August 31, 2017

Developments in Regenerative Medicine: FDA Announces Plans to Clarify Its Oversight of Cellular Therapy Products and Approves First Gene Therapy

Holland & Knight Healthcare Blog
Michael J. Werner

FDA Commissioner Scott Gottlieb, on Aug. 28, 2017, announced steps the agency is taking to begin rolling out policies and guidance documents to clarify its oversight over regenerative medicine products. Dr. Gottlieb emphasized that the FDA will work with companies to provide a speedy pathway to market in order to foster innovation in this cutting edge field, while also increasing enforcement activities against bad actors in the space.

Regarding the regulation of cell therapies and regenerative medicine, the FDA will be announcing a comprehensive policy framework that provides bright lines between individualized cellular therapies being performed by medical professionals as part of the practice of medicine (and, thus, not subject to FDA regulation) versus new treatments that constitute medical products and should be subject to FDA oversight. For those products requiring FDA oversight, the FDA’s goal is to provide an efficient pathway to evaluate safety and effectiveness in order to bring these novel products to market. It is likely this will include finalizing draft guidance published in 2015 regarding FDA’s application of key regulatory standards.

The framework will also include implementation of regenerative medicine provisions in the 21st Century Cures Act. Those provisions establish a new Regenerative Medicine Advanced Therapy (RMAT) designation that allows eligible manufacturers to access FDA expedited review programs. Importantly, Dr. Gottlieb’s statement says that certain gene therapy products will be eligible for the new designation.

At the same time, Dr. Gottlieb highlighted the threat posed by unscrupulous actors who have been attempting to take advantage of the lack of clarity in the regulation of regenerative medicine by marketing unproven and unsafe products that could pose a serious risk to patients. To this end, FDA is creating a working group that will aim to target these bad actors in order to protect the public health.

According to Gottlieb, FDA’s new policy framework will consist of:

  • A series of guidance documents, informed by public meetings that have already been held and are part the public record;
  • A compliance policy that will give current product developers a “very reasonable period of time” to interact with the FDA and come into compliance with relevant requirements;
  • A “novel approach” to FDA approval that takes into consideration the promise of regenerative medicine products, the cost of innovative associated with such products, the small companies engaged in the development of the products, and the difficulty of doing FDA registration trials, to inform an efficient and minimally burdensome process; and
  • Continued implementation of the 21st Century Cures Act RMAT Designation, which will provide certain gene therapy products with access to an expedited pathway for approval.

In a related matter, on Aug. 30, 2017, the FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). The drug, a cell-based gene therapy, is the first gene therapy available for marketing in the U.S. It is a genetically-modified autologous T-cell immunotherapy that uses an individual patient’s own T-cells to treat and potentially cure his or her cancer.

The product is priced at $475,000. Interestingly, the drug’s manufacturer announced it has made an arrangement with the Center for Medicare and Medicaid Services (CMS) that will allow for payment only when patients respond successfully to the drug.

For more information on these important developments, please contact the authors.

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