1. The NIH Roadmap

2. Imperatives for NIH Accelerate pace of discoveries in life sciences
More rapid translation from laboratories to patients and back
Develop novel approaches orders of magnitude more effective than current ones
Develop new strategies = NIH Roadmap

3. Roadmap Participants were asked:
What are today’s scientific challenges?
What are the roadblocks to progress?
What do we need to do to overcome roadblocks?
What can’t be accomplished by any single Institute – but is the responsibility of NIH as a whole?

4. The Problem
Bench
Bedside
Practice
System Broken

5. NIH Roadmap: three themes emerged
New Pathways to Discovery
Research Teams of the Future
Re-engineering the Clinical Research Enterprise

6. Criteria for Roadmap Initiatives
Is it ‘transforming’ -- will it change how or what biomedical research is conducted in the next decades?
Would its outcome enhance the ability of all ICs to achieve their missions?
Can the NIH afford to NOT to do it?
Will it be compelling to our stakeholders, especially the public?
Is it something that no other entity can or will do?

7. New Pathways to Discovery
Bench
Bedside
Practice
Building Blocks
and Pathways
Molecular Libraries
Bioinformatics
Computational
Biology
Nanomedicine

8. Biological Pathways and Networks
Understand in quantitative terms how gene regulatory networks, signal transduction pathways, and metabolic pathways are integrated to orchestrate normal development
Understand responses to internal and external stimuli in highly complex organisms
Understand how pathways and networks are perturbed in disease.

9. Bioinformatics and Computational Biology
Deploy a rigorous biomedical computing environment to analyze, model, understand and predict dynamic and complex biomedical systems.
Be able to integrate data and knowledge at all levels of organization.

10. Molecular Libraries Chemical Diversity -Publicly available database
-Biologically relevant chemical space optimally populated with naturally occurring and synthetic compounds
Screening
-Small molecule activators and inactivators available to researchers for individual functions of every gene product in human genome
Drug development as needed

11. Research Teams of the Future
Interdisciplinary
Research Teams;
Pioneer Award
Public-Private
Partnerships
Bench
Bedside
Practice
Building Blocks
and Pathways
Molecular Libraries
Bioinformatics
Computational Biology
Nanomedicine

12. Challenges to Interdisciplinary Research
The current system of academic advancement favors the independent investigator
Most institutions house scientists in discrete departments
Interdisciplinary research teams take time to assemble and require unique resources
a single PI is recognized; indirect costs from collaborating institutions become embedded in direct costs of the “lead” institution indirect costs follow the PI.

13. Interdisciplinary Curriculum Development Awards
Support the development of innovative courses and curricula designed to train interdisciplinary scientists
Curricula can be designed for undergraduate, pre-doctoral or postdoctoral students, or combinations of these.
Focus on programs that encourage the integration of quantitative, physical, behavioral, or social sciences with the traditional biomedical sciences

14. NIH Director’s Pioneer Award
New program to support individuals with untested ideas that are potentially groundbreaking
Encourages innovation, risk-taking
Totally new application and peer review process
Provides $500 K/year for 5 years
Expected to be highly competitive

15. Re-engineering Clinical Research
Interdisciplinary
Research
Pioneer Award
Public-Private Partnerships
Bench
Bedside
Practice
Building Blocks
and Pathways
Molecular Libraries
Bioinformatics
Computational
Biology
Nanomedicine
Integrated Research Networks
Clinical Research Informatics
NIH Clinical Research Associates
Clinical outcomes
Clinical Research Policy
Training
Translational
Research
Initiatives

16. Integration of Clinical Research Networks
Link existing networks so clinical studies and trials can be conducted more effectively
Ensure that patients, physicians, and scientists form true “Communities of Research”

17. Translational Research Centers
Provide sophisticated advice help scientists bring a new product from the bench to clinical use, including:
Laboratory studies to understand a therapy’s mechanisms of action
Pre-clinical drug synthesis, toxicity testing
Sophisticated manufacturing capacity
Expert advice to ensure that drug-development regulations are observed

18. Patient Reported Disease Outcomes
Establish a collaborative of investigators to improve measurement of patient-reported outcomes.
Focus on the collection of self-report data from a diverse population of individuals, including racial and ethnic minorities, having a variety of chronic diseases.
Compare the performance of specific items, instruments, and models across diverse clinical populations
Develop common metrics by which scores on new and existing instruments can be standardized and/or linked

19. National Electronic Clinical Trials/Research Network (NECTAR)
Common data standards, informatics
Software application tools for protocol preparation, IRB management, adverse event reports
Use existing networks to rapidly address questions beyond their traditional scope

20. National Clinical Research Associates
Goal:
Create a diverse national group of trained and certified community health care providers
Providers will enroll and follow their own patients
Means to accelerate translation of results into practice
Steps:
Determine feasibility: Barriers? Communities? Incentives needed?
Inventory training methods, best practices
Develop core competencies, certification
Future: training programs

21. Multidisciplinary K12 Career Development Program
Goal:
Promote development of investigators from a variety of disciplines (MD, DDS,PhD, RN, MPH..)
To be trained in multidisciplinary team settings
Features:
Up to 5 years of training
Core didactic courses, project-specific training
Mentored research experience in team settings
Faculty and mentor support to protect their time
Tuition support
Annual meetings

22. Current Polices, Procedures, Regulations = Major “Traffic Jam”
The clinical research community faces redundant and sometimes variable requirements to address fundamentally the same oversight concerns (e.g., AERs, COIs)
Variability exists not only among various Federal agencies, but also within the NIH and it hampers efficiency and effectiveness

23. Toward a Solution: NIH Role
NIH promotes the responsible conduct of high quality clinical research
Opportunity to convene other federal agencies and to facilitate dialogue and change

24. Roadmap Clinical Research Policy Coordination Initiative
Improve coordination and simplify requirements for clinical research in ways that enhance public trust
Adverse event reporting
Human subjects protections, including DSMB-IRB interactions and consent procedures
Auditing and monitoring clinical trials
HIPAA, privacy, conflict of interest policies
Investigator registration, financial disclosure
Standards for electronic data submission/reporting

25. Policy Coordination Implementation Plan
Need for sustained attention
Identify and prioritize issues
Maintain open communication with all stakeholders – investigators, patients, families, institutions, communities, policy-makers
Work collaboratively with sibling Agencies
Develop tools for investigators to facilitate compliance
Education and outreach

26. Roadmap Implementation Groups
Molecular Libraries and Imaging
Building Blocks, Biological Pathways and Networks
Structural Biology
Bioinformatics and Computational Biology
Nanomedicine
Interdisciplinary Research
High-risk Research
Public-Private Partnerships
Re-engineering the Clinical Research Enterprise

27. Roadmap Implementation
All Institutes and Centers committed to invest jointly in a pool of resources to support current and future Roadmap initiatives
$128 M in FY 2004 (DDF funds and 0.34% each ICs budget)
Over $2 B by FY 2009

28. Consultation www.nihroadmap.nih.gov
Participation, consultation, collaboration, and funding are needed from patients, health care providers, foundations, industry, academia, Federal partners …all stakeholders

29. The NIH Roadmap: A Work in Progress