UK Experimental research and clinical trials Working in partnership with industry

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New UKTI team – the Life Science Investment Organisation (LSIO) Combines private-sector expertise with Government teams Charged with:

The UKTI Life Science Investment Organisation

Creating focused messaging matching UK

strengths with global business needs

Translating benefits of the UK’s Life Science Strategy and capabilities for business

Catalysing and empowering the

UK Life Science community to work with

international business

UNLOCK YOUR GLOBAL BUSINESS POTENTIAL

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David CameronPrime Minister

Life Science Strategy and Innovation Health and Wealth are the UK government’s response to a global sector under pressure

“In December 2011 I made a firm commitment to re-establish the UK's global leadership in the life science sector, announcing the Government's ten-year Strategy for UK Life Sciences.”

“By more closely integrating the UK's unique strengths, I believe that we can improve healthcare for patients, attract new investment to the UK, and create new jobs and business opportunities in an increasingly competitive and international industry.”

The Strategy makes the most of the UK’s core strengths:

Basic science in universities Translational and clinical research excellence Industry and supply chain The National Health Service (NHS)

The UK is committed to supporting the Life Sciences and Healthcare sector both now and in the long-term future

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Unrivalled data and well-characterised patient cohorts A National Health Service (NHS) with more than 60 million patients

and electronic health records. Clinical datasets, well-characterised patient cohorts, stratification

tools, and biobanks.

Leading translational research facilities In England, £500 million annually spent on National Institute for

Health Research (NIHR) infrastructure to support health research and in particular experimental medicine research.

From complex early stage experimental research requiring innovative trial designs to larger late stage trials, the UK can be your commercial partner.

Translational research expertise with world – renowned KOLs

World-class science, universities, and principal investigators Globally renowned research charities committing £1bn in annual

research funding

The UK Opportunity: Globally Competitive

The UK offers your company access to:


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The UK government has introduced fiscal measures to stimulate innovation and growth for companies that opt to setup and / or operate in the UK

A business environment that supports and rewards innovation

Low corporation tax

rate currently standing at 23

per cent decreasing

further to 20 per cent by April

2015.

Patent Box: 10%

corporation tax on

qualifying profits

23%corporation

tax will drop

further to 20%


£180

m

£300

m

Biomedical Catalyst =

£180m programme of public funding

for growth

To stimulate R&D partnerships between universities businesses & charities

R&D tax credits for SMEs worth approximately 25p on every £1 (7p for large companies)

PBox

SUPPORTING INNOVATION

10%

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rpor

atio

n Ta

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Later-phase clinical research

The UK research ecosystem supports innovation flow from discovery science through to evidence generation and uptake

Translational research centresTranslating biological discovery into the clinical settingTranslational Research Centres including NIHR Biomedical Research Centres and Units, Translational Research Partnerships (TRPs) and Collaborations (TRCs)

Clinical Research NetworksEmbedded in the NHS supporting the delivery of multicentre clinical studies

UK Clinical Research Facilities and Experimental Cancer Medicine

CentresExpertly delivering early stage patient

studies

Medical Research Council and medical research charities.Research funding focused on the mechanisms of health and disease


UK Health dataIn the UK, your business will have access to unrivalled, clinically-coded health data and bio- repositories

Early-phase clinical researchPre-clinical research

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The UK has the visionary science, centres of academic and clinical excellence, key opinion leaders, experienced partners, and the tools to help your business

The UK is home to world-class discovery science..

Identify patient cohorts

Develop sensing and diagnostic technologies

Discover and validate biomarkers

Deliver engineering and informatics solutions

Understand disease biology

Develop novel targets for drug discovery


The Medical Research Council funds disease-focused consortia of academic and industry partners to enable and drive experimental medicine

The Wellcome Trust funds both basic biomedical research and translational projects.

The UK Health Departments fund translational research infrastructure designed to pull innovation from the laboratory into the clinical setting

Cancer Research UK and Cancer Research Technology also provide core oncology research funding, and offer translational funding and expert capabilities, and support patient engagement.

The UK can help your business..

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The UK is home to world-class translational research infrastructure that works in partnership with industry

..with translational research expertise and infrastructure to take your innovation from discovery to the clinic

NIHR Biomedical Research Centres and Units Expert translational researchers embedded in the leading University and hospital partnerships across England

Scotland Leading academic centres of excellence in drug discovery and experimental medicine. For example experimental cancer research expertise at Dundee.

Wales & NI provide industry with leading experimental medicine centres within leading hospital partnerships.

NIHR Translational Research Partnerships Structured partnerships of expert centres working in collaboration with industry on the early and exploratory development of new therapeutics


Access to patient samples and

biobanks

Access state of the art technology

platforms

Access well characterised

cohorts of patients

Conduct fast and efficient

experimental research

Conduct biomarker-led approaches to

patient stratification

Develop and test new therapeutic targets


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UK Clinical Research Faculties support inpatient and outpatient clinical research studies across a wide range of disciplines, led by investigators in both adult and paediatric specialities

Expert clinical research facilities support inpatient and outpatient clinical research


UK Clinical Research Facilities for Experimental Medicine are purpose-built, cutting-edge facilities, with specialist clinical, research and support staff, in locations where universities and NHS Trusts work together on dedicated programmes of patient-orientated experimental medicine research.

UK Experimental Cancer Medicine Centres brings together research experts in cancer biology with clinical oncologists and researchers to speed up the flow of innovations from the lab bench to the bedside. The ECMC network also includes Scotland, Wales and Northern Ireland through local partnership funding with Cancer Research UK.

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Quickly identify the right centres of excellence for a particular study

Conduct cost-effective and smarter clinical research

Design and deliver complex clinical studies

Connect to world-leading principal investigators

Speed up the flow of innovation from bench to bedside

Connect to expert research nurses experienced at running early stage trials


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Oxford Biomedica and the NIHR BRC at Cambridge collaborate on development of ProSavin, a new therapy for Parkinson’s diseaseo “This collaboration with Oxford BioMedica is a bold step forward not only in the use of

an advanced gene-based treatment. I’m most excited to be progressing this vital research with a UK biopharma company.” Professor Roger Barker, Theme Lead for Dementia and Neurodegenerative Disorders at NIHR Cambridge BRC

The NIHR Biomedical Research Centre at Oxford University Hospitals NHS Trust and Life Technologies Corporation is creating the first multi-gene DNA sequencing test to help predict cancer patients’ responses to treatment.o “With their expertise in translating genetics into clinical practice and with several

commercial partnerships with genomics technology companies successfully completed, the Oxford team were the obvious choice.” Josephine Harada, Product Management Leader, Life Technologies Corporation

NIHR BRC in collaboration with Proteome Sciences and Merck Millipore conducted a Alzheimer’s disease biomarker validation study. Preliminary data generated by the NIHR BRC for Mental Health and Proteome Sciences suggest that proteins in blood may act as biomarkers that could help to identify dementia in the pre-clinical phase. o This study is a major step towards a series of simple blood tests that would facilitate

the early diagnosis and management of Alzheimer’s.” Christopher Pearce, CEO of Proteome Sciences

The UK experimental medicine infrastructure in action

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The ECMC Combinations Alliance and AstraZeneca are collaborating on a Phase Ib/IIa clinical trial to examine an experimental drug called AZD4547 in combination with existing therapies . The trial will be run across the UK at ECMCs at the ICR, Imperial, Cardiff , Oxford and Glasgow. o “We believe that collaboration is crucial to finding solutions in the fight against cancer and

are pleased to partner with others in the UK who share our dedication to bringing new medicines to patients.” Andrew Foxley, clinical programme director for AZD4547 at AstraZeneca

Cancer Research UK and GSK have collaborated on a phase I trial, at the Institute of Oncology at St James’s University Hospital in Leeds and Bart’s and The London ECMC in London of GSK's 1070916A, an aurora kinase inhibitor.o “This deal with GSK demonstrates how Cancer Research UK and CRT can work with industry

to speed up the development of anti-cancer drugs that might otherwise remain on companies’ shelves.” Dr Keith Blundy, chief executive of CRT

The £4M Wellcome Trust-Wolfson Foundation Northern Ireland Clinical Research Facility (NICRF) opened in Sept 2013 focussing on cancer, nutrition and metabolism, vision science and respiratory research. This will allow more people than ever before to take part in clinical trials in Northern Ireland.

The UK experimental medicine infrastructure in action


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In depth understanding of disease mechanisms and pathways

Access to patient tissues and clinically annotated biobanks

Identify and validate Biomarkers for clinical study design

Explore and implement patient stratification approaches

Identify and recruit patient cohorts

Mechanism of Action studies

Phase Ib/IIa PoM and PoC studies

Feasibility, set-up and delivery of multi-centre clinical trials

Selected example UK resources

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Industry clinical trial requirements UK

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UK experimental medicine and late stage clinical research: Matching supply with demand


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Clinical Research Networks provide infrastructure to support clinical research in the NHS, giving patients access to high quality research studies and supporting the Life-Sciences Industry to develop potential new treatments

Experimental research can be rapidly and efficiently translated to extensive late stage clinical research networks across the UK

NIHR Clinical Research has a number of networks with therapeutic expertise that co-ordinate clinical trials in Cancer, Stroke, Mental Health, Diabetes, Medicines for Children Dementia and Neurodegenerative Diseases.

From 2010 to 2012, NHS Research Scotland (NRS) has worked with over 183 commercial clients to achieve in excess of 440 projects and 224 clinical-trial feasibility studies. Customers found the overall experience “good or excellent” in 90% of cases.

Through the Northern Ireland Clinical Research Network and Health Research Wales, your business can be connected to expertise and networks to conduct late stage clinical research in these regions


Rapidly set up multi-site studies

Speed up costing and contracting at sites

Identify the right sites drawing on more than 1000 primed investigators

Rapidly set up multi-site studies

Get advice on compatibility of protocols with current UK clinical practice

Access GCP trained nurses to support patient recruitment


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Novartis Accelerates Clinical Trials in Birmingham working within the existing NIHR Cancer Clinical Research Network to establish trials and deliver accelerated results via patient recruitment across 13 UK centres. Early results from Novartis Pharmaceuticals UK Ltd show a 50 per cent reduction in set-up time and significant reduction in costs per patient. Importantly, for the first time ever in its Oncology Unit, Novartis UK managed to secure the first visit by the first patient enrolled in the trial ahead of all centres participating globally.

Roche and the NIHR Medicines for Children Research Network collaborated to tackle systemic juvenile idiopathic arthritis. Roche sponsored a global study; looking at the effectiveness of a drug called RoActemra (tocilizumab) in children aged 2 to 17. The network recruited the entire patient cohort for the centre to time and target. RoActemra is now licenced (and saupported by NICE) for use in the clinic.

“The NIHR Clinical Research Network was helpful in being able to provide a research nurse to support a very quick setup. This meant the site could focus on the study at the right time and led to successful enrolment of eligible patients.” Stuart Bolt, Global Studies Leader, Roche

The UK late stage clinical research infrastructure in action


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In the UK, your business will have access to stable unrivalled, clinically-coded health data and bio- repositories.

…supported by a single health system with unrivalled, clinically-coded health data to drive delivery of clinical research

More than 60 million people are served by a National Health System

Example data resources:o UK Biobank is a unique resource of data and samples

linked to medical histories and health records from 500,000 adult participants

o The Scottish Health Informatics Programme (SHIP) tracks real time clinical information on all 239,000 people with diabetes in Scotland. This has been successfully employed to recruit patients to a range of managed clinical trials.

o NIHR BioResource enables recall to clinical studies by genotype and phenotype and helps stratify and select patients for trials

o Clinical Practice Research Datalink (CPRD) enables observational studies, clinical trial feasibility and protocol optimisation, and post-market surveillance

“We have had a very productive relationship with the Cambridge Bioresource (CBR) over the last two to three years where collaboration has resulted in provision of genetic resource, conduct of clinical and translational trials, and publication of both clinical and immuno-inflammatory mechanistic study results related to pharmacogenetic interactions. The initial interactions between us (industry) and CBR have been very successful and this is a collaboration we are keen to maintain and develop.” Simon McHugh, Director of Scientific Operations at GlaxoSmithKline


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This clinical research excellence is accessible through simplified industry access points.

IN NORTHERN IRELAND:

Health and Social Care Research and Development, funds the Northern Ireland clinical research network:o to support high quality clinical trialso promote research, develop

partnerships o ensure that targets are achieved

and maintained.

IN WALES: NISCHR Clinical Research

Centre (CRC) and Health Research Wales assist companies with the feasibility process, study setup and delivery of commercial research in Wales.

IN SCOTLAND: NHS Research Scotland

Permissions Coordinating Centre and Health Science Scotland (HSS) collaborate as single point of entry for multicentre permission applications. They are dedicated in identifying appropriate clinical trial sites, investigators and patients

IN ENGLAND: NIHR Office for Clinical Research

Infrastructure (NOCRI) ensures the companies can rapidly engage with multiple research centres and that studies are set up and delivered quickly and efficiently.

Through NOCRI or directly through the NIHR Clinical Research Networks, the UK’s late-phase clinical research infrastructure can be accessed quickly and efficiently.


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Government commitments:– Opening up the NHS to collaborate with your business on research and adoption– Ensuring that products and services are critically appraised to the highest standard

Transparent expert advice to support decisions on access to personal health information A streamlined health research approvals process Globally-respected regulators: The UK is home to the MHRA and the EMA Earlier Access and Adaptive Licensing are under consideration An expert stakeholder group meets quarterly to discuss regulatory innovation MHRA recently established an Innovation Office to promote early dialogue between the

MHRA and companies developing innovative products including: advanced therapies, stratified medicines, nanotechnology, advanced manufacturing, and novel drug/device combinations

Creating an Open and Flexible Regulatory Framework


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Our practical help and advice is free and confidential UKTI’s professional trade and industry advisers operate in the UK alongside a global network of experts based in all British diplomatic offices overseas. Links with centres of excellence (e.g. universities) Information on tax, regulatory and business planning issues Information on financial incentives if applicable Information on staff recruitment Site and Property search assistance Building key contacts – we can provide introductions to service providers, local, regional and

national government and trade organisations. Aftercare through on-going support Maximising your global potential – Once you are established in the UK, we can help your

company to take advantage of new business opportunities and branch out to new locations – both in the UK and overseas.

How UKTI can help you work with and establish new connections in the UK


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UK Experimental Research and Clinical Trials Working in Partnership with IndustrySuccess Stories

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A collaboration between GlaxoSmithKline, North West e-Health (NWeH), The University of Manchester, Salford Royal NHS Foundation Trust, NHS Salford’s local general practitioners, and local community pharmacists.

Testing the safety and effectiveness of a new treatment for asthma and COPD.

Draws on Salford’s e-Health records infrastructure, a clinical information system that provides a single, integrated electronic patient record across primary and secondary care.

Is recognised as a world first for the use of such data.

The Salford Lung Study: GlaxoSmithKline Tests the Effectiveness of Pre-License Medicine Using Real World Data in Manchester

“This study is a first in the world, testing a pre-license medicine in a real world setting and is a tribute to the partnerships we’ve created together, our collaborators and the health care professionals and people of Salford.” Dr David Leather, Medical Director of GSK’s respiratory Centre of Excellence


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The first medicine to treat the underlying cause of cystic fibrosis The NIHR has been supporting Vertex Pharmaceuticals

Incorporated (Vertex) to undertake a comprehensive and robust clinical and economic evaluation of KALYDECO™ (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis, for people with a specific genetic mutation.

The NIHR Royal Brompton Respiratory Biomedical Research Unit (BRU) was involved in the UK adult and paediatric clinical trials of KALYDECO which were carried out in the BRU funded Clinical Research Facility with additional support from the NIHR Medicines for Children Research Network.

Simon Lem, Country Manager, UK, Ireland and Scandinavia for Vertex said: “We are pleased to have worked with the NIHR’s clinical researchers and to have received the decision that the NHS will fund KALYDECO – this is great news for patients

Dr Jane Davies, honorary consultant in paediatric respiratory medicine at the NIHR Royal Brompton Respiratory Biomedical Research Unit said: “Kalydeco changes the way cystic fibrosis can be treated because for the first time, clinicians are now able to target the underlying cause of the disease, instead of just the symptoms and complications.”

Conducting a comprehensive and robust clinical and economic evaluation of KALYDECO™ (ivacaftor)


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Successfully concludes an alzheimer’s disease biomarker validation study: Preliminary data generated by the NIHR BRC for Mental Health

and Proteome Sciences suggest that proteins in blood may act as biomarkers that could help to identify dementia in the pre-clinical phase.

The capability to distinguish between the levels and progression of Alzheimer’s disease will enable drug developers to substantially improve patient care and people with mild cognitive impairment are likely to benefit the most from such disease-modifying drugs.

Professor Simon Lovestone, Director of the NIHR Biomedical Research Centre for Mental Health commented: “This is the largest study of plasma biomarkers to date that we are aware of, and builds on our earlier findings to identify diagnostic and prognostic signals in plasma. As new treatments for Alzheimer's disease are being developed, there is an increasing need for accurate and accessible markers of disease severity and progression. If our results are positive, we will move quickly to support the development of clinical tests based on these biomarker panels.”

Christopher Pearce, CEO of Proteome Sciences, said: “This study is a major step towards a series of simple blood tests that would facilitate the early diagnosis and management of Alzheimer’s. “

NIHR BRC in collaboration with Proteome Sciences and Merck Millipore


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Collaborate with industry to develop the world’s first multi-gene sequencing test for predicting cancer patients’ responses to treatment The world’s first multi-gene sequencing test for predicting cancer patients’

responses to treatment on the NHS has been launched in Oxfordshire in a partnership between Oxford University Hospitals NHS Trust and the University of Oxford.

The development of the test, which looks at 46 genes, has been co-funded by the TSB through a grant to NIHR Oxford Biomedical Research Centre (BRC), with other funding from Life Technologies Corporation, AstraZeneca and the Johnson & Johnson company Janssen Research & Development.

The test is performed on tumour tissue using Life Technologies’ Ion Personal Genome Machine at the BRC Molecular Diagnostics Centre lab in Oxford. The test costs around £300 but will save the cost of using expensive drugs in patients who will not respond.

A version of the test that involves around 150 genes is now being considered and it is hoped will be rolled out the 46 gene test more widely in the NHS at the moment.

NIHR Oxford Biomedical Research Centre (BRC)


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Led by Leukaemia & Lymphoma Research, the Trials Acceleration Programme (TAP) gives haematological oncology patients in the UK accelerated and wider access to early stage trials.

Working within the existing NIHR Cancer Clinical Research Network, TAP’s dedicated centralised management at the University of Birmingham minimises ‘red tape’ to establish trials and deliver accelerated results via patient recruitment across 13 UK centres.

Early results from Novartis Pharmaceuticals UK Ltd show a 50 per cent reduction in set-up time and significant reduction in costs per patient. Importantly, for the first time ever in its Oncology Unit, Novartis UK managed to secure the first visit by the first patient enrolled in the trial ahead of all centres participating globally.

In its initial year of operation TAP has ratified nine new trials including collaborations with eight pharmaceutical companies.

Novartis Accelerates Clinical Trials in Birmingham


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Created a data management platform to improve the understanding of mental health genetics and treatment

The centre, hosted by Cardiff University, has established the Wales Mental Health Network and is recruiting 6,000 volunteers over the next two years and will capture more than 300 clinical attributes for each person

IDBS’ platform will integrate clinical, biobanking and genetic data, providing a data management and analytics system for this and future studies

National Centre for Mental Health in Wales Partners with IDBS


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DestiNA Genomics, a spinout of the University of Edinburgh, is commercialising a revolutionary chemical-based system to detect nucleic acids and single nucleotide polymorphisms. The technology is unique and distinguishable from all existing enzymatic methods.

Scotland’s electronic records and ‘cradle to grave’ medical histories enable drug safety studies, clinical trials, biomarker discovery, and tracking well-phenotyped patients over time. Example: The national diabetes register, SCI-DC, tracks real time clinical information on all 239,000 people with diabetes in Scotland, updated daily.

Other key experimental medicine centres include:o Drug Discovery Unit (DDU) at University of Dundee. The

DDU offers drug discovery capabilities to translate biomedical research into candidate therapeutics.

o Kosterlitz Centre at University of Aberdeen. The centre acts as a hub linking the biomedical sciences to biomedical imaging, medicinal chemistry, clinical research and biopharmaceuticals.

o Rowling Centre at University of Edinburgh. The centre targets the discovery of treatments that will slow the progression of disease such as MS and MND.

o Wolfson Wohl Cancer Research Centre at the Beatson Institute, University of Glasgow. This international centre of excellence in the fight against Cancer incorporates expertise in genomics, biomarkers, target validation, drug development, pathology and preclinical trials.

Highlights from Scotland


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Four health boards in Scotland have teamed up with Pfizer to become the company’s second INSPIRE site driving clinical research in the UK: o The INSPIRE programme is a strategic network of sites

identified as highly productive and effective in conducting clinical trials.

o These sites have to meet strict criteria such as running trials to the highest standards within set timelines; ensuring dedicated, high-quality staff and resources; and expertise in key disease areas targeted by Pfizer’s R&D pipeline.

Working together for our nation’s health is key as we strive to achieve a more positive environment for life sciences in the UK, and it will play a key role in further strengthening the research culture within NHS Scotland.”

Dr David Montgomery, Oncology Medical Director, Pfizer Oncology UK

Farr Institute. The new institute is an interdisciplinary eHealth Informatics Research Centre, as an integral part of the UK-wide network. The institute is leveraging Scotland’s investment in electronic health records. Medical, population and computer scientists will combine their expertise to interpret large and complex health datasets in research environments that safeguard patient confidentiality.

Highlights from Scotland continued..


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The new Northern Ireland Centre for Stratified Medicine – the only one of its kind in the island of Ireland:

o Puts University of Ulster and the Western Health and Social Care Trust at the frontier of pioneering medical research into chronic degenerative diseases.

o The £11.5m investment will facilitate research that can better identify drugs or treatments that meet the specific needs of individual patients –and marks a significant advancement in this strategic research area.

In Northern Ireland a new Electronic Care Record has been developed which will ensure that no matter where you are being treated or cared for in the Northern Ireland Health and Social care system.

Cardiologists from both the Royal Victoria Hospital and Ulster Hospital Belfast can stratify patients for their suitability of a pioneering new technique. The technique uses specialised CT imaging to map heart disease progression which allows arteries to be visualised and determined for their suitability for this new procedure.

Highlights from Northern Ireland


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Novo Nordisk and Swansea have collaborated on 8 clinical trials in diabetes. In 7 of the those trials, recruitment was on time and target. One study was stopped early by the Company.

In all 8 studies, Swansea was able to recruit above the UK average number of patients per site.

In addition, approval times for the site in Swansea were below the national average (68 days v 85 days).

Results indicate successful research collaboration from feasibility and setup through to execution and delivery of results

Novo Nordisk collaborates with Swansea University on eight clinical trials in diabetes

UK Experimental research and clinical trials Working in partnership with industry

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