Proprietary and Confidential © AstraZeneca 2008FOR INTERNAL USE ONLY

Collaboration in Biobanking: sustainingR&D activities of the Pharmaceutical Industry

Julie Corfield, AstraZeneca R&D Charnwood BBMRI: Stakeholders forum 16th September 2009Renaissance Hotel, Brussels

Patients………Patients………Patients………PatientsUnmet needs

Cancer, Brain disorders, Inflammatory, Metabolic and Infectious diseases

R&D bottlenecks/IMI

Belonging to Europe……….sustaining R&D

Sustaining R&D activities in Pharma?Providing the foundation for the treatments of today

and the cures of tomorrow

Time

Risk

Cost

Change

Discovery. Effective risk assessment, failing drugs faster and improving productivity and cost-efficiency are becoming even more important…… a walloping average €895 million on researching and developing a single new medicine.

LEADERSHIP

Revisit the Current R&D Model? Estimated > 90% of medicines in use today have been discovered or developed by the industry. Late-stage attrition, with increasing development costs.

NEW OPERATING MODEL

Industry’s best hope for survival lies in innovation, its traditional strength. The business model of a vertically integrated approach to developing, manufacturing and selling drugs changed in favour of

outsourcing. INNOVATION, OUTSOURCING

…..unprecedented challenges. Business model is economically unsustainable and unsuited to act quickly enough to produce the types of innovative treatments that will be demanded. The industry requires a bold new vision and leaders who have the willingness to embrace a fundamentally new approach to

their business NEW APPROACH

‘Biomarkers increasingly important in development of new drugs ….conditions at molecular level. …..New molecular markers, new assays….to help us define new diseases, …… more specifically, more

differentially.’ BETTER DEFINED DISEASES, BIOMARKERS, PERSONALISED MEDICINE BIOBANKS

The big picture

Discoveryresearch

Preclinicaldevelop.

Translationalmedicine

Clinicaldevelop.

Pharmaco-vigilance

Discoveryresearch

Preclinicaldevelop.

Translationalmedicine

Clinicaldevelop.

Pharmaco-vigilance

DiscoveryResearch

Preclinical Develop-ment

Translationalmedicine Clinical

development Pharmaco-vigilance

Benefit/Risk

Assessment Target ID Cell/TissueRelevant to Disease

Hit Target

Validate Target

Safety

Efficacy

Bench to bedside

Biobanks

Clinical Studies

Biomarkers

Definitions

Biobank: is a place, a room, or container where human biological samples are physically stored, indefinitely or for a specified time. (This is distinct from the term ‘biobank’ when defined as a “collection of human

biological samples” in some legislation).

Biobanking:

the capability that covers the governance and infrastructure of biobanks with associated facilities and personnel who are engaged in the lifecycle management of human biological samples from acquisition and use for a defined purpose(s) and through to disposal.

Montreal

AstraZeneca global biobank organisation and local biobanks at R&D sites

Shanghai

CharnwoodBostonAlderley

Bangalore

Mölndal

Lund

Wilmington

Södertälje

Research Areas:

Cardiovascular; Cancer; Infection; Gastrointestinal;

Neuroscience (Central Nervous System; Pain Control/Anaesthesia)

Respiratory (COPD/Asthma) and Inflammation (Rheumatoid arthritis)

Global Head

Local HeadBiobank operations manager

Department operations managerSample owner

CustodianSample User

Collaboration in biobanking………

AZ biobank……..a capability from collaborations

Sample Sources

Healthy

Commercial

suppliers

Post

mortem

Patients

Legacy

AZ

Customers

TI/TV

Disease

strategy

PGx

Collaborations/

Investigators

Bespoke

Clinical

studiesLiving

Archived

1999 2009

Reduced risk/fewer scandals Increased demand for samples in AZIncreasing scrutiny/regulationWider scope for consent

Increasingly competitive R&DStandardisation/best practice….global project

Tactical

ProtocolsInformed consentEthicsContractSubject dataMonitoring/Sample trackingQuality/acceptance criteria

Biomarkers

RA/OA disease area biobanks: 2001 to 2006Profile

•2 collaborations in 2000; 12 collaborations in 2006• 2 R&D sites

Collaborations •UK: RA/OA joint replacement surgery •UK: Post mortem (knees) •UK: Case-controlled large joint OA; interaction of genes and environment •UK and Australia: OA biomarkers •Sweden: early RA •Sweden: biomarker validation in healthy volunteers•Nederlands: early RA •US: OA TI/TV, biomarkers•Commercial suppliers

Profile of biobanks/collections

Biobanks• Coded samples• Dynamic not static/stock- piled• Controlled release/access; custodians• Quality/molecular characterisation/library

• Donor characteristics• Medical history, concurrent disease, current/past medication, MRI, X-

ray

Plus lifestyle/environment

Sample Types• Synovium, cartilage, meniscus, bone, tendons, synovial fluid, blood

(plasma;serum; DNA), urine;

Sample Format• Fresh, arthroscopic samples; paraffin embedded; fresh frozen

Identification/validation of efficacy biomarkers for Chronic Obstructive Pulmonary Disease AstraZeneca R&D Lund

The Malmö Smoking Induced Systemic Inflammation Study (MSIS)

MSIS:An exploratory study of endothelial progenitor cells, circulating endothelia cells, endothelial function, inflammatory blood markers and physical exercise capacity in subjects with chronic obstructive pulmonary disease (COPD) and lung-healthy never-smokers

Patient flow57 included (randomised)• 10 severe COPD patients• 10 moderate COPD patients• 13 mild COPD patients• 12 healthy smokers• 12 healthy non-smokers

Biomarkers in plasma:

Nine out of thirty-one plasma markers are elevated in COPD patients in comparison with lung healthy individuals

Fostering Collaboration

AZ Biobanks

Internal Collaboration

MD/PhdQualifications

Academic research

Publications

Better understanding of disease

New targets

Opportunities for collaboration ………to sustain R&D?

Sample collections• Use of retrospective sample collections?

• Retrospective samples adequate quality?• Prospective samples costly

• Visibility/transparency of existing pre-competitive collections

• Not an unlimited supply of subjects to provide samples……need to engage patient groups

• More ‘networks’ to provide access to existing biobanks

Standards• Standardised protocols/laboratory manuals

• Adopt consistent approaches/technologies for analysis of samples

• Information standards

• Storage capability

What can be done to enable more collaboration?

More broadly

Ethics/regulation• Harmonisation of regulations• Greater consistency in process for informed consent and ethics committee

process……….broad consent

Infrastructure/approach• Access to experts/different skills and knowledge/centres of excellence• Creation of translational and multidisciplinary research collaborations to overcome

cultural differences• Translational research to validate initial findings and explore practical use• Greater co-ordination of activities across Europe and elsewhere

• Infrastructure development for large scale population studies…to enable implementation of protocols; collection of data and samples

• Continuing technology development• Training ‘gap’

Priorities to be addressed

• Ethical and legal issues……obstacle to collaboration; consent does not routinely allow sharing

• Governance to enable sharing and exchange of material

• Data and samples contained in biobanks are variable: content and quality

• Few standardised quality controlled protocols for data collection, sample storage and analysis and access. • Difficult to pool data

Customer Needs

The Future

Thank you