The NIH Roadmap
for Medical Research
Elias A. Zerhouni, M.D.
February 27, 2004
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?
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
Evolving Public Health
Challenges
Acute to chronic conditions
Aging Population
Health Disparities
Emerging Diseases
Biodefense
U.S. Health Expenditures
(Percentage of GDP)
18
Actual
Projected
Percent
16
14
12
10
1985 1987 1989 1991 1993 1995 1997 1999 2001 2003 2005 2007 2009 2011
Year
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
How was the Roadmap
developed?
Extensive consultations with stakeholders,
scientists, health care providers
– What are today’s scientific challenges?
– What are the roadblocks to progress?
– What do we need to do to overcome
roadblocks?
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.
NIH Roadmap for Medical Research
New Pathways
to Discovery
NIH
Research Teams
of the Future
Re-engineering the
Clinical Research Enterprise
Emerging Complexity of Biology
Need to understand
biological systems
Brent Cell, 2000
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
Multi- and Interdisciplinary Research will
be Required to Solve the “Puzzle” of
Complex Diseases and Conditions
Genes
Behavior
Diet/Nutrition
Infectious agents
Environment
Society
???
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
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
NIH Roadmap Strategy
Interdisciplinary Research
Pioneer Award
Nanomedicine
Public Private
Partnerships
Bench
Building Blocks
Pathways
Molecular Libraries
Bioinformatics and
Computational
Biology
Structural Biology
Nanomedicine
Bedside
Clinical
Research
Informatics
Translational
Research
Initiatives
Training
National Clinical Research
Associates
Practice
Integrated Research
Networks
Clinical outcomes
Re-engineering the
Clinical Research
Enterprise
Molecular
Libraries
and Imaging
Building Blocks,
Biological Pathways
and Networks
Clinical
Enterprise
Public-Private
Partnerships
Implementation
Groups
Bioinformatics and
Computational Biology
High-risk
Research
Interdisciplinary
Research
Research Teams
Structural
Biology
Nanomedicine
New Pathways
to Discovery
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
Roadmap Funding
dollars in millions
FY 2004 Funding = $128.3 (dollars in millions)
New Pathways
to Discovery
$64.1
$26.6
Research Teams
of the Future
NIH
$37.6
Re-engineering the Clinical
Research Enterprise
Roadmap Funding
dollars 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
0.34% 0.63%
To be competed for in a common pool
of initiatives by all researchers from every discipline
~0.9%
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
“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
“Yes, but we’re already doing those
things. What’s 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
There is no wrong time to
do the right thing.
NIH
Ideas
People
Resources
Leadership