We welcome publication of the Government’s England Rare Diseases Action Plan 2025 (Friday 28 February) and recognise its commitment for change to enable everyone living with a rare disease to access the health care they need.  

There are currently more than 150 types of rare kidney disease - affecting around 70 in 100,000 people - with the majority caused by genetic changes.   

Yet there are few treatments available and there is an urgent need for new therapies. 

Rare and genetic kidney diseases are responsible for nearly all cases of kidney failure in children, and account for more than a quarter of people needing dialysis or a transplant, thus contributing significantly to kidney failure rates. We believe there is a particular unmet need for treatment in children living with rare kidney disease. 

Kidney disease: A public health emergency

Our 2023 report warns that kidney failure could overwhelm the healthcare system by 2033. 

We support the four priorities in the Government framework of: 

• faster diagnosis 

• increased awareness of rare diseases among healthcare professionals 

• better co-ordination of care 

• improved access to specialist care, treatment and drugs. 

A call for more Government action

For patients with kidney disease, we call for further and faster Government action to ensure these priorities are met. In addition, we call for further investment in research and innovation in kidney health, improvements in equity and access to treatments. Finally, there must be greater access to clinical trials for all kidney patients, but particularly for children. 

“Real change can only be achieved through action. We look forward to seeing evidence that the Rare Diseases Action Plan is being implemented during the forthcoming year,” said Alison Railton, director of policy and public affairs at Kidney Research UK. 

“As a charity, we will continue to strive for our vision: a world free from kidney disease,” she added.