Registries, Databases & Clinical Networks David J Burn Newcastle University

Introduction Viable neuronal population Diagnostic certainty Prevalence – multicentre studies essential Therapeutic window – sensitivity vs. specificity Phenotypic variability Biomarkers & biobanking Patient-led vs. clinician case ascertainment

Registries & Databases What are they for? Who will use the data? – access model Feasible? Current? Confidential / secure?

Research & Feasibility Tool (RAFT) NIHR-DeNDRoN initiative Anyone interested in participating in dementia research registers details either online or via a telephone helpline Researchers use the system to establish whether their research proposal is feasible, based on the number of potentially suitable candidates registered & recruit appropriate candidates to their studies System developed in conjunction with key stakeholders including Alzheimer’s Society, Alzheimer’s Research UK, people affected by dementia, researchers & NHS Further development of the system will include linkage to NHS electronic patient records & integration with existing local research registers

EMSA Network Founded in January 1999 A consortium of scientific investigators from academic & research centres in Europe / Israel Aims to advance knowledge about the aetiology & pathogenesis of MSA EMSA-SG works with government & industry sponsors to develop & implement novel therapeutic interventions

UK MSA Network Lead: Henry Houlden Funder: MSAT Longitudinal clinical & imaging database & sample biobank Outcomes: – open access database – well phenotyped cohort platform for trials / biomarkers – global networking EMSA, MoDiMSA

UK MSA Network: Main Aims Recruit patients at all stages of MSA but with focus on early stage disease (UK MSA Register & Database) – clinical, demographic & environmental information – annual information on disease progression, milestones & quality of life – request for brain issue donation Collate serial MRI imaging, FP-CIT SPECT scan, autonomic etc. for each patient Collect blood for DNA, RNA, serum / protein ± CSF at diagnosis & two years later (UK MSA Biobank) – identification of future disease biomarkers to improve early diagnosis – develop surrogate markers of disease progression

Evolving the NIHR Clinical Research Network From 102 local networks Partial geographical coverage for many diseases 8 coordinating centres Different operating procedures & processes To 15 local networks Full geographical cover for all diseases 1 coordinating centre Consistent, coherent procedures & processes Why? Easy to understand for external stakeholders All clinical themes supported in all locations Flexibility to operate effectively with the changing NHS More effective learning organisation

Supporting PD & Movement Disorders Research from April 2014 Full national coverage from 15 Local Clinical Research Networks covering the whole of England At both national and local level there will be six research delivery divisions, each covering a set of related specialties Dementia & Neurodegeneration, Mental Health and Neurological Disorders will be in a single research delivery division PD will be part of the Dementia & Neurodegeneration (DeNDRoN) specialty

Growth in NIHR PD activity

From April 2014 DeNDRoN CC continue to provide national support for research delivery DeNDRoN LRNs being incorporated into new LCRNs Staff transferring to new organisations Business as usual Opportunities: in all locations, to learn from others, to further develop resources to support Movement Disorder research

What Does All this Mean for MSA? Continued availability to support delivery of MSA studies – UK MSA Network – recruitment & delivery of trials The PD-CSG is dead; long live the PD-CSG – links with Parkinson’s UK? – new investigator-led studies ABN-MD Special Interest Group

Conclusion is a time of change Exciting opportunities for MSA research – UK MSA Research Network Maximise strategic links with: – NIHR CRN / DeNDRoN – Parkinson’s UK – global networks

Current BritMODIS Structure IPMDSBritMODIS ABN MD SIG ABN BGS-MD Section BGS PD Nurse Specialists AHPs? BritMODIS “Executive”

BritMODIS: A Group for PD Training, Education & Research BritMODIS Executive Research & trials Clinical studies group Portfolio delivery Training, education & meetings Fellowships Evidence-based / guidelines Regional representation mapping to LCRNs where possible