The NIH Roadmap for Medical Research

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The NIH Roadmapfor Medical Research
Elias A. Zerhouni, M.D. February 27, 2004
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Questions, questions and more questions

• How and why was the NIH Roadmap developed?
• How is it being implemented?
• What are the initiatives?
• How will the Roadmap benefit my research area?

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Challenges for NIH

• Revolutionary and rapid changes in Science
• Increasing breadth of mission and growth
• Complex organization with many units(27
  institutes and centers, multiple program offices,
  e.g., OWHR, OAR, ORD, ...)
• Structured by Disease, Organ, Life stage,
  Disciplines .
• Rapid Convergence of Science

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Evolving Public Health Challenges

• Acute to chronic conditions

Aging Population
Health Disparities
Emerging Diseases
Biodefense
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U.S. Health Expenditures (Percentage of GDP)
18
Actual Projected
16
Percent
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12
10
1985
1987
1989
1991
1993
1995
1997
1999
2001
2003
2005
2007
2009
2011
Year
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Imperatives for NIH

• Accelerate pace of discoveries in life sciences
• Translate research more rapidly from laboratories
  to patients and back
• Explore novel approaches orders of magnitude more
  effective than current
• Develop new strategies NIH Roadmap

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How was the Roadmap developed?

• Extensive consultations with stakeholders,
  scientists, health care providers
• What are todays scientific challenges?
• What are the roadblocks to progress?
• What do we need to do to overcome roadblocks?

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What is the NIH Roadmap?

• A framework of priorities the NIH as a whole must
  address in order to optimize its entire research
  portfolio.
• A vision for a more efficient, innovative and
  productive system of biomedical and behavioral
  research.
• A set of initiatives that are central to
  extending the quality of healthy life for people
  in this country and around the world.

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NIH Roadmap for Medical Research
NIH
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Emerging Complexity of Biology
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Need to understand biological systems
Brent Cell, 2000
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The Biological Data of the Future

• Destructive
• Qualitative
• Uni-dimensional
• Low temporal resolution
• Low data density
• Variable standards
• Non cumulative

• Non-destructive
• Quantitative
• Multi-dimensional and spatially resolved
• High Temporal resolution
• High data density
• Stricter standards
• Cumulative

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Multi- and Interdisciplinary Research will be
Required to Solve the Puzzle of Complex
Diseases and Conditions
Genes Behavior Diet/Nutrition Infectious
agents Environment Society ???
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NIH Roadmap Research Teams of the Future Scale
and complexity of 21st C research require new
organizational models for scientific teams

• Multi-disciplinary and Inter-disciplinary Teams
• Larger, coordinated, resource sharing Teams
• Preserve the investigator(s)-initiated strategy

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Translational Research

• For historical reasons, clinical research has
  evolved haphazardly
• Started as cottage industry and select centers
• Now has more complex requirements regulation,
  technology, speed, efficiency
• Greater links to basic science
• Need transformation to move into the 21st Century
• Individual apprenticeship ? discipline of
  clinical research
• Uniform gauge ? harmonize rules, build
  infrastructure and create networks
• Focus on mentoring ? multidisciplinary teams
• The key value is access to well characterized
  cohorts of patients and biological samples

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NIH Roadmap Strategy
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Clinical Enterprise
New Pathways to Discovery
Research Teams
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Key elements of Roadmap funding and management

• All Institutes
• Participate with their scientific community in
  defining all components of the Roadmap
• Contribute equally and proportionately
• Participate directly in decision making and have
  a direct liaison to the Roadmap
• All Roadmap initiatives are offered for
  competition to researchers from all fields
• All research communities can compete for all
  initiatives
• The peer-review process will ensure appropriate
  expertise

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Roadmap Funding dollars in millions
FY 2004 Funding 128.3 (dollars in millions)
64.1
26.6
37.6
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Roadmap Fundingdollars in millions
FY04 FY05 FY06 FY07 FY08 FY09 Total
Pathways to Discovery 64 137 169 182 209 188 948
Research Teams 27 39 44 92 96 93 390
Clinical Research 38 61 120 174 214 227 833
Total 128 237 332 448 520 507 2,172
0.9
0.34 0.63
To be competed for in a common pool of
initiatives by all researchers from every
discipline
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NIH Roadmap Goal

• Accelerate basic research discoveries and speed
  translation of those discoveries into clinical
  practice
• Explicitly address roadblocks that slow the pace
  of medical research in improving the health of
  the American people

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How does the NIH Roadmap benefit my research
area?

• Speeding removal of major and fundamental
  roadblocks common to all diseases
• No Institute can solve these issues alone
• THIS IS A COMMON TRANS-NIH POOL OF TRANSFORMING
  INVESTMENTS OPEN TO ALL DISEASE AREAS FOR
  COMPETITION

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Yes, but were already doing those things.
Whats new?

• THE NIH ROADMAP POOLS RESOURCES FOR SPECIFIC
  ENABLING INVESTMENTS THAT INDIVIDUAL INSTITUTES
  COULD NOT UNDERTAKE
• Expanding molecular probe libraries publicly
  available to researchers by a factor of 7
• Increasing the number of publicly available
  molecular probes from less than 100,000 to over
  500,000
• Developing a common national research informatics
  platform allowing interoperability for data for
  all patients whether seen at a research hospital
  or in their own community (NECTAR)
• Improving the implementation of Breakthrough
  research trials through the creation of
  INTEGRATED research partnerships
• MORE RAPID DIFFUSION OF BEST PRACTICES TO PATIENTS

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• There is no wrong time to do the right thing.

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NIH
Ideas People Resources Leadership