MHRA signals new UK rulebook for rare therapies in 2026

Britain’s medicines regulator is changing how rare disease treatments move from lab bench to bedside. On 2 November 2025 the MHRA published a paper signalling a new rulebook to make testing, manufacturing and approvals quicker while keeping safety central. If you teach or study health policy, this is a live case study in how regulation keeps pace with science.

Why it matters is simple and human. One in 17 people in the UK-about 3.5 million-lives with a rare condition, but fewer than 5% of rare diseases have an approved treatment. The MHRA also points to long diagnostic journeys (on average 5.6 years), stark early childhood mortality, and heavy costs to families, the NHS and the wider economy, running into billions each year.

What might change under the new framework? The MHRA is exploring an early, single decision that could both start a clinical trial and allow tightly controlled patient use when initial evidence is compelling. That access would come with strict safety plans, scheduled evidence reviews and the power to adjust or withdraw approval as real‑world results come in. Consultation opens in early 2026 with the aim of an implementable model by late 2026.

Quick explainer: real‑world evidence means data from everyday care-clinic notes, registries and follow‑up visits-rather than only traditional randomised trials. The MHRA’s paper explicitly pairs any earlier access with tougher post‑market surveillance, so side‑effects are caught early and benefits are confirmed over time. Think of it as “approve, watch closely, and update the decision as the facts grow”.

Science catch‑up for the classroom: newer tools such as CRISPR and mRNA allow developers to design medicines for very small groups-even a single person carrying a specific genetic change. In plain terms, CRISPR is like a precise find‑and‑replace for DNA, while mRNA gives cells instructions to make a missing or helpful protein. That level of tailoring is why regulators need more flexible pathways for ‘n‑of‑1’ treatments.

This rare‑therapy push sits alongside wider UK research reforms. Parliament signed off new Clinical Trials Regulations in April 2025; after a 12‑month rollout and a technical re‑sign, they take full effect on 28 April 2026. The direction is faster studies with proportionate oversight-good news for rare disease research where every month counts.

You can already see the system speeding up. In October 2025, the MHRA reported average UK clinical trial approval times falling from 91 to 41 days, helped by digital platforms and process changes. Faster starts make it easier to run studies when patient numbers are tiny.

What this could look like for families: if a gene therapy for a very small group shows early promise, a team could begin a trial and, under a single early decision, offer supervised NHS use at the same time. You would receive clear information, regular safety checks and updates as evidence grows so choices are informed and revisited.

Safety remains the red line. The MHRA stresses that any earlier access would be time‑limited and tracked closely, with the ability to tighten, extend or stop use as data changes. The agency’s 2025 designation as a WHO‑Listed Authority underlines its international credibility-useful when sharing evidence and coordinating with other regulators.

Funding and fairness questions sit alongside regulation. The England Rare Diseases Action Plan trails a national operating framework for highly personalised ‘n‑of‑1’ therapies, including how to judge value and affordability and who might be eligible. Expect the MHRA’s rulebook to mesh with NICE’s evidence standards and NHS England’s commissioning decisions.

Joining up with the world: the UK’s International Recognition Procedure lets the MHRA consider trusted decisions from partner regulators where appropriate. For rare diseases, pooling small datasets across borders is essential-and the new paper flags plans to improve UK‑global evidence sharing to make that practical and safe.

Timeline to note for your revision: the MHRA paper and press release landed on 2 November 2025; public consultation is due early 2026; the refined model is targeted for late 2026; and the new UK Clinical Trials Regulations complete in April 2026. In short: policy in motion-watch this space through 2026.