Children remain underrepresented in drug research, says expert

January 11, 2017, Purdue University

Children continue to be underrepresented in drug and medical research, making them less likely to receive personalized health-care treatments for life-threatening conditions such as cancer and infectious disease, says Purdue University professor of chemistry Peter Kissinger. New tools are about to change this.

Prescribing and administering drugs is especially difficult because young children cannot describe their feelings with any precision, and supportive data from has been lacking. Many drugs are also used off label.

"One area where we believe we can make a difference very quickly is in where patients have limited blood volume and their health status can change very quickly," Kissinger said. "Studies have shown that we can now collect blood samples of as little as 0.1 percent of an ounce in a preprogrammed way that reduces labor, blood waste, and infection risk, while still allowing quality measurements. Some chemical diagnostics can now be made with a million-fold reduction in volume compared to 1980. This opens a number of opportunities for pediatric health care, especially for very ."

Kissinger reports that continued improvements in the way research is conducted with these will have long-term benefits.

Research over the last five decades has enabled more evidence-based decisions derived from . These studies provide averages for subpopulations that guides therapy, but imprecisely. We are just beginning to have the potential for quality data to support decisions one patient at a time. While traditional physical data such as body temperature and heart rate have great value, chemistry is driving the bus.

"The reduction in volume is made feasible by parallel advances in mass spectrometry and immunoassays. For example, ambient ionization methods developed in professor Graham Cooks' group at Purdue enabled our team to help found Prosolia Inc. This measurement technology is especially viable for drug determinations," said Kissinger, who later began Phlebotics Inc. to improve blood sampling for personalized medicine. "The combination of more efficient sampling and better measurement instruments provides a real opportunity to improve both pharmaceutical research and care for hospitalized children. To make it work, the data must be integrated into a decision support system that fits hospital workflows, considering the entire team of caregivers."

Using such methods for treatments and will be important to patient outcomes and to gain consent from parents for clinical trials.

"This is just the beginning of a multifactorial problem, which we are working together to address," he said. "There are still challenges and considerations in collecting real and meaningful data to improve health care outcomes for children."

Other reasons why children are often not the focus of clinical studies include:

  • Children are not small adults. Their physical characteristics change very rapidly from birth to their late teens. The rate of change confounds clinical trials of any length. For example, newborns vary biochemically month to month, and age is not a reliable metric to predict phenotype.
  • The ethical challenges of clinical trials with children are daunting to some and require informed consent from parents and/or guardians. Unlike for adults, healthy children are not included.
  • While Congress and the FDA have provided incentives, such as extended market exclusivity to pharmaceutical companies who add a pediatric component to trials for new drugs, the incentive is not often sufficiently compelling.

"Working with children's hospitals is a logical next step, but it is a very big step with respect to regulations, demonstrating safety and fitting into medical workflows," Kissinger said. "Fortunately, the underlying science is complete for many of these enhancements."

Networks of children's hospitals working together for pediatric research include the Eunice Kennedy Shriver National Institute of Child Health and Human Development, which is funded through the National Institutes of Health. It has organized a pediatric trials network and a neonatal research network among children's hospitals that share experiences and data. The 2003 Research to Accelerate Cures and Equity for Children Act was introduced to Congress requiring compliance with the Pediatric Research Equity Act in 2003. Last month the 21st Century Cure Act passed Congress and was signed into law, further supporting the potential for more precise and less error prone care.

"We have achieved much in the past decade to advance treatments for , but there is much more we can and should do," Kissinger said.

Explore further: Are parents willing to have their children receive placebos?

Related Stories

Are parents willing to have their children receive placebos?

November 15, 2016
Placebos are essential in any controlled clinical trial, providing a yardstick against which the test drug is measured. Placebos are even starting to be used as a treatment in their own right, as studies show that they make ...

Children dying preventable deaths from congenital heart disease

December 16, 2016
Over one million children are born with congenital heart disease (CHD) each year. When children with CHD receive timely treatment, 85% can survive into adulthood to live healthy, productive lives. Sadly, 90% of the children ...

Making medications safer for newborns

October 4, 2016
Although new drugs must be shown to be both safe and effective for approval by the Food and Drug Administration, sick newborns receive most of their drug treatment off-label and without the evidence provided for adults and ...

Support for research without prior consent in cases involving critically ill children

October 23, 2015
Public health experts at the University of Liverpool have shown that parents and medics support research without prior consent in the emergency treatment of critically ill children.

Study examines financial losses for inpatient care of children with Medicaid

September 12, 2016
Freestanding children's hospitals had the largest financial losses for pediatric inpatients covered by Medicaid, suggesting hospitals may be unlikely to offset decreased Disproportionate Share Hospital (DSH) payments from ...

Two treatments yield similar results for children after cardiac arrest

April 25, 2015
A large-scale, multicenter study has shown that emergency body cooling does not improve survival rates or reduce brain injury in infants and children with out-of-hospital cardiac arrest more than normal temperature control.

Recommended for you

Phone-addicted teens are unhappy, study finds

January 22, 2018
Happiness is not a warm phone, according to a new study exploring the link between adolescent life satisfaction and screen time. Teens whose eyes are habitually glued to their smartphones are markedly unhappier, said study ...

Baby brains help infants figure it out before they try it out

January 17, 2018
Babies often amaze their parents when they seemingly learn new skills overnight—how to walk, for example. But their brains were probably prepping for those tasks long before their first steps occurred, according to researchers.

NeuroNext biomarker study explores natural history of infantile-onset SMA

January 9, 2018
Research led by The Ohio State University Wexner Medical Center to define the natural history of infantile-onset spinal muscular atrophy (SMA) has been "critical" to accelerate the development of effective therapies and hasten ...

No link between childhood lead levels, later criminality

December 27, 2017
(HealthDay)— Exposure to higher levels of lead during early childhood can affect neurological development—but does that mean affected kids are doomed to delinquency?

Early puberty in girls may take mental health toll

December 26, 2017
(HealthDay)—A girl who gets her first menstrual period early in life—possibly as young as 7—has a greater risk for developing depression and antisocial behaviors that last at least into her 20s, a new study suggests.

Technology not taking over children's lives despite screen-time increase

December 21, 2017
With children spending increasing amounts of time on screen-based devices, there is a common perception that technology is taking over their lives, to the detriment and exclusion of other activities. However, new Oxford University ...


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.