1. Introduction to Orphan Drugs

  1.1 Orphan Drug Defined

  1.2 Rising Popularity of Orphan Drugs

2. Why Shift from Non-Orphan to Orphan Drugs?

  2.1 Exhausting Product Pipelines

  2.2 Profitability of Orphan Drugs

  2.3 Increasing R&D Investment

  2.4 Role of Economic Incentives

  2.5 Patent Protection & Market Exclusivity

3. Global Orphan Drug Market Outlook

  3.1 Market Overview

  3.2 Biological & Non-Biological Orphan Drugs

  3.3 Therapeutic Segmentation

  3.4 Regional Segmentation

4. US Orphan Drug Market Outlook

  4.1 Orphan Drug Designation Criteria

  4.2 Market Overview

  4.3 Reimbursement Policy

  4.4 Regulatory Framework

5. Europe Orphan Drug Market Outlook

  5.1 Orphan Drug Designation Criteria

  5.2 Market Overview

  5.3 Reimbursement Policy

  5.4 Regulatory Framework

6. Asia Orphan Drug Market Outlook

  6.1 Orphan Drug Designation Criteria

  6.2 Market Overview

  6.3 Reimbursement Policy

  6.4 Regulatory Framework

 7. Global Orphan Drug Pipeline Insight by Phase, Orphan Designated Disease & Country

  7.1 Clinical Phase: Research

  7.2 Clinical Phase: Preclinical

  7.3 Clinical Phase: Phase I

  7.4 Clinical Phase:  Phase I/II

  7.5 Clinical Phase:  Phase II

  7.6 Clinical Phase:  Phase II/III

  7.7 Clinical Phase:  Phase III

  7.8 Clinical Phase:  Preregistration

  7.9 Clinical Phase:  Registered

8. Key Issue to be Resolved

  8.1 High Initial Investment

  8.2 Clinical Study Hurdles

  8.3 Regulatory Hurdles

  8.4 Different Reimbursement Policy

  8.5 Distribution Challenges

9. Competitive Landscape

  9.1 Pfizer

  9.2 Roche

  9.3 Sanofi

  9.4 Rare Disease Therapeutics

  9.5 Glaxosmithkline

  9.6 Merck

  9.7 Novartis Pharmaceuticals

  9.8 Alexion

  9.9 Celgene

  9.10 Biogen Idec

  9.11 Eli Lilly

  9.12 Bristol Myers Squibb

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), 2012-2018

Figure 3-2: Biological & Non Biological Orphan Drug Segment (%), 2012 & 2018

Figure 3-3: Biological & Non Biological Orphan Drug Market (US$ Billion), 2012-2018

Figure 3-4: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2018

Figure 3-5: Regional Markets for Orphan Drugs, 2012 & 2018

Figure 4-1: US Market for Orphan Drugs (US$ Billion), 2012-2018

Figure 4-2: US - Orphan Drug Development by Clinical Phase (%)

Figure 4-3: US - Number of Orphan Drug by Clinical Phase

Figure 5-1: European Market for Orphan Drugs (US$ Billion), 2012-2018

Figure 5-2: Europe - Orphan Drug Development by Clinical Phase (%)

Figure 5-3: Europe - Number of Orphan Drug by Clinical Phase

Figure 6-1: Asia- Orphan Drug Development by Clinical Phase (%)

Figure 6-2: Asia - Number of Orphan Drug by Clinical Phase

Figure 6-3: Japan Orphan Drug/Medical Device Designation System

Figure 6-4: Japan Orphan Drug/Device Designation Process

Figure 7-1: Global - Orphan Drug Development by Clinical Phase (%)

Figure 7-2: Global - Number of Orphan Drug by Clinical Phase

Figure 7-3: Global - Number of Orphan Drug by Clinical Phase & Region

Figure 8-1: Orphan Drug Regulatory Hurdles

List of Tables

Table 2-1: Drugs Patent Expiry in 2014

Table 2-2: Drugs Patent Expiry in 2015

Table 2-3: Drugs Patent Expiry in 2016

Table 2-4: Orphan Drugs in the US and their Costs

Table 2-5: Orphan Drugs in the EU and their Costs

Table 2-6: Incentives for the Development of Orphan Drugs

Table 2-7: Orphan Drug Incentives by Country

Table 7-1: Global - Orphan Drugs in Clinical Development Phase: Research

Table 7-2: Global -Orphan Drugs in Clinical Development Phase: Preclinical

Table 7-3: Global - Orphan Drugs in Clinical Development Phase I

Table 7-4: Global - Orphan Drugs in Clinical Development Phase I/II

Table 7-5: Global - Orphan Drugs in Clinical Development Phase II

Table 7-6: Global - Orphan Drugs in Clinical Development Phase II/III

Table 7-7: Global - Orphan Drugs in Clinical Development Phase III

Table 7-8: Global - Orphan Drugs in Clinical Development Phase: Preregistration

Table 7-9: Global - Orphan Drugs in Clinical Development Phase: Registered