As the industry focuses its attention on the upcoming renewal of the Prescription Drug User Fee Act (PDUFA), there is a tendency to overlook two other significant pharmaceutical programs coming up for renewal and a related piece of legislation that has been introduced:
Incentives for ResearchFDA doesn't require prescription-drug manufacturers to test their products in children. The historical result has been that most prescription therapies in the United States do not have labeling for pediatric uses and do not contain specific recommendations for dosing in children. To remedy this lack of information, the FDA began requesting more data on pediatric medication use on a voluntary basis in 1994—but this effort had little impact. In 1997, Congress took up the issue when it authorized the FDA to offer financial incentives to pharma companies to develop pediatric data on their drugs as part of the FDA Modernization Act (FDAMA). This effort was augmented by Congress in 2002 in the Best Pharmaceuticals for Children Act (BPCA), which expires on September 30, 2007.
The outlines of these programs are similar: FDA decides which already marketed drugs require more research and asks the patent holder to conduct the studies. If the company completes the research, it receives six months of additional market exclusivity. If the company declines, FDA has the option of requesting that the research be performed under a grant from the Foundation for the National Institutes of Health—with no reward to the company.
The Pediatric Exclusivity Program has yielded important new information about uses of medications in children. By the end of 2006, findings from clinical studies initiated under the program resulted in new pediatric labeling for 122 drugs, according to Dianne Murphy, director of FDA's Office of Pediatric Therapeutics. These label changes included new child-safety information for 35 drugs, new or altered pediatric dosing information for 25 drugs, new dosing and instructions for younger pediatric populations for 82 drugs, and findings of a lack of efficacy for 24 drugs. By comparison, according to a US Government Accountability Office (GAO) study of the effects of BPCA, in the six years prior to FDAMA, only 11 studies of marketed drugs were completed—though 71 studies were promised.
At a hearing conducted by the Senate Committee on Health, Education, Labor, and Pensions this past March, pediatrician Richard Gorman, representing the American Academy of Pediatrics, pointed out cases in which research performed under BPCA had led to real improvements in the care of children. During his testimony, Gorman said the act had: