Federal court overturns FDA pediatric drug testing rule

Bills before the House and Senate would require drug testing in children, but passage is uncertain.

By Tanya Albert, AMNews staff. Nov. 18, 2002. Additional information

In light of an October court decision, pediatricians are renewing their push for legislation requiring drug companies to test products in children to ensure that medications are safe and effective for them.

The U.S. District Court for the District of Columbia ruled that the Food and Drug Administration had exceeded its authority when it created the 1998 Pediatric Rule, which allows the agency to require companies to conduct clinical trials in children.

The opinion agreed with the arguments made by the Arizona-based Assn. of American Physicians and Surgeons and two conservative public interest groups -- Consumer Alert and the Competitive Enterprise Institute. The organizations sued the FDA in December 2000.

The court said that only Congress could grant the FDA the authority over pharmaceutical firms that is found in the Pediatric Rule.

"This court does not pass judgment on the merits of the FDA's regulatory scheme," U.S. District Court Judge Henry H. Kennedy Jr. wrote for the court. "The Pediatric Rule may well be a better policy tool than the one enacted by Congress; it might reflect the most thoughtful, reasoned, balanced solution to a vexing public health problem. ... The issue is the rule's statutory authority, and it is this that the court finds lacking."

The 57,000-member American Academy of Pediatrics and other groups this spring already had started to lobby Congress to codify the Pediatric Rule. Efforts began when the FDA in March announced that it would suspend the rule. Government officials quickly changed their minds, and in April the Dept. of Health and Human Services announced that it would continue to enforce the rule.

But the October court decision has recharged the AAP's and lawmakers' efforts for Congress to pass Pediatric Rule legislation.

"It's very clear now what Congress has to do," said pediatrician Philip D. Walson, MD, who serves on the AAP's Committee on Drugs and Clinical Pharmacology and is director of clinical pharmacology at Children's Hospital Medical Center in Cincinnati. "They have to make it clear that the FDA has to protect children."

Lawmakers in both chambers already have introduced legislation, but it is unclear if anything will pass before the end of the year.

A bipartisan group of lawmakers, including Sens. Hillary Rodham Clinton (D, N.Y.) and Mike DeWine (R, Ohio), offered a measure in the Senate in late April. In August, the Health, Education, Labor and Pensions Committee unanimously passed it.

"The protections that we have relied on since 1998 to assure the safety and efficacy of your children's medicine have been taken away," Clinton said about the October decision. "This court ruling underscores the need for immediate congressional action."

In the House, Reps. Deborah Pryce (R, Ohio) and Connie Morella (R, Md.) introduced legislation in October, just before the court ruled. The bill was referred to the Energy and Commerce Committee, and at press time the panel had not taken up the issue.

Championing their causes

The physicians and surgeons association opposes the legislation. In addition to arguing that the FDA overstepped its bounds, the AAPS and others who filed the lawsuit against the Pediatric Rule said they did so because they thought it delayed approval of medications for adults and exposed children in clinical trials to unnecessary danger.

"We have a major ethical problem with testing in children," said Jane M. Orient, MD, AAPS executive director. "It is senseless for the FDA to require pediatric testing for drugs that expressly disclaim any use on children. Would you volunteer your child for experimental trials?"

Dr. Orient said doctors for all age groups have to decide what medications at what dosages they will use to treat patients; they have guidelines to help them make those judgments. She noted that testing isn't done on pregnant women or elderly people, either.

Instead of clinical trials that test medications on a small percentage of people, closely monitoring patients' reactions to drugs is a better way to gather information, she said.

"Children who receive a drug tested on adults, as an off-label prescription, arguably are at greater risk than children taking a drug that has already been extensively tested in a pediatric population," Dr. Orient wrote in a letter to Sen. Judd Gregg (R, N.H.), the ranking member of the Health, Education, Labor and Pensions Committee. "However, they are at no greater risk than an experimental subject would be, and they also reap the potential benefit of a more effective treatment.

"There is no reason to think that the supervision of a physician who is dedicated solely to patient welfare is less protective than that of a physician who is in the dual role of physician-researcher," she added.

But the AAP disagrees. Failure to test medications on children, whose bodies react to medications differently from adults' bodies, puts them at great risk, the AAP said.

Dr. Walson said there had been cases in which drugs that work in adults had no results in children or in which doses adjusted for children still had been harmful.

"Even those of us in pediatrics who thought we were making good, educated decisions have found out what we thought was rational wasn't," he said. "There were some major surprises for things we didn't predict."

That's why testing is important, Dr. Walson said. "We are learning over and over again that there are a lot of differences in children. It's amazing to say that it's not a good thing to test in children. Let's turn it around and imagine drugs were tested only in children, and doctors say to an adult, 'We have a good idea how to use it.' How long would adults stand for that?"

ADDITIONAL INFORMATION:

Case at a glance

Assn. of American Physicians and Surgeons et al. v. U.S. Food and Drug Administration et al.
Venue: U.S. District Court for the District of Columbia
At issue: Whether the FDA had the authority to create a rule that requires drugmakers to test their products in children. The court said no.
Potential impact: Those who filed the lawsuit say it is a great win because children shouldn't be used as guineas pigs. Others, including the American Academy of Pediatrics, say the ruling is a terrible blow to guaranteeing that medications prescribed to children are safe for them.

Weblink

Memorandum opinion of the U.S. District Court for the District of Columbia in Assn. of American Physicians and Surgeons v. U.S. Food and Drug Administration, in pdf (http://www.dcd.uscourts.gov/00-02898.pdf)

GOVERNMENT & MEDICINE