PR Newswire, 810 Seventh Avenue, New York, NY 10019 - Wednesday August 13, 1997 - 3:22 PM EDT

Currently, 80% of the drugs marketed in the U.S. have not been tested for safety and dosing in children. The absence of such studies poses a significant public health problem because doctors must choose between guessing the correct dosage of a drug not tested on children or depriving their pediatric patients of much-needed treatment.

"The Pediatric AIDS Foundation has worked diligently to draw national attention to this issue," said Susan DeLaurentis, Co-founder of the Pediatric AIDS Foundation. "For the past two years, we have aggressively tried to make sure that children are no longer forgotten. This may now become a reality."

Under the proposed mandate, the Food and Drug Administration will require pediatric-use data prior to drug approval for any medicine which may offer improved treatment for children over existing therapies or for medications that will be widely used by children. For drugs already on the market, this regulation would also codify FDA's authority to require, in compelling circumstances, that manufacturers conduct studies to support pediatric-use labeling for the approved indications.

Should a manufacturer fail to comply with either request, FDA would not hold up the drug for use in adults. Rather, they would bring an action in court against the manufacturer to require the necessary children's research be done.

This proposed rule would allow post-approved submission of pediatric data if FDA had safety concerns about testing the drug on children prior to approval. Likewise, the requirements could be waived if FDA found that the product is likely to be unsafe, or ineffective in pediatric patients, that pediatric studies were impossible or highly impractical or that reasonable efforts to develop a pediatric formulation had failed.

This regulation will provide physicians, pharmacists, and parents with the data needed to make treatment decisions based on scientific information aimed at their patient population. Therefore, sick children will have greater access to safe and effective drugs that could extend and improve the quality of their lives.

The Pediatric AIDS Foundation Response to the Pharmaceutical Manufacturers Association (PhRMA) On Testing Drugs for Safety for Children

In its press release dated August 12, 1997, PhRMA commits itself to working with the "Administration and anyone else to advance the goal of better medicine for children." The Pediatric AIDS Foundation (PAF) welcomes that commitment and looks forward to PhRMA's cooperation on this pressing problem.

However, PhRMA goes on in the press release to question the FDA's proposed regulations to require that drugs be tested for safety and dosing for children's use. In asking this question, the press release makes a number of serious errors, omissions, and misleading statements. This is an effort by PAF to correct the record.

Argument in Press Release:

Manufacturers are voluntarily testing drugs for children. "We question whether a government mandate is needed."

Response: The industry is not testing most drugs for children. 80% of the drugs already on the market have not been tested for children.

In 1991, only 56% of new drugs approved with potential usefulness in children were actually tested for children. In 1996, only 37% of new drugs approved with potential usefulness in children were actually tested for children.

During the period between 1991 and 1995, it is estimated that 60 drugs were approved that would be affected by the proposed regulation. Of those 60, 37 (61%) had no pediatric labeling.

Argument in Press Release:

The industry is primarily concerned about protecting the safety of children and avoiding risks of research.

Response: Children are placed at significant risk through industry practices of failure to test drugs. As the American Academy of Pediatrics has noted, the use of untested drugs "may place more children at risk than if drugs were administered as part of well-designed, controlled clinical trials."

Drugs that are untested in children are widely used already. Five million prescriptions a year are written for the top ten unstudied drugs alone.

Guidelines have been established by both HHS and the American Academy of Pediatrics about the protection of children in research.

The proposed regulation contains a specific waiver if there are reasonable grounds to believe that the drug is unsafe in children.

Argument in Press Release:

There are practical difficulties in testing drugs for children and in developing formulations appropriate for them.

Response: All drug research has some practical difficulties, not just children's research.

The proposed regulation offers a specific waiver of the children's research requirement if the study is highly impractical and another specific waiver if reasonable efforts to develop a pediatric formulation (when needed) fail.

Although industry argues that special formulations are often a problem, this argument is applicable only to very young children who cannot swallow pills. If problems in formulation were the reason that industry fails to do research on children, one would expect that research on older children would be routinely done. It is not. Manufacturers have largely failed to do research on children under the age of 12, and some have done no research on children under 16.

Argument in Press Release:

Adult drugs should not be delayed.

Response: Agreed, there should be no delay in drugs for adults. The only action proposed in the regulation if a manufacturer fails to do pediatric research is a court order requiring them to do it, but no delay in approving the drug for adults.

Conclusion

The current practice in most of the pharmaceutical industry is not to test drugs on children except in special circumstances. The Pediatric AIDS Foundation believes that presumption should be reversed: Drugs should be tested on children unless there is a reason not to. The proposed FDA regulation to be announced by the President today would accomplish that goal.

Statement of Susan DeLaurentis, Co-founder, Pediatric AIDS Foundation

"I want to thank the President, the First Lady, Vice President Gore and Secretary Shalala for raising this issue to the highest visibility and for taking this step to ensure that children are no longer left behind in the progress of biomedical research.

"The Pediatric AIDS Foundation was established to get drugs and research for children with HIV. We have been active on this issue because children with HIV couldn't get the same drugs as their parents. My best friend and fellow co-founder, Elizabeth Glaser, was shocked to find that the drugs that were available to her were not available to her daughter, Ariel.

"A decade later, that is still too often the case. Last year, there was great news for adults with AIDS -- protease inhibitors offered new hope. But the hope wasn't available to kids for almost a full year later. Only this spring, two of these drugs were finally approved for older children. One was a drug that did its adult trials long before its pediatric trials -- the usual story for most AIDS drugs. The other, however, was the drug manufactured by Agouron, a company that did the right thing by developing their drug for children at the same time as for adults.

"But still none of these new protease inhibitors have been approved for infants and newborns. This is especially tragic since recent studies show that the most promising time to control and potentially reverse the effects of HIV could be in a newly infected newborn.

"Children should not be an afterthought. Progress in research should include both adults and children. And while we have been primarily involved in AIDS, we recognize that this problem affects children with many other illnesses. So the solution should not be disease-specific. It should be for all diseases. It should be children-specific.

"I want to thank Senators Dodd and DeWine and Congressmen Greenwood and Waxman for bringing this longstanding problem to the public's attention, and we look forward to continuing to work with them when Congress returns. I would also like to personally thank the staff at the FDA, in the Administration and in particular, Tim Westmoreland and Bill Schultz for their hard work and commitment to the resolution of this untenable situation.

"Today's announcement will change history. From now on, children will not be automatically left out. From now on, it will be the rule that drugs are tested for children, unless there's a good reason not to.

"For that, I am grateful to the President, the First Lady, and the Vice-President, who have once more demonstrated that this is an Administration that truly cares about children."

SOURCE: Pediatric Aids Foundation

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