Editorial: Distinct Challenges of Paediatric From Adult-Onset Inflammatory Bowel Disease, Implications of Aggressive Disease Phenotypes and Early Biologic Therapy in Children—Authors' Reply

In their editorial, Drs. Molinaro and de Ridder discussed the challenges and implications of managing paediatric IBD (pIBD) [1], in light of our publication [2]. On one hand, there is an extensive disease phenotype in pIBD that, if left untreated, can impact a crucial period of growth. On the other hand, in real life, there is higher exposure to advanced and new therapies, some of which are being used off-label, shortly after those have been approved in adults. The growing use of off-label therapies in clinical practice in pIBD can offer much needed treatment options for children with refractory disease. Still, it also presents challenges regarding dosing, safety monitoring, and long-term effects [3, 4]. The use of off-label biologics in pIBD highlights the urgent need for more paediatric-specific clinical trials and expedited approval processes for these medications in children.

As highlighted by Croft et al. in their multi-stakeholder perspective on improving drug development for children and adolescents with IBD [5] and by Altepeter et al. [6], it is imperative to incorporate paediatric studies earlier in the drug development process and advocate for earlier inclusion of paediatric patients in clinical trials. In addition, these summaries suggested using real-world data to characterise the safety profiles of drugs in paediatric patients. This approach can complement traditional clinical trials and potentially accelerate the approval process for paediatric indications. Their summaries further emphasise the need for a collaborative approach involving clinicians, researchers, pharmaceutical companies and regulatory bodies to expedite the development and approval of new therapies for pIBD.

We can envision, for example, the inclusion of at least some of the paediatric population (above 12 or 14 years) at the initial stage, similar to the approach taken by recently approved treatments for eosinophilic oesophagitis. For instance, the initial study design for dupilumab involved both adults and children aged 12 and older [7], and it was subsequently approved by the FDA for use in both age groups. Similarly, future studies on IBD could be designed to include at least some of the paediatric population in the initial stages.

In conclusion, we thank the writers for their insightful commentary. Moving forward, we hope for more controlled studies and real-world evidence, as well as collaborative efforts, to contribute to improving the timely and safe treatment of children with IBD which will ultimately enhance their quality of life and long-term outcomes.

Maya Granot: conceptualization, writing – original draft, writing – review and editing. Batia Weiss: conceptualization, writing – original draft, writing – review and editing. Yael Haberman: conceptualization, writing – original draft, writing – review and editing.

This article is linked to Granot et al papers. To view these articles, visit https://doi.org/10.1111/apt.18264 and https://doi.org/10.1111/apt.18299.