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  1. 1. FDA AA - Title I PDUFA IV & FDA’s Performance Goals Friday, 30 November 2007 MBC Naseem Kabir Associate Director Regulatory Affairs
  2. 2. FDAAA & FDA’s PERFORMANCE GOALS Section B Performance Goals & Procedures for Advisory Review of DTC Television Advertising Section 104 (Fees Relating to Advisory Review of Prescription Drug TV Advertising) Section A I. Review Performance Goals II. NME Performance Goals III. Meeting Management IV. Clinical Holds V. Major Dispute Resolution VI. Special Protocol Assessments VII. Additional Procedures VIII. Enhancement and Modernization of the FDA Drug Safety System IX. Review of Proprietary Names to Reduce Medication Errors X. First Cycle Review Performance XI. Expediting Drug Development XII. Post-Marketing Study Commitments XIII. Improving FDA Performance Management XIV. Information Technology Goals Section 103 (Authority to Assess & Use Drug Fees) Performance Goals FDAAA (Title I)
  3. 3. PDUFA IV (Parts I-VII): What is New? <ul><li>FDA’s fee revenue amounts have increased </li></ul>$25 million -- Drug Safety $153.137 $86.434 Application/Supplement $153.137 $86.433 Product $153.137 $86.433 Establishment $392.7 million $259.3 million Total Fee Revenue Fiscal Year 2008 Fiscal Year 2007 Type of Fee Revenue
  4. 4. PDUFA IV (Parts I-VII): What is New? <ul><li>Additional $225 million of revenues for drug safety reflected in user fees </li></ul><ul><ul><li>$25,000,000 for fiscal year 2008; </li></ul></ul><ul><ul><li>$35,000,000 for fiscal year 2009; </li></ul></ul><ul><ul><li>$45,000,000 for fiscal year 2010; </li></ul></ul><ul><ul><li>$55,000,000 for fiscal year 2011; and </li></ul></ul><ul><ul><li>$65,000,000 for fiscal year 2012 </li></ul></ul>
  5. 5. PDUFA IV (Parts I-VII): What is New? <ul><li>Additional criteria for orphan drugs to request exemption from product and establishment fees </li></ul><ul><ul><li>The drug is owned / licensed and is marketed by a company that had less than $50,000,000 in gross worldwide revenue during the previous year </li></ul></ul><ul><ul><ul><li>Must submit certification that gross annual revenues < $50,000,000 for the preceding 12 months before the exemption was requested </li></ul></ul></ul>
  6. 6. PDUFA IV (Parts I-VII): What Remained the Same? <ul><li>FDA’s performance goals for application reviews </li></ul><ul><li>Orphan drugs continue to remain exempt from user fees for review of applications </li></ul><ul><li>Performance goals for clinical holds, major dispute resolution and SPAs </li></ul><ul><li>Requirement to pay annual prescription drug establishment fees and product fees </li></ul>
  7. 7. PDUFA IV (Parts I-VII): What Remained the Same? Manufacturing Supplements Original & Resubmitted NDAs/BLAs & Efficacy Supplements 90% in 6m 90% in 6m Class 2 90% in 2m 90% in 2m Class 1 Resubmitted Efficacy Supplements 90% in 6m 90% in 10m Original Efficacy Supplements 90% in 6m 90% in 6m Class 2 Resubmissions 90% in 2m 90% in 2m Class 1 Resubmissions 90% in 6m 90% in 10m Original Applications Priority Standard SUBMISSION COHORT 90% in 4m (PAS) 90% in 6m (CBE) FY 2008-2012
  8. 8. PDUFA IV (Parts I-VII): What Changed Slightly? <ul><li>An application/supplement that is submitted but refused for filing, or withdrawn before being accepted/refused for filing is subject to full review fee upon resubmission (unless waived/reduced) </li></ul><ul><li>FDA’s performance goals for meeting management remain unchanged except: </li></ul><ul><ul><li>Type B and C: 90% within 21 calendar days of FDA receipt </li></ul></ul>
  9. 9. PDUFA IV (Parts I-VII): What Changed Slightly? User fee increases 1,178,000 589,000 589,000 392,700 65,030 896,200 448,100 448,100 331,100 49,750 <ul><li>Applications </li></ul><ul><ul><li>Requiring clinical data </li></ul></ul><ul><ul><li>Not requiring clinical data </li></ul></ul><ul><ul><li>Supplements requiring clinical data </li></ul></ul><ul><li>Annual Establishment Fee </li></ul><ul><li>Annual Product Fee </li></ul>Fiscal year 2008 Fiscal year 2007 Submission Type
  10. 10. Part VIII: Enhancement and Modernization of the FDA Drug Safety System <ul><li>FDA to develop a 5-year plan to modernize drug safety activities & systems, emphasizing: </li></ul><ul><ul><li>new methodologies in the collection of AE info throughout the product life cycle; </li></ul></ul><ul><ul><li>epidemiology best practices and guidances; </li></ul></ul><ul><ul><li>CDER/CBER database acquisition and expansion; </li></ul></ul><ul><ul><li>validation, development of risk management / communication tools; </li></ul></ul><ul><ul><li>improved post-market IT systems / linkages, and </li></ul></ul><ul><ul><li>enhanced coordination between FDA offices and teams (CDER and CBER, pre-market and post-market) </li></ul></ul>
  11. 11. Part VIII: Enhancement and Modernization of the FDA Drug Safety System <ul><li>FDA will conduct and support activities designed to modernize PV by: </li></ul><ul><ul><li>Maximizing public health benefit of AE collection throughout product life cycle </li></ul></ul><ul><ul><ul><li>RFP published in 2008, awarded 2009, completed by 2011 </li></ul></ul></ul><ul><ul><ul><li>Contractors will critically review US, ex-US AE collection </li></ul></ul></ul><ul><ul><li>B. Developing an epidemiology ‘best practices’ and guidance document </li></ul></ul><ul><ul><ul><li>Public workshop in 2008; review current practice in US, ex-US </li></ul></ul></ul><ul><ul><ul><li>CDER and CBER to release joint guidance in 2010 </li></ul></ul></ul>
  12. 12. Part VIII: Enhancement and Modernization of the FDA Drug Safety System <ul><li>FDA will conduct and support activities designed to modernize PV by: (cont..) </li></ul><ul><ul><ul><li>C. Expanding database resources </li></ul></ul></ul><ul><ul><ul><ul><li>Not just AERS, but population-based epidemiologic data </li></ul></ul></ul></ul><ul><ul><ul><ul><li>Targeted PM surveillance, signal detection </li></ul></ul></ul></ul><ul><ul><ul><ul><li>FDA to purchase epi (observational) DB, hire additional epi staff </li></ul></ul></ul></ul><ul><ul><ul><li>D. Developing, validating risk management and risk communication tools </li></ul></ul></ul><ul><ul><ul><ul><li>Evaluate effectiveness of RiskMAPs </li></ul></ul></ul></ul><ul><ul><ul><ul><li>Public workshop in 2009: assess current risk management / risk communication options </li></ul></ul></ul></ul><ul><ul><ul><ul><li>Annual systematic review of select risk management programs & tools </li></ul></ul></ul></ul>
  13. 13. Part VIII: Enhancement and Modernization of the FDA Drug Safety System <ul><li>Impact on Industry: </li></ul><ul><ul><li>Outcome of the 5-year plan is likely to bring about a dramatic shift in PV, with consequences to all PV processes and procedures </li></ul></ul><ul><ul><li>Increasing importance of risk management PE will impact resources, training, and necessary skill sets </li></ul></ul><ul><ul><li>PE will become increasingly critical to support PM safety activities and commitments </li></ul></ul><ul><ul><li>IT systems thus created will need to be updated to accommodate new requirements </li></ul></ul><ul><ul><li>PDUFA IV represents a significant shift towards a life-cycle approach to risk management </li></ul></ul>
  14. 14. PART IX: REVIEW OF PROPRIETARY NAMES <ul><li>Objective is to reduce medication errors </li></ul><ul><li>Opportunity to submit brand name during the IND process (instead of during NDA) and to obtain FDA feedback earlier </li></ul><ul><li>Pilot program to be implemented in FY 09 </li></ul><ul><ul><li>Allows companies to submit their research on the brand names, which FDA can evaluate to provide decision </li></ul></ul>
  15. 15. PART X: First Cycle Review Performance <ul><li>Day-74 Letters: Notification of Issues Identified During Filing </li></ul><ul><ul><li>Will identify substantive review issues during the filing review (for original NDA/BLA applications and efficacy supplements) </li></ul></ul><ul><ul><li>Notification also sent if no substantive issues identified </li></ul></ul><ul><ul><li>Not indicative of deficiencies that may be identified later in the review cycle </li></ul></ul><ul><ul><li>To include planned review timelines (including dates for feedback on labeling and post-marketing commitment discussions) </li></ul></ul><ul><ul><ul><li>Allows companies to better plan workload </li></ul></ul></ul><ul><ul><ul><li>Solid strategy on submission content </li></ul></ul></ul><ul><ul><ul><li>Submission of major amendments may nullify timelines </li></ul></ul></ul>
  16. 16. PART XI: Expediting Drug Development <ul><li>FDA to develop guidance documents to further the Critical Path Initiative </li></ul><ul><ul><li>Clinical Hepatotoxicity – FY 2008 </li></ul></ul><ul><ul><li>Non-inferiority Trials – FY 2008 </li></ul></ul><ul><ul><li>Adaptive Trial Designs – FY 2008 </li></ul></ul><ul><ul><li>End of Phase 2(a) Meetings – FY 2008 </li></ul></ul><ul><ul><li>Multiple Endpoints in Clinical Trials – FY 2009 </li></ul></ul><ul><ul><li>Enriched Trial Designs – FY 2010 </li></ul></ul><ul><ul><li>Imaging Stds for Use as an EP in Clinical Trials – FY 2011 </li></ul></ul><ul><li>Ongoing collaboration with scientific community to develop guidances on: </li></ul><ul><ul><li>Predictive Toxicology </li></ul></ul><ul><ul><li>Biomarker Qualification </li></ul></ul><ul><ul><li>Missing Data </li></ul></ul><ul><li>FDA participation in public workshops to explore new approaches to develop a structured model for benefit/risk assessment. Goal is to evaluate pilots and development of guidance documents </li></ul>
  17. 17. PART XII: Post-Marketing Study Commitments <ul><li>FDA commits to: </li></ul><ul><li>Developing harmonized (CBER/CDER) SOPs 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 post-marketing study commitments” </li></ul><ul><ul><li>What will “voluntary” mean to industry? </li></ul></ul><ul><li>SOPs will be finalized prior to the end of FY 08 </li></ul><ul><li>Training will be provided to all CBER and CDER review staff on the SOPs as necessary through FY 12 </li></ul><ul><li>From industry perspective : </li></ul><ul><li>Must strategically consider consequences of PMCs and negotiate accordingly </li></ul><ul><li>Part XII extends possibility of FDA requesting PMCs for previously approved drugs </li></ul>
  18. 18. PART XIII: IMPROVING FDA PERFORMANCE MANAGEMENT <ul><li>FDA will conduct studies to: </li></ul><ul><ul><li>Assess the impact of the electronic submission and review environment on the efficiency and effectiveness of the overall process for the review of human drugs </li></ul></ul><ul><ul><li>Assess the progress toward full implementation of Good Review Management Principles, focusing on both FDA reviewer practices and industry sponsor practices affecting successful implementation </li></ul></ul>
  19. 19. Part XIV: IT Goals <ul><li>5-year plan will include: </li></ul><ul><ul><li>new safety system (previously referred to under Drug Safety) </li></ul></ul><ul><ul><li>a roadmap for all IT plans intended to support the goal of end-to-end electronic communications </li></ul></ul><ul><ul><li>Plan will be updated as FDA deems necessary to achieve the IT objectives </li></ul></ul><ul><li>Communications and Technical Interactions: </li></ul><ul><ul><li>FDA and industry stakeholders will meet on a quarterly basis to discuss: </li></ul></ul><ul><ul><ul><li>Discuss on-going implementation of the IT Plan </li></ul></ul></ul><ul><ul><ul><li>Status of IT metrics as available </li></ul></ul></ul><ul><ul><ul><li>Potential impacts of future activities on stakeholders </li></ul></ul></ul><ul><ul><ul><li>Revisions to the IT Plan </li></ul></ul></ul>
  20. 20. Part XIV: IT Goals <ul><li>Metrics & Measures </li></ul><ul><li>Numbers and types of applications received (NDA, BLA, IND, etc.) </li></ul><ul><li>Total number of submissions categorized by type of submission </li></ul><ul><li>Total number of submissions in valid electronic format in compliance with FDA standards </li></ul><ul><li>The number and type of failure areas for electronic submissions out of compliance </li></ul><ul><li>Total number of submissions received through the secure electronic single point of entry versus other methods </li></ul><ul><li>Total number of submissions received substantially on paper. </li></ul>
  21. 21. SECTION B FEES RELATING TO ADVISORY REVIEW OF DTC TV ADS <ul><li>Overview of Legislation </li></ul><ul><li>Authorizes FDA to assess/collect user fees for advisory review of drug DTC TV ads </li></ul><ul><ul><li>Medical device DTC TV ads not impacted at this time </li></ul></ul><ul><li>Voluntary Program </li></ul><ul><ul><li>Notify FDA in advance of # of DTC TV ads intended for next FY </li></ul></ul><ul><ul><li>FDA issued FR notice for companies wishing to participate in FY 2008 </li></ul></ul><ul><li>Legally binding commitment to pay fees </li></ul><ul><ul><li>≤ $83,000 per submission in FY2008 </li></ul></ul><ul><ul><li>to b e paid up front for # of reviews requested </li></ul></ul><ul><li>FDA will commit to providing advisory review in specified period of time </li></ul>
  22. 22. For Further Info <ul><li>My contact info </li></ul><ul><li>[email_address] </li></ul><ul><li>FDAAA website </li></ul><ul><li>http://www.fda.gov/oc/initiatives/advance/fdaaa.html#actions </li></ul><ul><li>PDUFA website </li></ul><ul><li>http://www.fda.gov/oc/pdufa/ </li></ul><ul><li>FDA Performance Goals </li></ul><ul><li>http://www.fda.gov/oc/pdufa4/pdufa4goals.html </li></ul>
  23. 23. Questions?