How to Run Effective Pediatric Clinical Trials

Pediatric clinical trials play a critical role in ensuring that medications, supplements, and treatments are safe and effective for children. Despite this, they remain one of the most underrepresented segments of clinical research. In fact, 50–90% of medicines prescribed to children are ‘off-label’, meaning they haven't been officially tested for pediatric use. This gap in research raises significant concerns about safety, efficacy, and trust.

As the industry works toward reducing global child mortality and improving pediatric healthcare outcomes, it's clear that we must better understand and address the challenges of pediatric clinical trials.

The Complex Challenges of Pediatric Clinical Trials

Running clinical trials involving children is no easy task. Researchers face a unique set of hurdles that can slow or stall studies before they even begin.

1. Ethical and Regulatory Barriers

Children are a vulnerable population, which means clinical studies involving them are held to higher ethical standards. In the U.S., researchers must submit a Pediatric Study Plan (PSP) under the Pediatric Research Equity Act. Similarly, studies in the EU require a Paediatric Investigation Plan (PIP) submitted to the European Medicines Agency. These documents are designed to ensure that every pediatric subpopulation is considered in terms of safety, dosing, and effectiveness.

One common mistake is assuming that a treatment approved for adults is automatically safe for children. It’s not. Pediatric clinical trials require independent validation, making proper planning and compliance essential.

2. Recruitment and Retention Difficulties

Patient recruitment remains one of the biggest challenges in pediatric trials. Parents and guardians often express:

• Fear of harm to the child

• Reluctance due to a lack of immediate benefit

• Discomfort with complex consent procedures

• Mistrust in the clinical trial process

On top of that, the pediatric population is generally smaller and healthier, further narrowing the pool of eligible participants.

Building Trust: Engaging Parents and Guardians

For pediatric clinical trials to succeed, trust must be at the center of every recruitment strategy. One of the primary reasons parents hesitate to enroll their children in studies is a general skepticism toward modern medicine and a lack of awareness about how trials work.

Meet Parents Where They Are

Rather than relying solely on traditional outreach, research sites should form partnerships with local clinics, schools, and nurseries. Building relationships in these community settings helps parents become familiar with the concept of clinical trials in a non-intimidating way.

Making Pediatric Clinical Trials More Accessible

In the digital age, participant engagement must evolve. If in-person events and community meetings are limited, there are still highly effective ways to reach parents and build trust.

1. Leverage Social Media

Social media is a powerful tool to raise awareness and connect with families. Platforms like Facebook, Instagram, and Twitter can help research sites humanize their message, share success stories, and promote ongoing studies in relatable language. With over 5 billion users on social media, the potential reach is enormous.

2. Create Digital Educational Resources

Providing accessible information can break down the fear factor. Visual aids, infographics, and FAQ pages, especially those tailored for parents, can be incredibly effective. Look to examples like the National Heart, Lung, and Blood Institute, which provides easy-to-digest materials for both adults and children.

3. Use Video Call Technology

With platforms like Zoom and Teams now ubiquitous, virtual telehealth meetings offer a convenient, personal way to engage with parents. These tools allow for real-time conversations, giving guardians a chance to ask questions and voice concerns directly, without leaving their homes.

Crafting a Human-Centered Recruitment Strategy

When recruiting participants for pediatric clinical trials, a data-driven strategy must also be deeply empathetic. Research shows that people respond more strongly to emotional appeals, especially when a cause hits close to home. Highlighting real stories, testimonials, and personal experiences can help shift perception and foster a sense of shared purpose.

Recruiters should aim to humanize the clinical trial experience, showing parents how their participation contributes to broader pediatric health outcomes, not just today, but for future generations.

The Future of Pediatric Clinical Trials

While the challenges of pediatric clinical trials are many, ethical, logistical, and emotional, they are not insurmountable. With the right planning, transparent communication, and a people-first approach, it’s possible to run ethical, effective studies that help close the gap in pediatric research.

For the health and safety of our children, this work isn’t just important, it’s essential.

Curious how we can help your brand run a pediatric clinical trial? Contact us today to learn more.