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Human Drugs Performance Goals 

<< Return to FY 2007 Budget Summary

 

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

1. Improve the efficiency and effectiveness of the new drug review program to ensure a safe and effective drug supply is available. (12001) (Output)

(Formerly: Ensure a safe and effective drug supply is available to the public.)

Measure 1A: Percentage of Standard NDAs within 10 Months.

2007 90% 10/08
2006 90% 10/07
2005 90% 10/06
2004 90% 97% of 94
2003 90% 100% of 82
2002 90% 99% of 84
Measure 1B: Percentage of Priority NDAs within 6 Months. (Output) 2007 90% 10/08
2006 90% 10/07
2005 90% 10/06
2004 90% 96% of 28
2003 90% 100% of 19
2002 90% 100% of 12

2. Increase the number of drugs that are adequately labeled for children and ensure the surveillance of adverse events in the pediatric population. (12026) (Output)

Measure: Number of written requests (WRs) issued for drugs that need to be studied in the pediatric population and number of drugs reported to the pediatric advisory committee on adverse events for drugs that receive pediatric exclusivity.

2007 7/7 1/08
2006 8/8 1/07
2005 8/7 12/14
2004 NA NA
2003 NA NA
2002 NA NA

3. Improve the efficiency and effectiveness of the generic drug review program to ensure safer and more effective generic drug products are available for Americans. (12003) (Outcome)

(Formerly: Ensure safe and effective generic drugs are available to the public.)

Measure: Number of months of the average FDA time to approval or tentative approval for the fastest 25% of original generic drugs application.

2007 Fastest 25% by .5 mos 1/08
2006 Fastest 25% by .5 mos 1/07
FY 05 Measure: Complete review and action upon fileable original generic drug applications within 6 months after submission date. 2005 90% 6/06
2004 85% 87% of 543
2003 80% 90% of 423
2002 65% 85% of 339

4. Improve the efficiency and effectiveness of the over-the-counter (OTC) drug review program to ensure a safe and effective drug supply is available. (12048) (Output)

Measure: Percentage of Rx-to-OTC Switch applications within 10 months of receipt in which there was complete review and action. Number of OTC monographs in which there was significant progress on completion.

2007 100%/5 1/08
2006 100%/6 1/07
2005 100%/6 100%/17
2004 100%/6 100%/8
2003 NA NA
2002 NA NA

5. Reduce time to marketing approval for new drugs and biologics.

Measure: Reduction in FDA approval time for the fastest 50 percent of priority New Molecular Entities/ Biologics Licensing Applications approved, using the 3-year submission cohort for FY 2005-2007.

* The reported results represent a three year average calculated using cohort data from the reported year and the two prior years.

2007 514 days 09/09
2006 NA 09/08
2005 NA 09/07
2004 NA 09/06
2003 NA 523 days*
2002 NA 520 days*
2001 NA 575 days*

6. Reduce the time to marketing approval or tentative approval for safe and effective new generic drugs.

Measure: Reduction in FDA time to approval or tentative approval for the fastest 70 percent of original generic drug applications approved or tentatively approved of those submitted using the three year submission cohort for FY 2005 - 2007.

* The reported results represent a three year average calculated using cohort data from the reported year and the two prior years.

2007 16.4 months 02/10
2006 NA 02/09
2005 NA 02/08
2004 NA 02/07
2003 NA 02/06
2002 NA 16.2 months*
2001 NA 17.6 months*
2000 NA 17.9 months*

Data Source: Review performance monitoring is being done in terms of cohorts, e.g., FY 2003 cohort includes applications received from October 1, 2002, through September 30, 2003. CDER uses the Center-wide Oracle Management Information System (COMIS) and New Drug Evaluation/Management Information System (NDE/MIS). FDA has a quality control process in place to ensure the reliability of the performance data in COMIS.

The Pediatric Exclusivity Database tracks all data regarding pediatric exclusivity as mandated by FDAMA and reauthorized by BCPA. Specifically, this database tracks the number of WRs issued and the number of products for which pediatric studies have been submitted and for which exclusivity determinations have been made. The Pediatric Page database captures all information regarding waivers, deferrals, and completed studies for applications that are subject to the Pediatric Research Equity Act.

Published monographs that establish acceptable ingredients, doses, formulations, and consumer labeling for OTC drugs.

Data Validation: The Center-wide ORACLE Management Information System (COMIS) is CDER's enterprise-wide system for supporting premarket and postmarket regulatory activities. COMIS is the core database upon which most mission-critical applications are dependent. The type of information tracked in COMIS includes status, type of document, review assignments, status for all assigned reviewers, and other pertinent comments. CDER has in place a quality control process for ensuring the reliability of the performance data in COMIS. Document room task leaders conduct one hundred percent daily quality control of all incoming data done by their IND and NDA technicians. Senior task leaders then conduct a random quality control check of the entered data in COMIS. The task leader then validates that all data entered into COMIS are correct and crosschecks the information with the original document.

CDER uses the Pediatric Exclusivity database and the Pediatric Research Equity Act Tracking System (PREATS) to track information such as number of written requests issued and the number of products for which pediatric studies have been submitted and for which exclusivity determinations have been made as well as information related to the PREA legislation.

Cross Reference: These performance measures support HHS Strategic Goal 2.
Long Term Goal: Increase capability to efficiently and cost-effectively maintain an information technology (IT) environment to support FDA business goals.
Measure FY Target Result

7. Create state-of-the-art information management systems and practices to move to a paperless environment (e-Government). (12051) (efficiency)

Measure: Percentage of ANDAs that contain some electronic portion.

2007
NA
NA
2006 NA NA
2005 35% 93%
2004 30% 72.5%
2003 NA NA
2002 NA NA
Data Source: The CDER Electronic Document Room. This is an Efficiency Goal.
Data Validation: CDER has instituted multiple layers of verification and validation for ensuring the accuracy of performance information. CDER relies on data extracted from information systems to support demonstrating performance toward most performance goals and targets. CDER has developed manuals of policies and procedures (MaPPs) or other standard operating procedures for using or entering data into information systems. There are quality controls built in to the information systems -controls that help ensure the integrity and accuracy of the data entered. CDER has a number of analysts who have expertise in extracting information from these systems. Their knowledge and experience working with the data, and their familiarity and experience with the business of the Center provide another layer of validation. Further, the Center requires a multi-level clearance process for verifying and validating the accuracy of the information provided in the annual performance report.
Cross Reference: This performance measure supports HHS Strategic Goal 8.
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

8. Enhance the protection of the American public against the effects of terrorist agents by facilitating the development of and access to medical countermeasures, providing follow-up assessments on therapies, and engaging in emergency preparedness and response activities. (12045) (Output)

(Formerly: Facilitate development and availability of medical countermeasures to limit the effects of the intentional use of biological, chemical, or radiologic/nuclear agents.)

Measure: Number of medical countermeasures in which there has been coordination and facilitation in development.

2007 5 1/08
2006 5 1/07
2005 5 11
2004 NA NA
2003 NA NA
2002 NA NA
Data Source: CDC/DHS Strategic National Stockpile (SNS) program, database from Department of Energy/REAC/TS (Oakridge), published guidance for Industry, published Federal Register Notices, www.fda.gov/FDAgov/Drugs/EmergencyPreparedness/BioterrorismandDrugPreparedness/default.htm.
Data Validation: CDER has instituted multiple layers of verification and validation for ensuring the accuracy of performance information. CDER relies on data extracted from information systems to support demonstrating performance toward most performance goals and targets. CDER has developed manuals of policies and procedures (MaPPs) or other standard operating procedures for using or entering data into information systems. There are quality controls built in to the information systems -controls that help ensure the integrity and accuracy of the data entered. CDER has a number of analysts who have expertise in extracting information from these systems. Their knowledge and experience working with the data, and their familiarity and experience with the business of the Center provide another layer of validation. Further, the Center requires a multi-level clearance process for verifying and validating the accuracy of the information provided in the annual performance report.
Cross Reference: This performance measure supports HHS Strategic Goal 2.
Long Term Goal: Sustain access to safe and effective new products by improving rapid, transparent and predictable science-based review of marketing applications.
Measure FY Target Result

9. Improve the Safe Use of Drugs in Patients and Consumers (12007) (Output)

(Formerly: Enhance postmarketing drug safety.)

 

 

 

 

 

 

2007
Evaluate new processes for communicating risk information and establish timeliness measures for time between identification of safety issues and action on those issues; Collaborate with the Centers for Medicare and Medicaid Services (CMS) on at least one study of a high priority safety issue in the Medicare population 1/08
2006
Standardize Agency processes and criteria for communicating risk information to patients and healthcare providers 1/07
2005
Review and provide comments on 100% of Risk Minimization Action Plans (RiskMAPs) for NMEs and for those products for which the sponsor or FDA initiated discussions, in accordance with applicable PDUFA goal dates. 100%
Data Source: CDC/DHS Strategic National Stockpile (SNS) program, database from Department of Energy/REAC/TS (Oakridge), published guidance for Industry, published Federal Register Notices, www.fda.gov/FDAgov/Drugs/EmergencyPreparedness/BioterrorismandDrugPreparedness/default.htm.
Long Term Goal: Improve problem detection and take timely and effective risk management actions with all FDA-regulated products.
Measure FY Target Result

10. Increase the efficiency of the Adverse Event Reporting Process by reducing the average cost associated with turning a submitted Adverse Event Report into a verified record in the database. (12053) (efficiency goal -pending OMB approval)

Measure: Unit Cost associated with turning a submitted Adverse Event Report into a verified record in the database.

2007
$14/per report
01/08
2006
NA
01/07
2005
NA
$17.35/per report
2004
NA
$19.30/per report
2003
NA
$21.91/per report
2002
NA
NA
11. Reduce medication errors in hospitals. Baseline data and performance targets under development. Expected completion - Sept 06
Data Sources: Adverse Event Reporting System (AERS), OMB Form 300 on Drug Safety, UFMS cost data
Data Validation: AERS, UFMS, and OCIO quality control processes
Cross Reference: This performance measure supports HHS Strategic Goal 2 and 5.
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

12. Improve the capability and efficiency of pharmaceutical development and manufacturing.

(12052 - Formerly 12016) (Output)

2007
NA
NA
2006
NA
NA
2005
cGMP: Continue progress in implementing an integrated quality management system; implement a risk-based site selection model for inspections based on results of pilot Progress reported below; pilot implemented
Data Source: Guidance documents. Relevant materials may be found on our website.
Data Validation: CDER has instituted multiple layers of verification and validation for ensuring the accuracy of performance information. CDER relies on data extracted from information systems to support demonstrating performance toward most performance goals and targets. CDER has developed manuals of policies and procedures (MaPPs) or other standard operating procedures for using or entering data into information systems. There are quality controls built in to the information systems-controls that help ensure the integrity and accuracy of the data entered. CDER has a number of analysts who have expertise in extracting information from these systems. Their knowledge and experience working with the data, and their familiarity and experience with the business of the Center provide another layer of validation. Further, the Center requires a multi-level clearance process for verifying and validating the accuracy of the information provided in the annual performance report.
Cross Reference: This performance measure supports HHS Strategic Goal 8.
Long Term Goal: Improve problem detection and take timely and effective risk management actions with all FDA-regulated products.
Measure FY Target Result

13. Increase risk-based compliance and enforcement activities to ensure drug product quality. (12020) (output)

FY 2007 Measure: The number of inspections conducted of foreign and domestic establishments identified as high-risk human drug manufacturers.

FY 2006 Measure: The number of inspections conducted of domestic establishments identified as high-risk human drug manufacturers.

2007
500 1/08
2006
483 1/07
2005
600 600
2004
376 481
2003
365 584
2002
NA NA
Data Source: Field Data Systems.
Data Validation: ORA uses two main information technology systems to track and verify field performance goal activities: the Field Accomplishments and Compliance Tracking System (FACTS) and the Operational and Administrative System Import Support (OASIS). FACTS includes 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.
Cross Reference: These performance measures support HHS Strategic Goal 2.

 

1. Improve the efficiency and effectiveness of the new drug review program to ensure a safe and effective drug supply is available. (12001)  (Formerly: Ensure a safe and effective drug supply is available to the public.) 

  • Context of Goal: This performance goal focuses primarily on improving the effectiveness and efficiency with which the FDA processes new drug applications. Central to that focus is FDA's commitment to meeting the goals and requirements of the Prescription Drug User Fee Act (PDUFA). The Public Health Security and Bioterrorism Preparedness and Response Act of 2002 reauthorized the collection of user fees to enhance the review process of new human drugs and biological products and established fees for applications, establishments, and approved products. FDA's timely performance of high-quality drug reviews in recent years reflects the importance of managerial reforms and substantial additional resources provided under the Prescription Drug User Fee Act (PDUFA). Consistent with the PDUFA requirements, a major objective of the human drugs program is to reduce the time required for review of all drugs. A key determinant in knowing if CDER is making progress in reducing time is to measure the time to "first action." The first action is the first regulatory action CDER takes (approvable, not approvable, or approval letter) at the end of the review of the original NDA submission (the first review cycle). The "first action time" refers to the time it takes to review and take an action on the original submission. This statistic is different from "total approval time" which is the time it takes from the original receipt of the application until it is approved, which may take more than one review cycle. "Total approval time" includes time spent reviewing an application in each of the review cycles plus the time taken by the sponsor to respond to the issues raised in the approvable/not approvable letter(s) and to re-submit the application for review. CDER's featured targets under this performance goal are to measure time to first action for "priority" submissions and "standard"submissions. Applications for drugs similar to those already marketed are designated standard, while priority applications represent drugs offering significant advances over existing treatments. (For example, drugs for Acquired Immune Deficiency Syndrome (AIDS) and cancer typically fall into the priority category.)
  • Performance: CDER will not have the final performance numbers for FY 2005 until October 2006. The latest information on CDER's performance toward the targets for this performance goal is from FY 2004. In FY 2004, CDER exceeded all PDUFA goals, including first actions on NDAs and BLAs. Review of Biologic License Applications (BLAs) for Therapeutic Biologic Products was transferred from CBER to CDER effective 10/1/2003, and these submissions are included in the table below.Performance toward the standard and priority NDA/BLA submissions, and other PDUFA goals, is provided in the following table:
 Fiscal Year 2004 First Action Review Performance
(Performance data as of September 30, 2005)
  Number Filed 2004 Performance Goal Final Performance
NDAs/BLAs      
Standard 94 90% in 10 mo. 97%
Priority 28 90% in 6 mo. 96%
NMEs/New BLAs      
Standard 15 90% in 10 mo. 100%
Priority 18 90% in 6 mo. 100%

 

2. Increase the number of drugs that are adequately labeled for children and ensure the surveillance of adverse events in the pediatric population. (12026)

  • Context of Goal: The context of the Pediatric Program's performance goal covers the activities and requirements of the various laws passed to ensure safe and effective drug products are available for children. Due to the inadequacy of pediatric use information found in the majority of prescription medications in the United States, Congress passed several legislative initiatives to promote drug development for children. In 1997, the Food and Drug Administration Modernization Act (FDAMA) was signed into law with Section 111 providing incentives to manufacturers who conduct studies in children. This incentive program, which provides six months of additional marketing exclusivity in return for conducting pediatric studies requested by the FDA, was reauthorized in January 2002 under the Best Pharmaceuticals for Children Act (BPCA). As a result of these initiatives, the number of ongoing pediatric clinical trials in the last 5 years has increased dramatically. Many of the studies reported to date have yielded new dosing and safety information in labeling. On December 3, 2003, the Pediatric Research Equity Act (PREA) was enacted. This law provides FDA the authority to require pediatrics studies for certain new and already marketed drug and biological products. PREA incorporates many elements of the former "Pediatric Rule" (63 FR 66632, Dec. 2, 1998) that was struck down in U.S. District Court for the District of Columbia on October 17, 2002. The effective date of PREA is retroactive to April 1, 1999, the same date the former Pediatric Rule became effective. Due to the retroactive nature of the legislation, a significant number of previously submitted applications are now subject to the requirements.
  • Performance: The target for FY 2005 performance was to issue at least 8 written requests for drugs that need to be studied in the pediatric population and report to the pediatric advisory committee on adverse events for 7 drugs that receive pediatric exclusivity. CDER issued Written Requests to sponsors of 12 on-patent drugs and Written Requests for 4 drugs on NIH's annual Priority List, as required by the Best Pharmaceuticals for Children Act. CDER reported to 2 Pediatric Advisory Committees on adverse events for 14 drugs that received pediatric exclusivity.

In addition, CDER accomplished the following activities in FY 2005:

  • Additional efforts were made related to Written Requests for the study of on-patent drugs in the pediatric population:
    • 40 amendments were issued to sponsors of existing Written Requests.
    • 3 on-patent Written Requests, declined by sponsors, were referred to the Foundation for the NIH.
  • Exclusivity determinations were made once final study reports were submitted:
    • Final study reports were submitted for 9 drugs
    • Exclusivity determinations were made for 14 drugs
    • Exclusivity was granted for 13 drugs
  • Final pediatric labeling information was determined and information disseminated:
    • 16 labeling changes were made and posted on the web
    • Information was disseminated through 22 outside presentations/liaison activities including 4 abstracts published, 6 scientific articles published, 4 poster presentations; and through 2 AAP News vignettes
  • Medical/clinical pharmacology reviews were posted on the web for 21 drugs at the time of action, under the provisions of Section 9 of the BPCA. Related FR notices were published.
  • CDER worked with NIH to publish the annual Priority List of Drugs in the Federal Register, January 27, 2005.

FDA is using several mechanisms to provide information on products for pediatric use:

  • The Best Pharmaceuticals for Children Act (BPCA), enacted in January 2002, requires that FDA make publicly available a summary of the medical and clinical pharmacology reviews of the pediatric studies conducted for supplements submitted under the BPCA. A total of 49 summaries are now posted, regardless of the regulatory action, at http://www.fda.gov/cder/pediatric/Summaryreview.htm.
  • BPCA mandates review of all adult and pediatric adverse event reports for a one-year period after pediatric exclusivity is granted and presentation of these reports to a pediatric advisory committee. As of March 31, 2005, reports have been presented for 34 drugs.
  • FDA isworking with companies to put more information on pediatric studies into the label even when the studies did not show efficacy for the indication studied.
  • The Pediatric Research Equity Act (PREA), enacted December 2003, gave FDA the authority to require pediatric studies of certain pharmaceutical products when such studies are needed to ensure the safe and effective use of the products in children. However, PREA does not require the same public disclosure of pediatric studies that is required under the BPCA.

 

3. Improve the efficiency and effectiveness of the generic drug review program to ensure safe and effective generic drug products are available for Americans. (12003) (Formerly: Ensure safe and effective generic drugs are available to the public.)

  • Context of Goal: Generic drugs are much appreciated for their cost-effectiveness. According to the Congressional Budget Office, they save consumers an estimated $8 billion to $10 billion a year compared with the price of trade-name products. The basic requirements for approval of generic and trade-name drugs are the same as new drug approvals, although the generic drug manufacturer does not need to repeat the safety and efficacy studies conducted by the developer of the original product. Prior to approval, generic drug sponsors are required to demonstrate bioequivalence to the innovator drug product by showing that the active ingredient in their product is absorbed at a rate and extent similar to the innovator counterpart. The approval time is measured from the date the application is received to the date a major action, either an approval or not approvable, is reached.

    This performance goal is an interim step toward achieving the Agency long-term outcome goal to reduce average time to marketing approval or tentative approval for safe and effective new generic drugs. The target for the long-term outcome goal is to reduce the average FDA time to approval or tentative approval for the fastest 70% of original generic drug applications by 1.5 months. The FY 2006 target involves making interim progress toward that target by decreasing the average time for a portion of the fastest approvals and tentative approvals by 0.5 months. Targets for FY 2003 - 2005 for this performance goal involve progressively increasing the percentage of generic drug applications reviewed and acted upon within six months after submission. Reviewing and acting upon more applications in less time should help drive down the average approval time. In FY 2002, the median approval time for generic drugs was 18.3 months. For FY 2003, the median approval time was down by one month to 17.3 months and down another month to 16.3 months for FY 2004.

  • Performance: FDA exceeded its goal for FY 2004 by acting on 87.4 percent of 543 original applications. FDA also exceeded its goal in FY 2003 by acting on 90 percent of 449 original applications.

 

4. Improve the efficiency and effectiveness of the over-the-counter (OTC) drug review program to ensure a safe and effective drug supply is available. (12048) (Formerly: Increase the number of drugs adequately labeled available for OTC use)

  • Context of Goal: Over-the-counter (OTC) drugs play an increasingly vital role in America's health care system. The trend to self-medicate has increased greatly in recent years as health care costs have risen and consumers want to be empowered to treat minor ailments with OTC drug products. However, safety, effectiveness, and proper labeling have not always been characteristic of OTC drug products in the United States. FDA's goal by 2010 is to complete its existing review of OTC drug products, to have considered a number of key foreign drugs for marketing in the United States, and to have considered a number of key potential "prescription (Rx)-to-OTC" switches. OTC drug monographs are "recipes" for marketing OTC drug products without the need for FDA pre-clearance. The monographs list the allowed active ingredients and the dosage or concentration, the required labeling, and packaging and testing requirements if applicable. The monographs save manufacturers costs and reduce barriers to competition, as they allow both large and small companies to enter the market place with OTC drug products that have to meet the same, uniform criteria. Final monographs (agency final rules) need to be completed for a number of large product categories (e.g., external analgesics, internal analgesics, antimicrobials, oral health care products, laxatives). In the next 7-10 years, FDA plans to complete the initial review of OTC monographs for 29 categories of drug products, thereby eliminating all unsafe and ineffective products from the OTC market.
  • Performance: FDA exceeded its goal by completing review and action on 100% of Rx-to-OTC switch applications within 10 months of receipt and making significant progress on 17 OTC monographs (Vaginal contraceptive drug products containing Nonoxynol 9; internal analgesic, antipyretic, and anti-rheumatic; laxatives; cold, cough, allergy, bronchodilator, and antiasthmatic healthcare antiseptics; food handlers antiseptics; consumer antiseptics; poison treatment; sunscreens; external analgesics; urinary analgesics; skin protectants; phenylpropanolamine; nasal decongestants; convenience size labeling rule; plaque and gingivitis; benzocaine/weight control). The expansion of the OTC drug review to evaluate foreign OTC drugs is expected to increase switch requests in the near future. While CDER is hoping for a 50 percent increase in applications; however, we do not control the number of applications submitted. FDA recognizes that some of these switch requests involve issues of "OTCness" - determination that the drug is appropriate for OTC use and developing appropriate labeling and other information (such as was done for OTC stop smoking aid products) for safe and effective consumer use of these products without the intervention of a health care professional.

 

5. Reduce time to marketing approval for new drugs and biologics.

  • Context of Goal: Reducing unnecessary delays in the approval time for safe and effective drugs that truly represent new therapies [i.e., NMEs and biologics] means earlier patient access for these medicines. Reducing unnecessary delays in drug approval also helps to both control the cost of new drug development, cited as a factor affecting the cost to consumers, and supports market competition among innovators. This is both good for the drug industry and good for consumers. New drug development presents uncertainties that increase the business risk and costs to the innovator. Higher costs can create barriers to competition both from new drugs with therapeutic value - but not blockbuster potential, and new innovators that don't have access to the capital available to more established pharmaceutical companies. Although some scientific and technical uncertainties are inherent and unavoidable in drug innovation, others can be reduced or eliminated, helping speed patient access to new drugs, and reducing the cost of drug development. FDA has begun major initiatives to reduce those sources of uncertainty.

    Additional initiatives are included in the Agency's Strategic Action Plan. Sponsors, for example, may be uncertain about what FDA expects to see in a high quality new drug application, because of a lack of interaction with FDA during development, or lack of clear, timely or consistent FDA-sponsor communication during review. As a result, the submitted application may have deficiencies that could have been avoided or addressed quickly, but instead create unnecessary delays as they are identified by FDA and then addressed by the sponsor. Although FDA has found that applications can often contain deficiencies that are not so readily addressed, clear understandings of FDA expectations and timely communication between FDA and application sponsors can increase the likelihood that the submitted application contains the necessary information for timely approval on the first review cycle.

    The targeted reductions in this FDA outcome goal represent approximately 10.5 percent reductions in total FDA review times for priority and standard NMEs and BLAs. Using Tufts estimates of potential cost reductions by phase of drug development1, a 10 percent reduction in regulatory review time yields a 1.6 percent reduction in total capital costs, now estimated at $802 million, translating to a savings of $12.8 million per NME approved.

  • Performance:The FDA approval time for the fastest 50 percent of priority NME and biologics licensing applications (BLAs) approved for the FY 2002-2004 cohort is 240 days as compared to 286 days for the baseline FY 2000-2002 submission cohort. This is a reduction of 46 days versus the FY 2005-2007 target of a reduction of 30 days.

 

6. Reduce the time to marketing approval or tentative approval for safe and effective new generic drugs.

  • Context of Goal: FDA achievement of this goal will create earlier access to lower cost drug alternatives for patients. The high cost of drugs limits patient access to treatment. The lower income and uninsured populations are particularly affected. Research has shown that 42 percent of the uninsured do not fill prescriptions because of financial reasons. While all state Medicaid programs provide outpatient prescription drug coverage, slightly more than one in four Medicaid patients ages 18-64 could not afford to fill at least one prescription, according to a study by the Center for Studying Health System Change (HSC). Increasing the availability of generic drugs will make many important treatments more affordable to the poor and the elderly and significantly improve access to treatment.

    Prescription drug expenditures remain one of fastest-growing segments of the U.S. health care system. In 2001, a 13.8 percent increase in drug spending accounted for one-fifth of the overall increase in health care spending. State Medicaid programs are particularly challenged with controlling escalating cost of pharmacy benefits and are in serious need of more generic alternatives to high cost brand name drugs to both reduce costs and increase access to treatment. Medicaid spending on outpatient drugs has increased by 18 percent a year from 1997 - 2000, which is close to three times greater than increases in medical care spending.

    Optimal access and use of generic drugs will enable policy decision makers to contain costs in both the Medicare and Medicaid programs. This will only become more important as more of the top selling brand name drugs go off patent over the next few years and if legislation for a Medicare drug benefit is passed by Congress. The National Institute for Healthcare Management has estimated that Medicaid programs could save $1 to $1.5 billion over the next few years if they were to increase their share of generic drug use to 55 percent of their total drug spending. According to researchers at Brandeis University, if a Medicare drug benefit were to be implemented and the use of generic drugs represented 50 percent of the total prescriptions, approximately $250 billion would be saved over 10 years.

    Generic drugs are typically priced between 20 - 50 percent lower than brand name competitors, which represent a significant cost saving to consumers.

  • Performance: The FDA approval time for the fastest 70 percent of original generic drug applications approved for the FY 2000-2002 cohort is 16.2 months as compared to 17.9 months for the baseline FY 1998-2000 submission cohort. This is a reduction of 1.7 months versus the FY 2005-2007 target of a reduction of 1.5 months. However, this progress may not hold in future years.

    Despite all of the efforts to make our review processes more efficient and to decrease review times, FDA is experiencing a growing backlog of applications awaiting review. Beginning in 2003, the number of submissions began increasing rapidly and for FY 2005, the Office of Generic Drugs (OGD) projects over 800 submissions, an over 220% increase from 2002. Recent reports in the press seem to indicate that this trend of unprecedented numbers of submissions may continue for the next few years in part due to increasing numbers of brand name drug patent expirations.

    Increases in appropriated funds in recent years were instrumental in enabling OGD to increase staff to address the backlog of applications that grew in the late 1990's due to insufficient staff levels. As a result of those funds and of the Agency's successful efforts to establish smarter and more efficient review processes, FDA has been able to meet or exceed the statutory goal of taking a first action in the statutory 180 day time frame. Further, as a result of appropriations increases in recent years we have been able to significantly shrink the backlog of applications while also shrinking review times from a median time of 27 months in 1995 to 15.7 months in 2004. Our success in 2004 was demonstrated by our ability to approve 380 generic products in one year - on average, a new generic drug or a new use for an existing generic product was approved each day that year.

    However, with the unprecedented and unpredicted surge in submissions to FDA, the backlog is growing again, and OGD expects that performance toward meeting the statutory timeframes will decrease. Further, FDA expects that progress toward meeting its more meaningful measure of the total time to approval of applications is also in jeopardy. The Generics Review long-term outcome goal targets the 3- year submission cohort for FY 2005-07, exactly the timeframe we expect will cover the incredible surge in submissions.

 

7. Create state-of-the-art information and knowledge management systems and practices to move to a paperless environment. (12051)

  • Context of Goal: The use of current technology will allow CDER to receive and review regulatory submissions more efficiently. In order to move to a paperless environment in an efficient and cost effective manner, we must develop standards for submission.
  • Performance: Due to the increase in electronic submissions since 1997, there has been a significant decrease in the average number of paper volumes per NDA submissions. CDER has been receiving an increasing volume of regulatory submissions in electronic format. In FY 2005, CDER significantly exceeded its goal for receipt of abbreviated new drug applications for generic products by receiving over 90% of the applications with some electronic portion.

 

8. Enhance the protection of the American public against the effects of terrorist agents by facilitating the development of and access to medical countermeasures, providing follow-up assessments on therapies, and engaging in emergency preparedness and response activities. (12045)

  • Context of Goal: The first therapy for those exposed to a biological, chemical, or radiological/nuclear agent is often a drug. FDA has been taking an aggressive and proactive approach to getting information on medical countermeasures into the labeling of already approved drugs. For example, gentamicin has not been FDA-approved for plague, yet is also widely recommend as a preferred therapy by experts. Human clinical trial data are needed to demonstrate safety and efficacy for specific treatments and to identify new therapeutic drug options. In the Federal Government's response to various agents of mass destruction, drugs will be mobilized from the CDC's Strategic National Stockpile (SNS). However, not all drugs in the SNS are FDA-approved for Counterterrorism uses. Identification of these deficits including development of a plan to address these deficits will move the Public Health Service closer to a goal of labeling all drugs that reside in the SNS for Counterterrorism uses.
  •  Performance: Funding over the last five years has strengthened CDER's capability to identify, prepare for, and respond to biological, chemical, and radiological/nuclear threats and incidents. FDA is engaged in many efforts to promote the development of medical countermeasures. The Agency encourages early and frequent interactions with sponsors, whether they are developing a novel compound or a new indication for a previously approved product. Regulatory mechanisms, such as Fast Track Designation, use of surrogate markers, or development under the Animal Efficacy Rule, and guidance documents are available to accelerate submission and review. FDA is actively working to expand the availability of safe and effective medical countermeasures for special populations (e.g., pregnant or lactating women, infants, elderly) through contracts that fund pharmacokinetic and safety studies of antibiotics likely to be used to prevent or treat illness following a terrorist attack. The following list describes the countermeasure performance for FY 2005:
    • Levaquin (levofloxacin) was approved for an additional indication of post-exposure prophylaxis of inhalational anthrax.
    • Cipro (ciprofloxacin) tablets, iv, solution, and oral suspension received approval for revised labeling for the Indications and Usage, Adverse Reactions, and Inhalational Anthrax-Additional Information sections of the package insert based on the information obtained from the Centers for Disease Control and Prevention program evaluation conducted after the bioterrorism events of October 2001. Four generic ciprofloxacin applications were approved.
    • ThyroShield (potassium iodide oral solution) was approved as a thyroid blocking agent for use in radiation emergencies. This oral solution is appropriate for use in children or in adults who cannot swallow tablets. In February 2005, CDER assisted DHHS in a BioShield procurement of ThyroShield for the Strategic National Stockpile.
    • Tentative approvals were granted for Manoplex (insoluble Prussian Blue), Kelacal (pentetate calcium trisodium injection, or calcium DTPA), and Kelazin (pentetate zinc trisodium injection, or zinc DTPA), for treatment of internal radiation contamination.
    • CDER continued to facilitate the ongoing human trials of gentamicin in plague in Africa, as well as the monkey studies of gentamicin, ciprofloxacin, levofloxacin, ceftriaxone, and doxycycline in pneumonic plague. These studies were funded in previous years by CDER through interagency agreements with the CDC and NIAID, respectively.

 

9. Improve the Safe Use of Drugs in Patients and Consumers. (12007)

  • Context of Goal: FDA recognizes now, more than ever, the need for protecting and advancing the public health, and the Agency has been focusing on new and better ways to perform this mission. Recently, the Department and FDA have announced new important efforts that the Agency is undertaking to improve its ability to monitor and respond to emerging drug safety information. These steps will ensure both a better internal process of deliberation of drug safety issues that ensures appropriate and independent consideration of all issues as well as a stronger ability to gather data about drug safety issues once a drug has been approved. Most importantly, we are working toward a policy of more transparency to ensure that patients and physicians have the most up-to-date and complete information necessary to make their treatment decisions. This new Drug Safety Initiative will give patients, healthcare professionals, and other consumers quick and easy access to the most up-to-date and accurate information on medicines and make FDA's drug review, approval, and monitoring programs as transparent a possible. FDA will be creating a Drug Watch Web Page that will include emerging information for both previously and newly approved drugs about possible serious side effects or other safety risks that have the potential to alter the benefit/risk analysis of a drug, affect patient selection or monitoring decisions, or that can be avoided through measures taken to prevent or mitigate harm. New communication channels will also include:
    • Healthcare Professional Information Sheets. One-page information sheets for healthcare professionals for all drugs on FDA's Drug Watch and all drugs with Medication Guides (FDA-approved patient labeling) containing the most important new information for safe and effective product use, such as known and potential safety issues based on reports of adverse events, new information that may affect prescribing of the drug, and the approved indications and benefits of the drug.
    • Patient Information Sheets. One-page information sheets for patients containing new safety information as well as basic information about how to use the drug in a consumer friendly format for all products on Drug Watch.

    To demonstrate our commitment and to measure our progress on this initiative, we have proposed a performance target for FY 2006 that focuses on the establishment of the new risk communication processes. For example, we will establish criteria for determining what drug products should be listed on the Drug Watch and for using drug labeling "black box warnings" to communicate safety information. The targeted increase for the Office of Drug Safety for FY 2006 will directly support performance toward the FY 2006 target. In FY 2007, with base budget resources, we expect to be able to continue progress on this initiative, and we commit to evaluating our new risk communication processes and to establishing timeliness measures for the time between when we identify a drug safety issue and the time when we communicate the risk information to the public. Further, in FY 2007, with an increase in funding for Drug Safety, we expect to be able to expand our understanding of, involvement in, and access to external population-based and "linked" databases (such as the CMS Medicare/Medicaid database) which represent the future of more thorough and continued monitoring of drug products after they are marketed. Key information regarding the safety of drug products is available in these types of databases.

  • Performance: The FY 2005 target for this performance goal involves reviewing and providing comments on Risk Minimization Action Plans (RiskMAPs) for new molecular entities (NMEs) and for those products for which the sponsor or FDA initiated discussions, in accordance with applicable PDUFA. CDER met this performance target. Further, CDER is making progress toward the new drug safety initiative described within the context of the goal to improve the safe use of drugs. For example, we areupdating drug safety information on certain drug products -- including new molecular entities, drugs with medication guides, and drugs with known safety issues -- and making that available to consumers in a new, user friendly format. We have recently updated our website (http://www.fda.gov/cder/drugSafety.htm) to reflect our advancements to date.

 

10. Increase the efficiency of the Adverse Event Reporting Process by reducing the average cost associated with turning a submitted Adverse Event Report into a verified record in the database. (12053)

  • Context of Goal: A crucial part of FDA's mission is to perform pre-market and post-market safety and efficacy assessments of human drugs and therapeutic biologics. Clinical trials that lead to formal marketing approval only begin to quantify the safety and efficacy of a given pharmaceutical compound or biological product. The collection and analysis of data by FDA staff must occur throughout the entire life cycle of the product in order to identify unexpected safety risks associated with the use of a human drug that could not have been predicted by clinical trials and biostatistical analysis. These unexpected safety problems, called adverse events, must be reported to FDA in order for the agency to carry out its mission of performing post-marketing safety surveillance (PMSS). The Adverse Event Reporting System (AERS) is a computing system that FDA staff uses to carry out the PMSS function.

    The AERS system is a critical component of FDA's post-marketing safety surveillance systems for all drug and therapeutic biologic products. The information captured in the AERS system allows FDA scientists and statisticians to search for patterns that may indicate an emerging safety hazard, which is the first step in analyzing the potential causes and formulating an effective risk management response. In FY 2005, about 94% of the adverse event reports relating to drugs and therapeutic biologics were submitted by manufacturers, who are required to submit expedited reports of serious events within 15 days, and periodic reports for less serious events. The remaining 6% of the drug and therapeutic biologic adverse event reports received by FDA are "direct" reports from health care providers, pharmacists, and citizens, which must be re-keyed into the AERS system. Overall, only about 29% of the total adverse event reports were submitted electronically in FY 2005. However, FDA received slightly over half of the expedited reports electronically.

    The manual entry of data into AERS is time-consuming and costly. Overall, the operating costs of the activities and systems covered by this goal represent approximately 12% of FDA's estimated total annual expenditures on post-market drug safety activities. The costs included in the measure include both information system operation and maintenance, as well as scientific and technical staff time to process the records and perform quality control.

    The current AERS system was released in November 1997 to support post-market safety surveillance. Initially, AERS captured information from over 200,000 adverse event reports per year and enabled electronic retrieval of information for agency reporting of adverse reactions to drugs and therapeutic biologics marketed in the United States. Since that time the total number of adverse event reports has grown to over 400,000 per year. Moreover, between 1992 and 2004, the number of manufacturer reports of serious and unexpected adverse events (the so-called Manufacturer 15-day reports, which represent a subset of the total number of adverse event reports) has grown almost 9-fold (see the figure below). The current cost of processing each AER presents a major obstacle to FDA's ability to keep up with and analyze the rapidly increasing volume of reports and to rapidly identify, assess, and manage emerging safety risks.

    FDA is making the AERS system more efficient by improving the data entry work processes and reengineering the system to increase the percentage of electronic submissions, to reduce the amount of re-keying, to increase the number of submissions that are "pre-MedDRA coded," along with other efficiencies. These system improvements will allow the FDA to reduce the average cost and time associated with turning a submitted Adverse Event Report into a verified record in the database. This improvement in efficiency will allow scientists and statisticians to access safety information sooner, and will free up resources that can be redirected to risk analysis activities that directly improve our ability to recognize and respond to drug safety problems.

  • Performance: The average cost associated with turning a submitted Adverse Event Report into a verified record in the database has been decreasing since FY 2003 due to FDA efforts to streamline its business processes and improve the information systems that are used to process records. In FY 2003, the cost per report was $21.91/per report. In FY 2004, the cost per report was $19.30/per report. In FY 2005, the cost per report was $17.35/per report. FDA expects to achieve further improvements in efficiencies due to improved automation of the submission and validation processes, and outreach to improve adoption of electronic submissions. The proposed FY 2007 target represents almost a 20% reduction in cost per adverse event report compared to the FY 2005 level.

Individual Safety Reports Received Graph [D]

 

11. Reduce medication errors in hospitals.

  • Context of Goal: In November 1999, the Institute of Medicine released a report estimating that as many as 98,000 patients die from medical errors in hospitals alone. Many of these deaths, as well as additional non-fatal illnesses, are associated with errors involving FDA regulated medical products, especially medications. A significant percentage of drug related mortality and morbidity results from errors that are preventable. In addition to their human cost, these errors impose significant economic costs on the U.S. health care system.

    The Secretary of Health and Human Services has directed FDA to promulgate the bar coding regulation to reduce preventable errors from medical products. This rule is anticipated to enable the uptake and use of bar code scanners that will allow a health professional to compare the bar code on a human drug product to a specific patient's drug regimen and then verify that the right patient is receiving the right drug, at the right dose, via the right route, at the right time. Research to date has demonstrated the ability of bar code scanners at the point of care to intercept errors in dispensing and administration of medication and prevent related adverse events. The implementation of this rule will be a big step forward for FDA in improving patient safety. The total cost of preventable adverse events has been estimated at $17 Billion. Preventing 11 percent of adverse drug events related to medication errors in half of all the hospitals in the U.S. will significantly reduce the related morbidity, mortality and health care costs.

  • Performance: Baseline data and performance targets under development. Expected completion - Sept 06.

 

12. Improve the capability and efficiency of pharmaceutical development and manufacturing. (12016)

  • Context of Goal: The focus of this performance goal for 2005 is on the Agency's current good manufacturing practices (cGMP) initiative. On August 21, 2002, FDA announced a major new initiative on regarding pharmaceutical manufacturing, "Pharmaceutical GMPs for the 21st Century: A Risk-Based Approach." The program has several ambitious objectives. One is to ensure that regulatory review and inspection policies are based on state-of-the-art pharmaceutical science and to encourage the adoption of new technological advances by the pharmaceutical industry. FDA will determine the best pathway to better integrate advances in quality management techniques, including quality systems approaches, into the Agency's regulatory standards and systems for the review and inspection processes. Additionally, risk-based approaches, that focus both industry and agency attention on critical areas, will be implemented.
  • Performance: Key activities toward accomplishing the performance goal for improving the capability and efficiency of pharmaceutical development and manufacturing are associated with the current Good Manufacturing Practices (cGMP) Initiative. On February 20, 2003, the Food and Drug Administration (FDA) released its progress report on a major initiative concerning the regulation of drug product quality. The two-year program, launched on August 21, 2002, applies to human drugs and biologics and veterinary drugs and has several objectives. One is to ensure that regulatory review and inspection policies are based on state-of-the-art pharmaceutical science and to encourage the adoption of new technological advances by the pharmaceutical industry. FDA is working toward integrating advances in quality management techniques, including quality systems approaches, into the Agency's regulatory standards and systems for the review and inspection processes. Additionally, implementation of risk-based approaches, that focus both industry and agency attention on critical areas are underway. Lastly, the Agency is committed to enhancing the consistency and coordination of its drug quality regulatory programs.

In FY 2005, FDA performed the following activities toward meeting this goal:

  • Evaluated comments on the draft quality system guidance document issued in September 2004, advanced the revision of the draft;
  • Established the CGMP Question and Answer Guidance Program under an internal SOP, initiated drafting of a CDER MaPP for the program;
  • Advanced to Step Three for the International Conference on Harmonisation (ICH) Q8 guidance project on Product Development and the ICH Q9 guidance project on Risk Management;
  • Established the ICH Q10 guidance project on quality systems and secured the approval of the concept paper on the project;
  • Completed the first Pharmaceutical Inspectorate class training component and initiated the on-the-job training/detail training component;
  • Contributed to the ongoing internal quality system reviews of warning letter procedures and recall procedures;
  • Completed review of comments on the proposal to revise the 21 CFR Part 11 Electronic signatures regulation, and initiated drafting of the revision;
  • Completed the pilot program for use of the risk-based computer model for selection of sites for CGMP inspection, found the pilot to be successful, added refinements to the model for including field alert activity and drug quality defect activity as factors affecting risk, and implemented the model as the routine risk tool for inspection site selection; used the model for preparation of the inspection plan being implemented for FY2006.

 

13. Increase risk-based compliance and enforcement activities to ensure drug product quality. [Inspections of foreign and domestic establishments identified as high risk human drug manufacturers.] (12020)

  • Context of Goal: Important features of the risk-based strategy for this goal will be reducing the occurrence of illness and death by focusing resources on manufacturing establishments and other industry components that present the highest risk.This will result in different inspection frequencies as establishment processes come under control and present lower risk, or as new risks are identified. We note that these goals were reported in previous years as inspection of a fixed percentage of the inventory of establishments. However, given the fluctuation in the inventory, the inspection resources available, and the risk-based prioritization approach that FDA is developing, we believe that it is more appropriate to state the goal in terms of the number of inspections of the highest-risk establishments. We have reformulated the goals accordingly, including prior years for comparability. This strategy will also allow FDA to better communicate to our stakeholders about drug safety risks.

    For FY 2005, FDA developed a more quantitative risk model to help predict where FDA's inspections are most likely to achieve the greatest public health impact. The model includes risk factors relating to the facility, such as compliance history, and to the type of drugs manufactured at the facility. For FY 2006, FDA will continue to improve the quantitative risk model, which may also include risk factors relating to the manufacturing processes and the level of process understanding. The targets continue the trend of measuring performance toward inspecting the highest-risk establishments.

    The risk prioritization scoring methodology was applied to about 800 non-US facilities manufacturing drugs for the US market (the number of drug facilities that received an inspection by FDA in recent years). Of these 800, approximately 500 scored high enough to be included in the domestic U.S. priority. In addition, about 50 percent of all non-U.S. sites are active pharmaceutical ingredient (API) manufacturers and about 55 percent of our annual inspections are of facilities that process APIs. FDA does not inspect non-U.S. facilities at the same frequency expected for U.S. facilities.

    For FY 2007, FDA proposes to inspect, as part of this goal, a combination of both foreign and domestic facilities that are ranked the highest risk by the risk prioritization scoring model. This inclusion of foreign facilities would permit more consistent coverage of non-U.S. sites predicted to have a similar public health impact as we have experienced as a result of our inspections of domestic U.S. sites in FY 2005 and FY 2006.

  • Performance: FDA met the FY 2005 goal by inspecting 600 high-risk firms.

 

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