Zeno Swijtink
11-23-2007, 09:45 AM
Most prescription drugs for children still are not tested on them
Rob Stein, Washington Post
Friday, November 23, 2007
A decade after the government began trying to ensure that prescription drugs used to treat children are effective and safe, doctors still have scant information to guide them when they administer many medications to kids.
Although federal regulators have enticed or forced pharmaceutical companies to conduct hundreds of studies that have produced vital results about more than 200 drugs, perhaps two-thirds of the thousands of medications given to children remain untested on them.
"Are there children dying because of this? I don't know. Are there children being less effectively treated because of this? Probably yes. But I can't tell you because I don't know," said Richard Gorman of the American Academy of Pediatrics. "That's the problem: We don't know what we don't know."
What researchers have discovered has been disturbing: A highly effective adult migraine drug turned out to be worthless in children, sometimes causing serious side effects, including strokes; an asthma inhaler could inhibit growth; doctors were giving far too little of a medicine used to prevent seizures.
The alarming gap in medical knowledge is the legacy of many factors. The testing of drugs in children was shunned for decades as unnecessary and unethical; Congress and the pharmaceutical industry failed to provide adequate funding; and conducting medical experiments on children is difficult.
The quandary stems from the same dynamics that left over-the-counter pediatric cold remedies on drug store shelves despite little evidence they helped and mounting evidence that they could be dangerous. Drug companies, regulators and researchers long thought doctors could safely extrapolate the results of studies in adults and simply scale down the doses.
"Up to the late 1990s, children were mostly left out of new drug development," said Ralph Kauffman, an emeritus professor of pediatrics at the University of Missouri School of Medicine. "It just wasn't thought necessary."
And pharmaceutical developers had little incentive to focus on children.
"Pediatric patients were always the orphans. People didn't pay enough attention to them. They're just not a big enough market share," said Lisa Mathis, associate director for the pediatric and maternal health staff at the FDA's office of new drugs.
But researchers started to realize that children react to many drugs in surprising ways.
"Children are different - they are not just small adults," said Gregory Kearns, a professor of pharmacology and pediatrics at the University of Missouri in Kansas City. "They are not just fractions of adults."
In 1997, Congress began to address the problem. The FDA Modernization Act gave the agency a crucial tool - it could offer companies six precious extra months to sell a drug without competition if they studied it in children.
Congress renewed FDA's authority in 2002 with the Best Pharmaceuticals for Children Act, which also established mechanisms for the FDA to work with the National Institutes of Health to start scrutinizing some drugs companies ignored or had no incentive to study because they had already had generic competitors.
The legislation called for Congress to appropriate $200 million for the NIH to study the highest-priority medications. And in response to criticism that the pharmaceutical industry was getting a windfall from profitable patent extensions, the legislation established a fund to help finance studies companies would not at the Foundation for the National Institutes of Health, a private nonprofit entity.
Advocates pushed for a written commitment from the industry to provide $6 million a year, said Elaine Zining, who was the American Academy of Pediatrics' chief lobbyist. Then-Rep. Billy Tauzin, R-La., chaired the House committee that negotiated the legislation.
"Unfortunately for children, that money never materialized," said Zining. Tauzin subsequently took over as the head of the Pharmaceutical Research and Manufacturers of America (PhARMA).
The foundation has raised just $4.2 million - barely enough to pay half of one study of one drug.
Industry representatives said there was never a formal obligation to contribute to the fund. Noting companies were spending tens of millions of dollars sponsoring studies of patented drugs, they blamed the shortfall primarily on the failure of Congress to appropriate the promised money.
In the absence of the congressional funding, the NIH has spent just $25 million a year for each of the last three years to study a handful of the drugs identified as the biggest concerns.
Of about 50 drugs that have been deemed high priority, the NIH has managed to start studies of 14 , including Ritalin, lithium and morphine. But only preliminary steps have been completed.
This article appeared on page A - 6 of the San Francisco Chronicle
Rob Stein, Washington Post
Friday, November 23, 2007
A decade after the government began trying to ensure that prescription drugs used to treat children are effective and safe, doctors still have scant information to guide them when they administer many medications to kids.
Although federal regulators have enticed or forced pharmaceutical companies to conduct hundreds of studies that have produced vital results about more than 200 drugs, perhaps two-thirds of the thousands of medications given to children remain untested on them.
"Are there children dying because of this? I don't know. Are there children being less effectively treated because of this? Probably yes. But I can't tell you because I don't know," said Richard Gorman of the American Academy of Pediatrics. "That's the problem: We don't know what we don't know."
What researchers have discovered has been disturbing: A highly effective adult migraine drug turned out to be worthless in children, sometimes causing serious side effects, including strokes; an asthma inhaler could inhibit growth; doctors were giving far too little of a medicine used to prevent seizures.
The alarming gap in medical knowledge is the legacy of many factors. The testing of drugs in children was shunned for decades as unnecessary and unethical; Congress and the pharmaceutical industry failed to provide adequate funding; and conducting medical experiments on children is difficult.
The quandary stems from the same dynamics that left over-the-counter pediatric cold remedies on drug store shelves despite little evidence they helped and mounting evidence that they could be dangerous. Drug companies, regulators and researchers long thought doctors could safely extrapolate the results of studies in adults and simply scale down the doses.
"Up to the late 1990s, children were mostly left out of new drug development," said Ralph Kauffman, an emeritus professor of pediatrics at the University of Missouri School of Medicine. "It just wasn't thought necessary."
And pharmaceutical developers had little incentive to focus on children.
"Pediatric patients were always the orphans. People didn't pay enough attention to them. They're just not a big enough market share," said Lisa Mathis, associate director for the pediatric and maternal health staff at the FDA's office of new drugs.
But researchers started to realize that children react to many drugs in surprising ways.
"Children are different - they are not just small adults," said Gregory Kearns, a professor of pharmacology and pediatrics at the University of Missouri in Kansas City. "They are not just fractions of adults."
In 1997, Congress began to address the problem. The FDA Modernization Act gave the agency a crucial tool - it could offer companies six precious extra months to sell a drug without competition if they studied it in children.
Congress renewed FDA's authority in 2002 with the Best Pharmaceuticals for Children Act, which also established mechanisms for the FDA to work with the National Institutes of Health to start scrutinizing some drugs companies ignored or had no incentive to study because they had already had generic competitors.
The legislation called for Congress to appropriate $200 million for the NIH to study the highest-priority medications. And in response to criticism that the pharmaceutical industry was getting a windfall from profitable patent extensions, the legislation established a fund to help finance studies companies would not at the Foundation for the National Institutes of Health, a private nonprofit entity.
Advocates pushed for a written commitment from the industry to provide $6 million a year, said Elaine Zining, who was the American Academy of Pediatrics' chief lobbyist. Then-Rep. Billy Tauzin, R-La., chaired the House committee that negotiated the legislation.
"Unfortunately for children, that money never materialized," said Zining. Tauzin subsequently took over as the head of the Pharmaceutical Research and Manufacturers of America (PhARMA).
The foundation has raised just $4.2 million - barely enough to pay half of one study of one drug.
Industry representatives said there was never a formal obligation to contribute to the fund. Noting companies were spending tens of millions of dollars sponsoring studies of patented drugs, they blamed the shortfall primarily on the failure of Congress to appropriate the promised money.
In the absence of the congressional funding, the NIH has spent just $25 million a year for each of the last three years to study a handful of the drugs identified as the biggest concerns.
Of about 50 drugs that have been deemed high priority, the NIH has managed to start studies of 14 , including Ritalin, lithium and morphine. But only preliminary steps have been completed.
This article appeared on page A - 6 of the San Francisco Chronicle