CBER Research:CBER Research:OBRR Office Site VisitOBRR Office Site Visit

Kathryn M. Carbone, MDKathryn M. Carbone, MDAssociate Director of ResearchAssociate Director of Research

CBER/FDACBER/FDA

CBER Research CBER Research Managing to Program GoalsManaging to Program Goals

Prepare for long-term programmatic Prepare for long-term programmatic needs AND for crisis responsivenessneeds AND for crisis responsiveness

Outcomes driven: Identifying and Outcomes driven: Identifying and resolving specific, high priority resolving specific, high priority scientific challenges in product scientific challenges in product evaluation evaluation – PRODUCT DRIVEN, expertise supportedPRODUCT DRIVEN, expertise supported

Focus on critical gaps in scientific tools Focus on critical gaps in scientific tools and knowledge for product evaluationand knowledge for product evaluation

Support product development for Support product development for critical, unmet public health needscritical, unmet public health needs

CBER Research SolutionsCBER Research Solutions Multidisciplinary, coordinated team research Multidisciplinary, coordinated team research

for regulatory challengesfor regulatory challenges– High Priority: Internal and External High Priority: Internal and External

CommunicationCommunication CBER Research Quality Initiative: Public and CBER Research Quality Initiative: Public and

expert evaluation of CBER Research through expert evaluation of CBER Research through peer reviewed journals, external laboratory Site peer reviewed journals, external laboratory Site Visits, Advisory Committee/stakeholder input, Visits, Advisory Committee/stakeholder input,

Increasing CBER Research impact, including Increasing CBER Research impact, including visibility (web site; workshops; meeting visibility (web site; workshops; meeting representation)representation)

Funding these efforts (OC & partnerships)Funding these efforts (OC & partnerships) Providing core research support: Molecular Providing core research support: Molecular

biology/proteomics core facility and animal biology/proteomics core facility and animal facilitiesfacilities

CBER RESEARCH MANAGEMENT INITIATIVE

REGULATORYAND PUBLIC

HEALTH PORTFOLIO

ANALYSIS & KEY SCIENTIFIC

CHALLENGES

CBERRESEARCH PRIORITIES

END OF FYSCIENTIFICPROGRAM REVIEW ANDANNUALREPORT

OFFICE RESEARCH

PLANS

ADVISORY COMMITTEEPRESENTATIONSAND OTHER INPUT•OUTCOMES•FUTURE PLANS

RegulatoryWorkloadAnalysis

REGULATORY AND

PUBLIC HEALTHPORTFOLIO

ANALYSIS & KEY SCIENTIFICCHALLENGES

KeyPolicy

Activities

Public Health& Emerging

Issues

CBERRESEARCH PRIORITIES

Regulatory Workload

Impact on FacilitatingProduct Development

Safety & Efficacy

ProductQuality

Public Health Impact Unique CBER Expertise

Impact onProduct Success

07 Research Priorities07 Research Priorities Cell Substrate Safety, Quality and Assays Cell Substrate Safety, Quality and Assays Vaccine AdjuvantsVaccine Adjuvants Influenza vaccine: safety, quality, assays/reagentsInfluenza vaccine: safety, quality, assays/reagents Disease models/surrogate markers Disease models/surrogate markers Unique toxicology model development for gene Unique toxicology model development for gene

therapytherapy Quality, characterization, distribution and fate of Quality, characterization, distribution and fate of

genes, stem cells genes, stem cells Bayesian/adaptive study designs for clinical trialsBayesian/adaptive study designs for clinical trials Improve post market surveillance techniquesImprove post market surveillance techniques New analytical tools/expertise in product New analytical tools/expertise in product

characterization, quality, clinical assessment (NMR, characterization, quality, clinical assessment (NMR, mass spec, omics) mass spec, omics)

Nanotechnology Nanotechnology Pathogen inactivation, removal, rapid microbial Pathogen inactivation, removal, rapid microbial

detectiondetection EID preparedness/assays EID preparedness/assays Characterization/safety of “blood substitutes”Characterization/safety of “blood substitutes”

CBERRESEARCH PLAN

OVRR, OBRROCTGT, OBE RESARCHPLAN

LeveragedResearchProjects

Communication of CBER scienceis critical for peer reviewof findings and enhancingimpact of innovations on theevaluation process

Scientific ExpertiseCross-OfficeTeams

ResearchCommunication& Coordination

Improving Internal FDA and External StakeholderExpertise Access

OFFICESCIENTIFIC PROGRAMANNUALREPORT

Research ProgramEvaluation

RESEARCHREVIEW &EVALUATION

ANNUALRESEARCHPROGRAMREPORTS: Web based•Achievements•Future PlansFOUR YEARRESEARCHPROGRAM EVALUATION•External Site Visit•PCE cyclical review

Annual Internal ReviewAnnual Internal Review Web-based Research ReportingWeb-based Research Reporting

– AchievementsAchievements– Future plansFuture plans

Reviewed by Office Leadership:Reviewed by Office Leadership:– Lab ChiefLab Chief– Division DirectorDivision Director– ADR/Office DirectorADR/Office Director

Rating Research Program SuccessRating Research Program Success AchievementsAchievements

– Return on research resources expendedReturn on research resources expended Direct impact of research on regulatory challengesDirect impact of research on regulatory challenges Quality: Peer reviewed publications & invited talksQuality: Peer reviewed publications & invited talks Contribution Guidances, policy, workshopsContribution Guidances, policy, workshops

– Impact on international harmonizationImpact on international harmonization– Review workload and qualityReview workload and quality

Future Research Plans-short & long termFuture Research Plans-short & long term– Unique CBER Expertise contributionUnique CBER Expertise contribution– Priority area & response to public health issuePriority area & response to public health issue– Direct impact on regulatory challengeDirect impact on regulatory challenge– Feasibility: Expertise, collaborations, infrastructureFeasibility: Expertise, collaborations, infrastructure– QualityQuality– Return on investmentReturn on investment

4 Year Cycle External Review:4 Year Cycle External Review:Scientific Expertise Site VisitsScientific Expertise Site Visits

Conducted by appropriate CBER’s Advisory Conducted by appropriate CBER’s Advisory Committee for each Product OfficeCommittee for each Product Office

Site Visit Team Chaired/Co-chaired by two Advisory Site Visit Team Chaired/Co-chaired by two Advisory Committee Members supplemented with appropriate Committee Members supplemented with appropriate outside Scientific Expertsoutside Scientific Experts

Held every four years for each Laboratory unit Held every four years for each Laboratory unit – Teams of Researcher-Reviewers & their Research Teams of Researcher-Reviewers & their Research

Programs within Product Office Programs within Product Office – Preparation and submission of Site Visit book by each Preparation and submission of Site Visit book by each

PI detailing achievements past four years and proposal PI detailing achievements past four years and proposal for future research next four yearsfor future research next four years

– Formal presentations to Site Visit TeamFormal presentations to Site Visit Team– Site Visit Team holds individual interviews with each PISite Visit Team holds individual interviews with each PI

Site Visit Report presented to and approved by full Site Visit Report presented to and approved by full Advisory Committee voteAdvisory Committee vote

Researcher-Reviewer and supervisors must respond Researcher-Reviewer and supervisors must respond to Site Visit Report comments and recommendations to Site Visit Report comments and recommendations within 6 monthswithin 6 months

4 Year Cycle Internal Review: Promotions 4 Year Cycle Internal Review: Promotions & Conversions Evaluation Committee& Conversions Evaluation Committee

Managed by Center ADR to provide Managed by Center ADR to provide recommendations to Center Director on personnel recommendations to Center Director on personnel actions & actions & cyclical reviewcyclical review assessments for all assessments for all Researcher-Reviewer StaffsResearcher-Reviewer Staffs

Formal SOP & procedures established in conjunction Formal SOP & procedures established in conjunction with Office of the Commissioner’s Peer Review with Office of the Commissioner’s Peer Review policypolicy

Chaired and staffed by Senior Researcher-Reviewers Chaired and staffed by Senior Researcher-Reviewers and Regulatory Scientists from each Product Officeand Regulatory Scientists from each Product Office

All Researcher-Reviewer staffs are evaluated every All Researcher-Reviewer staffs are evaluated every four years for cyclical progress review and for all four years for cyclical progress review and for all promotions or conversions to permanent staffpromotions or conversions to permanent staff

Formal regulatory workload & quality assessmentFormal regulatory workload & quality assessment Formal research productivity, mission relevance and Formal research productivity, mission relevance and

quality assessmentquality assessment

Stakeholder InputStakeholder Input

ADVISORYCOMMITTEE &

PUBLICINPUT

OFFICE SCIENTIFICPROGRAMANNUAL REPORT

OFFICE RESEARCHPLAN

REGULATORY ANDPUBLIC HEALTHPORTFOLIOANALYSIS & KEY SCIENTIFICCHALLENGES

RESEARCHPRIORITIES

Advisory Committee Office Research Advisory Committee Office Research Site Visit Reports 2005-6Site Visit Reports 2005-6

““Committee members who have been familiar with the Committee members who have been familiar with the research programs…over a period of years note that there research programs…over a period of years note that there has been a striking improvement over time…: has been a striking improvement over time…: – Focus and relevanceFocus and relevance……The research presented for The research presented for

this review had direct relevance to the Critical this review had direct relevance to the Critical Pathway of biologics product development and Pathway of biologics product development and availability. availability.

– QualityQuality. …. …the quality of the research has also the quality of the research has also improved;improved; many of the ongoing studies are equal in many of the ongoing studies are equal in quality to those in the intramural program at the quality to those in the intramural program at the National Institutes of Health (NIH) and of sufficient National Institutes of Health (NIH) and of sufficient caliber to compete for RO1 and other NIH grants.caliber to compete for RO1 and other NIH grants.

……strongly supports the FDA’s continued emphasis on the strongly supports the FDA’s continued emphasis on the importance of having a strong intramural research program importance of having a strong intramural research program to support its Critical Pathway program for effective and to support its Critical Pathway program for effective and efficient regulatory activities…efficient regulatory activities…

If we are to maintain our lead in health care development in If we are to maintain our lead in health care development in the USA, regulatory science needs to be given the priority it the USA, regulatory science needs to be given the priority it deserves, independent of the short-term political and deserves, independent of the short-term political and economic flurries that can derail progress.”economic flurries that can derail progress.”

Advisory Committee Office Research Advisory Committee Office Research Site Visit Reports 2005-6 IISite Visit Reports 2005-6 II

StrengthsStrengths– Productivity, scientific merit, mission Productivity, scientific merit, mission

relevantrelevant– Well recognized for outreach effortsWell recognized for outreach efforts– Complementary cross-Office expertiseComplementary cross-Office expertise– Success at recruitment and retentionSuccess at recruitment and retention– Core facilitiesCore facilities– Leveraging and collaboration (NIH, Leveraging and collaboration (NIH,

CDC, NTP, Academia)CDC, NTP, Academia)

Advisory Committee Office Research Advisory Committee Office Research Site Visit Reports 2005-6Site Visit Reports 2005-6

Concerns I:Concerns I: Increased regulatory workload and Increased regulatory workload and

decreasing supportdecreasing support Best mechanism: Balance managing Best mechanism: Balance managing

to mission relevance of research vs. to mission relevance of research vs. micromanagementmicromanagement

Covering many research bases vs. Covering many research bases vs. focus on quality in fewer areasfocus on quality in fewer areas

Development of an explicit Development of an explicit plan/strategic plan for 2-5 year plan/strategic plan for 2-5 year priority research needs with priority research needs with regulatory and stakeholder inputregulatory and stakeholder input

Advisory Committee Office Research Advisory Committee Office Research Site Visit Reports 2005-6Site Visit Reports 2005-6

Concerns II:Concerns II: MentoringMentoring Recruitment and retentionRecruitment and retention Increased research program visibilityIncreased research program visibility Continuing education supportContinuing education support Increased collaboration within and outside FDAIncreased collaboration within and outside FDA Increased FDA base funding support for Increased FDA base funding support for

researchresearch Creative leveraging support, e.g., FDA based Creative leveraging support, e.g., FDA based

Foundation for Research (like Jackson Foundation for Research (like Jackson Foundation for DOD)Foundation for DOD)

Need a public relations campaignNeed a public relations campaign Need a system of reward for successful Need a system of reward for successful

researchresearch

THANK YOUTHANK YOU