"Global Orphan Drug Market Future Outlook 2020" report highlights: Global Orphan Drug Market Overview Global Orphan Drug Market Segment Analysis FDA & EMA Regulation for Clinical Trials Orphan Designated Drugs Orphan Drug Designation Criteria & Reimbursement Policy by Region Comprehensive Insight on Global Orphan Drugs Clinical Pipeline & Patent Analysis by Company, Country, Indication & Phase Global Orphan Drugs Clinical Pipeline: 697 Drugs Majority Orphan Drugs in Phase-II Trials: 249 Drugs Marketed Orphan Drugs: 274 Drugs

1. Global Orphan Drug Market Future Outlook 2020
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Disease incidences are rapidly escalating across the globe and few rare diseases have emerged
for which effective medications are not available. Occurrence of such disease is not unusual
and their roots could be traced back to genetic or metabolic anomalies. They affect limited
number of individuals and widely uncommon in nature. Such diseases are called rare diseases
and corresponding drugs are known as orphan drugs. The orphan drug status to a particular
formulation depends upon the public health policy due to which number of individuals may
vary from country to country. This fact reflects the marketing opportunity in particular region
but largely limited market size is observed. Previously, the market for orphan drugs was not
as lucrative as it is today. Due to small patient population, the pharmaceutical companies
generally ignored them and hence these drugs were given the designation "orphan”. However,
in 1983, the Orphan Drug Act was passed in the US in order to encourage pharmaceutical
companies to develop orphan drugs followed by enactment of law for orphan drugs by
European Medical Agency in 1999.
Encouragingly, large numbers of orphan drugs have been introduced in global market and
many more are at different stages of clinical trials. Innovative technologies are being
employed for the development of these orphan drugs. For instance, bone marrow stem-cells
are being explored for treatment of Amyotrophic Lateral Sclerosis (ALS). Targeted RNAi
therapies for Duchenne Muscular Dystrophy (DMD) are under investigation. Market
introduction of orphan drugs based on these innovative therapies may take time but their
future seems optimistic due to increasing activity of pharmaceutical companies in this
segment. Moreover, owing to continuous effort of orphan drug developers for several decade,
many medical breakthroughs have been achieved which are also applicable for other diseases.
For instance, statins drugs developed for homozygous familial hypercholesterolemia is widely
prescribed for cardiac disorders.
Strong clinical pipeline of orphan drugs for different rare diseases using innovative
technologies makes them worthy candidates having high pharmacological and
commercialization potential. But, challenges in clinical trials may hamper their growth and
limit their marketing potential. Owing to low patient base it is not always possible to
generate high quality data in clinical trials. Regulatory authorities also understand this issue
due to which some relaxations have been made which is imperative in such scenarios. Their
clinical trials are quite risky and orphan drug developer may run the chances of having high
failure rates. This may lead to loss of valuable time and resources in clinical trials resulting in

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dominance of big pharmaceutical companies in this segment. However, few small/mid-sized
orphan drug developers could also be found in this segment which is developing autoimmune
and cancer therapeutics.
In recent years, several new rare diseases are being included in clinical trials for the
development of orphan drug candidate. Oncology, Genetic diseases, Autoimmune, pulmonary
disease, Central nervous system and others are main focus of orphan drug developers. All of
these diseases have large patient base but their rare forms doesn’t have suitable therapeutics
which reflects the marketing potential of orphan drugs in these segments. Out of these,
oncology segment has got maximum attention followed by rare genetic disorders, neurological
disorder and autoimmune disorders. In a bigger view, orphan drug segment is expected to
grow several folds in coming years due to several favorable factors. Introduction of innovative
technologies, favorable pricing, reimbursement, unmet medical necessities and strong clinical
pipeline could be attributed as important factors responsible for their growth.
"Global Orphan Drug Market Future Outlook 2020" report highlights:
• Global Orphan Drug Market Overview
• Global Orphan Drug Market Segment Analysis
• FDA & EMA Regulation for Clinical Trials Orphan Designated Drugs
• Orphan Drug Designation Criteria & Reimbursement Policy by Region
• Comprehensive Insight on Global Orphan Drugs Clinical Pipeline & Patent Analysis by
Company, Country, Indication & Phase
• Global Orphan Drugs Clinical Pipeline: 697 Drugs
• Majority Orphan Drugs in Phase-II Trials: 249 Drugs
• Marketed Orphan Drugs: 274 Drugs
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Table of Contents
1. Introduction to Orphan Drug
2. Favorable Market Dynamics for Orphan Drugs Commercialization
2.1 Economic Viability
2.2 Drug Patent Expiry
2.3 Financial & Non-Financial Incentives
2.4 Patent & Market Exclusivity
2.5 R&D Investment
3. Global Orphan Drug Market Overview
4. Global Orphan Drug Market Segment Analysis
4.1 Biological & Non-Biological Orphan Drugs
4.2 Therapeutic Segmentation
4.3 Regional Segmentation
5. Global Orphan Drug Clinical Pipeline Overview
6. US Orphan Drug Market Overview
6.1 Orphan Drug Designation Criteria
6.2 Market Overview
6.3 Reimbursement Policy
7. Europe Orphan Drug Market Overview
7.1 Orphan Drug Designation Criteria
7.2 Market Overview
7.3 Reimbursement Policy

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8. Asia Orphan Drug Market Overview
8.1 Orphan Drug Designation Criteria
8.2 Market Overview
8.3 Reimbursement Policy
9. FDA Regulation for Clinical Trials Orphan Designated Drugs
9.1 Content & Format of a Request for Written Recommendations
9.2 Provision for Granting & Refusing Written Recommendations
9.3 Content and Format of a Request for Orphan Drug Designation
9.4 Verification of Orphan Drug Status & Resident Agent for Foreign Sponsor
9.5 Timing of Requests for Orphan Drug Designation & Designation of Already Approved Drugs
9.6 Deficiency Letters and Granting Orphan Drug Designation
9.7 Refusal to Grant Orphan Drug Designation
9.8 Amendment & Change in Ownership to Orphan Drug Designation
9.9 Publication & Revocation of Orphan Drug Designations
9.10 Annual Reports of Holder of Orphan Drug Designation
9.11 Scope & FDA Recognition of Orphan Drug Exclusive Approval
9.12 Protocols for Investigations & Availability of Information
10. EMA Regulations for Clinical Trials of Orphan Designated Drugs
10.1 Committee for Orphan Medicinal Products
10.2 How to Apply for Orphan Designation in Europe
10.3 Marketing Authorization & Market Exclusivity
10.4 Transferring an Orphan Designation to another Sponsor
10.5 Mandatory Submission of Annual Report On Development
10.6 Incentives for Micro, Small and Medium-Sized Enterprises

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10.7 Fee Reductions for Designated Orphan Medicinal Products
10.8 Procedure for Orphan Designation & Incentives for R&D (Regulation (EC) No 141/2000)
11. Asian Regulations for Clinical Trials of Orphan Designated Drugs
11.1 Taiwan Rare Disease and Orphan Drug Act
11.2 Japan Orphan Drug Regulation
12. Global Orphan Drugs Clinical Pipeline Insight & Patent Analysis by Company,
Country, Indication & Phase
12.1 Unknown
12.2 Research
12.3 Preclinical
12.4 Clinical
12.5 Phase-I
12.6 Phase-I/II
12.7 Phase-II
12.8 Phase-II/III
12.9 Phase-III
12.10 Preregistration
12.11 Registered
13. Marketed Orphan Drugs Clinical Insight & Patent Analysis by Company, Country &
Indication
14. Discontinued & Suspended Orphan Drugs in Clinical Trials
14.1 No Development Reported
14.2 Discontinued
14.3 Preregistration-Submission Withdrawal
14.4 Market Withdrawal

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14.5 Suspended
15. Competitive Landscape
15.1 AOP Orphan
15.2 Agenus
15.3 Alexion
15.4 Bristol Myers Squibb
15.5 Biogen Idec
15.6 Celgene
15.7 Eli Lilly
15.8 Genethon
15.9 Genzyme Corporation
15.10 Glaxosmithkline
15.11 Merck
15.12 Novartis Pharmaceuticals
15.13 Orphan Europe
15.14 Pfizer
15.15 Prosensa
15.16 Rare Disease Therapeutics
15.17 Roche
15.18 Sanofi
15.19 Shire
15.20 Teva Pharmaceutical

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List of Figures
Figure 2-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development Time
Figure 2-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs
Figure 3-1: Global Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 3-2: US Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 3-3: Europe Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 3-4: Asia* Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 4-1: Biological & Non Biological Orphan Drug Segment (%), 2014 & 2020
Figure 4-2: Biological & Non Biological Orphan Drug Market (US$ Billion), 2013-2020
Figure 4-3: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2020
Figure 4-4: Regional Markets for Orphan Drugs, 2014 & 2020
Figure 5-1: Global - Orphan Drug Pipeline by Phase (%), 2015
Figure 5-2: Global - Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 5-3: Global - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
Figure 5-4: Global - No Development Reported in Orphan Drug Pipeline by Phase (Numbers),
2015
Figure 5-5: Global - Discontinued Orphan Drug Pipeline by Phase (%), 2015
Figure 5-6: Global - Discontinued Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 5-7: Global - Suspended Orphan Drug Pipeline by Phase (%), 2015
Figure 5-8: Global - Suspended Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 6-1: US Market for Orphan Drugs (US$ Billion), 2012-2020
Figure 6-2: US - Orphan Drug Pipeline by Phase (%), 2015
Figure 6-3: US - Orphan Drug Pipeline by Phase (Number), 2015

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Figure 6-4: US - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
Figure 6-5: US - No Development Reported in Orphan Drug Pipeline by Phase (Number), 2015
Figure 6-6: US - Discontinued Orphan Drug Pipeline by Phase (%), 2015
Figure 6-7: US - Discontinued Orphan Drug Pipeline by Phase (Number), 2015
Figure 6-8: US - Suspended Orphan Drug Pipeline by Phase (%), 2015
Figure 6-9: US - Suspended Orphan Drug Pipeline by Phase (Number), 2015
Figure 7-1: European Market for Orphan Drugs (US$ Billion), 2012-2020
Figure 7-2: Europe - Orphan Drug Pipeline by Phase (%), 2015
Figure 7-3: Europe - Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 7-4: Europe - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
Figure 7-5: Europe - No Development Reported in Orphan Drug Pipeline by Phase (Numbers),
2015
Figure 7-6: Europe - Discontinued Orphan Drug Pipeline by Phase (%), 2015
Figure 7-7: Europe - Discontinued Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 7-8: Europe - Suspended Orphan Drug Pipeline by Phase (%), 2015
Figure 7-9: Europe - Suspended Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 8-1: Asia Pipeline by Phase (%), 2015
Figure 8-2: Asia Pipeline by Phase (Number), 2015
Figure 8-3: No Development Reported Asia Pipeline by Phase (%), 2015
Figure 8-4: No Development Reported Asia Pipeline by Phase (Number), 2015
Figure 8-5: Discontinued Asia Pipeline by Phase (%), 2015
Figure 8-6: Discontinued Asia Pipeline by Phase (Number), 2015
Figure 8-7: Asia - Number of Orphan Drug by Clinical Phase
Figure 11-1: Japan Orphan Drug/Medical Device Designation System

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Figure 11-2: Japan Orphan Drug/Device Designation Process
Figure 15-1: AOP Orphan
Figure 15-2: Agenus Pipeline
Figure 15-3: Genethon Drug Pipeline
Figure 15-4: Genzyme Drug Pipeline
Figure 15-5: Orphan Europe Pipeline
Figure 15-6: Prosensa Drug Pipeline
Figure 15-7: Shire Drug Pipeline
Table 1-1: Orphan Drugs in the US and their Costs
Table 1-2: Orphan Drugs in the EU and their Costs
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