Critical Path Opportunities for Biologics Products Jesse L. Goodman, M.D. M.P.H. Director Kathryn M. Carbone, M.D. Associate Director for Research Center.

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Presentation transcript:

Critical Path Opportunities for Biologics Products Jesse L. Goodman, M.D. M.P.H. Director Kathryn M. Carbone, M.D. Associate Director for Research Center for Biologics Evaluation and Research

Innovations in the Science of Biologics Product Development Get more innovative biological products to patients Achieve robust biological product development pathways that are efficient and predictable. – –Many promising biologics products have no established development pathways, e.g., gene and cell therapies—a true opportunity How? – –Communicate and work closely with stakeholders – –Develop and evaluate new scientific toolkits that bring scientific advances into the product development process.

CBER Proactively Improving the Product Development Process Working with stakeholders to develop and prioritize needs for scientific tools and knowledge for product development CBER staff coordinating and collaborating with academic and industry scientists to respond to these needs – –Communicating with stakeholders, e.g., workshops & guidances Applying new science to chart a more predictable and efficient path for new biologics product evaluation Assessing progress and revising priorities in collaboration with stakeholders on a routine basis

Why CBER? Unique Role of the Center vis-a-vis Science of Biologics Product Evaluation CBER guidance based on science can foster innovation and improve chances of success of entire field of biological products CBER scientists are part of the review process during product development—they directly see the successes, failures, and missed opportunities due to lack of science CBER can play both a direct role as well as a convening and coordinating role for scientific needs across sponsors

CBER Critical Path Grand Opportunities New vaccine delivery systems, rapid use vectors, adjuvants, including vaccines for BT, EID New vaccine delivery systems, rapid use vectors, adjuvants, including vaccines for BT, EID Develop and make available well characterized cell banks (and related methods to assay for safety/adventitious agents) useful for vaccine and other biologics production Develop and make available well characterized cell banks (and related methods to assay for safety/adventitious agents) useful for vaccine and other biologics production Characterize cell therapies & link this information to standardized outcomes (e.g. stem cells) Characterize cell therapies & link this information to standardized outcomes (e.g. stem cells) Development and evaluation of methods for: Development and evaluation of methods for: –Multipathogen, rapid detection of microbial contamination of biologics including blood and tissue products –Pathogen inactivation for blood, plasma, tissues and other products Improving longevity/storage of blood and tissues Improving longevity/storage of blood and tissues

CBER Workshop CBER recently held an open, public workshop to discuss scientific opportunities to support evaluations of biologics product efficacy and safety CBER recently held an open, public workshop to discuss scientific opportunities to support evaluations of biologics product efficacy and safety Morning presentation by CBER Offices of Vaccines, Blood, Cell-Tissue-Gene Therapy and Compliance & Biologics Quality Morning presentation by CBER Offices of Vaccines, Blood, Cell-Tissue-Gene Therapy and Compliance & Biologics Quality Afternoon Breakout Discussions with members of academia, industry, patient advocacy groups and non-FDA government scientists and attendees Afternoon Breakout Discussions with members of academia, industry, patient advocacy groups and non-FDA government scientists and attendees Summaries of Breakout Sessions completed and manuscripts are being prepared for publication Summaries of Breakout Sessions completed and manuscripts are being prepared for publication

Critical Path: Improving the Predictability of Clinical Studies Study design & statistical analysis Study design & statistical analysis –Missing data, data mining, standardized outcomes, data bridging (animal-human; pediatric-adult), Risk Mgmt, multiple endpoints Preclinical safety assays Preclinical safety assays –Gene/Cell »Predicting cancer risks »Animal models: Adenovirus-associated respiratory distress model in liver disease (rat), Bioterrorism vaccines –Vaccine »Neurotoxicity models: Smallpox vaccine, Influenza vaccine, Polio vaccine, Mumps vaccine; HIV, WNV, SARS

Critical Path: Improving the Predictability of Clinical Studies Biomarkers & surrogate endpoints for efficacy Biomarkers & surrogate endpoints for efficacy –Vaccines for Bioterrorism, EID, Hep C, HIV »Animal models (Smallpox, anthrax, ebola) »Immune surrogates: Rotavirus, HIV, pneumococcus »Assays to distinguish immune response from natural, persistent infection vs. vaccination (HIV) –Gene and cell therapies »Noninvasive imaging techniques (w/CDRH) Correlation of product characteristics with clinical outcomes & subject biomarkers: Stem cells, vaccines Correlation of product characteristics with clinical outcomes & subject biomarkers: Stem cells, vaccines Postmarketing surveillance: Particularly important when surrogate endpoints are used Postmarketing surveillance: Particularly important when surrogate endpoints are used

Manufacturing: Better techniques and resources for product quality Product microbial assays Product microbial assays »TSE, Microarray for Mycoplasma, Spiroplasma, Ureaplasma, Xenotransplantation: PERV, bacteria in blood products Potency assays: Faster, easier, more quantitative Potency assays: Faster, easier, more quantitative –Smallpox vaccine/VIG assay, blood product assay Cell substrates: Developing the assays and qualified cell banks Cell substrates: Developing the assays and qualified cell banks –Along with assays for tumorigenicity, contamination with infectious agents, including TSE References, Standards, Statistical Approaches References, Standards, Statistical Approaches –Influenza vaccine, standard and pandemic –Adenovirus & Retrovirus gene vectors

Continuing the collaborative process of Critical Path discovery and application Continue open discussions of Biologics CP issues – –To develop CBER science future priorities and agenda – –Seek outside scientists for consultation, collaborative projects or to inspire for independent projects Transparency and accountability: – –CBER science organization, priorities and outcomes to be presented and reviewed in public Advisory Committee meetings in 2005 – –Continue regular formal external reviews of CBER science programs to seek evaluation of quality and to update priorities Critical Path Scientific advances to be communicated in guidances, policy and publications to support product development