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Biologics Performance Goals

<< Return to FY 2007 Budget Summary

 

Long Term Goal: Sustain access to safe and effective new products by providing rapid, transparent and predictable science-based review of marketing applications.
Measure FY Target Result

1. Complete review and action on standard original PDUFA NDA/BLA submissions within 10 months; and review and act on priority original PDUFA NDA/BLA submissions within 6 months of receipt. (13001)

Measure 1A: Percentage of Standard Applications within 10 Months. (Output)

2007 90% 6/08
2006 90% 6/07
2005 90% 6/06
2004 90% 100% of 6
2003 90% 100% of 4
2002 90% 100% of 6
Measure 1B: Percentage of Priority Applications within 6 Months. (Output) 2007 90% 4/08
2006 90% 4/07
2005 90% 4/06
2004 90% 100% of 1
2003 90% 100% of 4
2002 90% 100% of 3

2. Complete review and action on standard PDUFA efficacy supplements within 10 months; and review and act on priority PDUFA efficacy supplements within 6 months of receipt (13002)

Measure 2A: Percentage of Standard Efficacy Supplements within 10 Months. (Output)

2007 90% 6/08
2006 90% 6/07
2005 90% 6/06
2004 90% 100% of 7
2003 90% 100% of 13
2002 90% 83% of 7
Measure 2B: Percentage of Priority Efficacy Supplements within 6 Months: (Output) 2007 90% 4/08
2006 90% 4/07
2005 90% 4/06
2004 90% None Submitted
2003 90% 100% of 2
2002 90% 100% of 4

3. Complete review and action on complete blood bank and source plasma BLA submissions, and BLA supplements within 12 months after submission date. (13005)

Measure 3A: Percentage of BLA Submissions within 12 months. (Output)

2007 50% 11/08
2006 90% 11/07
2005 90% 11/06
2004 90% 100% of 1
2003 90% 100% of 5
2002 90% 100% of 5
Measure 3B: Percentage of BLA Supplements within 12 months. (Output) 2007 75% 11/08
2006 90% 11/07
2005 90% 11/06
2004 90% 100% of 542
2003 90% 100% of 530
2002 90% 99% of 469
Long Term Goal: Increase the number of safe and effective new products by increasing the predictability, efficiency and effectiveness of product development, including products for unmet medical and public health needs, emerging infectious diseases and counterterrorism.
Measure FY Target Result
4. Increase manufacturing diversity and capacity for pandemic influenza vaccine production through interacting with vaccine researchers and developers and issuing guidance and other documents and through global vaccine response coordination to facilitate the development and expedite the evaluation of cell-based technologies and dose-sparing approaches, such as the use of adjuvants. (13030) (Output) 2007 Issue one guidance or concept paper to facilitate development of non-egg-based influenza vaccines; evaluate the potency of monovalent influenza vaccines from at least three manufacturers by using quality system guidelines; demonstrate two new or improved methods for improved influenza vaccine manufacture; develop at least four influenza virus vaccine strains optimized for growth in non-egg culture systems by using quality systems guidelines. 11/08
2006 Develop a concept paper on clinical data needed to support license of new trivalent vaccines and of pandemic vaccines; draft a guidance on cell substrates to facilitate development of non-egg-based influenza vaccines; co-sponsor two workshops with WHO on pandemic vaccines. 11/07
2005 NA NA
Long Term Goal: Prevent harm from products by increasing the likelihood of detection and interception of substandard manufacturing processes and products.
Measure FY Target Result

5. Increase risk-based compliance and enforcement activities by inspecting the highest risk registered blood banks, source plasma operations and biologics manufacturing establishments to reduce the risk of product contamination; and by conducting human tissue inspections to enforce the new regulations. (13012)

Measure 5A: The number of inspections conducted of the highest-risk registered blood banks, source plasma operations and biologics manufacturing establishments. (Output)

2007 1,175 1/08
2006 1,128 1/07
2005 1,257 1,392
2004 1,319 1,444
2003 1,331 1,594
2002 1,331 1,419
Measure 5B: The number of human tissue inspections conducted to enforce the new regulations. (Output) 2007 325 1/08
2006 250 1/07
2005 NA NA
2004 NA NA
2003 NA NA
2002 NA NA
Data Source: CBER's Regulatory Management System and Field Data Systems.

Data Validation: The Center for Biologics Evaluation and Research (CBER) uses various databases to manage its diverse programs and to assess performance. The principal CBER database is the Regulatory Management System-Biologics License Application (RMS-BLA). The RMS-BLA is CBER's new VAX-based, Oracle database that is used to track all biologics license applications, and supplement submissions; provide information to facilitate the review process (product, application status, milestone tracking, facility, review committee, industry contacts, and other information); and produce a wide variety of management reports. The Regulatory Information Management Staff (RIMS) monitors and is responsible for maintaining data quality and integrity in RMS-BLA.

The Biologics Investigational New Drug Management System (BIMS) is CBER's VAX-based, Oracle database that is used to track all Investigational New Drug Applications (IND), Investigational Device Exemption (IDE), and Master Files (MF) submissions; provide product, application status, and other information to facilitate the review process; and produce a wide variety of management reports. There are numerous mechanisms established for quality control in Document Control Center , the application review offices, the Regulatory Information Management Staff, and several built into BIMS itself.

The Blood Logging and Tracking System (BLT) records and tracks the various applications reviewed by the Office of Blood Research and Review. The Office also has an NDA tracking system. The data retrieved from these systems are reviewed and validated by the RIMS and the application review offices. If errors are detected, they are corrected.

Federal regulations (21 CFR, Part 600.14 and 606.171) require reporting of deviations in the manufacture of biological products that affect the safety, purity, or potency of the product. The Biological Product Deviation Reports (BPDRs) (previously called error and accident reports) enable the Agency to evaluate and monitor establishments, to provide field staff and establishments with trend analyses of the reported deviations and unexpected events, and to respond appropriately to reported biological product deviations to protect the pubic health

The Biologics Program relies in the Office of Regulatory Affairs' Field Accomplishments and Tracking System (FACTS) to register and record biologics manufacturing establishment inspection and compliance data. FACTS versions 1 and 2 together will replace the several dozen applications that comprise the current Field Information System (FIS).

Cross Reference: These performance measures support HHS Strategic Goal 2.

 

Note about Baseline Data: In several years of the program, performance (Baseline Data) exceeds the projected performance goals. The PDUFA III goals were set forth in letters from the Secretary of Health and Human Services to Congressional Committee Chairmen. FDA developed these goals in consultation with the pharmaceutical and biological prescription drug industries. "NA" means the goal is not applicable in that fiscal year.

The PDUFA application-review performance goals measure time to first action, not final action. The term "complete review and action on" is understood to mean the issuance of a complete action letter after the complete review of a filed complete application. The action letter, if it is not an approval, will set forth in detail the specific deficiencies and, where appropriate, the actions necessary to place the application in condition for approval. The performance goals and this definition were developed in consultation with the industry and Congress, and are contained in the Secretary's commitment letter to the Chairman of the Energy and Commerce Committee of the House of Representatives, and the Chairman of the Labor and Human Resources Committee of the Senate. This definition enables to the Agency to approve only safe and effective products without having to issue not-approvable decisions on applications that are in some way not in condition for approval.

1. Complete review and action on standard original PDUFA NDA and BLA submissions within 10 months; and review and act on priority original PDUFA NDA/BLA submissions within 6 months of receipt. (13001)

  • Context of Goal: The Prescription Drug User Fee Act authorizes the FDA to collect fees from the prescription drug and biologic drug industries to expedite the review of human drugs and biologics so they can reach the market more quickly. Standard original BLAs are license applications for biological products, not intended as therapies for serious or life-threatening diseases. A priority BLA is a license application for a therapy to treat serious or life-threatening diseases.
  • Performance: CBER has met or exceeded these performance goals since 1994. These applications are tracked by year of receipt, which is the cohort year. The cohort-year review performance is not available until the prescribed review time, i.e., 10 months after receipt, is expired. The FY 05 performance data for standard applications will be available November 2006. In FY 2004, CBER exceeded its goal by completing review and action on 100% of 6 Standard applications within 10 months, and reviewing and acting on 100% of 1 Priority application within 6 months.

2. Complete review and action on standard PDUFA efficacy supplements within 10 months; and review and act on priority PDUFA efficacy supplements within 6 months of receipt. (13002)

  • Context of Goal: The PDUFA authorizes the FDA to collect fees from the prescription drug and biologic drug industries to expedite the review of human drugs and biologics so they can reach the market more quickly. A supplement is a change to an approved licensed product. An efficacy supplement provides information to FDA to modify the "approved effectiveness" in the labeling of a product such as a new indication, and normally includes clinical data.
  • Performance: CBER has met or exceeded most of these performance goals since 1994. The cohort-year review performance is not available until the prescribed review time, i.e.,10 months after receipt, is expired. The FY 05 performance data for standard efficacy supplements will be available June 2006. In FY 2004, CBER exceeded its goal by completing review and action on 100% of 7 Standard PDUFA efficacy supplements within 10 months.

3. Complete review and action on complete blood bank and source plasma BLA submissions, and BLA supplements within 12 months after submission date. (13005)

  • Context of Goal: In FY 2007, CBER has reduced the targets for this goal from 90% to 50% for blood bank and source plasma BLA submissions, and from 90% to 75% for BLA supplements. As a result of strategic redeployment, the target for this goal needed to be reduced.
  • Performance: These applications are tracked by year of receipt, which is the cohort year. The FY 05 performance data for supplements will be available November 2006. In FY 2004, CBER exceeded its goal by reviewing and acting on 100% of 1 complete submission within 12 months, and reviewing and acting on 100% of 542 supplements within 12 months after submission date.

4. Increase manufacturing diversity and capacity for pandemic influenza vaccine production through interacting with vaccine researchers and developers and issuing guidance and other documents and through global vaccine response coordination to facilitate the development and expedite the evaluation of cell-based technologies and dose-sparing approaches, such as the use of adjuvants. (13030)

  • Context of Goal: The Biologics Program has received appropriated funding to establish the infrastructure and surge capability to react to a potential disease pandemic. Influenza pandemics are explosive global events in which most, if not all, persons worldwide are at risk for infection and illness. Unlike the gradual changes that occur in the influenza viruses that appear each year during "flu season," a pandemic influenza virus represents a major, sudden shift in the virus' structure that increases its ability to cause illness in a large proportion of the population. During previous influenza pandemics, large numbers of people fell ill, sought medical care, were hospitalized and died. Because the current vaccines do not contain strains that will protect against such a pandemic, pandemic strain-specific-vaccines must be produced, likely on short notice. FDA has provided new and accelerated pathways to facilitate their rapid production and evaluation, and is working with partners to facilitate new technologies that could increase manufacturing flexibility and capacity. The Biologics Program pandemic prevention activities include:
    • Working with HHS to develop the operational implementation of the pandemic plan policies on vaccines;
    • Working with CDC, NIH, WHO and industry to facilitate availability of vaccines suitable for the H5N1 avian influenza viruses that continue to circulate in Asia;
    • Working with sponsors of INDs to facilitate the initiation of clinical trials through the review process as well as informal guidance as necessary;
    • Working with WHO and others to promote global harmonization and cooperation in pandemic vaccine development;
    • Involved with several groups, (CDC, academia, etc) to perform additional studies to prepare strains for influenza A virus subtypes with pandemic potential;
    • Perform serologic testing to determine whether current vaccines produce antibodies that can inhibit the new influenza viruses considered for use in vaccines. Similar work would be needed to prepare for pandemic situations that might arise from, for example, the continued circulation of avian influenza viruses in Asia; and
    • Produce, calibrate, and distribute reagents to be used in determining the potency of vaccines. For each new virus included in vaccine, the reagents include a virus-specific preparation of influenza antigens and a virus -specific antiserum. CBER also provides the antiserum to CDC and WHO for national and international surveillance of influenza viruses. Work has been done to prepare some reagents for influenza A subtypes H5 and H9. Because the identity of an influenza strain that may emerge into a pandemic cannot be predicted, additional reagents will need to be prepared.
  • Performance: FDA's role is critical in assuring that we have the needed tools to prepare for emerging infectious disease threats, such as safe and effective vaccines against pandemic flu. FDA will implement an enhanced and sustained preparedness effort to:
    • Collaborate with the private sector, CDC and NIAID to rapidly prepare and test pandemic influenza vaccine virus seed strains and provide related reagents to assure potency, effectiveness, safety and efficient manufacturing for all recognized pandemic threat strains;
    • Allow rapid review of new manufacturers, facilities and products, and use new vaccine technologies to facilitate HHS preparedness efforts, including HHS and NIH Requests for Proposals (RFPs) and contracts;
    • Advise national and international public health groups such as WHO, CDC, NIH and the National Vaccine Program Office in selecting new influenza viruses for vaccine manufacturing and to prepare for pandemic influenza; and,
    • Promote global regulatory and industrial cooperation, information sharing, guidance, harmonization and quality in pandemic vaccine development and manufacturing efforts.

5. Increase risk based compliance and enforcement by inspecting the highest risk registered domestic blood banks, source plasma operations and biologics manufacturing establishments to reduce the risk of product contamination; and, by conducting human tissue inspections to enforce the new regulations. (13012)

  • Context of Goal: Inspections for this goal are conducted to ensure compliance with Current Good Manufacturing Practices (CGMPs), and to ensure purity of biological products. There are currently an estimated 2,450 establishments in the Biologics program inventory covered under this regulation. The biologics inventory includes high-risk establishments such as blood collection facilities, plasma fractionator establishments, and vaccine manufacturing establishments.

    Beginning in FY 2006, the human tissue inspections have been added to this goal because they are of high priority due to the potential for associated adverse health events. FDA's responsibility for enforcing the new regulations and the need to quickly assess compliance makes tissues one of our highest priorities. Two new rules took effect regarding human tissue: one requiring tissue facilities to register with FDA became effective January 2004; while the "Donor Eligibility Rule" became effective May 2005.

    The field conducts establishment inspections and investigations to determine if human tissues for transplantation are in compliance with the tissue regulations. FDA determines if establishments are properly testing and screening tissue donors, and evaluates whether establishments are properly recovering tissues from donors as well as properly storing and transporting the tissues. Monitoring the recovery and processing of human tissue and the testing and screening of donors is critical to assure consumer protection from unsuitable tissue products and disease transmission which may endanger public health.

    Many of these firms are relatively new, small, unaware of the specifications of the new regulations, and have never been inspected previously. There are about 2,000 human tissue establishments currently registered.

  • Performance: In FY 2005, FDA exceeded this goal of 1,257 by inspecting 1,392 blood banks, source plasma and biologics manufacturing establishments.

 

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