The six-month market exclusivity provided to drugmakers which conduct studies of their products’ use in children under the 2002 Best Pharmaceuticals for Children Act (BPCA) is “absurdly generous,” according to Peter Lurie, deputy director of Public Citizen’s Health Research Group.

“Unless there is a strong reason to believe that pediatric usage will be minimal, conducting pediatric studies should be seen as the responsibility of all companies seeking to market a drug, not an undertaking for which companies should be rewarded, let alone as generously as they currently are,” Dr Lurie has told a hearing of the House Energy and Commerce Committee’s health subcommittee.

Moreover, he added, the Food and Drug Administration (FDA) should have the authority to compel such studies, no matter what the stage in the drug’s lifespan, without having to resort to patent extensions. The sole exception would be for drugs in which the pediatric use is off-label, in which case, a patent extension would be justified, but should not exceed three months for any drug and be lower for those with annual sales in excess of $1 billion, he said.

Support for new Act

The hearing was held to discuss the reauthorization of BPCA, which expires this year. Several legislators, including the subcommittee chairman, Democrat Frank Pallone, also felt the current market exclusivity provisions are too generous, and expressed support for the Senate’s reauthorization measure, S 1082, which would provide market exclusivity for just three months only on products with annual sales over $1 billion.

Marcia Crosse, director of health care at the General Accounting Office (GAO), presented the results of a GAO study conducted to examine what effects BPCA has had on the conduct of pediatric drug trials. It found that, while drug sponsors have initiated such studies for most of the patented drugs for which the FDA has requested them under the Act, no drugs have been studied when sponsors have declined these requests.

Also, few of the off-patent drugs which the National Institutes of Health (NIH) has said should be assessed for pediatric use have in fact been studied, said the study. It also found that about 87% of drugs receiving pediatric exclusivity under BPCA have had labeling changes, often because the studies found that children might have been exposed to ineffective drugs or dosing, overdosing or previously unknown side effects. However, the process for approving labeling changes was often lengthy; for 18 drugs that required labeling changes (about 40%), it took 238-1,055 days for information to be reviewed and changes to be approved.

'Extraordinary' success

The current program has been “extraordinarily successful” in improving medical care for children, said Lori Reilly, vice president for policy and research at the Pharmaceutical Research and Manufacturers of America (PhRMA). In less than 10 years, the program has resulted in studies on about 120 diseases and conditions and has led to new labeling on about 120 new or already-approved drugs for use in children, she added.

However, Donald Lombardi, chief executive of the non-profit Institute for Pediatric Innovation, told the panel that standard approaches for commercializing innovations, including the system for transferring federally-funded research results from academic and medical research institutions to the for-profit sector, do not work for pediatrics.

A solution requires novel forms of public-private, non-profit/for-profit collaboration to carefully assess what products are needed and to drive innovation toward these needs, he said, and called for legislation to be designed which provides incentives and support for such collaborations, while avoiding “unwanted consequences.”