Experts call for increased neonatal inclusion in pediatric drug trials

Clinical drug trials are a vital part of pharmaceutical manufacturers gaining approval for use by the U.S. Food and Drug Administration. A Commentary scheduled for publication in The Journal of Pediatrics assesses the issues surrounding the lack of clinical trials on medications used by children, most notably neonates, and how drug manufacturers and academic researchers could work together to create clinical trials that would benefit this underrepresented population.

Henry Akinibi, MD, and colleagues from Cincinnati Children's Hospital Medical Center and the University of Cincinnati believe that the lack of clinical drug trials for is caused by many factors, including additional regulatory requirements, no adult equivalents of many neonatal diseases to gain a starting point, lack of financial incentives for , and the unique physiology of neonates. As a result, many doctors prescribe adult-approved drugs for children in off-label and unapproved uses; unfortunately, without clinical trials, the safety of this practice often is unknown and could place neonates at risk for unknown complications.

Throughout the years, the federal government has created legislation in an attempt to gather more information on the medications prescribed for children; the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act were made permanent by Congress in August 2012. These types of legislation have led to over 400 pediatric drug-labeling changes since 1998. Because neonates were addressed in only 6% of these changes, they continue to be at risk for receiving ineffective medications, incorrect dosages, and developing unanticipated complications. Although these new laws now include a requirement that submit plans for pediatric studies at certain times in the drug-approval process (e.g., Phase 2), they do not require a neonatal inclusion. This is important because drawing conclusions from the results of in older children and applying them to neonates is not ideal and continues to leave this population at risk.

Indisputably, unstudied medications for neonates have played a major role in the positive outcomes and increased viability for infants born prematurely, and, overall, recent laws have resulted in great strides in pediatric trials. However, the neonatal population still lags in inclusion. Of over 120,000 studies at the National Institutes of Health repository, only 0.6% involves neonates; in total, only 3.4% of all registered pediatric studies involve neonates. Open communication and cooperative sharing between industry and academics are integral components to ensuring that drugs are safe and effective for all populations. According to the first author of the article, Jason R. Wiles MD, "Inclusion of neonatologists and neonatal pharmacologists in study designs, and early in the drug development process, would make data generated from such studies more relevant and useful."

add to favorites email to friend print save as pdf

Related Stories

Federal laws have enhanced pediatric drug studies

Feb 29, 2012

Federal laws that motivate or require drug and biologic developers to conduct pediatric studies have yielded beneficial information to guide the use of medications in children, says a new report by the Institute of Medicine. ...

Children neglected in clinical drug trials

Apr 29, 2012

Although children are more likely than adults to suffer from many diseases, few clinical trials are being conducted to test drugs in pediatric patients, according to a study to be presented Saturday, April 28, at the Pediatric ...

Children continue to be underrepresented in drug trials

Jul 23, 2012

(HealthDay) -- Even for conditions with a high pediatric disease burden, only a small proportion of clinical drug trials study pediatric patients, according to research published online July 23 in Pediatrics.

Recommended for you

Helping babies survive

Nov 21, 2014

A healthy baby is born in the Haydom Lutheran Hospital in Tanzania. She is given the name Precious and her proud mother is ready to take her back to the village. Many children born in the same hospital, or ...

Unstable child care can affect children by age four

Nov 20, 2014

A new study from UNC's Frank Porter Graham Child Development Institute (FPG) reveals that disruptions in child care negatively affect children's social development as early as age 4. However, the study also ...

Parental involvement still essential in secondary school

Nov 20, 2014

Although students become more independent as they rise through grade levels and parent-teacher interactions typically lessen as students age, parental involvement in a child's education during the secondary ...

User comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.