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CBER Executive Staff Briefing

CBER Executive Staff Briefing. Status and Priorities October 26, 2010 Robert Yetter, PhD Associate Director for Review Management Center for Biologics Evaluation and Research Food and Drug Administration. CBER Our Vision. Innovative Technology Advancing Public Health

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CBER Executive Staff Briefing

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  1. CBER Executive Staff Briefing Status and Priorities October 26, 2010 Robert Yetter, PhD Associate Director for Review Management Center for Biologics Evaluation and ResearchFood and Drug Administration

  2. CBER Our Vision Innovative Technology Advancing Public Health • Protect and improve public and individual health in the US and globally • Facilitate development, approval, and access to safe and effective products and promising new technologies • Strengthen CBER as a pre-eminent regulatory organization for biologics

  3. CBER Our Mission To ensure the safety, purity, potency, and effectiveness of biological products, including vaccines, blood and blood products, and cells, tissues, and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions, or injury; and help to defend the public against the threats of emerging infectious diseases and bioterrorism

  4. CBER Organization OFFICE OF THE CENTER DIRECTOR Director: Karen Midthun, MDAssociate Director for Policy: Diane Maloney, JD Associate Director for Review Management: Robert Yetter, PhD Associate Director for Research: Carolyn Wilson, PhD Associate Director for Quality Assurance: Sheryl Lard-Whiteford, PhD Office of ManagementDirector: James SiggDeputy: Deirdre Hussey Office of Vaccines Researchand Review Director: Norman Baylor, PhDDeputy: Marion Gruber, PhD Office of Cellular, Tissueand Gene Therapies Director: Celia Witten, MD, PhDDeputy: Stephanie Simek, PhD Office of Biostatistics and EpidemiologyDirector: Robert Ball, MD, MPHDeputy: Steven Anderson, PhD Office of Communication, Outreach and DevelopmentDirector: Lorrie McNeillDeputy: Joanne Binkley Office of Blood Researchand Review Director: Jay Epstein, MDDeputy: Ginette Michaud, MD Office of Complianceand Biologics QualityDirector: Mary MalarkeyDeputy (Acting): Jalena Specht, JD Divisions Divisions Divisions Divisions Divisions Divisions Divisions Manufacturers Assistance Leslie Wheelock Disclosure and Ovresight Mgt Susan Frantz-Bohn Communication & Consumer Affairs Diane Bartell Blood Applications: Richard Davey Hematology: Basil Golding Emerging & Transfusion-Transmitted Dis.: Hira Nakhasi Case Management: Robert SausvilleManufacturing & Product Quality: John Eltermann Inspections & Surveillance: Gilliam Conley Epidemiology: Rickey Wilson Biostatistics: Henry Hsu Cell & Gene Therapy: Raj Puri Clin. Eval. & Pharm./Tox.: Wilson Bryan/Mercedes Serabian Human Tissues: Ellen Lazarus Bacterial, Parasitic & Allergenic Products: Milan Blake Viral Products:Jerry Weir Vaccines & Related Product Applications:Wellington Sun Product Quality: William McCormick Planning, Eval & Budget Karen O’Brien Program Services David Leffler Scientific Advisors & Consultants William Freas Veterinary Services Philip Snoy

  5. Our Products • Blood Derivatives • Whole Blood • Blood Components • Selected Devices • Human Tissues • Vaccines (preventive and therapeutic) • AllergenicProducts • Cell & Gene Therapies • Xenotransplantation products

  6. CBER Strategic Priorities • Enhance the nation’s public health preparedness • Protect and promote public health globally • Keep patients and products safe • Improve the quality and safety of biological products • Bring innovative products to patients • Strengthen our human resources and performance

  7. CBER’s 2010 Actions to Address Key Agency Initiatives Safety Transparency National and Global Public Health Regulatory Science Enhanced Regulatory Processes

  8. Focus on 2011Priorities:Safety is Key

  9. CBER’s Integrated Safety Teams:Pooling our Expertise • 3 Product-based teams • Tissue Safety Team • Blood Safety Team • Vaccine Safety Team • With multi-disciplinary collaborations within CBER • Product office (blood, tissue or vaccines) • Office of Biostatistics & Epidemiology • Office of Compliance & Biologics Quality • Office of Communication, Outreach & Development • Office of the Center Director

  10. CBER Safety Teams (2) • Multi-disciplinary teams (epidemiologists, clinical/product reviewers, compliance/manufacturing experts, communications) to improve acquisition, analysis, and communication of safety information • Encompass entire product life cycle • Use data - evaluate emerging safety issues • Coordinate FDA response to issues with others • Enhance collaboration with other gov’ts., WHO, etc. • Proactive: contribute to research, policy, outreach

  11. FDAAA Safety Activities • Center Activities: • Cross-Office Center group • Part of Cross-Center Agency group • Focus on: • Safety Labeling Changes • REMS Considerations • Post-marketing Requirements

  12. CBER Post-Marketing Safety Collaborations • Vaccine Adverse Event Reporting System (with CDC) • Centers for Medicaid & Medicare Services (CMS): 45 million enrollees • Department of Defense • Vaccine Safety Database (managed by CDC) • Department of Veterans Affairs • Indian Health Service • Post-Licensure Rapid Immunization Safety Monitoring (linking state immunization registries with health care database medical records) • International collaborations (e.g., WHO and many regulatory and public health agencies worldwide)

  13. 2011 Priorities:Transparency*Overarching Agency Initiative to Enhance Communications in All Areas*CBER Continues to Facilitate the Development of Safe and Effective Biological Products

  14. Communication to Facilitate Product Development • Guidance • Workshops • Advisory Committees

  15. RECENT CBER GUIDANCESPathways for DevelopingInnovative Cell & Gene Therapy Products • Final: • Minimally manipulated, unrelated allogeneic placental/ umbilical cord blood intended for hematopoietic reconstitution for specified indications (10/09) • How to pursue licensure • Considerations for Allogeneic Pancreatic Islet Cell Products(9/09) • Draft: • Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications (10/09) • Patient access to life-saving products • Clinical Considerations for Therapeutic Cancer Vaccines (9/09) • Somatic Cell Therapy for Cardiac Disease (3/09)

  16. Workshops on Cellular Products • Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations -upcoming Nov 2, 2010 • Cord Blood Licensure: A Workshop – March 8-9, 2010 • FDA/NCI Workshop: Therapeutic Cancer Vaccines; Considerations for Early Phase Clinical Trials Based on Lessons Learned from Phase III - October 27, 2009

  17. Blood Safety Guidances Addressing Agents of Emerging Infectious Diseases • Guidance for Industry: Nucleic Acid Testing (NAT) for Human Immunodeficiency Virus Type 1 (HIV-1) and Hepatitis C Virus (HCV): Testing, Product Disposition, and Donor Deferral and Reentry(May 2010) • Guidance for Industry: Revised Preventive Measures to Reduce the Possible Risk of Transmission of Creutzfeldt-Jakob Disease (CJD) and Variant Creutzfeldt-Jakob Disease (vCJD) by Blood and Blood Products (May 2010) • Draft Guidance for Industry:Recommendations for the Assessment of Blood Donor Suitability, Blood Product Safety, & Preservation of the Blood Supply in Response to Pandemic (H1N1) 2009 Virus(11/13/2009) • Guidance for Industry:Use of Nucleic Acid Tests to Reduce the Risk of Transmission of West Nile Virus from Donors of Whole Blood and Blood Components Intended for Transfusion(11/6/2009)

  18. Blood and Tissue Safety Workshops Addressing Emerging Infectious Diseases & Blood Safety Public Workshops: • Quantitative Risk Assessment: Blood Safety and Availability Public Workshop -May 13, 2010 • Emerging Infectious Diseases: Evaluation to Implementation for Transfusion and Transplantation Safety –May 11 -12, 2010 • Emerging Arboviruses: Evaluating the Threat to Transfusion and Transplantation Safety – December 14-15, 2009

  19. Vaccines: Guidance/ Advisory Committee Meetings • Public Meeting on Animal Models—Essential Elements to Address Efficacy Under the Animal Rule; and Reopening of Comment Period (Docket No. FDA–2009–D–0007)-upcoming November 5, 2010 • Guidance for Industry:Characterization and Qualification of Cell Substrates and Other Biological Materials Used in the Production of Viral Vaccines for Infectious Disease Indications-March 2010 • Vaccines and Related Biological Products Advisory Committee (VRBPAC)-May 7, 2010 : • majority of the advisory committee members expressed support for the continued use of Rotarix and RotaTeq and stated that the vaccine’s benefits far outweigh the theoretical risks posed by PCV.

  20. 2011 Priorities:Global Collaboration to Improve Public Health • Emerging infectious diseases • Worldwide collaboration on emerging pubic health threats • Countermeasures against bioterrorism • Worldwide collaboration to address product quality, safety, and availability

  21. Challenge: H1N1 Pandemic • H1N1 response central to CBER’s international activities • CBER agreed to be Reference NRA for WHO vaccine prequalification for multiple H1N1 vaccines • CBER provides technical assistance to developing countries via WHO • FDA continues to work with international authorities so that data from other countries can inform US decision-making

  22. CBER’s WHO Engagements • CBER serves as reference NRA [National Regulatory Authority] for a number of WHO prequalified vaccines • Collaborating Center (CC) for Biological Standardization since 1998 • Large Spectrum of Activities as CC, e.g.: • Expert Committee on Biological Standards • Global Advisory Committee on Vaccine Safety • Advisory Committee on HIV Vaccines • Blood Regulators Network • PAHO Training on post-marketing surveillance • Work with WHO developing countries’ networks

  23. Regulatory Dialogue • Confidentiality arrangements with a number of regulatory agencies • European Medicines Agency (EMA) • Product “clusters” reflecting routine, on-going dialogue; newest cluster = Blood Products (Feb 2010) • Parallel scientific advice • Staff exchanges in areas of vaccines and cell, tissue & gene therapies • Health Canada • Sharing regulatory information on ad hoc basis • Mutual learning on training of staff

  24. Harmonization Efforts • ICH (USA, Europe, Japan) 2009/10 highlights • 1st ICH guideline on gene therapy taken up • Progress on interface between ICH and Standards Development Organizations • Expansion of Global Cooperation Group to additional individual countries • Global Harmonization Task Force (GHTF) • Efforts to harmonize medical device regulatory requirements & terminology • Asia-Pacific Economic Cooperation • Life Sciences Innovation Forum, regulatory harmonization efforts launched in 2009 • Pharmaceutical Inspection Cooperation Scheme

  25. Regulatory Science2011 Priorities:Using Science and Regulation to Advance Product Developmentand Improve Product Safety

  26. The CBER Model • CBER research contributes to regulatory policy, risk assessments, new methods & standards, and changes to product labeling • CBER researchers are fully integrated into review process • Together with others contribute to policy documents • Meet with sponsors and advisory committees • Evaluate adverse drug reactions and do risk assessment

  27. Influenza Program: CBER’s Role • Developsnew high-growth influenza virus reference strains for vaccines • Evaluatesmanufacturers’ influenza seed viruses before vaccine production • Preparesinfluenza virus reagents to determine potency of influenza vaccines • Expanded post-market ongoing safety assessment Photo: Wikimedia Commons

  28. Examples:CBER Research–BasedActivities to Facilitate Product Development • Rapid influenza hemagglutinin (HA) antibody reagent produced by recombinant techniques(no need to grow whole virus, then purify HA to assess vaccine potency) • Expedited influenza vaccine reagent calibration processand development of back-up procedures to ensure availability • New technologies to determine vaccine potency without using antibodies – for influenza vaccines (e.g., cell culture-derived, adjuvanted, DNA, recombinant protein-based, virus-like particles)

  29. Meeting Challenges to Advance and Enhance Regulatory Processes • Some Challenges: new & on-going • New laws/regulations (e.g., FDAAA, PREA, Patient Protection and Affordable Care Act • Biologics Price Competition and Innovation Act of 2009-“Biosimiliars”Sections 7001-7003 of the Patient Protection and Affordable Care Act (Public Law No. 111-148)  • PDUFA V- Reauthorization; Legislative authority expires Sept 2012 • Adoption/implementation of new standards (e.g., SPL, CDISC/SDTM and other computer standards) • Regulatory processes for innovative new products (especially for treatment of serious and/or rare conditions)

  30. Meeting Challenges to Advance and Enhance Regulatory Processes • Some Responses • Workgroups to address new laws, regulations • Enhanced reviewer training • Public Workshops and Guidance development • Approval Pathway for Biosimilar and Interchangeable Biological Products Public Meeting (upcoming Nov 2-3, 2010) • http://www.fda.gov/Drugs/NewsEvents/ucm221688.htm • Safety Reporting Rule (9/28/10) Final Rule: Investigational New Drug Safety Reporting Requirements for Human Drug and Biological Products and Safety Reporting Requirements for Bioavailability and Bioequivalance Studies in Humans • Standards development

  31. A Few Notable 2010 CBER Approvals • Menveo (meningococcal conjugate vaccine) to prevent invasive meningococcal disease • Prevnar 13 vaccine against more pneumococcal typesa broadened vaccine to prevent invasive pneumococcal disease in infants and young children • Prism automated blood screening antibody assay for automated detection of Chagas Disease • Provenge - autologous cellular immunotherapy for treatment of hormone refractory prostate cancer

  32. Acknowledgments • Karen Midthun • David Cho • Joan Wilmarth Blair • Mary Pat Leary

  33. Thank You for Your Attention – Questions?

  34. Resources/Contact • Consumer Questions about Products - Please direct all consumer questions about biological products to ocod@fda.hhs.gov • Manufacturers Assistance - Please direct all manufacturers' assistance questions to matt@fda.hhs.gov

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