Tests of medicines for children gain ground

Drug Store News, Nov 19, 1990

Tests of medicines for children gain ground

NEW YORK - A survey by the Pharmaceutical Manufacturers Association (PMA) on the development and testing of medicines for children shows that a strong effort in pediatric medicine testing is under way, despite such obstacles as small sample sizes and cost justification.

At a press conference that released results of the survey - which the PMA said is the first on the subject - officials said the survey found that 114 drugs and vaccines for children are currently being tested. John Petriciani, vp-medical and regulatory affairs, PMA, termed the activity "remarkable."

However, there is a need for more testing of drugs for children in key therapeutic categories like cardiovascular and high blood pressure, participants said.

The categories in which the most drugs are being tested are bacterial infections, where there are 26 clinical research projects under way, and cancer, where 25 projects were discovered. The survey also found that nearly two-thirds of the 127 clinical research projects being pursued (some drugs are being tested for more than one use) are in the final stages: Phase III human clinical tests, or at the FDA for review.

New categories

"The number of drugs and vaccines currently being tested in children is impressive and the pharmaceutical industry is to be commended for its efforts to provide needed medications for children," said Ralph E. Kauffman, M.D., chairman of the Committee on Drugs, American Academy of Pediatrics.

"But," Kauffman continued, "we still have a long way to go to make all necessary medications equally available to children." There are important categories which "by and large have not been labeled for use by children," Kauffman said. They include pain medications, sedatives/hypnotics, anesthetics, medications for arthritis and drugs to treat gastrointestinal illness.

Noting that "historically, children have not shared in therapeutic advances to the same extent adults have," Kauffman said a recent survey of the "1990 Physicians' Desk Reference" (PDR) by his committee showed that labeling for 75 percent of the medications contains disclaimers or precautions against use in children. Advisories against use in toddlers and infants were in 91 percent of the labels.

Steven J. Bongard, Ph.D., director of the Forum on Drug Development, Institute of Medicine/National Academy of Sciences, said that having adequately tested drugs for pediatric use is a "long-standing dilemma for the practicing physician."

Bongard said the problem can be complex: "Many of the drugs listed in the PDR serve no therapeutic use in children. Also, the FDA does not require pediatric testing of those drugs that are initially developed for the adult population.

Large numbers need testing

"Nevertheless," Bongard continued, "there are a large number of these drugs that do provide children with therapeutic benefits, but have never been tested or approved for these purposes."

Testing drugs in children presents unique hurdles, including finding enough children with the conditions being tested, and cost-justifying drug testing and development for a smaller market, participants said.

Efforts are under way to promote continued development. Kauffman said the AAP Committee On Drugs is drawing a prioritized list of medications that should be tested for children. Among other efforts, the committee is supporting incentives to make the drug development economically worthwhile, such as an extension of exclusivity for products which include pediatric labeling.

Bongard said that the National Institute of Child Health and Human Development has received approval from its advisory council to develop a network of "six to ten" pediatric pharmacology centers which drug developers would pay for and use for clinical trials. The centers should help recruiters enlist patients for testing, as well as provide basic laboratory support.

Besides the research on baterial infections and on cancer, projects found by the PMA survey include nine on viral infections, 11 on genetic disorders, and seven each on AIDS and growth disorders.