1. CBER Research: OBRR Office Site Visit Kathryn M. Carbone, MD Associate Director of Research CBER/FDA

2. CBER Research Managing to Program Goals  Prepare for long-term programmatic needs AND for crisis responsiveness  Outcomes driven: Identifying and resolving specific, high priority scientific challenges in product evaluation –PRODUCT DRIVEN, expertise supported  Focus on critical gaps in scientific tools and knowledge for product evaluation  Support product development for critical, unmet public health needs

3. CBER Research Solutions  Multidisciplinary, coordinated team research for regulatory challenges –High Priority: Internal and External Communication  CBER Research Quality Initiative: Public and expert evaluation of CBER Research through peer reviewed journals, external laboratory Site Visits, Advisory Committee/stakeholder input,  Increasing CBER Research impact, including visibility (web site; workshops; meeting representation)  Funding these efforts (OC & partnerships)  Providing core research support: Molecular biology/proteomics core facility and animal facilities

4. CBER RESEARCH MANAGEMENT INITIATIVE REGULATORY AND PUBLIC HEALTH PORTFOLIO ANALYSIS & KEY SCIENTIFIC CHALLENGES CBER RESEARCH PRIORITIES END OF FY SCIENTIFIC PROGRAM REVIEW AND ANNUAL REPORT OFFICE RESEARCH PLANS ADVISORY COMMITTEE PRESENTATIONS AND OTHER INPUT OUTCOMES FUTURE PLANS

5. Regulatory Workload Analysis REGULATORY AND PUBLIC HEALTH PORTFOLIO ANALYSIS & KEY SCIENTIFIC CHALLENGES Key Policy Activities Public Health & Emerging Issues

6. CBER RESEARCH PRIORITIES Regulatory Workload Impact on Facilitating Product Development Safety & Efficacy Product Quality Public Health Impact Unique CBER Expertise Impact on Product Success

7. 07 Research Priorities  Cell Substrate Safety, Quality and Assays  Vaccine Adjuvants  Influenza vaccine: safety, quality, assays/reagents  Disease models/surrogate markers  Unique toxicology model development for gene therapy  Quality, characterization, distribution and fate of genes, stem cells  Bayesian/adaptive study designs for clinical trials  Improve post market surveillance techniques  New analytical tools/expertise in product characterization, quality, clinical assessment (NMR, mass spec, omics)  Nanotechnology  Pathogen inactivation, removal, rapid microbial detection  EID preparedness/assays  Characterization/safety of “blood substitutes”

8. CBER RESEARCH PLAN OVRR, OBRR OCTGT, OBE RESARCH PLAN Leveraged Research Projects

9. Communication of CBER science is critical for peer review of findings and enhancing impact of innovations on the evaluation process

10. Scientific Expertise Cross-Office Teams Research Communication & Coordination Improving Internal FDA and External Stakeholder Expertise Access

11. OFFICE SCIENTIFIC PROGRAM ANNUAL REPORT Research Program Evaluation RESEARCH REVIEW & EVALUATION ANNUAL RESEARCH PROGRAM REPORTS: Web based Achievements Future Plans FOUR YEAR RESEARCH PROGRAM EVALUATION External Site Visit PCE cyclical review

12. Annual Internal Review  Web-based Research Reporting –Achievements –Future plans  Reviewed by Office Leadership: –Lab Chief –Division Director –ADR/Office Director

13. Rating Research Program Success  Achievements –Return on research resources expended  Direct impact of research on regulatory challenges  Quality: Peer reviewed publications & invited talks  Contribution Guidances, policy, workshops –Impact on international harmonization –Review workload and quality  Future Research Plans-short & long term –Unique CBER Expertise contribution –Priority area & response to public health issue –Direct impact on regulatory challenge –Feasibility: Expertise, collaborations, infrastructure –Quality –Return on investment

14. 4 Year Cycle External Review: Scientific Expertise Site Visits  Conducted by appropriate CBER’s Advisory Committee for each Product Office  Site Visit Team Chaired/Co-chaired by two Advisory Committee Members supplemented with appropriate outside Scientific Experts  Held every four years for each Laboratory unit –Teams of Researcher-Reviewers & their Research Programs within Product Office –Preparation and submission of Site Visit book by each PI detailing achievements past four years and proposal for future research next four years –Formal presentations to Site Visit Team –Site Visit Team holds individual interviews with each PI  Site Visit Report presented to and approved by full Advisory Committee vote  Researcher-Reviewer and supervisors must respond to Site Visit Report comments and recommendations within 6 months

15. 4 Year Cycle Internal Review: Promotions & Conversions Evaluation Committee  Managed by Center ADR to provide recommendations to Center Director on personnel actions & cyclical review assessments for all Researcher-Reviewer Staffs  Formal SOP & procedures established in conjunction with Office of the Commissioner’s Peer Review policy  Chaired and staffed by Senior Researcher-Reviewers and Regulatory Scientists from each Product Office  All Researcher-Reviewer staffs are evaluated every four years for cyclical progress review and for all promotions or conversions to permanent staff  Formal regulatory workload & quality assessment  Formal research productivity, mission relevance and quality assessment

16. Stakeholder Input ADVISORY COMMITTEE & PUBLIC INPUT OFFICE SCIENTIFIC PROGRAM ANNUAL REPORT OFFICE RESEARCH PLAN REGULATORY AND PUBLIC HEALTH PORTFOLIO ANALYSIS & KEY SCIENTIFIC CHALLENGES RESEARCH PRIORITIES

17. Advisory Committee Office Research Site Visit Reports 2005-6  “Committee members who have been familiar with the research programs…over a period of years note that there has been a striking improvement over time…: –Focus and relevance…The research presented for this review had direct relevance to the Critical Pathway of biologics product development and availability. –Quality. …the quality of the research has also improved; many of the ongoing studies are equal in quality to those in the intramural program at the National Institutes of Health (NIH) and of sufficient caliber to compete for RO1 and other NIH grants.  …strongly supports the FDA’s continued emphasis on the importance of having a strong intramural research program to support its Critical Pathway program for effective and efficient regulatory activities…  If we are to maintain our lead in health care development in the USA, regulatory science needs to be given the priority it deserves, independent of the short-term political and economic flurries that can derail progress.”

18. Advisory Committee Office Research Site Visit Reports 2005-6 II  Strengths –Productivity, scientific merit, mission relevant –Well recognized for outreach efforts –Complementary cross-Office expertise –Success at recruitment and retention –Core facilities –Leveraging and collaboration (NIH, CDC, NTP, Academia)

19. Advisory Committee Office Research Site Visit Reports 2005-6 Concerns I:  Increased regulatory workload and decreasing support  Best mechanism: Balance managing to mission relevance of research vs. micromanagement  Covering many research bases vs. focus on quality in fewer areas  Development of an explicit plan/strategic plan for 2-5 year priority research needs with regulatory and stakeholder input

20. Advisory Committee Office Research Site Visit Reports 2005-6 Concerns II:  Mentoring  Recruitment and retention  Increased research program visibility  Continuing education support  Increased collaboration within and outside FDA  Increased FDA base funding support for research  Creative leveraging support, e.g., FDA based Foundation for Research (like Jackson Foundation for DOD)  Need a public relations campaign  Need a system of reward for successful research

21. THANK YOU