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Rules and Regulations- Not the Only Obstacles to Pediatric Trials :

October 3, 2016

In a recently released article in Pediatrics, Dr. Joseph Pathma and colleagues bring us a fascinating study, “Researchers’, Regulators’, and Sponsors’ Views on Pediatric Clinical Trials: a Multinational Study.”

In a recently released article in Pediatrics, Dr. Joseph Pathma and colleagues bring us a fascinating study, “Researchers’, Regulators’, and Sponsors’ Views on Pediatric Clinical Trials: a Multinational Study.” (10.1542/peds.2016-1171) You definitely don’t need to be a researcher to enjoy reading this one. Anyone who prescribes medications for children will be interested.

The views of various stakeholders who are involved with clinical research are diverse. Yet perhaps not surprisingly one of the key controversies and tensions relates to how to protect the rights of children (who are participants in much needed clinical research) while also contending with  rules and regulations that are “infrastructural barriers”,  and could be perceived of as suffocating, discouraging or preventing the research they seek to regulate. Stakeholders in this article recommend some very interesting ways forward, including more international collaboration, more embedding of trials within clinical care, and streamlining of regulatory approvals. 

Formulating research trials to addressing global child healthcare needs is an optimal and achievable goal, but researchers and regulators will indeed need to think broadly, develop collaborations across borders, and establish high levels of cultural competency.  In the US, minors participate in research via a process of “Assent”, which is very broadly defined in the Code of Federal Regulations. Many local US Institutional Review Boards require written documentation of Assent, with an Assent document for 7-13 year olds and use of the adult or parental Informed Consent document for 14-17 year olds, even though this is not specifically required by the Code of Federal Regulations (Office for Human Research Protections).  The parent or legal guardian, however, must give permission (46.117 of subpart A of 45 CFR part 46) and (depending on the risks and benefits of the research) their permission must be documented in writing. In the US a child can decline to participate in research even if their parent has given permission, although for certain types of research (if the prospect of great benefit is obtainable only by participation in the research), the requirement for Assent may be waived and the child cannot dissent 45 CFR 46.408(a)

If you have been following the intricacies of what it takes for  child participation in clinical research in the US, now try moving your study to another country. If you move to another Western country, many cultural issues related to research will be similar though not the same.  In developing countries, however, the process of assent and consent will likely be quite different, not due to regulatory obstacles but due to cultural differences.  For example, a child may not be expected to provide independent assent regardless of the study’s benefits, and instead of seeking parental consent in a private location such as an exam room, a village meeting might be more appropriately called to explain the study to parents in a group setting. The informed consent form will likely require translation into at least one additional language, and transportation and logistics like refrigeration of medications and biological samples may be challenging and threaten to overshadow the trial itself. Vaccine trials, for example, benefit children and can be integrated into clinical care, yet are a living example of how challenging it can be to conduct needed pediatric research internationally.

Those who conduct pediatric research internationally are a unique and highly motivated group of medical scientists, and it would indeed be a great benefit to children worldwide if an international IRB with representatives from both high income and LMICs (low and middle income countries) could serve as the central or reliant IRB of record for international researchers. This would facilitate, for example, local context review, access to verifiable translation services and contact with persons who understand local regulatory and government requirements, all of which would hopefully allow for rapid yet culturally respectful and scientifically sound protocol review and approval.

Readers seeking more information can examine the “Harmonized Clinical Guidelines for the Conduct of Clinical Trials of Medicines in Pediatric Populations (Clinical Trials in Pediatric Population ) and the Food and Drug Administration Considerations for Pediatric Studies for Drugs and Biological Products (General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Products).

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