Recently, FDA Commissioner Dr. Scott Gottlieb announced the launch of several FDA initiatives including activities to implement the 21st Century Cures Act (the Act) enacted last December.

Innovation Initiative

The agency has launched a “Comprehensive Innovation Initiative” designed to modernize the regulatory process to ensure safe and effective products reach patients as quickly as possible. The Act gave FDA significant new authorities and resources to accomplish this mission. The agency has posted its implementation work plan. It can be found here. It lists almost 70 specific action items the FDA will undertake over the coming weeks and months.

Some specific items include:

  • Implementation of the Regenerative Medicine Advanced Therapy, or RMAT designation. This new process provides another pathway to access FDA’s existing expedited programs, and is available for certain cell therapies, therapeutic tissue engineering products, and certain combination products. In addition, the agency will be publishing new guidance related to the regulation of these technologies this fall.
  • Device regulation. The Center for Devices and Radiological Health (CDRH) has exempted more than 70 Class I device types from the requirement to submit a 510(k) submission. CDRH also proposed exempting another 1,000+ Class II device types from having to submit a 510(k) submission based on an initial determination that premarket review is not necessary. Finally, CDRH amended its current regulations to allow more devices to qualify for a humanitarian device exemption for small patient populations.
  • Patient-focused drug development. FDA has developed a plan for the development and issuance of patient-focused drug development guidance to facilitate a more systematic approach to gathering and using patient perspectives to inform FDA’s regulatory decision-making.

Orphan Drugs

The FDA has released a plan to address the backlog of orphan drug requests. There are currently 200 requests pending from manufacturers. Orphan drugs are those designed to treat less than 200,000 patients in the US.

Under the plan, the FDA will prioritize completing reviews of all orphan drug designation requests that are older than 120 days. After clearing those reviews, the agency pledged to respond to all new requests within 90 days. To meet these goals, Gottlieb has instructed the FDA to establish a special SWAT team and take other steps to address scientific and regulatory issues.

Generic Drugs

Improving the process by which generic drugs reach the market quickly has become an increasingly important issue for FDA. Dr. Gottlieb has noted that an important way to reduce prescription drug costs is to increase generic competition. Dr. Gottlieb has recently announced a Drug Competition Action Plan. As first steps, the FDA launched two new initiatives to encourage generic drug development and increase generic drug access. They are:

  • A revision to its priority review procedures to allow for the prioritized review of generic drug applications until there are three approved generics for a given drug product. The agency said it revised this policy based on data that indicates consumers see significant price reduction when there are at least three FDA-approved generics available. More details can be found here.
  • Publication of the “List of Off-Patent, Off-Exclusivity Drugs without an Approved Generic,” which the agency will continue to refine and periodically update. FDA is publishing this list of approved new drug application (NDA) drug products which are off patent and off exclusivity and for which the agency has not approved a generic drug application. The agency also intends to expedite the review of any generic drug application for a product on this list to ensure that they come to market as expeditiously as possible. More details can be found here.

The agency plans to take additional, related actions in the near future, and will continue to communicate with stakeholders as these new elements are implemented.

FDA is launching a series of initiatives to change the regulation of digital health technologies. More details can be found here.

Clients are urged to follow developments closely as FDA implements these programs and launches new initiatives in the coming weeks and months.