Drug safety and efficacy in children is rarely addressed in drug trials for major diseases

Jul 23, 2012, 11:05 ET from Boston Children's Hospital

BOSTON, July 23, 2012 /PRNewswire-USNewswire/ -- There are major discrepancies between the amount of pediatric clinical research for several major global diseases and the number of children who suffer from those diseases. While children bear nearly 60 percent of the burden of 10 major global diseases, only about 12 percent of the clinical trials for those diseases include or focus on children.

Such are the results of an exhaustive analysis by researchers led by Kenneth Mandl, MD, MPH, and Florence Bourgeois, MD, MPH, at Boston Children's Hospital of data from five years' worth of clinical trials. The discrepancies, the authors conclude, may be related to a relative lack of industry funding and support for pediatric clinical studies.

The investigators published their findings online today in the journal Pediatrics.

It is well known that children and adults respond to medications differently. However, clinical research focused on adults far outstrips that on pediatric patients, creating a knowledge gap when it comes to medication efficacy and safety for children with common conditions like asthma, migraine headaches, depression and malaria.

"Children are severely underrepresented in clinical research, despite several national and global efforts aimed at addressing their medical needs," said Mandl, who, along with Bourgeois, works in Boston Children's Division of Emergency Medicine; he also directs the Intelligent Health Laboratory in Boston Children's Informatics Program. "Without proper information on pediatric safety and efficacy, doctors are often left with little choice but to extrapolate from adult studies when prescribing medications for children."

To measure the level of clinical research for diseases with a high proportion of pediatric patients, Mandl, Bourgeois and their colleagues surveyed studies registered in ClinicalTrials.gov, a database of federally and privately supported clinical trials conducted in the United States and around the world. The team focused on drug trials from 2006 to 2011 for the 10 highest burden conditions as listed in the World Health Organization's Global Burden of Disease study.

The discrepancy between pediatric burden and pediatric research, while present for all 10 conditions, was most stark when looking at diseases primarily seen in middle- to low-income countries (e.g., lower respiratory infections, diarrheal disease, HIV/AIDS). The team also found that adult trials were more likely to focus on safety and to be conducted collaboratively across multiple centers than pediatric trials.

"Our study focused on the 10 conditions with the highest prevalence in the WHO data, but the discrepancy may actually be higher for those that are more rare," Bourgeois explained. "This could just be the tip of the iceberg."

Mandl and Bourgeois believe that the mechanisms for funding pediatric clinical research may contribute to the differences. More than 58 percent of the pediatric trials they surveyed were conducted with only government or nonprofit funding, while nearly 65 percent of adult trials had industry funding.

"We believe industry should play a larger role in supporting and conducting clinical trials for pediatric populations," Mandl said, "and take steps to ensure that children's needs are reflected in the clinical research enterprise."

This study was supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (grants 5T32HD040128 and 1R21HD072382) and the National Library of Medicine (grants 5R01LM007677 and 5G08LM009778).

Boston Children's Hospital is home to the world's largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults since 1869. More than 1,100 scientists, including nine members of the National Academy of Sciences, 11 members of the Institute of Medicine and nine members of the Howard Hughes Medical Institute comprise Boston Children's research community. Founded as a 20-bed hospital for children, Boston Children's today is a 395 bed comprehensive center for pediatric and adolescent health care grounded in the values of excellence in patient care and sensitivity to the complex needs and diversity of children and families. Boston Children's also is the primary pediatric teaching affiliate of Harvard Medical School. For more information about research and clinical innovation at Boston Children's, visit: http://vectorblog.org/.

Keri Stedman
Boston Children's Hospital

SOURCE Boston Children's Hospital