Improve care, support and treatment for children with rare diseases

3.5 million people in the UK will be affected by a rare disease at some point in their lives. 95% of rare diseases have no treatment and 50% of those affected are children; 1 in 3 do not live to see their 5th birthday. These children can wait years for a diagnosis and some just never receive one.

Care for children with rare diseases is suffering hugely due to COVID19. The current UK Strategy for Rare Diseases ends this year and work to provide a new and modernised framework has stalled. Sign this petition to urge Government to deliver a comprehensive, modern and measurable rare disease framework before the end of 2020, so the most vulnerable children in our society can get the improvements to care and support they so desperately need. Sign to make their lives better and to show you care.

COVID-19 has had, and continues to have, a profound effect on the NHS, patients and their families. It has also had a heavy impact on government business, with new processes and structures needing to be established rapidly and some work pausing to enable civil servants to be redeployed in the short term to support the UK’s response to the pandemic. Despite this, the Department for Health and Social Care (DHSC) and NHS England (NHSE) remain focused on the need for continued momentum in improving the lives of rare disease patients and are committed to introducing a new UK Rare Diseases Framework by the end of 2020 for the benefit of all patients; notably children who live with a rare disease. NHSE is also considering the impact of COVID-19 on rare disease patients, including understanding what services can continue to be delivered through virtual communication technology such as telephone consultation and videoconferences. Where services do need to be delivered face-to-face, NHSE will work with providers to ensure that patients have a safe journey through the hospital to the treatment area.

The United Kingdom Strategy for Rare Diseases, published in 2013, set out a strategic vision for improving the lives of patients and promoted actions to ensure that people living with a rare disease, including children, receive high-quality care. The Strategy is due to expire in 2020 and will be replaced by the future UK Rare Diseases Framework. All four nations have signed up to the Framework development, ensuring that we continue to work collaboratively across the UK with a coherent, national vision. The Framework will be followed by nation-specific actions plans that will detail the specific commitments each government will take to meet the shared principles of the UK Rare Diseases Framework.