Federal legislation has improved the safety and design of drug studies in children, but there is room for improvement, particularly in conducting long-term follow-up studies of approved products, and in studies of newborns.
That was the finding of an Institute of Medicine review committee that examined studies of drugs and biologic products in children that have be conducted since the 1997 enactment of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA).
The BPCA provides economic incentives for drug companies to respond to Food and Drug Administration requests for studies in children, while the PREA allows the FDA to require pediatric studies in certain situations. Both laws are scheduled to be reauthorized this year.
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Although access to information from pediatric drug studies has improved, the studies remain limited in areas, such as the lack of long-term safety data on drugs that kids take for chronic conditions like asthma.
Since the two laws went into effect, the FDA has approved 400 labeling changes for drugs related to their use in children, according to the report. The report did not address the impact of BPCA and PREA on clinical practice or on the off-label use of medication in children.
Planning, initiating, and completing studies in a timelier manner would increase the benefits for children, the report authors noted. But they added that not all studies under BPCA and PREA succeed for many reasons, including insufficient numbers, weak study designs, and reluctance of doctors and parents to enroll children in clinical trials of approved adult drugs.
Although public access to information from pediatric drug studies has greatly improved under the two laws, such studies remain limited in several key areas, namely the lack of long-term safety data on drugs that children take for chronic conditions and on the long-term side effects on growth and development in children who take medications for short periods during infancy and early childhood.
In addition, there remains a lack of data on the use of drugs in infants aged 28 days and younger, and in premature or sick newborns, according to the report.
In 2010, Congress extended BPCA incentives to include biologics; therefore, it is too soon to evaluate any impact in this area, the authors noted.
The IOM review committee made suggestions for further improvements to pediatric drug studies, including:
• Improved public access to study findings.
• Improved timeliness of pediatric studies.
• Added emphasis on areas with insufficient research, including drug studies in newborns and long-term safety studies of children using medication for short-term and chronic conditions.
• Improved design and execution of pediatric studies.
• Added encouragement for the study of biologics in children.
• Improved clarity and understanding of the expected health benefits of pediatric studies requested under BPCA.