Great Ormond Street Hospital calls for Europe deal that protects paediatric research and UK’s ability to treat most complex childhood diseases

World leading children’s paediatric research and treatment centre, Great Ormond Street Hospital (GOSH), is urgently calling on the Government to secure a European deal which ensures the UK retains its brightest minds and crucial research funding, to save young lives and ensure transformative treatments and cures continue to be developed.

GOSH highlights the serious risk of losing EU staff, large-scale EU funding and long standing European collaborations for the transformative diagnoses, cutting edge treatments and care that children with rare and complex diseases so urgently need.

Losing EU funding for research programmes and clinical trials could critically delay the development, and availability of revolutionary treatments and cost the lives of some of the vulnerable children that GOSH treats, whose conditions are so serious that every day is vital. Seventy-five per cent of rare diseases affect children and thirty per cent of children with a rare disease die before their fifth birthday. For many of these children research is their only hope.

Without EU research staff, centres like GOSH will lose experts who currently drive transformations in paediatric care and treatment. Without highly skilled EU medical staff the NHS would see deficits to its workforce and severe disruptions to the care of sick children. GOSH and the ICH have substantial numbers of EU staff – 24 per cent of research staff, 25 per cent of medical staff and 16 per cent of nurses.

Dr Peter Steer, Chief Executive of Great Ormond Street Hospital, said:

“We are deeply concerned about what leaving Europe means for our many EU staff and our research programmes. We call on the Government to prioritise the best European deal to protect these essential staff and research programmes, now and in the future. For the vulnerable, very ill children we care for, every day counts.

“The UK is known for the strength of its biomedical research.  At GOSH our research base enables us to develop transformative treatments and cures and save lives. We do not want to see this eroded by uncertainty about the status of key staff and funding.”

Graeme, is father to three-year-old Nina, who was treated for a rare, aggressive disease of the immune system, severe combined immunodeficiency (SCID), which left her with life-threatening, persistent infections. She was successfully treated on a trial for untested gene therapy, with a 30 per cent survival chance.

"Pioneering research and treatments from Great Ormond Street Hospital saved our three-year-old daughter Nina’s life. These treatments are now being further refined and developed to save the lives of thirteen other children from all over Europe.

"It is critical that funding is allocated to the most effective areas of research, to combat rare genetic illnesses that children like Nina have. GOSH treats children with the most difficult illnesses known to man. Research must never be compromised or lose priority if we’re to continue to save, and value, our children's lives."

GOSH further outlines the risks to paediatric care and research:

• Restricting freedom of movement for EU patients taking part in collaborative UK-EU clinical research risks losing a larger patient base. This is crucial to studying and gaining an understanding of rare diseases and speciality areas such as metabolics, oncology and gene therapy, to identify new treatments earlier. These treatment discoveries also translate into crucial opportunities for adult medicine.
• The EU currently provides uniform regulations and safety infrastructure around research and clinical trials that ensure safety for children on these trials. 
• The diagnosis, treatment and management of rare childhood diseases requires global collaboration at all levels. Restricted access to research groups and institutions across and outside Europe, risks the effective and timely sharing of research insight and results which drive new global treatments for children and adults. Scientists will need to find lengthy, complex ways around this to achieve participation.
• Drug treatments for children with rare diseases could be affected and the costs of some drugs may increase.
• Renegotiating entry into EU funding programmes will be expensive, lengthy and could, even then, lead to restrictions. The UK government would need to substantially contribute to all or part of the Framework Programme for Research to become an Associated Country.

“Living with OI, I have seen first-hand how treatment for conditions like mine can dramatically improve the quality of life children have. Just within my lifetime, researchers have started to develop a treatment, using funding from the EU, which can modify the genes of a foetus before birth, completely relieving the symptoms of OI. This essentially eradicates the disease. In my opinion this is an absolutely fantastic contribution to society, and honestly something we truly cannot live without.”