FDA promises “nimble” regulatory system for gene editing

By Joan Conrow

June 8, 2018

The US Food and Drug Administration (FDA) has pledged to support the advance of new biotechnology tools, such as genome editing and synthetic biology, through an “innovative and nimble” regulatory approach.

“We’re committed to developing a framework that allows these innovations to safely advance, to fulfill the potential envisioned by those who are pioneering these approaches, and to inspire public confidence in these methods,” stated a blog post authored by FDA Commissioner Dr. Scott Gottlieb and Deputy Commissioner Anna Abram.

“The advance of these technologies holds significant public health promise. Unlocking their full potential and competitiveness depends on the trust we build now and in the years to come,” the post stated.

The agency vowed “to take new steps to help developers understand their responsibility to ensure product safety and we’ll identify ways to help reduce unnecessary regulatory burden and undue barriers to bring potential beneficial products to commercialization while ensuring their safety.”

To that end, the FDA plans to adopt an “efficient and predictable science- and risk-based regulatory framework that provides developers with regulatory clarity and predictability and maintains public confidence in our regulatory system,” the post stated.

The FDA asserted that these new biotechnology tools “hold enormous potential to improve human and animal health, animal welfare, and food security. And researchers and companies based in the United States helped pioneer these technologies. They position the US as a global leader of this rapidly growing and highly promising field.”

The post specifically referenced advances in agricultural biotechnology, including the use of gene editing to develop plants that can resist disease and environmental stress while offering such consumer benefits as better yields, more product variety and healthier nutrient profiles.

It also described how gene editing can improve the health and welfare of livestock, as well as “alter animals to minimize or prevent their ability to spread human disease…and produce human drugs, devices, or biologics, including tissues or organs for xenotransplantation.”

The agency’s statement reinforces the position taken by the US Department of Agriculture, which in late March also praised the “enormous promise” of plant breeding innovation and reconfirmed the agency has no plans to impose new or additional regulation on crops developed through new breeding techniques, such as gene editing.

To achieve its goals of advancing biotech innovation and modernizing the regulatory system, the FDA last month formed a new Biotech Working Group, comprised of FDA staff, that will develop an action plan that outlines steps for implementing a science- and risk-based regulatory framework, strong public education and outreach, and increased engagement with domestic and international partners to achieve regulatory alignment and efficiency.

The FDA also sees a role for the agency in facilitating the progression of research and development, due to “the wide spectrum of products that we regulate, and the in-depth scientific and policy engagement that the agency has with innovators and counterpart regulatory agencies around the world.”

“As our knowledge of genome editing applications increases over different product areas, we expect to build on those even greater synergies and increase our understanding to help with assessments of risks to human and animal health.”