Pharmaceutical Innovation: The Role of Academia

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Pharmaceutical Innovation The Role of Academia

• Annetine C. Gelijns, Ph.D.
• Eric A. Rose, M.D.
• Deborah D. Ascheim, M.D.
• Corey Magnell, M.D.
• Richard Nelson, Ph.D.
• Alan J. Moskowitz, M.D.
• Columbia University

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Advances in Molecular Genetic Bases of Disease

• New opportunities for drug development
• Drug targets increased from 500 to gt 5000
• H. genome 30K genes 60K targets (agonism,
  antagonism)

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Changing University-Industry Interface

• Universities have strengthened ties with industry
• Academic faculty actively involved in
  commercialization of knowledge
• Critical questions costs and benefits of the
  changing interface?

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Translational Research Productivity
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Research Priorities

• Institutional interplay led to important drugs
• But progress in some diseases disappointing
• Questions about research priorities
• Are important medical needs being addressed?
• What is value of me-too drugs?
• Are clinically important, but unvalidated,
  targets being avoided?

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Threats to Mission of Universities

• University patenting of biological targets
  research tools
• Openness of communication
• Shift in the research agenda

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Focus of Presentation

• Need for policy reform to accelerate development
  of new drugs for important diseases?
• Roles and contributions of private and public
  sector?
• Traditional image does not capture dynamics of
  pharmaceutical innovation

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Research Activities
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Clinical Observation

• Descriptive
• AIDS
• Epidemiologic
• Framingham
• Pathologic
• Battered child syndrome
• Combinations of above
• Tobacco related illness
• H. pylori peptic ulcer disease

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Fundamental Sciences of Health

• Genetics molecular bio revolution spawned
  biotech sector
• From inception intimately tied to academic
  scientists
• Tacit knowledge geography matters (Zucker et al)

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Basic Mechanisms of Disease Overlapping Roles

• Pathways and targets
• Academia is major source
• Animal labs for physiological research
• Patient population genetic abnormalities/polymorp
  hisms
• Research tools
• CU 40 of Invention Reports 40 of patents
  gt50 of licenses
• Industry also a source of research tools and
  basic mechanisms

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Synthesis of New Drugs

• Practice before 1992
• 21 high Rx impact drugs 1965-1992 (Cockburn
  Henderson, 98)
• 16 drugs had public sector involvement in
  enabling discovery
• 2 compounds synthesized in public labs
  (cisplatin, foscarnet)
• 15 important drugs 1967-1992 (Mansfield, 91
  98)
• 2 drugs synthesized and patented by publicly
  funded researchers

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Important Drugs Synthesized in 1980sCockburn
Henderson
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Clinical Evaluation

• Trials designed for
• market entry
• Placebo controlled or
• Head to head comparison to first mover
• market expansion
• therapeutic optimization
• Head to head comparison with relevant
  alternatives
• Enroll diverse study population
• Broad range of outcomes
• Incremental benefits of additive therapies

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Role in Clinical Evaluation?

• Industry sponsored trials
• 30 RD budget
• Clinical enrollment/data
• Diminishing roles of AMCs
• Design management
• Inhouse
• CROs
• Academia

• Govt sponsored trials
• 10 NIH budget (all technologies)
• Trial design and management, primarily academia
• New uses for off-patent drugs
• Therapeutic optimization

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Indications Discovered Post-Marketing
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Important Economic Phenomenon?

• Accounted for 40 of revenues from 1995 block
  buster drugs
• Similar pattern for medical devices
• Much of this learning by doing occurred in AMCs
• Feedback to upstream RD

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Conclusions

• Extensive interactions between academia and
  industry
• Linkages among different types of research
  practice
• Many new opportunities for drug development

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Conclusions

• Currently linkages often occur opportunistically
• Enhanced drug generation, in the public interest,
  requires strategic approach maximize the upsides
  and minimize the downsides of effective
  collaboration