Orphan Drug Development for Rare Diseases
iv
Table 7-7. LATAM.........................................................................................................................121
Table 7-8. APAC/ASEAN.............................................................................................................121
Table 7-9. Orphan Designation in Other Countries. .....................................................................122
Table 7-10. LATAM.........................................................................................................................123
Table 7-11. APAC/ASEAN.............................................................................................................124
Table 7-12. Clinical Trial and Marketing Authorization Requirements in Other Countries. ..........125
Table 7-13. LATAM.........................................................................................................................126
Table 7-14. APAC and ASEAN......................................................................................................127
Table 7-15. Market Access/Reimbursement/Pricing in Other Countries.........................................128
Figures
Figure 1-1. Patient Engagement From Early to Late-Stage, Review, Approval, and Postapproval. ...12
Figure 1-2. Overview of the Orphan Drug Approval Process............................................................15
Figure 1-3. Orphan Designation Process. ..........................................................................................16
Figure 1-4. Overview: Emapalumab FDA Approval. ........................................................................29
Figure 1-5. Overview: Avelumab FDA Approval Overview. .............................................................31
Figure 2-1. Sponsor’s Guide to an Orphan Designation....................................................................46
Figure 2-2. Orphan Medicine Development......................................................................................52
Figure 2-3. FDA and EMA Approval Overview of Zolgensma (onasemnogene abeparvovec). ........53
Figure 4-1. Strategy of SAKIGAKE as a Package. ............................................................................72
Figure 4-2. General Timeframe of SAKIGAKE. ..............................................................................75
Figure 4-3. Scheme for Rapid Authorization of Unapproved Drug...................................................77
Figure 4-4. Accelerated Review System Summary.............................................................................78
Figure 4-5. Role of MHLW, PMDA, and NIBIO in the Development of Orphan Drug
Development ..................................................................................................................80
Figure 4-6. The Orphan Drug Designation Procedure.......................................................................81
Figure 4-7. Data Required to be Submitted for Qualifying the Orphan Designation. ......................82
Figure 4-8. Hypothetical Case Study for Clinical Development in Japan..........................................83
Figure 4-9. Typical Case 1—Company-Oriented Clinical Development. .........................................84
Figure 4-10. Typical Case 2—Company-Oriented Clinical Development. .........................................84
Figure 4-11. Ideal Case of Academia-Oriented Research....................................................................84
Figure 4-12. Pragmatic Case for Drug Repositioning-I (New Indication for Known Drugs). ............84
Figure 4-13. Pragmatic Case for Drug Repositioning-II (New Indication for Known Drugs)............84
Figure 4-14. Pragmatic Case for New Molecular Entity......................................................................85
Figure 4-15. Spinraza (Nusinersen) PMDA Approval.........................................................................86
Figure 5-1. Flowchart to Determine Eligibility for Standard Orphan Designation.
(Excluding Financial Viability and new Dosage Form Medicine)...................................92
Figure 5-2. Overview of Zolgensma Approval in the US, EU, Japan, Australia, and Canada............99
Figure 6-1. Overview of Zolgensma Approval in the US, EU, Japan, Australia, and Canada..........110
Abbreviations and Acronyms. ...............................................................................................................135
Glossary.................................................................................................................................................143
Index.....................................................................................................................................................151
iv
Table 7-7. LATAM.........................................................................................................................121
Table 7-8. APAC/ASEAN.............................................................................................................121
Table 7-9. Orphan Designation in Other Countries. .....................................................................122
Table 7-10. LATAM.........................................................................................................................123
Table 7-11. APAC/ASEAN.............................................................................................................124
Table 7-12. Clinical Trial and Marketing Authorization Requirements in Other Countries. ..........125
Table 7-13. LATAM.........................................................................................................................126
Table 7-14. APAC and ASEAN......................................................................................................127
Table 7-15. Market Access/Reimbursement/Pricing in Other Countries.........................................128
Figures
Figure 1-1. Patient Engagement From Early to Late-Stage, Review, Approval, and Postapproval. ...12
Figure 1-2. Overview of the Orphan Drug Approval Process............................................................15
Figure 1-3. Orphan Designation Process. ..........................................................................................16
Figure 1-4. Overview: Emapalumab FDA Approval. ........................................................................29
Figure 1-5. Overview: Avelumab FDA Approval Overview. .............................................................31
Figure 2-1. Sponsor’s Guide to an Orphan Designation....................................................................46
Figure 2-2. Orphan Medicine Development......................................................................................52
Figure 2-3. FDA and EMA Approval Overview of Zolgensma (onasemnogene abeparvovec). ........53
Figure 4-1. Strategy of SAKIGAKE as a Package. ............................................................................72
Figure 4-2. General Timeframe of SAKIGAKE. ..............................................................................75
Figure 4-3. Scheme for Rapid Authorization of Unapproved Drug...................................................77
Figure 4-4. Accelerated Review System Summary.............................................................................78
Figure 4-5. Role of MHLW, PMDA, and NIBIO in the Development of Orphan Drug
Development ..................................................................................................................80
Figure 4-6. The Orphan Drug Designation Procedure.......................................................................81
Figure 4-7. Data Required to be Submitted for Qualifying the Orphan Designation. ......................82
Figure 4-8. Hypothetical Case Study for Clinical Development in Japan..........................................83
Figure 4-9. Typical Case 1—Company-Oriented Clinical Development. .........................................84
Figure 4-10. Typical Case 2—Company-Oriented Clinical Development. .........................................84
Figure 4-11. Ideal Case of Academia-Oriented Research....................................................................84
Figure 4-12. Pragmatic Case for Drug Repositioning-I (New Indication for Known Drugs). ............84
Figure 4-13. Pragmatic Case for Drug Repositioning-II (New Indication for Known Drugs)............84
Figure 4-14. Pragmatic Case for New Molecular Entity......................................................................85
Figure 4-15. Spinraza (Nusinersen) PMDA Approval.........................................................................86
Figure 5-1. Flowchart to Determine Eligibility for Standard Orphan Designation.
(Excluding Financial Viability and new Dosage Form Medicine)...................................92
Figure 5-2. Overview of Zolgensma Approval in the US, EU, Japan, Australia, and Canada............99
Figure 6-1. Overview of Zolgensma Approval in the US, EU, Japan, Australia, and Canada..........110
Abbreviations and Acronyms. ...............................................................................................................135
Glossary.................................................................................................................................................143
Index.....................................................................................................................................................151