Innovative approaches to investigator-initiated, multicentre paediatric clinical trials in Canada

Area of Science:

• Pediatric Clinical Trials
• Drug Development
• Health Services Research

Background:

• Clinical trial data is essential for safe and effective pediatric medicine use.
• Conducting pediatric clinical trials presents unique design and implementation challenges.
• Established pediatric clinical trial infrastructure exists in the UK, USA, and Europe.

Purpose of the Study:

• To describe partnerships and operational considerations for developing pediatric clinical trial infrastructure in Canada.
• To detail the design and execution of four parallel, multi-province emergency room pediatric trials.
• To identify lessons learned and solutions for facilitating pediatric drug development in Canada.

Main Methods:

• Conducted four emergency room pediatric trials across four Canadian provinces with separate sponsors.
• Implemented multisite contract development and centralized, risk-based data monitoring.
• Focused on ethical review processes and patient engagement strategies.

Main Results:

• Successfully executed parallel, multi-site pediatric trials demonstrating feasibility.
• Identified critical operational elements including contract management, data monitoring, ethical review, and patient engagement.
• Gathered insights into challenges and potential solutions for Canadian pediatric drug development.

Conclusions:

• Establishing robust pediatric clinical trial infrastructure in Canada requires strategic partnerships and operational planning.
• Addressing challenges in multisite coordination, ethical review, and patient engagement is key to successful pediatric drug development.
• The described model offers a framework for future pediatric clinical trials in Canada, improving access to essential medicines for children.