New FDA framework points to boost for cell and gene therapies
- The Food and Drug Administration has released a regenerative medicine policy framework designed to "spur innovation, efficient access to potentially transformative products, while ensuring safety & efficacy."
- The two final documents and two draft documents will clarify which products are covered by the regulatory approach, and propose a more efficient process for evaluating safety and efficacy. The suite of documents also focuses on enforcement actions against products that are potentially harmful.
- FDA Commissioner Scott Gottlieb describes it as 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."
After decades of research, cell and gene therapies are finally emerging onto the marketplace and showing potential for regenerative medicine in serious conditions. Two cell-based gene therapies, Novartis AG's Kymriah (tisagenlecleucel) and Gilead Sciences, Inc.'s Yescarta (axicabtagene ciloleucel) have recently reached the market, and a number of others are getting close.
"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 Gottlieb.
With innovative therapies that have this kind of promise, the regulatory authorities are facing some major challenges. They need to create a balance between speeding life-changing treatments to patients and protecting patients from unforeseen harms.
"But this field is dynamic and complex… 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," he added.
The two final guidance documents in the framework clarify which cell- and tissue-based therapies would be exempted from the regulations, and which are subject to FDA premarket authorization. These also look at enforcement for products that pose a significant safety concern.
The draft guidelines focus going forward is on supporting development and access. The 'Evaluation of Devices Used with Regenerative Medicine Advanced Therapies' plans to simplify and streamline the regulatory requirements for devices used in the recovery, isolation and delivery of regenerative medicine advanced therapies (RMATs), including defining those used with a specified therapy as combination products.
'Expedited Programs for Regenerative Medicine Therapies for Serious Conditions' is the part of the framework that could have the biggest impact on the industry, by defining the programs that could promote the development of RMATs. These include RMAT designation, which was created under the 21st Century Cures Act, priority review and accelerated approval.
"In the longer term this bodes well for companies developing innovative cell and gene therapies, as the FDA appears to be accommodating and supportive of helping companies get through the regulatory process in an accelerated and streamlined manner. Overall, we see the types of initiatives… as favorable for industry as companies should be able to work collaboratively with FDA to accelerate the process, use novel endpoints, and have much smaller studies than normal," wrote Jefferies analyst Michael Yee in a note to clients. "We have said we expect more FDA approvals and in a faster time-frame than expected for many gene therapy and cell therapy players which literally makes the time to market faster and easier for these companies."
The International Society for Cellular Therapy (ISCT), the global professional society for the cell therapy sector, has stated its support for the framework, but has urged a sense of caution.
"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 Catherine Bollard, president of ISCT. "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."
- Food and Drug Administration Statement
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