Research in the Office of Cellular, Tissue and Gene Therapies: Vision and Overview

1. Jesse Goodman, M.D., M.P.H. Director, Center for Biologics Evaluation and Research September 29, 2005 Research in the Office of Cellular, Tissue and Gene Therapies: Vision and Overview

2. Vision for CBER INNOVATIVE TECHNOLOGY ADVANCING PUBLIC HEALTH • Protect and improve public and individual health in the US and, where feasible, globally • Facilitate the development, approval and access to safe and effective products and promising new technologies • Strengthen CBER as a preeminent regulatory organization for biologics

3. Products Regulated by CBER • Blood, blood components and derivatives • Vaccines (preventive and therapeutic) • Allergenics • Cell and Gene Therapies • Tissues • Xenotransplantation • Related Devices (including certain IVDs)

4. Critical Path Initiative • Identify and focus on regulatory and scientific opportunities to improve product development and availability • Opportunity to promote and preserve a science-based CBER and FDA • Seeking input to identify opportunities, collaborators, and priorities • October 2004 workshop • Site visits with advisory committees

5. Unique Role of FDA in Critical Path • FDA scientists are involved in review during product development and see the successes, failures, cross-cutting challenges and missed opportunities • We strive for FDA guidance documents to be science-based and to foster innovation and improve chances of efficient and successful development of safe and effective products • Scientific expertise in certain areas is critical to making informed decisions about proposed studies and about the safety and effectiveness of products before and after approval

6. CBER Research Working Group and Leadership Retreat • Consistent with the Critical Path Initiative, CBER RWG developed ideas and principles to strengthen and support CBER science and its alignment and management to our mission • CBER research leadership (including Office Associate Director’s for Research (ADRs) & Directors) held a retreat in September to discuss the input & define next steps • Agreement was reached on: • Guiding Principles for Offices and the Centers • Creation of a CBER Research Leadership Council to Coordinate and Develop Implementing the Principles • Priorities for accomplishment in the next 12 months

7. Draft Guiding Principles • The CBER research program is highly collaborative and includes laboratory, epidemiological, statistical and clinical sciences. • Its scope will encompass the scientific basis of pre-clinical and clinical studies, manufacturing, regulatory submissions, inspections, postmarketing surveillance and guidances. • The research will be high quality, efficient, and directed and managed to provide outcomes that address scientific and regulatory challenges in product development, safety, effectiveness and quality.

8. Implementation • The ADRs in each relevant office will be empowered to serve as the Office Directors’ lead representative in research planning and management for each office, which will remain the locus of management • The ADRs will form a new Research Leadership Council, also including the Center ADR, OCBQ, Core Facility and Vet Services

9. Research Leadership Council • In next 12 months: Propose to Center leadership and facilitate implementing consistent processes for research management including: • Stakeholder input • Prioritization and goal setting • Tools for regulatory workload assessment and evaluation of research • Develop Cross-cutting Research Priorities and Communication strategies, and Center-wide matrix programs where appropriate • Work w/ Center Director, ADR, Office Directors and their leadership teams, including Division Directors, to support the research enterprise

10. Goals of Today’s OCTGT Research Program Review • Assess strengths, weaknesses, opportunities, and needs of the OCTGT research program as a whole • Assess preparedness for present and future scientific challenges • Make recommendations that help us further dynamic and responsive research programs intended to facilitate development of safe and effective biological products

11. Objectives of OCTGT Research Program Review cont. • Identify needs and strategies for current and future biologics research • Increase visibility and transparency • Communicate how research program is integrated into regulatory process • Communicate contributions of OCTGT research to product development and availability • Get your input identifying needs and prioritizing use of our limited resources • Consider opportunities for research expansion, redirection, new collaborations • Recommendations for attracting qualified experts to OCTGT: who and how best to?

12. CBER: Innovative Technology Advancing Public Health • CBER has responsibility for some of the most important medical products with major public health and preparedness impacts: blood, vaccines, stem cells and tissues: safety and availability issues • FDA expertise and collaboration can play a critical role in facilitating development of very promising innovative medical products (e.g. cellular & gene therapies, cell and xeno transplants) with great potential to contribute to health

13. Thank you, again! • Expertise, partnership, and wise use of resources are essential • We appreciate and welcome your input! • CBER: INNOVATIVE TECHNOLOGY ADVANCING PUBLIC HEALTH