Center for Biologics Evaluation and Research (CBER) PPT
- 1. 1 CBER Center for Biologics Evaluation and Research Dr. Dasari Vasavi Devi, Associate Professor, Department of Pharmaceutical Analysis, Annamacharya College of Pharmacy, Rajampet.
- 2. 2 CONTENTS Organizations of USFDA Introduction History Current Role Vision Mission Responsibilities of CBER Key Functions Divisions of CBER Achievements CBER Regulates complex Biological Products CBER Research Goals CBER- BAA Priorities References
- 3. 3 Department of Health and Human Services US Food and Drug Administration (FDA) CDER(Center for Drug Evaluation and Research ) CBER(Center for Biologics Evaluation and Research) CDRH (Center for Devices and Radiological Health) CFSAN(Center for Food Safety and Applied Nutrition) CVM (Center for Veterinary Medicine) National Center for Toxicological Research Office of the Commissioner Office of Regulatory Affairs
- 4. 4 The Center for Biologics Evaluation and Research (CBER) is one Center within the Food and Drug Administration, an Agency within the United States Government's Department of Health and Human Services (HHS).
- 5. 5 CBER is to protect and enhance the public health through the regulation of biological and related products including blood, vaccines, allergenics, tissues, and cellular and gene therapies. Biologics, in contrast to drugs that are chemically synthesized, are derived from living sources (such as humans, animals, and microorganisms), are not easily identified or characterized, and many are manufactured using biotechnology. These products often represent cutting-edge biomedical research and, in time, may offer the most effective means to treat a variety of medical illnesses and conditions that presently have few or no other treatment options.
- 6. 6 CBER's review of new biological products, and for new indications for already approved products, requires evaluating scientific and clinical data submitted by manufacturers to determine whether the product meets CBER's standards for approval. After a thorough assessment of the data, CBER makes a decision based on the risk-benefit for the intended population and the product's intended use. CBER's legal authority resides in the Public Health Service Act and in specific sections of the Federal Food, Drug and Cosmetic Act. CBER advances the public health.
- 7. 7 Although medical products are required to be safe, safety does not mean zero risk, since all medical products are associated with some level of risk. A safe biological product is one that has reasonable risks, given the patient's condition, the magnitude of the benefit expected, and the alternatives available. The choice to use a biological product involves balancing the benefits to be gained with the potential risks. CBER is committed to a product approval process that maximizes the benefits and minimizes the risks to patients of the biological product.
- 8. 8 CBER is committed to providing up-to-date information to the public, healthcare professionals, the media and product manufacturers about the products CBER regulates through the Biologics Web pages. Site content is developed and reviewed by CBER staff prior to publication. The Biologics License Application (BLA) is a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce (21 CFR 601.2) in US.
- 9. 9 BIOLOGICAL PRODUCTS Derived from human, animals, plants and microorganism sources. This includes blood and blood components, tissues allergenic extracts, vaccines, drugs derived using biotechnology and certain diagnostic products.
- 10. 10 HISTORY Pre-FDA Era: Early 20th Century 1. 1902 - The Biologics Control Act: This act marked the first federal regulation of biologics in the United States. It was passed following a public health tragedy in which contaminated diphtheria antitoxin caused multiple deaths. The Hygienic Laboratory of the U.S. Public Health Service was designated as the oversight body for biologics production, laying the groundwork for future biological regulation. 2. 1930 - Establishment of the FDA: The Food, Drug, and Insecticide Administration was renamed the FDA. Biologics regulation continued under the Public Health Service but in coordination with the FDA. Early CBER Formation 3. 1972 - Transfer of Biologics Regulation to the FDA: The responsibility for regulating biologics moved from the National Institutes of Health (NIH) to the FDA. This transfer included vaccines, blood products, and other biologics. The Division of Biologics Standards became part of the FDA as the Bureau of Biologics. 4. 1982 - Formation of the National Center for Drugs and Biologics: This center consolidated the Bureau of Drugs and the Bureau of Biologics, integrating biologics and drug regulation. 5. 1988 - Creation of CBER: The Center for Biologics Evaluation and Research (CBER) was officially formed as a separate entity within the FDA, focusing solely on biologics.
- 11. 11 Key Milestones in CBER History 6. 1990s - Focus on Emerging Biotechnologies: CBER expanded its regulatory oversight to include novel biotechnologies, such as recombinant DNA products and gene therapies. 7. 1997 - Food and Drug Modernization Act: This act streamlined regulatory pathways, enhancing CBER's ability to review and approve new biologics efficiently. 8. 2003 - Separation of Therapeutics and Devices: Regulation of therapeutic biologics, such as monoclonal antibodies, was transferred to the FDA's Center for Drug Evaluation and Research (CDER). CBER continued to oversee vaccines, blood products, and gene/cellular therapies. 9. 2009 - Response to the H1N1 Pandemic: CBER played a critical role in reviewing and approving H1N1 influenza vaccines, demonstrating its capacity for rapid response to public health crises. Modern Era 10. Recent Innovations: CBER has been at the forefront of regulating advanced therapies such as: CAR-T cell therapies for cancer. Gene editing products like CRISPR-based therapies. COVID-19 vaccines and therapies during the pandemic. 11. Commitment to Safety and Innovation: CBER continues to ensure the safety, efficacy, and availability of biologics, collaborating with international regulatory agencies to harmonize standards.
- 12. 12 Current Role CBER oversees a wide range of biologics and supports the advancement of scientific innovation in areas such as: Personalized medicine. Advanced biotechnologies. Public health preparedness. CBER’s evolution reflects its vital role in safeguarding public health while adapting to advancements in medical science and biotechnology.
- 13. 13 VISION The Center for Biologics Evaluation and Research (CBER) uses sound science and regulatory expertise to: Protect and improve public and individual health in the United States and, where feasible, globally; Facilitate the development, approval of, and access to safe and effective products and promising new technologies; and Strengthen CBER as a preeminent regulatory organization for biologics.
- 14. 14 MISSION The mission of the Center for Biologics Evaluation and Research (CBER) is to ensure the safety, purity, potency, and effectiveness of biological products including vaccines, allergenics, blood and blood products, and cells, tissues, and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions, or injury. Through our mission, we also seek to protect the public against the threats of emerging infectious diseases and bioterrorism. Protect and promote public health. Facilitate the development of safe and innovative biological products. Ensure compliance with federal regulations for manufacturing and distribution.
- 15. 15 RESPONSIBILITIES OF CBER CBER regulates a wide variety of biological products, including: Vaccines: For infectious diseases (e.g., influenza, COVID-19). Blood and Blood Products: Ensures safety and availability of blood, plasma, and blood derivatives. Cellular and Gene Therapies: Monitors cutting-edge therapies like CAR-T cells and gene-editing treatments. Tissues and Tissue Products: Ensures safety in transplantation of human tissues. Allergenic Products: Regulates products like allergy shots or diagnostic tests for allergies. Xenotransplantation Products: Oversees the use of animal cells, tissues, or organs in human transplantation. Monoclonal antibodies and other therapeutic proteins are regulated by the FDA Center for Biologics Evaluation and Research (CBER).
- 16. 16 CBER REGULATING THE BIOLOGICAL PRODUCTS Activities includes: Monitoring the pre-clinical and clinical testing of new biological products, and evaluating their safety and effectiveness before marketing. Licensing biological products and manufacturing establishments, including blood banks. Research on AIDS medications, diagnostic tests, and vaccines. Compliance monitoring, lot releasing, and post market surveillance.
- 17. 17 APPROVAL CBER staff reviews clinical research and laboratory testing data to determine if the biologic is safe and effective for its intended use. In order for a biological product to be approved for marketing in the U.S., an applicant must submit a Biologics License Application (BLA).
- 18. 18 BLA Animal studies and human clinical trails performed. How the biologic is manufactured, processed and packaged, including information on the quality control methods used during its manufacture. Labeling that will be used with the product. Once a biological product is approved, its identity and manufacturing process cannot change without prior FDA approval.
- 19. 19 BLOOD SUPPLY Assuring the safety of, and the public confidence in, the nation’s blood supply is one of CBERs main priorities. There are five overlapping safe guards in place to help protect the safety of blood. Quarantine of untested blood Donor screening. Donor deferral registries. Blood testing Investigations of problems
- 20. 20 Some of the products regulated by CBER are devices. These include products used in the collection and processing of blood products, such as blood bags, centrifuges, and test kits that are used to screen donated blood for infectious diseases such as HIV and hepatitis.
- 21. 21 AUTHORITY: Resides in section 351 and 361 of the Public Health Service Act. Section 351: Licensing the biological products that travel in the interstate commerce of the United states. Deny or suspend or cancel any current license if the manufacturer does not comply with the requirements. Section 361: Make and enforce the regulations to control the interstate speed of communicable diseases.
- 22. 22 KEY FUNCTIONS Regulatory Oversight: Reviews applications for biological product approval, including Biologics License Applications (BLAs). Monitors post-market safety and efficacy of biologics through pharmacovigilance programs. Scientific Research: Conducts research to develop new testing methods and improve the understanding of biologics. Supports innovation in biological sciences. Public Health Preparedness: Works on emergency responses, such as vaccine approval during pandemics like COVID-19 or H1N1 influenza. Inspections and Compliance: Inspects manufacturing facilities to ensure adherence to Good Manufacturing Practices (GMPs). Ensures that biologics meet rigorous safety and quality standards.
- 23. 23 DIVISIONS OF CBER CBER operates through several offices, each with specialized functions: Office of Blood Research and Review (OBRR): Focuses on blood products, plasma, and related safety issues. Office of Vaccines Research and Review (OVRR): Handles the regulation of vaccines for diseases like measles, influenza, and COVID-19. Office of Cellular, Tissue, and Gene Therapies (OCTGT): Oversees the rapidly growing field of regenerative medicine and gene editing. Office of Compliance and Biologics Quality (OCBQ): Ensures compliance with regulatory standards and quality assurance. Office of Biostatistics and Epidemiology (OBE): Conducts statistical evaluations and epidemiological studies to assess product safety and effectiveness.
- 24. 24 ACHIEVEMENTS COVID-19 Response: Led the evaluation and approval of vaccines (e.g., mRNA vaccines from Pfizer-BioNTech and Moderna) during the pandemic. Advanced Therapies: Approved pioneering therapies like CAR-T cell therapy for cancer treatment and gene therapy for rare genetic disorders. Blood Safety: Implemented strategies to reduce the risk of transfusion- transmissible infections. CBER Reports are a summary of regulatory, research, and administrative accomplishments for the last 2 fiscal years and related reports.
- 25. 25 CBER REGULATES COMPLEX BIOLOGICAL PRODUCTS
- 26. 26 CBER RESEARCH GOALS •Develop and evaluate technology and tools to support non-clinical evaluation of medical products. Priority 1 •Enhance the validity and efficiency of clinical evaluation through use of innovative statistical, analytical, and modeling approaches. Priority 2 •Proactively address public health challenges and emerging infectious diseases. Priority 3 •Advance scientific capabilities to assess novel technologies and innovative medical products to inform regulatory oversight. Priority 4 Advancing the scientific basis for regulation of biologics, human tissues and blood by:
- 27. 27 CBER - BAA(BROAD AGENCY ANNOUNCEMENT) PRIORITIES I. Harness regulatory science to modernize development and evaluation of FDA- regulated products Advanced Manufacturing Approaches Analytical and Computational Methods II. Harness regulatory science to strengthen post-market surveillance and labeling of regulated products RWD to serve as RWE Utilizing and Validating Artificial Intelligence Adverse Event Reporting and Surveillance
- 28. 28 REFERENCE 1. https://www.fda.gov/vaccines-blood-biologics/guidance- compliance-regulatory-information-biologics/biologics- guidances (Access on Nov 25 2024). 2. Emily Braunstein, PhD., CBER Research Program Manager. Center for Biologics Evaluation and Research (CBER) Presentation, www.fda.gov.