On December 13, 2016, President Obama signed into law the 21st Century Cures Act (the “Cures Act”). We detailed the Cures Act’s provisions affecting hospitals in a prior Health Law Pulse post and will focus on a more in-depth analysis of its impact on the U.S. Food and Drug Administration (the “FDA”) here. The Cures Act authorizes $500 million over 10 years for the FDA to fund the development of new drugs and devices and enhance clinical trials. As more thoroughly discussed below, the Cures Act directs the FDA to (1) hire more qualified individuals, (2) modify certain drug/device approval pathways, and (3) alter certain clinical trial requirements.

FDA hiring efforts

The Cures Act authorizes the FDA to appoint qualified individuals to scientific, technical, and professional positions to bolster the FDA’s efforts to develop and regulate medical products. It further expands the number of research-related positions in the FDA and allows salary increases to attract skilled employees.

Modified drug/device approval pathways

The Cures Act strives to streamline the approval process for certain drugs/devices. Specifically, the Cures Act:

  • Creates an expedited approval pathway for breakthrough devices.
  • Enumerates five types of medical software that generally will not be regulated as medical devices by the FDA due to their low level of risk to patients.
  • Establishes a review pathway at the FDA for drug development tools such as biomarkers that may help decrease both drug development time and failure rates.
  • Requires the FDA to hold a public meeting and issue guidance to assist sponsors in incorporating complex adaptive and other novel designs into new drug applications.
  • For combination products, requires the FDA to meet with sponsors and agree how best to study the combination product to meet the standard for approval.

Clinical trial design requirements

To further its goals of improving clinical trial design, the Cures Act:

  • Requires the FDA to evaluate the feasibility of using “real world” evidence to support the approval of a new indication for a previously approved drug and to support or satisfy post-approval study requirements.
  • Allows the FDA to modify informed consent requirements for clinical trials with minimal risk.
  • Permits the FDA to rely upon qualified data summaries to support the approval of an application for a new indication of an already approved drug, provided there is sufficient existing data demonstrating the safety of the drug.
  • Requires the FDA to, where appropriate, standardize the requirements of the HHS Human Subject Regulations and the FDA Human Subject Regulations.
  • Enables the FDA to grant accelerated approval for regenerative advanced therapy drugs. Among other requirements, such drugs include “cell therapy, therapeutic tissue engineering products, human cell and tissue products, and [certain] combination products using any such therapies or products….”

It is unclear how the pending selection of President Trump’s nominee for FDA Commissioner, as well as his recently announced hiring freeze for federal employees, will impact the implementation of the Cures Act by the FDA. We will, therefore, provide updates to this analysis as new information becomes available.

The complete text of the Cures Act is available here.