Manufacturers Seek Strategies for Ensuring Quality of Innovative Therapies

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Continued industry investment in advanced biologics will raise further regulatory challenges and require innovative manufacturing systems.

The challenges in producing cellular and gene therapies and other cutting-edge products that meet standards for safety, efficacy, and quality was high on the agenda for biopharmaceutical companies at PDA/FDA annual joint regulatory conference held on Sept. 11–12, 2017 in Washington, DC. Peter Marks, director of the Center for Biologics Evaluation and Research (CBER), opened the three-day meeting with an update on how the regulatory agency is expediting the development and approval of human cellular and tissue products. A main CBER initiative involves vetting new regenerative medicine advanced therapies (RMATs), including those utilizing gene-editing technology to treat malignancies and diseases.

Similarly, the National Institutes of Health (NIH) is tapping its expertise in cell biology and tissue engineering to advance regenerative medicine, explained Rosemarie Hunziker of the National Institute of Biomedical Imaging and Bioengineering. She noted that these products require very different manufacturing and dosing processes and raise multiple challenges for scale-up and for keeping cells alive during processing. Some of these issues will be explored by the new National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), which is being established at the University of Delaware with support from NIH and the National Institute of Standards and Technology (NIST).

At a later session, Deborah Hursh, senior investigator in CBER’s Office of Tissues and Advanced Therapies (OTAT), described how this relatively new and growing office is devising standards for characterization and potency assays for regenerative products, stem cell therapies, therapeutic vaccines, gene therapies, antivenins and certain combination products. Hursh addressed the importance of controlling the manufacturing process for these products and noted challenges in achieving the right level of product characterization based on critical quality attributes. Patricia Hughes, acting branch chief in the Office of Product Quality in the Center for Drug Evaluation and Research (CDER), discussed new developments in utilizing single-use systems and continuous biomanufacturing to improve microbial control and increase manufacturing efficiency, consistency and flexibility for innovative products.


Continued industry investment in advanced biologics will raise further regulatory challenges and require innovative manufacturing systems. Marks commented that knowledge gained from early CAR-T and other cutting-edge products should lead eventually to more standardized manufacturing systems. The first applications require greater attention to how a new treatment can target cells, but better understanding of manufacturing processes should emerge as these therapies become more familiar. PDA is continuing the conversation at a December conference on Cell and Gene Therapy in San Diego.