Rare therapies and UK regulatory considerations
A paper outlining MHRA’s intentions to make it quicker and easier to get rare disease therapies tested, manufactured and approved in the UK.
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The UK is well-positioned to become a global leader in developing, regulating, and integrating rare therapies into national healthcare pathways. This position paper outlines the current approach by UK regulators and partners to design a forward-looking framework that meets the needs of patients with rare diseases, while ensuring robust evidence generation, system sustainability, and international convergence.
Consultation on this framework will begin in early 2026, with the objective of establishing a refined and implementable model by late 2026.
This work would not have been possible without the support of the Rare Disease Consortium, which is made up of:
- Government / regulators: MHRA, NICE, DHSC, NHS England
- Patient and advocacy groups: Genetic Alliance UK, Beacon, Unique, Mila’s Miracle Foundation
- Academia and research: University of Oxford, Newcastle University (Rare Diseases Research UK), Great Ormond Street Hospital, UC Berkeley (Innovative Genomics Institute)
- Industry: LifeArc, Catapult Cell and Gene Therapy, AstraZeneca (Alexion), Biogen, Alnylam, Ipsen, Mereo BioPharma, BIA, ABPI, Weatherden, Vertex, BioMarin, Syncona, UCB.
Additional contributors include the Rare Therapies Launchpad.