Paediatric drugsJournal Article
10 May 2025
The evidence to support the efficacy and safety of pharmacological treatments for chronic non-cancer pain in children is limited. In practice, clinicians are often required to establish therapeutic plans using data extrapolated from adult studies, which may not apply to younger patients.
Recent systematic reviews and meta-analyses indicate minimal evidence of benefit for these treatments in children; however, the low quality of studies included in these reviews complicates the conclusions that can be derived from them.
In this article, we focus on safety, an outcome as critical as efficacy in clinical trial design but often designated as secondary or even exploratory.
Specifically, we examine methods for assessing adverse events in clinical research and propose a practical approach for evaluating these events in everyday practice.
Additionally, we outline our strategy to conduct a risk-benefit analysis at the individual patient level, highlighting the importance of using a composite risk-benefit metric rather than assessing these outcomes separately.
This approach enables real-time monitoring of both drug-related symptom relief and adverse effects, facilitating clinically meaningful risk-benefit discussions with patients and their families.
Finally, we advocate for improvements in clinical trial design for pediatric chronic pain treatments, particularly around adverse events. Future trials should incorporate standardized definitions, comprehensive risk-benefit evaluations, and transparent outcome reporting.
Implementing these changes may enhance decision-making by balancing the safety and the effectiveness of pharmacological treatments for children and adolescents with chronic pain.
Declarations. Conflicts of Interest: Dominique Dundaru-Bandi, Kacper Niburski, Rebecca Pitt, Nada Mohamed, Victor Hugo Gonzalez Cardenas, Lisa M. Einhorn, and Pablo Ingelmo have no conflicts of interest that might be relevant to the contents of this manuscript. Author Contributions: Dominique Dundaru-Bandi participated in the writing of the manuscript. Kacper Niburski, Rebecca Pitt, and Nada Mohamed contributed to the conceptualization and data analysis. Victor Hugo Gonzalez Cardenas was responsible for data management. Lisa M. Einhorn and Pablo Ingelmo contributed to the conceptualization, writing, and data analysis. All authors read and approved the final version of the manuscript and fulfilled the criteria for authorship. Data Availability Statement: Data sharing is not applicable to this article as no datasets were generated or analyzed during the current study. Ethics and Consent: Not applicable. Code Availability: Not applicable. Funding: The research and education activities of Dr. Ingelmo are supported by grants of the Montreal Children’s Hospital Foundation and by the Louise and Alan Edwards Foundation.
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