Frequently Asked Questions About Therapeutic Biological Products

These frequently asked questions have been developed for the CDER Small Business and Industry Assistance Web site to help small pharmaceutical businesses understand the regulatory process for therapeutic biological products.

1. What is a biological product? Biological products, like other drugs, are used for the treatment, prevention or cure of disease in humans. In contrast to chemically synthesized small molecular weight drugs, which have a well-defined structure and can be thoroughly characterized, biological products are generally derived from living material--human, animal, or microorganism-- are complex in structure, and thus are usually not fully characterized. Section 351 of the Public Health Service (PHS) Act defines a biological product as a “virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or analogous product, … applicable to the prevention, treatment, or cure of a disease or condition of human beings.” FDA regulations and policies have established that biological products include blood-derived products, vaccines, in vivo diagnostic allergenic products, immunoglobulin products, products containing cells or microorganisms, and most protein products. Biological products subject to the PHS Act also meet the definition of drugs under the Federal Food, Drug and Cosmetic Act (FDC Act). Note that hormones such as insulin, glucagon, and human growth hormone are regulated as drugs under the FDC Act, not biological products under the PHS Act
2. What Center has the regulatory responsibility for therapeutic biological products? Both the FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have regulatory responsibility for therapeutic biological products, including premarket review and oversight. The categories of therapeutic biological products regulated by CDER (under the FDC Act and/or the PHS Act, as appropriate) are the following
   • Monoclonal antibodies for in vivo use.
   • Most proteins intended for therapeutic use, including cytokines (e.g., interferons), enzymes (e.g. thrombolytics), and other novel proteins, except for those that are specifically assigned to the Center for Biologics Evaluation and Research (CBER) (e.g., vaccines and blood products). This category includes therapeutic proteins derived from plants, animals, humans, or microorganisms, and recombinant versions of these products. Exceptions to this rule are coagulation factors (both recombinant and human-plasma derived).
   • Immunomodulators (non-vaccine and non-allergenic products intended to treat disease by inhibiting or down-regulating a pre-existing, pathological immune response).
   • Growth factors, cytokines, and monoclonal antibodies intended to mobilize, stimulate, decrease or otherwise alter the production of hematopoietic cells in vivo.

Please refer to the Transfer of Therapeutic Biological Products to the Center for Drug Evaluation and Research for updates that further define the categories of biological products that are regulated by CDER and CBER.

Additional Information

• "Changes to an Approved Application for Specified Biotechnology and Specified Synthetic Biological Products" (PDF - 33 KB)
• "Content and Format of INDs for Phase I Studies of Drugs Including Well-Characterized, Therapeutic, Biotechnology-derived products" (PDF - 42 KB)
• "Providing Clinical Effectiveness of Human Drugs and Biological Products"
• "Points to Consider in the Manufacture and Testing of Monoclonal Antibody Products for Human Use" (PDF - 140 KB)