CBER Today and Tomorrow: Challenges and Opportunities

The Center for Biologics Evaluation and Research (CBER) has responsibilities for complex biologic products, including gene and cellular therapies, blood products, vaccines, and human tissues. At the Alliance’s meeting with Dr. Peter Marks, CBER’s Director, he shared his vision for the Center — both challenges and opportunities. In particular, he noted how much medical progress has been achieved in areas regulated by CBER and how much more advancement is expected in the next 5 to 10 years. He pointed to regenerative medicine as an area that is expanding and evolving quickly, with significant encouragement from CongressIn a preview of his more detailed remarks, Dr. Marks spoke about the need for new approaches to manufacturing -- both in specific areas (discussed below), but also in a more general sense. Right now, most products involve an initial manufacturing stage to serve early clinical needs (pilot scale) and then a significantly larger, scaled-up version to serve late clinical trials and marketing. One important goal is to see how CBER can contribute to compressing this process, perhaps with companies able to get to a final/scalable product at an early stage. He noted that this was a particular problem, but also a great opportunity, in the orphan drug area where production volume will never be that great.Dr. Marks then talked about a number of CBER’s “applied scientific and regulatory initiatives.” One of these is a renewed effort to reduce pathogens in the blood supply. Not only are better methods needed to screen blood against well-known pathogens, but the blood supply system needs to be prepared for emerging threats, such as Zika and Ebola. More rapid and less expensive testing with greater detection power is also a priority in order to become less dependent on individuals disclosing risk factors when giving blood.Another initiative, which will be done with 21st Century Cures monies, is to create standards for the development of human tissue and cellular products. They are working on these standards with NIH and the National Institute of Standards and Technology (NIST). Closely aligned with this are CBER’s program for innovative development of regenerative medicine products. Dr. Marks also discussed CBER’s efforts to improve the manufacturing of gene therapy vectors. Methods being used (lentiviral and adeno-associated viruses) are over 20 years old and demand is outstripping the capacity to produce these two vectors. Similarly, CBER is interested in improving the manufacture of vaccines. While this has several elements, the keys are transition to continuous manufacturing and creating scale-up/scale-down processes to accommodate the variable demand for vaccines.Dr. Marks also spoke about his efforts to expand internal “applied science” research programs and his goal of having more partnerships with external academic institutions and industry. He also mentioned (as Dr. Shuren and Dr. Woodcock had also mentioned) that a hoped-for by-product of the manufacturing initiatives is to make it advantageous to manufacture in the US rather than overseas.Editorial note: The Analysis and Commentary section is written by Steven Grossman, Deputy Executive Director of the Alliance for a Stronger FDA.