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Impact of Drug Approval Pathways for Paediatric Inflammatory Bowel Disease
Eileen Crowley1,2, Christopher Ma2,3,4, Marija Andic2
1Department of Pediatrics, Division of Pediatric Gastroenterology & Hepatology, Children's Hospital Western Ontario, Western University, London, ON, Canada.
Insights
Access to new medications for pediatric inflammatory bowel disease (IBD) is delayed, with five drugs approved years after adult use. This necessitates improved trial designs and enrollment strategies for faster access to therapies.
Area of Science:
- Pediatric gastroenterology
- Drug development and regulation
- Inflammatory Bowel Disease (IBD) research
Background:
- Timely access to medications is crucial for pediatric inflammatory bowel disease (IBD) patients.
- Current drug development timelines for pediatric IBD are suboptimal, leading to significant delays after adult drug approval.
- This results in widespread off-label prescribing without clear dosing guidelines for children.
Purpose of the Study:
- To summarize the time lag between adult and pediatric approval for IBD therapies.
- To outline the regulatory approval pathways of the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) for pediatric IBD drugs.
- To identify areas for improvement in drug approval processes for pediatric IBD.
Main Methods:
- A review of publicly available data from the EMA and FDA.
- Identification of therapeutic agents approved for pediatric IBD between 2005 and 2021.
Main Results:
- Only five drugs are currently approved for pediatric IBD.
- There are substantial delays between adult and pediatric drug approvals for IBD therapies.
- The full impact of current drug development processes on pediatric IBD outcomes remains to be seen.
Conclusions:
- Further improvements are needed in drug development frameworks for pediatric IBD.
- Consideration of patient age in trial enrollment and the adoption of novel, efficient trial designs are essential.
- These changes aim to enhance access to newer therapies for pediatric IBD patients.
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.
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