FDA's Authority to Ensure That Drugs Prescribed to Children Are Safe and Effective

Publication Date: May 2007

Publisher: Library of Congress. Congressional Research Service

Author(s):

Research Area: Health

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Abstract:

The Best Pharmaceuticals for Children Act (BPCA, P.L. 107-109) and the Pediatric Research Equity Act (PREA, P.L. 108-155) allow the Food and Drug Administration (FDA) to offer financial and regulatory incentives to manufacturers for testing their products for pediatric use. Unless Congress acts, both programs will end on October 1, 2007. FDA has approved for adult use many products never tested in children. Yet clinicians often prescribe them for children believing that the safety and effectiveness demonstrated with adults would hold for younger patients. However, this off-label prescribing results in children receiving ineffective drugs or too much or too little of a potentially useful drug. Some side effects are unique to children, or children of specific ages, including effects on growth and development. Studies show that drugs vary in bioavailability in children, which depends on the maturation and development of organs and other factors.

About 75% of drugs have not had pediatric studies. The market has not been able to overcome the economic, ethical, legal, and mechanical obstacles that make manufacturers reluctant to conduct these tests. FDA had tried to spur pediatric drug research through administrative action. With the FDA Modernization Act of 1997 (FDAMA, P.L. 105-115), Congress provided an incentive: in exchange for a manufacturer's completion of pediatric studies according to an FDA written request, FDA would extend its market exclusivity for that product for six months. BPCA gave this program a five-year reauthorization in 2002. From 1997 through November 2006, FDA sent 338 written requests to patent-holding manufacturers; 44% responded and 91% of those conducted the studies and were granted pediatric exclusivity. This resulted in 118 labeling changes -- for 87% of the drugs granted exclusivity, but only 35% of the drugs for which FDA requested studies. BPCA also set up study referral processes through the National Institutes of Health (NIH) for offpatent drugs and on-patent drugs for which the manufacturer declined FDA's request. Those programs have yet to yield labeling changes.

To get pediatric use information on the drugs that manufacturers were not studying, in 1998, FDA published the Pediatric Rule requiring that manufacturers submit pediatric testing data at the time of all new drug applications. In 2002, a federal court declared the rule invalid, holding that FDA lacked the statutory authority to promulgate it. Congress gave FDA that authority with PREA. PREA covers drugs and biological products and includes provisions for deferrals, waivers, and the required pediatric assessment of an approved marketed product. From 2003 through 2006, FDA approved more than 372 drugs and biologics; there have been 63 PREA-related labeling changes.

The FDA Revitalization Act (S. 1082, as reported) contains pediatric research provisions, including the continuing of BPCA and PREA. Updates of this report will reflect legislative activity.