WASHINGTON - Drug makers are a step closer to having to conduct clinical trials for adult products that could be used off-label in the pediatric population.
In a unanimous consent Wednesday night, the Senate passed the "Pediatric Research Equity Act," handing the FDA authority to mandate pediatric studies in new drugs, biologics and some products already on the market. The legislation essentially would restore the 1998 "Pediatric Rule" that was struck down in October 2002 when a U.S. District Court judge said the FDA didn't have the power to force companies to conduct such studies.
Supporters of the legislation hope the House will have a chance to take up the legislation before the month-long summer recess begins today. (The Senate recesses for the summer Aug. 1.)
Either way, winning support from the Senate is a major coup for the American Academy of Pediatrics, the Chicago-based 57,000-member organization that has lobbied for 40 years to get this type of legislation passed.
In a prepared statement, Stephen Edwards, AAP's president, said, "The Senate gave back what should never have been taken away, the guarantee that the medicines our children take will be rigorously tested. When it comes to protecting the health and safety of children, it doesn't get much better than this."
Alan Holmer, president and CEO of the Washington-based Pharmaceutical Research and Manufacturers of America, and Sen. Bill Frist (R-Tenn.), a surgeon and Senate majority leader, released prepared statements supporting the legislation.
But Sam Kazman, general counsel at the Washington-based Competitive Enterprise Institute, characterized the legislation as problematic. "From a policy standpoint, we think this is a big mistake both in terms of drug development and the future of off-label research because, for starters, it adds something else the companies have to consider, and it's got an interesting disincentive to doing off-label research," he told BioWorld Today.
He said the mandate could cause companies to rethink certain off-label research if it means they could be hit with testing demands. "In a sense, doing off-label research may come back to bite a company, and that's a serious disincentive," he said.
The CEI is one of three groups responsible for persuading the U.S. District Court to strike down the Pediatric Rule of 1998. Co-plaintiffs were the Association of Physicians and Surgeons, of Tucson, Ariz., and Consumer Alert, of Washington. The AAP and the Elizabeth Glaser Pediatric AIDS Foundation in Washington are appealing the decision.
FDA Commissioner Mark McClellan told a House subcommittee in March that the Bush administration supports pediatric testing and had planned to pursue the matter via Congress as opposed to appealing the court decision. (See BioWorld Today, March 10, 2003.)
The Senate's legislation covers all new drugs and biologics that could be used in children (Alzheimer's drugs or other products specific to adults would not need to be studied). However, companies may request an FDA waiver or deferral under the new legislation.
Certain deferral rights could be exercised in the event that additional studies would delay introduction of a product to the adult market. Studies also could be deferred if trials in adults or older children need to be finished before testing for younger children or infants can be safely conducted.
The FDA also would have authority to waive the requirement if a company could prove the studies would be impractical, the product would not represent a meaningful therapeutic benefit over existing products or if the product would be not likely to be used in the pediatric population.
If the bill is eventually signed into law, Kazman worries it would create serious consequences. "Now you may be seeing other groups like geriatrics go to congressmen and say, if children are entitled to this, why aren't we," he said. "I think politicians are going to have a difficult time not heeding those groups, and in a sense, you are going to be expanding the range of special populations to cover just about everyone."
The legislation contains a "sunset," or expiration, date of 2007, meaning it would have to be reauthorized by the Congress periodically.
AAP believes the Senate's legislation complements the "Best Pharmaceuticals for Children Act," a law that provides financial incentives to drug companies if they voluntarily test medicines frequently prescribed to children. Specifically, the act gives a company six months of market exclusivity in exchange for conducting pediatric trials.
Also, the legislation would require companies to follow through on pediatric trials that have been on hold or have not started since the Pediatric Rule was struck down.