FDA’s Policy on Regenerative Medicine Therapies

On Nov. 16, 2017, FDA Commissioner Scott Gottlieb, MD, issued a statement regarding the agency’s policy on regenerative medicines. The agency is creating a pathway for product developers to ensure the safety of patients. Gottlieb announced a framework for how the agency will apply existing laws and regulations to these therapies.

The use of cell-based therapies in regenerative medicines may be transformative for patients, but they come with unique challenges for researchers, healthcare providers, and regulators. Since 2005, when FDA first established a regulatory framework for regenerative medicines, advances have been made in the field. Cells and tissues can now be engineered to grow healthy, functional organs; new genes can be used to fight disease; and adult stem cells can generate new cells to replace those lost to injury or illness.

FDA wants to support these innovations while ensuring public safety. To do this, the agency is considering having academic investigators follow the same manufacturing protocols and share combined clinical trial data in support of FDA approval. This would ensure that “small investigators who are working with cells that are being manufactured in ways that render them subject to our current laws and regulations-because the cells are, for example, more than ‘minimally manipulated’-can nonetheless seek the FDA’s approval through a less burdensome process,” Gottlieb said in the statement.

The agency also released four guidance documents intended to implement the Regenerative Medicine Advanced Therapy (RMAT) designation program. This program is designed to expedite development and review of these therapies.

“To be clear, we remain committed to ensuring that patients have access to safe and effective regenerative medicine products as efficiently as possible. We are also committed to making sure we take action against products being unlawfully marketed that pose a potential significant risk to their safety. The framework we’re announcing today gives us the solid platform we need to continue to take enforcement action against a small number of clearly unscrupulous actors,” Gottlieb stated.

Source: FDA