Skip to content

Study suggests rare kidney diseases treatments could dramatically reduce the burden of kidney disease on patients and the NHS

14 March 2024

In a major new study published in The Lancet, a team of researchers led by Professor Danny Gale from University College London used information from the largest rare kidney disease registry in the world - the UK National Registry of Rare Kidney Diseases (RaDaR) - to show that finding better treatments for rare kidney diseases could have a far greater impact on reducing the need for dialysis and transplant than previously thought. 

Professor Daniel Gale
Professor Daniel Gale

Highlighting the impact of rare kidney diseases 

While each disease might be rare on its own, collectively, rare kidney diseases are common. Globally, more than 25% of adults and 50% of children with kidney failure have a rare disease.    

Because they affect a relatively small number of people, it is difficult to gather enough information about rare diseases to ensure evidence-based decision making, and to get funding to research and develop treatments. This is particularly true for many kidney conditions as patients often live with them for a long time, so their information may be scattered across different databases, systems and healthcare providers, making it difficult to access all at once.

What is a registry and what is RaDaR?

A registry is a collection of information about a specific group of people. Registries are particularly useful for studying rare diseases because they collect information from people who have these conditions into one place, making it much easier to find patterns, learn more about diseases, identify groups eligible for clinical trials and ultimately develop better treatments.    

RaDaR was set up in 2010 with funding from Kidney Research UK and Kidney Care UK and is supported and coordinated by the UK Kidney Association. RaDaR securely stores important long-term information about people in the UK with rare kidney diseases, including where they live, any treatment they are receiving and how they are responding to that treatment, as well as relevant blood test and imaging results.   

RaDaR is the largest rare kidney disease registry in the world and has now recruited over 30,000 patients, aged 0-96 years from 108 UK hospitals across 29 rare disease categories. The goals of RaDaR are to:  

  • Learn more about how rare kidney diseases develop and progress over time.  
  • Study the long-term effects of treatments.  
  • Create a system to help with research on specific rare diseases and other smaller registries.  

Using RaDaR data to guide clinical practice

Danny and the team studied kidney function decline, kidney failure and survival in 27,285 patients with 28 categories of rare kidney diseases. They also looked at how their health compared with that of 2.81 million patients who have chronic kidney disease (CKD) due to other causes (such as diabetes and high blood pressure).  

The impact of focusing on rare diseases

Results showed that, over five years, people with rare kidney diseases were 28 times more likely to reach kidney failure than patients with CKD from other causes, but they were much less likely to die both during the earlier stages of CKD and whilst on dialysis.  

This means that people with rare kidney diseases represent a larger share of the population of people receiving dialysis or transplant than we previously realised, so finding effective therapies for rare kidney diseases could have a huge benefit on the overall demand for dialysis and transplant. 

Dr Katie Wong, first author of the study, said: “In general, rare kidney disease patients are much younger than those with CKD and are much less likely to die from related conditions such as cardiovascular disease. We know that they can live a long life with a successful kidney transplant, but there’s also an opportunity to prevent kidney failure in the first place with targeted treatment. I hope that the robust, large-scale data for each of the 28 diseases covered by RaDaR will inform trial design and make developing new treatments a less uncertain endeavour for drug companies.” 

Saving money as well as lives

Last year, Kidney Research UK produced a health economics report, which estimated that, in 2023, the direct cost to the NHS of dialysis and kidney transplant was £1.05 billion (not including the £225 million spent on transport for patients on in-centre dialysis) and £293 million, respectively.  

As well as the benefits to health and quality of life, these new results from RaDaR suggest that tackling rare kidney diseases would dramatically reduce the demand for dialysis and transplant which would also have a huge economic impact on healthcare systems.  

Danny said: “This study underscores the importance of recognising the pivotal role rare kidney diseases play in the overall burden of kidney failure. I hope that this will be a call to arms to show how important rare kidney diseases are and the many potential benefits of focusing on these conditions. Treatments for many of these diseases are either available or in development, so I think we now have a golden opportunity to substantially reduce the burden, both for patients and the NHS, of kidney failure.” 

Elaine Davies, director of research operations at Kidney Research UK said: “As well as highlighting the importance of finding treatments for rare kidney diseases, these new results will allow patients and doctors to make more informed decisions about their care and will be invaluable for the planning of future clinical trials. RaDaR is a fantastic resource that has provided and will continue to offer vital insights into rare kidney diseases, and we are proud to support it.”   

Read more research news

Scroll To Top