Table of Content

1. What Are Orphan Drugs?


2. Global Orphan Drug Designation Criteria

2.1 US
2.2 Europe
2.3 Asia & Australia
2.3.1 Japan
2.3.2 Taiwan
2.3.3 South Korea
2.3.4 Australia


3. Market Exclusivity & Patent Protection for Orphan Drugs


4. Global Orphan Drug Reimbursement Policy

4.1 US
4.2 Europe
4.3 Asia


5. Global Orphan Drug Market Opportunity Insight 2026

5.1 Global Orphan Drug Sales Opportunity
5.2 Market by Class Variation
5.3 Market by Therapeutic Application
5.4 Market by Regions


6. FDA Regulation for Clinical Trials Orphan Designated Drugs

6.1 Content & Format Of A Request For Written Recommendations
6.2 Provision For Granting & Refusing Written Recommendations
6.3 Content And Format Of A Request For Orphan Drug Designation
6.4 Verification Of Orphan Drug Status & Resident Agent For Foreign Sponsor
6.5 Timing Of Requests For Orphan Drug Designation & Designation Of Already Approved Drugs
6.6 Deficiency Letters And Granting Orphan Drug Designation
6.7 Refusal To Grant Orphan Drug Designation
6.8 Amendment & Change In Ownership To Orphan Drug Designation
6.9 Publication & Revocation Of Orphan Drug Designations
6.10 Annual Reports Of Holder Of Orphan Drug Designation
6.11 Scope & FDA Recognition Of Orphan Drug Exclusive Approval
6.12 Protocols for Investigations & Availability of Information


7. EMA Regulations for Clinical Trials of Orphan Designated Drugs

7.1 Committee for Orphan Medicinal Products
7.2 How to Apply for Orphan Designation in Europe
7.3 Marketing Authorization & Market Exclusivity
7.4 Transferring An Orphan Designation To Another Sponsor
7.5 Mandatory Submission Of Annual Report On Development
7.6 Incentives For Micro, Small And Medium-Sized Enterprises
7.7 Fee Reductions For Designated Orphan Medicinal Products
7.8 Procedure for Orphan Designation & Incentives for R&D ( Regulation (EC) No 141/2000 )


8. Asian Regulations for Clinical Trials of Orphan Designated Drugs

8.1 Taiwan Rare Disease and Orphan Drug Act
8.2 Japan Orphan Drug Regulation


9. Global Orphan Clinical Pipeline Overview

9.1 By Company
9.2 Drug Class
9.3 Formulation
9.4 Indication
9.5 Region
9.6 Priority Status
9.7 Patient Segment
9.8 By Phase


10. Unknown Phase - Orphan Drugs Clinical Pipeline By Company, Country & Indication

10.1 Overview
10.2 Clinical Pipeline Insight


11. Research Phase - Orphan Drugs Clinical Pipeline By Company, Country & Indication

11.1 Overview
11.2 Clinical Pipeline Insight


12. Preclinical Phase - Orphan Drugs Clinical Pipeline By Company, Country & Indication

12.1 Overview
12.2 Clinical Pipeline Insight


13. Clinical Phase - Orphan Drugs Clinical Pipeline By Company, Country & Indication

13.1 Overview
13.2 Clinical Pipeline Insight


14. Phase-I - Orphan Drugs Clinical Pipeline By Company, Country & Indication

14.1 Overview
14.2 Clinical Pipeline Insight


15. Phase-I/II - Orphan Drugs Clinical Pipeline By Company, Country & Indication

15.1 Overview
15.2 Clinical Pipeline Insight


16. Phase-II - Orphan Drugs Clinical Pipeline By Company, Country & Indication

16.1 Overview
16.2 Clinical Pipeline Insight


17. Phase-II/III - Orphan Drugs Clinical Pipeline By Company, Country & Indication

17.1 Overview
17.2 Clinical Pipeline Insight


18. Phase-III - Orphan Drugs Clinical Pipeline By Company, Country & Indication

18.1 Overview
18.2 Clinical Pipeline Insight


19. Preregistration - Orphan Drugs Clinical Pipeline By Company, Country & Indication

19.1 Overview
19.2 Clinical Pipeline Insight



20. Registered - Orphan Drugs Clinical Pipeline By Company, Country & Indication

20.1 Overview
20.2 Clinical Pipeline Insight

21. Marketed Orphan Drugs Clinical Insight By Company, Country & Indication

21.1 Overview
21.2 Clinical Pipeline Insight

22. Competitive Landscape

22.1 AOP Orphan
22.2 Agenus
22.3 Alexion
22.4 Bristol Myers Squibb
22.5 Biogen Idec
22.6 Celgene
22.7 Eli Lilly
22.8 Genethon
22.9 Genzyme Corporation
22.10 Glaxosmithkline
22.11 Merck
22.12 Novartis Pharmaceuticals
22.13 Orphan Europe
22.14 Pfizer
22.15 Prosensa
22.16 Rare Disease Therapeutics
22.17 Roche
22.18 Sanofi
22.19 Shire
22.20 Teva Pharmaceutical



List of Figures


Figure 5-1: Global - Orphan Drugs Market Value (US$ Billion), 2018-2026
Figure 5-2: US - Orphan Drugs Market Value (US$ Billion), 2018-2026
Figure 5-3: Europe - Orphan Drugs Market Value (US$ Billion), 2018-2026
Figure 5-4: Asia* - Orphan Drugs Market Value (US$ Billion), 2018-2026
Figure 5-5: Biological & Non Biological Orphan Drug Segment (%), 2018 & 2026
Figure 5-6: Biological & Non Biological Orphan Drug Market (US$ Billion), 2018-2026
Figure 5-7: Global Orphan Drugs Market by Therapeutic Area, 2018 & 2026
Figure 5-8: Regional Markets for Orphan Drugs, 2018 & 2026
Figure 8-1: Japan Orphan Drug/Medical Device Designation System
Figure 8-2: Japan Orphan Drug/Device Designation Process
Figure 9-1: Global - Orphan Drugs Clinical Pipeline by Company (Number), 2020 till 2026
Figure 9-2: Global - Orphan Drugs Clinical Pipeline by Drug Class (Number), 2020 till 2026
Figure 9-3: Global - Orphan Drugs Clinical Pipeline by Formulation (Number), 2020 till 2026
Figure 9-4: Global - Orphan Drugs Clinical Pipeline by Indication (Number), 2020 till 2026
Figure 9-5: Global - Orphan Drugs Clinical Pipeline by Region (Number), 2020 till 2026
Figure 9-6: Global - Orphan Drugs Clinical Pipeline by Priority Status (Number), 2020 till 2026
Figure 9-7: Global - Orphan Drugs Clinical Pipeline by Patient Segment (Number), 2020 till 2026
Figure 9-8: Global - Orphan Drugs Clinical Pipeline by Phase (Number), 2020 till 2026
Figure 9-9: Global - Orphan Drugs Clinical Pipeline by Phase (%), 2020 till 2026
Figure 10-1: Share of Initial Development Drug in Total Orphan Drug Pipeline, 2020
Figure 11-1: Share of in Research Phase Orphan Drugs in Total Pipeline, 2020
Figure 12-1: Share of in Preclinical Phase Orphan Drugs in Total Pipeline, 2020
Figure 13-1: Share of in Clinical Phase Orphan Drugs in Total Pipeline, 2020
Figure 14-1: Share of in Phase-I Orphan Drugs in Total Pipeline, 2020
Figure 15-1: Share of in Phase-I/II Orphan Drugs in Total Pipeline, 2020
Figure 16-1: Share of in Phase-II Orphan Drugs in Total Pipeline, 2020
Figure 17-1: Share of in Phase-II/III Orphan Drugs in Total Pipeline, 2020
Figure 18-1: Share of in Phase-III Orphan Drugs in Total Pipeline, 2020
Figure 19-1: Share of in Preregistration Orphan Drugs in Total Pipeline, 2020
Figure 20-1: Share of in Registered Orphan Drugs in Total Pipeline, 2020
Figure 21-1: Share of in Marketed Orphan Drugs in Total Pipeline, 2020