We welcome the recent release of a new paper setting out the Medicines and Healthcare products Regulatory Agency’s (MHRA) intention to overhaul the rules for rare disease treatments in the UK.
Key takeaways from the announcement include:
- Reform of regulations for rare disease treatments to speed up access and innovation, with less than 5% of rare diseases currently having approved therapies.
- A new framework aimed at removing barriers, such as small patient populations, limited trial data and low commercial incentives, which MHRA says have slowed development and approval.
- The planned adoption of a combined approval process for clinical trials and marketing, enhanced monitoring and a stronger use of empirical evidence.
- The new plan is to be in conjunction with the Government’s Rare Disease Action Plan, which relies on using UK leadership on areas such as genomics, NHS data and research.
- The scheme aims to turn the UK into a global hub for rare-disease treatment.
- Plans for the creation of a new body called the Rare Disease Consortium involving patients, industry, academia and regulators.
Sue Farrington, Chief Executive, Scleroderma & Raynaud’s UK (SRUK) said:
“The MHRA’s plans to modernise the regulatory framework for rare disease treatments is a welcome update. For too long, people living with rare and complex conditions such as scleroderma have faced unacceptable delays in diagnosis and in accessing effective therapies. This review is a positive step towards addressing those challenges and creating a more responsive system that works for patients.
“The proposed changes, particularly the move towards earlier approval pathways and greater use of real-world evidence, could make a tangible difference in getting promising treatments to those who need them most. It will be vital, however, that patients and charities, like SRUK, remain closely involved in shaping how these reforms are implemented. Their invaluable experience provides essential insight into what truly matters in treatment development and access.
“We look forward to working with the MHRA and the new Rare Disease Consortium to help turn this ambition into practical progress.”