Dr Scott GottliebScott Gottlieb, MD
The FDA has released a joint statement from its commissioner, Scott Gottlieb, MD, and Peter Marks, MD, PhD, the director of the Center for Biologics Evaluation and Research, regarding the policies that are being developed to address the growing development of cell and gene therapies.1

Ultimately, they laid out their plan to work with sponsors to maximize the expedited programs such as the regenerative medicine advanced therapy (RMAT) designation, to develop a series of clinical guidance documents intended to aid active product development and addressing potential issues, and to advance additional efficient pathways to approval.

According to the statement, the agency is expecting more than 200 Investigational New Drug (IND) applications each year by 2020, and the approval of 10 to 20 new cell and gene therapies per year by 2025, “building upon our total of more than 800 active cell-based or directly administered gene therapy INDs currently on file with the FDA.”

“At this time, for products covered by a user fee program, including cell and gene therapy products, our review of existing medical product applications and associated policy development is funded by limited carryover user fee balances. We’ll continue to update the public on how we’re approaching our work,” Gottlieb and Marks said.

The pair stated that the agency’s goal is to add 50 additional clinical reviewers to the current group that is overseeing the investigations, developments, and review of these therapeutics.

Gottlieb and Marks called this a reflection of “a turning point in the development of these technologies and their application to human health,” comparing it to the surge of antibodies developed in the late 1990s and their introduction into the mainstream as the foundations of modern treatment regimens.

“In the case of antibodies, it was a product innovation that sparked an inflection point in the advance of those products, after which antibody drugs became a mainstay of medical care,” they said.

They explicitly pointed to the widespread implementation of safe and effective platforms for humanizing these antibodies, which led to the eventual development of successful fully human monoclonal antibodies. In the instance of these gene and cell therapies, they compared their introduction as an innovation on par with the aforementioned antibodies, giving credit to the development of safe and effective viral vectors for gene and cell therapy delivery.

“Gene therapy products now have the potential to cure intractable diseases, and fundamentally alter the trajectory of many other vexing illnesses,” Gottlieb and Marks said. “To advance these opportunities, the FDA plans to introduce additional new policy guidance and other advances in our drug development framework in 2019. Today, we want to take an opportunity to preview that policy agenda and offer some perspective on the focus of our policies over the coming year as it relates to these technologies.”

They did acknowledge the concern about the number of companies and individuals operating outside of regulatory compliance, which, in some instances, has led to significant safety issues.  The agency declared that it is planning additional enforcement actions this year to address the products which pose the most significant risks and harm to patients.

Gottlieb and Marks said that they “believe these cell-based and gene therapy technologies hold tremendous promise for addressing some of the most intractable diseases. But with their novelty, also comes new uncertainties and some unique, theoretical risks.” Their efforts, they noted, are intended to ultimately aid those who “proactively address these potential risks, while we outline a modern and efficient pathway for the continued development of these innovations.”
REFERENCES
1. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance
development of safe and effective cell and gene therapies [press release]. Published January 15, 2019. fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629493.htm Accessed January 15, 2019.