RCE Section Review March 7, 2008

1. RCE Section ReviewMarch 7, 2008

2. RCE Team • Rona Hirschberg • Susan Garges • Bill Angus • Michael Schaefer • JoJo Stemple

3. RCE Program Goals • Basic research to understand category A-C and EID agents • Translational capacity leading to drugs, vaccines, and diagnostics • Expand the pool of researchers and technical personnel for biodefense and EID research • Regional facilities for research • Assist emergency responses

4. Topics for Today • RCE Program Evaluation – Rona • RCE Recompetition – Mike • Product Development and Emergency Response Activities – Bill • RCE Database – JoJo • RCE Highlights -- Sue

5. RCE Interim Program Evaluation

6. Provide evidence to assess program success Inform development of RFA Enhance the effectiveness of the program as a whole Pilot test measures, data collection and analysis protocols to inform future, definitive evaluations of the RCE Plan for future, definitive evaluations Enhance evaluation capacity Evaluation Goals

7. Overall Process Obtain stakeholder input regarding program success factors Define a representation of what the program should do (logic model) Determine critical areas of the program to evaluate Develop specific measures relating to the evaluation areas Gather and analyze data, prepare report Implement findings for improvement

8. Logic Model with 8 Focal Areas Activities Outputs Outcomes Inputs Impact Translate and Apply Science to Practice Manage Funds flexibly and efficiently Innovative & Flexible response to changing needs and opportunities Leveraged funds from elsewhere (Integration & Synergy) Establish Management structures & processes RCE Program Funding Product Development (Translational Activities) Capacity to serve as a regional resource Scientific Knowledge on Category A,B,C organisms (Research ) Collaboration & Communication Leadership with expertise (Integration & Synergy) Expanded cadre of investigators (Recruit and Train staff) Better response to infectious diseases that threaten humans Build Infrastructure Readiness to Respond in an Emergency Synergy between Centers (Integration & Synergy) Biodefense Priorities Recruit & Train staff Researchresultson Category A,B,C organisms Conduct Researchon Category A,B,C organisms

9. Areas for Evaluation Conduct research Research knowledge Communication/collaboration/synergy Support emergency response Translational activities Innovation/flexibility Bringing people to the field Leveraging funds

10. Collaboration and Communication

11. Summary of Participation and Collaboration Over 290 institutions involved in 48 states and Puerto Rico, 488 PIs and 563 projects 41% of project teams are cross institutional 44% of follow-on (non RCE) projects involve cross institutional teams 51% of publications have cross institutional co-authorship Only 4 of 477 papers had cross-RCE authorship

13. Research Conducted on Category A, B and C Agents

14. * (partial year)

15. * (partial year)

18. Scientific Knowledge on Category A, B and C Agents

19. Total Publications

20. Total Number of RCE and NIAID Publications on Category A Agents, 2004-2007

21. Observed and Expected Citations for All RCE Program Publications by Year, 2004-2007

22. Selected Journals, their Journal Impact Factor and Number of RCE Publications in those Journals.

23. Publications RCE publication productivity is increasing over time. RCE publications are well regarded, as indicated by citations from other publications. RCE articles have been published consistently in frequently cited journals. Citation of RCE publications is significantly higher than for comparable articles in the same journals and fields

24. Overall Conclusions Program has had a wide reach Collaboration, communication and synergy are occurring Research on appropriate organisms and subjects Research is contributing to scientific knowledge Program structure allows for innovation and flexibility

25. Conclusions, con’t Expanded cadre of investigators has resulted Readiness to respond has been shown, but inconsistent Early indicators of progress toward products is evident Leveraging of RCE funds is occurring

26. RCE Recompetition

27. New RFA Regional Centers of Excellence  for Biodefense and Emerging Infectious Diseases Research (RCE) [U54] http://grants.nih.gov/grants/guide/rfa-files/RFA-AI-08-002.html • Released Dec. 21, 2007 • Applications due June 3, 2008 • Open to all applicants, new and continuing • Expect to fund up to 10 centers about 3/09

28. Funds Available and Who May Apply • $91M for up to 10 awards • Awards expected to range from $3-10M direct costs (mistake in RFA) • New and competing continuation applications will be accepted

29. Consortium • An interactive group of investigators at more than one institution • Mix of expertise and capabilities • Big enough to get the job done • Small enough to work • One applicant organization, one PI

30. Required Elements of an RCE Application • Background, Strategic plan, and Management Structure • Research program • Scientific facilities cores • Developmental research plan • Career development and training programs • Biocontainment needs and facilities • Emergency response plans • Evaluation plans

31. Regions - DHHS -RegionI: CT, ME, MA, NH, RI, VT -Region II: NJ, NY, PR, VI -Region III: DE, D.C., MD, PA, VA, WV -Region IV: KY, MS, NC, TN, AL, FL, GA, SC -Region V: IL, IN, MI, MN, OH, WI -Region VI: AR, LA, NM, OK, TX -Region VII: IA, KS, MO, NE -Region VIII: CO, MT, ND, SD, UT, WY -Region IX: AZ, CA, HI, NV -Region X: AK, ID, OR, WA

32. Regional hub of activity Must serve as a regional facilities resource Must respond at the regional level in the event of a biodefense emergency Foster new research projects and human resource development with a regional basis Linkages to regional institutions Does not need to include all institutions in the region May include institutions and investigators from other regions Funded Regional Centers will interact with each other Will be part of a national network Regionality

33. Research Themes • One or a few • Focal points for RCE activities • Based on strengths • Provide coherence and synergy • Important review criterion • Organisms, platforms, approaches, etc.

34. Research Program • Integrated research program leading to practical solutions for category A-C and EID agents • Basic > translational > early clinical • At least five projects, a mix of types • Project = R01-like or P01-like • Integrated with themes • Balance of organisms and approaches • State-of-the-art approaches • Projects phase in and out as the research evolves

35. Scope • Category A-C and EID lists • Mostly BioD, at least 51% • Zoonotic diseases only if related to human disease • Will not fund: surveillance, public health, animal health, health care delivery, environmental detection, etc. • Less emphasis on well studied issues: anthrax, smallpox, influenza • Do not duplicate other programs: genomics, bioinformatics, FWDN, etc.

36. Clinical Studies and Trials • Clinical studies. Clinical Terms of Award apply. Needs program approval. • Clinical trials. Clinical Terms apply. • Phase 1 only • In application or later • Needs DMID input and approval • Discuss with program first • No foreign clinical trials • See RFA for what to submit • Use existing infrastructure

37. Product Development • Translational activities beyond proof-of-concept level • Ready for serious pre-clinical studies • Requires a PD plan with milestones • Requires involvement of appropriate consultants • Consider forming partnerships for moving forward

38. Foreign Components • Allowed as part of a domestic project • Must integrate into a parent project • Up to $100K direct costs per project per year • Must offer unique opportunities • Need a strong justification • No foreign clinical trials

39. Facilities Cores • Integral to the strategic plan and research • Number and kinds appropriate for each RCE • Basic and translational, but not clinical • Available for a biodefense of EID emergency • Regional resource • Management plans and key personnel • Leverage and linkages • Need strong justification • May not include research

40. Developmental Research Projects • Small, short term start-up projects • Funded at RCE level • Capitalize on progress in field • Important for flexibility • May “graduate” into Center projects or R01s • Need clear plans and procedures • Key element of success • Projects are not submitted as part of the application

41. Career Development and Training • Increase human resources for biodefense and EID research • At least two projects: one for individuals and one for groups • Complement the research activities and themes • Innovative approaches • Many options • Individual mentored research projects are not submitted as part of the application

42. Timetable

43. How RCEs Will Be the Same • Same overall goals • Same general structure • Continued emphasis on flexibility and synergy • Emphasis on portfolio of basic to applied research • Goal of product concepts • Regionality

44. How RCEs Will Change • Expand to include EID as well as A-C pathogens • Allow some foreign interactions • Focus more on A-C and EID issues that are not covered by other programs • Required evaluation component • Greater focus on themes to promote synergy and collaboration • Align priorities with new strategic plan

45. RCE Emergency Response

46. Emergency Response

47. Activities • Compiled list of resources that can be used in an emergency • Participation in a public health emergency • Compiled list of experts who can help in an emergency. • RCE membership on state/local committees tasked with emergency response. • Identified designated RCE contact person for each locality. • Participation in simulations (in the field) or in table top exercises • Conducted public outreach.

48. Emergency Response Examples Spinach Related E-coli Outbreak (Multiple states, 2006) NWRC offered rapid, in-depth whole genome sequencing of a prototypical outbreak strain. The NWRCE analysis complemented the sequence analysis performed at Michigan State University by providing greater genetic detail in areas of ambiguity Suspected Tularemia (St. Louis, 2006) Activated the MRCE Emergency Management Group phone tree and disseminated information to Washington University School of Medicine and our hospitals. Also assisted local health departments with the development of an appropriate case definition to enhance surveillance. Surge Capacity NERCE-affiliated investigators have been registered with the CDC under the Massachusetts SLI select agent registration, allowing work as back-up personnel in their laboratories in the event of an biological emergency

49. Trans-RCE Emergency Response • In March 2006, GLRCE and SERCEB convened teleconference of ER personnel from all ten RCEs. • Database of contact information for ER leadership nationwide developed • Discussion areas for national collaboration: • Potential for the RCEs to provide high-end surge capacity in areas such as diagnostics, care of research animals, plans for researcher displacement, augmentation of field epidemiology capabilities if requested by public health authorities • Provision of content expertise, education on a national level • Development of centralized media training for RCE personnel.

50. Communications • Media Training for Bioscientists (MARCE) • Scenario Exercise, Case Studies, Mock News Conference • Policy Ethics and Law Core (SERCEB) • Dual use, public health ethics in pandemics, social and legal implications of biodefense research • Law, Policy and Ethics Core (WRCE) • Federal policy, law and regulations, human subjects testing, intellectual property