Thank you to everyone who joined our webinar on innovative trial designs for rare disease research, held on Tuesday 9 December 2025. The session explored practical and methodological approaches that can support robust, efficient, and patient-centred trials in settings where participant numbers are limited.
Our speakers — James Wason and Victoria Homer — discussed a range of methodological innovations, including adaptive designs, synthetic and external controls, and approaches tailored to both semi-rare and very rare conditions. They also introduced current work within the ARDT Trial Design Team and the CAPTIVATE node, covering ongoing methodology projects and opportunities for researchers to collaborate with the team.
Watch the Recording
The full recording of the webinar is now available to view:
Topics Covered
The webinar included:
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An introduction to the LifeArc ARDT programme and the challenges of rare disease trials
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Efficient randomised designs and improved statistical analyses for semi-rare conditions
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Alternatives to randomised trials for very rare diseases
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Adaptive designs, including a case study from the FORMAT trial
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The augmented binary method and its impact on reducing sample size
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Platform trial structures and shared control approaches
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The use of synthetic and external controls, with examples from Primary Biliary Cholangitis
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Opportunities for embedding rare disease research within broader disease areas
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Overview of CAPTIVATE methodology projects and recent stakeholder workshops
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How to work with the ARDT Trial Design Team for design support and collaboratio
Contact Us
If you would like to discuss design support, explore collaboration opportunities, or submit a trial proposal, please contact us at:
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centreardt@contacts.bham.ac.uk
