Impact of Drug Approval Pathways for Paediatric Inflammatory Bowel Disease

Abstract

Background and aims: Timely access to approved medications is a priority in paediatric inflammatory bowel disease [IBD]. To date, the timing of drug studies in paediatric IBD has been suboptimal, with most studies conducted long after approval has been granted for adult IBD. This delay in approval leads to extensive off-label prescribing of medications in children, often without clear guidance on optimal dosing. The European Medicines Agency [EMA] and U.S. Food and Drug Administration [FDA] have implemented drug development frameworks in an attempt to address these challenges. However, access to information on these regulatory pathways in paediatric IBD is limited. We summarised the time from adult to paediatric approval of IBD therapies, outlining the regulatory approval pathway between the EMA and FDA, with the goal of identifying areas for improvement.

Methods: We reviewed publicly accessible data from the EMA and the FDA to identify therapeutic agents approved over 2005-2021 for paediatric IBD.

Results and conclusions: Five drugs are currently approved for use in the paediatric IBD population, with long interval delays after adult approval. The impact of these drug development processes in paediatric IBD is awaited. Further consideration needs to be given to the age of enrolment along with novel, more efficient trial designs in an effort to improve access for paediatric IBD patients to newer therapies.

Keywords: Clinical trials; paediatrics.