immune-mediated conditions. Although some of these diseases are diagnosed in the pediatric population, research with these age groups is limited.

Since 1972, the Food and Drug Administration (FDA) has been responsible for the regulation of biologics. FDA licenses biological products under the Public Health Service Act licensing provisions and approves drugs under the federal Food, Drug, and Cosmetic (FDC) Act approval provisions. Under the FDC Act, certain old, relatively simple, biologically based products (e.g., insulin and human growth hormone) have long been regulated by the Center for Drug Evaluation and Research (CDER) through the New Drug Application process rather than through the Biologics License Application process of the Public Health Service Act (FDA, 2009c). In 2003, CDER also assumed responsibility for certain biologics. These are sometimes referred to as “therapeutic biologics,” although responsibility for regulation of other therapeutic biologics, such as intravenous immune globulins, remained with the Center for Biologics Evaluation and Research (CBER). CDER-regulated biologics include monoclonal antibodies for in vivo use, cytokines, growth factors, enzymes, immunomodulators, thrombolytics, certain therapeutic proteins, and nonvaccine immunotherapies (FDA, 2009d, 2010). Regulation of allergenics, blood and blood components (including recombinant proteins of blood components), gene therapy products, certain human cellular and tissue-based products (including stem cells and tissues for implantation or transplantation), vaccines, and nonhuman cells or tissues for transplantation remains under the authority of CBER (FDA, 2009a). This paper focuses on the biologics regulated by CDER and the CBER-regulated biologics that are derived from blood and blood components, with the exception of vaccines.


Generally described, biologics are “isolated from a variety of natural sources—human, animal, or microorganism—and may be produced by biotechnology methods and other cutting-edge technologies” (FDA, 2009e, unpaged). The regulatory definition provided in the Public Health Service Act (as amended in 2010) states that a biologic is “a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein (except any chemically synthesized polypeptide), or analogous product, or arsphenamine or derivative of arsphenamine (or any other trivalent organic arsenic compound) applicable to the prevention, treatment, or cure of a disease or condition of human beings” (42 USC 262(i)).

Biologics differ from conventional drugs in complexity and source. Unlike small-molecule drugs, which are produced by chemical reactions and

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