LifeArc and Genetic Alliance UK have launched a report calling for the UK Government, NHS, researchers, industry, patient groups and policymakers to work together to transform the lives of more than 3.5 million people in the UK living with a rare disease.
LifeArc assembled a Taskforce of stakeholders from across the UK rare disease and life sciences landscape to develop the report ‘Accelerating R&D for rare disease in the UK: An opportunity to change millions of lives.’ The report lays out the systemic failures of the existing landscape and makes several crucial recommendations to streamline the R&D ecosystem, create an attractive environment for investment, and ultimately, drive progress for people with rare diseases.
“The challenges we face are clear – but so is the potential for meaningful change. We can no longer wait for tomorrow. We must act today to demand better, to work together and to transform the system for the millions of people living with rare diseases.”
Dr Sam Barrell, CEO of LifeArc
One such person is Idrees Hanif, who was diagnosed with the rare condition, Niemann-Pick, at the age of 5. Idrees’s father, Qasim, explains, “Scans revealed an enlarged liver, spleen and a build-up in his lungs, his organs were the size you’d see in a teenager. When the doctor described the symptoms and the fact that life expectancy is very short – we were devastated, our world came tumbling down.”
In April 2025, shortly after Idrees’s diagnosis, news of a potential treatment emerged. However, the rollercoaster continued for Idrees’s family when the treatment was rejected by the National Institute for Health and Care Excellence (NICE). “Knowing your grandson has a devastating disease is only made worse by knowing there is a treatment but one he can’t access,” added Nasreen Hussain, Idrees’ grandmother.
Currently, Idrees is receiving the treatment under special ‘compassionate use grounds’, with the pharmaceutical company supplying the drug. “He’s now flourishing, but we live with the constant fear of what happens if that’s taken away. This report matters – its recommendations could help fix the system and make a real difference for families like ours,” said Nasreen.
The report comes at a pivotal time as the UK Government considers the future of the Rare Disease Framework, which expires in 2026. Its recommendations include embedding rare disease needs into the UK’s health data infrastructure, ensuring that new treatments are reviewed and approved in a more joined-up and coordinated way, creating a ‘concierge-style’ support service to assist rare disease innovators and refreshing the existing UK Rare Disease Framework.
“Rare conditions are common, and the people who live with them deserve better. We want a future where people with rare diseases receive radically improved care in the way they are diagnosed, treated and cared for. This report provides practical, evidence-based solutions to make that a reality.”
Nick Meade, Chief Executive of Genetic Alliance UK
The report was launched today (Monday 7 July) at an event in Westminster.
For further information and to download the report, follow the link below.
Media contact
Andrew Stewart
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