When Congress enacted the Best Pharmaceuticals for Children Act (BPCA) in 1997, it was accepted that nearly two-thirds of the drugs being prescribed for children had never been tested for safety or efficacy on children, and doctors were simply guessing at the proper pediatric dosages. To try to remedy the lack of pediatric testing, the law gave pharmaceutical companies six months of extended marketing rights in return for conducting tests on children.

Ten years later, Congress is due to reauthorize BPCA this year, and controversy has erupted over whether the program’s benefit to children is outweighed by the windfalls many pharmaceutical companies enjoy from the extended period of copyright exclusivity.That profit can be substantial—much more than the cost of testing--if the manufacturer is protected from competition on a blockbuster drug, though smaller companies that make less popular drugs say they barely break even, according to a research report published in  the Journal of the American Medical Association.

As of 2007, the BPCA program has generated more than 300 pediatric studies, and more than 115 products have undergone labeling changes for pediatric use. That makes the program popular with pediatricians, who told the Senate Committee on Health in March that the studies sparked by the law and the better labeling of drugs are “a tremendous improvement over the shrugging shoulders and the resigned look and the soft sigh when we had to say, ‘I’m sorry, we just don’t know enough about this drug in children.’”

Drug companies are equally pleased. In a statement to the committee, the Pharmaceutical Research and Manufacturers of America (PhRMA) said the pediatric exclusivity program “has greatly advanced the medical care of children by helping spur research.” PhRMA noted that requests by the federal Food and Drug Administration (FDA) for pediatric studies have included a wide range of therapeutic areas, including treatment of fever, skin conditions, heart disease, HIV, cancer, endocrine problems, gastrointestinal disorders, and others.

The FDA is authorized by law to request such studies, and the Government Accountability Office (GAO) reported in March that drug sponsors have initiated pediatric studies for 173 of the FDA’s 214 written requests for studies of on-patent drugs. Most drugs granted pediatric exclusivity also had labeling changes, often because the pediatric studies found that children may have been exposed to ineffective drugs, ineffective dosing, overdosing, or previously unknown side effects.

The FDA regularly makes available summaries of medical and clinical pharmacology reviews of pediatric studies that have been conducted in compliance with BPCA. For example, in January this year, the FDA notified that studies had been completed for the drugs AZOPT, BETAXON, and GLEEVA; and in April the agency announced the availability of pediatric studies of CELEBREX, COLAZAL, ELOXATIN, EMTRIVA, SUPRANE, and TOPROL-XL. Summaries of all the pediatric studies that have been conducted so far are available at www.fda.gov/cder/pediatric/index.htm.

But there is work yet to be done, according to the National Institutes of Child Health and Human Development in the National Institutes of Health, which in March published a priority list of drugs for which it believes pediatric studies are urgently needed, including drugs that are used in the treatment of pediatric cancers, pediatric hypertension, and asthma.

In addition to debating whether to reauthorize the Better Pharmaceutical for Children Act this year, Congress has to decide about another law—the Pediatric Research Equity Act (PREA)—which gives the FDA statutory authority to request companies to perform clinical trials on medications used by children. That law is also set to expire this year, and Senator Hillary Clinton (D-NY) has introduced legislation to make the FDA’s authority to request studies permanent.

“The reasons for reauthorization of BPCA and PREA are clear, numerous, and resounding. Together, they provide both an incentive and a requirement crucial to the success of a robust pediatric program,” Samuel Maldonado, head of Johnson & Johnson’s Pediatric Drug Development Center, told the Senate committee.

Saying he is concerned about the profits to pharmaceutical companies from six months of market exclusivity in return for pediatric testing, committee member Senator Sherrod Brown (D-OH) is suggesting a compromise—he would let drugmakers choose between three months of market exclusivity or six months with 10 percent of profits used to finance pediatric research centers. “I think this proposal would ensure a continued flow of pediatric testing while multiplying the benefits of the pediatric exclusivity program—for the good of our children, for the good of taxpayers, and for the good of the public health,” Brown said.

Bills introduced in Congress to reauthorize the Better Pharmaceuticals for Children Act and the Pediatric Research Equity Act can be read and tracked at website http://thomas.loc.gov.

A research report, “Economic Return of Clinical Trials Performed Under the Pediatric Exclusivity Program,” appeared in the February 7, 2007, issue of the Journal of the American Medical Association.