The Use of Non Profit, Industrial Consortia to Improve Pharmaceutical Industry R & D

InInter SAE Consortium Ltd.

The Use of Non Profit, Industrial The Use of Non Profit, Industrial Consortia to Improve Pharmaceutical Consortia to Improve Pharmaceutical

Industry R & DIndustry R & D

““Lessons Learned”Lessons Learned”

Wellcome Trust’s “Pre-Competitive Boundaries and Open Innovation in Drug Discovery and Development”

Meeting

Arthur HoldenArthur Holden

Founder, Chairman, and CEOFounder, Chairman, and CEO


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Introduction • Executive not scientist• 30 years of leadership experience – commercial &

non-profit consortia; start-ups to mid size to large corporations– Baxter International (1984-1994)– Celsis International (1994-1998)– First Genetic Trust (2000-2006)– Illumina (2006-2007)

• Formed and managed numerous large-scale consortia – The SNP Consortium, Ltd. (1999-2002)– The International SAE Consortium, Ltd. (2007- 2012)– Supported many others (MSC, HapMap, etc.)


My Comments

• Consortia Context– Model– TSC– SAEC

• Some Lessons Learned

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Context


“Pharmaceuticals” Context Declining Productivity & novel therapeutics

Increasing Costs & Competition at “Market Entry”

“Block Buster” mentality softening

Scale “Innovation” ???

> Healthcare pricing pressures in all core markets “pay for performance” paradigm of the future

End of “biotech risk subsidy” by the public markets

etc, etc, etc.

Increasing R & D productivity is critical.

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“R & D Sourcing Options”

In-house Operations

ExternalCommercial Partnerships

External Academic/Gov.Relationships

R & D Consortia

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Industrial Biomedical Consortium“Generic Model”

IndustrialMembers

Clear Project/Unifying Goal(s)/ Finite

Timeframe

Organized by Professional Management & Supported by Quality Processes

SkilledExternalPartners

PrivateFoundations/Governmental

Bodies

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Why Industrial Biomedical Consortia [IBCs]?• Fill a research gap, not being effectively filled by

existing players [government or private]

• Establish common/often risky “research platforms”, which help all researchers, while meeting specific industrial scientific requirements [pre-competitive/pro-competitive]

• Establish industry standards

• Reduce legal/IP barriers

• Gain greater efficiency/effectiveness (scale) via pooling of talents and resources across industry


Two Consortia

The SNP Consortium1999-2002

The SAE Consortium2007-2012

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Common Characteristics (TSC/SAEC)

• Defined project finite timeframe

• Non-Profit [501c3] > “public benefit orientation”

– Open data access

– No IP constraints

• “Fixed, uniform” membership contributions

• Designed for “leverage” and “time to result”

• Collaboration with government (if advantageous to results)

• Active (Committee) Participation and Investment by Members

• “Governance by majority” 1 member/1 vote

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SNP Consortium -- Mission & Results“The SNP Consortium worked to advance the field of medicine and

the development of genetic based diagnostics and therapeutics, through the creation of a high quality, dense genome wide single nucleotide polymorphism (SNP) map, which is available to all parties at no cost.”

The Results: Created a high quality HG SNP map for GWAS

Identify 300,000 SNPs [ 2.7M Common SNPs]

Map 150,000 SNPs [ 2.5M Common SNPs]

Broad, evenly spaced well annotated map within two years

Maximize public accessibility [~ 2.4M “unencumbered” SNPs]

Provided order and orientation data to assembled base WG project sequence and a significant “backbone” to the “HapMap”

$42M project, which exceeded expectations with $9M in budget favorability

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Drivers of the SNP Consortium

Industry standard SNP map strong industry

support

Universal access to base genomic/SNP data public

domain (IP considerations)

Facilitate pharmaco-genomics / genetics

association, linkage & haplotyping studies

Economics:

Favorable economy of scale>>cost & risk sharing

Favorable cost to access high quality SNP datasets

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DCCDCC

WhiteheadWhitehead SangerSanger Wash UWash U

StanfordStanford

SNPsSNPs

Mapping DataMapping Data

[email protected]@CSHL.org

ConsortiumConsortium

PublicPublicdbSNP/EBIdbSNP/EBI

Reformatted SNPs & MapsReformatted SNPs & Maps

SNPsSNPsSNPs & Mapping DataSNPs & Mapping Data

SNPsSNPs

The SNP Consortium -DCC Summary


iSAEC -- Mission & Results“The iSAEC will identify and validate DNA-variants useful in predicting the risk of drug induced serious adverse events.”

The Results (to date): Initial GWAS for Drug Induced Serious Skin Rash identifying marker(s) of potential

significance

Assembled 2nd largest DILI cohort, merged with DILIN, completed initial GWAS identifying numerous significant marker(s) within/across drugs

Initial GWAS for Drug Induced PQT/TdP, merged with LeDueq Network, and identified marker(s) of potential significance

Completed four public data releases (clinical and GWAS data) with FDA

> 200 genetic markers associated with DILI and SSR in the public domain.

Favorable to budget in phase by $2M

Organized Phase 2 focused on “drug induced immunologic SAEs” associated with specific drugs and ethnicities, and forming novel SAE research channels vital to drug-specific SAE research.

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Drivers of the iSAECNeed to improve new product (safety) productivityScale required to execute safety pharmacogenetics (top R

& D management priority) common markers in public domain

Need to standardize “SAE phenotypes” Need to develop new and innovative methods to source

cases and controls Pooled technology assessment & risk (e.g. GWAS, WG

sequencing) FDA’s Industrial Advisory Board recommendation$ resources required (poor public funding, given the

health impacts)

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Scientific/ClinicalSMC

iSAEC Organization and Committees

Research Collaborators [Academic & Commercial]

Legal/IP

BOD

Outsourced Service Suppliers [Legal & PR]

PR/ Comm.

SAE [s] Data Anal.

Genotyping Core [Expression Analysis,

Inc]CEO

Bioinformatics Core [Columbia University]

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iSAEC Web Sitehttp://www.saeconsortium.org

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iSAEC Data Access Process

Investigator Request Form (online) System admin

Automated Data AccessAssessment

DatabaseData Use Agreement(Online/Hardcopy)


Lessons Learned


10 Lessons Learned (Success Factors)

1. A clear unifying objective is a must.

2. Ensure the effort is both “pre-competitive” and “pro-competitive.”

3. Have a quality “draft” operating concept/plan before recruiting members [“one shot rule”].

4. Establish a uniform membership requirements & development strategy with potential members.

5. Establish dedicated management early in the effort /don’t reinvent the wheel.

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10 Success Factors cont.

6. Organize well defined committees with high quality, dedicated leaders

7. Outsource…outsource… outsource to the best external advisors/ investigators via “performance based” contracts

8. Generate results that exceed expectations

9. Make it fun and say “thank you” in meaningful ways, and….

10. Know when to fold … begin with an end in mind!

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•Needed Science!•Easily Understood•Measurable•Focused vs General•All essential for member recruitment

Have a Clear, Quality Objective which Unifies the Membership[…Full Genome Representative SNP Map… Safety related markers in the public domain]

Learning #1

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•Pre-Competitive (moving target)

•Pro-Competitive (legal anti-trust perspective)

•“Public Good” orientation (Tax/Charity status)

•“Neutral data access policy” “public” access to data

The focus should be “pre-competitive” and “pro-competitive.”

Learning #2

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•Well organized “formation phase” with potential members

•Scientific Plan [including necessary feasibility pilots]

• Informatics Plan•Legal/IP Policies•Operating

Plan/Performance Metrics/Contingencies

•Budget [Proactive Risk Factor Mgnt]

Develop a high-quality plan [before recruiting membership]

Learning #3

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• High Priority a Must!• Fixed Dues Structure

(Annual Sensitivity [$1M down to <$.4M/year)

• Do not vary membership tiers for industrial members

• Non industrial members >> same $ terms for voting rights

• Member Diversification has pros & cons

Establish a Clear Membership Requirement & Development Strategy with potential members

Learning #4

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•Formation phase leadership

•Members are investing in both the leader/project

•Defining the Organization & Key Processes

•Sourcing strategies•Pre-formation

negotiations•“Closing” Membership•Communication is Key•Everything takes more

Effort than Planned

Establish dedicated management ASAP

Learning #5

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• SMC, Legal/IP, & Sub-committees key

• Functional/Scientific Leadership is vital

• Time Commitment is Greater than Expected

• Senior Management Support of Manager Commitment

Organize clear committees with high quality dedicated leaders

Learning #6

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• Increases expertise & skills

• Leverage existing infra-structure/investments/skill

• Performance based contracts no grants!

• Reduces project risk• Increases management

flexibility• Careful assessment of

the risks of doing the Consortium if Outsourcing is not an Option

Outsource…outsource… outsource

Learning #7


• Confirms membership investment creates additional consortia opportunities

• “Consortia fatigue” is real major cause is poorly managed/under performing consortia

• Winning together is fun …

Generate results that exceed expectations!

Learning #8

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• Member help is essential

• “Recognition starvation” is an epidemic

• Everybody likes “special events” … but cost-effectiveness is vital in today’s environment

Say “thank you” in meaningful ways and make the consortium a fun experience

Learning #9

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• Finite objectives within a fixed time frame attractive to members

• Consortia are strategic initiatives vs on-going concerns

• New Consortia require different leadership

• Effective BOD with the appropriate experience & seniority—Key!

Don’t try to make a consortium into something it shouldn't be … know when to fold

Learning #10


Final Caveats• Wellcome Trust involvement and funding

important validation to pharmcos• Involve stakeholder governmental bodies (TSC—NIH,

iSAEC—FDA, EMEA, PMDA), without slowing the effort.

• Limits to company leverage in these “leaner times” … funding a quality consortium is very hard these days (more freeloaders)

• Vital that we learn from best efforts take these into new, important consortia efforts to improve pharma productivity

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AcknowledgmentsMembers Brian Spear [Abbott] Rick Scheyer [Daiichi Sankyo] Lon Cardon [GSK] Nadine Cohen [J&J] Joanne Meyer [Novartis] Aidan Power [Pfizer] Klaus Lindpaintner [Roche] Robert Dix [Sanofi-Aventis] Leonardo Sahelijo [Takeda] Michael Burczynski [Wyeth] Janet Woodcock [FDA] ShaAvhree Buckman [FDA] Michael Dunn [Wellcome Trust]

SMC Ann Daly & DILIGEN [Newcastle] Mariam Molokhia & EUDRAGENE [London] Matt Nelson [GSK] Sally John [Pfizer] Yufeng Shen [Columbia] Itsik Pe’er [Columbia] Aris Floratos [Columbia] Mark Daly [Harvard/Broad] David Goldstein [Duke] Eric Lai [ex-GSK] Donald Halbert [Abbott] Joe Walker [D-S] Nadine Cohen, Quingqin Serena Li, & Adrian Thomas [J&J] Joanne Meyer & Steve Lewitzky [Novartis] Klaus Lindpaintner & Karen Wilcock [Roche] Steven Kovacs [Sanofi-Aventis] Leonardo Sahelijo [Takeda] Ted Burczynski & Maha Karnoub [Wyeth] Robert O'Neill & Steve Wilson [FDA] Andrea Califano [Columbia] Allen Roses [Duke] John Senior [FDA]


Appendix Slides


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Industrial Consortia Some Operational Tenants

• Unifying objective industry and public good [501 c 3]

• Focused projects, with clear objectives & strong operational management

• Strong “quality” and “time to result” orientation • Clear and uniform “membership requirements” • Extensive leverage of members’ skills via well

organized sub-committees • Strive to collaborate with the best quality external

advisors & investigators on an international basis• Public release of data after appropriate IP

management actions to ensure “openness”

The Use of Non Profit, Industrial Consortia to Improve Pharmaceutical Industry R & D

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