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  1. Vaccines, Blood & Biologics

FY 2018 Report from the Director

Dr. Peter Marks sitting at a desk smiling

Fiscal Year 2018 was productive and exciting at the Center for Biologics Evaluation and Research (CBER). We continued to support the promise of 21st Century medicine, advancing science and technology in the service of public health.

One of the highlights of FY 2018 was the approval of two gene therapies: Luxturna, the first directly administered gene therapy for a specific genetic disorder, an inherited retinal disease, and Yescarta, a cell-based gene therapy for certain types of adult, large B-cell lymphoma. We also approved HEPLISAV-B, a Hepatitis B vaccine to prevent infection caused by all known subtypes of hepatitis B virus in adults 18 through 70 years of age, Shingrix, a vaccine for the prevention of shingles in adults 50 years of age and older, and Fluarix Quadrivalent, to prevent influenza disease in children as young as 6 months of age. In addition, CBER/FDA granted an Emergency Use Authorization to the Department of Defense to enable the use of Freeze-Dried Plasma to treat hemorrhage or coagulopathy of U.S. military personnel injured during military combat when plasma is not available or its use is not practical.

FDA’s ongoing contributions to controlling Zika virus included CBER’s approval of the cobas Zika Test and the Procleix Zika Virus Assay (Nucleic Acid Tests), the first donor screening tests for the direct detection of Zika Virus RNA in human plasma from individual donors.

The report below highlights these and other CBER accomplishments that reflect our responses to new and ongoing scientific and regulatory challenges, such as those posed by advanced therapies, emerging infectious diseases, and threats to the blood supply. It also reflects how we are carefully focusing our own research and stakeholder outreach so they support our efforts to meet those challenges. Just to highlight one aspect of stakeholder outreach undertaken this year, we launched the INTERACT program (CBER INitial Targeted Engagement for Regulatory Advice on CBER producTs). These meetings enable sponsors to obtain preliminary informal consultation with the Agency at an early stage of development prior to a pre-IND meeting.

Our successes this year also included meeting or exceeding the expectations of the Prescription Drug User Fee Act VI and the Medical Device User Fee Amendments, as detailed below. CBER also initiated or completed efforts on behalf of the 21st Century Cure Act, including patient experience in regulatory decision making, providing grants to research institutions, drafting guidances for regenerative medicine, and contributing to a guidance on antibacterial and antifungal drug development.

CBER is pleased to provide this report, which demonstrates the Center’s continuing dedication to our vision and our mission of meeting the challenge of improving public health, nationally and globally.

Peter Marks, M.D., Ph.D.
Center for Biologics Evaluation and Research

Advancing access to safe and effective products

CBER advanced public health in 2018 by achieving or exceeding expectations in its scientific and regulatory overview of new products. These achievements included the following accomplishments:

Prescription Drug User Fee Act VI (PDUFA)

  • All four standard schedule (non-priority) and one priority Biologic License Applications received in FY 2018 were under review and within the review-time goal at the close of FY 2018.
  • CBER acted within the goal timelines for all 11 BLA PDUFA applications received in FY 2017, as of September 2018. Among the six applications with standard schedules, four were approved, one was in Complete Response status, and one was withdrawn; among five Priority schedule applications, three were approved, one was in Complete Response status, and one was withdrawn.

Medical Device User Fee Amendments (MDUFA)

  • CBER exceeded the FY 2018 goal of 95% approval for 510(k) submissions by issuing a decision on 100% of submissions received in the FY 2018 cohort within 90 FDA days, as of June 30, 2018.
  • CBER exceeded the FY 2018 goal of 95% for the “within-90-FDA-days” approval of Real Time PMA Supplements by issuing a MDUFA decision on 100% of submissions received within that time limit, as of June 30, 2018.
  • CBER exceeded the FY 2018 goal of 95% approval of PMA 180-day supplements by issuing a MDUFA decision on 100% of submissions within that time limit.

21st Century Cures Act investments

  • CBER completed its coordination of the Innovation Fund as required by the 21st Century Cures Act, and invested $14 million of its resources by the end of FY 2018. As part of our commitment to achieving the goals of the 21st Century Cures Act, CBER awarded nearly $3 million in grants to support research at five institutions aimed at developing more innovative, consistent, and dependable manufacturing of biological products.

Mutual Recognition Agreement (MRA) with foreign regulatory agencies

  • CBER assessed regulatory agencies of 14 countries to ensure their inspection of medical products regulated by the Center could be recognized by the FDA, as per required by the MRA. CBER continued to assess the remaining European Union member states to meet December 2018 and July 2019 commitments.

Emergency Use Authorization (EUA) for freeze-dried plasma product to treat military personnel

  • CBER/FDA granted an EUA to the Department of Defense (DoD) to enable the emergency use of Pathogen-Reduced Leucocyte-Depleted Freeze-Dried Plasma manufactured by the Centre de Transfusion Sanguine des Armées (French FDP). The product is used to treat hemorrhage or coagulopathy of U.S. Military personnel injured during military combat when plasma is not available or its use is not practical. The granting of the EUA demonstrates FDA’s commitment to the joint program that was started in FY 2018 to further enhance FDA’s support of DoD to ensure the efficient development of medical products to treat injured military personnel, focusing first on products regulated by CBER.

Approval of major, innovative, complex biologic products

FY 2018 was a productive year for approvals of novel biologic products, especially cell and gene therapies. CBER also approved new vaccines and continued to approve blood screening devices for emerging infections. The introduction of these products reflect the commitment of CBER scientists to support the development of products made using advanced and novel technologies.

Office of Vaccines Research and Review

  • Fluarix Quadrivalent (Influenza Virus Vaccine), for the prevention of influenza disease caused by influenza subtype A viruses and type B viruses in people 6 months of age and older. (January 11, 2018)
  • HEPLISAV-B (Hepatitis B Vaccine [Recombinant], Adjuvanted), for the prevention of infection caused by all known subtypes of hepatitis B virus in adults 18 through 70 years of age. (Nov. 9, 2017)
  • Shingrix, (Zoster Vaccine Recombinant, Adjuvanted), for the prevention of herpes zoster (shingles) in adults aged 50 years and older. (October 20, 2017)

Office of Tissues and Advanced Therapies

  • RECELL Autologous Cell Harvesting Device, indicated for the treatment of acute thermal burn wounds in patients 18 years of age and older. (Sept. 20, 2018)
  • JIVI (Antihemophilic Factor [Recombinant], PEGylated aucl, for use in previously treated adults and adolescents (12 years of age and older) with hemophilia A (congenital Factor VIII deficiency) for: 1) On-demand treatment and control of bleeding episodes; 2) Perioperative management of bleeding; 3) Routine prophylaxis to reduce the frequency of bleeding episodes. (August 29, 2018)
  • PANZYGA (immune globulin intravenous, human – ifas), indicated for the treatment of primary humoral immunodeficiency (PI) in patients two years of age and older, and for treatment of chronic immune thrombocytopenia. (Aug. 2, 2018)
  • Hematopoietic Progenitor Cells, Cord (HPC-C), indicated for use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution, in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. (June 21, 2018)
  • ANDEXXA (Coagulation factor Xa (Recombinant) inactivated-zhzo), indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. (May 3, 2018)
  • Plasma Cryoprecipitate (For Further Manufacturing Use) (May 3, 2018)
  • FIBRIN SEALANT (Human), indicated as an adjunct to hemostasis for mild to moderate bleeding in adults undergoing surgery when control of bleeding by standard surgical techniques (such as suture, ligature, and cautery) is ineffective or impractical. (Feb. 5, 2018)
  • LUXTURNA (voretigene neparvovec), an adeno-associated viral (AAV) vector gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). This approval was for the third gene therapy approved by CBER and the first directly administered gene therapy for a specific genetic disorder. (Dec. 19, 2017)
  • YESCARTA (axicabtagene ciloleucel), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or relapsed after, at least two other kinds of treatment. This is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma. (Oct. 18, 2017)

Office of Blood Research and Review

  • Imugen Babesia microti Arrayed Fluorescent Immunoassay (AFIA), the first donor screening tests for the detection of antibodies to Babesia microti (B. microti) in human plasma samples, and the Babesia microti Nucleic Acid Test (NAT), for the detection of B. microti DNA in human whole blood samples. Babesiosis parasites are transmitted by tick bites and by transfusion of blood or blood components from an infected donor. Because the tests could prevent the transmission of Babesia infection through blood transfusion, they were granted Priority Review status. (March 6, 2018)
  • cobas Zika Test (Nucleic Acid Test), the first donor screening test for the direct detection of Zika Virus RNA in human plasma from individual donors, including donors of whole blood, and blood components for transfusion, as well as other living donors, to prevent virus transmissions. (Oct. 5, 2017)
  • Albuminex (Human) 5% and 25%, indicated for adults and children for hypovolemia, ascites, hypoalbuminemia including from burns, acute nephrosis, acute respiratory distress syndrome, and cardipulmonary bypass.

Advancing Product Development & Ensuring Product Safety

CBER not only facilitates the development and availability of new medical products; through workshops, meetings, and conferences, it also shares and seeks important new knowledge about diseases and treatments and works to ensure the safety of products already on the market.

Center for Biologics Evaluation and Research

Office of Vaccines Research and Review

  • “Science and Regulation of Live Microbiome-Based Products Used to Prevent, Treat, or Cure Diseases in Humans” (Sept. 17, 2018)
    Public workshop in cooperation with the National Institute of Allergy and Infectious Diseases to exchange information with the scientific community about the clinical, manufacturing, and regulatory considerations associated with live microbiome-based products, when administered to prevent, treat, or cure diseases or conditions in humans.
  • Vaccines and Related Biological Products Advisory Committee (VRBPAC) meetings
    • Discussion of approaches to demonstrate effectiveness of group B streptococcus vaccines during pregnancy to protect newborn infants (May 1, 2018)
    • Selection of influenza virus strains for inclusion in the 2018/2019 influenza vaccine (March 1, 2018)
  • Consultation meeting at the World Health Organization on the composition of influenza vaccines for the Northern Hemispheredisclaimer icon (Geneva, Switzerland, Feb. 19-21, 2018).
  • Participation in WHO’s Global Advisory Committee for Vaccine Safety (GACVS). This committee provides independent, authoritative, scientific advice to WHO on vaccine safety issues of global or regional concern with the potential to affect in the short- or long-term national immunization programs. (June 2018)
  • Participated in ten meetings of the interagency collaboration Influenza (Flu) Risk Management Meeting (FRMM) between October 1, 2017 and August 31, 2018 to discuss:
    • seasonal influenza surveillance
    • influenza vaccine stockpiling program
    • H7N9 influenza virus surveillance
    • generation of candidate vaccine viruses
    • reagents for seasonal and pandemic influenza vaccines
    • influenza vaccine effectiveness studies
    • progress made in the Seasonal Influenza Vaccine Improvement (SIVI) program, an interagency collaboration to maintain the effectiveness of influenza vaccines despite the mutation of circulating viral strains.

Office of Tissues and Advanced Therapies

  • NIST-FDA Genome Editing Workshop on measurement and standards needs for genome editing, especially for therapeutic products. The goal was to clarify regulatory needs and challenges, and to inform the efforts of the recently launched NIST Genome Editing Consortium, regarding safety of genome editing and standardization of reporting. (April 23-24, 2018)
  • Convened the 21st U.S. - Japan Cellular and Gene Therapy Conference, “Neurodegenerative Diseases: Biology, Cellular and Gene Therapy” in collaboration with the Ministry of Education Culture, Sports, Science, and Technology, Japan. The goal of the conference was to exchange ideas on cutting edge and diverse areas of biomedical research and enhance opportunities for collaborations among scientists from the U.S. and Japan. CBER clarified U.S. regulatory approaches to the clinical development of cellular and gene therapies. (March 1, 2018)
  • NIST-FDA Flow Cytometry Workshop: Building Measurement Assurance in Flow Cytometry, which focused on identifying problems in clinical and manufacturing settings and exploring potential solutions and needs in standards development. (October 25, 2017)
  • Public workshop “Immune Globulin Potency in the 21st Century” to discuss new challenges for Industry to meet U.S. potency requirements for Immune Globulin (IG) products and to identify measures to address these challenges. The workshop included presentations and panel discussions by experts from academic institutions, industry, and government agencies. (Nov. 8-9, 2017)

Office of Blood Research and Review

Office of Biostatistics and Epidemiology

Active Surveillance Initiatives

Biological Effectiveness and Safety (BEST) Initiative

An expansion of the CBER program within the Sentinel Initiativedisclaimer icon to help build a national program for detecting in near real time, rare adverse events and for conducting safety assessments of biological products. BEST provides CBER with access to electronic health record (ERH) sources from >20 million patients for rapid safety and effectiveness studies of blood, advanced therapeutics, and vaccines. (See OBE section, 2017 Director’s Letter: award of two contracts).

  • Performed query studies (>200 simple, 18 medium, 15 complex) on blood components to identify transfusions and adverse events (AEs), such as transfusion-related acute lung injury.
  • Used new natural language processing (NLP) method to identify transfusion-associated circulatory overload (TACO) and post-transfusion sepsis cases; validated cases using ERH record review.
  • Demonstrated use of NLP to automate AE reporting to the FDA Adverse Event Reporting System (FAERS).
  • Biologics Effectiveness and Safety (BEST) Sentinel Initiative Industry Day
    Meeting with industry to review CBER requirements and needs to build additional capacity for: 1) biologics post-market, near real-time safety and effectiveness surveillance; 2) access to large sources of clinical patient-centered data, particularly de-identified electronic health records; 3) innovative approaches to conduct surveillance using sophisticated query tools, machine learning, artificial intelligence, and natural language processing. Contracts were awarded later in FY 2018. (Feb. 12, 2018)

Transfusion-Transmitted Infections Monitoring Systemdisclaimer icon (TTIMS)

TTIMS is a blood monitoring system to evaluate risks of HIV, hepatitis B, and hepatitis C in >60% of US blood supply.

  • Analyzed the first 30 months of donor testing data; conducted HIV recency testing (time elapsed before a measurable amount of antibody to, or genetic material of, the infectious organism is produced) and donor risk questionnaires.
  • Determined by interim analyses that no apparent increase in HIV prevalence or risk had occurred since the start of TTIMS.

Centers for Medicare & Medicaid Services collaboration

This product safety and effectiveness surveillance collaboration provides access to Medicare data for about 58 million mostly elderly people in the US.

  • Completed a study in over 13 million persons 65 years of age and older that showed cell-culture and high-doses of influenza vaccine were marginally more effective than egg-based quadrivalent influenza vaccines in preventing hospitalization and emergency room visits during the 2017-18 season.
    • The cell-culture vaccine was 10.7% more effective; the high-dose vaccine was 8.4% more effective
  • Completed a rapid surveillance of annual influenza vaccine and Guillain-Barre syndrome that found no increased risk during the 2017-18 influenza season.

Centers for Disease Control and Prevention and Vaccine Safety Datalink (VSD) collaboration

VSD is a collaborative project between CDC’s Immunization Safety Office and eight health care organizations that monitors vaccine safety and studies rare and serious adverse events following immunization among 10 million persons in the US.

Science of Patient Input Initiative (SPI)

SPI includes methods and approaches of systematically obtaining, analyzing, and using information that captures patients’ experiences, perspectives, needs, and priorities in support of the development and evaluation of medical products.

The Office of Biostatistics and Epidemiology expanded its support of FDA efforts to integrate patient input on treatment preferences and outcomes into medical product development and regulatory decision-making, through its Science of Patient Input Initiative.

  • Completed a Sentinel study assessing feasibility of identifying and characterizing a cohort of patients with hemophilia; used a mobile app to survey hemophilia patients.
  • Launched three patient preference studies for prospective CBER products in the following disease areas: osteoarthritis, sickle cell disease, and brittle diabetes. These studies will provide important insights into patient perspectives and into the use of patient preference information (PPI) for regulatory decision making. PPI can answer questions about which benefits and risks are most important to patients, and which benefit-risk trade-offs they find acceptable. These efforts support patient-focused drug development requirements under 21st Century Cures and similar commitments under PDUFA VI.
    • Sponsored or represented CBER in multiple inter-center initiatives and public workshops aimed at developing guidance documents for including patient experience data in submissions to FDA in order to inform regulatory decision making, as per the 21st Century Cures Act and PDUFA VI.
    • Workshop: “Advancing Use of Patient Preference Information as Scientific Evidence in Medical Product Evaluation”
    • Draft Guidance and Workshop: "Patient-Focused Drug Development: Collecting Comprehensive and Representative Input“
    • Presentations at multiple national and international meetings on incorporation of patient input into regulatory decision making.


  • Partnered with CDER to organize the FDA public workshop “Promoting the Use of Complex Innovative Designs in Clinical Trials.” This workshop discussed complex adaptive clinical trial designs, Bayesian and other innovative clinical trial designs, and clinical trial simulations for confirmatory trial design and planning. (March 20, 2018)
  • Partnered with FDA’s Center for Drug Evaluation and Research (CDER) to support a public workshop convened by the Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University, “Utilizing Innovative Statistical methods and Trial Designs in Rare Disease Settings.” Attendees discussed innovative statistical approaches to rare disease product development, such as using prior data from early phase trials to inform Phase 3 trial design and using patient registries. (March 19, 2018)
  • Partnered with CDER on launching a pilot program for reviewing Complex and Innovative Trial Design (CID) proposals, fulfilling a PDUFA VI commitment.

CBER Guidances for Industry

CBER releases guidance documents as part of its effort to inform stakeholders of the Center’s current interpretation of its regulations and ways that regulated industry can remain in compliance. The Center also collaborates with other FDA Centers on joint guidances that include issues with broader intra-agency regulatory implications. A selection of these guidances is noted below.

Office of Blood Research and Review

Office of Tissues and Advanced Therapies

CBER in cooperation with other centers (Selected examples)

Mission-Related Research

The research laboratories at CBER create new knowledge that is essential to the fulfillment of our mission of ensuring that products we regulate are safe, pure, potent, and effective—and that new products will be available to the public.


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