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Poster presentation | Open | Published:

UK paediatric rheumatology and its clinical trials network

Background

There is striking paucity of a rigorous evidence-base for the treatment of paediatric musculoskeletal diseases. The Medicines for Children Research Network (MCRN) improves coordination and quality of clinical trials and related studies across the UK. It has joined in partnership with the Arthritis Research Campaign (arc), the major UK charitable funder of musculoskeletal research, to form the MCRN/arc Paediatric Rheumatology CSG.

Methods

The MCRN has extensive expertise in supporting non-commercial, pharmaceutical-sponsored and investigator-led studies. The CSG is a multi-disciplinary group comprising clinicians, academics, basic scientist, pharmacy, and clinical trials unit support. Through consultation with stakeholders, its remit includes: developing a comprehensive portfolio for clinical trials/related studies covering the spectrum of paediatric rheumatology; ensuring consumer involvement; upholding Good Clinical Practice.

Results

The CSG has developed topic-specific groups (TSGs) outlined in Table 1. Key "themes" across TSGs include: acceptability of intervention; quality of life; health economics; cardiovascular risk; skeletal health; rare diseases; orphan drugs; international collaboration. Key "tools" include: characterised phenotype cohort studies; biomarkers; genotype/phenotype/proteomics; pharmacogenomics; pharmacokinetic studies; mechanism of disease; Phase II and III studies. Strategic development of protocols is underway. Regionally based networks assist with feasibility, site selection, financial, ethical and governance arrangements, staff recruitment, training, participant recruitment and trial monitoring. Table 2 illustrates studies already MCRN adopted and supported in this way.

Table 1 Topic-specifc Groups
Table 2 Studies recently adopted by MCRN

Conclusion

An integrated national research network provides an excellent infrastructure to collaborate closely with international trial networks (e.g. CARRA, PRINTO) while fostering a unique contribution to the transformation of clinical care of children.

Author information

Correspondence to MW Beresford.

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Keywords

  • Orphan Drug
  • Paediatric Rheumatology
  • Clinical Trial Unit
  • Trial Network
  • Consumer Involvement