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

Elias A. Zerhouni, M.D.February 27, 2004

The NIH Roadmapfor Medical Research

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

Acute to chronic conditions

Evolving Public Health Challenges

Health Disparities

Emerging Diseases

Aging Population

Biodefense

U.S. Health Expenditures(Percentage of GDP)

18

16

14

12

101985 1987 1989 1991 1993 1995 1997 1999 2001 2003 2005 2007 2009 2011

Year

Per

cen

t

ActualProjected

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 Pathwaysto Discovery

Re-engineering theClinical Research Enterprise

Research Teamsof the Future

NIH

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

GenesBehaviorDiet/NutritionInfectious agentsEnvironment Society???

NIH Roadmap Research Teams of the Future

Scale and complexity of 21st C research requirenew organizational models for scientific teams

Multi-disciplinary and Inter-disciplinary TeamsLarger, coordinated, resource sharing

TeamsPreserve the investigator(s)-initiated

strategy

Translational ResearchFor 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

Bench Bedside Practice

Building Blocks PathwaysMolecular LibrariesBioinformatics and Computational BiologyStructural BiologyNanomedicine

TranslationalResearchInitiatives

Clinical ResearchInformatics

Integrated ResearchNetworksClinical outcomes

TrainingNational Clinical Research Associates

Interdisciplinary ResearchPioneer Award Nanomedicine

Public PrivatePartnerships

NIH Roadmap Strategy

Re-engineering the Clinical Research

Enterprise

Public-PrivatePartnerships

High-riskResearch

Interdisciplinary Research Nanomedicine

Bioinformatics and Computational Biology

StructuralBiology

Building Blocks, Biological Pathways

and Networks

Molecular Libraries

and Imaging

ImplementationImplementationGroupsGroups

New Pathways to DiscoveryResearch Teams

ClinicalEnterprise

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

New Pathwaysto Discovery

Re-engineering the Clinical Research Enterprise

Research Teamsof the Future

NIH

$64.1

$26.6 $37.6

FY 2004 Funding = $128.3 (dollars in millions)

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

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

0.34% 0.63% ~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 7Increasing the number of publicly available molecular probes from less than 100,000 to over 500,000Developing 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.

IdeasPeople

ResourcesLeadership

NIH