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2.4.1 Program Description, Context and Summary of Performance

The mission of the Biologics Program is to ensure the safety, purity, potency, and effectiveness of biological products (primarily vaccines and blood products) for the prevention, diagnosis, and treatment of disease or injury. The products that the Biologics Program regulates are on the leading edge of technology. Rapid scientific advances in biochemistry, molecular biology, cell biology, immunology, genetics, and information technology are transforming drug discovery and development, paving the way for unprecedented progress in developing new medicines to conquer disease.

The number of Investigational New Drug Applications (INDs) and Investigational Device Exemptions (IDEs) received by the Biologics Program has increased 31% from FY 1996 to FY 2001. INDs and IDEs are an indication of future license application workload. Sponsors submit INDs/IDEs prior to beginning clinical trials to determine the safety and efficacy of the product in humans.

While scientific advances of new biological products promise great health benefits for U. S. consumers, FDA must ensure that these products are safe. FDA is also responsible for ensuring the safety of the nation's blood supply by minimizing the risk of infectious disease transmission and other hazards, while facilitating the maintenance of an adequate supply of whole blood and blood products. These challenges are represented by the Program's two strategic goals for the 21st century:

• Ensure the expeditious availability of safe and effective biologics, for the prevention, diagnosis, and treatment of disease or injury.
• Reduce the risk of biologics products on the market through assuring product quality and correcting problems associated with their production and use.

FDA is responsible for ensuring that vaccines and related products (such as botulinum toxin, skin test reagents for tuberculosis, and allergenic products) are safe and effective and adequately labeled. Vaccines against diseases such as Hepatitis B, polio, Haemophilus influenzae type b, mumps, measles, rubella, diphtheria, tetanus, pertussis, and chicken pox are recommended for all U.S. children, and vaccines against influenza and pneumococcal infections are recommended for all adults more than 65 years of age. Periodic tetanus and diphtheria booster vaccinations are recommended for all adults. The use of influenza vaccine among adults has, in recent years, increased markedly (to a current use of about 80 million doses/year). Additional vaccines are recommended for special groups (for example, persons with Hepatitis A) or for travelers to particular areas of the world (for example, Salmonella typhi or Japanese encephalitis virus vaccines). Many additional vaccines are in various stages of investigation (for example, HIV or Herpes simplex virus vaccines), and their INDs are being reviewed.

2.4.2 Strategic Goals

Strategic Goal Component 1:
Strategic Goal 1: Ensure the expeditious availability of safe and effective biologics, for the prevention, diagnosis, and treatment of disease or injury.

A. Strategic Goal Explanation

The FDA is responsible for reviewing and approving biologics covered under the Prescription Drug User Fee Act (PDUFA). These products are primarily vaccines and therapeutics. FDA is also responsible for reviewing and approving biologic products not covered by PDUFA. The non-PDUFA biological products are primarily blood and blood products, biotechnology-derived hematologics, allergenic products, and devices associated with their manufacture and use.

To provide the U.S. public with quicker access to new biologics, FDA consults closely with product sponsors early in product development and makes prompt decisions on important new biological product applications. FDA will continue to make timely decisions in reviewing PDUFA product license applications (PLAs), Biologic License Applications (BLAs), and New Drug Applications (NDAs) and their supplements (performance goals 13001-13004). FDA will also continue to make timely decisions in reviewing non-PDUFA biologics, primarily blood and plasma products (performance goal 13005).

FDA is in the process of establishing a comprehensive new system through the tissue action plan to regulate human cells, tissues, and cellular and tissue-based products. The goal of the new approach, published in the Federal Register on February 28, 1997, is to improve protection of the public health without imposing unnecessary restrictions on research and development, or on the availability of new products. This system is expected to lead to increasing the safety of transplanted human cells, tissues, and cellular and tissue-based products, while encouraging the development of new products.

PDUFA Products: FDA worked with various stakeholders, including representatives from consumer, patient, health provider groups, and the pharmaceutical and biological prescription drug industries, to develop a reauthorization proposal for PDUFA that would build upon and enhance the success of the program. Title 5, Subtitle A, of the Public Health Security and Bioterrorism Preparedness and Response act of 2002 (Public Law #107-188) was enacted on June 12, 2002. The Act extends PDUFA through 2007.

The PDUFA authorized revenues from fees paid by the pharmaceutical industry to expedite review by the FDA of human drug applications, including biologics.

Fees that FDA collects from drug and biologic firms are used to reduce the evaluation time for certain human drug, including biologics, applications without compromising review quality. FDA primarily spent these PDUFA funds to hire personnel to review applications and update the information technology (IT) infrastructure supporting the review process. PDUFA provides FDA with the resources necessary to sustain the larger application review staff. It also provides FDA with additional funds to acquire the resources needed to achieve the more stringent performance goals.

The PDUFA time frames and performance goals are the result of in-depth negotiations between the drug industry and FDA. Industry and FDA determined that both the time frames and the percentage goals are realistic, achievable with the additional user fee resources, and desirable. The PDUFA time frames for drug applications differ in some cases from the Food, Drug and Cosmetic Act (FD&C) statutory requirements. Biologics applications are covered by the Public Health Service Act, which does not have any statutory time frames. Industry is pleased with the certainty of timely action and response from the FDA review process, and the net result of a higher percentage of applications being approved faster. Patients benefit by having more therapies available more quickly. Performance goals for PDUFA applications are based on the PDUFA time frames. Some of the more stringent PDUFA goals were phased in over several years.

Non-PDUFA Products: The Biologics Program also reviews and approves license applications for products not covered by PDUFA. The mission of the Blood Program is to ensure that blood, blood products, biotechnology-derived hematologics, and devices associated with their manufacture and use, are safe, effective, and adequately labeled.

The blood supply is critical to the nation's health care system, and the United States has the safest blood supply in the world. Each year approximately 14 million blood units are drawn from volunteer donors for use in more than 3.5 million Americans. FDA vigorously continues to strengthen its efforts to protect the nation's blood supply and to minimize any risk to patients of acquiring the human immunodeficiency virus (HIV), hepatitis, Creutzfeldt-Jakob disease (CJD), and other blood-borne diseases.

Factors which affect the Agency's ability to achieve the performance goals are: the quality and complexity of applications, the number of applications received, and commitments which take researchers/reviewers away from their assigned review work, such as regulation/guidance writing.

B.   Summary of Performance Goals

Performance Goals	Targets	Actual Performance	Reference
1. Complete review and action on 90% of standard original PDUFA NDA/PLA/BLA submissions within 10 months; and review and act on 90% of priority original PDUFA NDA/PLA/BLA submissions within 6 months of receipt. (13001)	Standard Applications within 12 months: FY 04: NA FY 03: NA FY 02: NA FY 01: 90% FY 00: 90% FY 99: 90% Standard Applications within 10 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 70% FY 00: 50% FY 99: 30% Priority Applications within 6 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 90% FY 00: 90% FY 99: 90%	Standard Applications within 12 months: FY 04: FY 03: FY 02: FY 01: 100% of 5 FY 00: 100% of 10 FY 99: 100% of 5 Standard Applications within 10 months: FY 04: FY 03: FY 02: 09/03 FY 01: 100% of 5 FY 00: 100% of 10 FY 99: 100% of 5 Priority Applications within 6 months: FY 04: FY 03: FY 02: 04/03 FY 01: 100% of 3 FY 00: 100% of 4 FY 99: 100% of 1	4
2. Complete review and action on 90% of standard PDUFA efficacy supplements within 10 months; and review and act on 90% of priority PDUFA efficacy supplements within 6 months of receipt. (13002)	Standard Applications within 12 months: FY 04: NA FY 03: NA FY 02: NA FY 01: 90% FY 00: 90% FY 99: 90% Standard Applications within 10 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 70% FY 00: 50% FY 99: 30% Priority Applications within 6 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 90% FY 00: 90% FY 99: 90%	Standard Applications within 12 months: FY 04: FY 03: FY 02: FY 01: 100% of 14 FY 00: 100% of 11 FY 99: 100% of 8 Standard Applications within 10 months: FY 04: FY 03: FY 02: 09/03 FY 01: 100% of 14 FY 00: 100% of 11 FY 99: 100% of 8 Priority Applications within 6 months: FY 04: FY 03: FY 02: 05/03 FY 01: 100% of 2 FY 00: 100% of 2 FY 99: 100% of 2	4
3. Complete review and action on 90% of PDUFA manufacturing supplements within 6 months of receipt, and review and act on 90% of PDUFA manufacturing supplements requiring prior approval within 4 months of receipt. (13003)	Within 6 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 90% FY 00: 90% FY 99: 90% Within 4 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 70% FY 00: 50% FY 99: 30%	Within 6 months: FY 04: FY 03: FY 02: 05/03 FY 01: 94% of 410 FY 00: 97% of 349 FY 99: 96% of 218 Within 4 months: FY 04: FY 03: FY 02: 03/03 FY 01: 95% of 186 FY 00: 92% of 241 FY 99: 93% of 259	4
4. Complete review and action on 90% of Class 1 resubmitted original PDUFA applications within 2 months; and review and act on 90% of Class 2 resubmitted original PDUFA applications within 6 months of receipt. (13004)	Class 1 resubmissions within 2 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 70% FY 00: 50% FY 99: 50% Class 2 resubmissions within 6 months: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 90% FY 00: 90% FY 99: 90%	Class 1 resubmissions within 2 months: FY 04: FY 03: FY 02: 100% of 2 FY 01: 100% of 6 FY 00: 100% of 1 FY 99: 100% of 2 Class 2 resubmissions within 6 months: FY 04: FY 03: FY 02: 05/03 FY 01: 100% of 10 FY 00: 100% of 8 FY 99: 100% of 12	4
5. Complete review and action on 90% of complete blood bank and source plasma PLA/BLA submissions, and 90% of PLA/BLA supplements within 12 months after submission date. (13005)	Complete Submissions: FY 04: 90% FY 03: 90% FY 02: 90% FY 01: 90% FY 00: 85% FY 99: 60% Supplements: FY 04 90% FY 03: 90% FY 02: 90% FY 01: 90% FY 00: 90% FY 99: 90%	Complete Submissions: FY 04: FY 03: FY 02: 11/03 FY 01: 100% of 7 FY 00: 100% of 12 FY 99: 100% of 10 Supplements: FY 04: FY FY 02: 11/03 FY 01: 99% of 417 FY 00: 100% of 559 FY 99: 99% of 780	4
6. Facilitate the availability of safe and effective biological products to prevent, diagnose, and treat sicknesses or injuries associated with a terrorist attack. (13019)	FY 04: Issue guidance document regarding recommendations for deferral of blood donors who may have been exposed to smallpox. FY 03: Issue guidance document regarding recommendations for deferral of blood donors vaccinated for smallpox.Review and approve a supplement to the license application for use of a 1 to 5 dilution of Dryvax, the FDA licensed smallpox vaccine, to expand the total number of available smallpox vaccine doses. FY 02: NA	FY 04: FY 03: FY 02: NA	2, 4
TOTAL FUNDING: ($ 000)	FY 04: 134,427 FY 03: 160,550 FY 02: 140,731 FY 01: 114,849 FY 00: 111,968	Numbers in the Reference column corresponds to the relevant strategic goal in the HHS Strategic Plan

C.   Goal-By-Goal Presentation of Performance

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 90% of standard original PDUFA NDA, PLA, and BLA submissions within 10 months; and review and act on 90% of priority original PDUFA NDA/PLA/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 PLAs or BLAs, are license applications for biological products, not intended as therapies for serious or life-threatening diseases. A priority PLA/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., 12 months after receipt, is expired. The FY 2002 data for standard applications within 12 months will be available after November 2003. The FY 2002 data for standard applications within 10 months will be available after September 2003.
Data Sources: CBER's Regulatory Management System

2. Complete review and action on 90% of standard PDUFA efficacy supplements within 10 months; and review and act on 90% of 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 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., 12 months after receipt, is expired. The FY 2002 data for standard applications within 12 months will be available after November 2003. The FY 2002 data for standard applications within 10 months will be available after September 2003
Data Sources: CBER's Regulatory Management System

3. Complete review and action on 90% of PDUFA manufacturing supplements within 6 months of receipt, and review and act on 90% of PDUFA manufacturing supplements requiring prior approval within 4 months of receipt. (13003)
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. A manufacturing supplement provides FDA information relating to a proposed expiration date change, formulation revision, manufacturing process change, packaging change, or controls change.
Performance: CBER has met or exceeded these performance goals since 1994. These applications are tracked by year of receipt, which is the cohort year.
Data Sources:CBER's Regulatory Management System

4. Complete review and action on 90% of Class 1 resubmitted original PDUFA applications within 2 months; and review and act on 90% of Class 2 resubmitted original PDUFA applications within 6 months of receipt. (13004)
Context of Goal: 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 resubmitted original application is a complete response to an action letter addressing all identified application deficiencies. Class 1 resubmitted applications are applications resubmitted after a complete response letter that include one or more of the following items: final printed labeling; draft labeling; safety updates; stability updates; commitments to perform Phase IV (postmarketing) studies; assay validation data; final release testing; a minor re-analysis of data; other minor clarifying information; or other specific information requested by the Agency. Class 2 resubmissions include any other items.
Performance: These applications are tracked by year of receipt, which is the cohort year. FDA's FY 2002 performance for review of Class 1 resubmissions within 2 months was 100%.
Data Sources: CBER's Regulatory Management System

5. Complete review and action on 90% of complete blood bank and source plasma PLA/BLA submissions, and 90% of PLA/BLA supplements within 12 months after submission date. (13005)
Context of Goal: Blood bank and source plasma applications are not covered by PDUFA. The non-PDUFA review resources in CBER are not protected from cuts as the PDUFA resources are by the PDUFA legislation. CBER's non-PDUFA review resources have been cut in recent years to meet unfunded pay raises, increased current service costs, and other budget actions.
Performance: 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., 12 months after receipt, is expired. The FY 2002 data for review of complete submissions and for major supplements will be available after November 2003.
Data Sources: CBER's Regulatory Management System

6. Facilitate the availability of safe and effective biological products to prevent, diagnose, and treat sicknesses or injuries associated with a terrorist attack. (13019)
Context of Goal: An essential element of the Counterterrorism initiative includes the expeditious development and licensing of biological products to diagnose, treat or prevent outbreaks from exposure to the pathogens that have been identified as bioterrorist agents. These products must be reviewed and approved prior to the large-scale productions necessary to create and maintain a stockpile. Staff must guide the products through the regulatory process, including the manufacturing process, pre-clinical testing, clinical trials, and the licensing and approval process. Experts in these areas must expedite the licensing and approval process for these products. Pathogens that have been identified as potential biological warfare agents include those that cause smallpox and anthrax. CBER is responsible for the review of biologic products including vaccines, blood and blood products, as well as therapeutic biologic products.
Guidance documents assist the Center staff and regulated industry in identifying issues and areas where more specific direction can be outlined and thus contribute to expediting the review process. Issues that need to be explored with respect to the safety and availability of blood and blood products include viral testing and removal in blood, blood products, and plasma derivatives in the event that exposed individuals donate prior to detection; and, the impact on the safety of the blood supply from donations by individuals vaccinated with live vaccines. Identifying areas in the regulatory process where more specific direction regarding requirements is needed facilitates the review process.
Performance: New goal/not available
Data Sources: CBER's Regulatory Management System and the Regulations and Policy Staff

Strategic Goal 2:
Reduce the risk of biologics products on the market through assuring product quality and correcting problems associated with their production and use.

A. Strategic Goal Explanation

FDA is required by law to conduct biennial inspections of all licensed establishments to determine compliance with Current Good Manufacturing Practice (CGMP) regulations and to ensure compliance with applicable product and establishment standards and license commitments. In addition, FDA inspects all manufacturing facilities, which are unlicensed and/or under contract to a licensed establishment. FDA conducts biomedical research inspections to review pivotal clinical trial data, and in inspections of new tissue-cellular based products. By accomplishing the performance goal 13012, the Biologics Program will ensure that biologics establishments are in compliance with regulations and that the products produced in those establishments are safe and pure.

Factors, which affect the FDA's ability to achieve the performance goals, are unanticipated crises such as product tampering, which require immediate investigative and enforcement actions and take inspectors/investigators away from their planned assignments.

The availability of qualified scientific personnel to review, evaluate and investigate postmarket adverse events affects the Agency's ability to make sound and timely decisions concerning recalls and withdrawals.

B.   Summary of Performance Goals

C.   Goal-By-Goal Presentation of Performance

7. Meet the biennial inspection statutory requirement by inspecting 50% of the approximately 2,700 registered blood banks, source plasma operations and biologics manufacturing establishments to reduce the risk of product contamination. (13012)
   Context of Goal: This includes inspections done by FDA directly, or through state contracts or partnership agreements. The law requires FDA to conduct inspections of certain manufacturing facilities once every two years. The inspections are conducted to ensure compliance with Current Good Manufacturing Practices (CGMPs), and ensure the purity of the biological products. There are currently 2,693 establishments in the Biologics Program inventory covered under this statute. The establishments include high-risk establishments such as blood collection facilities, plasma fractionator establishments and vaccine manufacturing establishments. There are 1,665 additional establishments in the Biologics Program inventory not covered under this statute.
   Performance: In FY 2002, FDA inspected 52% of the establishments in the Official Establishment Inventory, exceeding the goal of 50%. The drop in inspection coverage from 64% in FY 1999 to 52% in FY 2002 is attributed to changes in risk priorities. Some resources were re-allocated to other high-priority areas such as tissues. Due to a few problems resulting from the transition to a new database (FIS to FACTS) in FY 2000, some adjustments in counting the inventory and inspectional coverage were necessary.
   Data Sources: Program-Oriented Data System, Official Establishment Inventory.

Contact Information:
Planning Staff, Office of Planning, FDA
Phone: 301-827-5210

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