At the Center of Innovative Safe and Effective New Biologics

By: Karen Midthun, M.D. 

We’ve all read news reports of research achievements that promise exciting and powerful new treatments such as gene therapies to treat diabetes and cell therapies to rebuild failing hearts.  

These medical products are biologics and may seem like the stuff of science fiction. But they could transform the way doctors treat certain illnesses and conditions. Other, more traditional biologics, such as vaccines and blood products, may play an important role in protecting against bioterrorism

As the part of FDA that has regulatory authority over many of these products, the Center for Biologics Evaluation and Research (CBER) plays a critical role in their development through its regulatory oversight and research. CBER comprises a variety of offices, including three product offices responsible for 1) gene, cell, and tissue therapies, 2) vaccines and allergy products and 3) blood and blood products. 

Biologics differ from chemically manufactured drugs; they are usually derived from living sources (humans, animals, microorganisms). Work in this field is demanding because of the unique challenges biologics pose to manufacturers as well as to the reviewers at CBER. For example, the sensitive makeup of some biologics (e.g., DNA and cells) means they can’t be sterilized, so great care is needed in manufacturing them. In addition, biologics are complex and often based on new scientific knowledge that CBER reviewers must be familiar with. 

One way CBER meets the challenges of reviewing these products is by conducting its own research, which creates new knowledge and helps advance development of innovative medical products. CBER shares this knowledge by publishing results in peer-reviewed journals, drafting guidance documents to assist in product development, and sharing information through global collaboration and conferences with colleagues in research institutes, government agencies, industry, and stakeholders. Scientists at CBER can bring this added knowledge to their review of new products. CBER research  enhances CBER reviewers’ ability to evaluate a product’s safety and effectiveness—and to spot problems in product development that might not be recognized by manufacturers. 

Thanks to all of this work, CBER has approved important new products over the past couple of years, including 1) the first over-the-counter test that enables consumers to determine their own HIV (the AIDS virus) status in about 30 minutes—in private, 2) five stem cell products derived from cord blood (placenta-umbilical cord) for use in rebuilding the populations of blood forming cells and the immune system; 3) three influenza vaccines that protect against four strains of influenza virus rather than the previous limit of three strains, and 4) two other influenza vaccines, which are produced using mammalian cells or insect cells, rather than with eggs, as is customary. These two new production techniques for influenza vaccine offer the potential for faster startup of the manufacturing process than egg-based manufacturing, as well as the ability to rapidly address an unexpected, spreading epidemic. These vaccines also offer an alternative for individuals who are allergic to egg products. 

Importantly, CBER’s product safety efforts don’t stop after a product goes to market. The Office of Biostatistics and Epidemiology works with collaborators such as Medicare and the FDA Sentinel program on projects that can help evaluate whether certain adverse reactions are caused by approved treatments. 

As medical innovation accelerates in the 21st Century, CBER will continue to be at the forefront of ensuring the development of innovative safe and effective biologics. 

Karen Midthun, M.D., is Director of FDA’s Center for Biologics Evaluation and Research

Recent Related Posts