EuroBioForum 2013 2nd Annual Conference 27-28 May 2013 - Hilton Munich City, Munich, Germany http://www.eurobioforum.eu/2013 ======================================= # NATIONAL PERSPECTIVES # Scotland: 2b or not 2b – delivering Smart Clinical Trails Graeme Boyle Interim Director Stratified Medicine Scotland & Senior Programme Manager at Health Science Scotland ======================================= http://www.eurobioforum.eu

1. 2B or not 2B
The case for Smart Clinical Trials
Graeme Boyle, Stratified Medicine Scotland
EuroBioForum, Munich, May 27, 2013

2. Health Science Scotland

3. Lost in Translation?
Basic
research
Prototype
discovery
Preclin
Dev.
Early
trials
Late trials
HTA,
HRS
Use in
NHS
TMRC
Health Science Scotland
1st gap in translation 2nd gap in translation
HTA: health technology assessment
HSR: health services research
Adapted from Cooksey report Chart 7.1, page 105
SMC/SHTG/MHRASULSA/SINAPSE
NRS/PCC

4. Scotland - World Class
Therapeutic Areas – e.g.
• CV – Metabolic
• Oncology
• Inflammatory/Immune
• Stem Cell/Regenerative Medicine
Infrastructure – e.g.
• NRS/CRF
• BioBanks
• Health Informatics
• Stratified Medicine

5. Issue 1 – Collaborative Research
Adapted from Tufts Center Report

6. Stratified Medicine Scotland - Innovation
Centre
“The right treatment, to the right patient,
at the right cost………”

7. Stratified Medicine = better outcomes
It will allow us to offer
• The right drug
• To the right patient
• For the right disease
• At the right time
• With the right dose
• Targeted treatment based on
genetic profile
• Develop diagnostic markers
for disease
• Reduce the costs of
clinical trials through better
cohort selection
Reduction in drug
toxicity
Faster and cheaper to
bring drugs to market
Targeted drug design
Earlier illness
detection & prevention
Determine disease
variants

8. Scotland’s USP
Genetics
Provider Informatics

9. Exemplar Programme 2: Heart disease & diabetes
Exemplar Programme 3: RhA & respiratory
Exemplar Programme 1: Oncology
C
o
m
p
a
n
y
A
Tissue/Bio-repositories/Datasets
Biomedical Informatics/e Health
Imaging
Genomic/”omic’ medicine
Innovation Centre Business Platform
The Model
C
o
m
p
a
n
y
B
C
o
m
p
a
n
y
C

10. Stratify patients
Patient sample sent
to CRO for
genotyping
Drug Trials Get Smarter
Mutation A Enrolled
Rejected (Treatment
Delayed)
Clinical Trial
agent targets
mutation A
NGS Panel
Trial B
Trial A
Trial D
Trial C
Mutation B
Mutation A
Mutation D
Mutation C
No Mutation
Enrollment through centralized
testing (old)
Enrollment through care-
embedded testing (future)
Patients with
disease
OLD
NEW
Modified from

11. Long Term Vision
To transform management of chronic disease globally by
accelerating biomedical research, high quality health care
provision and economic growth.Key Segment:Molecular Diagnostics
Precisetherapy
requiresprecise
diagnostics
Thewaywelookat diseaseischanging Assemblingthenecessarytechnologies
tolead
Responders
Patient populations
Mammography MolecularBiopsyTools
Tumor
+
IHCStain
GenesequencingMolecular pathology
Imaging Molecular Diagnostics
Alzheimer’s Disease Age matched control
[18F]AH110690 PETImaging
90 –120 min summation
Alzheimer’s Disease Age matched control
[18F]AH110690 PETImaging
90 –120 min summation
Operations&
commercialization
Molecular imaging
agents&equipment
Technology&
content
Enabling
workflow
invivo
diagnostics
GRC
Molecular
Pathology
Digital
Pathology
Clarient
...tobea leader inthenext diagnostic
frontier
Toxic
impact
No
impact
A
B
Genomic profile of tumourActionable mutations
KRAS
(G12D)
p16/CDKN2A
(loss)
TP53
(V274F)
Modified from

12. Issue 2 – Creating Economic Value
SME
New and
existing
Research
Partner
End User of IP
arising
New Entrants
Supply Chain
Partner
Technology
Solution
Provider

13. Characteristics of Scotland
• Population 5M
• Rural and inner city
• Stable population
• Collaboration – Aberdeen, Edinburgh, Dundee,
Glasgow, St Andrews
• Single coherent health care provider
• Unique patient identifier

14. Collecting data from cradle to grave …
Maternity
BIRTH
DEATH
Neonatal Record SMR11
Child health surveillance
Immunisation
GP consultations
Dental SMR13
Out patients SMR00 Hospital Admissions SMR01
Mental Health SMR04
Prescribing Screening
Community care SMR50
Cancer registrations SMR06
Cancer registry

15. NRS W
NRS NE
NRS SE
NRS E
Biorepository Network
Strategic national collections
• Rheumatoid arthritis
• Renal cancer
• Type 1 diabetes
• Generation Scotland/ SHHS
National/ local planned collections
• Generic consent
• Strategy driven
• Future focus
Bespoke collections
• Specific consent
• Project based
• Investigator ‘owned’
Infrastructure development
• Inventory management system
• Patient record linkage
• Enhanced storage capacity
• Facilitated rapid access
Pathology archive

16. Issue 2: European Biorepository Network

17. Imaging
phenotype
(PACS)
laboratory
phenotype
(SCI store)
Storage Area Network
Mortality
(GROS)
Hospital episodes
(SMR: ISD)
Identification
(CHI)
Prescribing
NHS Data stores
Primary Care?
Dumb terminals
SAFE HAVEN
Secure storage
power protection
camera surveillance
NHS staff
Integrated datasets
Accredited access
Health Informatics Centres

18. Issue 3:
Connecting Health Informatics

19. Issue 4 – Information Commons

20. The Opportunity – Window
Actively Working but 1-2 years offMatured capability
Potential but no plans to develop Unlikely to provide access due to policy or technical Unknown
Readiness to implement electronic
Health Records – Stratified Medicine
GSK recognises Scotland’s current leadership in electronic Public Health
Records … this IC will provide the opportunity to secure that position
through its application to Clinical Trials to stratify patient groups..
Patrick Vallance, President Pharmaceutical R&D, GSK

21. European Markets for Stratified
Medicine
Source:Advancing Access to Personalized Medicine: A Comparative Assessment of European
Reimbursement Systems – Personalised Medicine Coalition

22. Issue 5: Developing skills
• Modern apprenticeship
• Further education level
The Stratified Medicine Scotland consortium has identified
the key training needs required to support the exploitation
of stratified medicine by Scottish industry.
• Graduate level (Health science/informatics)
• Post Graduate (Health science/informatics)
• Entrepreneurship
• Senior level/industry exchange (GSK, P Vallance)
• Industry/Academia mentoring

23. Issue 6: Ethics and Public Engagement

24. Challenges & Opportunities
• Collaborative Research
• Economic Impact
• Biorepository linkages
• Informatics
• Skills Development
• Public Engagement &
Ethics
Modified from ESF: Personalised Medicine for the European Citizen

25. Danke schön
Thank You
Merci
takk
Dank u
mulțumesc
Спасибо
Grazie
& Tapadh Leibh