2B or not 2B

The case for Smart Clinical Trials

Graeme Boyle, Stratified Medicine Scotland

EuroBioForum, Munich, May 27, 2013

Health Science Scotland

Lost in Translation?

Basic research

Prototype discovery

PreclinDev.

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/MHRA SULSA/SINAPSE

NRS/PCC

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

Issue 1 – Collaborative Research

Adapted from Tufts Center Report

Stratified Medicine Scotland - Innovation Centre

“The right treatment, to the right patient,

at the right cost………”

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

Scotland’s USP

Genetics

Provider Informatics

Exemplar Programme 2: Heart disease & diabetes

Exemplar Programme 3: RhA & respiratory

Exemplar Programme 1: Oncology

Company

A

Tissue/Bio-repositories/Datasets

Biomedical Informatics/e Health

Imaging

Genomic/”omic’ medicine

Innovation Centre Business Platform

The Model

Company

B

Co mpany C

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

Long Term Vision

To transform management of chronic disease globally by accelerating biomedical research, high quality health care

provision and economic growth.

12 GE Title or job number

7/13/2011

Key Segment: Molecular Diagnostics

Precise therapy

requires precise

diagnostics

The way we look at disease is changing Assembling the necessary technologies

to lead

Responders

Patient populations

Mammography Molecular Biopsy Tools

Tumor

+

IHC Stain

Gene sequencing Molecular pathology

Imaging Molecular Diagnostics

Alzheimer’s Disease Age matched control

[18F]AH110690 PET Imaging

90 –120 min summation

Alzheimer’s Disease Age matched control

[18F]AH110690 PET Imaging

90 –120 min summation

Operations &

commercialization

Molecular imaging

agents & equipment

Technology &

content

Enabling

workflow

in vivo

diagnostics

GRC

Molecular

Pathology

Digital

Pathology

Clarient

Molecular profiling segment for cancer expected to

grow from $15B in 2010 to $47B in 2015

... to be a leader in the next diagnostic

frontier

Toxic

impact

No

impact

A

B A

B

Genomic profile of tumour Actionable mutations

KRAS

(G12D)

p16/CDKN2A

(loss)

TP53

(V274F )

Modified from

Issue 2 – Creating Economic Value

SME

New and existing

Research Partner

End User of IP arising

New Entrants Supply Chain

Partner

Technology Solution Provider

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

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

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

Issue 2: European Biorepository Network

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

Issue 3: Connecting Health Informatics

Issue 4 – Information Commons

The Opportunity – Window

Actively Working but 1-2 years off Matured 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

European Markets for Stratified Medicine

Source:Advancing Access to Personalized Medicine: A Comparative Assessment of European

Reimbursement Systems – Personalised Medicine Coalition

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

Issue 6: Ethics and Public Engagement

Challenges & Opportunities

• Collaborative Research

• Economic Impact

• Biorepository linkages

• Informatics

• Skills Development

• Public Engagement & Ethics

Modified from ESF: Personalised Medicine for the European Citizen

Danke schön

Thank You Merci takk

Dank u mulțumesc Спасибо

Grazie & Tapadh Leibh