vii
Table 5-2. TPP Panel for the Clinical use and Efficacy attributes (mainly the primary endpoint) of a Liquid
Immunoglobulin G Preparation in the Treatment of Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP). ..........................................................................................................................................51
Table 5-3. TPP Panel Summarizing the Safety Information a Liquid Immunoglobulin G Preparation in the
Treatment of Primary Immunodeficiency. ................................................................................................................52
Table 5-4. Specific Areas to be Covered by the TPP Asset Characterization Module. ................................................54
Table 6-1. Recommended Duration of Repeat-Dose Toxicity Studies to Support the Conduct of Clinical Trials. .....71
Table 7-1. Centralised Procedure Requirements Based on Product Type................................................................... 81
Table 7-2. Regulatory Authorities by Country.......................................................................................................... 81
Table 8-1. Study Outline Document Template......................................................................................................... 91
Table 10-1. Countries With Orphan Drug Regulations and Population Prevalence................................................ 110
Table 10-2. Overview of Orphan Drug Designation Incentives.............................................................................. 111
Table 10-3. Global Health Authority Orphan Drug Office..................................................................................... 113
Table 10-4. Orphan Drug Regulations by Country. ...............................................................................................114
Table 10-5. Orphan Drug Templates and Submission Tips..................................................................................... 114
Table 13-1. Communication Objectives for Sponsor-Regulator Interactions. .........................................................146
Table 13-2. Examples of Open vs. Content-Specific Questions to Regulators......................................................... 147
Table 13-3. Summary of Regulatory Agency Meeting Types. ..................................................................................152
Table 14-1. US Pathways and Exclusivity. ..............................................................................................................156
Table 14-2. FDA Drug and Biologic NDA Application Review Timeframes. .........................................................156
Table 14-3. FDA Programs to Expedite Approval of Drug/Biologic Products Intended to Treat
Serious Conditions................................................................................................................................................. 160
Table 14-4. EU Application Procedures for Drugs and Biologics............................................................................ 162
Table 14-5. EU Standard Timelines for Centralised Applications. ..........................................................................162
Table 14-6. EU Standard Mutual Recognition Procedure Timelines....................................................................... 163
Table 14-7. EU Standard Decentralised Procedure Timelines. ................................................................................164
Table 14-8. Drug MAA Types in EU...................................................................................................................... 165
Table 14-9. EU Mechanisms for Accelerated Drug and Biologics Development and Approval............................... 166
Table 14-10. Japan Drug Filing Fees....................................................................................................................... 168
Table 14-11. Human Drug (Pharmaceutical and Biological) Submission and Application Review Timeframe
and Fees as of April 1, 2019. ..................................................................................................................................171
Table 14-12. TGA Application and Evaluation Fees in Australian Dollars for Prescription Medicines as of
December 2019...................................................................................................................................................... 178
Table 14-13. Overview of the TGA Application Process and Timeframes. ..............................................................180
Table 14-14. TGA Fees in Australia Dollars for Sponsoring Biologicals. .................................................................180
Table 15-1. Comparison of Regulatory and HTA Requirements. ...........................................................................191
Table 15-2. Overview of Specific Country Requirements. ......................................................................................197
Table 17-1. CM Considerations by Phase of Product Lifecycle............................................................................... 223
Table 17-2. Lifecycle Evolution of Oral Antidiabetic Drugs in the US and EU. .....................................................227
Table 17-3. Remicade US Indication Expansion in the First Five Years After Launch. ............................................228
Table 18-1. Examples of Structured Regulatory Goals. ...........................................................................................238
Table 18-2. Best Practice Examples for Clear Regulatory Writing. ..........................................................................241
Table 5-2. TPP Panel for the Clinical use and Efficacy attributes (mainly the primary endpoint) of a Liquid
Immunoglobulin G Preparation in the Treatment of Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP). ..........................................................................................................................................51
Table 5-3. TPP Panel Summarizing the Safety Information a Liquid Immunoglobulin G Preparation in the
Treatment of Primary Immunodeficiency. ................................................................................................................52
Table 5-4. Specific Areas to be Covered by the TPP Asset Characterization Module. ................................................54
Table 6-1. Recommended Duration of Repeat-Dose Toxicity Studies to Support the Conduct of Clinical Trials. .....71
Table 7-1. Centralised Procedure Requirements Based on Product Type................................................................... 81
Table 7-2. Regulatory Authorities by Country.......................................................................................................... 81
Table 8-1. Study Outline Document Template......................................................................................................... 91
Table 10-1. Countries With Orphan Drug Regulations and Population Prevalence................................................ 110
Table 10-2. Overview of Orphan Drug Designation Incentives.............................................................................. 111
Table 10-3. Global Health Authority Orphan Drug Office..................................................................................... 113
Table 10-4. Orphan Drug Regulations by Country. ...............................................................................................114
Table 10-5. Orphan Drug Templates and Submission Tips..................................................................................... 114
Table 13-1. Communication Objectives for Sponsor-Regulator Interactions. .........................................................146
Table 13-2. Examples of Open vs. Content-Specific Questions to Regulators......................................................... 147
Table 13-3. Summary of Regulatory Agency Meeting Types. ..................................................................................152
Table 14-1. US Pathways and Exclusivity. ..............................................................................................................156
Table 14-2. FDA Drug and Biologic NDA Application Review Timeframes. .........................................................156
Table 14-3. FDA Programs to Expedite Approval of Drug/Biologic Products Intended to Treat
Serious Conditions................................................................................................................................................. 160
Table 14-4. EU Application Procedures for Drugs and Biologics............................................................................ 162
Table 14-5. EU Standard Timelines for Centralised Applications. ..........................................................................162
Table 14-6. EU Standard Mutual Recognition Procedure Timelines....................................................................... 163
Table 14-7. EU Standard Decentralised Procedure Timelines. ................................................................................164
Table 14-8. Drug MAA Types in EU...................................................................................................................... 165
Table 14-9. EU Mechanisms for Accelerated Drug and Biologics Development and Approval............................... 166
Table 14-10. Japan Drug Filing Fees....................................................................................................................... 168
Table 14-11. Human Drug (Pharmaceutical and Biological) Submission and Application Review Timeframe
and Fees as of April 1, 2019. ..................................................................................................................................171
Table 14-12. TGA Application and Evaluation Fees in Australian Dollars for Prescription Medicines as of
December 2019...................................................................................................................................................... 178
Table 14-13. Overview of the TGA Application Process and Timeframes. ..............................................................180
Table 14-14. TGA Fees in Australia Dollars for Sponsoring Biologicals. .................................................................180
Table 15-1. Comparison of Regulatory and HTA Requirements. ...........................................................................191
Table 15-2. Overview of Specific Country Requirements. ......................................................................................197
Table 17-1. CM Considerations by Phase of Product Lifecycle............................................................................... 223
Table 17-2. Lifecycle Evolution of Oral Antidiabetic Drugs in the US and EU. .....................................................227
Table 17-3. Remicade US Indication Expansion in the First Five Years After Launch. ............................................228
Table 18-1. Examples of Structured Regulatory Goals. ...........................................................................................238
Table 18-2. Best Practice Examples for Clear Regulatory Writing. ..........................................................................241