National call in translational paediatric rare disease research

Great Ormond Street Hospital Children’s Charity is inviting project grant applications, from across the UK, which support translational research into rare diseases and which will impact patients at Great Ormond Street Hospital (GOSH) and elsewhere.

Great Ormond Street Hospital Children’s Charity is committed to supporting high quality research with the potential to improve the health and well-being of children at GOSH, through better diagnosis, treatment, improved care and cures. Our particular interest is the rapid translation of research for patient benefit. 

Scope of call

Rare diseases are currently under-researched and under-resourced and they can often leave patients and their families feeling isolated and vulnerable. Rare diseases have a low prevalence but a high level of complexity and for rare syndromes, diagnosis may be difficult and delayed due to limited scientific knowledge and clinical recognition of the disease. Seventy five percent of rare diseases affect children. GOSH specialises in the care of children with rare syndromes and hosts 19 nationally commissioned services for such conditions, the largest number in a single NHS Trust. The charity is committed to investment in rare disease research and in addition to this call is developing a new Centre for Children’s Rare Disease Research, an £85 million research facility due to be completed in 2017.  

Research that will improve the diagnosis of rare diseases will be prioritised and for the purposes of this call the definition of a rare disease is one that affects fewer than five in 10,000 people.

We particularly encourage highly translational proposals that:

  • Address problems that will have an impact at the point-of-care.

  • Are based on practical research that will have an impact on health services and delivery.

  • Have a robust study design and established access to the relevant patient population.

  • Are multidisciplinary in nature and demonstrate real partnership between clinical and academic researchers.

  • Have the potential to leverage Great Ormond Street Hospital Children's Charity funding and lead to a larger grant.

Funding available

Great Ormond Street Hospital Children's Charity is inviting project grant applications from across the UK that have the potential to impact patients at GOSH and elsewhere. Up to £1 million is available to spend under this call and it is anticipated that we will fund a range of proposals from different investigators and institutions. Funding will be provided for the direct costs of research (such as research staff and consumables) and will not cover consultant PAs or team-leader salary time. Typically, projects will be up to three years duration.

Further details are available through the Additional Information and Guidance Notes and on the Great Ormond Street Hospital Children's website www.gosh.org/research

The deadline for submission of outline applications is 5pm, Wednesday 6 November 2013.