vi
Case Studies
Case Study 8-1. Teduglutide and the Selection of a Clinically Meaningful Endpoint................................................ 94
Case Study 8-2. Adverse Events of Special Interest.................................................................................................... 95
Case Study 8-3. Excluding Unacceptable Risk.......................................................................................................... 96
Case Study 13-1. Vascepa for the Treatment of Hypertriglyceridemia..................................................................... 151
Figures
Figure 5-1. Target ProductProfile (TPP) Planning Process. .......................................................................................45
Figure 5-2. Target Product Profile............................................................................................................................. 48
Figure 8-1. Clinical Development Program Reverse-Engineering Principle............................................................... 90
Figure 8-2. Sample Timeline With one Option for Displaying High-Level Information on Program Timing. ..........92
Figure 10-1. Sponsor’s Guide to an Orphan Designation........................................................................................ 109
Figure 14-1. EMA Centralised Procedure Flowchart With Major Milestones. ........................................................163
Figure 14-2. Flow Chart of Drug and Device Review in Japan. ..............................................................................167
Figure 14-3 Japan’s New Approval System for Commercialization of Cellular Therapy Products............................. 169
Figure 14-4. Standard Registration Process with TGA. ...........................................................................................173
Figure 14-5. TGA Priority Registration Process Diagram........................................................................................ 175
Figure 14-6. TGA Provisional Registration Process Diagram. .................................................................................177
Figure 14-7. TGA Regulation of Biologicals. ..........................................................................................................179
Figure 15-1. Multidisciplinary Product Development............................................................................................. 188
Figure 15-2. Key Interactions Between Marketing and Regulatory. ........................................................................190
Figure 15-3. Clinical Development Strategy in Asia. ..............................................................................................194
Figure 15-4. The CTD Triangle.............................................................................................................................. 195
Figure 15-5. Global Technology System for Safety Monitoring.............................................................................. 199
Figure 16-1. Typical Advisory Committee Meeting Seating Arrangement............................................................... 206
Tables
Table 3-1. Sources of Regulatory Intelligence. ..........................................................................................................23
Table 3-2. Websites for Healthcare Product Regulations by Region. .........................................................................24
Table 3-3. Regulatory Intelligence Databases............................................................................................................ 26
Table 3-4. Professional Associations for Regulatory Professionals.............................................................................. 26
Table 3-5. Clinical Trial Databases........................................................................................................................... 27
Table 3-6. Examples of Web Pages for Subscribing to Regulatory Updates............................................................... 28
Table 3-7. Regulatory Agency Twitter Handles......................................................................................................... 28
Table 4-1. Key Global Regulatory Plan Elements......................................................................................................... 32
Table 4-2. Changes a Global Regulatory Plan Should Anticipate, Mitigate or Manage. ............................................32
Table 4-3. Regulatory Intelligence Tool Examples to Understand the Competitive Product Landscape..................... 39
Table 4-4. Regulatory Journals for Understanding Shifting Regulatory Policy. .........................................................40
Table 4-5. Global Regulatory Plan Components—Sample Table of Contents........................................................... 43
Table 5-1. Introductory/Top Table Presenting Certain Drug Key Elements .............................................................50
Case Studies
Case Study 8-1. Teduglutide and the Selection of a Clinically Meaningful Endpoint................................................ 94
Case Study 8-2. Adverse Events of Special Interest.................................................................................................... 95
Case Study 8-3. Excluding Unacceptable Risk.......................................................................................................... 96
Case Study 13-1. Vascepa for the Treatment of Hypertriglyceridemia..................................................................... 151
Figures
Figure 5-1. Target ProductProfile (TPP) Planning Process. .......................................................................................45
Figure 5-2. Target Product Profile............................................................................................................................. 48
Figure 8-1. Clinical Development Program Reverse-Engineering Principle............................................................... 90
Figure 8-2. Sample Timeline With one Option for Displaying High-Level Information on Program Timing. ..........92
Figure 10-1. Sponsor’s Guide to an Orphan Designation........................................................................................ 109
Figure 14-1. EMA Centralised Procedure Flowchart With Major Milestones. ........................................................163
Figure 14-2. Flow Chart of Drug and Device Review in Japan. ..............................................................................167
Figure 14-3 Japan’s New Approval System for Commercialization of Cellular Therapy Products............................. 169
Figure 14-4. Standard Registration Process with TGA. ...........................................................................................173
Figure 14-5. TGA Priority Registration Process Diagram........................................................................................ 175
Figure 14-6. TGA Provisional Registration Process Diagram. .................................................................................177
Figure 14-7. TGA Regulation of Biologicals. ..........................................................................................................179
Figure 15-1. Multidisciplinary Product Development............................................................................................. 188
Figure 15-2. Key Interactions Between Marketing and Regulatory. ........................................................................190
Figure 15-3. Clinical Development Strategy in Asia. ..............................................................................................194
Figure 15-4. The CTD Triangle.............................................................................................................................. 195
Figure 15-5. Global Technology System for Safety Monitoring.............................................................................. 199
Figure 16-1. Typical Advisory Committee Meeting Seating Arrangement............................................................... 206
Tables
Table 3-1. Sources of Regulatory Intelligence. ..........................................................................................................23
Table 3-2. Websites for Healthcare Product Regulations by Region. .........................................................................24
Table 3-3. Regulatory Intelligence Databases............................................................................................................ 26
Table 3-4. Professional Associations for Regulatory Professionals.............................................................................. 26
Table 3-5. Clinical Trial Databases........................................................................................................................... 27
Table 3-6. Examples of Web Pages for Subscribing to Regulatory Updates............................................................... 28
Table 3-7. Regulatory Agency Twitter Handles......................................................................................................... 28
Table 4-1. Key Global Regulatory Plan Elements......................................................................................................... 32
Table 4-2. Changes a Global Regulatory Plan Should Anticipate, Mitigate or Manage. ............................................32
Table 4-3. Regulatory Intelligence Tool Examples to Understand the Competitive Product Landscape..................... 39
Table 4-4. Regulatory Journals for Understanding Shifting Regulatory Policy. .........................................................40
Table 4-5. Global Regulatory Plan Components—Sample Table of Contents........................................................... 43
Table 5-1. Introductory/Top Table Presenting Certain Drug Key Elements .............................................................50