Pediatric Clinical Trials

an increase in the conduct of pediatric trials

I have not had the opportunity to work on a pediatric trial, however in the past year several people in my network have worked on these trials. I thought it was interesting that more of my old colleagues were working on clinical trials and after a capabilities presentation from ReSearch Pharmaceutical Services last week I am realizing that the trend may actually be correlated with an increase in the conduct of pediatric trials.

The RPS group provided historical data summarizing the number of active pediatric trials from in 2012 and then from a snapshot from the first quarter of 2013. Essentially, they showed a doubled expansion in the number of new pediatric trials.

A little background on the clinical trials for wee ones

In 1997, legislation was passed to permit “economic incentives for pediatric studies of approved drugs, including the 6-month period of pediatric exclusivity to be added to existing exclusivity and patent protection on a drug if studies fairly respond to a pediatric Written Request even if the data submitted are inconclusive or do not support a labeling change.” In 2002, the Best Pharmaceuticals for Children Act was created and then later amended in 2007.

So a sponsor can do a pediatric trial on an approved drug and receive 6-month patent exclusivity regardless of whether or not efficacy is demonstrated. This act was made permanent in the summer of 2012 with passage of the Food and Drug Administration Safety and Innovation Act

Note that in Europe according to European Medicines Agency regulation, all sponsors are required to prepare a pediatric development plan after Pharmacokinetic studies are completed in adults.

Testing drugs on kids is totally ethical

Or maybe not. The FDA asks that new medications and devices are tested in children (vulnerable persons) only when there is minimal risk. Children are afforded special protections beyond those provided for competent adults. The thing about early clinical development is that the safety profile is still an unknown; it isn’t possible to assure that the risk is minimal. The purpose of the early trials often isn’t to demonstrate medical benefit; it is most purely to assess safety. Therefore, children are not typically included in early clinical trials if there are inappropriate risks. There are not typically demonstrated benefits in early clinical trials so it is impossible to assert that benefits outweigh potential risks.

The exception follows from the guidelines that although healthy children are not included in early clinical trials, new interventions can be studied in critically-ill children provided that their participation in the trial isn’t precluding them from treatment with an approved alternative therapy and putting them at a potential disadvantage for recovery/treatment.
By and large, the amount of pediatric data at the time of approval is limited and the FDA typically requests sponsors to conduct post-marketing surveillance studies for drugs and devices that are likely to be used in a pediatric population.

Mother (or Guardian) knows best

But, if appropriate, the child must also assent to be in the study. For clinical trials in children, consent must be obtained by the parent or guardian and for “participants of appropriate intellectual maturity” assent must be obtained from the child participant (21 CFR Sec. 50.3r).  In any case, the child participant should be made aware of their right to withdraw consent at any time.

Kids are half-size adults, so they get half the dose

Yeah, not necessarily. There are some ethical considerations regarding running Pharmacokinetic (PK) studies in children.

blood sample clinical trial

Children truly are smaller than adults, so there are a lot of operational concerns such as the feasibility and safety of drawing the amount and frequency of blood required to demonstrate PK equivalence, the flavor of solutions or size of oral therapies, etc. You can reference the draft guidance from 1998 for further discussion on selecting appropriate doses for our pint-sized companions.

Learn More about Pediatric Trials

For further reading on pediatric trials, please visit the FDA pediatric product development section of the CDER website:

The European Medicines Agency Guidance on Pediatric Investigation Plans is located on their website here:

If you have questions, comments, or feedback on this topic, I would love for you to leave a comment or contact me via email.

You may also like: From the Lead CRA Blog

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About The Author


Nadia Bracken, lead contributor to the Lead CRA blog and the ClinOps Toolkit blog, is a Clinical Program Manager in the San Francisco Bay Area.


  • Matt Miller

    April 30, 2015

    This piece brings up some very important considerations when clinical research is being conducted in children. There are many assumptions when it comes to conducting pediatric research, such as issues with dosing amounts and consent. But this article pinpoints my feelings on the issues exactly. A great read.

  • Babi

    July 5, 2013

    Peds studies have become an important arena in the HIV space and in ex-US areas i.e sub-saharan Africa or Asia where children need to get access to life saving drugs like antiretroviral therapies (ARTs) where they still don’t have access. It’s important for companies to develop in this space. Having worked previously in the HIV space and seeing patients still having access issues to drugs it’s become more of a public health issue and sponsor responsibility to put capital on having more PEDs trials and allowing children to participate in life saving options. So yes, companies have a responsively to develop in this space ensuing the regs are followed and executing on proper clinical trials for children under the age of 18.

  • Marten CRA

    June 18, 2013

    I’ve experienced several times that children enrolled in a study wanted to quit, but their parents more or less forced them to continue. In these cases it has been children with very rare and serious diseases, where good treatment doesn’t exist. Of course it’s “in their own interest” to participate, as their parents tries to tell them, but how do you tell if a 5 year old really wanna participate or not? What is best practice here? I’ve gottentheimpression that children may say one thing to the trial personel when the parent is inthe room, but anotherthing when they talk to e.g. the study nurse alone. Tricky situation…

    • Nadia


      June 18, 2013

      I have limited experience in Pediatric Studies but your question touches on the same ethical boundaries addressed in Jodi Picoult’s novel, My Sister’s Keeper. In the story, a child is refusing to consent to medical procedures that her parents believe are “in her best interest” and she petitions to be legally emancipated so she can make medical decisions for herself. I think back to that novel often when discussing assent because I found it so thought-provoking. You’ve rightly stated that it is tricky because there are both laws and ethics to consider when children are enrolled in clinical research. I don’t have a strong position but I thank you for this idea as a potential future blog topic and I encourage you to read the book.

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