Washington Editor

WASHINGTON - A Senate committee unanimously approved legislation that would force biotechnology companies to conduct pediatric trials on products that could be used off-label in children.

Sam Kazman, general counsel and expert on FDA policy for the Competitive Enterprise Institute (CEI), told BioWorld Today the legislation would result in "very bad trade-offs."

"Namely, this will have an impact on drug development," he said. "There's just no way around it. Even if the pharmaceutical industry is not publicly opposing this, you just cannot be adding more and more risks and requirements for any new chemical entity that's taken through the approval process and think it's not going to have an effect."

CEI, based in Washington, is the non-profit organization that convinced a federal court to strike down the Pediatric Rule of 1998 that gave the FDA authority to force companies to conduct clinical trials on adult drugs used for the same purpose in kids. The government decided against appealing the matter in court, instead choosing to work with Congress on legislation that would achieve the same result. (See BioWorld Today, March 10, 2003.)

And last week, the Senate Health, Education, Labor and Pensions Committee approved legislation, sponsored by Sen. Mike DeWine (R-Ohio), that mirrors the Pediatric Rule. Referred to as "The Pediatric Equity Act," DeWine's legislation allows companies to seek waivers on specific products and provides companies with an appeals process. However, the legislation gives the FDA authority to pull a product if a company refuses to conduct trials. It applies to all new drugs and biologics whose intended use in pediatrics is the same as in adults.

"If the drug is currently going through the approval process, then you have to persuade the FDA to give you a waiver, and if you don't get it, then you may well have to do lots more testing," Kazman said. "That's a risk that's being created that's not there now. Even if you get a drug out and there's an unforeseen use in pediatric populations - that's a blessing for pediatricians because it's another drug - but for a company, it may turn out to be a liability."

But DeWine doesn't view it that way.

The senator released a prepared statement saying, "We are introducing this bill so that our children are no longer a therapeutic afterthought. Children are not small adults. They metabolize drugs very differently and require different dosages than adults. Yet, because most drugs lack adequate pediatric safety information, every year millions of American parents are forced to play Russian roulette with their children's health - they reach in the medicine cabinet and hope for the best. Our children deserve better."

Indeed, Sen. Hillary Clinton (D-N.Y.), also a member of the Health, Education, Labor and Pensions Committee, released a statement saying, "Four years ago, I strongly urged appropriate testing and labeling of prescription drugs for children because common sense says that medicine for children should be proven safe and effective for children."

Aside from Clinton, other co-sponsors are Sens. Edward Kennedy (D-Mass.), Judd Gregg (R-N.H.) and Christopher Dodd (D-Conn.).

If DeWine's bill becomes law, supporters believe it will complement 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.

The American Academy of Pediatrics (AAP) in Chicago, a group that has worked for 40 years to get drug makers to test products in children, endorsed DeWine's legislation.

However, Marjorie Tharp, AAP's public affairs manager in Washington, told BioWorld Today the organization is not pleased with the "sunset amendment," which places an expiration date of 2007 on the legislation.

Amanda Flaig, a spokeswoman for DeWine, told BioWorld Today the senator is not happy with the amendment either, but he went along with it in order to get the legislation pushed through.

Meanwhile, CEI believes there are other ways of convincing companies to conduct pediatric trials. Kazman said the financial incentives provided under Best Pharmaceuticals and government-funded testing (through the National Institutes of Health) are better solutions.

"The worst effects of [DeWine legislation] will be that drugs won't get developed or they will be delayed," he said. "So for a drug that right now appears to be a marginal prospect, adding these new risks may turn the marginal prospect into a no-go drug."

The ruling to strike down the Pediatric Rule was appealed by AAP and the Elizabeth Glaser Pediatric AIDS Foundation of Washington. Kazman said AAP lost a request to speed the appeal in the U.S. Court of Appeals of the District of Columbia Circuit. CEI, along with co-plaintiffs the Association of American Physicians and Surgeons, of Tucson, Ariz., and Consumer Alert of Washington, have moved to dismiss the appeal on the grounds that AAP doesn't have the right to take it forward.