世界のオーファンドラッグ（希少疾患治療薬）治験及び制度動向...市場調査レポートについてご紹介

1. What Are Orphan Drugs?
1.1 Introduction To Orphan Drug
1.2 Favorable Parameters For Orphan Drugs Acceptance
1.3 Economic Viability Of Orphan Drugs
1.4 Patent & Market Exclusivity Advantage

2. Global Orphan Drug Market Outlook
2.1 Market Overview
2.1.1 By Biological & Non-Biological Orphan Drugs
2.1.2 By Therapeutic Segmentation
2.1.3 By Regional Segmentation
2.2 Global Orphan Drug Clinical Pipeline Overview

3. Orphan Drug Designation Criteria
3.1 US Orphan Drug Designation Criteria
3.2 Europe Orphan Drug Designation Criteria

4. Reimbursement Policy For Orphan Drug
4.1 US Orphan Drug Reimbursement Policy
4.2 Europe Orphan Drug Reimbursement Policy

5. US (FDA) Regulatory Framework For Orphan Drugs
5.1 Content & Format Of A Request For Written Recommendations
5.2 Provision For Granting & Refusing Written Recommendations
5.3 Content And Format Of A Request For Orphan Drug Designation
5.4 Verification Of Orphan Drug Status & Resident Agent For Foreign Sponsor
5.5 Timing Of Requests For Orphan Drug Designation & Designation Of Already Approved Drugs
5.6 Deficiency Letters And Granting Orphan Drug Designation
5.7 Refusal To Grant Orphan Drug Designation
5.8 Amendment & Change In Ownership To Orphan Drug Designation
5.9 Publication & Revocation Of Orphan Drug Designations
5.10 Annual Reports Of Holder Of Orphan Drug Designation
5.11 Scope & FDA Recognition Of Orphan Drug Exclusive Approval
5.12 Protocols for Investigations & Availability of Information

6. Europe (EMA) Regulatory Framework For Orphan Drugs
6.1 Committee for Orphan Medicinal Products
6.2 How to Apply for Orphan Designation in Europe
6.3 Marketing Authorization & Market Exclusivity
6.4 Transferring An Orphan Designation To Another Sponsor
6.5 Mandatory Submission Of Annual Report On Development
6.6 Incentives For Micro, Small And Medium-Sized Enterprises
6.7 Fee Reductions For Designated Orphan Medicinal Products
6.8 Procedure for Orphan Designation & Incentives for R&D ( Regulation (EC) No 141/2000 )

7. Asia Regulatory Framework For Orphan Drugs
7.1 Taiwan Rare Disease & Orphan Drug Act
7.2 Japan Orphan Drug Regulation

8. Global Orphan Drug Pipeline by Company, Country, Indication & Phase
8.1 Unknown
8.2 Research
8.3 Preclinical
8.4 Clinical
8.5 Phase-I
8.6 Phase-I/II
8.7 Phase-II
8.8 Phase-II/III
8.9 Phase-III
8.10 Preregistration
8.11 Registered

9. Marketed Orphan Drug by Company, Country & Indication

10. Discontinued & Suspended Orphan Drugs in Clinical Trials
10.1 No Development Reported
10.2 Discontinued
10.3 Market Withdrawal
10.4 Suspended

11. Competitive Landscape
11.1 AOP Orphan
11.2 Agenus
11.3 Alexion
11.4 Bristol Myers Squibb
11.5 Biogen Idec
11.6 Celgene
11.7 Eli Lilly
11.8 Genethon
11.9 Genzyme Corporation
11.10 Glaxosmithkline
11.11 Merck
11.12 Novartis Pharmaceuticals
11.13 Orphan Europe
11.14 Pfizer
11.15 Prosensa
11.16 Rare Disease Therapeutics
11.17 Roche
11.18 Sanofi
11.19 Shire
11.20 Teva Pharmaceutical

Figure 1-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development Time
Figure 1-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs
Figure 2-1: Global Orphan Drugs Market Value (US$ Billion), 2013-2018
Figure 2-2: US Orphan Drugs Market Value (US$ Billion), 2013-2018
Figure 2-3: Europe Orphan Drugs Market Value (US$ Billion), 2013-2018
Figure 2-4: Asia* Orphan Drugs Market Value (US$ Billion), 2013-2018
Figure 2-5: Biological & Non Biological Orphan Drug Segment (%), 2014 & 2018
Figure 2-6: Biological & Non Biological Orphan Drug Market (US$ Billion), 2013-2018
Figure 2-7: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2018
Figure 2-8: Regional Markets for Orphan Drugs, 2014 & 2018
Figure 2-9: Global Orphan Drug Pipeline by Phase (%), 2015
Figure 2-10: Global Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 2-11: No Development Reported Global Orphan Drug Pipeline by Phase (%), 2015
Figure 2-12: No Development Reported Global Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 2-13: Discontinued Global Orphan Drug Pipeline by Phase (%), 2015
Figure 2-14: Discontinued Global Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 2-15: Suspended Global Orphan Drug Pipeline by Phase (%), 2015
Figure 2-16: Suspended Global Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 7-1: Japan Orphan Drug/Medical Device Designation System
Figure 7-2: Japan Orphan Drug/Device Designation Process
Figure 11-1: AOP Orphan
Figure 11-2: Agenus Pipeline
Figure 11-3: Genethon Drug Pipeline
Figure 11-4: Genzyme Drug Pipeline
Figure 11-5: Orphan Europe Pipeline
Figure 11-6: Prosensa Drug Pipeline
Figure 11-7: Shire Drug Pipeline

Table 1-1: Orphan Drugs in the US and their Cost
Table 1-2: Orphan Drugs in the EU and their Cost