Precision Medicine In Pediatrics: Biomarkers and Assay Development

By Eva Raschke

Over the past two decades, there has been a remarkable increase in the availability of therapies explicitly approved by the FDA to treat conditions in children. Notably, the proportion of approved cancer therapies with pediatric indications more than doubled between 2017 and 2021 compared to the previous five years. Despite these advancements, there remains a significant unmet need for additional safe and effective medicines for the prevention and treatment of diseases affecting children. Precision medicine, which involves tailoring medical treatment to the individual characteristics of each patient based on their genetic or biomarker profiles, aims to improve efficacy and limit toxicity when a therapy is unlikely to be beneficial. This approach is particularly crucial in pediatrics because children are not simply "small adults." They often exhibit different disease symptoms, responses to medication, and recovery patterns compared to adults.

In this article, we delve into the potential of precision medicine for pediatrics to emphasize the importance of biomarkers and assay development. Explore how these tools can help identify the most effective treatments for children, reduce adverse effects, and ultimately improve health outcomes for our youngest patients.