Drug Testing in Children : Part 2

by Food and Drug Administration (FDA)

(Page 2 of 2)

Q. Those steps might help with drugs now under development and testing. What about drugs already on the market?

A. We have a way of dealing with that, too. Currently, according to the regulations, no drug may be labeled for use in children unless it has been subjected to "adequate and well-controlled studies" in children. But the regulations also give FDA the authority to waive that requirement, if there have been adequate and well-controlled studies in adults.

Q. Under what circumstances will FDA consider granting this waiver?

A. We have specific criteria. First, the disease has to be the same in adults. Then, the drug must have been well studied in adults. To use the drug appropriately in children, you need to have information on dosing and on adverse reactions, which may be different in children.

Q. Can you explain the new regulation about the "pediatric use" section in drug labeling for physicians?

A. We published a final regulation Dec. 13, 1994, revising the present pediatric use section in physician labeling for all marketed drugs for diseases for which the safety and effectiveness trials in adults are relevant for children. The change will allow use of information from other sources, such as blood level data or adverse event data — if we think it appropriate.

Q. Have FDA's increased efforts begun to pay off?

A. We are encouraged. In the past few years, we have seen a definite increase in the number of new drugs approved specifically for children. FDA has approved major new drugs for pediatric diseases. Exosurf and Survanta are pulmonary surfactants for respiratory distress syndrome in newborn babies. Adagen is an enzyme, ADA, to replace an enzyme missing in children with a severe immunodeficiency. Chemet is an oral drug for treatment of lead poisoning in children. FDA has classified these drugs as important new therapies.

We are seeing a burst of pediatric studies; other drugs for pediatric diseases are being studied or are close to approval.

Q. Do you see any areas where there is a particular need?

A. One in particular stands out: drugs for newborns. With the advent of neonatal intensive-care units and increased survival of tiny babies, we are seeing the need for new drugs to treat these small creatures. One success involves the pulmonary surfactants, drugs that help the survival of premature infants whose lungs have not yet fully developed. Pharmaceutical manufacturers have been vigorous in developing surfactants. We at FDA have considered it important to make these life-saving therapies available as rapidly as possible. We approved two treatment INDs for therapy before approval, and the first surfactant was approved for marketing in a near-record five months after an application was submitted.

Q. Dr. Botstein, what should parents do if they have any questions about drugs their children are taking?

A. If parents have any questions about drugs, such as the proper dosage or possible side effects, they should always check with their physician or pharmacist.

Q. Can parents do anything to help make more drugs available for children?

A. Perhaps the most important thing they can do is to consider allowing their own child or children to participate in clinical drug testing if the opportunity is available. People often think of the practical difficulties — such as the need to draw blood samples — of studying drugs in the young. But we have found that parents are often eager to have their children participate, especially if they feel they can help family members or other children.

New Rules for Children's Medicines

New measures to provide health-care professionals with the information necessary to more safely prescribe medicine for children were announced by FDA in December 1994. Previously, most prescription drugs were tested primarily in adults and the labeling lacked information about appropriate use in children.

A 1979 regulation had required full clinical trials in the pediatric population as the basis for labeling for children. A new rule, published in the Dec. 13, 1994, Federal Register, revised the "Pediatric Use" section to allow companies in some situations to base pediatric labeling on data extrapolated from adequate and well-controlled adult studies together with other information on treating children. However, the rule makes it clear that such pediatric use statements can be made only if the course of the disease and the drug's effects are sufficiently similar in children and adults.

Also, under the new rule, manufacturers must reexamine existing information to see if pediatric labeling of already marketed products can be modified on the basis of adult studies and other available data. If so, the manufacturers must take the necessary steps to modify existing labeling.

In addition, the new regulation clarifies the agency's authority to request specific pediatric use information. For example, FDA might request pediatric use data for a drug that is widely used, represents a safety hazard, or is therapeutically important in children. The rule, however, does not limit the manner in which a practitioner may prescribe an approved drug.

A special pediatric subcommittee will track implementation of the new regulations and take steps to ensure that the various possibilities of pediatric testing and use are explored. FDA's Center for Drug Evaluation and Research has taken steps to increase the number of pediatric studies included in submissions for new prescription medicines. The agency also will work closely with the National Institute of Child Health and Human Development to conduct pediatric studies and will work with sponsors of new products to ensure that necessary pediatric data are included.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.