1. The NIH Roadmap for Medical Research
Elias A. Zerhouni, M.D.
February 27, 2004

2. 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?

3. 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

4. Evolving Public Health Challenges
Acute to chronic conditions
Aging Population
Health Disparities
Emerging Diseases
Biodefense

5. U.S. Health Expenditures
(Percentage of GDP) 18
Actual
Projected 16
Percent 14 12 10
1985
1987
1989
1991
1993
1995
1997
1999
2001
2003
2005
2007
2009
2011
Year

6. 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

7. 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?

8. 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.

9. NIH Roadmap for Medical Research

10. Emerging Complexity of Biology

11. Need to understand biological systems
Brent Cell, 2000

12. 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

13. Multi- and Interdisciplinary Research will be Required to Solve the “Puzzle” of Complex Diseases and Conditions
Genes
Behavior
Diet/Nutrition
Infectious agents
Environment
Society
???

14. 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

15. 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

16. NIH Roadmap Strategy Bench Bedside Practice Interdisciplinary Research
Building Blocks Pathways
Molecular Libraries
Bioinformatics and
Computational
Biology
Structural Biology
Nanomedicine
Translational
Research
Initiatives
Clinical
Informatics
Integrated Research
Networks
Clinical outcomes
Training
National Clinical Research Associates
Interdisciplinary Research
Pioneer Award
Public Private
Partnerships

17. Clinical
Enterprise
New Pathways to Discovery
Research Teams

18. 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

19. Roadmap Funding dollars in millions
FY 2004 Funding = $128.3 (dollars in millions)
NIH
$64.1
$26.6
$37.6

20. 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 38 61
120
174
214
227
833
128
237
332
448
520
507
2,172
~0.9%
0.34% %
To be competed for in a common pool
of initiatives by all researchers from every discipline

21. 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

22. “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

23. “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

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

25. NIH
Ideas
People
Resources
Leadership