World Patient Safety Day: Safer Research for Children

Each year on September 17th, the World Health Organization recognizes World Patient Safety Day to raise awareness and promote action to reduce patient harm. This year’s theme, “Ensuring safe care for every newborn and child,” highlights the importance of protecting the youngest and most vulnerable study participants, from birth through age nine. 

In clinical research, this theme resonates deeply. Pediatric and neonatal trials play a critical role in advancing treatments, yet they present unique challenges. Children are not “small adults,” and their participation requires careful consideration of safety, ethics, and family impact. To mark World Patient Safety Day, our experts in pediatrics and neonatology share what “patient safety from the start” means in practice. 

Designing Trials with Children in Mind 

Safety must be embedded at the earliest stages of clinical research design. For adults, trial protocols may be complex but manageable. For children, especially newborns and infants, extra layers of planning are required. 

What works on paper can break down in practice. A protocol that requires multiple blood draws at precise intervals may seem feasible in theory, but it can contradict the realities of caring for a three-year-old. Study teams should continuously ask: Does this design work for the patient and their family? 

Because young children cannot advocate for themselves, researchers often must rely on input from parents, advocacy groups, and experienced clinicians. Engaging these voices early ensures that protocols reflect the needs of children, rather than simply adapting adult models. 

Minimizing Burden and Discomfort 

One recurring theme is the importance of reducing unnecessary pain and study burden. For neonates, this might mean coordinating study procedures with existing care routines, such as aligning trial-related exams with regular touch times in the NICU. It could also mean avoiding extra heel sticks by scavenging leftover blood from routine clinical lab draws whenever possible. 

Older children face different challenges. Some trial assessments require sedation (e.g., MRIs), which adds risk and anxiety for families. Others require in-depth physical assessments or long days at clinical sites, forcing parents to juggle childcare, work, and travel. By accounting for these practical realities, sponsors and CROs can design studies that are safer, more feasible, and more compassionate. 

Building Safety into Protocol Design 

Beyond logistics, trial protocols themselves must embed safety guardrails. Common strategies include: 

• Age and weight-based enrollment cohorts – starting with older or larger children before enrolling younger or more fragile patients. In neonatal studies, for example, enrollment can be stratified by birth weight—starting with larger, more stable infants and adding the smallest, most fragile neonates only after safety is confirmed. This staged approach helps ensure the highest level of protection. 

• Stepwise progression – pausing between patients and smaller cohorts to review safety data before expanding to younger or lower weight groups. 

• Healthy volunteer studies – testing new interventions in healthy children before introducing them to higher-risk populations, when appropriate. 

• Risk–benefit balance – assessing whether the potential benefits justify the risks, especially in rare diseases or life-threatening conditions. 

Consent, Assent, and Engagement 

Ethics is a cornerstone of pediatric research. Informed consent from parents (and assent from older children, when possible) is mandatory, but truly safe studies go beyond paperwork. 

Innovative tools such as illustrated storyboards or sticker calendars help children understand and track their participation. While not required, these tools engage families and can help reduce fear and empower patients to feel involved in their care. Encouraging children to speak up when something feels uncomfortable and supporting parents with clear, compassionate communication are equally important parts of safety. 

Measuring and Monitoring Safety 

In pediatric studies, collecting accurate patient-reported outcomes can be especially challenging, making other clinical and caregiver-reported measures even more critical. Safety must often be tracked through lab results, clinical exams, and parent observations. 

Investigators play a crucial role in interpreting safety signals. For very young patients, this often means relying on lab data and observation, while older children can begin to share their own perspectives. Throughout clinical trials, the insights of caregivers remain essential. Ongoing monitoring, frequent check-ins, and responsive adjustments are critical to keeping children safe throughout a trial. 

The Role of Families and Advocacy Groups 

Families are not just participants; they are partners. Including parents in the design phase helps sponsors anticipate real-world challenges, from travel demands to emotional stress. Advocacy groups can amplify these perspectives, ensuring that trial designs reflect the lived experiences of families. 

Pediatric research presents unique considerations that may not be immediately apparent when compared to adult studies. Advocacy groups help bridge this gap by highlighting the unique burdens families face, ensuring those perspectives are built into trial design. This collaboration builds trust and ensures studies are both scientifically rigorous and ethically sound. 

Balancing Risks and Benefits 

Every clinical trial involves risk, but in pediatrics the calculus is even more complex. Sometimes, risks may be justified, such as in life-threatening neonatal conditions with no effective treatments. Other times, proposed interventions may carry more risk than benefit, especially if safer alternatives exist. 

In practice, certain procedures, like central lines or invasive monitoring, may be deemed unnecessarily risky given the study design. In such cases, experience and judgment are critical to protecting children from harm. Ultimately, safety in pediatric research requires constant evaluation of the risk–benefit ratio, a balance that evolves as new therapies and data emerge. 

Collaboration Across the Ecosystem 

Ensuring patient safety in pediatric trials is not the responsibility of any single stakeholder. Sponsors, CROs, regulators, investigators, advocacy groups, and families must work together. Independent oversight bodies like Institutional Review Boards (IRBs) and Ethic Committees provide additional safeguards, scrutinizing protocols to ensure risks are minimized. Experience also matters. Collaborating with teams who have managed pediatric and neonatal trials before can help avoid common pitfalls. 

Advancing Pediatric Safety on World Patient Safety Day 

This year’s theme, “patient safety from the start,” is more than a slogan. It is a reminder that protecting children in research requires thoughtful design, compassionate engagement, and collaboration at every stage. 

From reducing unnecessary procedures to involving families as true partners, each step toward safer pediatric trials helps build a future where innovative therapies can be developed without compromising the well-being of our youngest patients. 

On World Patient Safety Day, we celebrate the progress made and reaffirm our commitment to ensuring safe, ethical, and effective care for every child, from the very start. To discuss your pediatric study needs, contact us. 

ABOUT PREMIER RESEARCH:  

Premier Research, a global clinical research, product development, and consulting company, is dedicated to helping innovators transform life-changing ideas and breakthrough science into new medical treatments. We offer strategic solutions across the entire development life cycle, from pre-clinical through commercialization, specializing in smart study design and full-service clinical trial management.     

Leveraging technology and therapeutic expertise, we deliver clean, conclusive data with a focus on reducing development timelines, securing access to the right patients, and effectively navigating global regulations to ensure submission-ready results.     

As an organization that puts patients first, we pride ourselves on helping customers answer the unmet needs of patients across a broad range of medical conditions. Visit premier-research.com.