Research in the Office of Vaccines Research and Review: Vision and Overview Jesse Goodman, M.D., M.P.H. Director, Center for Biologics Evaluation and Research May 19, 2006

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

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)

CBER Strategic Priorities Pandemic influenza, emerging infectious disease and counterterrorism preparedness Enhance product safety through better prevention, detection and response including use of safety teams and modern informatics Bring safe and effective innovative products to patients efficiently Improve manufacturing and product quality using 21 st century technologies and preventive and risk based approaches

Critical Path Initiative Identify and focus on regulatory and scientific opportunities to improve product development and availability – consistent with CBER's policy 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

Unique Role of FDA Science 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 and policies to be science-based and to foster innovation and improve chances of efficient and successful development of safe and effective products Scientific expertise is critical to making informed decisions about proposed studies and about the safety and effectiveness of products before and after approval –Unique responsibilities and opportunities

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 2006

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 product innovation, pre-clinical and clinical studies, manufacturing, regulatory submissions, inspections, post-marketing 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.

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

Objectives of OVRR Research Program Review cont. Increase visibility and transparency –Communicate how research program is integrated into and with regulatory process –Communicate contributions of OVRR research to product development, quality and availability –Get your input identifying highest needs, where OVRR can best contribute and in prioritizing use of our limited resources Consider opportunities for redirection, leveraging, new collaborations Recommendations for attracting (and retaining) qualified experts, who excel in both science and review, for OVRR: who and how best to?

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 scientific expertise and collaboration can play a critical role in facilitating development of very promising innovative medical products with great potential to contribute to health

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