New analysis shows 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% of the burden of 10 major global diseases, only about 12% of the clinical trials for those diseases include or focus on children, according to Boston Children's Hospital doctors Kenneth Mandl and Florence Bourgeois’s analysis of five years of clinical trial data.

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 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 U.S. 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% of pediatric trials they surveyed were conducted with only government or nonprofit funding, while nearly 65% of adult trials had industry funding.

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