Biotechnology
Advances in stem cell biology have greatly accelerated research in regenerative medicine, defined as “a field that involves replacing, engineering, or regenerating human cells, tissues, or organs to establish, restore, or enhance normal function.” While there is still much progress to be made, specifically more widespread clinical benefits, the usage of cell-based products in regenerative medicine is key to innovation in the field.
The US FDA issues and enforces regulations to prevent the introduction and spreading of infectious diseases, and because the regulatory framework is based on risk levels, human cells, tissues, and cellular and tissue-based products are split into categories that require and do not require premarket approval. Biologic products are regulated under this framework as requiring to be studied for investigational new drugs, with manufacturers of biologics needing to submit a biologics license application to the FDA for approval before marketing the product or using it in clinical trials. This process can be drawn out and cumbersome, so the FDA has established expedited programs for new therapy development, especially if the therapy is aimed at life threatening conditions. One of these programs is the expedited program for Regenerative Medicine Advanced Therapies (RMAT), part of the 21st Century Cures Act. As of the end of 2017, the FDA had processed 43 requests for products to designated as RMAT and receive expedited approval, acting on 35 of them and granting the designation to 13 of them.
The FDA is also incorporating new expedited processes for companies to obtain approval, such as providing tools to incentivize small groups of doctors to collaborate on a regenerative medicine product, which will lead to each of the members of the groups receiving a biologics license. The manufacturers of the product would need to agree on a common manufacturing process and a common clinical trial process, and then the sites that are participating in the clinical trial would submit efficacy data as part of a biologics application. If there is a favorable benefit-risk profile, the FDA may use that data to determine the safety and efficacy of the drug, expediting the process.
