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International Biobanking:

Oportunities and Challenges for

Private-Public Collaboration

David R. Cox M.D.,Ph.D.

Senior Vice President and Chief Scientific Officer

Biotherapeutics and Bioinnovation Center

Pfizer Inc

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FUNDEMENTAL PRINCIPLES OF INTERNATIONAL

BIOBANKING

Open access with wide-scale data sharing and collaboration across countries

Altruism, with promotion of the common good

Consent, with respect for cultural diversity

Protect of the interests of a diverse array of stakeholders

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Biobanks and the Pharmaceutical

Industry

Disease as opposed to population focus

Clinical trial-related translational research

Valuable information regarding treatment outcomes

Private

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Key Insights

The pharmaceutical industry needs to engage the broader scientific and healthcare community in a more collaborative fashion in order to achieve its goals

Money alone does not provide access to the critical collaborative relationships

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Phase 2 Survival Remains a Major Challenge to Increasing R&D

Productivity

Unsustainable economics: Industry average cost per NME

>$2.3 B (2007) ~35 NCE into the clinic to yield

1 NME

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Reducing Drug AttritionReducing Drug Attrition

Molecular Targets

Target to clinic: Poor alignment of molecular

understanding to clinical need Animal models inconsistently

predictive of human disease and outcomes

Clinical Outcome

Clinic to target: Defined unmet clinical need Quantitative translation from

human genetic insights, to optimized therapy based on

an understanding of biology

Human Genetics

Traditional Discovery

VS.

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The SNP ConsortiumThe SNP Consortium International HapMap ProjectInternational HapMap Project

Genotyping ChipsGenotyping ChipsAdapted from David Altshuler, Harvard Medical School, 2007

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Human Genetics-Based Approaches Differ on Scale

and Focus

Large population strategy: International Hap Map Project – genomic atlas from “normal

individuals” Catalog human variability to identify patterns of genes linked to health

and disease Common genetic variants with modest genetic effects Sampling strategy requiring 10,000 to 300,000 samples Utilize large existing disease cohorts with limited clinical outcome data Significant data already exists for common disorders that now requires

additional biology to fully characterize optimal target space

Rare DNA variant strategy: Focus on humans with traits of interest – patients with extreme

phenotypes of a clinical outcome Variant will better identify pathophysiological pathways that are the

basis of the disorder Rare variants yield larger genetic effects than common variants

enabling study of a wide range of clinical outcomes Loss-of-function variant defines direction of therapeutic perturbation Sampling strategy requiring 200 to 300 individuals for each trait Utilize existing, well characterized disease cohorts with the most

important clinical outcomes

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Major Challenges

Linking biobank materials to multiple sources of healthcare information, while protecting all stakeholders

Reconciling altruism and open collaboration with intellectual property and profit

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The Public Has Mixed Feelings Regarding Genetic Research

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NCAA's sickle cell test plan raises fears

Erin Allday, Chronicle Staff WriterMonday, September 14, 2009

A recent NCAA recommendation to screen college athletes for sickle cell trait - the gene that cancause sickle cell disease - is raising the hackles of some experts who say testing is probablyunnecessary, and may even lead to inadvertent discrimination against minority players. Sickle celldisease is a blood disorder that can cause severe pain, stroke and death, but sickle cell trait is almostalways benign, and many people never know whether they carry the gene. About 8 percent of black peopleand about 1 percent of Latinos have sickle cell trait, but it's rare among white people, affecting only about1 in 10,000. The United States has a long history of discrimination against people with sickle cell trait, saidTroy Duster, a sociologist at UC Berkeley and New York University. In the 1960s, people who tested positiveweren't allowed into the Air Force Academy, and into the '70s people were denied insurance or certain jobs,he said. It's irresponsible to screen people when there's little scientific evidence that the gene causes deathand no specific precautions athletes can take to protect themselves, Duster said. "When you screen someone,the question is, for what? What are you going to do with that information?" Duster said. "The NCAA is sayingthey want education, but education requires research, and there's no research."

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Key Priorities Going Forward

Harmonization as opposed to standardization

Linking biobanks with mutliple sources of health information

Harmonizing the legal and ethical frameworks of multiple countries

Public engagement

Sustainability through the cooperation of mutliple diverse stakeholders

Private sector contributions of knowledge and data in addition to money