S3, E4. Shifting sands: the evolving world of rare disease policy

Welcome to Episode 4 of Season 3 of Rarely Heard.

2026 is a big year for rare diseases in the UK. The extension of the rare disease framework to February 2027 has created a window of opportunity for the community to make the case for a renewed and revitalised piece of policy for rare disease.

This comes at the time when the government has published its ten-year plan for Health in England, when the national medicines regulator, the MHRA, is creating new pathways for rare disease medicines, and when NICE have reviewed cost effectiveness thresholds. Alongside this, the UK has seen a number of new rare disease research networks, designed to accelerate the development of new rare disease therapies. All of this creates an exciting moment in the rare ecosystem, where the UK could position itself as a leader in rare disease research and care, to the benefit of all those people living with a rare disease.

In this episode, Beacon CEO and a panel of experts from Genetic Alliance UK, LifeArc, the MHRA and the UK Bioindustry Association discuss UK rare disease policy in 2026, and the opportunities for the whole rare disease community.

Please listen below!