Five Strategies to Tailor Your Pediatric Clinical Trials to the Child’s Needs

Implementing these approaches can keep pediatric study design from becoming an obstacle for drug developers.

When participating in clinical trials, pediatric populations require distinct considerations for their safety and well-being — which can present unique challenges for drug developers. Unlike trials focused on adult populations, pediatric trial design requires additional safeguards to:

• Enable special protections for the safety of this population
• Account for the normal growth and development that children will undergo throughout the study
• Understand the patient and caregiver experience as they progress from recruitment to trial completion

Addressing these challenges upfront to ensure clinical trials are tailored to meet the specific needs of children doesn’t have to be another obstacle for drug developers. A number of strategies can be adopted to put the needs of the child at the center of the study. Partnering with a contract research organization (CRO) that has deep experience in pediatric clinical trials enables drug developers to successfully implement these strategies, which also have the potential to enhance study startup time, ease the burden on pediatric patients and their caregivers, and ultimately improve retention rates.

Five strategies to tailor your pediatric clinical trials to the child’s needs

1. Cultivate a deep understanding of the nuances of pediatric populations

Pediatric populations encompass various age groups, each with its own characteristics and preferences. The U.S. Food and Drug Administration defines these groups as:

• Neonates: newborns up to 1 month
• Infants: 1 month–2 years
• Children: 2–12 years
• Adolescents: 12–16 years

Pediatric clinical trials must account for different age groups and developmental stages, as children’s physiology and response to treatments can vary significantly. Designing appropriate dosing, assessments and outcome measures for different age groups can be complex, which is why it is crucial to understand the physiological and developmental differences among them.

While regulatory guidelines often provide specific recommendations for each age group to ensure the ethical conduct of pediatric clinical trials, incorporating patient preferences is another avenue for customizing the study. For example, different pediatric groups may have different preferences for treatment options — where one may prefer a liquid over pills, others may prefer an orally disintegrating tablet.

By having a firm grasp of the characteristics of a study’s target pediatric population, elements of the trial can be tailored accordingly.

2. Engage patients and caregivers early in the study design

Involving pediatric patients and their caregivers in the study design process is crucial — and the earlier, the better. A patient-centered approach ensures that participants’ needs, values and priorities are considered when designing the trial protocol, interventions and assessments.

In terms of trial outcomes, the patient and caregiver perspective may differ from the clinical perspective, and it’s critical to understand those differences from the start. It’s important to ensure the treatments are safe and effective, while also aligning with what outcomes are important to the patients and their caregivers.

Seeking their input early in the process and understanding their perspectives enables drug developers to ensure that the trial design aligns with the needs and priorities of the children and their caregivers.

3. Leverage decentralized solutions

Decentralized trial elements, such as home health visits and the use of digital tools and wearables, can minimize patient burden and improve retention rates. These solutions offer increased convenience and flexibility, allowing pediatric patients to participate in the trial from the comfort of their own homes.

Beyond accommodating patients with limited mobility or those who live long distances from study sites, an added benefit of decentralized trial solutions is the potential for less disruption in the patient’s day-to-day activities. Often, pediatric patients don’t want to be seen as different from their peers, and incorporating decentralized elements like wearables may enable them to participate in more inconspicuous ways.

4. Ensure sites are suited for pediatric populations

Staff at clinical trial sites should be experienced and adept at interacting with families and performing procedures on children. Creating a welcoming atmosphere can go a long way in alleviating anxiety and fostering a positive experience for pediatric participants. This can range from having appropriately sized furniture for children, to toys for waiting siblings to play with, to specially designed comics to impart study information.

Scheduling visits strategically is also key to keeping the patients’ needs at the forefront of the study. The visit’s order of operations matters to a pediatric patient. Experienced sites know that if a blood draw is required, it may be best to reserve time for that later in the visit, to prevent the patient from being upset from the start. Conversely, if a cognitive assessment is part of a site visit, it’s best to schedule that early in the appointment to avoid end-of-visit fatigue.

5. Communicate frequently and transparently

Establishing and maintaining trust with pediatric patients and their caregivers is essential to study success. Transparent and age-appropriate communication builds necessary trust and ensures that patients and caregivers remain well-informed throughout the trial.

When patients and caregivers are clear on what is expected of their participation and are knowledgeable about the types of procedures and assessments they’ll take part in, they may be more cooperative and satisfied with their overall experience.

And building trust goes both ways. With open lines of communication, patients and caregivers may be more proactive about reporting any changes in health or well-being and asking questions that may arise throughout the study.

A wealth of pediatric clinical trial experience

When enacting strategies to tailor pediatric clinical trials to the child’s needs, experience matters. The PPD clinical research business of Thermo Fisher Scientific has demonstrated its expertise in taking extraordinary measures to improve the lives of pediatric patients. Our Rare Disease and Pediatric Center of Excellence provides deep pediatric knowledge and experience to drug developers across all therapeutic areas. More than 60 dedicated experts in pediatrics — including 28 board-certified pediatricians and 28 statisticians with pediatric specialization — come together with our partners to ensure that pediatric clinical trials are executed with the highest quality and with the patient top of mind.

Our pediatric experts are empowered to optimize trials based on past experience, leading to studies that are more efficient, cost effective and patient centric.