NHSBT R&D Strategy: Advancing Healthcare Research

1. 2015 – 2020 R&D Strategy November 2014 Nick WatkinsAssistant Director – R&D

2. 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

3. We have an academically excellent research programme....... Universities of Liverpool/Leeds University of Cambridge University of Oxford University College London University of Bristol Impact • 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

4. 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.

5. 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

6. 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.

7. 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;

8. 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

9. 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.

10. 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;

11. Cumulative Number of QUOD Donors 289 donors to date

12. 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)

13. 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:

15. 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.

16. 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;

17. 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;

18. 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

19. Post-2015 R&D Programme Translational data science Microbiology BTRU – Stem cells First-in-man trial of cultured red cells Clinical trial in coagulopathy BTRU – Blood cells (TBC) BTRU - ODT Long-term impact of donation BTRU - Donors QUOD National BioBank INTERVAL Donor behaviour

20. 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.

21. 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;

22. 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 NIHR-BTRU 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

23. 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;

24. 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)