ensuring pediatric testing of biological products, including consideration of any incentives, such as those provided under this section or section 351(m) of the Public Health Service Act.”2
This chapter outlines the incentives for pediatric studies included in BPCIA and explains why it is too early to assess the impact of these incentives or offer recommendations. It also summarizes information about biological products that have been studied, are being studied, or are pending study with children and then identifies a small number of products that appear not to have been the subject of pediatric studies. As context for this chapter, Appendix C describes some differences between small-molecule drugs and biologics and reviews information about the use of biologics by children. Appendix D includes tables listing 96 biological products with summary information about pediatric studies identified in each product’s labeling or in a public database of clinical trials.
Biologics Price Competition and Innovation Act
The primary objective of BPCIA was to create a pathway to licensure for biological products that are demonstrated to be biologically similar (biosimilar) to and, in some cases, interchangeable with a previously licensed biologic.3 In 1984, when Congress created a pathway for the approval of less expensive generic versions of drugs regulated under the Food, Drug, and Cosmetic (FDC) Act, no analogous pathway was created for products regulated under the PHS Act. At the time, modern biotechnology was in its early days, so the lack of such a pathway was not a particularly pressing issue.
Congress has defined the terms biosimilar and interchangeable. As summarized by FDA in 2010,
A biological product may be demonstrated to be “biosimilar” if data show that the product is “highly similar” to the reference product notwithstanding minor differences in clinically inactive components and there are no
2 Section 351(m) covers incentives for pediatric studies of biologics added to the PHS Act by BPCIA.
3 As described in Chapter 3, for regulatory purposes, 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 older products that were originally derived from human or other animal sources (e.g., insulin, human growth hormone, and certain enzymes) are regulated under the FDC Act rather than the PHS Act and were covered by BPCA from the outset.