Vaccines, Blood & Biologics
Highlights of CBER’s Medical Countermeasure Regulatory Science and Research Program
Increasing the nation’s preparedness to address threats as a result of bioterrorism and pandemic and emerging infectious diseases
CBER’s regulatory science and research priorities focus on developing a comprehensive approach to rapid pathogen detection, enhancing preparedness for seasonal and influenza vaccines, and facilitating licensure of medical counter-measures for treating or preventing diseases due to bioterrorist attacks. The purpose of our regulatory science program is to provide both proactive solutions to fill gaps in scientific data, tools, or approaches needed to perform regulatory review, while also providing a cadre of scientific experts that are available to be responsive to public health emergencies that may require a laboratory response.
- Rapid Pathogen Detection
The threat posed by transmissible infectious microorganisms in biological products has led to the establishment of a variety of safety measures: Donors of blood and tissues must be tested for pathogens and deferred if necessary; biologic products must be tested for bacteria and fungi during manufacture whenever feasible; vaccines, cell and gene therapies, and the cell substrates used to manufacture these products must be tested for a wide variety of infectious agents that are not amendable to methods for inactivating or removing them during manufacture. Furthermore, newly emerging infectious agents could also threaten product safety and availability due to the spread of disease vectors (e.g., dengue), travel and immigration (e.g., malaria), terrorism (e.g., anthrax), or previously rare or unknown agents (e.g., XMRV and Q-fever).
CBER performs proactive, comprehensive research to respond to new and emerging safety threats by:
- Working collaboratively within the center and with sister agencies in DHHS to identify, assess, monitor and prioritize emerging threats
- Creating a scientific team to facilitate development and availability of reagents, samples and methods for developing, validating, and deploying screening tests for emerging priority pathogens by public-private collaboration
- Assessing and encouraging development of new technologies that enable rapid, sensitive, specific, high throughput, cost-effective screening of blood, tissues and their donors, and other biologics for known, emerging and unknown pathogens, including monitoring and testing during manufacturing
- Developing and evaluating methods for broad pathogen detection
- Assessing, developing, and improving new methods to inactivate pathogens without impairing product function
- Improving microbial safety of human tissue products by developing and evaluating better processing conditions, pathogen inactivation, and/or pathogen detection
- Preparedness for Pandemic Influenza
CBER ensures the safety, quality, and potency of both seasonal and pandemic influenza vaccines.
CBER prepares for pandemic influenza while enhancing yearly review for seasonal influenza vaccines by:
- Developing and evaluating improved tools and methods to enhance safety, effectiveness, quality, and potency testing of influenza vaccines, including faster methods to develop reference reagents to allow for more rapid response in pandemics
- Identifying and implementing new approaches to reliably generate seed stocks that replicate more efficiently, thus speeding vaccine manufacture
- Developing and evaluating preclinical methods to screen novel adjuvants for adverse effects prior to clinical trials, thus increasing the availability of highly effective vaccines during a pandemic
- Evaluating the safety of new production methods for influenza vaccines, such as use of mammalian cell substrates
- Preparedness for Bioterrorism
CBER focuses on vaccines and therapeutics for treating bioterrorism agents and regenerative medicine products to treat victims of chemical or radiological terrorist attacks.
- Bioterrorism Agents
The ethical prohibition against performing clinical studies of vaccine efficacy by challenging healthy volunteers with a bioterrorism agent means that regulatory review and licensure of most vaccines and therapeutics for bioterrorism agents must be based on nonclinical data.
CBER facilitates compliance with the Animal Rule to facilitate licensure of products for treating or preventing bioterrorist attacks by:
- Developing and evaluating nonclinical models to study pathogenesis and identify relevant correlates of immunity
- Creating methods and models to study potential toxic effects of vaccine antigens, adjuvants, and other components of vaccines
- Determining biomarkers of pathogenicity and developing new methods to evaluate and ensure safety of live vaccines
- Studying mechanisms of vaccine-related adverse events, ways to mitigate them, and biomarkers of predisposition to adverse events
- Creating methods to evaluate and improve immunogenicity, potency, and efficacy of vaccines
- Studying innate and adaptive immunity against viral and bacterial diseases, and mechanisms of immunopathology (including allergy), and developing new approaches to inducing protective immunity
- Chemical or Radiological Agents
CBER regulates products used to treat persons injured by chemical or radiological acts of terrorism. These products include cell-based therapies for wound repair, tissue regeneration, and reconstitution of bone marrow.
CBER prepares for evaluation of novel regenerative medicine products for treating victims of chemical or radiological terrorist attacks by:
- Improving safety and efficacy of stem cell-based products and combination and tissue engineering medical products by using systems biology, modern methods of cell characterization (e.g., genomics, epigenetic analysis, proteomics, cell surface phenotypic analysis, and measures of in vitro function), and assessment and development of animal models to identify biomarkers for appropriate cell differentiation state and function, localization, and controlled proliferation.