About FDA

FY 2015 Report from the Director

The Center for Biologics Evaluation and Research (CBER) made significant contributions to public health during FY 2015.

Among the important new medical products CBER approved were vaccines against meningococcal disease, treatments for a bleeding disorders and inhalation anthrax, and the first system for reducing pathogens in donated platelets.

We also continued our support of the global fight against Ebola virus by expediting both reviews of multiple Investigational New Drug Applications for vaccine candidates and meetings with sponsors to provide timely guidance on various aspects of vaccine development.

I am pleased to be able to share details on these and other achievements in this report, my final one as director of CBER. As always, I am extremely proud of the role our center plays in promoting public health nationally and globally.

Advancing access to safe and effective new products

Office of Vaccines Research and Review

OVRR approved important vaccines against meningococcal disease and human papillomaviruses, as well as a combination pediatric vaccine to protect against diphtheria, tetanus, pertussis, and poliomyelitis.

  • Trumenba™: First vaccine licensed in the United States to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroup B in individuals 10 through 25 years of age.
  • Bexsero™:  Second vaccine licensed to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroup B in individuals 10 through 25 years of age.
  • Gardasil 9™: Vaccine indicated for the prevention of certain diseases caused by nine types of Human Papillomavirus (HPV), five more HPV types than Gardasil (previously approved by the FDA). Gardasil 9 has the potential to prevent approximately 90 percent of cervical, vulvar, vaginal and anal cancers.
  • Quadracel™: Vaccine indicated for active immunization against diphtheria, tetanus, pertussis and poliomyelitis for use in children 4 through 6 years of age.

Office of Blood Research and Review

OBRR approved products to manage bleeding disorders, reduce pathogen contamination of donated platelets, treat anthrax and snake bites, screen potential blood donors for viruses that cause serious disease, and to help control surgical bleeding.

  • Obizur Antihemophilic Factor (Recombinant): For the treatment of bleeding episodes in adults with acquired hemophilia A, a blood clotting disorder.
  • Anthrasil Anthrax Immune Globulin Intravenous (Human): Treatment for inhalation anthrax in combination with appropriate antibacterial drugs.
  • Anavip™: Crotalidae Immune F(ab')2 (Equine): Treatment for management of adult and pediatric patients to neutralize rattlesnake venom. 
  • MP™ Diagnostics HTLV Blot 2.4, Human T-cell Lymphotropic Virus-I/II (HTLV-I/II) test: First FDA-licensed supplemental test for this virus in human serum or plasma;  intended to confirm HTLV infection in blood donors and to differentiate between HTLV-I and HTLV-II following a positive result on an FDA-licensed HTLV-I/II blood donor screening test.
  • Raplixa: Treatment to help control bleeding in adults from small blood vessels when standard surgical techniques, such as suture, ligature or cautery, are ineffective or impractical.

Advancing Regulatory Science and Innovation

Office of Vaccines Research and Review

Making use of its clinical trial and regulatory decision-making expertise, OVRR played a major role in the global response to the Ebola epidemic by providing sponsors with advice on designing and conducting vaccine clinical trials, and expediting review of investigational vaccines and clinical trials. The Office also demonstrated the effect of a monoclonal antibody against poliovirus in mice and the effect of an adjuvant in improving human immune responses of a vaccine against the H7N9 influenza virus.

  • Workshop: “Immunology of Protection from Ebola Virus Infection” in collaboration with NIAID, DOD, CDC, and BARDA

Discussions of Ebola virus and vaccine immunology took place to inform future clinical, scientific, and regulatory decision-making.

Discussions on licensure, demonstrations of efficacy, post-marketing studies of effectiveness, research studies of Ebola in Africa.

  • IND reviews for Ebola vaccine candidates and vaccine clinical trial protocols
    • Expedited reviews of multiple Investigational New Drug Applications (INDs) for Ebola vaccine candidates.
    • Held many pre-IND meetings with sponsors within two weeks of the request to provide guidance on various aspects of vaccine development.
    • Expedited reviews of Phase 2 and Phase 3 clinical trial protocols for some candidate vaccines.
    • Participated in four WHO-sponsored joint reviews to address concerns and advise African regulators in the host countries of Liberia, Sierra Leone, and Guinea.  
  • CBER scientists participated in an assessment that suggested that a vaccine using the adjuvant Icomatrix™ promotes higher quality antibody immune responses against H7N9 avian influenza in humans than vaccine that does not contain the adjuvant.
  • CBER scientist demonstrated that a chimeric human/chimpanzee monoclonal antibody conferred pre- and post-exposure protection against type 1 poliovirus in transgenic mice

CBER study suggested that hybrid human/chimpanzee monoclonal antibodies that had been previously isolated in collaboration with the National Institutes of Health could be used in combination with drugs, a vaccine, or both, to improve their efficacy against polioviruses.

Office of Blood Research and Review

OBRR held a meeting to support development of treatment for a respiratory disease caused by lack of a blood protein, contributed to the development of blood screening assays for variety of viruses, and a faster way to identify specific strains of influenza viruses.

  • Public meeting: Patient-Focused Drug Development for Alpha-1 Antitrypsin Deficiency”: Patient-Focused Drug Development meeting to obtain input on the symptoms and other impacts that matter most to patients with alpha-1 antitrypsin deficiency, which increases risk for emphysema. FDA also sought patient perspectives on current approaches to treating this disorder.
  • Developing infectious organism genotype reference panels: 
    • Panels for four dengue virus types (DENV-1, 2, 3 and 4) have been used to produce a CBER Lot Release panel for validation of future licensed assays and are undergoing evaluation as candidate WHO International standards for the four DENV serotypes: these have also been provided to test kit manufacturers for development of blood screening assays;
    • HIV-1 RNA genotype reference panel was developed. 
    • Chikungunya virus genotype reference panel was developed
  • Convened Blood Products Advisory Committee to discuss strategies for implementing serological and nucleic acid testing for babesiosis organism in blood donors. (May 13, 2015)
  • Transmissible Spongiform Encephalopathies Advisory Committee (TSEAC) meeting at which FDA sought the advice of the TSEAC on the FDA policies to reduce risk of transfusion-transmitted Variant Creutzfeldt-Jakob disease (vCJD)--specifically, whether FDA’s geographically based donor deferral policies should be revised.
  • Public Workshop, “New Methods to Predict the Immunogenicity of Therapeutic Coagulation Proteins,” was held to discuss recent scientific progress in identifying the genetic determinants for an unwanted immune response to therapeutic coagulation proteins and to identify and discuss potential new methods to predict it. 

Office of Cellular, Tissue, and Gene Therapies

  • Collaborated with the National Academies of Science and Institute of Medicine on a consensus study of the ethical and social aspects of genetic modification of eggs and embryos to prevent transmission of disease caused by mutations in the mitochondria. The key aspect of the issue is the proposed transfer of nucleic genetic material from eggs or zygotes of a woman with such mitochondrial mutations to an egg or zygote of a woman with normal mitochondria.  A resulting child would have DNA from three individuals, the mother, father, and donor of the egg or zygote.
  • Convened a joint public meeting of the Cellular, Tissue and Gene Therapies Advisory Committee and the Oncologic Drugs Advisory Committee on April 29, 2015 to discuss talimogene laherparepvec (Imlygic™), an oncolytic immunotherapy for the treatment of patients with injectable regionally or distantly metastatic melanoma.

Office of Biostatistics and Epidemiology

OBE contributed to studies on the effectiveness and adverse effects of influenza vaccines and co-chaired a public meeting on innovations in clinical trials.

  • Initiated a follow-up study to assess febrile seizures after influenza vaccination for the 2013â€�14 and 2014-15 seasons in the FDA Sentinel Postmarket Rapid Immunization Safety Monitoring (PRISM) system.  
  • Published (with OVRR) results of retrospective study to assess effectiveness of high-dose inactivated influenza vaccine among elderly.  
  • Helped to organize and co-chaired public workshop, “Innovations in the Science and Practice of Clinical Trials.”
  • Collaborated with OBRR and NHLBI to establish the Transfusion Transmitted Infections Monitoring System (TTIMS) as of October 1, 2015. TTIMS enables FDA to evaluate HIV, Hepatitis B and C virus incidences, prevalence, and behavioral risk factors in those who present to donate blood or blood components.
  • Successfully completed a Sentinel study that concluded there was no association between venous thromboembolism (VTE) and vaccination with Gardasil.
  • In conjunction with the Center for Medicare & Medicaid Services, continues near real-time surveillance of GBS after influenza vaccination using Medicare claims data, an important tool for U.S. 
  • Initiated a follow-up study on Kawasaki Disease and PCV13 Pneumococcal Conjugate Vaccine in PRISM.
  • Presented at the 6th Annual Sentinel Initiative Public Workshop in February 2015 to highlight achievements of FDA’s Sentinel Initiative and explore the future of Sentinel as a national resource to support medical product surveillance.

International Affairs

Office of Vaccines Research and Review

OVRR provided critical guidance and advice to regulatory authorities and vaccine sponsors working to prevent the spread of Ebola, HIV, and influenza.

  • Collaborated with WHO and other national regulatory authorities to draft the WHO guidance documents "Post-licensure Assessment of Safety for Malaria Vaccines” and “Considerations for the Quality, Safety, and Efficacy of Ebola Vaccine”.  Also consulted on the WHO guidance “Use of Convalescent Whole Blood and Plasma Collected from Patients Recovered from Ebola Virus Disease for Transfusion as an Empirical Treatment during Outbreaks”.
  • Provided expert advice at 16 WHO consultations and related international meetings to assist national regulatory authorities in developing countries with development and evaluation of vaccines to protect against HIV, influenza, and Ebola.

Office of Blood Research and Review

Provided guidance and expert advice to WHO on treatments for Ebola, an assay to measure a blood coagulation protein, and assays for several infectious agents, such as West Nile Virus.

Office of Cellular, Tissue, and Gene Therapies

OCTGT led meetings on molecular imaging in cellular therapy studies of gene therapy products.

  • Convened the 18th U.S.-Japan Conference on Molecular Imaging in Cellular Therapy

The goal focused on cutting edge areas of biomedical research and enhancing opportunities for collaborations among scientists from the US and Japan, specifically, molecular imaging in cellular therapy.

  • Hosted and chaired a meeting with international regulators from the International Pharmaceutical Regulators Forum (IPRF) Gene Therapy Working Group (GTWG) to discuss bio-distribution studies for gene therapy products (May 2015).

Guidances and Rules

Office of Vaccines Research and Review

Provides information and recommendations on electronic submission of postmarketing safety reports involving vaccine products into the Vaccine Adverse Event Reporting System.

Office of Blood Research and Review

Provides recommendations to submitters and FDA reviewers in preparing and reviewing premarket notification submissions for human leukocyte antigen (HLA) in vitro diagnostic device test kits to match donors and recipients in transfusion and transplantation, whether for a single locus or for multiple loci simultaneously.

Provides blood establishments that collect blood or blood components, including Source Plasma, with revised donor deferral recommendations for individuals with increased risk for transmitting human immunodeficiency virus (HIV) infection.

Provides blood collection establishments and transfusion services recommendations for testing for bacterial contamination of platelets intended for transfusion.

Provides manufacturers of licensed whole blood and blood components intended for transfusion or for further manufacture, including Source Plasma, with recommendations intended to help determine which reporting mechanism is appropriate for submission of changes to an approved Biologics License Application.

Describes requirements for donor eligibility and donation suitability to ensure the safety, purity, and potency of the blood and blood components used for transfusion or for further manufacture.

Office of Cellular, Tissue, and Gene Therapies

Provides establishments that make donor eligibility determinations for donors of human cells, tissues and cellular and tissue-based products with updated recommendations concerning donor testing for evidence of infection with syphilis.  

Provides sponsors of virus or bacteria-based gene therapy products and oncolytic viruses or bacteria with recommendations on conducting viral shedding studies during preclinical and clinical development.

Assists sponsors and investigators in designing early-phase clinical trials for cellular therapy and gene therapy products, while giving current recommendations regarding clinical trials in which the primary objectives are the initial assessments of safety, tolerability, or feasibility of administration of investigational products.

Guides investigational new drug application sponsors and applicants for a biologics license application (BLA), or a supplement to a BLA, on determining whether to submit an Environmental Assessment (EA) for gene therapies, vectored vaccines, and related recombinant viral or microbial products.  

Provides sponsors, clinicians, and other establishments that manufacture and use human cells, tissues, or cellular or tissue-based products (HCT/Ps) from adipose tissue, with recommendations for complying with the regulatory requirements for HCT/Ps.

Provides human cells, tissues, and cellular and tissue-based product (HCT/P) manufacturers, healthcare providers, and FDA staff, with recommendations for meeting the criterion of minimal manipulation.

Provides manufacturers of human cells, tissues, and cellular and tissue-based products (HCT/Ps) with recommendations for complying with the requirements for investigating and reporting adverse reactions involving communicable disease in recipients of HCT/Ps that are regulated solely under section 361 of the Public Health Service Act (PHS Act) and 21 CFR Part 1271.

Amends certain regulations regarding donor eligibility, including the screening and testing of donors of particular HCT/Ps, and related labeling. The rule proposes these changes in response to enhanced understanding in this area and in response to comments from stakeholders regarding the importance of embryos to individuals and couples seeking access to donated embryos.

CBER Guidances and Rules with the Center for Drug Evaluation and Research

Provides information and recommendations on drug and biological product development when human efficacy studies are not ethical or feasible. The regulations that set forth the pathway for approval of these products are commonly referred to as the Animal Rule.

Provides answers to common questions from sponsors interested in developing biosimilar products, biologics license application (BLA) holders, and other interested parties regarding FDA’s interpretation of the Biologics Price Competition and Innovation Act of 2009 (BPCI Act).

Assists sponsors to demonstrate that a proposed therapeutic protein product is biosimilar  to a reference product for purposes of the submission of a marketing application

Describes the Agency’s current thinking on factors to consider when demonstrating that a proposed therapeutic protein product is highly similar to a reference product licensed under section 351(a) of the Public Health Service Act (PHS Act) for the purpose of submitting a marketing application under section 351(k) of the PHS Act.

Provides answers to common questions from sponsors interested in developing proposed biosimilar products, biologics license application (BLA) holders, and other interested parties regarding FDA’s interpretation of the Biologics Price Competition and Innovation Act of 2009 (BPCI Act).

   Requires all applicants of covered, approved prescription drug or biological products other than blood or blood components for transfusion, all applicants of blood or blood components that manufacture a significant percentage of the U.S. blood supply, and all manufacturers of covered prescription drugs marketed without an approved application, to notify FDA electronically of a permanent discontinuance or an interruption in manufacturing of the product that is likely to lead to a meaningful disruption in supply of the product in the United States.

Describes the conditions under which FDA does not intend to take action for violations of sections 351 of the PHS Act and sections 502(f)(1) and where specified, section 501(a)(2)(B) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), when a state-licensed pharmacy, a Federal facility, or an outsourcing facility dilutes, mixes or repackages certain biological products without obtaining an approved BLA.

General Accomplishments for Operational Excellence

  • Implementation of more than 70% of the FY 2015 Program Alignment objectives outlined in the program Action Plans and Streamlining Plans of the Biological Products Action Plan on time.

This plan is intended to “facilitate increased operational and program alignment as FDA transitions to distinct commodity-based and vertically-integrated regulatory programs with well-defined leads, coherent policy and strategy development, and well-designed and coordinated implementation.” Among the accomplishments for FY 2015 were:

  • developing a charter for the CBER/ORA Action Plan Steering Committee
  • delegating authority for Class 1 recalls from ORA to CBER
  • streamlining import alerts for CBER-regulated products and developing a strategic plan for imports of CBER-regulated products

Performance on Product Reviews and Product Availability

CBER completed or exceeded performances goals related to premarket product reviews as identified in the Prescription Drug User Fee Act (PDUFA), Medical Device User Fee Amendments (MDUFA), Biosimilar User Fee Act (BsUFA), Generic Drug User Fee Act (GDUFA), Animal Drug User Fee Act (ADUFA),  and Animal Generic Drug User Fee Act (AGDUFA).

  • Completed 100% of standard and priority BLA/NDA reviews within the specified review time frames
  • Acted on 100% of standard and priority Efficacy Supplements within the specified time frames. 
  • Exceeded the performance target for 510(k) submissions and Real Time Supplements, issuing a MDUFA decision on 100% of submissions received (FY 2015 receipt cohort) within 90 FDA days. 
  • Exceeded the performance requirement in funding 22 medical countermeasures (MCM) regulatory science projects on emerging infectious diseases, pandemic influenza, and other chemical, biological, radiological, and nuclear threat (CBRN) issues.  In addition, two regulatory science projects were awarded Ebola supplemental funding and two were awarded challenge grant funding through FDA’s Medical Countermeasures Initiative Program.

 

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