Clinical Research: Phase 1 - Phase 4

The Top 5 Concerns for Pediatric Study Participants

Patient and parent engagement is crucial for successful pediatric drug trials. That’s why the best strategies are ones that encourage proactive involvement beginning at the early stages of protocol development through study conduct and eventual results-sharing. And that includes input.

We partnered with iCAN in 2015 to ask pediatric patients, their family members, and even their friends for feedback on an upcoming flu treatment trial. These were the five biggest concerns they had, along with our suggestions on how to address them.

1. Incomplete understanding.

Many patients and parents weren’t sure as to which study procedures were to be carried out and what they involved. The chance of being assigned a placebo was a particular source of confusion.


  • Better consenting and assenting tools to present study information in a more patient- and parent-friendly way. Depending on demographics and local regulatory requirements, these may include flip boards, cartoons or videos.
  • A doll as a visual aid to explain procedures to younger patients.
  • Consent workshops to help staff without previous pediatric clinical research experience.

2. Anxiety about meeting different people for different procedures.

The number of clinicians, assistants, and technicians involved in a clinical trial can be overwhelming, especially to young children.


  • Provide young patients with a booklet for them to fill out that includes information about themselves and their families. This tool gives children an easy way to introduce themselves to each new person.

3. Having to bring the study drug to school.

Most students these days are prohibited from carrying medication with them, so even older teens must work with a school nurse while participating in clinical trials. This can be considered a hassle for both patients and parents that’s large enough for them to balk at enrollment.


  • Study drugs that are easy to distribute and packaged to allow for bringing appropriate doses to school.
  • Include instructions suitable for a school nurse and a process for documenting this training.
  • Consider a dosing schedule that minimizes the amount of drug needed to be taken during the school day.

4. Boredom during and between study procedures.

This applies to both patients and any siblings who may be accompanying them. Of course, parents aren’t exempt from boredom, either.


  • Provide entertainment suitable for multiple ages, such as video games, books, or movies.
  • Schedule assessments with minimal waiting.
  • Explain possible waits to adults beforehand so that they come prepared.

5. The impact of study participation on the patient’s academic performance, hobbies and social life.

Much of the strongest survey feedback came down to this one overarching concern. Making sure patients understand possible effects ahead of time is a major factor for patient retention.


  • Design protocols that take the patient and family’s normal life into account.
  • Discuss any potential impacts during the consenting process. Minimize the possibility of surprises that can cause patient dropout.

By addressing these concerns during study design, sponsors can help ensure success for the future of pediatric drug development. Read more about involving kids in clinical research in our white paper.