As part of what the US Food and Drug Administration (FDA) called “a comprehensive policy framework for the development and oversight of regenerative medicine products,” four guidance documents – two draft, two final – were published yesterday.
“We’re at the beginning of a paradigm change in medicine,” FDA commissioner Scott Gottlieb said in a statement. “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.”
There have been huge advances in cell and gene therapies, along with stem cell-based regenerative therapies over the past few years, and within the past three months the agency has approved two one-time treatments based on a patient's own genetically modified T cells – Novartis’ Kymriah (tisagenlecleucel) and Kite/Gilead’s Yescarta (axicabtagene ciloleucel).
However, such therapies are dynamic and complex, Gottlieb continued. “[They have] presented unique challenges to researchers, health care providers, and the FDA as we seek to provide a clear pathway for those developing new therapies in this promising field, while making sure that the FDA meets its obligation to ensure the safety and efficacy of the medical products that patients rely upon.”
The first draft guidance sets out requirements for devices used in the recovery, isolation, and delivery of regenerative medicines, while a second draft document gives information to sponsors looking to submit their product through an expedited programmes, and includes advice for getting on the Regenerative Medicine Advanced Therapy (RMAT) designation created by the 21st Century Cures Act.
One final guidance document intends to clarify when cell and tissue-based products removed from and implanted into the same individual are exempt from established regulations, while the second issues guides industry on the FDA’s definitions of “minimal manipulation” and “homologous use.”
Boon for cell and gene makers
The framework was greeted positively by the Alliance for Regenerative Medicine (ARM), a US advocacy group that lobbies on behalf of advanced therapy manufacturers.
Michael Werner, ARM co-founder and senior policy adviser said in a statement sent to Biopharma-Reporter: “These guidances are further evidence of the FDA's commitment to ensuring safe and effective treatments reach patients as quickly possible, while modernising the agency’s regulatory approach to take into account the innovative nature of regenerative medicine products.
He added: “Therapeutic developers now have further clarity on the regulatory process requirements to meet the FDA’s high product approval standards. The ongoing implementation of the RMAT designation also is a boon to cell and gene therapy manufacturers, as they can apply to qualify for FDA’s expedited approval pathways, with special attention paid to the uniqueness of these products.”
Safety concerns and rogue clinics
The FDA’s policies came on the same day the Agency sent a consumer warning regarding unapproved and unproven stem cell clinics and treatments.
“The US Food and Drug Administration is concerned that some patients seeking cures and remedies are vulnerable to stem cell treatments that are illegal and potentially harmful,” the warning said.
“The FDA is increasing its oversight and enforcement to protect people from dishonest and unscrupulous stem cell clinics, while continuing to encourage innovation so that the medical industry can properly harness the potential of stem cell products.”
In August, the Agency stepped up its crackdown on “unscrupulous” stem cell firms and clinics warning a Florida-based clinic and seizing vials of a smallpox vaccine in California used to create an unapproved product.