Central regions’ event

30 October 2015

Shaping personalised

medicine in the NHS

Housekeeping

Earth Air

Fire Water

A personalised medicine strategy for the NHS

Professor Sue Hill OBE

Chief Scientific Officer for England, NHS England

Medicine became a science by

combining clinical observation

with pathology and function [physiology]

and through the application

of the chemical, biological

and physical sciences

Personalised medicine

– its foundation in modern medicine

Sir William Osler, (often dubbed ‘the father of modern medicine’)

recognised the importance of a personalised approach

to medicine more than 100 years ago

It is much more important to know what

sort of a patient has a disease than

what sort of a disease a patient has.

He also recognised the mechanism to get there

Personalised medicine is a move away from a ‘one size fits all’ approach to the treatment and care of patients with a particular condition, to one which uses emergent approaches in areas such as diagnostic tests, functional genomic technologies, molecular pathway, data analytics and real time monitoring of conditions to better manage patients’ health and to target therapies to achieve the best outcomes in the management of a patient’s disease or predisposition to disease.

Defining personalised medicine

Increased efficiency

through coupling of therapy

with companion diagnostic

eg Herceptin Identification of those groups

interventions are effective

– makes sub-population

treatments more economic

Personalised medicine

Prognostics to predictdisease

Targeted prevention &

new strategies

More precisediagnoses

Stratification & therapies

for population subgroups

Theranostics – combined

diagnostics & therapy

More participatory

role for patients

The future vision for personalised

medicine in the NHS

• This personalised approach addresses the aims of

the Five Year Forward View and the future challenges

and priorities for the health system.

These include:

― improved prevention based on underlying predisposition

― earlier diagnosis of disease as a result of identifying

abnormality earlier

― more precise diagnosis based on cause

― targeted interventions through the use of companion diagnostics to

identify and stratify effective treatments

• 5YFV recognises that NHS sustainability requires ‘revolutionary

change in service provision’ – informed by research and

innovation, with a new focus on prevention and earlier

detection of disease and delivery by new ways of working.

Delivering the aims of the

Five Year Forward View

• ‘One size fits all’ care particularly struggles with much of the chronic inflammatory and degenerative disease facing society, These non-communicable diseases are increasing as survival improves

• Life style influences are creating a time bomb in non communicable diseases for example in cancer, obesity, diabetes, hypertension

• Drug treatments based and developed through the ‘one size fits all’ business model are ineffective in between 30 and 60% of patients (current NHS drugs bill is over £12 billion)

• Adverse reaction to medicine is responsible for1 in 15 hospital admissions

Issues in disease burden and drug efficacy

Condition Efficacy

Rate (%)

Alzheimer’s 30

Asthma 60

Diabetes 57

Hep C 47

Cancer 25

Osteoporosis 48

Rheumatoid arthritis 50

Schizophrenia 60

Limits of pharmaceutical effectiveness

Issues in drug development

New possibilities through

technological & informatics advances

Advances are opening up a range of new

possibilities:

• The explosion of new technology to interrogate complex cellular

processes –the multiomics (genomics, transcriptomics,

proteomics, epigenomics, microbiomics, metabolomics)

and the exposome

• New non-hierarchical approaches to phenotyping complex

disease (eg cluster analysis, machine learning)

• Applications of informatics to interrogate large data-sets from

biological collections, clinical trials and linked population-based

case records and prescribing practice

Data TypeLarge-scale Structural Changes

Balanced Translocations

Distant Consanguinity

Uniparental Disomy

Novel / Known Coding Variants

Novel / Known Non-

coding Variants

Targeted gene

sequencing

SNP+ arrays

Array CGH*

Exome

Whole Genome

+ Single Nucleotide Polymorphism * Comparative Genomic Hybridisation

100,000 Genomes Project and

Whole genome sequencing

10,000

100,000

1,000,000

10,000,000

100,000,000

1,000,000,000

10,000,000,000

0 0.5 1 1.5 2 2.5

GenotypingWhole genome3.3bn basesBoth exons and introns

Exome 10m basesExons only

Panels <10m bases

Subset of exons

The 100,000 Genomes Project

in practice

Diagram courtesy of Nature

The emergence & rapid evolution of

multiomics technology platforms

‘Next’ Genomics

The multiomics

cascade

The importance of

broader clinical context

Genome sequencing in the absence of other diagnostic and clinical information is insufficient

Each ‘healthy person’ has

• 22,000 genetic variants

• 9,000 non-synonymous genetic variants

• 100 rare missense genetic variants

• 5 rare protein-truncating genetic variants

• 0-2 de novo variants

Rare genetic variation is not necessarily pathogenic variation

Phenotypic clinical data and diagnostics are crucial to making sense of the genomic data

Integrated diagnostic data – the

foundation of the new approach to healthcare

• The millions of diagnostic, scientific and other interventions each year generate an enormous amount of clinical data

• Integration and analysis of this data will enable the clinical characterisation required to deliver personalised medicine – improving outcomes, particularly around non-communicable disease

• This coordinated dataset provides huge opportunities for new insight, analysis, interventions and prognostic possibilities as well as for machine learning and will be centralto future healthcare

Imaging

Biochemical &

biomarker data

Tissue

samples

Physiological

tests

Other clinical

measurements

Genomic sequence

data

Multiomics information

Eg metabolomics,

epigenetics

relevant data collected

as a series over time

Economic, social &

population data

Harnessing population information

for patient benefitCreation of a new taxonomy of medicine requires an Information Commons in which data on large populations of patients become broadly available for both clinical and research use and a Knowledge Network that adds value to these data by highlighting their inter-connectedness and integrating them with evolving knowledge of fundamental biological processes

Reclassification of human disease by identifiable causal pathways

Strategic approach -tailoring treatment & management to a patient’s individual makeup

‘One size fits all’

treatments &

intervention

• ‘One size fits all’ treatment based

on symptoms

• Organ/ speciality organisation of

services and professions

• Limited use of genomic and

molecular markers

• Diagnostic and other clinical data

not linked

• New taxonomy of medicine based on underlying cause and personal response

• Comprehensive linked diagnostics to give a full picture of patient

• Tailored, more-effective therapies for better outcomes

• Integrated clinical services taking a ‘whole body‘ approach

Now 2020

Individually-

tailored

approach

Increasingly precision interventions based

upon carefully identified subgroups within the

broader population

18

Improving outcomes through personalisation

Targeted disease preventionIdentification of predisposition markers or

underlying processes can predict future disease

Early disease detection2-8 yrs before onset & symptoms become

obvious with low cost stratification

Accelerated diagnosisbased on underlying cause and incidental findings – rather than

just grouped symptoms

Targeted therapyIdentification of effective personalised treatments

Improves outcomes

• Greater efficiency from streamlined care pathways • Earlier and more precise diagnosis and treatment• Fewer and less complicated surgical interventions• Fewer patients getting cancer and other diseases

£

www.england.nhs.uk

Personalisation in practice

- management of genetic diabetes

Basic details (eg BMI)

& simple pathology

(eg HbA1c)

Probability calculation

Urinary C-peptide creatinine ratio

testing/ Antibody testing for pancreatic

autoimmunity (rules out Type 1)

Genetic testing

Permanent diabetes and

developmental delaySulphonylurea therapy

Wolcott Rallison

SyndromeLiver Transplant

IPEX syndrome Bone Marrow Transplant

Syndromic pancreatic

agenesis

Insulin and exocrine

supplements

Multi-organ autoimmune

disease ? STAT3 inhibitor

KCNJ11 p.V59M

EIF2AK3 p.E371*

FOXP3 c.227delT

GATA6 c.1448

-1455del

STAT3 p.T716M

DIAGNOSIS TREATMENT

Combination of phenotypical characterisation (including established diagnostics)

with genomic testing identifies precise diagnosis and treatment options

Molecularly stratified cancer care

20

Antibody

Therapy

Small

Molecules

Clinical Trials

Novel drugs

Chemotherapy

Radiotherapy

Addressing the dementia challenge

• Stratification is not limited to prediction of response.

• Grouping patients by diagnosis, prognosisetc. can also enable greater mechanistic insight and support the assessment of novel stratification hypotheses.

• Dementias pose a critical societal challenge and suffer from a paucity of therapeutic options.

• MRC has established the Dementias Platform UK in order to:

― Stage the progression of Neurodegeneration(ND)

― Identify the physiological and molecular drivers of ND progression

― Join up mechanistic understanding across ND in a whole body context

― Translate into therapeutic & public health interventions

Tackling adverse drug reactions

• Adverse drug reactions are responsible for 1 in 15 hospital admissions

• Characterising an individuals immune system can inform potential issues

Associations of Serious Adverse Drug Reactions with HLA Alleles

Diagram courtesy of Wolfson Centre for Personalised Medicine,

Liverpool & MRC Centre for Drug Safety Science

….back to basics?

• Significant variation in commissioning of

all diagnostics in the current system

• Evidence from Atlas of Variation and CSO

Commissioning Information project

• Examples of NICE recommended diagnostics but not not

universally adopted

― Faecal calprotectin (abdominal pain pathway)

― Natriuretic peptide (heart failure pathway)

• Quality requirements for diagnostic services need to be

explicit in commissioning and contractual requirements

Unwrapping the complexity of

current pathways & commissioning

Developments in diagnostic provision

- changes in the ‘where & when’

FUTURE

DIAGNOSTIC

PROVISION

At home or in the

High Street

Tele-monitoring

of individuals

Local Diagnostic

Hubs

Networked

Services

Tele-reporting & image/ data

sharing

Centralised Diagnostics

Hubs

Multimodal and

Multifunctional

Divergent evolution driven by scientific and technological advance moving at pace

Scientific & technological advance and

innovation driving changes in provision

Continuum of Delivery

linked through IT

Centralisation

(‘Big Science’)

High-capital investments

delivered at small number

of locations driven by

- robotic platforms,

- molecular diagnostics for

human and microbes,

- multimodality imaging,

- highly specialised

physiological investigations

and models,

- remote monitoring and

telereporting etc.

Enables:

• Scale

• Efficiency

• Centralised delivery

Minaturisation

Devices that are carried

by/within patients or close

to patient as part of

patient centered

localisation with bio-

nanotechnology driving

point of care and hand

held testing devices

measuring multiple

pathological and

physiological parameters,

Capsule endoscopy, frugal

technologies

Enables:

• Near-patient & self-

testing and home

monitoring

• Distributive model

including high street

provision and within

GP surgeries

PLUS:Health apps &

telemonitoring

Multiomics

– inc proteomics

& metabolomics

Combined imaging

eg PET/MR

Spearheading new ways of working - the NHS GMC legacy

NHS GMCs

Requires input across many

clinical specialities –much more

multidisciplinary coordination

Standardisation of laboratory

protocols and practice to ensure

national comparability

Capture of data from multiple systems and

bringing together against common

datasets and standards

A step change in the processing and analysis

of information

Pioneering network structure

- with lead organisation & LDPs to serve

their population

Consent and PPI Involvement

Aligning our advances against 5

year forward view

• Across the diagnostic and care continuum from Point of care to high-throughput technologies

• The right tech in the right place with consolidated expertise• Underpinned by responsive commissioning framework & clear economic case• Driven by engaged clinical leaders

New models of care

• Improvement across all diagnostics

• Linkage systems for diagnostic and clinical data

• Consolidation of services where appropriate

• Changes driven by contractual and other levers

Existing services

• Adoption of cutting-edge technology linked to role of AHSNs

• New insights through sharing and analysis of data

• Knowledge base to drive clinical practice & scientific research

Science & innovation

• Alignment of initiatives outside of NHS England

• Responsive regulatory and advisory system

• Clear guidance and support for commissioners and providers

• Support for workforce developments

Policy & Strategy

Recognising the factors in adoption

of innovation and clinical change• Where changes are cost saving for a given trust or commissioner

they may take place at any time

• Adoption of changes which are cost-increasing for individual organisations but health-improving are influenced by guidance, price setting, contracts and operational provider decision making

Working across the landscape

NHS England’s role is about setting the strategy and an integrated work programme with each part of the system having a role to play in implementation

DH

HEEGenomics England

Other Professional

Bodies

NHS England

NHS Commissioners

NHS Providers

Higher Education

NICE

Charities and patient groups

MHRA

AHSNsMed Tech &

Pharma

NHS Improvement (Monitor/TDA)

Medical Royal

Colleges

The

economic

case for

Personalised

medicine

Greater prognostic

identification

Earlier diagnosis

Timely treatment reduces

diagnostic cycles and

patient visits

Reduced cost of ineffective medicine

Reduced costs of adverse drug

reactions

Lower drug development

costs as target groups better

identified

Given the

growing

burden of

disease,

we need to

manage and

mitigate

the risks of

inaction

• Ambition: How fast, and how far, should we move to a personalised NHS?

• Implications: What will the impact be on those parts of the system which you know best?

• Challenges: What has to be in place to allow us to move forward?

• Strategy: Who needs to work with whom to make this happen? What should NHS England do (that only it can do)?

Issues for consideration

Personalised medicine: considerations for providers –

a laboratory and personal perspective

Professor Michael Griffiths

Clinical Director, Genetics and Pathology Directorate,

Birmingham Women’s Hospital NHS Foundation Trust

Personalised medicine - definitions

• National Institutes of Health (NIH):

– Personalised medicine is an emerging practice of medicine that uses an individual's genetic profile to guide decisions made in regard to the prevention, diagnosis, and treatment of disease

Personalised medicine – wider defn

• National Cancer Institute (NCI):

– A form of medicine that uses information about a person’s genes, proteins, and environment to prevent, diagnose, and treat disease. In cancer, personalized medicine uses specific information about a person’s tumor to help diagnose, plan treatment, find out how well treatment is working, or make a prognosis.

Concept

• Not treating all patients the same

• Selecting optimal treatment for each patient

• Driven by tests

More than

• More than genetics

• other omics, other diagnostics

• More than cancer

• But will focus on these

• More than simple companion diagnostics (CDx) and targeted therapies

• Is already happening – two non-CDx examples……

Example: IGH/TCR clonality for MRD in ALL

• Acute Lymphoblastic Leukaemia (ALL) – clonal rearrangements of the IGH and TCR genes– unique to each patient

• Testing involves:– PCR and sequencing of the IGH and TCR regions– Design of a patient specific assay– Use of the assay to measure Minimal Residual Disease (MRD) after initial

treatment (d29).– Level of MRD stratifies patient for subsequent treatment

• More intense • Less intense (within trial)

• Example of a truly personalised test determining treatment

http://www.2minutemedicine.com/

Example: Chronic myeloid leukaemia (CML)

• Caused by a novel gene – formed by fusion of two normal genes via translocation– BCR-ABL1

• Average survival 3-5 years until introduction of first Tyrosine Kinase Inhibitor (TKI) therapy in 2001.

– Imatinib (Glivec)

• Most patients respond well and have normal lives.• Still need additional treatments:

– Some patients are intolerant to imatinib– Some patients develop resistance due to additional mutations in the BCR-ABL1 gene

• 5 TKIs now available– Different sensitivity to resistance mutations– Different side affect profiles

• impact of patient co-morbidities

• Availability of choice important• Me too drugs relevant• Typically only one at a time

– – so rationing availabiltiy

TKI F359V E279K

Imatinib: Partially sensitive Partially sensitive

Nilotinib: Partially sensitive Partially sensitive

Dasatinib: Sensitive Sensitive

Bosutinib: Sensitive Sensitive

Ponatinib: Sensitive Not yet known

http://www.medical-labs.net/

Monitoring response

M244V NilotinibImatinib

Patient with initial responseIncreasing levels prompted analysis for resistance mutations – M244VPositive response to switch to alternative TKI - Nilotinib

• Blue line shows level of disease

• Pink line sensitivity of test • Overlapping lines - no

detectable disease

Patients can be complex

Bosutinib

Nilotinib

Imatinib Dasatinib

Y253H Y253H

Patient with long history of CMLLost response to imatinib – mutation responsible identified – Y253HGood response to Dasatinib, but discontinued - intolerantNilotinb, as expected, not effective (based on Y253H)Good response to Bosutinib, but discontinued – intolerantConsidered unfit for transplantOff TKI and disease returning – still has Y253H

Limited options:• Imatinib or Nilotinib

could provide sub-optimal therapy despite Y253H

• Ponatinib - but only approved on CDF for T315I mutations

Companion diagnostics (CDx)

• Companion diagnostics - a therapy linked to a specific test– E.g. HER2 status for Herceptin in breast cancer

• Issues:– Requesting the test implies choosing right patient for drug– Alleged cost savings based on restricting expensive therapies

• But test “negative” patients still need treating

– Increasing portfolio of therapeutics• Multiple single test CDx not scaleable or sustainable (or affordable?)• “negative” patients will have alternative therapies

– Really need panel based profiling of all CDx targets• Choice of therapy not restriction• Right drug for patient (not right patient for drug)

• Not cost saving but optimal use of resources, improved outcomes

Example: Matrix trial

• Trial linked to CRUK Stratified Medicine Programme (phase 2)

• Lung cancer• Genetic testing based on 28 gene panel

– Nationally co-ordinated through three Tech Hubs

• Mutation status for eligibility for (currently) 8 arms of the trial

• Small numbers in each arm looking for large effects

• Flexible – relatively easy to add additional arms

PM - Not just CDx and targeted therapies• More accurate diagnosis

– ultimately moving towards genomics classification of cancer

• Prognosis– Patient centred – important information that patients want– Stratification

• Stratification for generic therapy– High risk, Standard risk, Low risk– May be treated differently e.g. Acute Myeloid Leukaemia (AML)

• Selection for targeted therapies (CDx)– But the right therapy based on

• Tumour characteristics• Patient characteristics

– Co-morbidities– Pharmaco-genomics

• Monitoring– CML examples already shown – potential for all leukaemia, and all cancer!

• Surveillance– Early detection of relapse/recurrence – re-treat while well– Pre cancer surveillance in high risk populations?

Cell free circulating tumour DNA (cfDNA)

• Present in plasma (urine, etc)– Transient, but constantly replenished– Product of cell death

• Alternative source for characterisation of tumours– non-invasive– reducing heterogeneity issues

• Will allow monitoring of solid tumour burden– similar to that in leukaemia

• Biological tool to rapidly assess response (days/weeks) rather than long term outcomes (years)

Perceived barriers?• Commissioning mechanisms?

– Inconsistent funding of molecular diagnostics in cancer– PBR - retrospective counting – anti-growth– Little, if any, anticipation

• Anticipation?– Invest in infra-structure and training in advance of need– Training of clinical scientist in genetics has decreased since MSC

• Absence of test approval mechanisms?– UKGTN available for rare disease test approval– Need similar mechanism (not NICE) for cancer molecular diagnostics

• Evidence to support test commissioning?– Evidence to support approval of tests

• does not need to be as rigorous as for therapeutics– Commissioning should be proactive (as well as anticipatory)

• commission evidence including Health Economics• diagnostic services don’t have the resources• diagnostics industry – different financial capacity to pharma.

– Industry – still needs to support pump-prime projects to create evidence of utility

• Trials?– Real world data on utility of tests may be more relevant than trial data – pilot implementation projects

Opportunities

• Patient benefits– Improved outcomes– More effective use of resources

• Potential for trial redesign– e.g. Matrix

• Industry – sponsored evaluations– NHS reluctance to support new tests without evidence

• Implement routine genomic profiling of tumours– Create the infrastructure to deliver PM– Create the evidence to accelerate impact of PM– UK could become a test bed for trials

• Routine identification of patient eligibility for biomarker driven trials

• Increase access to latest therapeutics (free?)• Impact on wealth creation – platform for diagnostics

Personalised medicine: considerations for providers

Hilary Fanning

Deputy Director of Delivery and Director of Research,

Development and Innovation, University Hospitals

Birmingham NHS Foundation Trust

Future state?

Challenges

Paving the way…

Personalised medicine: considerations for providers

Realising the potential of stratified medicine

Professor Charles Craddock

Director of BMT Unit and Professor of Haemato-

oncology, University Hospitals Birmingham NHS

Foundation Trust

The DNA revolution has transformed our understanding

of the molecular basis of human disease

Watson and Crick solved the structure of DNA in 1953

Brian Druker

50+ years investment in basic biomedical research have resulted in the

development of a wave of novel potentially highly effective therapies in

human disease

Time Magazine May 2004

Delivering translational medicine: a global challenge

National and international context

• After decades of investment in basic research it is now apparent that the major factor limiting rapid development of new therapies is a global deficit of effective translational medicine centres

• As a consequence patients are not able to access potentially life saving novel therapies

• At the same time the inability to rapidly assess new drugs and medical devices has been identified as a critical rate-limiting step by the pharmaceutical and biotechnology industries

• Government and NHS have identified life sciences as a key priority, so that the UK becomes the global hub for life sciences; the location of choice for investment; and life sciences are a key contributor to economic growth

• Birmingham is one of the few global cities with all the attributes required

The life sciences now represent a multi-billion pound industry that

the UK is uniquely placed to lead

Accelerating access for

NHS patients to new

medical innovations

“Unlocking the power of the NHS to be a "test-bed" for new medical innovations could dramatically reduce the time it

takes for patients to gain access to new treatments”

George Freeman MP, Minister for Life Sciences, November 2014

Requirements for a globally competitive translational medicine

programme - Birmingham’s strengths and challenge

High quality clinical teams

World class clinical infrastructure

One of the largest catchment regions in Europe

Strong, translationally focused basic science programme

High quality genomics and informatics programmes

X Infrastructure and personnel required to deliver rapidly deliver clinicial trials with embedded genomics

Development of the Centre for Clinical Haematology

in Birmingham

• Birmingham serves a 6 million catchment region - massive

• Potential for early phase trial delivery in haemato-oncology

• 1999: clinical resource rudimentary consisting of three outpatient rooms, two secretaries’ offices

• Strong support by Trust and University for the development of a translational haematology unit and identification of central site on campus, “if you can find the money”

• £2.2 million grant to create Centre for Clinical Haematology awarded by Advantage West Midlands

• Dependent on:

• creation of 105 jobs

• trialling of 25 new drugs

• assists to new and established businesses.

Entrance to the Centre for Clinical

Haematology

Outpatients and Consulting Rooms

Consultant and Secretaries

Offices

D Cameron, No 10 Life Sciences paper 2011

National Trials Acceleration Programme (TAP)

Core funding for:

• Central Co-ordination Hub in Birmingham

• A funded network of early phase leukaemia centres

• Serving metropolitan areas within the UK - covering catchment area of 20 million

• Dedicated research nurses and data managers

Aims:

• To open four or five new phase I/II haematology trials each year

• To open trials within 6-12 months of award letter

• To complete, analyse and publish the results of each trial no more than two years after recruitment of the first patient

Summary of time to trial set-up

Trial Award date Date trial

opened to

recruitment

Days to opening

MAJIC 01 Oct 2011 09 Aug 2012 313

RAVVA 01 Oct 2011 28 Sep 2012 363

Brevity 25 Nov 2011 10 Feb 2014 808

CyCLLe 29 Mar 2012 29 Apr 2013 396

RomAZA 22 Nov 2012 30 Sep 2013 312

VIOLA 08 Apr 2013 06 Feb 2014 303

IciCCLe 29 Mar 2012 12 Jun 2014 805

ELASTIC 22 Nov 2012 15 Oct 2014 692

MATCHPOINT 22 Nov 2012 02 Dec 2014 740

TIER 27 Nov 2013 03 Feb 2015 433

TAP Centres

• Co-ordinated network of trials

centres

• Large enough catchment areas to

allow rapid recruitment

Belfast City Hospital

Cardiff University Hospital

The Christie, Manchester

Churchill Hospital Oxford

Gartnavel General Hospital, Glasgow

Hammersmith Hospital, London

King’s College London (King’s, Guy’s, St Thomas’)

Nottingham University Hospital

Queen Elizabeth Hospital, Birmingham

Royal Liverpool University Hospital

Southampton General Hospital

St Bartholomew’s Hospital, London

St James’ University Hospital, Leeds

Summary of recruitment

Trial Recruitment target Recruitment to

date

MAJIC 306 (290 previously) 259

RAVVA 260 (180 previously) 259

Brevity 30 (+ extension) 36

CyCLLe 10 6

RomAZA 36 14

VIOLA 27 14

IciCCLe 40 40

ELASTIC 37 4

MATCHPOINT 30 4

TIER 40 1

Calibre 40 2

RomiCAR 60 1

0

5

10

15

20

25

30

35

40

Cu

mm

ula

tiv

e A

ccru

al

Time Period

IcICLLe cumulative recruitment to 16-Mar-2015

Actual Accrual

Cumulative Target

With this FPFV, UK has now achieved the

first Ruxolitinib IIT patient in the world,

beating Germany in the process

This is a tremendous achievement for

MAJIC and Novartis, and has been

highlighted to Novartis global colleagues in

the US

This is a first for the UK Oncology BU

| MAJIC and TAP | Ibrahim ElHoussieny and Barbara McLaughlan|

28 September 2012

A gold for the UK

Strategy for UK life sciences, one year on

Developing a TAP model in stem cell transplant and

immunotherapy - UPTAKE

• Despite major progress in donor identification, <50% patients alive

at three years post-transplant

• Numerous studies addressing GVHD, infection prophylaxis,

relapse are required

• Prospective transplant studies across the globe are slow to recruit

• Cell Therapy Catapult is game-changer for UK but slow trial

recruitment threatens delivery

• UPTAKE model agreed as key UK priority by partners: Anthony

Nolan, BSBMT, NHSBT, LLR, Cell Therapy Catapult

Central Hub

Transplant Centre

Specialist Transplant

Centre

Transplant Centre

Transplant Centre

Specialist Transplant

Centre

Transplant Centre

Specialist Transplant

Centre

Transplant Centre

Transplant Centre

Specialist Transplant

Centre

Specialist

Transplant Centres

Able to recruit to

Cell Therapy

Catapult trials

Proposed structure of UPTAKE

• Key deliverables of four year programme by December 2020

• Key deliverables of four year programme by December 2024

No. of trials set-up/opened

No. of trials fully recruited

Number of trials ready for publication

BSBMT trials 12 6 0

Cell Therapy Catapult trials

12 9 3

Total 24 15 3

Deliverables

No. of trials set-up/opened

No. of trials fully recruited

Number of trials

submitted for publication

BSBMT trials 12 12 12

Cell Therapy Catapult trials

12 12 12

Total 24 24 24

The Institute of Translational Medicine

• £24 million ITM developed in partnership between government, UHB and University

• Funded as part of £1 billion City Deal in recognition of Birmingham’s strategic strengths in the life sciences

• Commissioned to fund a five storey building which co-locates all the key components of an effective translational infrastructure

• Three floors with dedicated office space for key clinical academic specialities

• One floor housing a dedicated Cancer Unit, containing office space for cancer specialties and a link to the Birmingham Cancer Trials Unit

• One floor dedicated to stratified medicine in oncology and non-malignant disease and health economic appraisal unit

• An integrated clinical research office and Commercial Hub

• Will generate more than £100 million free drugs and in excess of 600 jobs

Strategy for UK life sciences, one year on

Conclusions

• The UK’s combination of a world-class science base and an

integrated NHS represent huge strategic advantages in

delivering a world-class stratified medicine programme

• TAP models are critical to drive rapid development and

recruitment of trials of novel agents with embedded genomics

• Key to its success are the combination of a regulatory hub,

networked large catchment region and collegiate clinical

academic leadership

• Establishment of a nested programme of regional and national

TAPS delivering trials to regulatory level, in collaboration with

pharma and NICE, has the potential to become a key delivery

arm of the government’s life sciences vision

The AHSN role

Dr Chris Parker CBE

Managing Director, West Midlands AHSN

christopher.parker@wmahsn.org

Core objectives of AHSNs

1. Focus on the needs of patients and local populations

2. Build a culture of partnership and collaboration

3. Speed up adoption of innovation into practice to

improve clinical outcomes and patient experience

4. Create wealth

National AHSN coverage

5.6m populationDiversity +++

WMAHSN priorities 2014/15

• Long term conditions, a whole system,

person-centred care approach

• Mental health

• Drug safety

WMAHSN priorities 2014/15

• Long term conditions, a whole system,

person-centred care approach

• Mental health

WMAHSN priorities 2015/16

• Wellness and prevention of illness

• Long term conditions, a whole system,

person-centred care approach

• Mental health

WMAHSN priorities 2015/16

• Wellness and prevention of illness

• Long term conditions, a whole system,

person-centred care approach

• Mental health

WMAHSN priorities 2015/16

• Wellness and prevention of illness

• Long term conditions, a whole system,

person-centred care approach

• Mental health crisis care

WMAHSN priorities 2015/16

• Wellness and prevention of illness

• Long term conditions, a whole system,

person-centred care approach

• Mental health crisis care

• Advanced diagnostics, genomics and

precision medicine

Core objectives of AHSNs

1. Focus on the needs of patients and local populations

2. Build a culture of partnership and collaboration

3. Speed up adoption of innovation into practice to

improve clinical outcomes and patient experience

4. Create wealth

Core objectives of AHSNs

1. Focus on the needs of patients and local populations

2. Build a culture of partnership and collaboration

3. Speed up adoption of innovation into practice to

improve clinical outcomes and patient experience

4. Create wealth

Core objectives of AHSNs

1. Focus on the needs of patients and local populations

2. Build a culture of partnership and collaboration

3. Speed up adoption of innovation into practice to

improve clinical outcomes and patient experience

4. Create wealth

Core objectives of AHSNs

1. Focus on the needs of patients and local populations

2. Build a culture of partnership and collaboration

3. Speed up adoption of innovation into practice to

improve clinical outcomes and patient experience

4. Create wealth

WMAHSN Innovation and Adoption

Service

investmentROI = Return on involvement

Key messages

• 100k Genome Project/transformation

diagnostics, genomics and precision medicine

• Focused on regional population

• Main effort is adoption of innovation

• Catalyst for collaboration

• Commercialisation

West Midlands AHSN

Office 12, Ground Floor

Institute of Translational Medicine

Heritage Building (Queen Elizabeth Hospital)

Mindelsohn Way

Edgbaston

Birmingham

B15 2TH

Tel: 0121 371 8061

info@wmahsn.org and www.wmahsn.org

Personalised medicine –

a patient’s perspective

James Cunningham

Panel discussion

Professor Sue Hill, Professor Michael Griffiths, Hilary

Fanning, Professor Charles Craddock

Lunch and

workshop session

Meeting Point

Thank you and have

a safe journey