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FDA’s Critical Path Research Initiative & Intro to the CBER Research Program

FDA’s Critical Path Research Initiative & Intro to the CBER Research Program. Kathryn M. Carbone, M.D. Associate Director for Research CBER/FDA. Despite basic biomedical research investment going up. Attending to the Critical Path for medical product development. BLA. IND.

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FDA’s Critical Path Research Initiative & Intro to the CBER Research Program

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  1. FDA’s Critical Path Research Initiative & Intro to the CBER Research Program Kathryn M. Carbone, M.D. Associate Director for Research CBER/FDA

  2. Despite basic biomedical research investment going up....

  3. Attending to the Critical Path for medical product development BLA IND

  4. FDA Critical Path Research Initiativewww.fda.gov/oc/initiatives/criticalpath.htm • Identify, focus upon and manage to regulatory & scientific opportunities to improve product development process and availability • Potency/effectiveness/standards • Safety • Consistency/manufacturing/quality • Needed policy and guidance • Preserve a science based FDA

  5. Why FDA? • Unique perspective of the Agency vis-a-vis leads to a valuable role in convening and coordinating Critical Path Research • Combination of FDA intramural, FDA intramural/extramural collaborations, and extramural research efforts

  6. Integral Role of Research to Inform Policy of Product Evaluation

  7. Subcommittee for the External Review of CBER Research, 2/98 • “The Researcher/Reviewer Model is essential to providing CBER with top-level expertise in a regulatory culture.” • Working closely with CBER Regulatory Scientists and Clinical Review Scientists to perform high quality evaluation of novel biological products

  8. Multitasking at the FDA: Research Supports Regulatory Mission • Research Programs organized by Product Offices • CBER researchers are fully integrated into the regulatory process (~50% average time) = “Researcher-Regulator” model • Review INDs and BLAs • Development of Policy and Guidance Documents • Meeting with Sponsors and Advisory Committees • Participation in Pre-license and Biennial Inspections • Evaluation of Adverse Drug Reactions and Risk Assessment • Performing research relevant to product evaluation of safety, efficacy, manufacturing: Developing/evaluating scientific tools & knowledge

  9. Mission Relevanceof Research Programs • Hundreds of Biologics Licensing Applications and Investigational New Drug Applications directly supported by research programs • More than 50% of the Research Programs have applicability to evaluation of Counterterrorism-relevant biological products • CBER research in the public domain supports development of safe and effective biologics across entire product classes

  10. Managing Research Programs at CBER • Evaluation of past achievements and future plans • External Laboratory/Res-Reg Site Visits: Four year cycle • Internal Management reviews: Yearly cycle using Annual Research Program reporting: E.g., Publications, Regulatory Policy/Guidances, Invited talks, Research QA/QC • Office Research Site Visit to be conducted in FY05 & ‘06 • Developing cross-Office Coordinated Research Expertise Teams • CBER researchers provided with intramural support • Must compete for select sources of extramural funding to support research programs

  11. CBER Research: Supporting Innovation • WNV blood donor screening advances: enhanced IND NAT testing = 1000+ units detected • New tests and standards for biologic products: HIV, hepatitis, blood typing, blood cross-matching,, IGIV immune globulin, a-1 proteinase, thrombin, WNV • New safety evaluations: HBOC oxidative toxicity; prion detection and removal

  12. Examples of CBER Critical Path Investment Opportunities • Develop/make available well characterized cell banks (and methods to assay for safety/adventitious agents) for vaccine and biologics production – & update guidance • Characterization of cell therapies & links to standardized clinical/lab outcomes (e.g. HPSCs) • New assays, standards, biomarkers, surrogates for complex biologics safety, efficacy and quality • Methods & validation of pathogen inactivation for blood, plasma, tissues and other products • Multipathogen and rapid detection methodologies • Improving longevity/storage of blood and tissues • Flu vaccine assays, standards and reagents • Enhanced clinical trial design/analysis

  13. CBER: INNOVATIVE TECHNOLOGY ADVANCING PUBLIC HEALTH • We are proud of our staff and our role in public health, biodefense, product safety and availability. • New technologies need innovative and interactive regulation, new models, standards and assays. • Expertise and partnerships essential. • We welcome your input. Contact me: carbonek@cber.fda.gov or 301-827-0372

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