Invitation for industry-collaborative projects

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Rare Diseases Translational Research Collaboration
Invitation for industry-collaborative projects
Contents
Rare Diseases Translational Research Collaboration ..................................................................... 1
Invitation for industry-collaborative projects .................................................................................... 1
Overarching aims of the NIHR RD TRC ...................................................................................... 2
Invitation for industry-collaborative projects ................................................................................. 2
Project eligibility ............................................................................................................................ 2
Criteria for support of in-depth phenotyping of a NIHR RD-TRC cohort ..................................... 3
Funding limits and duration .......................................................................................................... 3
Application and assessment process........................................................................................... 3
Overarching aims of the NIHR RD TRC
Over 7% of the UK population are affected by one of >5000 different rare diseases, with ~80% being
single gene disorders. However, each specific disease is rare, and affects <5 in 10,000 of the
population. The overall goal of the NIHR RD-TRC is to support in-depth phenotyping of patients with
rare diseases and to develop new treatments and speed up diagnosis.
Whilst the focus of the NIHR RD-TRC is on in-depth phenotyping, a number of resources are
available for acquiring genomic data, including the infrastructure and sequencing capacity provided by
the NIHR BioResource – Rare Diseases and Genomics England Ltd.
The NIHR RD-TRC will support in-depth phenotyping research within a number of themes. Each
theme is led by an investigator within a NIHR Biomedical Research Centre (BRC) / Biomedical
Research Unit (BRU) / Clinical Research Facility (CRF):
Prof. Stan Kaye
Prof. Hugh Watkins
Prof. Nicholas Wood
Eye Disease:
Professor Graeme Black
Dr. Andrew Webster
Prof. David Jones
Gastrointestinal:
Prof. Adrian Thrasher
Immunological Disorders:
Prof. Stephen O’Rahilly
Metabolism:
Musculoskeletal Disorders: Prof. Bryan Paul Wordsworth
Prof. Michael Hanna
Neuromuscular Disorders:
Non-Malignant Haematology: Prof. Irene Roberts
Prof. Timothy Barrett
Paediatric (Cross-Cutting):
Cancer:
Cardiovascular:
Dementia & Neurodegen:
Renal Disease:
Respiratory Disease:
Prof. Fiona Karet
Prof. Eric Alton
Skin:
Prof. John McGrath
NIHR Royal Marsden BRC
NIHR Oxford BRC
NIHR UCLH BRC
NIHR Moorfields BRC
NIHR Newcastle BRC
NIHR GOSH BRC
NIHR Cambridge BRC
NIHR Oxford MSK BRU
NIHR UCLH BRC
NIHR Oxford BRC
NIHR/Wellcome Trust Birmingham
CRF
NIHR Cambridge BRC
NIHR Royal Brompton Respiratory
BRU
NIHR GSST BRC
Within each theme a number of in depth phenotyping projects are currently funded which are led by
Investigators working in NIHR Biomedical Research Centres, Biomedical Research Units or Clinical
Research Facilities (BRC/BRU/CRFs). For an up to date list of funded projects and project lead
investigators please see: http://rd.trc.nihr.ac.uk/themes-2/
By increasing the volume of in-depth phenotyping data and correlating this with the relevant genomic
data the NIHR RD-TRC aims to provide greater understanding of rare diseases and of the
mechanisms and support translational research into rare diseases. This will allow insight into which
interventions, new or existing, are likely to be effective in preventing or treating these or other
diseases. By working in collaboration with industry partners, the NIHR RD TRC projects aims to
increase in-depth understanding of rare diseases in alignment with the development of new therapies
and diagnostics.
Invitation for industry-collaborative projects
The NIHR RD-TRC recognises the importance of working with industry in order to successfully deliver
health research and progress the development of new treatments for rare diseases. The invitation for
industry-collaborative projects will aim to establish collaborative projects in rare diseases between
investigators working within NIHR BRC, BRU and CRF’s and industry partners.
Project eligibility
The NIHR RD-TRC funding available through this invitation is for in-depth phenotyping only. Where a
proposal involves other research methodology (for example, a clinical trial or acquisition of genomic
data) only the phenotyping aspects of a study will be funded by the NIHR RD-TRC. Other costs must
be met by the industry partner or another appropriate funding source. Some support to deliver the
study may be available through the NIHR RD-TRC Theme Coordinators.
Applications that build on and extend projects that are currently funded by the NIHR RD-TRC will also
be considered.
Criteria for support of in-depth phenotyping of a NIHR RD-TRC cohort
Funding will be awarded based on the following criteria:

A named lead investigator who has a track record in rare disease translational research and
is within a NIHR BRC/BRU/CRF.

A named industry partner who will be in a position to support and provide additional resource
for the study.

Application endorsed by a NIHR BRC/ BRU or CRF Director. Any funding will be awarded to
the NIHR BRC/BRU/CRF host NHS Trust.

A clearly articulated, important translational research question with the potential to have
impact within 4 years.

Evidence from prior knowledge that the research has the potential to advance diagnosis or
therapy of rare diseases.

Evidence of appropriate existing local clinical and research infrastructure.

Commitment from the lead investigator to develop and use common documentation for
consent and information, and to information exchange.

To link with, or use, OpenClinica for data capture and add to the NIHR RD-TRC OpenClinica
data set.
Funding limits and duration
NIHR contribution will be determined based on the quality of the application value for money as well
as added value. Maximum NIHR RD-TRC funding of £200,000 is available over two years. Projects
must be completed by 31 March 2017.
Application and assessment process
If the proposed study aims to make a significant contribution to the objectives of the NIHR RD-TRC
and you have an identified research and/or collaborative opportunity, the NIHR RD-TRC would
encourage your submission.
The NIHR RD-TRC invitation for industry collaborative projects uses a two-stage application process.
It is open for submissions of Expressions of Interest (EoIs) at any time until further notice. Full
applications are expected to be submitted within 4 months of an invitation being issued.
Expressions of interest from Investigators or Industry should be provided using the Expression of
Interest form.
EoIs will be reviewed by a sub-section of the NIRH RD-TRC Strategic Oversight Group (SOG) to
determine whether they are within the scope of the scheme. Successful EoIs will be invited to submit
a full proposal requesting additional detail related to the proposal and a detailed breakdown of the
study costs and industry contribution, including resources provided ‘in kind’. Full applications will
undergo peer review prior to review by the SOG. The NIHR RD-TRC will endeavour to review
applications as quickly as possible. For EoIs we aim to provide a decision regarding eligibility for full
application one week after submission. For full applications we expect to provide a decision one
month following receipt of the full application.
Submission: Completed EoI or applications forms should be submitted to the Operational
Director, Dr. Marijcke Veltman, at mwmv2@medschl.cam.ac.uk
Linking Industry with the NIHR Infrastructure
If you require further information or would like an informal discussion regarding a proposed project in
advance of submitting an expression of interest or full application please contact NIHR Office for
Clinical Research Infrastructure (NOCRI).
For assistance in sourcing a relevant NIHR investigator to explore the possibility of collaborating on a
project, the industry partner should complete the EoI form stating that they require assistance to
identify a partner. NOCRI will seek willing academic collaborators to work with the industry partner
within 2 weeks of the application submission and will facilitate set up of an initial meeting. Following
this, activities and the decision to collaborate in the application will be left between the two parties
involved.
nocri@nihr.ac.uk
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