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FY 2013 Report from the Director

The Center for Biologics Evaluation and Research (CBER) made substantial progress in fulfilling its mission to ensure the safety, purity, potency, and effectiveness of biological products during fiscal year 2013.

I’m pleased to provide details of our work, including recent approvals, strides made in achieving the mandates of the Food and Drug Administration Safety and Innovation Act (FDASIA) and other regulatory objectives, work advancing the safety and effectiveness of medical products, collaborations addressing preparedness for emerging infectious diseases and pandemic influenza, and efforts furthering FDA’s global product safety strategy.
 

 Advancing access to safe and effective new products

CBER made important new healthcare products available to the public during fiscal year 2013 through timely review of many investigational applications for biologics and devices and the approval of many marketing applications.


The Office of Vaccines Research and Review licensed five new vaccines, including two of the first influenza vaccines produced using innovative technologies to be licensed in the US. These technologies, cell culture and recombinant DNA, have the potential to speed the start-up of vaccine manufacturing in the event of a pandemic, thus offering the public more rapid access to potentially life-saving biologic products.
 

  • Flucelvax is the first seasonal influenza vaccine licensed in the US produced using cell culture technology instead of the traditional egg-based process.  By making an adequate supply of readily available and previously tested cells for vaccine production it helps to ensure a faster manufacturing startup.
     
  • Flublok is the first seasonal influenza vaccine produced using recombinant DNA technology, which speeds vaccine production because it does not depend on an egg supply or the availability of the intact influenza virus.
     

OVRR also licensed three quadrivalent influenza vaccines (vaccines designed to protect against four different influenza viruses):
 

  • Fluzone Quadrivalent, Fluarix Quadrivalent, and FluLaval Quadrivalent are the first inactivated influenza vaccines containing four strains and available in the US for the prevention of influenza caused by two influenza A subtype viruses and two type B viruses.  Fluzone is indicated for individuals 6 months of age and older; Fluarix and FluLaval are indicated for individuals 3 years of age and older.
     

CBER also approved three Efficacy Supplements for other types of vaccines, i.e., applications to make certain changes (e.g., add a new indication) to an approved marketing application:
 

  • Prevnar 13 prevents invasive pneumococcal disease caused by serotypes contained in the vaccine; the supplement extended its use to children 6 years through 17 years of age.
     
  • Ixiaro prevents Japanese encephalitis, a viral infection of the brain that is endemic in several tropical and subtropical regions in Asia and that affects over 50,000 people annually. The supplement extends the use of this vaccine to individuals 2 months to under 17 years of age.  
     
  • Menveo, the first four-strain vaccine for the prevention of invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, Y and W-135 in individuals as young as 2 months of age.
     

The Office of Blood Research and Review approved important products for treating a variety of serious blood diseases along with devices and tests to ensure blood product safety.
 

Therapies

  • Botulism Antitoxin Heptavalent is for treating botulism after exposure to botulinum neurotoxin. This is the first botulism antitoxin targeting all seven known types of botulinum nerve toxin that cause botulism, and the first plasma derivative approved using the Animal Rule.
     
  • Kcentra, Prothrombin Complex Concentrate (Human), is a combination of blood clotting factors that reverses the effects of oral anticoagulation therapy from a Vitamin K antagonist such as warfarin when an adult individual experiences acute bleeding (e.g., bleeding after an injury). Since Kcentra does not require blood group typing or thawing, this product can be administered faster than is possible with frozen plasma.
     
  • RIXUBIS [Coagulation Factor IX (Recombinant)] is the first recombinant product that is specifically indicated for routine prevention or reduction in the frequency of bleeding episodes in adult patients with Hemophilia B.
     
  • Alere Determine HIV-1/2 Ag/Ab Combo assay is the first rapid HIV test that simultaneously detects both HIV-1/2 antibodies and HIV-1 p24 antigen. The test can be used by trained professionals in outreach settings (e.g., community-based organizations) to identify HIV-infected individuals who might not be tested in traditional health care settings.
     
  • DG Gel® cards is for use in identifying red blood cell antigens (i.e., molecules that help determine blood types) and detecting and identifying antibodies against red blood cells. They constitute the second US-licensed blood typing and antibody detection and identification system using gel column agglutination technology to assist in blood typing discrepancies.
     

Blood safety device

  • iTrace for Blood Centers  was approved for use in enhancing blood safety by preventing the release of unsuitable blood products. It is the first device employing Radio Frequency Identification technology cleared for use in blood establishments to track and monitor blood products.
     

The Office of Cellular, Tissue and Gene Therapies approved three hematopoietic stem cell products.
 

  • HPC Cord Blood (Life South; June 2013), ALLOCORD (SSM Cardinal Glennon Children’s Medical Center; May 2013),  and DuCORD (Duke University; October 2012) are cord blood products that are potentially life-saving treatment options for patients with disorders affecting the hematopoietic (blood-cell- making) system that are inherited, acquired during life, or that result from cancer treatment. These products comprise stem cells that can produce new red blood cells and immune system cells when transplanted into people unrelated to the donors of these cells. 
     

Completion of FY 2013 mandates of Food and Drug Administration Safety and Innovation Act (FDASIA) and other regulatory objectives

  
CBER successfully fulfilled all of its FY 2013 deliverables mandated under FDASIA , including:
 

  • Critical contributions to three reports to Congress:
     

Performance on Product reviews

CBER met or exceeded expectations in its review of applications for medical devices and biologics during FY 2013:
 

  • Issued a review decision on 100% of 510(k) submissions for medical devices within 90 days as stipulated by the Medical Device User Fee Amendments, 2012, exceeding the target of 91% within that timeframe.
     

Issuance of Guidance Documents

CBER released five guidances during FY 2013 that advanced medical product safety, effectiveness, and innovation.
 

Two guidance documents from the Office of Cellular, Gene, and Tissue Therapies (OCTGT) are the first comprehensive set of recommendations to sponsors and investigators on the required preclinical data and on the design of early phase clinical trials for cellular and gene therapy products.

Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products (Draft Guidance, November 2012)

This guidance provides sponsors and individuals who design and implement preclinical studies with recommendations on the substance and scope of preclinical information needed to support clinical trials for investigational cellular therapies, gene therapies, therapeutic vaccines, xenotransplantation, and certain biologic-device combination products reviewed by the Office of Cell, Tissue, and Gene Therapies.  When finalized, this guidance will supersede the recommendations in section VIII in the final guidance entitled “Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy” dated March 1998.

Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products (Draft Guidance, July 2013)

This guidance provides recommendations to assist in designing early phase clinical trials of cellular and gene therapy products. Its goal is to clarify expectations of the Office of Cellular, Gene, and Tissue Therapies regarding clinical trials in which the primary objectives are the initial assessments of safety, tolerability, or feasibility of administration of investigational products. The scope of this guidance is limited to products for which the office has regulatory authority.

An additional two guidance documents from OCTGT provide recommendations for the submission of Investigational New Drug (IND) and Biologics License Applications (BLA) of unlicensed HPC, Cord Blood for rebuilding the red and white blood cell populations in individuals with blood diseases that disrupt this process.

Guidance for Industry and FDA Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System (Draft, June 2013)

Guidance for Industry: Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients With Disorders Affecting the Hematopoietic System (Draft, June 2013)

A fifth guidance, from the Office of Blood Research and Review, provides recommendations for the use of FDA-licensed nucleic acid tests (NATs) to screen blood donors for hepatitis B virus (HBV) deoxyribonucleic acid (DNA) and for product testing, donor management, requalification of previously deferred donors, and product labeling. NATs analyze variations in the sequence, structure, or expression of deoxyribonucleic acid (DNA) and ribonucleic acid (RNA) in order to diagnose disease or medical conditions, infection with an identifiable pathogen, and determine genetic carrier status. The guidance reflects the view of CBER that the use of an FDA-licensed HBV NAT is necessary to reduce the risk of HBV transmission. 
 

Use of Nucleic Acid Tests on Pooled and Individual Samples from Donors of Whole Blood and Blood Components, including Source Plasma, to Reduce the Risk of Transmission of Hepatitis B Virus (Final Guidance,  November 2012.
 

Advancing the Safety and Effectiveness of Medical Products

Product assessments using the Mini-Sentinel system

CBER initiated three large-scale post-marketing safety signal evaluations of CBER-regulated vaccines with Mini-Sentinel's Post-Licensure Rapid Immunization Safety Monitoring (PRISM) system and completed the study of intussusception (potentially life-threatening telescoping of one part of the intestine into a nearby portion, causing blockage) following vaccination against rotavirus by RotaTeq. The findings prompted the first safety labeling change made by FDA as a result of a Mini-Sentinel protocol-driven evaluation.  CBER presented final results to the Center for Disease Control and Prevention’s Advisory Committee on Immunization Practices in June 2013
 

CBER also initiated the first protocol-based evaluation of a blood-derived product using Mini-Sentinel, which will examine the risk of thromboembolic events after immunoglobulin administration.
 

Comparison of the effectiveness of high- and low-dose influenza vaccine in elderly Medicare beneficiaries

CBER initiated a study, in collaboration with the Centers for Medicare and Medicaid Services (CMS) and the CDC, to compare the effectiveness of Fluzone High-Dose influenza vaccine and the standard dose influenza vaccine among the US elderly during the 2010-11, 2011-12 and 2012-13 influenza seasons.  Early pilot results suggested that the Fluzone High-Dose vaccine is more effective in persons 85 years of age or older.  Analyses are ongoing, with final results expected in 2014.
 

Evaluating new methods to evaluate vaccine potency

In FY 2013 CBER evaluated three new methods for determining influenza vaccine potency and measuring the potency of inactivated influenza vaccines from several manufacturers: enzyme-linked immunosorbent assay, surface plasmon resonance, and label-free, antibody-free mass spectroscopy. This work increased manufacturing diversity and capacity for influenza vaccine production. It is part of our overall goal of preventing safety problems by modernizing science-based standards and tools to ensure high-quality manufacturing, processing, and distribution of products we regulate. This also reflects our ongoing response to the Government Performance and Results Act of 1993 (GPRA, Performance Goal 234101).
 

Advancing Regulatory Science and Innovation

CBER had significant achievements in its efforts to advance regulatory science and innovation. Among those efforts were new types of treatment for infectious diseases and reviewing new and ongoing in-house research projects:
 

Advancing Preparedness for Emerging Infectious Diseases and Pandemic Influenza, including Medical Countermeasures

CBER made significant contributions to the preparedness of the US and other nations for emerging infectious diseases:  
 

  • Key participant in the U.S. federal public health response to the appearance of a novel influenza virus with pandemic potential, H7N9, reported in other countries:
     
    • Leadership on a cross-HHS working group on the development of potentially safe and effective vaccines.
       
    • Preparation of potency reagents and provided to vaccine manufacturers.
       
    • Collaboration with NIH to develop a protocol for the conduct of human clinical trials to obtain preliminary safety and immunogenicity data which will inform use of the vaccine in populations.
       
  • Held a public meeting of VRBPAC to discuss and make recommendations pertaining to the safety and immunogenicity of the first adjuvanted vaccine for the prevention of H5N1 influenza in individuals 18 years and older. 
     
  • Convened VRBPAC to discuss and make recommendations on the safety and efficacy of a Hepatitis B Vaccine.
     
  • Constructed stably bioluminescent derivatives of clinically relevant Stapylococcus aureus MRSA strains for use in imaging MRSA infections.
     
  • Developed two genetically modified live attenuated (weakened) Leishmania donovani parasites (the cause of the parasitic disease leishmaniasis) to test as vaccine candidates. These vaccine candidates are safe, stimulated the immune system, and protected against virulent parasite challenge in animal models. In laboratory studies they also triggered a strong protective immune response in immune system cells from healthy humans, which suggests they could offer protection if used as vaccines in humans.
     
  • Conducted a site visit to the United Kingdom to assess the potential for an anthrax vaccine production facility (Anthrax Vaccine Precipitated) to supply vaccines to the US.
     

Advance FDA’s Global Product Safety Strategy

CBER also undertook significant projects as part of its global role in advancing product safety. These projects reflect the critical contribution FDA makes to the safety and efficacy of healthcare products worldwide.
 

    • Played a leadership role in the development of written standards and a guidance document on the nonclinical safety assessment of adjuvanted vaccines. (Adjuvants enhance the immune response triggered by the vaccine itself.) This document, once finalized, will provide guidance to global vaccine manufacturers regarding testing and safety assessments of adjuvanted vaccines, thereby facilitating availability of these products throughout the world. 
       
    • Participated as a member of the WHO Expert Committee on Biological Standardization and the WHO Blood Regulators Network, whose achievements included:
       
      • Establishing international standards for dengue viruses and West Nile virus. 
         
      • Developing a consensus statement supporting the inclusion of whole blood and red blood cells on the WHO Model List of Essential Medicines
         
      • Developing a consensus position statement on blood donor deferral for men who have had sex with other men.   
         
  • Participated in the feasibility assessment and early development of an international vaccine safety surveillance network designed to assess rare and complex adverse events. 
     
  • Provided expert faculty to address vaccine safety and regulatory decision making for the WHO training course, “Pharmacovigilance:  the study of Adverse Drug Reactions and Related Problems.” 
     
  •  Attended and provided expert advice at 19 WHO meetings to assist National Regulatory Authorities in developing countries with the development of vaccines to protect against diseases including dengue, influenza and typhoid fever.