Secretary of State for Health and Social Care visits GOSH
30 Mar 2022, 1:40 p.m.
On Tuesday 29 March, Rt Hon Sajid Javid MP Secretary of State for Health and Social Care, visited our hospital to host a roundtable discussion on rare diseases.
The roundtable was designed as an opportunity for the Secretary of State to hear from people with lived experiences of rare diseases in the community, as well as from researchers, clinicians and people in industry.
Key points of discussion included health inequalities, the complexity of needs of children with rare diseases, and how we can have more research that translates to better care for children.
The Secretary of State was welcomed to Great Ormond Street Hospital (GOSH) by chief executive Matthew Shaw, who also attended the roundtable. The event follows the recent publication of the Rare Diseases Action Plan by the UK Government. This plan set out a shared vision for addressing health inequalities and improving the lives of people living with rare diseases across the UK.
Visiting Great Ormond Street Hospital I heard first-hand from those affected by rare diseases. The Zayed Centre for Research into Rare Disease in Children at the hospital provides world-leading care and it was a pleasure to meet patients and some of the fantastic staff treating them.
1 in 17 people will be affected by a rare disease at some point in their life and our action plan published earlier this year sets out how we will speed up diagnosis, improve access to specialist treatment and better coordinate care. We will continue to work closely with those affected, those providing treatment and those involved in research to progress this plan.
Research into rare diseases at Great Ormond Street Hospital
75% of rare diseases affect children, and at GOSH we treat over 28,000 children with rare and ultra-rare disease.
We currently have over 500 research projects looking into rare and complex diseases. These include work on gene therapy for rare immunodeficiencies, faster diagnosis of genetic conditions, and fetal surgery to treat spinal bifida.
In the last five years alone our research has contributed to the regulatory approval of 11 new treatments across Europe and the US. This means our research benefits not only children at GOSH but millions of people worldwide.
We are incredibly proud of our rare disease research at GOSH. This research helps us provide the best possible care for our patients and helps to change the lives of seriously ill children and young people. Discussions like this roundtable helps bring together a range of voices and shine a spotlight on health inequalities so that we can work together to achieve the best outcomes for the 3.5 million people in the UK, and many more across the world, who have a rare disease.
After the round table, Sajid Javid visited our paediatric intensive care unit (PICU) to see the invaluable work our staff members do in caring for critically ill children. Here he spoke with staff members about the complexity and challenges of the children we see, and also briefly spoke to a couple of patient families on the ward.
Sajid Javid last visited GOSH in September when he provided a blood sample for a genomic sequencing programme aimed at tackling disparities in health.
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