2015 2020 RD Strategy Nick Watkins Assistant Director

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

Universities of Liverpool/Leeds University of Oxford University of Cambridge Impact We have an academically excellent research programme. . . . University College London University of Bristol Our 17 Principal Investigators: • World leaders in their fields; • > 1, 000 publications over 10 years; • 9 patents filed. 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 d. Cell 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/? Gi. A • 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 – £ 75 k identified so far to support ex-vivo perfusion study; • Build upon the successful establishment of the QUOD National Bio. Bank: – 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; – £ 200 k 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; – £ 300 k 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 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 Bio. Bank INTERVAL Donor behaviour

Current status and approval • Strategic objectives approved by the ET (5 th November); • R&D Committee discussed on 24 th November; • Final strategy and more detailed proposals to be reviewed by R&D Committee on 6 th 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, Re. Gen 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)