N-of-1 Hub

Generalisability is one of the most frequently cited concerns in discussions of N of 1 trials. Critics argue that findings from individual-level experiments cannot meaningfully inform practice beyond the person studied, even when trials use standardised protocols and are analysed in aggregate (pooled N of 1 trials). This concern is often treated as self-evident. Yet it prompts a critical question: why is generalisability considered a central limitation of N of 1 designs, while it is...

Study Design A new study by Victoria Defelippe and colleagues demonstrates that continuous Bayesian outcome estimation in N of 1 trials can accelerate clinical decision-making while maintaining statistical rigor. The research examined whether treatment effects can be evaluated before the pre-specified end of a trial, potentially reducing trial duration and patient burden without sacrificing certainty. The study combined two approaches: (1) simulation of an N of 1 trial in severe epilepsy, and...

More than 3.5 million people in the UK live with a rare disease (1), yet care remains fragmented and access to targeted therapy is still limited. For health professionals, this creates significant challenges in clinical management and patient support. The Rare Therapies Launch Pad (RTLP), established in 2023, is the UK’s response to this unmet need, providing a structured pathway for developing and delivering highly individualised therapies for ultra-rare and life-limiting conditions....