Regulation of Regenerative Medicines: A Global Perspective
iv
Chapter 10 Regulation of Regenerative Medicines in the US. ..............................................................99
By Stephen Westover, RAC and William Sietsema, PhD
Chapter 11 Understanding the Regulatory Framework for Regenerative Medicines in the
European Union and the United Kingdom ......................................................................117
By Christiane Niederlaender, PhD
Chapter 12 Regulation of Cell and Gene Therapies in Canada. ..........................................................129
By Aileen J. Zhou, MSc, PhD
Chapter 13 Regulation of Regenerative Medicines in Japan................................................................143
By Tomoki Yokoyama, MSc and Brett Snyder, RAC
Chapter 14 Cell and Gene Therapy Product Regulation in China.......................................................163
By Yingying Liu, MSc, Murphy Mao, MSc, Linxi Wu, PhD, and Anette Hjelmsmark, MSc
Chapter 15 Regulation of Regenerative Medicines in Australia and New Zealand.............................177
By Orin Chisholm, PhD, SFHEA, FRAPS
Chapter 16 Regulatory Environment of Cell, Tissue, and Gene Therapy Products in Singapore. .......191
By Stefanie Fasshauer, MBA
Chapter 17 The Future of Regenerative Medicine. ..............................................................................203
By Kristin Van Goor, PhD, Dylan Bechtle, MS, Snehal Naik, PhD, and Rasika Kalameghan, PhD
Figures
Figure 5-1. Vector Heterogeneity .........................................................................................................42
Figure 5-2. Multi-Luminance Mobility Test (MLMT), a Novel Endpoint Developed for Patients
With Inherited Retinal Dystrophy (IRD) Enrolled in Gene Therapy Trials .....................44
Figure 6-1. Framework to Assess the Risk of Gene Therapy-Related Delayed Adverse Events. ...........53
Figure 8-1. Essential Steps for Identifying Product-Specific CQAs and Their Clinical Relevance. ......74
Figure 8-2. Sequential Elements of Achieving Manufacturing Control................................................76
Figure 8-3. Examples of the Existing Platforms Used in Manufacturing Cell and Gene Therapy
Products .............................................................................................................................77
Figure 9-1. Logistics Overview of Autologous, Allogeneic, and Gene Therapies. .................................86
Figure 9-2. Four Main Transportation Lanes of Regenerative Medicines.............................................87
Figure 9-3. Shipping Container Label ..................................................................................................88
Figure 9-4. Status Information in a Tracking Portal Derived From an Active Tracking Device ...........89
Figure 9-5. Supply Chain of Autologous Therapies...............................................................................90
Figure 9-6. Supply Chain of Allogeneic Therapies. ...............................................................................93
Figure 9-7. Allogeneic Logistics and the Need for Storage Facilities ...................................................94
Figure 9-8. Supply Chain of Gene Therapies.........................................................................................95
Figure 9-9. Transportation Lanes of Regenerative Medicines...............................................................96
Figure 10-1. Application of HCT/P Regulations..................................................................................101
Figure 10-2. High-Level Organizational Structure of the Center for Biologics Evaluation and
Research (March 2022).....................................................................................................103
Figure 10-3. Organizational Structure of the Office of Tissues and Advanced Therapies
(March 2022). ...................................................................................................................104
iv
Chapter 10 Regulation of Regenerative Medicines in the US. ..............................................................99
By Stephen Westover, RAC and William Sietsema, PhD
Chapter 11 Understanding the Regulatory Framework for Regenerative Medicines in the
European Union and the United Kingdom ......................................................................117
By Christiane Niederlaender, PhD
Chapter 12 Regulation of Cell and Gene Therapies in Canada. ..........................................................129
By Aileen J. Zhou, MSc, PhD
Chapter 13 Regulation of Regenerative Medicines in Japan................................................................143
By Tomoki Yokoyama, MSc and Brett Snyder, RAC
Chapter 14 Cell and Gene Therapy Product Regulation in China.......................................................163
By Yingying Liu, MSc, Murphy Mao, MSc, Linxi Wu, PhD, and Anette Hjelmsmark, MSc
Chapter 15 Regulation of Regenerative Medicines in Australia and New Zealand.............................177
By Orin Chisholm, PhD, SFHEA, FRAPS
Chapter 16 Regulatory Environment of Cell, Tissue, and Gene Therapy Products in Singapore. .......191
By Stefanie Fasshauer, MBA
Chapter 17 The Future of Regenerative Medicine. ..............................................................................203
By Kristin Van Goor, PhD, Dylan Bechtle, MS, Snehal Naik, PhD, and Rasika Kalameghan, PhD
Figures
Figure 5-1. Vector Heterogeneity .........................................................................................................42
Figure 5-2. Multi-Luminance Mobility Test (MLMT), a Novel Endpoint Developed for Patients
With Inherited Retinal Dystrophy (IRD) Enrolled in Gene Therapy Trials .....................44
Figure 6-1. Framework to Assess the Risk of Gene Therapy-Related Delayed Adverse Events. ...........53
Figure 8-1. Essential Steps for Identifying Product-Specific CQAs and Their Clinical Relevance. ......74
Figure 8-2. Sequential Elements of Achieving Manufacturing Control................................................76
Figure 8-3. Examples of the Existing Platforms Used in Manufacturing Cell and Gene Therapy
Products .............................................................................................................................77
Figure 9-1. Logistics Overview of Autologous, Allogeneic, and Gene Therapies. .................................86
Figure 9-2. Four Main Transportation Lanes of Regenerative Medicines.............................................87
Figure 9-3. Shipping Container Label ..................................................................................................88
Figure 9-4. Status Information in a Tracking Portal Derived From an Active Tracking Device ...........89
Figure 9-5. Supply Chain of Autologous Therapies...............................................................................90
Figure 9-6. Supply Chain of Allogeneic Therapies. ...............................................................................93
Figure 9-7. Allogeneic Logistics and the Need for Storage Facilities ...................................................94
Figure 9-8. Supply Chain of Gene Therapies.........................................................................................95
Figure 9-9. Transportation Lanes of Regenerative Medicines...............................................................96
Figure 10-1. Application of HCT/P Regulations..................................................................................101
Figure 10-2. High-Level Organizational Structure of the Center for Biologics Evaluation and
Research (March 2022).....................................................................................................103
Figure 10-3. Organizational Structure of the Office of Tissues and Advanced Therapies
(March 2022). ...................................................................................................................104