ing the study and approval of new drugs and biologics and their labeling, and mechanisms for monitoring drug safety after products are approved for marketing. It then describes major features of BPCA and PREA. The discussion of PREA includes a short comparison of differences in requirements for pediatric drug studies between the United States and Europe. The chapter concludes with some suggestions for policy makers as they consider the reauthorization of BPCA and PREA in 2012.1Chapter 4 describes another part of the regulatory framework for pediatric studies—regulations concerning the protection of human participants in research. Chapter 8 provides more information about BPCIA, the implementation of which was still in its early stages at the time this report was being completed.

BASIC REGULATORY FRAMEWORK FOR DRUG
DEVELOPMENT, APPROVAL, AND SURVEILLANCE

Definition of Drugs and Biologics

As defined in the FDC Act, drugs are

articles recognized in the official United States Pharmacopoeia, official Homoeopathic Pharmacopoeia of the United States, or official National Formulary, or any supplement to any of them; and (B) articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals; and (C) articles (other than food) intended to affect the structure or any function of the body of man or other animals; and (D) articles intended for use as a component of any article specified in clause (A), (B), or (C). (21 USC 321(g)(1))

This definition encompasses both small-molecule chemical compounds (what are conventionally called “drugs”) and biologics.2

For regulatory purposes under the PHS Act, as amended by BPCIA in 2010, 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)). A few biologics have been and still are regulated under the FDC Act. These include a small

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1 After this report was released in February 2012, Congress reauthorized BPCA and PREA in June 2012. The FDA Safety and Innovation Act changed several provisions described in this chapter. A comparison of the previous and current legislation can be found in AAP, 2012.

2 In 1972, the Secretary of what is now the Department of Health and Human Services gave FDA the explicit authority to apply the requirements of the FDC Act to biologics (37 FR 4004, cited in Carver et al., 2010).



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