The NIHR NIHR@Leeds; drivers of change Professor Stephen Smye Acknowledgements; Professor Dame Sally Davies FRS, Chief Medical Officer Professor Chris Whitty, Chief Scientific Adviser, DH Dr Louise Wood, Director of Science, Evidence and Research Dr Jonathan Sheffield, CEO, NIHR CRN Professor Peter Bower, NIHR CRN National Specialty Lead for HSR Exciting environment Expanding research environment Primary, seconday, end of life care as well as community setting

Building on 10 remarkable years The National Institute for Health Research (NIHR) is funded by the Department of Health to improve the health and wealth of the nation through research. The NIHR plays a key role in the Government’s strategy for economic growth, attracting investment by the life-sciences industries through its world-class infrastructure for health research. Together, the NIHR people, programmes, centres of excellence and systems represent the most integrated health research system in the world.

NIHR@Leeds; drivers of change Science Distribution of research Training System & Process ODA Life Sciences Industry Exciting environment Expanding research environment Primary, seconday, end of life care as well as community setting

Emerging science & health (Gen)omics Stratified medicine Regenerative medicine Government’s 8 Great Technologies – 2 have direct links to DH priorities (genomics & regenerative medicine). All these (and more) are relevant aspects of converging science & technology for health – no single technology but potentially important improvements to current approaches to prevention, diagnosis and treatment. DH Strategy Unit leading some work considering :- sensors & wearables; 3D printing; Robotics; Implantable / ingestible technology; synthetic biology, nanotechnology; blockchain & distributed ledgers. (Gen)omics – all genes, proteins and other biomarkers in a patient or even their microbial flora. Dependent on advances in gene sequencing, big data and computing to understand “systems biology”. Applications include: Large population genomics initiatives like 100,000 Genomes Project to embed genomic medicine in routine healthcare Liquid biopsy / single cell sequencing (cancer, prenatal diagnosis) Lab-on-a-chip, rapid near patient testing for AMR, diagnostics & drug prescribing Microbiomics - links between (gut) microflora and chronic health conditions including obesity Regenerative medicine – new advances in stem cells, gene therapy and gene editing to repair & replace tissues, applications include: Treating rare / degenerative disease, improved joint & limb repairs, artificial tissues & organs (3D printing) Immunotherapy for cancer – CAR-T cells are showing huge promise Gene editing – huge breakthroughs for basic science, adult gene therapies BUT debate over embryo research and germline therapies Stratified/Precision Medicine – A move away from ‘one size fits all’ medicine to diagnostic tests, genomic technologies and data analytics to target therapies to achieve the best outcomes in the management of a patient’s disease or predisposition to disease Improved understanding of diseases mechanisms More precise diagnosis involving panels or tests Therapies based on rules – likely response Pharmacogenetics - right dose, right drug, first time Deliver more targeted, predictable and effective outcomes

National Institute for Health Research: research infrastructure Invention Evaluation Adoption Early-phase clinical Research Late-phase clinical Research NIHR Biomedical Research Centres EM infrastructure in matched to excellent infrastructure Simplify NIHR Clinical Research Facilities Experimental Cancer Medicine Centres Med Tech and IVD Cooperatives Bench to bedside and back. Supporting life sciences industry, whether CRO, pharma, med tech- small or large. The whole infrastructure is there to support the product lifecycle. NIHR Clinical Research Network Collaboration for Leadership in Applied Health Research and Care

Trials Adaptive N-of-1 Real world “..an adaptive design clinical study is defined as a study that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data (usually interim data) from subjects in the study” http://www.fda.gov/downloads/Drugs/Guidances/ucm201790.pdf N-of-1 “ a key component will be transforming everyday clinical care into solid N-of-1 trials” Schork NJ 2015 Nature 520, 609-11 Real world Exciting environment Expanding research environment Primary, seconday, end of life care as well as community setting

Applied Health Research - New Models of Care Multispecialty Community Providers - moving specialist care out of hospitals into the community Integrated primary and acute care systems  - joining up GP, hospital, community and mental health services Acute care collaboration - local hospitals working together to enhance clinical and financial viability Enhanced health in care homes  - offering older people better, joined up health, care and rehabilitation services Urgent and emergency care  - new approaches to improve the coordination of services & reduce pressure on A&E departments. Charles Tallack, NHS(E)

Public health Exciting environment Expanding research environment Primary, seconday, end of life care as well as community setting

Must take account of distribution of need Must take account of distribution of need. Dementia in women (L), heart disease (R)

Population 85 and over: 1992, 2015, 2033 (ONS).

The NIHR: Public “Around three-quarters of the public say they would be willing to share their anonymised medical records, or their anonymised genetic information, for the purposes of a medical research study. The primary concern among those who are unwilling relates, perhaps unsurprisingly, to confidentiality and privacy…..” Exciting environment Expanding research environment Primary, seconday, end of life care as well as community setting

Methodological expertise Precision Medicine Research Infrastructure The Leeds Centre for Personalised Medicine and Health Dissemination& Adoption Clinical Efficacy & Utility Safety & Clinical Validity Preclinical & Measurement Validity “The Push” New Technology Drugs Devices Diagnostics “The Pull” Clinical Need NHS Academia Industry Stakeholders Early Modelling (Commercial and NHS opportunity) Patient Benefit Economic Value Methodological expertise Health Economics, Health Informatics, Medical Statistics, Metrology, Systems Biology, Quality Management, Artificial Intelligence Precision Medicine Research Infrastructure Patient Cohorts, Biobanks, “Big Data”, Analytical Platforms (e.g. Omics), High Performance Computing Cost-Effectiveness & Market Approval Data Science