TABLE 1.
Summary of Pediatric Medication Safety Research Challenges and Innovative Solutions to Address These Challenges
| Challenge | Potential Solutions |
|---|---|
| Pediatric drug development and drug trials | |
| • Difficulty recruiting patients with disease of interest and across pediatric age continuum | • Opportunistic clinical trials that capitalize on routine care procedures |
| • Limited blood volume in neonates | • Master protocols that allow for collection of data for multiple drugs, indications, and/or biomarkers |
| • Application of pharmacokinetic/pharmacodynamic modeling and simulation methods to optimize clinical trial design | |
| • Sparse sampling and ultralow volume bioanalytical assays to facilitate blood testing | |
| Conducting CER in pediatric populations | |
| • Identifying and enrolling children who meet eligibility criteria and who are at various developmental stages | • Multisite collaborations of hospitals and health systems that agree to share EMR data |
| • EMR issues, including inconsistencies in free text data, incomplete or missing data, and limited ability to extract data across EMR vendors, and incomplete longitudinal data | • Data harmonization across EMRs that minimizes missing data (CER2) |
| Child and parent engagement on study teams | |
| • Identifying and engaging children and parents who are representative of the clinical population | • Pediatric collaborative care networks |
| • Logistical and/or scheduling issues | • Involving children and parents in the earliest stages of a study |
| • Describing research studies to parents and children | • Teleconference technologies and scheduling meetings after school or after work to accommodate family schedules |
| • Identifying ways for parents and children to contribute to all aspects of a study | • Break-out groups that allow children and parents to express their priorities/concerns separately |
| Improving communication with children and parents | |
| • Provider discomfort communicating with youth and parents | • National guidelines about youth-friendly medication communication (You’re Welcome; example from United Kingdom) |
| • Relying on parent, child, or provider reports of communication quality | • Creating youth-friendly physical spaces to enhance communication |
| • Conducting direct observations of child-parent-provider communication | |
| Assessing child-reported outcomes and ADEs | |
| • Caregivers provide proxy report of child’s health and functioning even when child is able to provide such reports | • Online libraries of validated child-report measures (Pediatric PRO-CTCAE, PROMIS) |
| • Data on what constitutes “normal” laboratory values and ADEs in youth are lacking | • Pharmacovigilance programs to standardize systems to monitor ADEs in clinical care settings |