More Than You Can Imagine: The Unseen Work That Gets Rare Disease Medicines to Patients

By building tailored partnerships with innovators and drawing on deep local expertise, a new European model is unlocking the potential of rare disease medicines and reaching patients across all 32 countries. Across Europe, patients with rare diseases are being let down,” admits Adam Plich, and it isn’t due to a lack of pharma innovation. The … Continued

The socioeconomic burden of a rare disease in the UK is ~£70,000 per patient per year ;1 it’s critical to acknowledge this cost to recognise the full value of new treatments. In 2025, we reviewed published evidence for ten rare conditions and estimated that the socioeconomic burden of a rare condition in the UK is … Continued

Rare conditions affect 1 in 17 people in the UK. We need an equitable healthcare system to meet their needs. Scarcity of rare Genetic Alliance UK’s Equity for Rare report, launched on Rare Disease Day 2026, highlights that small patient populations are an immutable characteristic of these conditions. This leads to systemic challenges: low priority … Continued