Pediatric studies proposed for drugs

The Food and Drug Administration announced a proposal that will require that new drugs and biologics be labeled for safe use in children. If approved, the mandate may also apply to some medicines that are already approved and in use.

Pharmaceutical manufacturers will be required to conduct clinical pediatric studies for any medicines which may offer improved treatment for children over existing therapies, or for medicines that will be widely used in children.

According to the Pediatric AIDS Foundation, as many as 80% of drugs currently marketed in the United States remain untested for dosing and safe use in children. As a result, pediatricians may be reluctant to prescribe breakthrough products for young patients, or they may prescribe the products in inappropriate doses.

The FDA decided to propose a formal mandate because pharmaceutical companies were not doing enough pediatric studies on their own. The lack of voluntarily submitted data was particularly troubling to the FDA because it had simplified regulations for pediatric studies in 1994.

"Kids deserve the same access to newly developed drugs that their parents get," said Donna Shalala, secretary of the Department of Health and Human Services. "With this proposal, we will have the power to ensure that pediatricians and other health care providers who treat children have the best scientific information available on which to base their medical decisions."

The FDA approves approximately 30 new medicines each year. Under the proposed new rule, about half of these products will require pediatric testing.

Industry reactions

The change in policy, which was announced by President Clinton in August, was applauded by the National Association of Chain Drug Stores, the American Academy of Pediatrics, the Children's Defense Fund and the Pediatric AIDS Foundation.

The Pharmaceutical Research and Manufacturers of America, although supportive, was somewhat less enthusiastic. It questioned whether the proposal would slow approval of new medicines for adults.

"We think the FDA is reasonable about this but we want to make sure that it won't delay the introduction of new drugs," said Anne Oman, an assistant vice president of communications for PhRMA. "Hypothetically, if a company has a drug out for asthma and it's only been tested in teenagers but not in young children, then, in order to market it, the company has to apply for a waiver. All of the paperwork has to be done before [the company] gets approval, and all that may take time. Then it's not only adults who will suffer, but children and teenagers as well."

Overall, however, PhRMA is comfortable with the proposed changes. Oman summarized: "The more studies you do, the more you know, and that's great."

The FDA did say it would allow post-approval submission of data if it had safety concerns about testing a drug on children prior to approval. It could also waive the requirement if the FDA found "the product was likely to be unsafe or ineffective in pediatric patients, that pediatric studies were impossible or highly impractical, or that reasonable efforts to develop a pediatric formulation had failed."

The FDA will consider written comments on the initiative from the public and the industry. It will then develop a final ruling. PR