Neurodegeneration specialty profile
Delivering Neurodegenerative Diseases Research in the UK
Neurodegenerative disease research is fully embedded within NHS clinical practice. We have well-established, highly regarded clinical services for patients with neurodegenerative disease and their carers, with clinical communities having research delivery as a key priority, such as the Parkinson’s Excellence Network and the Motor Neurone Disease Clinical Studies Group. We have a network of study sites across the UK, with study delivery expertise in all three major disease areas – Parkinson’s disease (PD), motor neurone disease (MND) and Huntington’s disease (HD), encompassing the whole range of study complexity.
The Clinical Research Network (CRN) provides researchers with the practical support they need to make research happen in the NHS and the wider health and social care environment. As well as providing research delivery staff, we also bring together communities of clinical practice to provide national networks of research expertise. Neurodegeneration, which is part of the Dementia and Neurodegenerative Diseases (DeNDRoN) Specialty, is one of 31 such communities and is made up of leading research-interested clinicians and practitioners at both national and local levels. Our job is to ensure that the studies we support in neurodegenerative conditions such as PD, MND and HD are delivered to time and target.
All regions in England and the devolved nations have representation in our specialty leadership group, which includes nationally and internationally recognised key opinion leaders who have acted as Chief Investigators for UK and international multi-centre studies. Our industry lead for commercial studies, together with our stratification lead, provides strategic direction as well as an operational link between industry, the NIHR and researchers, ensuring that as a nation we have specialist sites who are trial-ready for the innovative therapeutics being developed.
We take a multidisciplinary approach to developing the next generation of researchers through training and mentorship, building skills amongst a broad range of professionals so that capacity for study delivery sits well within clinical care pathways.
Access to participants
Our specialty provides access to research for the majority of patients with neurodegenerative conditions attending NHS services in the UK. The cohort study initiatives supported by the major charities and investigators (such as PROBAND and DISCOVERY in PD, PROSPECT for atypical parkinsonism, the MND National Register and ENROLL-HD, TRACK-HD and TRACKON-HD in HD) ensure that we have trial-ready potential participants who are well phenotyped, and also, increasingly, genotyped.
Regional research registers also facilitate patient access (such as PRO-DeNDRoN in PD and MotoDeNDRoN in MND). We also work closely with charities to identify access and recruitment needs and solutions, ensuring research access is as inclusive as possible.
High quality study delivery
In 2018/19, over 45,900 participants were recruited into 262 Dementias and Neurodegeneration (DeNDRoN) studies within 159 secondary care NHS trusts. Of these, 12,330 participants were recruited into neurodegeneration and 33,588 participants into dementia studies.
The Dementias and Neurodegeneration Speciality delivered 80 per cent of studies to time to target in 2018/19. There is expertise in the delivery of Phase II and III studies, including multi-site international, randomised clinical trials.
Our study sites are distributed across the UK, matching the patient population. We undertake feasibility using high quality data analytics, and increasingly sites are implementing data solutions for feasibility assessment.
The UK neurodegenerative disease research portfolio is diverse. Expertise exists across all key domains: application of brain imaging; research into pathology and genetics; qualitative research including the development of person-centered care; and epidemiological work.
We have strong links with national charities such as Parkinson’s UK, the Motor Neurone Disease Association and the Huntington’s Disease Association, including patient representation on our specialty group. These links facilitate patient involvement in research and provide important synergies with providing service excellence across the UK. Several studies on the CRN portfolio have been funded by these charities and, through the efforts of our specialty group, have recruited to time and target.
We work collaboratively with pharma to deliver investigator initiated studies and support trainee networks to ensure development of the next generation of researchers.
The CRN uniquely offers a collegiate research community, operating for the benefit of our participants, taking on new challenges in study delivery and sharing best practice and solutions.
Optipark was a commercial study which successfully recruited 29 per cent above the UK targets across 20 sites, with 16 out of the 20 reaching or exceeding their local targets. This study demonstrates the wide network of sites available in the UK to successfully deliver commercial studies.
Motor neurone disease:
The SOD-1 study was investigating a first-in-man novel therapeutic in a genetic MND subtype. It was delivered in a single centre, but relied on our national network to identify participants with the necessary specific genotype. This study over-recruited, demonstrating capability for recruiting to studies based on genotype and ability to deliver novel biological therapies. This SOD-1 gene therapy trial provides an excellent, highly promising example of a personalised medicine study in a genetically stratified neurodegenerative disorder.
A ground-breaking study of intrathecally-delivered antisense oligonucleotide (ISIS 443139) involved 4 UK sites, delivering 20 of the 46 global recruits. This demonstrates capability to adopt new therapeutic innovations and deliver a unique therapeutic using an unfamiliar delivery system, to share learning between sites across the network to ensure successful study delivery.
Contact our Study Support Service
- Access our Study Support Service online at: www.nihr.ac.uk/showcase/services/study-support-service.htm
- Phone: 00 44 113 34 34 555
- Email: email@example.com