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Appendix A: PDUFA IV Performance Goals FY 2008 – FY 2012
FY 2008 PDUFA Performance Report Table of Contents
The table below summarizes, by fiscal year, the performance measures set forth in the letters referenced in the Food and Drug Administration Amendments Act of 2007 (PDUFA IV). Goal summaries for the earlier years of PDUFA can be found in the Appendix of earlier PDUFA Performance Reports.
I. Review Performance Goals
| On-time Performance Level for Fiscal Year of Filing or Receipt | |||||
|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | |
| Review and act on priority original NDAs and BLAs within 6 months of receipt.[2] | 90% on time | ||||
|
Review and act on standard original NDAs and BLAs within 10 months of receipt. [2] |
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| Review and act on priority efficacy supplements within 6 months of receipt.[2] | |||||
| Review and act on standard efficacy supplements within 10 months of receipt.[2] | |||||
| Review and act on all manufacturing supplements within 6 months of receipt and those requiring prior approval within 4 months of receipt.[3] | |||||
| Review and act on Class 1 resubmitted original applications within 2 months of receipt. | |||||
| Review and act on Class 2 resubmitted original applications within 6 months of receipt.[2] | |||||
| Review and act on Class 1 resubmitted efficacy supplements within 2 months of receipt. | |||||
| Review and act on Class 2 resubmitted efficacy supplements within 6 months of receipt.[2] | |||||
[2] Receipt of a major amendment in the last 3 months extends the goal date by 3 months. Under PDUFA II this extension applied to original NDAs and BLAs only. Under PDUFA III, it also applies to efficacy supplements and Class 2 resubmitted NDAs, BLAs, and efficacy supplements.
[3] Receipt of a major amendment in the last 2 months extends the goal date by 2 months (PDUFA III submissions only). This extension applies only to manufacturing supplements.
II. NME Performance Goals
The performance goals for priority and standard original NMEs will be the same as for all of the original NDAs but will be reported separately.
For biological products, for purposes of this performance goal, all original BLAs will be considered to be NMEs.
III. Procedural and Processing Goals
| Performance Area | FDA Activity | Performance Goal | Performance Level FY 2008 – FY 2012 |
|---|---|---|---|
| Meeting Management | Meeting Requests -- Notify requestor of formal meeting in writing (date, time, place, and participants). | Type A Meetings Within 14 days of receipt of request. | 90% on time |
| Type B Meetings within 21 days of receipt of request. | |||
| Type C Meetings within 21 days of receipt of request. | |||
| Scheduling Meetings -- Schedule meetings within goal date or within 14 days of requested date if longer than goal date. | Type A Meetings within 30 days of receipt of request. | ||
| Type B Meetings within 60 days of receipt of request. | |||
| Type C Meetings within 75 days of receipt of request. | |||
| Meeting Minutes -- FDA prepared minutes, clearly outlining agreements, disagreements, issues for further discussion and action times will be available to sponsor. | Within 30 days of meeting. | ||
| Clinical Holds | Response to sponsor’s complete response to a clinical hold. | Within 30 days of receipt of sponsor’s response. | |
| Major Dispute Resolution | Response to sponsor’s appeal of decision. | Within 30 days of receipt of sponsor’s appeal. | |
| Special Protocol Question Assessment and Agreement | Response to sponsor’s request for evaluation of protocol design. | Within 45 days of receipt of protocol and questions. |
IV. Review of Proprietary Names To Reduce Medication Errors
| Performance Area | Initiative | Commitment | Performance Level and/or Implementation Timeline by Fiscal Year -- Not applicable X Action due |
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|---|---|---|---|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | |||||
| Enhancement and Modernization of the Drug Safety System | Development of 5-Year plan and Communication and Technical Interactions | FDA will publish a draft 5-year plan by March 31, 2008. | X | -- | -- | -- | -- | ||
| FDA will publish the final 5-year plan no later than December 31, 2008. | -- | X | -- | -- | -- | ||||
| Conduct and publish an annual assessment of progress against the 5-year plan by September 30, 2009. | -- | X | -- | -- | -- | ||||
| Conduct and support activities designed to modernize the process of pharmaco-vigilance | Maximize the public health benefit of adverse event collection throughout the product lifecycle. Publish a request for proposals (RFP) by September 30, 2008. Award contracts during FY 2009. Complete contract studies by FY 2011. | X | X | X | X | -- | |||
| Epidemiology best practices and guidance document development During FY 2008 hold a public workshop to identify epidemiology best practices. Develop joint CDER and CBER draft guidance by the end of FY 2010. Issue final guidance in FY 2011. | X | -- | X | X | -- | ||||
| Enhancement and Modernization of the Drug Safety System (continued) | Conduct and support activities designed to modernize the process of pharmaco-vigilance (continued) | Develop and validate risk management and risk communication tools. During FY 2008 develop a plan to identify risk management tools and programs and conduct assessments of current tools and RiskMAPS. During FY 2009 hold a public workshop to obtain stakeholder input on evaluations. Starting in FY 2009 conduct annual effectiveness reviews of risk management programs and tools. | X | X | -- | -- | -- | ||
| Review Performance Goals – Drug/Biological Product Proprietary Names | Review of proprietary names submitted during IND phase (as early as end-of-phase 2) | Within 180 days of receipt. Notify sponsor of tentative acceptance or non-acceptance. | -- | 50% | 70% | 90% | |||
| Review of proprietary names submitted with NDA/BLA | Within 90 days of receipt. Notify sponsor of tentative acceptance or non-acceptance. | ||||||||
| Guidance Document Development | By the end of FY 2008, FDA will publish a final guidance on the contents of a complete submission package for a proposed proprietary drug/biological product name. | X | -- | -- | -- | -- | |||
| Review Performance Goals – Drug/Biological Product Proprietary Names (continued) | Guidance Document Development (continued) | By the end of FY 2009, FDA will prepare a MaPP (Manual of Policies and Procedures) to ensure that FDA internal processes are consistent with meeting the proprietary name review goals. | -- | X | -- | -- | -- | ||
| By the end of FY 2010, FDA will publish a draft guidance on best practices for naming, labeling and packaging drugs and biologics to reduce medication errors. Final guidance will be published by the end of FY 2011. | -- | -- | X | X | -- | ||||
| By the end of FY 2012 FDA will publish a draft guidance on proprietary name evaluation best practices. Publication of final guidance on proprietary name evaluation best practices will follow as soon as feasible. | -- | -- | -- | -- | X | ||||
| Pilot Program | During PDUFA IV, FDA will develop and implement a pilot program to enable pharmaceutical firms participating in the pilot to evaluate proposed proprietary names and submit the data generated from those evaluations to the FDA for review. | FDA will hold a public technical meeting to discuss the elements necessary to create a concept paper describing the logistics of the pilot program, the contents of a proprietary name review submission, and the criteria to be used by FDA to review submissions under the pilot program. Subsequently, by the end of FY 2008, FDA will publish the concept paper. | X | -- | -- | -- | -- | ||
| Pilot Program (continued) | During PDUFA IV, FDA will develop and implement a pilot program to enable pharmaceutical firms participating in the pilot to evaluate proposed proprietary names and submit the data generated from those evaluations to the FDA for review. (continued) | By the end of FY 2009, FDA will begin enrollment into the pilot program. | -- | X | -- | -- | -- | ||
| By the end of FY 2011, or subsequent to accruing two years of experience with pilot submissions, FDA will evaluate the pilot program. | -- | -- | -- | X | -- | ||||
| Other Activities | FDA and industry are interested in exploring the possibility of “reserving” proprietary names for companies once the names have been tentatively accepted by the Agency. | By the end of FY 2008, FDA will initiate a public process to discuss issues around “reserving” proprietary names. | X | -- | -- | -- | -- | ||
| FDA will provide the full source code and supporting technical documentation for the Phonetic and Orthographic Computer Analysis (POCA) tool and make it available on disk for use by industry and others from the general public by end of FY 2008. | X | -- | -- | -- | -- | ||||
V. FIRST CYCLE REVIEW PERFORMANCE PROPOSAL
| Performance Area | Initiative | Commitment | Performance Level and/or Implementation Timeline by Fiscal Year -- Not applicable X Action due |
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|---|---|---|---|---|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | ||||||
| Notification of Issues Identified during the Filing Review | For original NDA/BLA applications and efficacy supplements, FDA will report substantive review issues (or lack thereof) identified in the initial filing review to the sponsor by letter, telephone conference, facsimile, secure e-mail, or other expedient means. | FDA will provide the sponsor a notification of substantive review issues (or lack thereof) within 14 days after the 60-day filing date. | 90% | |||||||
| Notification of Planned Review Timelines | For original NDA/BLA applications and efficacy supplements, FDA will inform the applicant of the planned timeline for review of the application. The information conveyed will include a target date for communication of feedback from the review division to the applicant regarding proposed labeling and postmarketing study commitments (PMCs) the Agency will be requesting. | Original BLAs and NME NDAs within 14 calendar days after the 60 day filing date. | -- | 90% | ||||||
| Efficacy supplements for new/expanded indications within 14 calendar days after the 60 day filing date. | -- | -- | 90% | |||||||
| All original NDAs within 14 calendar days after the 60 day filing date. | -- | -- | -- | 90% | ||||||
| All efficacy supplements within 14 calendar days after the 60 day filing date. | -- | -- | -- | -- | 90% | |||||
| Report on Review Timeline Performance | FDA will report its performance in meeting goals for notification of review timelines in the annual PDUFA performance report. | -- | X | |||||||
| FDA will report its performance in meeting review timelines for labeling and PMCs in the annual PDUFA performance report. | -- | X | ||||||||
| Engage an independent consultant to analyze FDA’s success in meeting review timelines. A final report will be due to FDA by March 31, 2011. | -- | X | ||||||||
| Standard Operating Procedures and Training | FDA will develop harmonized (CBER/CDER) standard operating procedures (SOPs) regarding the notification of planned review timelines. Training will be provided to all CBER and CDER review staff on the harmonized (CBER/CDER) standard operating procedures. | These SOPs will be finalized and implemented by the end of FY 2008. | X | -- | -- | -- | -- | |||
| Standard Operating Procedures and Training (continued) | Training | All new review staff and refresher training will be provided to all review staff as necessary through FY 2012. | X | X | X | X | X | |||
VI. Expediting Drug Development
| Performance Area | Initiative | Commitment | Performance Level and/or Implementation Timeline by Fiscal Year -- Not applicable X Action due |
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|---|---|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | |||
| Guidance Development | FDA will develop and publish for comment draft guidances on the following topics by the end of the indicated Fiscal Year of PDUFA-IV. FDA will complete the final guidances within one year of the close of the public comment period. | Clinical Hepatotoxicity | X | -- | -- | -- | -- |
| Non-inferiority Trials | X | -- | -- | -- | -- | ||
| Adaptive Trial Designs | X | -- | -- | -- | -- | ||
| End of Phase 2(a) Meetings | X | -- | -- | -- | -- | ||
| Multiple Endpoints in Clinical Trials | -- | X | -- | -- | -- | ||
| Enriched Trial Designs | -- | -- | X | -- | -- | ||
| Imaging Standards for Use as an End Point in Clinical Trials | -- | -- | -- | X | -- | ||
| Ongoing Scientific Collaboration | Workshops | FDA will participate in workshops with scientific stakeholders to further the science toward development of guidance documents in the following areas: Predictive Toxicology, Biomarker Qualification, Missing Data | X | X | X | X | X |
| Benefit/Risk Assessment | Workshops and Public Meetings | Participate in workshops and public meetings to explore new approaches to a structured model for benefit/risk assessment. Determine if pilots should be conducted or guidance documents issued. | X | X | X | X | X |
VII. Postmarketing Study Commitments
| Performance Area | Initiative | Commitment | Performance Level and/or Implementation Timeline by Fiscal Year -- Not applicable X Action due |
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|---|---|---|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | ||||
| Postmarketing Study Commitments | FDA will develop harmonized (CBER/CDER) standard operating procedures that articulate the Agency’s policy and procedures (e.g., timing, content, rationale and vetting process) for requesting that applicants agree in writing to voluntary postmarketing study commitments. | The SOPs will be finalized prior to the end of FY 2008. | X | -- | -- | -- | -- | |
| In developing these SOPs, the Agency will take into consideration the findings of the contractor study of current Agency procedures to be completed during FY 2007. FDA will make available a releasable version of the final report within 2 months of receipt from the contractor. | X | X | -- | -- | -- | |||
| Training will be provided to all CBER and CDER review staff on the harmonized (CBER/CDER) standard operating procedures. Training will continue for all new review staff and refresher training will be provided to all review staff as necessary through FY 2012. | X | X | X | X | X | |||
XIII. IMPROVING FDA PERFORMANCE MANAGEMENT
| Performance Area | Initiative | Commitment | Performance Level and/or Implementation Timeline by Fiscal Year -- Not applicable X Action due |
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|---|---|---|---|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | |||||
| Improving FDA Performance Management |
Studies will include: 1. Assessment of the impact of the electronic submission and review environment on the efficiency and effectiveness of the overall process for the review of human drugs. 2. Assessment of the progress toward full implementation of Good Review Management Principles, focusing on both FDA reviewer practices and industry sponsor practices affecting successful implementation. 3. Assessment by an independent accounting firm of the review activity adjustment methodology (as described in section 736(c)(2) that is applied in FY 2009 with recommendations for changes, if warranted. |
Complete the assessment of the review activity adjustment methodology in FY 2009 prior to fee setting for FY 2010. Complete the electronic review and GRMPs assessments as appropriate during PDUFA IV. | --- | X | --- | --- | --- | ||
V. INFORMATION TECHNOLOGY GOALS
| Initiatives | Implementation Deadline by Fiscal Year -- Not applicable X Action due |
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|---|---|---|---|---|---|---|---|
| 2008 | 2009 | 2010 | 2011 | 2012 | |||
| Develop and periodically update an IT plan, covering a rolling five-year planning horizon. | X | X | X | X | X | ||
| Develop, implement, and maintain new information systems consistently across all organizational divisions participating in the process for the review of human drug applications, and in compliance with the IT plan, the FDA’s program-wide governance process, the FDA’s target enterprise architecture, and with HHS enterprise architecture standards. The consistency of development, implementation, and maintenance of new information systems will be determined by the FDA based on considerations of program efficiency and effectiveness. Emphasis will be placed on the consistency of interactions with regulated parties and other external stakeholders | X | X | X | X | X | ||
| Update technical specifications and IT-related guidance documents as necessary to reflect consistent program-wide implementation of new information systems supporting electronic information exchange between FDA and regulated parties and other external stakeholders. | X | X | X | X | X | ||
| Extend the capability of the secure electronic single point of entry to include two-way transmission of regulatory correspondence. | X | X | X | X | X | ||
|
Establish an automated standards-based regulatory submission and review environment for INDs, NDAs, and BLAs, and their supplements, that enables the following functions over the life cycle of the product: (1) Electronic IND, NDA, and BLA submissions received by FDA can be archived to enable retrieval through standardized automated links; (2) Electronic IND, NDA, and BLA submissions can include cross-references to previously submitted electronic materials through standardized automated links; and (3) Archived electronic IND, NDA, and BLA submissions can be retrieved through standardized automated links. |
X | X | X | X | X | ||
| Establish a system for electronic exchange and management of human drug labeling information in a modular manner (e.g., at the label section level) that is based on FDA standards and that enables revision tracking. | X | X | X | X | X | ||
| Establish standards-based information systems to support how FDA obtains and analyzes post-market drug safety data and manages emerging drug safety information, as described in Section VIII addressing the enhancement and modernization of the FDA drug safety system. | X | X | X | X | X | ||
Definitions of Terms
A. The term “review and act on” means 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. B. Under PDUFA I and II, receipt of a major amendment to original NDAs and BLAs in the last 3 months extended the goal date by 3 months. Under PDUFA III, this extension also applies to efficacy supplements and Class 2 resubmitted NDAs, BLAs, and efficacy supplements. Receipt of a major amendment to a manufacturing supplement in the last 2 months extends the goal date by 2 months (PDUFA III submissions only). C. A resubmitted original application is a complete response to an action letter addressing all identified deficiencies. D. Class 1 resubmitted applications are applications resubmitted after a complete response letter (or a not approvable or approvable letter) that include the following items only (or combinations of these items):
1. Final printed labeling
2. Draft labeling
3. Safety updates submitted in the same format, including tabulations, as the original safety submission with new data and changes highlighted (except when large amounts of new information, including important new adverse experiences not previously reported with the product, are presented in the resubmission)
4. Stability updates to support provisional or final dating periods
5. Commitments to perform Phase 4 studies, including proposals for such studies
6. Assay validation data
7. Final release testing on the last 1‑2 lots used to support approval
8. A minor reanalysis of data previously submitted to the application (determined by the agency as fitting the Class 1 category)
9. Other minor clarifying information (determined by the agency as fitting the Class 1 category)
10. Other specific items may be added later as the agency gains experience with the scheme and will be communicated via guidance documents to industry E. Class 2 resubmissions are resubmissions that include any other items, including any item that would require presentation to an advisory committee. F. A Type A Meeting is a meeting that is necessary for an otherwise stalled drug development program to proceed (a “critical path” meeting). G. A Type B Meeting is a 1) pre‑IND, 2) end of Phase 1 (for Subpart E or Subpart H or similar products) or end of Phase 2/pre‑Phase 3, or 3) a pre‑ NDA/BLA meeting. Each requestor should usually only request 1 each of these Type B meetings for each potential application (NDA and BLA) (or combination of closely related products, i.e., same active ingredient but different dosage forms being developed concurrently). H. A Type C Meeting is any other type of meeting.
Next page: Appendix B: List of Approved Applications
