By Lisa Seachrist
WASHINGTON — The fact that over 50 percent of the drugs routinely given to children for asthma have never been tested in them and as a result come with no information regarding pediatric dosing served as the backdrop for an FDA hearing on proposed pediatric testing regulations.
The agency gathered pediatricians, bioethicists, patient advocates and industry representatives at a public hearing to get feedback on its plan to require that all new drugs be tested in children.
The problem, put simply by Wendy Goldberg, an activist with Patient/Family Representative and mother of an asthmatic six-year-old who takes nine medications that have never been tested in children, is that "I feel like I am testing drugs on her every day and nobody gets the benefit of that test.
"I can't believe it is impossible to test these drugs in children because parents are too scared to have their children in clinical trials," Goldberg said. "I don't think you could scare me anymore than I already am."
Those testifying at the hearing unanimously supported the agency's efforts to get adequate pediatric information about new and existing medications and urged the agency to empanel an advisory group to help them sort out the types of drugs that should be tested.
"Because this is such a high priority for the agency and the administration and the issues are complex, we decided to have this hearing," said William Schultz, deputy FDA commissioner for policy.
Prior Efforts Didn't Stir Enough Action
The agency established new rules in December 1994, streamlining the requirements drug sponsors had to meet in order to add pediatric use sections to their drug labels. Like the proposed plan, the 1994 rules allowed pediatric labeling based on efficacy data from adult trials in combination with pharmacokinetic and pharmacodynamic studies in children, provided the course of disease and mechanism of drug action were deemed identical in both adults and children.
Schultz, however, noted the agency didn't get the kind of response it had hoped for from the pharmaceutical and biotechnology industries. As a result, the agency proposed the new regulations, which were announced with great fanfare by President and Mrs. Clinton in a White House ceremony in August.
The proposed rule would require drug manufacturers to submit pediatric pharmacokinetic data that can detail dosages and side effects specific to children with all new drug applications. The requirement for additional studies would be waived in cases where the disease doesn't occur in children, if there is reason to believe the drug is unsafe or ineffective in children, or if the patient population is too small or too geographically diverse to make such studies cost effective.
"The proposed rules would also give the agency the authority to compel studies for drugs already in use," Schultz said.
Susan DeLaurentis, cofounder of the Pediatric AIDS Foundation, congratulated the FDA for taking steps to include children in biomedical research, but claimed the agency wasn't going far enough.
"The number of waivers available to the manufacturers needs to be minimized," DeLaurentis said. "This is about changing the paradigm by which the research is done in the first place. We should be starting from the position that all drugs should be tested in children and the exceptions should be rare."
D. Gail McCarver, a professor of pediatrics at Wayne State University, in Detroit, agreed with DeLaurentis' view and noted that an advisory panel could determine when waivers are appropriate as well as help the FDA and drug manufacturers determine which age groups of children should be tested.
While noting the agency's intent to provide more medications to children was laudable, Stephen Spielberg, a director of research for Merck & Co., of Whitehouse Station, N.J., suggested the FDA has all the regulatory authority it needs in the current regulations.
"My belief is that the 1994 rule has allowed us to make great strides with regard to pediatric testing," Spielberg said. "It will simply take some time in the development process for those strides to be realized."
Lawmakers Also Attacking Problem
Even so, Spielberg supported the notion of an advisory panel as well as urged early communication between the agency and the drug sponsor to be sure pediatric testing exposes the fewest number of children to the minimum level of risk.
Spielberg also mentioned the importance of financial incentives that Congress has put in place in its FDA reform bills.
In the Senate, Sens. Michael DeWine (R-Ohio) and Christopher Dodd (D-Conn.) have sponsored the Better Pharmaceuticals for Children Act, which provides six months' additional market exclusivity for drugs tested in children. Their provision was incorporated into the FDA reform bill, S. 830. Reps. Jim Greenwood (R-Pa.) and Henry Waxman (D-Calif.) are spearheading such efforts in the House.
The comment period for the proposed regulations is open until Nov. 13, 1997. *