(London, 3rd) The UK government will undertake a major reform of the regulatory framework for rare disease treatments, aiming to accelerate the entire chain from research and development to patient access. This initiative is a key part of the UK's strategy to advance the life sciences sector.
The UK Medicines and Healthcare products Regulatory Agency (MHRA) stated on the 2nd that there are currently around 3.5 million rare disease patients in the UK, but less than 5% of rare diseases have approved treatment options. On average, it takes 5.6 years for patients to be diagnosed, and 30% of affected children die before the age of 5.
The MHRA said that rare diseases not only place a heavy burden on patients and their families, but also cause an estimated annual economic loss of about £14.9 billion (approximately RM81.955 billion) to the UK economy.
In response, the UK government will lead the establishment of a new body—the Rare Disease Treatments Alliance. Members of this Alliance will include patients and their representatives, as well as academics and industry experts, to jointly address the challenges of rare diseases.
At the same time, the MHRA has proposed several innovative regulatory approaches. The most notable is the exploration of an "early single approval" mechanism—that is, under circumstances of limited but highly promising clinical evidence, a one-off approval will be granted for both clinical trials and market authorization, accompanied by stringent real-time safety monitoring and evaluation. This will greatly streamline the approval process.
The MHRA stated that these reforms will also promote global evidence sharing, helping the UK become a benchmark for global innovation in rare disease treatment.
Multiple patient organizations and industry bodies have welcomed the move.
Jayne Spink, Chief Executive of Genetic Alliance UK, said that for many families, a rare disease diagnosis often means there are no effective treatments. This reform is a crucial step in changing that reality and will also enhance the UK’s international competitiveness in rare disease treatment.