About FDA

FY 2012 Report from the Director

Karen Midthun, MD

I am pleased to report that during fiscal year 2012, the Center for Biologics Evaluation and Research (CBER) made substantial progress in fulfilling its mission.
This letter reports highlights of progress made during the past year. The accomplishments described reflect the extraordinary efforts of CBER employees in executing the Center’s regulatory and research responsibilities. Working together, CBER staff have successfully advanced the development of important new products through informed oversight and innovative research.
Access to Safe and Effective New Products
Our efforts to make important new healthcare products available to the public in a timely manner were reflected by the many investigational applications for biologics and devices that CBER reviewed and by the marketing applications that CBER approved in FY 2012.
Product Approvals
CBER exceeded its Prescription Drug User Fee Act (PDUFA) performance goals for reviewing standard and priority Biologics License Applications (BLA) during FY 2012 by completing 100 percent of reviews within specified time frames. The Center also exceeded its Medical Device User Fee Act (MDUFA) goals for 510(k) applications (devices substantially equivalent to legally marketed devices) by completing 100 percent of reviews within 90 days.
CBER’s product offices, the Office of Vaccines Research and Review (OVRR), the Office of Blood Research and Review (OBRR), and the Office of Cellular, Tissue and Gene Therapies (OCTGT), in collaboration with the Office of Biostatistics and Epidemiology (OBE) and the Office of Compliance and Biologics Quality (OCBQ), each contributed to a number of important approvals.
OVRR approved two important vaccines this year:
  • FluMist Quadrivalent is the first seasonal influenza vaccine containing four strains of the influenza virus: two influenza A strains and two influenza B strains. Influenza vaccines have traditionally protected the public from two influenza A strains and one influenza B strain. The inclusion of a second B strain in FluMist Quadrivalent increases the likelihood of adequate protection against circulating influenza B strains.

  • MenHibrix is a combination vaccine for infants and children ages 6 weeks through 18 months for prevention of bacterial meningitis caused by Neisseria meningitidis serogroups C and Y and Haemophilus influenzae type b. Diseases caused by the bacteria Neisseria meningitidis (meningococcal disease) and Haemophilus influenzae type b (Hib disease) can be life-threatening. These bacteria can infect the bloodstream causing sepsis, and can infect the lining that surrounds the brain and spinal cord causing meningitis. In young children, Neisseria meningitidis and Haemophilus influenzae type b are important causes of bacterial meningitis.
OBRR approved several important products for blood donor screening and for testing for HIV including:
  • The OraQuick In-Home HIV Test - this test is a single-use rapid assay for detecting antibodies to HIV in human oral fluid specimens. It is an over-the-counter test that enables consumers to perform the test and interpret the results in private. It is the first in-home HIV test of its kind to be approved by FDA.

  • The Abbott ESA Chagas - this test is an additional, more specific test for use on human serum or plasma specimens previously found to be repeatedly reactive using a licensed screening test for antibodies to Trypanosoma cruzi (T. cruzi). T. cruzi causes Chagas disease, a serious and potentially fatal parasitic infection.

  • The Avioq HTLV-I/II Microelisa System - this test identifies the presence of HTLV-I and II in blood donors and can also be used as an aid in clinical diagnosis.

  • The PROCLEIX ULTRIO Plus Assay – this assay tests for the presence of genetic material from HIV, hepatitis B and hepatitis C and was approved as a supplemental test that confirms infection of specimens from human donors, including donors of blood, blood components, plasma, and organs that are repeatedly reactive on licensed donor screening tests for these viruses.
OCTGT approved two important cellular therapy products:
  • The first and second licensed allogeneic (donated) cord blood hematopoietic progenitor cell therapies (HEMACORD and HPC Cord Blood). These products are used to reconstitute both red blood cell and immune system cell populations in patients with disorders affecting the hematopoietic (blood-cell-producing) system that are inherited, acquired, or result from cancer treatments.
Guidance Documents
Another of CBER’s strategies for supporting development of safe and effective products is the publication of guidance documents that explain FDA’s current thinking on a variety of relevant topics. The following is a list of particularly important guidance documents that illustrates the broad scope of CBER’s responsibility in regulating biological products:
CBER, working together with CDER, played a significant role in developing three draft guidance documents related to biosimilar biological products:
CBER drafted and published guidance documents aimed at enhancing the safety and availability of blood including:
In support of development of vaccines designed to treat cancer, CBER published the following guidance:
CBER also provided general guidance to manufacturers of a variety of cells, tissues, and cellular and tissue-based products including:
Advance Safety and Effectiveness of Medical Products
In FY 2012 CBER expanded its product surveillance efforts through the use of Mini-Sentinel, a pilot project providing the opportunity for active adverse event surveillance that FDA is using to guide development of the Sentinel System for monitoring the safety of medical products regulated by the agency. The expansion of surveillance efforts also included the launch of a contract for the Blood Safety Continuous Active Surveillance Network (Bloodscan) and continuing the evaluation of vaccine safety in the Post-licensure Rapid Immunization Safety Monitoring (PRISM) program.
OVRR and the OBE began the evaluation of three different vaccines (RotaTeq, Rotarix, and Gardasil) using the Mini-Sentinel system. These evaluations are aimed at providing better information to help clarify potential safety concerns that have arisen from other surveillance systems. They will also enable FDA to better assess any potential risk of intussusception (bowel obstruction) from the rotavirus vaccines (RotaTeq or Rotarix) and venous thromboembolism (VTE, blood clots deep in the body) from Gardasil (human papillomavirus vaccine). While the increase in the rate of reported VTE following administration of Gardasil is not statistically significant, and other risk factors might explain the blood clots, CBER is conducting this safety assessment to more thoroughly evaluate this observation.
In another surveillance project, OBRR and OBE, in collaboration with the Centers for Medicare and Medicaid Services and HealthCore Integrated Research Database, found evidence that some antibody-containing products used to treat individuals who have weakened immune systems and certain other blood diseases are associated with an increased risk of potentially fatal blood clots. CBER shared this information with health care professionals and the public.
Another way CBER improved public safety during FY 2012 was by completing implementation of Direct Recall Classification (DRC). DRC refers to the classification of biological product recalls directly by personnel in CBER rather than through the more laborious, time-consuming methods that require additional work by personnel in each FDA field office involved in the recall. For the agency, as well as for the public, DRC decreases the resources needed to classify and publish recalls, facilitating the reallocation of resources to other agency priorities related to protecting the public health.
Advancing Regulatory Science and Innovation
Much of our work at CBER is designed to support the development of safe and effective biological products by advancing regulatory science and promoting scientific innovation. In FY 2012 we articulated this part of our mission through the development and posting on our external website of a comprehensive five-year plan for research focused on advancing the development of biological products.
Among the many goals of the plan are strategies for research promoting new techniques and tools for regulatory testing of product safety and potency, as well as strategies for new product development.
For example, CBER scientists developed a non-human primate model for pertussis (whooping cough) that will enable studies that significantly improve understanding of protective immune responses to this disease. This model also has the potential to facilitate development of new pertussis vaccines at a time when there is a resurgence of this disease in the United States.  
In an effort to incorporate new science into biological product testing, CBER also updated the rule governing sterility testing to make it more efficient and less burdensome. The final sterility rule supports state-of-the-art testing technologies that give accurate and reliable results, often more quickly and with less effort than methods required in the older regulations. Moving forward, the amendments to the sterility testing rule will provide manufacturers of biological products the flexibility, as appropriate, to keep pace with technological and scientific advances.
Advance Preparedness for Emerging Infectious Diseases and Pandemic Influenza, including Medical Countermeasures
A significant amount of our innovation and regulatory research work was directed to medical countermeasures (MCM), which are those products intended to protect or treat the public in the event of chemical, biological, or radiological threats, including emerging infectious diseases.
For example, OVRR pursued research toward the development of a vaccine that could safely and effectively target a wide variety of influenza viruses. Our scientists also developed new methods to evaluate the ability of influenza vaccines to stimulate the immune system. In addition, they developed new methods to measure the potency of influenza vaccines.
OBRR scientists also contributed to CBER’s MCM work by continuing their efforts to adapt an existing blood testing technology for the identification of genetic material from pathogens in blood samples to be more broadly applicable. This contributes to the Center’s efforts in protecting the public health by ensuring a safe blood supply.
Advance FDA’s Global Product Safety Strategy
In addition to the significant contributions to public health in the United States, much of CBER’s research and regulatory work has global impact. Indeed, the World Health Organization (WHO) re-designated CBER as a WHO Collaborating Center for Biological Standardization for 2012 through 2016. In this important role, the Center helps to establish reference standards for biological products, such as vaccines and laboratory tests, develops written standards for biologicals, supports implementation of these standards worldwide, and conducts research that advances standardization of biologicals.
CBER also continued its global collaboration by sharing results of its regulatory science research with other WHO Collaborating Centers and the global community. This collaboration helped to advance product development for influenza and other vaccines, improve methodologies for influenza strain selection, and enhance safety monitoring of the public following vaccination.
In summary, this brief look at some of our major accomplishments of FY 2012 reflects the hard work and skills of all CBER employees. I am grateful for their contributions and dedication to the Center’s mission. I look forward to continued achievements in the coming year.
Karen Midthun, M.D.
Center for Biologics Evaluation and Research
United States Food and Drug Administration


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