LifeArc Centre for Acceleration of Rare Disease Trials held a webinar on innovative trial designs for rare disease research, 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.

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.

Topics Covered

The webinar included:

• An introduction to the LifeArc ARDT programme and the challenges of rare disease trials

• Efficient randomised designs and improved statistical analyses for semi-rare conditions

• Alternatives to randomised trials for very rare diseases

• Adaptive designs, including a case study from the FORMAT trial

• The augmented binary method and its impact on reducing sample size

• Platform trial structures and shared control approaches

• The use of synthetic and external controls, with examples from Primary Biliary Cholangitis

• Opportunities for embedding rare disease research within broader disease areas

• Overview of CAPTIVATE methodology projects and recent stakeholder workshops

• How to work with the ARDT Trial Design Team for design support and collaboration

Webinar Recording Now Available!