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2015 – 2020 R&D Strategy
Nick Watkins
Assistant Director – R&D
November 2014
Our Strategic intent
• To support large, strategic research initiatives for
the benefit of NHSBT and healthcare in the UK and
beyond
• To deliver an innovative and translational R&D
programme which is embedded within corporate
objectives
We have an academically excellent research
programme.......
6
Universities of
Liverpool/Leeds
University of
Oxford
University of Cambridge
University College
London
Impact
5
4
3
2
Mol Eng
Stem Cells
ODT
Platelets
Red cells
Appropriate Use
Microbiology
Our 17 Principal Investigators:
• World leaders in their fields;
• > 1,000 publications over 10 years;
• 9 patents filed.
0
Donor Health
1
University of
Bristol
Benchmark
“....the research potential of the current staff at NHSBT should be considered excellent....”
CWTS, University of Leiden
The R&D Programme 2010-15 delivered
improvements in patient care....
Gene therapy to
treat haemophilia
dCell Dermis for
leg ulcers
Prion filter evaluations
Cord blood
improvements
Benefit of platelets in
cancer patients
Improved stem
cell/organ matching
DCD allocation
Pre-operative use of red
cells in sickle patients
Smoker’s lungs
HTLV detection
Pathogen inactivation of plasma
...in support of operational strategies.
Overview of New Themes/funding
• Donor health/population genomics - -NIHR
• Virology/microbiology -funded from blood price
• Patient Blood management – funded from blood
price
• Regenerative medicine/blood cells ?NIHR/price
• Organ donation/transplantation – NIHR/?GiA
• Stem cells & immunotherapies – NIHR
• Tissues – funded from prices
Research priorities in organ donation and
transplantation
• Support research that will lead to increased rates of donation from
deceased and living donors or will improve or increase quality
and numbers of organs transplanted or improve the quality and
length of survival of transplant recipients, in line with our published
strategic objectives outlined in ‘Taking Organ Transplantation to 2020’.
• Work closely with clinical partners at both national and international
levels to promote our aims by provision of skills, knowledge,
information, resources, expertise within our current resources.
• Promote research and clinical audit within ODT and in collaboration
with stakeholders.
• Ensure NHSBT is recognised, nationally and internationally, as a
leader for research in organ donation and transplantation.
New: NIHR Blood and Transplant
Research Units –funded for 5
years
– To create an environment where world-class
research, focused on our needs and those of
the patients we serve, can thrive;
– To translate advances in research into benefits
for users of NHSBT’s services;
– To focus on areas of greatest priority;
– To provide high quality research evidence to
inform decision-making;
Creation of 4 NIHR Blood and Transplant
Research Units
Open competition – international panel
– Donor Health and Population Genomics: Cambridge
– Will address major questions about the health of blood donors;
– Produce evidence-based strategies to enhance donor safety;
– Ensuring sustainability of blood supply.
– Organ Donation and Transplantation: Cambridge/Newcastle
– Develop and evaluate novel approaches and technologies;
– Increase the availability of suitable donor organs for transplantation
– Improving graft survival.
– Stem cells and immunotherapies: UCL
– Facilitate the clinical translation of scientific advances;
– Enable optimal donor selection
– Develop new and improved stem cell-based treatments
Competition in regenerative medicine (blood cells) launched now
NIHR BTRU in ODT
• £3.8 M over 5 years awarded to establish a Blood and Transplant
Research Unit in organ donation and transplantation:
– Director Prof Andrew Bradley, University of Cambridge;
– Dr Andy Fisher, University of Newcastle;
• Will develop and evaluate novel approaches and technologies that
increase the availability of suitable donor organs for transplantation,
while improving graft survival;
• Key objectives will be:
– improve donor management and evaluate novel interventions in
deceased donors;
– develop novel approaches for assessing donor thoracic and
abdominal organ quality;
– evaluate normothermic ex-vivo perfusion as an approach for
assessing the function of extended-criteria thoracic organs and
kidneys;
– reduce the demand for re-transplantation through improved
understanding of donor/recipient compatibility.
Additional activities in organ donation and
transplantation
• Service activities are funded through Grant-in-Aid:
– Develop budget to support service development
– £75k identified so far to support ex-vivo perfusion study;
• Build upon the successful establishment of the QUOD National
BioBank:
– Commence research studies using collected samples;
– Continue collection of samples;
– Agree cost recovery model through charging for samples;
Cumulative Number of QUOD Donors
289 donors to date
Strategic Goals in other themes (2015 –
2020)
– Delivering clinical trials to support patient blood
management:
–Commission a new trial on the management/diagnosis of
acquired coagulopathy;
– Enhancing our programme of research in
transfusion/transplantation microbiology and virology to
maintain blood, tissue and organ safety:
–Recruit two replacement PIs/leverage work with PHE
– Improve production of cultured blood cells to support a
first-in-man trial (subject to funding)
Two new cross-cutting themes
(2015 – 2020)
– Establishing a Behavioural Research programme to identify
behavioural change interventions which significantly increase
donation and consent rates:
– Establishing a Translational Data Science programme to build
and exploit big data resources which improve our services:
We are already undertaking some
behavioural change research
•
AFFINITIE – NIHR Programme grant on enhanced audit and
feedback to change clinician behaviour (Stanworth)
•
Behavioural Research Strategy Group Established (Chair Sally
Johnson)
–
Initial focus on consent (authorisation) for organ donation
– Commission academic groups through competitive process to
undertake interventional behavioural change studies;
– Aligned to current activities to support Taking Transplantation
to 2020;
– £200k p.a.
Translational data science
• We are a global leader in the use of outcome data in organ
donation and transplantation;
• We are developing capabilities in blood donation and stem cells;
• Can bring benefits in an affordable manner:
– Better evidence-based use of our services by understanding
outcome data from large cohorts of patients;
– Better recruitment and retention of blood donors by
stratification;
– Improved understanding of donor driven component/product
variation;
– Increased retention of donors by maintaining large-research
active cohorts;
Realising the benefits of translational data
science
• Leadership through establishing a new 5-year academic post
• Embed our “Big Data” team in a productive academic environment:
– Critical mass of relevant clinical bioinformatics skills;
– High performance computing environments;
• Relevant governance framework and information security;
• Successful delivery of demonstration projects;
– Seize the opportunity to more fully integrate our data with hospital data to
better understand patient outcomes;
– We need to develop the capabilities to do this;
– £300k p.a.
• Pilot studies of long term outcomes in blood donors and living kidney donors
• Aligned to Personalised health and care 2020: a framework:
– Recently announced by the DH National Information Board:
– Make England a leading digital health economy;
– Develop new resources to support research and maximise the benefits of
new medicines and treatments;
Infrastructure goals
• To provide facilities, infrastructure and resources to support
an innovative research programme
– Buildings, equipment
• To ensure that our workforce have the skills and expertise to
deliver the R&D Programme
–
–
–
–
Clinical fellows
Tenure track?
Research nurses
Equality & diversity opportunities
Post-2015 R&D Programme
Microbiology
First-in-man trial of
cultured red cells
Translational data
science
BTRU – Stem cells
Clinical trial in
coagulopathy
BTRU – Blood cells
(TBC)
BTRU - ODT
Long-term impact
of donation
BTRU - Donors
QUOD National
BioBank
INTERVAL
Donor behaviour
Current status and approval
• Strategic objectives approved by the ET (5th November);
• R&D Committee discussed on 24th November;
• Final strategy and more detailed proposals to be reviewed
by R&D Committee on 6th March 2015;
• Final approval by the Board in March 2015.
Questions?
• The Chairs of Organ Advisory Groups are asked to:
– Comment on the proposals in the strategy;
– Identify ways in which national studies in organ
donation/transplantation could be delivered/funded;
Our Strategic Goals (2015 – 2020)
• Delivering clinical trials to support patient blood management
• Enhancing our programme of research in transfusion/transplantation
microbiology and virology to maintain blood, tissue and organ safety
• Improving the production of cultured blood cells to support their clinical use
• Establishing a Behavioural Research programme to identify behavioural
change interventions which significantly increase donation and consent rates
• Establishing a Translational Data Science programme to build and exploit big
data resources which improve our services
• Enhancing our R&D Programme through the successful delivery of NIHRBTRU objectives
• To provide facilities, infrastructure and resources to support an innovative
research programme
• To ensure that our workforce have the skills and expertise to deliver the R&D
Programme
Inputs/drivers to 2015-2020 strategy:
• Horizon scanning and priority setting by our research
strategy groups;
• Discussions with external stakeholders;
• Changes to NIHR funding;
• Changes to internal R&D funding;
• Changes in the external environment (care.data, 100,000
genomes project, ReGen Medicine);
• Discussions with International blood services;
Research must have a clear link to
improvements in patient care
Successful gene therapy for haemophilia B
“This is a potentially life-changing
treatment for patients with this disease and
an important milestone for the field of gene
therapy” Amit Nathwani
Image: Trial participant Sebastian Misztal
(Credit: UCLH/UCL NIHR Biomedical
Research Centre)