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Biologics Performance Detail FY 2012 OPA

Table of Contents FY 2012 OPA

Long Term Objective: Advance Biologics Safety and Effectiveness

 

Measure

FY

Target

Result

233201: Complete review and action on standard original PDUFA NDA/BLA submissions within 10 months of receipt. (Output)

2012

90%

Nov 30, 2013

2011

90%

Nov 30, 2012

2010

90%

Nov 30, 2011

2009

90%

100%

(Target Exceeded)

2008

90%

100%

(Target Exceeded)

2007

90%

100%
(Target Exceeded)

233202: Complete review and action on priority original PDUFA NDA/BLA submissions within 6 months of receipt. (Output)

2012

90%

Apr 30, 2013

2011

90%

Apr 30, 2012

2010

90%

Apr 30, 2011

2009

90%

75%

(Target Not Met)

2008

90%

100%
(Target Exceeded)

2007

90%

100%
(Target Exceeded)

233203: Complete review and action on standard PDUFA efficacy supplements within 10 months of receipt. (Output)

2012

90%

Nov 30, 2013

2011

90%

Nov 30, 2012

2010

90%

Nov 30, 2011

2009

90%

100%

(Target Exceeded)

2008

90%

100%

(Target Exceeded)

2007

90%

100%
(Target Exceeded)

233205: Complete review and action on complete blood bank and source plasma BLA submissions within 12 months after submission date. (Output)

2012

90%

Nov 30, 2013

2011

90%

Nov 30, 2012

2010

90%

Nov 30, 2011

2009

90%

100%

(Target Exceeded)

2008

90%

100%

(Target Exceeded)

2007

90%

100%
(Target Exceeded)

233206: Complete review and action on complete blood bank and source plasma BLA supplements within 12 months after submission date. (Output)

2012

90%

Nov 30, 2013

2011

90%

Nov 30, 2012

2010

90%

Nov 30, 2011

2009

90%

99%
(Target Exceeded)

2008

90%

100%

(Target Exceeded)

2007

90%

99%
(Target Exceeded)

 

Measure

Data Source

Data Validation

233201
233202
233203
233205
233206

CBER’s regulatory management systems

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). RMS-BLA is CBER’s VAX-based (Virtual Address eXtension), Oracle database 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 used to track all Investigational New Drug (IND) Applications, 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 the Document Control Center, the application review offices, RIMS, and several mechanisms are built into BIMS.  The Blood Logging and Tracking System (BLT) records and tracks various applications reviewed by the Office of Blood Research and Review (OBRR).  OBRR also has a New Drug Application (NDA) tracking system. Data retrieved from these systems are reviewed and validated by 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 Report (BPDR) (previously called error and accident report) enables CBER to evaluate and monitor establishments, provide field staff and establishments with trend analyses of the reported deviations and unexpected events, and assist CBER in responding appropriately to reported biological product deviations.

 


Measure

FY

Target

Result

234101: Increase manufacturing diversity and capacity for pandemic influenza vaccine production. (Output)

2012

Evaluate and compare new methods to determine the potency of influenza vaccines

Nov. 30, 2012

2011

Apply novel technologies including mass spectrometry, an analytical technique for the determination of the elemental composition of a molecule, to quantify the absolute amount of hemagglutinin, a substance that causes red blood cells to aggluntinate, in the reference standards that are used to determine influenza vaccine potency.

Nov 30, 2011

2010

Completed and evaluated the pilot vaccine adverse-effects program and participated in an international workshop on alternative methods to reduce, refine, and replace the use of animals in vaccine potency and safety testing.

All targets met.

2009

Started a pilot program to develop and evaluate new methods to detect possible adverse effects, both pre-specified and non-pre-specified, of newly licensed vaccines, including pandemic influenza vaccines, in large population databases. Participated in at least one international workshop or conference.

All targets met.

2008

Facilitated development and evaluation of one new pandemic influenza vaccine and one new trivalent vaccine; demonstrated an improved method for evaluating the safety, potency or immunogenicity of influenza vaccines; and participated in one international workshop.

All targets met

2007

Issued guidance on clinical data to support licensure of pandemic influenza vaccines; evaluated potency of five influenza vaccines; demonstrated methods for improved influenza manufacture. 

All targets met

 

Measure

Data Source

Data Validation

234101

CBER’s Office of Vaccines Research and Review; and CBER’s Medical Director for Emerging and Pandemic Threat Preparedness

The data are validated by the appropriate CBER offices and officials.

 

Measure

FY

Target

Result

234202: Number of registered domestic blood bank and biologics manufacturing inspections.   (Output)

2012

1,000

December, 2012

2011

1,000

December, 2011

2010

1,000

1,073

(Target Exceeded)

2009

870

1,001

(Target Exceeded)

2008

870

1,014
(Target Exceeded)

234203: Number of human tissue establishment inspections.   (Output)

2012

533

December, 2012

2011

533

December, 2011

2010

518

564

(Target Exceeded)

2009

380

434

(Target Exceeded)

2008

325

383
(Target Exceeded)

2007

325

427
(Target Exceeded)

 

Measure

Data Source

Data Validation

234202

234203 

Field Data Systems

ORA use the following two main information technology systems to track and verify field performance goal activities: Field Accomplishments and Compliance Tracking System (FACTS) and Operational and Administrative System Import Support (OASIS). FACTS include data on the number of inspections; field exams; sample collections; laboratory analyses; and the time spent on each. OASIS, which is coordinated with U.S. Customs and Border Protection, provides data on what FDA regulated products are being imported, as well as, where they are arriving. It also provides information on compliance actions related to imports. FDA is currently developing the Mission Accomplishment and Regulatory Compliance Services (MARCS) system. MARCS will incorporate the capabilities of these two field legacy systems and include additional functionality.

1.   Complete review and action on standard original PDUFA NDA and BLA submissions within 10 months of receipt.  (233201)

 

Context:  The Prescription Drug User Fee Act (PDUFA) authorizes the FDA to collect fees from the prescription drug and biologic drug industries to expedite the review of human drugs and biologics to shorten the time needed for these products to reach the market.  Standard original BLAs are license applications for biological products, not intended as therapies for serious or life-threatening diseases. In FY 2012, FDA continues to maintain the target for this goal, which meets the performance commitments in the HHS Secretary’s letter to Congressional leaders.   

 

Performance: FDA tracks PDUFA performance by year-of-receipt, which FDA calls the cohort year. Complete performance data are not available until the prescribed review time, i.e., 10 months after receipt, is expired.  In FY 2009, CBER exceeded its goal by completing review and action on 100 percent of 8 standard applications within 10 months of receipt and has met or exceeded this performance goal since 1994.  The FY 2010 performance data for this goal will not be available until November 2011.

 

2.      Complete review and act on priority original PDUFA NDA/BLA submissions within 6 months of receipt. (233202)

 

Context:  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 BLA will receive priority review if the product would be a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious or life-threatening disease.  In FY 2012, FDA continues to maintain the target for this goal, which meets the performance commitments in the HHS Secretary’s letter to Congressional leaders.   

 

Performance: FDA tracks PDUFA performance by year-of-receipt, which FDA calls the cohort year.  Complete performance data are not available until the prescribed review time, i.e., 6 months after receipt, is expired.  In FY 2009, CBER did not achieve its goal by completing review and action on 75 percent of 4 priority applications within 6 months of receipt.  The goal for one application was missed because critical new data were submitted by the sponsor near the PDUFA review deadline.  CBER decided to continue the review and to not issue a Complete Response letter to meet the PDUFA review deadline because of the public health importance of the vaccine. The FY 2010 performance data for this goal will not be available until April 2011.

 

3.      Complete review and action on standard PDUFA efficacy supplements within 10 months of receipt.  (233203)

 

Context:  PDUFA authorizes the FDA to collect fees from the prescription drug and biologic industries to expedite the review of human drugs and biologics to shorten the time needed for these products to reach the market.  An efficacy supplement is a change to an approved licensed product to modify the “approved effectiveness” of a product, such as, a new indication which normally requires clinical data.   In FY 2012, FDA continues to maintain the target for this goal, which meets the performance commitments in the HHS Secretary’s letter to Congressional leaders.   

 

Performance: FDA tracks PDUFA performance by year-of-receipt, which FDA calls the cohort year. Complete performance data are not available until the prescribed review time, i.e., 10 months after receipt, is expired.   In FY 2009, CBER exceeded its goal by completing review and action on 100 percent of 16 standard PDUFA efficacy supplements within 10 months of receipt.  CBER has met or exceeded most of these performance goals since 1994.  The FY 2010 performance data for this goal will not be available until November 2011.

4.      Complete review and action on complete blood bank and source plasma BLA submissions within 12 months after submission date. (233205)

 

Context:  For FY 2012, CBER maintains the goal of reviewing and acting upon complete blood bank and source plasma BLA submissions at 90% within 12 months after submission.  Since CBER receives only a few complete blood bank and source plasma submissions, the actual performance may be significantly different than the target. 

 

Performance:  CBER tracks performance by year-of-receipt, which FDA calls the cohort year.  Complete performance data are not available until the prescribed review time, i.e., 12 months after receipt, is expired.  In FY 2009, CBER exceeded its goal by reviewing and acting on 100 percent of 2 submissions within 12 months of receipt. The FY 2010 performance data for this goal will not be available until November 2011.

 

5.      Complete review and action on complete blood bank and source plasma BLA supplements within 12 months after submission date. (233206)

 

Context:  In FY 2012, CBER maintains the goal of reviewing and acting upon complete blood bank and source plasma BLA supplement submissions within 12 months after submission.  User fee resources are not available for blood bank and source plasma application review.

 

Performance:  CBER tracks performance by year-of-receipt, which FDA calls the cohort year.  Complete performance data are not available until the prescribed review time, i.e., 12 months after receipt, is expired.  In FY 2009, CBER exceeded its goal by reviewing and acting on 99 percent of 346 supplements within 12 months of receipt.  The FY 2010 performance data for this goal will not be available until November 2011.

 

6.      Increase manufacturing diversity and capacity for pandemic influenza vaccine production.  (234101)

 

Context: Influenza pandemics are explosive global events in which most, if not all, persons worldwide are at risk for infection and illness.  Pandemic influenza strains, such as avian or H1N1 influenza, can rapidly change.  Vaccines will need to be produced for pandemic influenza strains on a short notice; therefore, FDA needs to provide new and accelerated pathways to facilitate their rapid production and evaluation.   This goal changes on a yearly basis to ensure continued progress in preparation for a pandemic outbreak.  The FY 2012 pandemic preparedness target will be to evaluate and compare new influenza-vaccine potency methods.

 

Performance:  In FY 2010, CBER accomplished its targets for this goal. 

 

7.   Number of registered domestic blood bank and biologics manufacturing inspections.  (234202)

 

Context: FDA will enhance its risk-based compliance and enforcement activities by increasing inspections of registered manufacturers of biological products, which are essential for meeting national public health objectives.  These products involve complex manufacturing processes and are in limited supply in some cases.  Inspections for this performance goal are conducted to ensure compliance with current Good Manufacturing Practices (cGMPs) requirements and applicable standards, and to ensure the safety, purity and potency of biological products.  The biologics inventory includes blood establishments, plasma derivative manufacturing establishments, and vaccine manufacturing establishments, especially seasonal and pandemic influenza vaccines.  In FY 2010, the target was increased to 1,000 inspections to reflect historical accomplishments. In FY 2012, the target is being maintained at the FY 2011 level.

Performance:  In FY 2010, FDA exceeded this high risk inspection goal of 1,000 by inspecting 1,073 blood banks and biologics manufacturing establishments.

 

8.   Number of foreign and domestic human tissue establishment inspections.   (234203)

 

Context:  Beginning in FY 2006 as a result of new regulations, the human tissue inspection goal was created.  FDA’s responsibility for enforcing the new regulations and the need to quickly assess compliance makes tissues one of the 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 tissue inspections to determine if human tissues for transplantation are in compliance with FDA tissue regulations and to assure consumer protection from unsuitable tissue products and disease transmission which may endanger public health.  In FY 2009, FDA increased this goal by 55 additional tissue inspections, over the FY 2008 target, in order to cover more of the firms that registered as a result of the new regulations.  In FY 2010, the target was increased by 138 inspections to reflect the FY 2009 Appropriations. In FY 2011, the target is being increased by 15 inspections for a new target of 533 inspections.  In FY 2012, the target is being maintained at the FY 2011 level.

 

Performance:  In FY 2010, FDA exceeded the human tissue goal of 518 by conducting 564 inspections under new regulations.

 

Next page: Animal Drugs and Feeds Performance Detail FY 2012 OPA