WASHINGTON - Following the Senate's lead, the House of Representatives passed legislation giving the FDA authority to force drug companies to conduct clinical trials of adult drugs used off label in the pediatric population.
President Bush is expected to sign the legislation. On passage in the House Wednesday, Tommy Thompson, secretary of Health and Human Services, and Mark McClellan, FDA commissioner, released a joint statement praising Congress for its move to protect children.
"Children are a unique population with special medical needs," the statement said. "Access to drugs that have been properly tested for pediatric use will give us all more confidence that the drugs we use to treat our children will be safe and will work as expected."
The American Academy of Pediatrics, a 57,000-member Chicago-based organization, is pleased. After all, the AAP has worked 40 years to get such legislation passed, Marjorie Tharp, AAP's Washington spokeswoman, told BioWorld Today.
The legislation covers all biologics and pharmaceuticals that can be used in children (for example, drugs specific to Alzheimer's disease would not be included). However, companies may request an FDA waiver or deferral under the new legislation.
Once Bush signs it, the Pediatric Research Equity Act (Senate bill 650 and House bill 2857) will become effective immediately and retroactive to April 1999. In a unanimous vote, the Senate passed the legislation in July. (See BioWorld Today, July 25, 2003.)
In 1998, the FDA implemented something similar called the Pediatric Rule, which required companies to run trials in children. But a year ago, a U.S. District Court for the District of Columbia overturned the rule, saying the FDA lacked sufficient statutory authority to require such studies.
Supporters of the legislation lobbied for the retroactive element so that companies would be required to follow through on pediatric trials that have been on hold or have not started since the Pediatric Rule was struck down.
The Washington-based Competitive Enterprise Institute (CEI), one of the groups responsible for convincing the U.S. District Court to strike down the Pediatric Rule of 1998, opposes the legislation, saying it will add another layer to the drug-review process.
Sam Kazman, general counsel at CEI, said the new law will discourage off-label research because if companies get any indication that their product works in children, the FDA will force them to conduct pediatric trials.
Also, Kazman fears the law opens the door for others - for example, the geriatrics population - to begin lobbying Congress for laws requiring trials specific to them.
The Pediatric Research Equity Act contains a "sunset," or expiration, date of 2007, meaning it would have to be reauthorized by Congress periodically. While supporters weren't happy with that part of the bill, opponents, like Kazman, said a sunset provision is better than "'til death do us part."
Tharp said the rule complements the Best Pharmaceuticals for Children Act, a law that gives financial incentives to companies that voluntarily decide to test drugs in children. The Pediatric Rule includes drugs not covered by Best Pharmaceuticals, which also must be reauthorized in 2007.