N-of-1 Hub

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....

What is the main takeaway? This article in the British Journal of General Practice examines the pharmacological factors that determine whether an N-of-1 trial can realistically be carried out in primary care. Diezi et al. identify seven key factors: onset of action, washout, titration rate, withdrawal or rebound potential, reversibility of effect, identifiability, and galenic formulation (how the medicine is made and delivered, for example tablet vs capsule or slow release). Through clinical...

As the demand for personalised medicine grows, there is increasing interest in methods that prioritise individual patient responses over group-level findings. Traditional clinical trials remain essential for evaluating broad treatment effects, but they often fail to capture how an intervention works for a specific patient over time. This is where N-of-1 trials come in, allowing for repeated within-person measurements to assess patterns in symptoms, behaviours, and treatment effects. However,...