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 16 Percent 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

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 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

Clinical Enterprise New Pathways to Discovery Research Teams

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) NIH $64.1 $26.6 $37.6

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% 0.63% To be competed for in a common pool of initiatives by all researchers from every discipline

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.

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