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New FDA Regulatory Pathway Imperative for Cellular Therapy Advancement in U.S.

Wednesday, December 2, 2015

Washington, D.C. – To keep the United States at the forefront of medical innovation, federal policies need to incentivize new technologies to treat and cure patients. Groundbreaking regenerative cellular therapies represent a promising new approach to address degenerative organ and tissue disease. Therapeutic cells have the potential to halt progression of degenerative joint disease in the knee or the hip or help improve a failing heart or damaged cornea. However, significant FDA regulatory challenges prevent cellular therapy from fulfilling this promise. A new report by the Bipartisan Policy Center offers recommendations to accelerate the availability of safe and effective cellular therapies to Americans and improve U.S. competitiveness in the global marketplace.

In the report, Advancing Regenerative Cellular Therapy: Medical Innovation for Healthier Americans, project co-chairs, including former Senate Majority Leader Bill Frist, MD and former Representative Bart Gordon, with guidance from an expert panel, call for a new regulatory pathway that would promote the progress of cellular therapy in the U.S. This strategy is rooted in a set of core principles—all of which center on the need to promote public health while simultaneously protecting public safety.

“Recent scientific advances in cellular therapy have the potential not to just treat disease, but to cure it,” said Frist. “There will be challenges in bringing therapies from the bench to the bedside. But for regenerative medicine to be realized in the United States, we must ensure that our regulatory approach to cellular therapy hastens rather than hampers the emergence of this promising new field. To do this, we must adopt a more specific, modernized, relevant, rational, and responsible regulatory framework for cellular therapy.”

BPC’s proposal recommends strengthening the rights of patients to be treated with their own cells, empowering physicians to treat their patients with the best available therapies and innovative techniques, and enabling industry to support physicians and patients with safe, proven technologies. To assure that patients have access to innovative, safe, and effective therapies, BPC’s proposal recommends that the premarket approval process be modified for a unique class of therapeutic cells, including the patient’s own cells and the cells of a donor that do not induce an adverse immune response.

“Europe and Japan have outpaced the United States in their policies to grant patients safe access to cellular therapies,” said Gordon. “The U.S. has a strategic, scientific, and moral imperative to regain its lead in this space.”

Under current law, the FDA regulates cell-based therapies with a statutory framework based on those for chemical drugs, vaccines, and biologics, forcing these types of therapies into an ill-fitting regulatory regime. Additionally, FDA regulatory requirements—designed for products manufactured and sold on a mass scale—cannot be readily satisfied when it comes to treatments that are personalized to individual patients and more common in the practice of medicine.

“BPC’s proposal facilitates the FDA’s capacity to more rigorously and effectively evaluate the safety and efficacy of well-characterized therapeutic cells,” said Janet Marchibroda, director of the BPC’s health innovation initiative. “These recommendations represent a critical first step in ensuring that FDA’s regulatory framework reflects the rapidly emerging science related to cellular therapies.”

This proposal builds on BPC’s July report, Advancing Medical Innovation for a Healthier America, which contains viable policy actions that Congress can take to reduce both the time and cost of developing and delivering safe and effective medical products to patients.

KEYWORDS: FOOD AND DRUG ADMINISTRATION, BILL FRIST, JANET MARCHIBRODA, BART GORDON, 21ST CENTURY CURES