In a special report published in the New England Journal of Medicine on March 8th, Peter Marks, M.D., Ph.D., and Scott Gottlieb, M.D. of the U.S. Food and Drug Administration (FDA) provide an overview of the regulatory context for regenerative medicine and outline the ways in which the agency has expedited the development of new therapies through instruments like fast-track designation, priority review, accelerated approval, and designation as a breakthrough therapy.
The report signals an openness on the part of the FDA to continue incorporating concepts and principles that promise to accelerate the approval process for new biologics without compromising patient safety. Among the concepts proposed is the proposal of a new process by which small investigators and firms can expedite the approvals process for life-saving therapies.
Under the traditional process, a biologic is produced in a central manufacturing facility and shipped to multiple sites, where clinical trials are conducted under the same protocol. A single application is submitted to the FDA, and if the product is eventually approved, a single license is issued.
The alternative process outlined in the report allows multiple manufacturers to produce biologics under the same protocol, and then treat patients enrolled in clinical trials at their individual sites. In this case, safety and efficacy data are pooled from all sites, and submitted as part of the biologics application for each individual site. If the product is approved, each site receives its own biologics license.
The FDA’s willingness to receive and consider clinical trials data based on treatment using biologics produced at different sites marks a significant shift towards the decentralization of cell and gene therapy manufacturing. For now, this alternative model applies to relatively simple products, and sites receive individual licenses based on their facility-specific manufacturing data. However, in the long run, demonstrating that different manufacturing sites can produce what is, for all intents and purposes, the same biologic, may enable a single company to manufacture in a decentralized fashion and receive a single license.
At Akron Biotech, we are driven by a desire to provide the highest quality ancillary materials in the industry while also supporting efforts to reduce the cost of producing these life-saving therapies. We welcome the FDA’s willingness to deploy new instruments, concepts, and principles that expedite the approval of safe and efficacious therapies and tissues, and will support small investigators and manufacturers that opt to decentralize production.
The article titled, “Balancing Safety and Innovation for Cell-Based Regenerative Medicine,” was published in the New England Journal of Medicine.