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FDA’s “Critical Path” Initiative

FDA’s “Critical Path” Initiative. Kathryn M. Carbone, M.D. Assoc. Director for Research, CBER, FDA www.fda.gov/oc/initiatives/criticalpath.htm. Tomorrow’s Medical Breakthroughs.

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FDA’s “Critical Path” Initiative

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  1. FDA’s “Critical Path” Initiative Kathryn M. Carbone, M.D. Assoc. Director for Research, CBER, FDA www.fda.gov/oc/initiatives/criticalpath.htm

  2. Tomorrow’s Medical Breakthroughs • Many serious diseases afflict our population and are waiting for better treatments: autism, addictive disorders, Alzheimer's disease, AIDS, bipolar disorders, cancer, cystic fibrosis, heart diseases, diabetes, morbid obesity, multiple sclerosis, muscular dsytrophy, rheumatoid arthritis, osteoarthritis, systemic lupus, schizophrenia, stroke, and many more

  3. Three Dimensions of the Critical Path • Assessment of Safety – how to predict if a potential product will be harmful? • Preclinical assays • Clinical trial design • Proof of Efficacy -- how to determine if a potential product will have medical benefit? • Biomarkers, e.g., Surrogate markers of efficacy • Industrialization – how to manufacture a product at commercial scale with consistently high quality? • Predict “scale up” problems early • Quality: Standards & Assays

  4. Proposal: FDA Proactively Improving the Product Development Process • Apply new science to improve predictability in product development, not just scientific discovery • Scientific knowledge and scientific tools to more efficiently assess candidate products

  5. Critical Path Research: Outcome of Improving Prediction • Reduce cost by focusing resources on the best candidate products early in development • More quantitative, less empiric, more predictable

  6. FDA & The Critical Path Initiative Bringing attention and focus to the need for targeted scientific efforts to modernize the techniques and methods used to evaluate the safety, efficacy and quality of medical products as they move from product selection and design to mass manufacture.

  7. Why FDA? Unique Role of the Agency vis-a-vis the Critical Path • FDA scientists are involved in review during product development--they see the successes, failures, and missed opportunities • FDA guidance documents are based on science and foster innovation and improve chances of success. • Convening and coordinating role for new biomarker and clinical method development

  8. ROLE OF FDA IN THE PROCESS

  9. Examples of Opportunities with CBER Products • Develop improved potency assays for specific allergens • Gain better understanding of allergen extract purity issues, e.g., role of natural immunomodulatory agents • Effects of viral infection on responses to common allergens • Develop and make available well characterized cell banks (and related methods to assay for safety/adventitious agents) useful for vaccine and other biologics production • Characterization of cell therapies & links to standardized outcomes (e.g. HPSCs) • Methods & validation of pathogen inactivation for blood, plasma, tissues and other products • Multipathogen and rapid detection methodologies for biologics including blood and tissue products • Improving longevity/storage of blood, blood products and tissues • Others as determined by working with stakeholders

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