The Medicines & Healthcare products Regulatory Agency (MHRA) have announced today that they are launching a public consultation on the new Rare Disease Therapies Regulatory Framework.
Respond with your views on the consultation by 30th July!
With 3.5 million people living in the UK with a rare disease (when combined with carers, this equates to more than the population of London) and only 5% of these rare diseases currently having approved treatment, this framework is huge news for the rare disease community.
Many patients and families with rare diseases experience years of uncertainty, a long and often difficult journey to diagnosis, with limited options once a condition is identified.
The current system for developing and approving medicines was not built with rare diseases in mind. Traditional approaches rely on large clinical trials and extensive datasets; something that is often impossible when patient populations are small, geographically dispersed, and sometimes not yet fully understood.
Currently, the average journey for reaching a diagnosis for a rare disease is 5.6 years, with significant delays and barriers in receiving treatment.
The new framework aims to address these unique challenges for rare diseases through multiple methods, including streamlining clinical trials.
While traditional rare disease programmes usually take 10 – 12 years to reach approval, this framework hopes to reduce these timelines, bringing therapies to patients faster.
Our CEO, Rick Thompson, said:
There are millions of people in the UK living with a rare condition, struggling to secure research or treatment. This new regulatory framework has the chance to be transformative for rare diseases – inspiring more research and ensuring that more therapies have the chance to reach patients who need them.
I encourage all stakeholders to engage with the MHRA consultation. Together, we can help to ensure these transformative ideas are implemented in a manner that works for developers and underscores their importance to those affected by rare diseases.
The framework is designed for therapies treating rare diseases, which occur in around 1 in 50,000 or fewer in the UK, where there are ‘clear and measurable barriers to conventional development’.
The new proposed framework has been developed so far by the Rare Disease Consortium, which has now opened up the process for wider input.
All contributions will help the UK form a world-leading framework for rare disease therapies.
We strongly encourage patients, families, professionals and organisations across the sector to take part.
Your views will help shape how rare disease therapies are regulated in the UK for years to come.
Respond with your views on the consultation here by 30th July and learn more about the Rare Disease Therapies Framework here!
Season 3, Episode 4: Shifting sands – the evolving world of rare disease policy
For more context on rare disease policy, have a listen to the latest episode of our podcast, Rarely Heard!
In this episode, you can hear from a panel of experts from Genetic Alliance UK, LifeArc, MHRA and the UK BioIndustry Association (BIA), who sat down with our CEO, Rick Thompson, to discuss UK rare disease policy in 2026.
MHRA’s Executive Director, Julian Beach, speaks about the new framework and why it demonstrates genuine regulatory innovation.
He highlights how the framework will uniquely position the UK within the rare disease landscape.
Listen to the podcast to learn more about this exciting new framework now!
