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The Medicines and Healthcare products Regulatory Agency (MHRA) has asked for views on plans to speed up access to rare disease treatments.
A statement published by the MHRA on 21 May 2026 said its proposed ‘Rare disease therapies regulatory framework’ would incorporate a compressed regulatory pathway, which was “expected to reduce the cost of developing rare disease therapies and potentially make medications more affordable”.
“More than 3.5 million people in the UK — around one in 17 — live with a rare disease. Yet fewer than 5% of rare diseases currently have an approved treatment.
“Developing therapies for rare diseases is genuinely hard. Patient numbers are small, scientific understanding is often limited, and the financial case for investment is weak.
“The regulatory rules that govern clinical trials and drug approvals were designed around common diseases — they assume large patient populations and the kind of definitive clinical trial data that is simply impossible to generate when a condition affects approximately one in 50,000 or fewer people,” the statement added.
Conditions eligible for the proposed new framework are those where no more than 1 in 50,000 people are affected, and there are quantifiable barriers to conducting a standard clinical trial. Eligibility criteria include disease severity and unmet need.
“Central to the proposal is a new investigational marketing authorisation, which would combine clinical trial approval with a progressive route to market authorisation approval,” the statement said.
“This means that where there is limited evidence, patients could gain access to innovative treatments earlier, subject to approval by the National Institute for Health and Care Excellence (NICE) on NHS use.”
The MHRA announced its commitment to reform rare disease therapies in November 2025.
Helen Knight, director of medicines evaluation at NICE, said: “The MHRA’s proposed ‘Rare disease therapies regulatory framework’ potentially complements NICE’s approach that enables NHS patients to receive innovative and promising medicines while additional evidence is gathered on how well they work in practice.”
The proposed framework emphasises patient engagement, and people affected by rare diseases will be involved throughout the treatment development process, the MHRA said.
Former prime minister Lord Cameron, chair of the Oxford-Harrington Rare Disease Centre Advisory Council, said: “I know all too well from my own experience caring for our dear son, Ivan, that for millions of patients living with rare diseases and their families, time matters. Too often patients and their loved ones face years of uncertainty, worry and heartache, with few or no treatment options available to them.
“Today’s announcement from the MHRA is an important and hopeful step towards changing that, while helping the UK become a powerhouse of drug development for genetic and rare diseases.”
The consultation closes on 20 July 2026.
