Why PTN Research Matters and Patient Safeguards in PTN Research

Kelly Wade, MD, PhD, Children’s Hospital of Philadelphia, Co-Chair of the PTN Safety and Ethics Core

Why is it important to do research in infants and children?

The only way to safely dose medicine for children is to study the medicine in children. When it comes to how the body handles a medication, we have to remember that children are not small adults and infants are not even small children. Fortunately, we now have the technology to measure medication concentrations in just a few drops of blood and can coordinate the timing of a blood draw with one already being done for clinical care. By doing so, we can determine how to dose these medications in infants and children while minimizing any risks posed to study participants.

What safeguards are in place to protect the safety of children enrolled in PTN studies?

Many safeguards are in place to protect the safety of patients enrolled in all research studies. All research studies are voluntary, all procedures involved in research are fully disclosed during the informed consent process (see below), and all studies adhere to a strict confidentiality policy. Children will receive the same care whether or not they are participating in research. All children are monitored for any safety and adverse events, and these events are reviewed by a monitoring committee that will stop a study if harm is detected.

In fact, clinical trial programs have been shown to have public health benefits not only for trial participants, but also for patients in hospitals with infrastructure to conduct clinical trials and for those who subsequently benefit from the insights gained through the research.

Regarding the PTN specifically, all drugs being studied by the network are currently being prescribed to children; our research is aimed at finding the most effective and safe dose of medications already in use.

What is the informed consent process for pediatric trials and why does it exist?

The informed consent process is a personal discussion between a parent/guardian and a specially trained person working on the study. Children ages 7 and older are invited to assent to participate in studies. Parents/guardians are informed of why they are being approached, why the research is being done, and what procedures will occur for research purposes. The risks and possible benefits of such research are disclosed. Families are also given information about how to withdraw from research studies and who to contact if concerns arise. Families must acknowledge with a signature that they understand that their participation is fully voluntary. Research cannot begin prior to informed consent being obtained so that researchers can be certain that families know exactly what to expect from the study and what is expected of them.

What effect does the work of the PTN having on everyday clinical practice?

Data from PTN trials will provide evidence for proper dosing of commonly used medications, improving everyday medicine for children in both hospital and outpatient settings. Ultimately, we will make health care safer and more effective for babies and kids.