In mid-November, the US Food and Drug Administration issued a comprehensive regenerative medicine policy framework for the development and oversight of regenerative medicine products, including novel cellular therapies. The framework is intended to spur innovation, provide efficient access to potentially transformative products, and also ensure safety & efficacy, according to the agency.
Four guidance documents (two draft and two final) outline the framework, which builds on the agency’s existing risk-based regulatory approach and includes clearer descriptions of which products are regulated, a proposed science-based process for efficiently helping to ensure the safety and effectiveness of these therapies and an explanation of how FDA will focus its enforcement actions against those products that raise potential significant safety concerns.
“We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease,” said FDA Commissioner Scott Gottlieb, M.D. “But this field is dynamic and complex. As such, it has 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. With the policy framework the FDA is announcing today, we’re adopting a risk-based and science-based approach that builds upon existing regulations to support innovative product development while clarifying the FDA’s authorities and enforcement priorities. This will protect patients from products that pose potential significant risks, while accelerating access to safe and effective new therapies.”
The International Society for Cellular Therapy (ISCT), a global society of clinicians, regulators, technologists, and industry partners dedicated to bringing safe cellular therapies to patients, recently issued a statement in support of the new FDA framework. ISCT supports “continuing clarification and action from the FDA of the regulation of the cell therapy industry,” according to the association. The society has, in fact, advocated for stronger legislative oversight and improved frameworks for regulating the new cell therapy industry.
In particular, ISCT global leadership “welcomes the FDA’s stronger stance on unproven therapies and the additional clarifications on legislation around regenerative medicine.” It is also pleased that “FDA qualifications for fast approvals for gene and cell therapies proving sustained therapeutic benefits and efficacy will also provide considerable incentive to all stakeholders in the sector to innovate new cell and gene therapies.”
However, ISCT would like FDA to be cautious in its introduction and application of the new framework. “The FDA needs to balance bringing those operating outside the regulatory pathways to compliance, taking action against those that remain outside the licensing and regulatory frameworks, and continuing to foster the ongoing innovation and considerable potential for the majority of the sector operating within the regulatory frameworks,” said ISCT President Catherine Bollard. “An over-zealous regulatory application could increase development and manufacturing bureaucracy and costs and time to market, and delay the validation of products and facilities.”
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