Although rare diseases are individually infrequent, one in 17 people will be affected by a rare disease at some point in their lives. This equates to nearly a million children in the UK and Ireland.
So, what can be done? The mapping of human genome, changes to EU orphan drug regulations and the launching of the UK rare disease strategy in 2015 led to an explosion of interest in rare diseases. This has stimulated new and innovative areas of research in the field of immunotherapeutics, genomic and translational.
However, funding sources can still be a challenge to locate, especially for basic disease research such as epidemiology, diagnostics, and service evaluation. By targeted funding these areas there is the potential to speed diagnosis and contribute to improving service delivery and public health policy changes.
This will improve the lives of those children and families affected by rare disease for which living with such conditions has a large, emotional, financial and social impact.
The objective of the Rare Disease Development Fund is to support research and surveillance projects and to help develop and deliver education and training programmes on rare disease and rare complications - all with the aim of improving health outcomes for children and young people affected by rare diseases.
The fund will facilitate the development of:
For paediatricians to carry out translational or basic science research, such as surveillance studies through the British Paediatric Surveillance Unit (BPSU)
Grants and bursaries
Grants or BPSU-approved studies on rare diseases that affect children and that address a question of clinical or public health importance
Bursaries for child health professionals (paediatric trainees, academic trainees, new consultants, allied health professionals) to support attendance at training courses, conferences or events to present data
Education and training
Development and delivery of programmes to support the paediatric workforce to better provide services for children and young people with rare diseases, including face-to-face and online courses in collaboration with BPSU
Development and dissemination of patient resources in collaboration with Rare Disease UK, RCPCH &Us and other stakeholders and patient groups
We invite you to be a part of this vision to influence the future of childhood rare disease research, and the health outcomes for children and young people affected by them.
We welcome personal donations (from both RCPCH members and non-members), corporate gifts, grants and funding from trusts and foundations and other charities. Please contact firstname.lastname@example.org, if you wish to discuss supporting a specific area or topic.
If you pay income or capital gains tax in the UK you increase the value of your donation to us by 25% through a Gift Aid declaration. It allows us to claim back tax paid on the donation, which we can then put toward the College’s projects.
For information on how we process your data in relation to donations, see our privacy notice.