Collectively common: the devastating impact of rare kidney diseases in the UK
Our independently produced report reveals the impact of rare kidney diseases.
The report shows that people with rare kidney diseases face significant diagnosis delays, inconsistent care and long waits for new treatments, with children in particular left at greater risk of medical, educational, and economic disruption.
Key findings from the report
People with rare kidney diseases face significant diagnosis delays, inconsistent care and long waits for new treatments, with children in particular left at greater risk of medical, educational, and economic disruption.
- Report shows over 160,000 rare kidney disease patients face diagnosis delays and significant gaps in care.
- Children with rare kidney diseases can wait more than a decade longer than adults to access new treatments, and those on dialysis are on average missing more than half the school year, every year.
- Rare kidney diseases cost the NHS £263m annually in dialysis alone – yet families remain overlooked in health policy.
Recommendations for action
14 recommendations have been made to deliver faster diagnosis, ensure children are included in
clinical trials, and commit investment into research and services so families are no longer left behind.
The recommendations focus on six priority areas of focus.
Diagnostic challenges
Rare kidney diseases may mimic common conditions and are often asymptomatic at early stages, leading to delayed diagnoses or misdiagnoses. Delays in diagnosis can lead to further disease progression,and misdiagnoses can lead to inappropriate treatment, leaving patients with worse outcomes and closer to kidney failure.
Healthcare challenges
NHS care pathways are not well adapted to the needs of people with rare kidney diseases, particularly children. Renal clinical networks and paediatric renal services improve coordination but lack national consistency, and there is inadequate support for transition from paediatric to adult care. In addition, regional access to specialist care is inconsistent. Rare kidney disease care should be prioritised as the UK Government develops new policy to replace the current UK Rare Diseases Framework and ensure it is implemented across the four nations, to manage the disproportionate impact of rare kidney disease.
Access to new medicines
The limited availability of disease-specific treatments means many people with rare kidney diseases rely on supportive or generalised treatments with considerable side effects. There is some hope, however, with a number of clinical trials ongoing for new targeted medicines. It is essential that regulatory and reimbursement pathways for medicines are optimised for rare diseases to ensure patients receive access to new treatments as soon as possible.
Clinical trials
Enrolment in a clinical trial offers participants early access to new medicines and can boost UK R&D investment. However, in a recent review of 13 clinical trials for rare kidney diseases, 40% did not have a UK site, meaning UK patients and the UK economy are not benefiting as much as they could be.Furthermore, the exclusion of children from rare kidney disease trials means they are unable to benefit from new therapies at the same time as adults.
Enhance RaDaR
RaDaR is a world-leading patient registry and an invaluable resource for the study of rare kidney diseases in the UK. However, enrolment into the database is time-consuming, and there is no government funding to support doing so. This means we lack an accurate picture of the true prevalences of rare kidney diseases in the UK, and the data we do have are geographically skewed. Furthermore, patients who might benefit from participating in research are missing out.
Lack of economic evidence
Primary research into the economic impact of rare kidney diseases is lacking. A systematic review of the economic literature focused on rare kidney diseases published over the last ten years found 33 studies globally, but none covered the UK.
