and it published to its website a chart of Deliverables with statutory deadlines.

Gottlieb focused on a range of efforts that included increased reliance on data modelling for the development of more effective clinical trials, so-called in silico trials. The FDA’s Center for Drug Evaluation and Research (CDER) is moving to deepen its simulation efforts in light of a new era of personalized medicine and genomic understanding. The EU announced a concerted focus on simulation programs in 2013.

“Right now, FDA is collaborating with scientists to develop such natural history models in Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, and muscular dystrophy,” in a play to better model aspects of the placebo arm in clinical trials, Gottlieb reports. The work is partly aimed at lowering development and approval costs, which for pharmaceuticals are currently astronomical.

“We therefore need to be mindful of the costs of our regulatory processes, to the degree that these costs also affect the availability of new innovations, and the way that they are ultimately priced,” Gottlieb wrote. In order to keep up with its own efforts to model and simulate increasingly large data sets, he expressed a need for the FDA to “continue to invest in high performance computing.”

The FDA has also implemented a new Regenerative Medicine Advanced Therapy (RMAT) designation for cell therapies and tissue engineering products. The designation “must be made either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND.” So far, Gottlieb notes that the FDA has received over 20 requests for the designation and granted 4.

In accordance with the 21st Century Cures Act (and the new Commissioner’s philosophy), the FDA’s Center for Devices and Radiological Health (CDRH) has exempted hundreds of device types from having to submit a 510(k) submission, effectively limiting the necessity of premarket review. The CDRH has also amended regulations “allow more devices to qualify for a humanitarian device exemption for small patient populations.” That wing of the FDA is also working on its own in silico regulatory models for device evaluation.

Early in his blog post, Gottlieb again teased that the FDA would soon unveil “a comprehensive Innovation Initiative” aimed at ensuring modern and efficient regulatory processes. “We’re at the beginning of a transformative era in science and medical technology,” the Commissioner wrote, underscoring his FDA’s “enthusiasm and commitment to [the 21st Century Cures Act]-both its letter and its spirit.

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