Testing kids’ drugs

Four years ago, Congress offered marketing incentives to entice drug manufacturers to conduct studies on the effects of drugs on children. A report from the Food and Drug Administration in May concludes both that the program has been effective in some areas and could be easily improved in others. This encouraging news should lead Congress to support an updated version of the act.

Only a small portion of all drugs used to treat children has been studied specifically to measure their effect on pediatric patients. The FDA can require these studies for new drugs in some circumstances but not in all cases and not on older drugs. This sometimes has made prescribed dosages more guesswork than science, particularly for medicine given to very young children. The danger of providing the wrong dosage is obvious and can prevent doctors from prescribing potentially more effective treatment, yet a decade ago, when the FDA tried voluntary measures to improve labeling for children’s medicines, it got little response from drug manufacturers.

In 1997, Congress offered to tack on six months of exclusivity on patent protections if the manufacturers would conduct pediatric studies on their products. Based on an FDA list of drugs that needed studying, the incentive had the desired effect. To date, at least 58 studies on pediatric drugs have been conducted through this program, with 25 exclusivity extensions granted. Improved labeling is expected on drugs to treat diabetes, pain, asthma, hypertension and many others. But the incentive wasn’t effective in all cases.

Drugs no longer covered by patent protections, for instance, weren’t covered by the act and neither were drugs with very low sales. Studies on certain drugs used to treat young children could not be conducted until studies on older children had been completed. Clearly, the act needed to be improved.

The Senate has before it a response to these shortcomings in the form of a bill called “Best Pharmaceuticals for Children,” which is co-sponsored by Sen. Susan Collins. It extends the exclusivity provision and provides the ability of the FDA to award contracts for studies on drugs unaffected by the provision. The added authority for the FDA should broaden the reach of the act, providing important information on drugs taken by children.

A FDA review of this program in January concluded, “The pediatric exclusivity provision has done more to generate clinical studies and useful prescribing information for the pediatric population than any other regulatory or legislative process to date.” The improved version of the act, now before the Senate, could make it even more effective.