Global Pediatric Development of Drugs, Biologics, and Medical Devices
iv
Chapter 9 Regulatory Requirements—Switzerland. .......................................................................107
By Ruchi Gupta, MS and Azzurra Ravizza, MSc
Section V: Asia Pacific
Chapter 10 Drug Development—Japan............................................................................................ 113
By Monique Carter, MS, RAC
Chapter 11 Regulations—Australia and New Zealand. ....................................................................119
By Kathrin Schalper, PhD, RAC
Chapter 12 Regulations—China. ......................................................................................................129
By Yingying Liu, MSc
Chapter 13 Drug Development—India. ...........................................................................................143
By Ashley Butler, MHA
Section VI: Rest of the World
Chapter 14 Drug and Device Requirements—Rest of the World (RoW). .......................................155
By Jing Zhou, MS
Tables
Table 2-1. Pediatric Subpopulations. ...................................................................................................5
Table 2-2. Timeline for Pediatric Drug Regulation in the US. ...........................................................6
Table 2-3. Overview of BPCA and PREA.......................................................................................... 9
Table 2-4. Types of US FDA Meetings............................................................................................. 13
Table 2-5 Initial Pediatric Study Plan Template and Suggested Content from the PSP Guidance. ..18
Table 2-6. Grounds for Waivers and Deferrals. .................................................................................21
Table 2-7. Example from Section 6, Table of Nonclinical and Clinical Development for
Drug X or Biological Product Y. ......................................................................................22
Table 2-8. Examples of Organ System Development Differences..................................................... 25
Table 2-9. Overview of Age-Dependent Development of Organ System by Species........................ 26
Table 3-1. Priority Review Vouchers Awarded Under the Pediatric Rare Disease Program.............. 37
Table 3-2. FDA’s Expedited Programs for Serious Conditions......................................................... 41
Table 4-1. Pediatric Subpopulations. .................................................................................................46
Table 4-2. Additional Pediatric Subpopulations................................................................................ 46
Table 4-3. Examples of Medical Devices Based on Classification ....................................................49
Table 5-1. Key Pediatric Drug Development Events and Policies in Canada.................................... 54
Table 6-1. Countries in Latin America and Caribbean Region......................................................... 59
Table 6-2. Overview of Reference Countries in Latin America and Caribbean Region:
Demographics................................................................................................................... 61
Table 6-3. National Regulatory Authorities of Regional Reference (NRAr) in Latin America
and the Caribbean Region................................................................................................ 63
Table 6-4. Countries of Regional Reference in Latin America and the Caribbean Region with
Orphan Drug Regulation and Population Prevalence. .....................................................63
Table 6-5. Major Rare Disease Patient Advocacy Organizations in Countries of Regional
Reference in Latin America and the Caribbean Region................................................... 64
Table 7-1. Timeline of Key Paediatric Initiatives Undertaken in the EU.......................................... 75
Table 7-2. Overview of Information Required in a PIP. ...................................................................83
iv
Chapter 9 Regulatory Requirements—Switzerland. .......................................................................107
By Ruchi Gupta, MS and Azzurra Ravizza, MSc
Section V: Asia Pacific
Chapter 10 Drug Development—Japan............................................................................................ 113
By Monique Carter, MS, RAC
Chapter 11 Regulations—Australia and New Zealand. ....................................................................119
By Kathrin Schalper, PhD, RAC
Chapter 12 Regulations—China. ......................................................................................................129
By Yingying Liu, MSc
Chapter 13 Drug Development—India. ...........................................................................................143
By Ashley Butler, MHA
Section VI: Rest of the World
Chapter 14 Drug and Device Requirements—Rest of the World (RoW). .......................................155
By Jing Zhou, MS
Tables
Table 2-1. Pediatric Subpopulations. ...................................................................................................5
Table 2-2. Timeline for Pediatric Drug Regulation in the US. ...........................................................6
Table 2-3. Overview of BPCA and PREA.......................................................................................... 9
Table 2-4. Types of US FDA Meetings............................................................................................. 13
Table 2-5 Initial Pediatric Study Plan Template and Suggested Content from the PSP Guidance. ..18
Table 2-6. Grounds for Waivers and Deferrals. .................................................................................21
Table 2-7. Example from Section 6, Table of Nonclinical and Clinical Development for
Drug X or Biological Product Y. ......................................................................................22
Table 2-8. Examples of Organ System Development Differences..................................................... 25
Table 2-9. Overview of Age-Dependent Development of Organ System by Species........................ 26
Table 3-1. Priority Review Vouchers Awarded Under the Pediatric Rare Disease Program.............. 37
Table 3-2. FDA’s Expedited Programs for Serious Conditions......................................................... 41
Table 4-1. Pediatric Subpopulations. .................................................................................................46
Table 4-2. Additional Pediatric Subpopulations................................................................................ 46
Table 4-3. Examples of Medical Devices Based on Classification ....................................................49
Table 5-1. Key Pediatric Drug Development Events and Policies in Canada.................................... 54
Table 6-1. Countries in Latin America and Caribbean Region......................................................... 59
Table 6-2. Overview of Reference Countries in Latin America and Caribbean Region:
Demographics................................................................................................................... 61
Table 6-3. National Regulatory Authorities of Regional Reference (NRAr) in Latin America
and the Caribbean Region................................................................................................ 63
Table 6-4. Countries of Regional Reference in Latin America and the Caribbean Region with
Orphan Drug Regulation and Population Prevalence. .....................................................63
Table 6-5. Major Rare Disease Patient Advocacy Organizations in Countries of Regional
Reference in Latin America and the Caribbean Region................................................... 64
Table 7-1. Timeline of Key Paediatric Initiatives Undertaken in the EU.......................................... 75
Table 7-2. Overview of Information Required in a PIP. ...................................................................83