PL. 107-109 of the BPCA reauthorizes the pediatric studies provision of the 1997 Modernization Act to improve the safety and efficacy of pharmaceuticals for children. It continues to encourage pharmaceutical companies to conduct studies of on-patent drugs that are used in pediatric populations (but that are not labeled for such use) by extending their market exclusivity. In addition, P.L. 107-109 authorizes studies for off-patent drugs by the federal government or other entities with the expertise to conduct pediatric clinical trials.

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After several years of clinical studies, important label changes have had an impact on the use of these drugs in children (Table 1).

2002 and 2003

In January 2003, the Secretary of DHHS revealed that 12 commonly prescribed drugs would now require clinical testing for use in children. The governmental agencies (DHHS and FDA) have planned to support the testing of the drugs starting this year. Pediatric drug testing is required, according to the BPCA, as signed into law in 2002. The law provides for DHHS agencies or pharmaceutical companies to sponsor pediatric tests of certain drugs that have already been approved for marketing but have never been tested specifically for their effects in children. These drugs are the dozen highest-priority agents needing pediatric review. DHHS contends that now is the time to understand fully the effects of these medications in children (younger than 21 years of age). Read the rest of this entry »

1979

The FDA, working under the aegis of the Congress, began to persuade pharmaceutical companies to provide pediatric labeling information on drugs if such data were available. Initially, a 1979 FDA regulation required full clinical trials in pediatric populations as the basis for drug labeling for chil-dren, but progress in achieving this goal was made very slowly. The 1979 regulation permitted pediatric claims only if there had been adequate and well-controlled studies of the drug in children. This regulation, contrary to its purpose, stymied the hope that drug labels would provide adequate information for using drugs in children. For the words “Pediatric Use” to appear on the label, there needed to be “substantial evidence derived from adequate and well-controlled studies.”

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PROBLEMS OF DRUG TESTING IN CHILDREN

Children obviously need medicines, medical health professionals agree, but they are considered “therapeutic orphans” when it comes to drug development. Most drugs are studied, approved, and labeled for use in adults. To use one of these approved drugs for the same disease in children, it is important to know which doses work best in children and which kinds of adverse reactions are likely to occur. Other drugs are used for diseases specific to children, and adequate and well-controlled studies of their effectiveness and safety are needed. The physician labeling on the product normally includes a disclaimer that safety and effectiveness have not been established for use in children. That is the usual procedure with most of today’s drugs. Even morphine, which is a standard, necessary pain medication, has not included explicit labeling for use in children.

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