Our work will have a clear impact on multiple beneficiaries. Patients will benefit from a better understanding of the molecular pathophysiology of musculoskeletal disorders that will deliver new biomarkers for the diagnosis and outcome prediction, and new individualised and target-directed treatments some of which may be delivered by re-purposing of already existing medicines. Assuming success in commercialisation (or the discovery of a new indication for an existing licensed drug), benefit will extend to the public sector (via the University) and the commercial sector (the manufacturer of the product).
Our major focus is to deliver excellence in care and ensure that the research we do is always directed towards answering the priority questions our patients and their families have.
- To improve the health and well-being of children
By providing an internationally excellent children's centre, for conduct of experimental medicine studies and early phase clinical trials, which will be an important resource for the pharmaceutical industry.
- To establish a platform of expertise
Expertise that is internationally competitive and fully integrated with existing paediatric rheumatology, collaborative strengths and NIHR infrastructures.
- To develop a national consortium
This will support existing experimental treatment resources, to enhance the delivery of “first in childhood disease” studies of novel therapies, diagnostics and prognostic biomarkers.
- To understand the mechanisms of adverse drug reactions
This will allow the development of mechanism-based biomarkers to improve the benefit-risk ratio of medicines.
Achieving our goals
The EATC4Children is underpinned by nationally and internationally recognised expertise in:
- Paediatric rheumatology and musculoskeletal health, in early and late phase clinical trials in children and young people
- Capability and infrastructure to sustain every stage of drug development, in a manner consistent with EU Regulations developing Paediatric Investigation Plans (PIPs)
- Drug development, drug safety, pharmacogenomics, stratification of medicines and training in paediatric clinical pharmacology
- Development of trial designs using novel adaptive trial designs, supported by the MCRN Clinical Trials Unit and the NW MRC Methodology Hub
- Basic science, translational research and training.
Through partnership collaborations with the Versus Arthritis adult-based Clinical Studies Group’s, NOCRI Translational Research Partnership, and MRC/ABPI Inflammatory Arthritis Initiative, the EATC4Children optimises potential impact across the clinical and regulatory age groups (child through adolescents to adult).