The NIH Roadmap

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

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

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

21. Multidisciplinary K12 Career Development Program

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

29. The NIH Roadmap:A Work in Progress