1. Strategies for Preparing for Meetings with FDA Susan M. Mondabaugh, Ph.D. Vice President, Regulatory Affairs Hurley Consulting Associates Ltd. Chatham, NJ

2. Presentation Goals Overview of Types of Sponsor Meetings Overview of Types of Sponsor Meetings Sponsor Best Practices Sponsor Best Practices Pitfalls to Avoid Pitfalls to Avoid

3. Sponsor – FDA Meetings PDUFA 2 Defined 3 Types of Meetings: Type A Type A Needed Immediately for Otherwise Stalled Development Program Needed Immediately for Otherwise Stalled Development Program Type B Type B Pre-IND, End-of-Phase II, Pre-NDA Meetings Pre-IND, End-of-Phase II, Pre-NDA Meetings Type C Type C Any Other Meeting Not a Type A or Type B Any Other Meeting Not a Type A or Type B

4. Managing the Project Team Identifying the Need for an FDA Meeting: What Questions Do We Need Addressed? What Questions Do We Need Addressed? When Do We Need It? When Do We Need It? Do We Need a Face-to-Face Meeting? Is a Teleconference an Option? Do We Need a Face-to-Face Meeting? Is a Teleconference an Option? What is the Goal of a Meeting? What is the Goal of a Meeting? What is the Desired Outcome? What is the Desired Outcome?

5. Pre-IND Meetings Special Considerations: First-in-Man or Phase II/III Study First-in-Man or Phase II/III Study Design and Scope of Nonclinical Program Design and Scope of Nonclinical Program Fast-Track Designation Request Fast-Track Designation Request Orphan Drug Designation Request Orphan Drug Designation Request Specific Safety Issues Specific Safety Issues Not Needed for Every Drug

6. End of Phase II Meetings Agree on Efficacy Criteria for Phase III Studies That Will Be Basis of Approval Agree on Efficacy Criteria for Phase III Studies That Will Be Basis of Approval Endpoints Endpoints Duration of Studies Duration of Studies Number of Studies Number of Studies Comparators Comparators Agree in Principle That Studies Will Support Target Indications Agree in Principle That Studies Will Support Target Indications Discuss Pediatric Requirements Discuss Pediatric Requirements Identify Any Other Requirements Identify Any Other Requirements

7. Pre - NDA Meetings Format and Content of NDA Format and Content of NDA Efficacy and Safety Data Presentations Efficacy and Safety Data Presentations Statistical Analyses Statistical Analyses Datasets and Programs Datasets and Programs Electronic or Paper Electronic or Paper Status of Pediatric Program Status of Pediatric Program Standard or Priority Review Standard or Priority Review Special Issues Special Issues

8. Other Meetings General Guidance General Guidance Agree on FDA Protocol Comments Agree on FDA Protocol Comments Share Information Share Information Renegotiate Phase III Clinical Requirements or Phase IV Commitments Renegotiate Phase III Clinical Requirements or Phase IV Commitments Specific Safety Issues Specific Safety Issues Negotiate Labeling Negotiate Labeling

9. Alleviating Meeting Stress

10. Best Practices Start With the End in Mind! Ultimate Goal is the Patient Ultimate Goal is the Patient Company Goals and Objectives Company Goals and Objectives

11. Meeting Request Follow Guidance Document Follow Guidance Document Clearly Identify Goals and Objective of Meeting Clearly Identify Goals and Objective of Meeting Well-Structured Questions Well-Structured Questions FDA Attendees Needed FDA Attendees Needed Appropriate Technical Representation and Decision Makers Appropriate Technical Representation and Decision Makers Be Realistic Be Realistic

12. Meeting Briefing Package Appropriate Background Information so FDA Can Address the Questions Appropriate Background Information so FDA Can Address the Questions Focused Focused Concise Concise Brief Brief Reviewer Friendly Reviewer Friendly Allow Time for Management Review Prior to Submission Allow Time for Management Review Prior to Submission

13. Meeting Preparation Identify Issues Identify Issues Identify Key Negotiation Points and Acceptable Fallback Positions Identify Key Negotiation Points and Acceptable Fallback Positions No Presentation Unless Requested No Presentation Unless Requested Prepare Only Key Slides That Might Be Needed Prepare Only Key Slides That Might Be Needed Rehearsal Rehearsal Logistics and Travel Logistics and Travel Time of FDA Meeting Time of FDA Meeting

14. Other Preparation Contents of Briefing Package Contents of Briefing Package Relevant Guidance Documents and Regulations Relevant Guidance Documents and Regulations Regulatory History of Any Issues Regulatory History of Any Issues Regulatory Playing Field (Other Drugs) Regulatory Playing Field (Other Drugs)

15. Elements for a Successful Meeting Role of Regulatory Affairs Role of Regulatory Affairs Prepare Responses for Likely Questions Prepare Responses for Likely Questions Identify Potential Speakers Identify Potential Speakers Be Honest Be Honest Use Consultants Judiciously Use Consultants Judiciously Designate Note Takers Designate Note Takers Summarize Key Agreements Summarize Key Agreements

16. Pitfalls to Avoid Introducing New Data Introducing New Data Not Being Prepared Not Being Prepared Being Late Being Late Not Being Empowered to Make Commitments Not Being Empowered to Make Commitments Not Controlling Company Speakers or Consultants Not Controlling Company Speakers or Consultants Arguing Over Policy or Regulation Arguing Over Policy or Regulation

17. Post-Meeting Debrief and Summarize Debrief and Summarize Write Meeting Minutes Write Meeting Minutes Submit Company Minutes and Follow-up to Obtain FDA’s Submit Company Minutes and Follow-up to Obtain FDA’s Review Minutes for Misunderstandings or Discrepancies Review Minutes for Misunderstandings or Discrepancies FDA’s Version is the Official Minutes