Evolving Public Health Challenges

1. The NIH Roadmapfor Medical ResearchBelindaSeto, Ph.D.Deputy DirectorNational Institute of Biomedical Imaging and Bioengineering

2. Evolving Public Health Challenges Acute to Chronic Conditions AgingPopulation Health Disparities EmergingDiseases Biodefense

3. NIH opportunities and challenges… • 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. How was the Roadmap developed? • Extensive consultations with stakeholders, scientists, health care providers • Discussions addressed: • 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?

5. 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 set of initiatives that are central to extending the quality of healthy life for people in this country and around the world. • A vision for a more efficient and productive system of biomedical and behavioral research.

6. Roadblocks Bench Bedside Public

7. Standard Model Laboratory Research Clinical Research Public Health Translational Research Population Research

8. The Way it Should Work Patient-oriented Clinical Research Laboratory Research Population-based Clinical Research Clinical Trials

9. Three themes emerged…. NIH

10. New Pathways to Discovery Bench Bedside Public Building Blocks and Pathways Molecular Libraries and Imaging Bioinformatics and Computational Biology Structural Biology Nanomedicine

11. New Pathways to Discovery Initiatives within this theme address technologies and approaches necessary to meet contemporary research challenges. Including: • Grasping the emerging complexity of biology • Understanding biological systems • Accessing biological data, technologies, and other scientific resources • Promoting Multi-and Interdisciplinary research

12. Potential Outcomes of Molecular Libraries & Imaging Probes Activities • Development of research tools (molecular probes and novel assays) to facilitate studies of biology and pathophysiology • Advances in biological research leading to the identification and validation of novel biological targets for therapeutics development • Discovery of biological markers to monitor disease progression and to predict treatment response

13. National Centers for Biomedical Computing • Partnerships of: • Computer scientists • Biomedical computational scientists • Experimental and clinical biomedical and behavioral researchers • Focused on software rather than hardware • Each National Center to have Driving Biological Projects • Programs in preparation for partnerships between individual investigators and National Centers

14. There is a need to understand complex biology at a systems level Brent Cell, 2000

15. Research Teams of the Future Initiatives within this theme provides mechanisms for interdisciplinary research, high-risk strategies and public-private partnerships. Including: • Encouraging Multi- and Interdisciplinary teams • Supporting larger, coordinated, resource sharing teams • Preserving investigator-initiated strategy • Promoting investigators to take creative, unexplored avenues of research

16. Exploratory Centers (P20s) for Interdisciplinary Research • Planning centers will combine various aspects of current disciplines to provide new ways of thinking about and addressing key and complex problems in the biomedical sciences • Institutions challenged to resolve local barriers to interdisciplinary research including assignment of credit for co-investigators and maintenance of complex research teams • NIH challenged to resolve barriers to interdisciplinary research including recognition of “co-PI”, developing interdisciplinary study sections and developing interdisciplinary (and cross-IC) research management teams

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

18. NIH Director’s Pioneer Award • New program to support individuals with untested, potentially groundbreaking ideas! • Encourages innovation, risk-taking • Totally new application and peer review process • Expected to be highly competitive • Expanded eligibility – (not only traditional biomedical investigators) • Provides $500,000/year for 5 years

19. 2004 NIH Director’s Pioneer Award Recipients • Larry Abbott, Ph.D., Brandeis University, Waltham, MA • George Daley, M.D., Ph.D., Children’s Hospital Boston, Boston, MA • Homme Hellinga, Ph.D., Duke University Medical Center, Durham, NC • Joseph McCune, M.D., Ph.D., J. David Gladstone Institutes, San Francisco, CA • Steven McKnight, Ph.D., University of Texas Southwestern Medical Center, Dallas, TX • Chad Mirkin, Ph.D., Northwestern University, Evanston, IL • Rob Phillips, Ph.D., California Institute of Technology, Pasadena, CA • Stephen Quake, Ph.D., California Institute of Technology, Pasadena, CA • Sunney Xie, Ph.D., Harvard University, Cambridge, MA • The nine recipients represent a broad spectrum of scientific disciplines

20. Reengineering the Clinical Research Enterprise Initiatives within this theme address the need for developing new strategies to enhance the infrastructure and capacity for clinical research and reenergize the clinical research workforce. Including: • Promoting better integration of existing clinical research networks, including NECTAR • Supporting translational research • Encouraging the development of technologies to improve the assessment of clinical outcomes • Harmonizing regulatory processes • Enhancing training for clinical researchers

21. Translational Research: I Research Cores: • Product development planning • Regulatory advice and assistance for pre-IND phase • Facilities to manufacture, formulate and test small molecules and biologicals • Resources for animal pharmacology and toxicology Status: • Pilot phase, for small molecules only, being implemented in FY’05 through NCI RAID

22. Translational Research: II 3 Types of Regional Translational Research Centers: • Clinical services (regulatory, data management, patient recruitment …) provided regionally • Core technologies provided nationally • Combined centers Status: • Planning grants to be funded, FY’05-’06 (~20/yr) • Centers to be funded FY’06-’08 (~8/yr)

23. Clinical Workforce Training Double the size of the intramural (medical student) clinical research training program • Program initiated in FY’04 Develop conceptual model and assess feasibility of a Nationwide (community-based) Clinical Research Associates Program • Contracts issued in FY’04-soon to be funded Multidisciplinary Clinical Research Career Development Program (K12) • Grants funded in FY’04

24. Clinical Research Networks and National Informatics Network (NECTAR) Inventory and evaluate best practices in research Networks and Informatics Systems • Contract issued in FY’04-soon to be funded Integrate and expand (currently existing) clinical research networks with new or well developed electronic informatics components • Responses to BAA reviewed…ready for 04 funding

25. Patient-Reported Outcomes Measurement Information System (PROMIS) Computer based adaptive testing to increase sensitivity of patient-reported symptoms • Data coordinating center and ?5 collaborating research sites funded in FY’04

26. Harmonization of Regulatory Requirements for Clinical Research Scope of work: • Adverse event reporting • Human subjects protection • Central IRBs • Auditing and monitoring clinical trials • HIPAA, privacy, conflict of interest policies • Investigator registration, financial disclosure • Standards for electronic data submission/reporting Status: • Office created in OSP

27. National Clinical Research Associates • Diverse national group of trained and certified disease-focused, community-based providers linked to academic translational research centers • Primarily made up of physicians with prior clinical research training who left academia • Will enroll and follow their own patients using a standardized information system • Will accelerate translation of results into practice • Studying feasibility: Barriers? Communities? Incentives needed?

28. Engaging the Public in Clinical Research Priorities • Building trust • On-going communication between research community & public • Education • NIH Public Trust Initiative

29. Roadmap Funding(dollars in millions) Directors Fund 35 60 ?? Roadmap initiative is about .9% of NIH budget Totals on table may not add due to rounding

30. FY 2005 Request = $28,757 Million Roadmap Percent to Total NIH Budget Non-Roadmap 99.2% ($28,520 Million) Roadmap 0.8% ($237 Million)

31. Roadmap Requests for Applications (RFA's)Funding Opportunities for FY05: Molecular Libraries and Imaging

32. Roadmap Requests for Applications (RFA's)Funding Opportunities for FY05: Building Blocks, Biological Pathways Structural Biology Bioinformatics and Computational Biology

33. Roadmap Requests for Applications (RFA's)Funding Opportunities for FY05: Nanomedine* * Limited “RFA” will be competitive only to those who have been awarded and successfully completed the “Concept Development Memo” phase Interdisciplinary Research High Risk Research

34. Roadmap Requests for Applications (RFA's)Funding Opportunities for FY05: Re-engineering the Clinical Research Enterprise

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

36. The NIH Roadmap:A Work in Progress