On National Child Health Day, and every day, I feel grateful for the work I do at Covance, and how it impacts the lives and health of children. I also recognize this event as an opportunity to spread knowledge about the work still remaining.
Often, when I tell people outside of the medical profession that I manage pediatric clinical research trials, the first response is usually: “I don’t know how you could ever put a child in a trial with investigational medicines. You don’t know if the medicines work or not.” From there, the conversation naturally opens for me to discuss my role in pediatric research and provide education on the importance of clinical trials for pediatric healthcare.
Addressing the elephant in the room with pediatric medicine
Historically, nearly 50% of the medications given to pediatric patients have never been formally researched in a clinical trial setting. That means we don’t understand how a child’s body processes, metabolizes, or reacts to these medications and, as a result, these medicines have no information on their label for dosing pediatric patients. In these cases, physicians practice what is called “off-label dosing” where they must make their best guess on the doses and amounts required.
The younger a child is, the greater the likelihood of off-label dosing. For example, with preterm infants, nearly 90% of their medications have never formally been tested in a preterm or neonatal population. We don’t clearly understand the appropriate dosing regimen, tolerability, or other safety parameters, and, therefore, these most vulnerable patients are often being given doses of medication based on historical use, not on well-defined research performed within their population(1).
One may assume that smaller doses are needed for a smaller body. But kids aren’t tiny adults. For example, we know that infants have very different metabolic and nutrition needs as compared to an adult. Or consider an adolescent going through puberty, who may have unpredictable shifts in growth, hormones and metabolism. These critical variables can impact how medicines are processed and utilized within the body.
Without formally researching medications in the continuum of pediatric populations, we leave our children at risk for adverse events, or sub-optimal dosing that doesn’t effectively or efficiently treat, address or prevent a condition or a disease.
Understanding the role clinical trials have in children’s health
In the 1990s, regulators in the United States and Europe began developing regulations, which were ultimately made permanent. These regulations require pediatric clinical trials for all new medications seeking adult labeling and approval, with the caveat that the illness is also found in the pediatric population.
These regulations have made a tremendous impact on pediatric medicine. During 2007-2016, there were approximately 300 pediatric clinical trials resulting in 107 labeling changes that included pediatric dosing information(2).
Beyond determining safe and effective dosing regimens, knowledge gained from clinical trials impacts another important facet of pediatric medicine. Patients and their families now have a voice in their care, as these clinical studies also collect outcomes measures, a type of healthcare quality measurement, gathered directly from the families. This additional information helps clinicians understand what is important to children – and their families – when they are ill and provides crucial insights on how to improve their paths to wellness.
Our ongoing reminder to improve pediatric health
While National Child Health Day is a reminder of the basic care and preventive measures that parents and healthcare providers should take to keep children healthy, I see this day as a valuable reminder to our medical community: Our work is not yet done. We have so much to learn about how to care for children and how their bodies react to illness and treatments.
Without pediatric trials, we place undue risk on our most precious and vulnerable population. Today’s clinical research trials are well thought out, highly regulated and contribute valuable information to our ever-expanding body of knowledge in healthcare and medicine. That’s why it’s essential to include children and their families in the research process. Together, we can appreciate the patient perspective and understand the impact treatments have on the ongoing health and lives of our pediatric populations.
1.Milne CP, Davis J (2014). The Pediatric Studies Initiative: After 15 Years Have We Reached the Limits of the Law? Clinical Therapeutics. Vol 26 (2).
2. Avant D, et al. J Pediatr. Oct. 10, 2017, http://dx.doi.org/10.1016/j.jpeds.2017.08.048