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Orchestrating a National Translational Research Strategy

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Title: Orchestrating a National Translational Research Strategy


1
Orchestrating a National Translational Research
Strategy
  • John Bell

2
UK Health Research Analysis
O.S.C.H.R.
  • Published May 2006
  • First ever comprehensive national analysis of
    health research funding
  • 11 largest Government and charity funders of
    health related research in the UK
  • Collected peer-reviewed research funded 2004/2005
    950m/9500 awards
  • All types of research activity and all areas of
    health and disease

3
UK Health Research Analysis
O.S.C.H.R.
4
UK Health Research Analysis
O.S.C.H.R.
5
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6
Drivers for Change
O.S.C.H.R.
7
Key Findings
O.S.C.H.R.
O.S.C.H.R.
  • UK Health research system has many strengths
  • But
  • Risk of failing to meet full economic, health and
    social benefits of UK public investment
  • No overarching health research strategy
  • Key gaps in the translation of health research
  • Funding of health research from concept to
    practice could be more coherent
  • Cultural, institutional and financial barriers to
    translating research.

8
A Single Health Research Strategy
O.S.C.H.R.
O.S.C.H.R.
  • A new, sustainable, strategic framework for
    health research and cultural change
  • Continued investment in basic biomedical science
  • A Health Research Ring-fence
  • Commitment and engagement across all four UK
    Administrations
  • New investment targeted in key strategic areas

9
Government Investment in Health Research
Millions GBP
10
OSCHR Partners developing a single UK Health
Research Vision
11
OSCHR
Public Health Board
Translational Medicine Board
E-Health Records Research Board
Human Capital
Infrastructure
12
UK Health Research Priorities
O.S.C.H.R.
Survey of unmet medical need
Evaluation of Scientific Opportunity
Health economic impact
13
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14
England Forecast Increase in Diabetes
Prevalence by Local Authority District, 2001-2020
The Burden of Disease and Illness in the UK S.
Green, R Miles. April 2007 Source Yorkshire
Humber Public Health Observatory 2007
15
The Burden of Disease and Illness in the UK S.
Green, R Miles. April 2007
16
England and Wales Age-standardised rates for
three major causes of death (per million
population), 1971-2005
The Burden of Disease and Illness in the UK S.
Green, R Miles. April 2007 Source Office for
National Statistics 2007
17
England and Wales Cancer Mortality Trends
Age-standardised Mortality rates per Million
Population, 1991-2005
The Burden of Disease and Illness in the UK S.
Green, R Miles. April 2007 Source Office for
National Statistics 2007
18
Health Research Opportunities 2
  • Stratification of phenotype
  • Regeneration and replacement
  • Tracking response to intervention
  • Measure, understand and modify environmental and
    inherited influences on health
  • Exploitation of world leading position in
    hypothesis-generating science to deliver improved
    health
  • Early detection of the opportunity for effective
    intervention
  • Primary prevention
  • Behaviour modification
  • Understanding the burden of illness
  • Development of new interventions

19
Rebuilding Basic Science Infrastructure
O.S.C.H.R.
MRC Laboratory for Molecular Biology, Cambridge
20
Translational Pipeline
21
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22
O.S.C.H.R.
  • Translational Medicine
  • Experimental Medicine and Exploratory
    Development, including imaging, biomarkers MRC
  • Methodology for large and small clinical trials
    MRC
  • Large trials and evaluations of therapeutics,
    devices, diagnostics, and other interventions
    (overlapping with public health) NIHR
  • Clinical training NIHR

23
The complex environment of translational medicine
24
Translational Medicine Enabling Technology
  • Imaging
  • Biomarkers
  • Drug Safety
  • Experimental Medicine
  • GenotypePhenotype

25
Developmental Pathway funding Scheme (MRC)
O.S.C.H.R.
Phase I
HTS
L.I. ? L.O.
Pre- clinical models
P.o.C.
Biologics
Regulatory Support
Safety and Toxicology
Manufacturing and Formulation
26
Biomedical Research Centres (NIHR)
O.S.C.H.R.
  • 5 Centres selected in competition
  • 100 million p.a. support for infrastructure and
    personnel
  • Increase capacity in experimental medicine and
    exploratory development

27
Well-characterised Small Cohorts (MRC NIHR)
O.S.C.H.R.
  • Common disease cohorts (e.g. COPD,
    osteoarthritis, heart failure, stroke, hepatitis
    C, HIV, Alzheimers disease)
  • Phenotyping using imaging, physiology, genetics
    and genomics
  • Disease progression monitoring
  • Maintained for experimental medicine and
    exploratory development

28
Molecular Diagnostics The new genetics in
clinical practice
29
Translational GeneticsBridging the Gap
30
Next Generation Sequencing
Oxford Nanopore
454 Life Sciences
SOLEXA / ILLUMINA
31
Sequencing Projects
  • POTENTIAL TLN OUTCOMES
  • Improved test for 5-10 genes
  • New UK /European test
  • New UK /European test
  • Infection surveillance in hospitals
  • Improved test novel application
  • New UK /European test
  • Identification common variants
  • Pharmacogenetics tests
  • Signal transduction pathways, Stratified medicine
  • PROJECTS
  • Sudden Cardiac Death
  • Retinal Degeneration
  • Mental Retardation
  • Pathogens
  • HNPCC cancer
  • CHD
  • Type 2 diabetes
  • Renal cancer
  • Melanoma

32
Sudden Cardiac Death Syndromes
  • Hypertrophic and dilated cardiomyopathies, long
    QT syndrome
  • Heterogeneous single gene conditions - autosomal
    dominant
  • Incidence 1500-11000
  • Condition treatable once diagnosed lifestyle,
    beta blockers, defribillators
  • Oxford GKP programme
  • Up to 5 genes currently tested for HCM, DCM or
    LQT
  • Potential to increase referrals (cardiologists,
    coroners) expand genes tested (10)
  • Technology upgrades required to support this
  • Once validated can be applied to other
    established NHS genetic tests (eg BRCA1/2)

33
Retinal Degeneration
  • Inherited eye conditions
  • Defects in photoreceptors and retina leading to
    progressive visual loss
  • Genes known, but currently lack of comprehensive
    testing
  • 25-30 genes known for autosomal recessive
    retinitis pigmentosa
  • 200-300 genes for ARRP, ADRP, XLRP and other
    relevant eye disorders

34
Pathogen Surveillance
Clinical applications Novo virus, MRSA, C
difficile, TB
At national level identify new epidemic
strains At local level identify endemic
outbreaks Individually identify pathogen to
inform clinical intervention
35
Array CGH-NHS Potential
  • Develop as first line test for chromosomal
    anomalies
  • Multi-sample formats and high density
  • Cost implications and commissioning (gt50 cost
    efficiency)
  • Extend Applications
  • Speech and language / autism
  • Congenital heart disease
  • Leukaemia
  • Pre-implantation genetic diagnosis
  • Cancer
  • Diagnosis, prognosis, treatment selection

36
SLI037 456kb 3p26.3 loss, 607kb Xp22.11 gain
3p26.3
Male Proband
Father
Male sib (affected)
37
Large Scale Evaluation
  • Therapeutics
  • Diagnostics
  • Devices
  • Other interventions

38
E-Health
O.S.C.H.R.
  • Contribute to developing Connecting for Health
    for research purposes (Research Capability
    Program)
  • Pilot studies with databases in UK (GPRD),
    Scotland and Wales
  • Federate databases across UK

39
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40
Benefits - Research
  • Epidemiology scale follow up of patient cohorts.
  • Content rich databases allowing integration of
    data
  • Evaluation of efficacy and toxicity of
    therapeutics in real populations
  • Rapid ascertainment for clinical trials
  • Novel cohort methodology for evaluations of all
    forms of interventions

41
Integration of patient data
Laboratory data
Genomic data
E-Health Record
Imaging
GP record
Hospital admission
42
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43
E-Health
  • Effective systems of record linkage in Scotland,
    Wales and parts of England (North west and
    Birmingham) but international competition is
    growing
  • Research capability program is delivering tools
    for research purposes that will work in most
    systems
  • CfH is unlikely to be the platform in its current
    form but multiple exisiting record systems will
    work together
  • Governance of data could be limiting factor
  • We have lost the international lead

44
Why have we lacked in Public Health Research?
  • Multiple disciplines (epidemiology, infectious
    disease, modelling, behavioural psychology)
  • Much is outside the health system and Department
    (education, transport, workplace environment)
  • Multiple disease areas (cardiovascular, cancer,
    infectious disease, mental health, diabetes)
  • Inconsistent and sparse funding
  • No career track for professionals

45
Public Health
Infectious DISEASE
Infectious Disease
Infectious Disease
Chronic Disease
Discovery
Discovery
Surveillance
Evaluation
Evaluation
46
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47
Going Global with Public Health
48
Can we do better in Public Health?
  • Multi-agency/multi-departmental funding
  • Leadership in a few major areas (addictions and
    mental health, infections, obesity, ageing)
  • Obtain national experiments from others
    (transport, education)
  • Work with industry

49
The Economy.
50
Maintaining a Competitive Commercial Health
Sciences Sector
  • Pharma pipelines are poor and late stage failures
    are frequent
  • Efficacy is low in unstratified populations
  • Marketing exceeds innovation
  • Clinical Trials are too slow and expensive
  • Biotech business model is broken. Gestation is
    too long and Venture Capital is scarce
  • Not enough partnerships

51
Patient Recruitment HPS-Thrive
  • Too slow
  • One size fits all
  • Too many sites

Costs US 1.0 UK 0.6 China 0.3
52
Stratification Imperatives
  • Rheumatoid Arthritis (Mkt 16 billion)

Biologics Target
Infliximab TNF
30-40 non response
Remicade TNF
30-40 non response
Humira TNF
30-40 non response
Kineret IL-1
Limited efficacy
Rituxan CD20
Orencia CTLA-4
Limited efficacy
Actemra IL6-R
10 super responders
53
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54
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55
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56
Benefits for Industry Translational Medicine
O.S.C.H.R.
  • Creating new insights into pathways relevant to
    disease, providing new targets for industry for
    therapeutic intervention.
  • Validating targets and pathways using both small
    molecules and antibodies
  • Development of new and preclinical models that
    more appropriately mimic disease pathogenesis
  • Managing programmes that are surplus to
    requirements but informative to industry through
    exploratory development
  • Development of new diagnostics, therapeutics or
    devices up to and including exploratory
    development that could be further exploited by
    industry
  • Development of better tools for the evaluation of
    preclinical and clinical safety
  • Exploratory development programmes
  • Content-rich large-scale trials
  • Methodology innovation for trials

57
OSCHR Future Challenges and Opportunites
  • Effective communication of the role of OSCHR and
    the entire UK funding landscape
  • Commercial Interactions
  • Public Health Research
  • E-Health Records Research
  • Capacity Building
  • The Economy!
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