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
- The Pediatric Exclusivity Program, which grants pharmaceutical companies additional marketing exclusivity in exchange for
performing needed research on the use of their products in children, is up for renewal and possibly permanent extension. Senator
Chris Dodd (D-CT) introduced the legislation in early March.
- The Pediatric Research Equity Act, which gives the Food and Drug Administration (FDA) the ability to require pediatric drug
studies under certain circumstances, is up for renewal, and Senator Hillary Clinton (D-NY) has introduced legislation (the
Pediatric Research Improvement Act) to renew nad modify it.
- Dodd has also introduced the Pediatric Medical Device Safety and Improvement Act of 2007, which brings the research-for-market-exclusivity
concept to the realm of devices.
Extending the patent life of a drug in exchange for clinical information about how it works in children has helped improve
the practice of pediatric medicine. But the approach remains controversial, and it is reasonable to expect that key elements
of the programs will come up for debate in the weeks to come. The criticism that you will hear most often is that pharma spends
too little—and makes too much—on pediatric studies.
Incentives for Research
FDA 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:
- given pediatricians the ability to give the correct dose of pain-relief medicine, such as Neurontin, to children with chronic
pain who previously received inadequate doses
- warned intensive care–unit physicians that a drug (Propofol) used for sedation in ICUs had twice the mortality rate as another
- given pediatricians and child psychiatrists important information on both the relative effectiveness and serious side effects
of antidepressant medications, like Prozac and Paxil, in adolescents
- given children increased relief of pain through medicines taken by mouth, breathed into the lungs, given through the vein,
and absorbed through the skin
- alerted both pediatricians and parents about unexpected side effects of medications that have allowed for a more complete
discussion of both the risks and benefits of a particular therapeutic course.