Rare Diseases Market

Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas

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    March 2025

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Chapter Outlines

Chapter 2  provides a general introduction to rare diseases. We have highlighted the indispensable role played by several regulatory organizations in accelerating this market. The chapter also elaborates upon the various drivers responsible for the growth in this market.

Chapter 3  provides a comprehensive market landscape of drugs in development for rare diseases. This chapter includes an elaborate analysis of pipeline drugs which are currently in different stages of development. The detailed analysis includes information on the targeted indications, types of molecules and the current phases of development. The chapter also provides the historic ladder for the orphan drug designations conferred to several drugs in the US and the European markets.

Chapter 4  offers an insightful discussion of six most popular indications targeted by the drugs under development. This analysis revolves around current landscape, prevailing competition, anticipated shift in the market trend and the key focus for the future. 

Chapter 5  provides detailed company profiles of the top pharmaceutical companies focusing in the field of rare diseases. Each company profile includes information such as financial performance, geographical presence, marketed / pipeline drugs, recent collaborations specific to rare diseases and the initiatives taken by the firm towards the treatment of rare diseases.

Chapter 6  summarises the overall report. In this chapter, we have provided a recap of the key takeaways and our overall opinion on where the market is headed in the mid-long term.

Chapter 7  provides the list of companies covered in this report.

Table of Contents

1. PREFACE
1.1. Overview
1.2. Research Methodology
1.3. Chapter Outlines
 
2. INTRODUCTION
2.1. Overview
2.2. Scope of the Study
2.3. Methodology
2.4. Key Highlights
 
3. RARE DISEASE: PIPLEINE ANALYSIS
3.1. Chapter Overview
3.2. Drugs for Rare Diseases
3.2.1. Drugs for Rare Diseases: Distribution by Highest Phase of Development
3.2.2. Drugs for Rare Diseases: Distribution by Therapeutic Area
3.2.3. Drugs for Oncological Rare Diseases: Distribution by Highest Phase of Development
3.2.4. Drugs for Rare Diseases: Distribution by Nature of Drug
3.3. Regulatory Committees: Impetus to the Rare Disease Drug Market
3.3.1. US Orphan Drug Designations: Timeline
3.3.2. EU Orphan Drug Designations: Timeline
 
4. RARE DISEASES: MARKET POTENTIAL
4.1. Chapter Overview
4.2. Market Potential
4.3. Rare Indications: Market Potential
 
4.3.1. Ovarian Cancer
4.3.1.1. Overview
4.3.1.2. Facts
4.3.1.3. Example Marketed Drugs
4.3.1.4. Pipeline Drugs
4.3.1.5. Roots Analysis’ Perspective
 
4.3.2. Chronic Lymphocytic Leukemia
4.3.2.1. Overview
4.3.2.2. Facts
4.3.2.3. Example Marketed Drugs
4.3.2.4. Pipeline Drugs
4.3.2.5. Roots Analysis’ Perspective
 
4.3.3. Multiple Myeloma
4.3.3.1. Overview
4.3.3.2. Facts
4.3.3.3. Example Marketed Drugs
4.3.3.4. Pipeline Drugs
4.3.3.5. Roots Analysis’ Perspective
 
4.3.4. Gastric Cancer
4.3.4.1. Overview
4.3.4.2. Facts
4.3.4.3. Example Marketed Drugs
4.3.4.4. Pipeline Drugs
4.3.4.5. Roots Analysis’ Perspective
 
4.3.5. Acute Myeloid Leukemia
4.3.5.1. Overview
4.3.5.2. Facts
4.3.5.3. Example Marketed Drugs
4.3.5.4. Pipeline Drugs
4.3.5.5. Roots Analysis’ Perspective
 
4.3.6. Metastatic Breast Cancer
4.3.6.1. Overview
4.3.6.2. Facts
4.3.6.3. Example Marketed Drugs
4.3.6.4. Pipeline Drugs
4.3.6.5. Roots Analysis’ Perspective
 
5. RARE DISEASES: COMPANY PROFILES
5.1. Chapter Overview
5.2. Key Contributors: Potential Analysis
5.3. Key Contributors: R&D Spend
5.4. Key Contributors: Research Spend on Rare Diseases
5.5. Key Contributors: Distribution by Therapeutic Area
 
5.6. Company Profiles
5.6.1. Roche Laboratories
5.6.1.1. Company Overview
5.6.1.2. Financial Performance
5.6.1.3. Rare Disease Drug Portfolio
5.6.1.4. Partnerships
5.6.1.5. Recent Developments
5.6.1.6. Company Initiatives
 
5.6.2. Bristol Myers Squibb
5.6.2.1. Overview
5.6.2.2. Financial Performance
5.6.2.3. Rare Disease Drug Portfolio
5.6.2.4. Partnerships
5.6.2.5. Recent Developments
5.6.2.6. Company Initiatives
 
5.6.3. Celgene
5.6.3.1. Company Overview
5.6.3.2. Financial Performance
5.6.3.3. Rare Disease Drug Portfolio
5.6.3.4. Partnerships
5.6.3.5. Recent Developments
5.6.3.6. Company Initiatives
 
5.6.4. AstraZeneca
5.6.4.1. Company Overview
5.6.4.2. Financial Performance
5.6.4.3. Rare Disease Drug Portfolio
5.6.4.4. Partnerships
5.6.4.5. Recent Developments
5.6.4.6. Company Initiatives
 
5.6.5. Eli Lilly
5.6.5.1. Company Overview
5.6.5.2. Financial Performance
5.6.5.3. Rare Disease Drug Portfolio
5.6.5.4. Partnerships
5.6.5.5. Recent Developments
5.6.5.6. Company Initiatives
 
5.6.6. Pfizer
5.6.6.1. Company Overview
5.6.6.2. Financial Performance
5.6.6.3. Rare Disease Product Portfolio
5.6.6.4. Partnerships
5.6.6.5. Recent Developments
5.6.6.6. Company Initiatives
 
5.6.7. GSK
5.6.7.1. Company Overview
5.6.7.2. Financial Performance
5.6.7.3. Rare Disease Drug Portfolio
5.6.7.4. Partnerships
5.6.7.5. Recent Developments
5.6.7.6. Company Initiatives
 
5.6.8. Boehringer Ingelheim
5.6.8.1. Company Overview
5.6.8.2. Financial Performance
5.6.8.3. Rare Disease Drug Portfolio
5.6.8.4. Partnerships
5.6.8.5. Recent Developments
5.6.8.6. Company Initiatives
 
5.6.9. Amgen
5.6.9.1. Overview
5.6.9.2. Financial Performance
5.6.9.3. Rare Disease Drug Portfolio
5.6.9.4. Partnerships
5.6.9.5. Recent Developments
5.6.9.6. Company Initiatives
 
5.6.10. AbbVie
5.6.10.1. Overview
5.6.10.2. Financial Performance
5.6.10.3. Rare Disease Drug Portfolio
5.6.10.4. Partnerships
5.6.10.5. Recent Developments
5.6.10.6. Company Initiatives
 
5.6.11. Novartis
5.6.11.1. Company Overview
5.6.11.2. Financial Performance
5.6.11.3. Rare Disease Drug Portfolio
5.6.11.4. Partnerships
5.6.11.5. Recent Developments
5.6.11.6. Company Initiatives
 
5.6.12. Sanofi
5.6.12.1. Company Overview
5.6.12.2. Financial Performance
5.6.12.3. Rare Disease Drug Portfolio
5.6.12.4. Partnerships
5.6.12.5. Recent Developments
5.6.12.6. Company Initiatives
 
5.6.13. Takeda
5.6.13.1. Company Overview
5.6.13.2. Financial Performance
5.6.13.3. Rare Disease Drug Portfolio
5.6.13.4. Partnerships
5.6.13.5. Recent Developments
5.6.13.6. Company Initiatives
 
5.6.14. Ono Pharmaceuticals
5.6.14.1. Company Overview
5.6.14.2. Financial Performance
5.6.14.3. Rare Disease Drug Portfolio
5.6.14.4. Partnerships
5.6.14.5. Recent Developments
 
5.6.15. Otsuka
5.6.15.1. Company Overview
5.6.15.2. Financial Performance
5.6.15.3. Rare Disease Drug Portfolio
5.6.15.4. Partnerships
5.6.15.5. Recent Developments
 
5.6.16. Eisai
5.6.16.1. Company Overview
5.6.16.2. Financial Performance
5.6.16.3. Rare Disease Drug Portfolio
5.6.16.4. Partnerships
5.6.16.5. Recent Developments
5.6.16.6. Company Initiatives
 
5.6.17. Daiichi Sankyo
5.6.17.1. Company Overview
5.6.17.2. Financial Performance
5.6.17.3. Rare Disease Drug Portfolio
5.6.17.4. Partnerships
5.6.17.5. Recent Developments
5.6.17.6. Company Initiatives
 
5.6.18. Shire
5.6.18.1. Company Overview
5.6.18.2. Financial Performance
5.6.18.3. Rare Disease Drug Portfolio
5.6.18.4. Partnerships
5.6.18.5. Recent Developments
5.6.18.6. Company Initiatives
 
5.6.19. Johnson & Johnson
5.6.19.1. Company Overview
5.6.19.2. Financial Performance
5.6.19.3. Rare Diseases Drug Portfolio
5.6.19.4. Partnerships
5.6.19.5. Recent Developments
5.6.19.6. Company Initiatives
 
5.6.20. Kyowa Hakko Kirin
5.6.20.1. Company Overview
5.6.20.2. Financial Performance
5.6.20.3. Rare Disease Drug Portfolio
5.6.20.4. Partnerships
5.6.20.5. Recent Developments
 
5.6.21. Astellas
5.6.21.1. Company Overview
5.6.21.2. Financial Performance
5.6.21.3. Rare Disease Drug Portfolio
5.6.21.4. Partnerships
5.6.21.5. Recent Developments
5.6.21.6. Company Initiatives
 
5.6.22. Biogen Idec
5.6.22.1. Company Overview
5.6.22.2. Financial Performance
5.6.22.3. Rare Disease Drug Portfolio
5.6.22.4. Partnerships
5.6.22.5. Recent Developments
5.6.22.6. Company Initiatives
 
5.6.23. Chugai Pharmaceuticals
5.6.23.1. Company Overview
5.6.23.2. Financial Performance
5.6.23.3. Rare Disease Drug Portfolio
5.6.23.4. Partnerships
5.6.23.5. Recent Developments
5.6.23.6. Company Initiatives
5.6.24. Gilead Sciences
5.6.24.1. Company Overview
5.6.24.2. Financial Performance
5.6.24.3. Rare Disease Drug Portfolio
5.6.24.4. Partnerships
5.6.24.5. Recent Developments
5.6.24.6. Company Initiatives
 
5.6.25. Mitsubishi Tanabe Pharma
5.6.25.1. Company Overview
5.6.25.2. Financial Performance
5.6.25.3. Rare Disease Drug Portfolio
5.6.25.4. Partnerships
5.6.25.5. Recent Developments
 
5.6.26. Sumitomo Dainippon
5.6.26.1. Company Overview
5.6.26.2. Financial Performance
5.6.26.3. Rare Disease Drug Portfolio
5.6.26.4. Partnerships
5.6.26.5. Recent Developments
 
5.6.27. ChiesiFarmaceutici
5.6.27.1. Company Overview
5.6.27.2. Financial Performance
5.6.27.3. Rare Disease Drugs Portfolio
5.6.27.4. Partnerships
5.6.27.5. Recent Developments
5.6.27.6. Company Initiatives
 
5.6.28. Teva
5.6.28.1. Company Overview
5.6.28.2. Financial Performance
5.6.28.3. Rare Disease Drug Portfolio
5.6.28.4. Partnerships
5.6.28.5. Recent Developments
5.6.28.6. Company Initiatives
 
5.6.29. Baxter
5.6.29.1. Overview
5.6.29.2. Financial Performance
5.6.29.3. Rare disease Drug Portfolio
5.6.29.4. Partnerships
5.6.29.5. Recent Developments
5.6.29.6. Company Initiatives
 
5.6.30. Grifols
5.6.30.1. Company Overview
5.6.30.2. Financial Performance
5.6.30.3. Rare Disease Drug Portfolio
5.6.30.4. Partnerships
5.6.30.5. Recent Developments
5.6.30.6. Company Initiatives
 
5.6.31. Bayer
5.6.31.1. Company Overview
5.6.31.2. Financial Performance
5.6.31.3. Rare Diseases Drug Portfolio
5.6.31.4. Partnerships
5.6.31.5. Recent Developments
5.6.31.6. Company Initiatives
 
5.6.32. Actelion
5.6.32.1. Company Overview
5.6.32.2. Financial Performance
5.6.32.3. Rare Disease Drug Portfolio
5.6.32.4. Partnerships
5.6.32.5. Recent Developments
 
5.6.33. Ipsen
5.6.33.1. Company Overview
5.6.33.2. Financial Performance
5.6.33.3. Rare Disease Drug Portfolio
5.6.33.4. Partnerships
5.6.33.5. Recent Developments
 
5.6.34. Nippon Shinyaku Pharmaceuticals
5.6.34.1. Company Overview
5.6.34.2. Financial Performance
5.6.34.3. Rare Disease Drug Portfolio
5.6.34.4. Partnerships
5.6.34.5. Recent Developments
 
5.6.35. Merck KGaA
5.6.35.1. Company Overview
5.6.35.2. Financial Performance
5.6.35.3. Rare Disease Drug Portfolio
5.6.35.4. Partnerships
5.6.35.5. Recent Developments
5.6.35.6. Company Initiatives
 
5.6.36. UCB
5.6.36.1. Company Overview
5.6.36.2. Financial Performance
5.6.36.3. Rare Disease Drug Portfolio
5.6.36.4. Company Initiatives
 
5.6.37. Commonwealth Serum Laboratories (CSL)
5.6.37.1. Company Overview
5.6.37.2. Financial Performance
5.6.37.3. Rare Disease Drug Portfolio
5.6.37.4. Partnerships
5.6.37.5. Recent Development
5.6.37.6. Company Initiatives
 
5.6.38. Merck & Co.
5.6.38.1. Company Overview
5.6.38.2. Financial Performance
5.6.38.3. Rare Disease Drug Portfolio
5.6.38.4. Partnerships
5.6.38.5. Recent Developments
5.6.38.6. Company Initiatives
 
5.6.39. Sigma-Tau Pharmaceuticals
5.6.39.1. Company Overview
5.6.39.2. Rare Disease Drug Portfolio
5.6.39.3. Pipeline Drugs
5.6.39.4. Partnerships
5.6.39.5. Recent Developments
5.6.39.6. Company Initiatives
 
5.6.40. Alexion
5.6.40.1. Company Overview
5.6.40.2. Financial Performance
5.6.40.3. Rare Disease Drug Portfolio
5.6.40.4. Partnerships
5.6.40.5. Recent Developments
5.6.40.6. Company Initiatives
 
5.6.41. Recordati
5.6.41.1. Company Overview
5.6.41.2. Financial Performance
5.6.41.3. Rare Disease Drug Portfolio
5.6.41.4. Partnerships
5.6.41.5. Recent Developments
5.6.41.6. Company Initiatives
 
5.6.42. Novo Nordisk
5.6.42.1. Company Overview
5.6.42.2. Financial Performance
5.6.42.3. Rare Disease Drug Portfolio
5.6.42.4. Partnerships
5.6.42.5. Recent Developments
5.6.42.6. Company Initiatives
5.6.43. Shionogi Inc
5.6.43.1. Company Overview
5.6.43.2. Financial Performance
5.6.43.3. Rare Disease Drug Portfolio
5.6.43.4. Partnerships
5.6.43.5. Recent Developments
5.6.43.6. Company Initiatives
 
5.6.44. Vertex Pharmaceuticals
5.6.44.1. Company Overview
5.6.44.2. Financial Performance
5.6.44.3. Rare Disease Drugs Portfolio
5.6.44.4. Partnerships
5.6.44.5. Recent Developments
 
5.6.45. Meiji Seika Pharma Co, Ltd.
5.6.45.1. Company Overview
5.6.45.2. Financial Performance
5.6.45.3. Rare Disease Drug Portfolio
5.6.45.4. Partnerships
 
5.6.46. Ferring Pharmaceuticals
5.6.46.1. Company Overview
5.6.46.2. Rare Disease Drug Portfolio
5.6.46.3. Recent Developments
5.6.46.4. Company Initiatives
 
5.6.47. Orion Pharma
5.6.47.1. Company Overview
5.6.47.2. Financial Performance
5.6.47.3. Rare Disease Drug Portfolio
5.6.47.4. Partnerships
5.6.47.5. Recent Developments
 
5.6.48. Santen
5.6.48.1. Company Overview
5.6.48.2. Financial Performance
5.6.48.3. Rare Disease Drug Portfolio
5.6.48.4. Recent Developments
 
5.6.49. Teijin Pharmaceuticals
5.6.49.1. Company Overview
5.6.49.2. Financial Performance
5.6.49.3. Rare Disease Drug Portfolio
5.6.49.4. Partnerships
5.6.49.5. Recent Developments
 
5.6.50. Lundbeck
5.6.50.1. Company Overview
5.6.50.2. Financial Performance
5.6.50.3. Rare Disease Drug Portfolio
5.6.50.4. Partnerships
5.6.50.5. Recent Developments
5.6.50.6. Company Initiatives
 
6. CONCLUSION
6.1. The Emerging Focus in a Widely Untapped Market
6.2. Several Stakeholders have Collaborated to Lead the Initiatives
6.3. Big Pharma are the Major Contributors
6.4. Industry- Academia Coalition: Expunge the MarketConstraints
6.5. Rare Disease Market: Overall Opportunity is Big
 
7. APPENDIX: LIST OF COMPANIES

List of Figures

Figure 2.1 Rare Disease Drugs Market: Key Takeaways
Figure 3.1 Rare Disease Drugs: Distribution by Highest Phase of Development
Figure 3.2  Rare Disease Drugs: Distribution by Target Indication
Figure 3.3  Rare Disease Drugs for Oncology: Distribution by Highest Phase of Development
Figure 3.4  Rare Disease Drugs: Distribution by Nature of Drug
Figure 3.5  Rare Disease Drugs: US Orphan Drug Designation Timeline
Figure 3.6  Rare Disease Drugs: EU Orphan Drug Designation Timeline
Figure 4.1  Rare Diseases: Market Potential
Figure 4.2 Ovarian Cancer Pipeline Drugs: Distribution by Nature of Drug
Figure 4.3  Ovarian Cancer Pipeline Drugs: Distribution by Highest Phase of Development
Figure 4.4  Ovarian Cancer: Market Potential
Figure 4.5 CLL Pipeline Drugs: Distribution by Nature of Drug
Figure 4.6  CLL Pipeline Drugs: Distribution by Highest Phase of Development
Figure 4.7  CLL: Market Potential
Figure 4.8  Multiple Myeloma Pipeline Drugs: Distribution by Nature of Drug
Figure 4.9  Multiple Myeloma Pipeline Drugs: Distribution by Highest Phase of Development
Figure 4.10  Multiple Myeloma: Market Potential
Figure 4.11  Gastric Cancer Pipeline Drugs: Distribution by Nature of Drug
Figure 4.12  Gastric Cancer Pipeline Drugs: Distribution by Highest Phase of Development
Figure 4.13  Gastric Cancer: Market Potential
Figure 4.14  AML Pipeline Drugs: Distribution by Nature of Drug
Figure 4.15  AML Pipeline Drugs: Distribution by Highest Phase of Development
Figure 4.16  AML: Market Potential
Figure 4.17  Metastatic Breast Cancer Pipeline Drugs: Distribution by Nature of Drug
Figure 4.18  Metastatic Breast Cancer Pipeline Drugs: Distribution by Highest Phase of  Development
Figure 4.19  Metastatic Breast Cancer: Market Potential
Figure 5.1  Key Contributors: Pipeline Strength
Figure 5.2  Key Contributors: R&D Spend
Figure 5.3  Key Contributors: Proxy R&D Spending (USD Billion)
Figure 5.4  Key Contributors: Distribution by Therapeutic Area
Figure 5.5  Roche Revenues, 2009-2014 (CHF Billion)
Figure 5.6  Roche Revenues: Distribution by Business Segments, 2011 – 2014 (CHF Billion)
Figure 5.7  Roche Pharmaceutical Revenues: Distribution by Therapeutic Areas, 2014 (CHF Billion)
Figure 5.8  Roche Pipeline Drugs: Distribution by Nature of Drug
Figure 5.9  Roche Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.10  Roche Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.11  BMS Revenues, 2009-2014 (USD Billion)
Figure 5.12  BMS Revenues: Distribution by Therapeutic Areas, 2014 (USD Billion)
Figure 5.13  BMS Revenues: Distribution by Reported Segments, 2012-2014 (USD Billion)
Figure 5.14  Celgene Annual Revenues, 2009-2014 (USD Million)
Figure 5.15  Celgene Revenues, 2012-2013: Distribution by Business Segment (USD Million)
Figure 5.16  Celgene Pipeline Drugs: Distribution by Nature of Drug
Figure 5.17  Celgene Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.18  Celgene Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.19  AstraZeneca Revenues, 2009-Q3 2014 (USD Million)
Figure 5.20  AstraZeneca Revenues: Distribution by Therapeutic Areas, 2012 – 2013 (USD Billion)
Figure 5.21  AstraZeneca Pipeline Drugs: Distribution by Nature of Drug
Figure 5.22  AstraZeneca Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.23  AstraZeneca Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.24  Eli Lilly Revenues, 2009- 2014 (USD Million, %)
Figure 5.25  Eli Lilly Revenues: Distribution by Business Segments, 2012-2013 (USD Million, %)
Figure 5.26  Eli Lilly Human Pharmaceutical Segment Revenues: Distribution by Operating Segments, 2012- 2013 (USD Million, %)
Figure 5.27  Eli Lilly Pipeline Drugs: Distribution by Nature of Drug
Figure 5.28  Eli Lilly Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.29  Eli Lilly Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.30  Pfizer Annual Revenues, 2011-2014 (USD Million)
Figure 5.31  Pfizer Revenues: Distribution by Business Segments, 2014 (USD Million)
Figure 5.32  Pfizer Revenues: Distribution by Therapeutic Areas, 2012-2013 (USD Million)
Figure 5.33  Pfizer Pipeline Drugs: Distribution by Nature of Drug
Figure 5.34  Pfizer Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.35  GSK Annual Revenues, 2009-2014 (GBP Billion, %)
Figure 5.36  GSK Annual Revenues: Distribution by Business Domains, 2014-2012 (GBP Billion, %)
Figure 5.37 GSK Pharmaceutical Revenues: Distribution by Business Domains 2014, (GBP Billion)
Figure 5.38  GSK Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.39  GSK Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.40  GSK Pipeline Drugs: Distribution by Nature of Drug
Figure 5.41 Boehringer Ingelheim Net Revenues, 2009-2013 (EUR Million, %)
Figure 5.42  Boehringer Ingelheim: Distribution by Business Segments, 2012-2013 (EUR Million)
Figure 5.43  Boehringer Pipeline Drugs: Distribution by Nature of Drug
Figure 5.44  Boehringer Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.45  Boehringer Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.46  Amgen: Annual Revenues, 2009 –2014 (USD Billion)
Figure 5.47  Amgen Pipeline Drugs: Distribution by Nature of Drug
Figure 5.48  Amgen Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.49  Amgen Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.50  AbbVie Annual Revenues, 2012 –2014 (USD Billion)
Figure 5.51  AbbVie Pipeline Drugs: Distribution by Nature of Drug
Figure 5.52  AbbVie Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.53  AbbVie Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.54  Novartis Revenues, 2009-2013 (USD Billion, %)
Figure 5.55   Novartis Revenues: Distribution by Business Segments, 2013 (USD Billion)
Figure 5.56  Novartis Pipeline Drugs: Distribution by Nature of Drug
Figure 5.57  Novartis Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.58  Sanofi Net Revenues, 2009- 2014 (EUR Million, %)
Figure 5.59  Sanofi Revenues: Distribution by Business Segment, 2012-2014 (EUR Million)
Figure 5.60  Sanofi Revenues: Distribution by Therapeutic Areas H1, 2014 (EUR Million)
Figure 5.61  Genzyme Revenues, H1 2014 (EUR Million)
Figure 5.62  Sanofi: Revenues from Rare Disease Drugs, H1 2014 (EUR Million)
Figure 5.63  Sanofi Pipeline Drugs: Distribution by Nature of Drug
Figure 5.64  Sanofi Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.65  Sanofi Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.66 Takeda Revenues, 2010 –Q3 2015 (JPY million, %)
Figure 5.67  Takeda Pipeline Drugs: Distribution by Nature of Drug
Figure 5.68  Takeda Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.69  Takeda Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.70  Ono Pharmaceuticals Revenues, 2009-2013 (JPY Million, %)
Figure 5.71  Ono Pipeline Drugs: Distribution by Nature of Drug
Figure 5.72  Ono Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.73  Ono Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.74  Otsuka Net Revenues, 2009-Q2 2014 (JPY Million, %)
Figure 5.75  Otsuka Revenues: Distribution by Business Segment, 2012-Q2 2014 (JPY Million)
Figure 5.76  Otsuka Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.77  Otsuka Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.78  Eisai Annual Revenues, 2009-Q3 2014 (JPY Billion)
Figure 5.79  Eisai Revenues: Distribution by Reporting Segments, FY 2012-Q3 2015 (JPY Billion)
Figure 5.80  Eisai Pipeline Drugs: Distribution by Nature of Drug
Figure 5.81  Eisai Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.82  Eisai Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.83  Daiichi Sankyo Net Revenues, 2009 – Q3 2014 (JPY Million)
Figure 5.84  Daiichi Pipeline Drugs: Distribution by Nature of Drug
Figure 5.85  Daiichi Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.86  Daiichi Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.87  Shire Annual Revenues, 2009-Q3 2014 (USD Million)
Figure 5.88  Shire Revenues: Distribution by Reporting Segments, 2012-2014 (USD Million)
Figure 5.89  Johnson &Johnson Annual Revenues, 2009-2014 (USD Billion)
Figure 5.90  Johnson &Johnson Revenues: Distribution by Business Segments, 2014 (USD Billion)
Figure 5.91  Kyowa Hakko Kirin Annual Revenues, 2009-2014 (JPY Million, %)
Figure 5.92  Kyowa Kirin Pipeline Drugs: Distribution by Nature of Drug
Figure 5.93  Kyowa Kirin Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.94  Kyowa Kirin Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.95  Astellas Revenues, 2009- Q3 2014 (JPY Billion)
Figure 5.96  Astellas Revenues: Distribution by Business Segment, 2013-2014 (JPY Billion)
Figure 5.97  Biogen Idec Annual Revenues, 2009-2014 (USD Million)
Figure 5.98  Biogen Idec Revenues: Distribution by Segment, 2013-2014 (USD Million)
Figure 5.99  Biogen Idec Revenues: Distribution by Therapeutic Areas, 2014 (USD Million)
Figure 5.100 Chugai Annual Revenues, 2009-2014 (JPY Billion)
Figure 5.101 Chugai Revenues: Distribution by Product Domains, 2012-2014 (JPY Billion)
Figure 5.102 Chugai Pipeline Drugs: Distribution by Nature of Drug
Figure 5.103 Chugai Pipeline Drugs: Distribution by Therapeutic Areas
Figure 5.104 Chugai Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.105 Gilead Sciences Net Revenues, 2009 –2014 (USD Million)
Figure 5.106 Mitsubishi Tanabe Annual Revenues, 2009- Q3 2014 (JPY Billion)
Figure 5.107 Mitsubishi Tanabe Pharmaceuticals Revenues: Distribution by Segments, Q1-Q3 2014 (JPY Billion)
Figure 5.108 Sumitomo Dainippon Annual Revenues, FY2009-Q32014 (JPY Billion)
Figure 5.109 Sumitomo Dainippon Revenues: Distribution by Business Segments, FY 2012-FY 2013 (JPY Billion)
Figure 5.110 Sumitomo Dainippon Revenues: Distribution by Geographical Segments, FY 2012-FY 2013 (JPY Billion)
Figure 5.111 Chiesi Annual Revenues, 2009-2013 (EUR Million)
Figure 5.112 Teva Net Revenues, 2009- 2014 (USD Billion, %)
Figure 5.113 Teva Revenues: Distribution by Business Segments, 2012- 2014 (USD billion)
Figure 5.114 Teva Specialty Revenues: Distribution by Therapeutic Areas (USD Billion, 2014)
Figure 5.115 Teva Pipeline Drugs: Distribution by Therapeutic Area
Figure 5.116 Teva Pipeline Drugs: Distribution by Highest Phase of Development
Figure 5.117 Baxter Annual Revenues, 2009-2014 (USD Billion)
Figure 5.118 Baxter Revenues: Distribution by Business Segment, 2012-2014 (USD Billion)
Figure 5.119 Baxter Revenues: Distribution by Sub-Segments, 2014 (USD Billion)
Figure 5.120 Grifols Annual Revenues:  2009-Q3 2014 (EUR Million, %)
Figure 5.121 Grifols Revenues–Distribution by Business Segments, Q1-Q3 2014 (EUR Million, %)
Figure 5.122 Bayer Revenues, 2009-Q3 2014 (EUR Billion, %)
Figure 5.123 Bayer Revenues: Distribution by Business Segment, 2012-Q3 2014 (EUR Billion)
Figure 5.124 Bayer Revenues: Distribution by Healthcare Segments Q1-Q3 2014 (EUR Million)
Figure 5.125 Actelion Annual Revenues, 2009-2013 (CHF Million)
Figure 5.126 Actelion Revenues: Distribution by Business Segment, 2012-2013 (CHF Million)
Figure 5.127 Ipsen Revenues, 2009- Q3 2014 (EUR Million, %)
Figure 5.128 Ipsen Revenues: Distribution by Business Segments, 2014 (EUR Million) 
Figure 5.129 Ipsen Specialty Revenues: Distribution by Therapeutic Area, 2013-2014 (EUR Million)
Figure 5.130 Ipsen Primary Care Revenues: Distribution by Therapeutic Area, 2013-2014 (EUR Million)
Figure 5.131 Nippon Revenues, 2009- Q3 2014 (JPY Million, %)
Figure 5.132 Nippon Revenues: Distribution by Business Segment, 2012-2014 (JPY Million)
Figure 5.133 Nippon Pharmaceutical segment Revenues: Distribution by Therapeutic Areas, 2012-2014 (JPY Million)
Figure 5.134 Merck Group Revenues, 2009- Q3 2014 (EUR Million, %)
Figure 5.135 Merck Revenues: Distribution by Business Divisions, 2013-Q3 2014(EUR Million)
Figure 5.136 UCB Annual Revenues, 2009-Q3 2014 (EUR Million)
Figure 5.137 CSL Net Revenues, 2009-2014 (USD Million, %)
Figure 5.138 CSL Revenues: Distribution by Business Segment, 2013-2014 (USD Million)
Figure 5.139 Merck Revenues, 2009-2014 (USD Billion)
Figure 5.140 Merck Revenues: Distribution by Business Segment, 2014 (USD Billion)
Figure 5.141 Alexion Revenues, 2009- 2014 (USD Million)
Figure 5.142 Recordati Annual Revenues, 2009-Q3 2014 (EUR Million)
Figure 5.143 Novo Nordisk Annual Revenues, 2009 –2014 (DKK Million)
Figure 5.144 Novo Nordisk Annual Revenues: Distribution by Business Segment, 2013- 2014 (DKK million)
Figure 5.145 Shionogi Annual Revenues, 2009-Q3 2014 (JPY Billion, %)
Figure 5.146 Vertex Revenues, 2009-2014 (USD Million, %)
Figure 5.147 Meiji Seika Revenues, 2009-Q3 2014 (JPY Billion, %)
Figure 5.148 Meiji Seika Revenues: Distribution by Business Segment, 2012-2014 (JPY Billion)
Figure 5.149 Meiji Seika Pharmaceutical Revenues: Distribution by Sub segments, Q1-Q3 2014, (JPY Billion)
Figure 5.150 Orion Revenues, 2009- 2014 (EUR Million, %)
Figure 5.151 Orion Revenues: Distribution by Business Segment, 2012-2014:  (EUR Million)
Figure 5.152 Orion Pharmaceutical Revenues: Distribution by sub-divisions, 2012- 2014 (EUR Million)
Figure 5.153 Santen Annual Revenues, 2009-Q3 2014 (JPY Million)
Figure 5.154 Santen Revenues: Distribution by Business Segment, 2012- Q3 2014 (JPY Million)
Figure 5.155 Teijin Net Revenues, 2009- Q2 2015 (JPY Billion, %)
Figure 5.156 Teijin Revenues: Distribution by Business Segment, 2012- Q3 2015 (JPY Billion)
Figure 5.157 Lundbeck Annual Revenues, 2010- Q3 2014 (DKK Million, %)

List of Tables

Table 3.1 Rare Disease Drugs: Clinical Pipeline
Table 4.1 Ovarian Cancer: Key Facts
Table 4.2  Ovarian Cancer: Example Marketed Rare Disease Drugs 
Table 4.3  Ovarian Cancer: Pipeline Rare Disease Drugs 
Table 4.4  CLL: Key Facts
Table 4.5  CLL: Example Marketed Rare Disease Drugs 
Table 4.6  CLL: Pipeline Rare Disease Drugs 
Table 4.7 Multiple Myeloma: Factual Peep
Table 4.8  Multiple Myeloma: Example Marketed Rare Disease Drugs 
Table 4.9 Multiple Myeloma: Pipeline Rare Disease Drugs 
Table 4.10  Gastric Cancer: Key Facts
Table 4.11  Gastric Cancer: Example Marketed Rare Disease Drugs 
Table 4.12 Gastric Cancer: Pipeline Rare Disease Drugs 
Table 4.13  Acute Myeloid Leukaemia: Key Facts
Table 4.14 AML: Example Marketed Rare Disease Drugs 
Table 4.15 AML: Pipeline Rare Disease Drugs 
Table 4.16 Breast Cancer: Key Facts
Table 4.17 Metastatic Breast Cancer: Marketed Rare Disease Drugs 
Table 4.18 Metastatic Breast Cancer: Pipeline Rare Disease Drugs 
Table 5.1  Roche: Rare Disease Marketed Drugs
Table 5.2:  Roche: Rare Disease Pipeline Drugs
Table 5.3  BMS:  Rare Disease Marketed Drugs
Table 5.4  BMS:  Rare Diseases Pipeline Drugs
Table 5.5  Celgene: Rare Disease Marketed Drugs
Table 5.6  Celgene:  Rare Disease Pipeline Drugs
Table 5.7  AstraZeneca/ MedImmune:  Rare Disease Marketed Drugs
Table 5.8 AstraZeneca/ MedImmune- Rare Disease Pipeline Drugs
Table 5.9  Eli Lilly: Rare Disease Marketed Drugs
Table 5.10  Eli Lilly: Rare Disease Pipeline Drugs
Table 5.11 Pfizer:  Rare Disease Marketed Drugs
Table 5.12  Pfizer-Rare Disease Pipeline Drugs
Table 5.13  GSK: Rare Disease Marketed Drugs 
Table 5.14  GSK: Rare Disease Pipeline Drugs 
Table 5.15  Boehringer Ingelheim: Rare Disease Pipeline Drugs
Table 5.16  Amgen: Rare Disease Marketed Drugs
Table 5.17  Amgen: Rare Disease Pipeline Drugs
Table 5.18  AbbVie: Rare Disease Marketed Drugs
Table 5.19  AbbVie: Rare Disease Pipeline Drugs
Table 5.20  Novartis: Rare Disease Marketed Drugs
Table 5.21  Novartis: Rare Disease Pipeline Drugs
Table 5.22  Sanofi/ Genzyme:  Rare Disease Marketed Drugs
Table 5.23 Sanofi/ Genzyme:  Rare Disease Pipeline Drugs
Table 5.24  Takeda/ Millennium: Rare Disease Pipeline Drugs
Table 5.25 Ono Pharmaceuticals:  Rare Disease Pipeline Drugs 
Table 5.26 Otsuka:  Rare Disease Pipeline Drugs
Table 5.27  Eisai:  Rare Disease Marketed Drugs
Table 5.28 Eisai:  Rare Disease Pipeline Drugs 
Table 5.29  Daiichi Sankyo: Rare Disease Pipeline Drugs
Table 5.30  Shire:  Rare Disease Marketed Drugs
Table 5.31  Shire:  Rare Disease Pipeline Drugs
Table 5.32  Johnson & Johnson: Rare Disease Marketed Drugs
Table 5.33  Johnson & Johnson: Rare Disease Pipeline Drugs
Table 5.34  Kyowa Hakko Kirin: Rare Disease Pipeline Drugs
Table 5.35  Astellas: Rare Disease Marketed Drugs
Table 5.36  Astellas:  Rare Disease Pipeline Drugs
Table 5.37  Biogen Idec:  Rare Disease Marketed Drugs
Table 5.38  Biogen Idec:  Rare Disease Pipeline Drugs
Table 5.39  Chugai:  Rare Disease Pipeline Drugs
Table 5.40 Gilead Sciences: Rare Disease Marketed Drugs
Table 5.41 Gilead Sciences: Rare Disease Pipeline Drugs
Table 5.42 Mitsubishi Tanabe:  Rare Disease Pipeline Drugs
Table 5.43 Sumitomo Dainippon:  Rare Disease Pipeline Drugs
Table 5.44  Chiesi: Rare Disease Pipeline Drugs
Table 5.45  Teva: Rare Disease Marketed Drugs
Table 5.46  Teva: Rare Disease Pipeline Drugs
Table 5.47  Baxter:  Rare Diseases Marketed Drugs
Table 5.48  Baxter: Rare Diseases Pipeline Drugs
Table 5.49 Grifols:  Rare Disease Marketed Drugs
Table 5.50 Grifols: Rare Disease Pipeline Drugs
Table 5.51 Bayer:  Rare Disease Marketed Drugs
Table 5.52 Bayer:  Rare Disease Pipeline Drugs
Table 5.53  Actelion: Rare Disease Marketed Drugs
Table 5.54  Actelion:  Rare Disease Pipeline Drugs
Table 5.55 Ipsen:  Rare Disease Pipeline Drugs
Table 5.56 Nippon Shinyaku:  Rare Disease Pipeline Drugs
Table 5.57 Merck KGaA:  Rare Disease Marketed Drugs
Table 5.58 Merck KGaA:  Rare Disease Pipeline Drugs
Table 5.59  UCB:  Rare Disease Pipeline Drugs
Table 5.60  CSL: Rare Disease Marketed Drugs
Table 5.61  CSL:  Rare Disease Pipeline Drugs
Table 5.62  Merck: Rare Disease Pipeline Drugs
Table 5.63 Merck Partnerships
Table 5.64 Sigma-Tau: Rare Disease Marketed Drugs
Table 5.65 Sigma-Tau:  Rare Disease Pipeline Drugs
Table 5.66  Alexion:  Rare Disease Marketed Drugs 
Table 5.67  Alexion:  Rare Disease Pipeline Drugs
Table 5.68 Recordati:  Rare Disease Marketed Drugs
Table 5.69  Recordati:  Rare Diseases Pipeline Drugs
Table 5.70  Novo Nordisk: Rare Disease Marketed Drugs
Table 5.71  Novo Nordisk: Rare Disease Pipeline Drugs
Table 5.72 Shionogi: Rare Disease Pipeline Drugs
Table 5.73 Vertex:  Rare Disease Pipeline Drugs
Table 5.74 Meiji Seika:  Rare Disease Pipeline Drugs
Table 5.75 Ferring:  Rare Disease Pipeline Drugs
Table 5.76 Teijin:  Rare Diseases Pipeline Drugs
Table 5.77  Santen:  Rare Disease Pipeline Drugs
Table 5.78 Teijin:  Rare Disease Pipeline Drugs
Table 5.79 Lundbeck: Rare Disease Marketed Drugs

Listed Companies

The following companiesand organizations have been mentioned in the report.

  1. Abbott
  2. AbbVie
  3. Abraxis Bioscience Ltd
  4. AC Immune
  5. Accleron
  6. Actelion
  7. Active Biotech
  8. AesRx LLC
  9. Agensys
  10. Agios Pharmaceuticals 
  11. Alexion
  12. Alnara Pharmaceutical
  13. Alnylam
  14. Ambit Biosciences
  15. American BioScience Inc
  16. Amgen
  17. Amicus Therapeutics 
  18. Amor Chem
  19. Amylin Pharmaceuticals
  20. Araclon Biotech
  21. Archimex
  22. ArmaGen
  23. ArQule Inc
  24. Array Biopharma
  25. Arresto Biosciences
  26. Aslan Pharmaceuticals
  27. Astellas Pharma Inc. 
  28. Astex Pharmaceuticals 
  29. AstraZeneca
  30. Avanir
  31. Aventis
  32. Aveo Pharmaceuticals 
  33. Avid Radiopharmaceuticals
  34. Aviesan
  35. Babraham Institute
  36. Banner Alzheimer's Institute
  37. Barcelona Alzheimer’s Treatment and Research Center
  38. Basilia Pharmaceuticals 
  39. Baxter
  40. Bayer
  41. Bial Pharmaceuticals
  42. Bill & Melinda Gates Foundation 
  43. BIO Ventures 
  44. Bioarctic Neuroscience AB
  45. Biogen Idec
  46. BioMarin
  47. Biotie Therapies 
  48. Biovail Laboratories International 
  49. BioXcellence
  50. Blackswan Foundation
  51. BMS
  52. Boehringer Ingelheim
  53. Boston’s Children Hospital 
  54. Brazilian Association for Leukemia and Lymphoma 
  55. Brazilian Association for Thalassemia
  56. Britannia Pharmaceuticals
  57. Broad Institute
  58. Calistoga Pharmaceuticals
  59. Camurus
  60. Cancer Research Technology
  61. Cambridge Institute
  62. Cangene Corporation
  63. Caritas Argentina 
  64. Carter
  65. Catalyst Biosciences
  66. Cavis Pharma
  67. Cedars Sinai Medical Center
  68. Celgene
  69. Cell Therapeutics
  70. Celldex Therapeutics 
  71. Center for iPS Cell Research and Application 
  72. Centocor Inc
  73. Cephalon
  74. Ceptaris Therapeutics.  
  75. Chatham Therapeutics
  76. Chelsea Therapeutics
  77. Chemocentryx Inc.
  78. Chiasma
  79. Chiesi
  80. Children’s Hospital and Research Centre
  81. Chiron  Corporation 
  82. Chugai
  83. Cincinnati Children Hospital 
  84. CIRAD
  85. Cleveland Clinic
  86. Coherus Biosciences
  87. Committee for Medicinal Products for Human Use
  88. Community Chest 
  89. Concordia Pharmaceuticals Inc
  90. Cordis Corporation
  91. CSL
  92. Curie Institute
  93. Cystic Fibrosis Research Inc.
  94. Cystinosis Research Foundation
  95. Cytos Biotechnology
  96. Daiichi Sankyo
  97. Dainippon Pharmaceuticals 
  98. DaniscoBioActives
  99. Debiopharm
  100. Diagnostic Division of the Shionogi &Co. Ltd
  101. Dimension Therapeutics 
  102. Dong A Pharmaceuticals
  103. Drais Pharmaceuticals
  104. DS Pharma Animal Health Co
  105. Duke Clinical Research Institute
  106. Duke University 
  107. Edison Pharmaceuticals 
  108. Eisai
  109. Eli Lilly
  110. Enobia Pharma Corp
  111. EnVivo Pharmaceuticals
  112. Envoy Therapeutics
  113. Enzon Pharmaceuticals 
  114. Epizyme Inc. 
  115. EspeRare Foundation
  116. Eunefron
  117. European Crohn’s and Colitis Organization
  118. European Organisation for Rare Diseases
  119. European Parkinson’s Disease Association
  120. European Porphyria Network (EPNET)
  121. European Respiratory Society Congress 
  122. European Society for Paediatric Research Congress
  123. EURORDIS 
  124. EuroWilson
  125. Evotec
  126. Exelixis
  127. Ezose Sciences Inc
  128. Fast Forward LLC
  129. Ferring Pharmaceuticals 
  130. Fondazione Telethon 
  131. Foundation Medicine
  132. F-star Alpha Ltd. 
  133. Fujisawa Pharmaceuticals
  134. Fumapharm AG. 
  135. Fundacio ACE 
  136. Galapagos
  137. Galderma
  138. Galecto Biotech AB 
  139. GebertRuf Foundation
  140. Gedeon Richter Plc. 
  141. Genentech
  142. Glenmark Pharmaceuticals
  143. Gilead Sciences
  144. Gladstone Institute 
  145. GlaxoSmithKline
  146. Global Innovation Pharmaceuticals
  147. Gloucester Pharmaceuticals 
  148. GlycoMetrics
  149. Grifols
  150. GTx Inc
  151. GW Pharmaceuticals Plc 
  152. Halozyme
  153. Harvard and Massachusetts Institute of Technology
  154. Helsinn Group 
  155. Hemobras
  156. Hologic, Inc
  157. Hospira
  158. HudsonAlpha Institute
  159. Human Combinatorial Antibody Laboratory
  160. Hutchison China MediTech Ltd.
  161. Idec Pharmaceuticals
  162. ILDONG Pharmaceuticals 
  163. Immunex Corp. 
  164. Impax Pharmaceuticals 
  165. Inception Sciences
  166. Incyte Corporation
  167. Infinity
  168. Innovive Pharmaceuticals 
  169. INSERM
  170. Insmed
  171. Inspiration Biotech 
  172. Institute of Experimental Neurology 
  173. Intellect Neurosciences 
  174. Intercept Pharmaceuticals 
  175. InterMune
  176. International Organization of Multiple Sclerosis Nurses
  177. International Pancreatic Cancer Patient Advocacy Committee
  178. International Union against Tuberculosis and Lung Disease 
  179. Invida Group
  180. Ipsen
  181. Ipsos
  182. Iris Pharmaceuticals
  183. Isis Pharmaceuticals 
  184. Italian Centre for Cystic fibrosis
  185. Janssen Pharmaceuticals 
  186. Japan Research Foundation for Clinical Pharmacology
  187. Japan Tobacco Inc.
  188. Jerini AG 
  189. Johnson & Johnson
  190. Jude’s Children Research Institute
  191. Kamada Ltd. 
  192. KEK (The High Energy Accelerator Research Organization)
  193. Kirin Amgen
  194. Kirin Brewery Company 
  195. Kirin Co. Ltd
  196. Kirin-Amgen
  197. Kissei Pharmaceuticals Co. Ltd
  198. Knopp Neurosciences
  199. Kotobuki Pharmaceuticals Ltd
  200. Kyoto University
  201. Kyowa Hakko Kirin 
  202. LabCorp Beijing 
  203. Leukemic Children’s Association 
  204. Ligand Pharmaceuticals
  205. Liverpool School of Tropical Medicine
  206. Lotus Tissue Repair, Inc. 
  207. Lumena Pharmaceuticals 
  208. Luminex Corporation
  209. Lundbeck
  210. Lymphoma Academic Research Organization
  211. Macusight
  212. Manchester Collaborative Centre 
  213. Marnac Inc. 
  214. Massachusetts General Hospital
  215. Mayo Clinic
  216. Medarex
  217. MediKidz Limited
  218. MedImmune
  219. Medivation
  220. Medtronic 
  221. Meiji Seika 
  222. Merck
  223. Merck Serono
  224. Merrimack Pharmaceuticals 
  225. MGI Pharma 
  226. Michael J Fox Foundation 
  227. Micromet
  228. Millennium
  229. Minophagen Pharmaceuticals 
  230. Mitsubishi Tanabe Pharma
  231. Moderna Therapeutics 
  232. MorphoSys AG 
  233. Mount Sinai Medical Center
  234. Mylan Pharmaceuticals 
  235. Myogen
  236. Nagasaki University 
  237. National Cancer Institute
  238. National Eye Institute
  239. National Institute for Health and Care Excellence (NICE) 
  240. National Institute of Advanced Industrial Science and Technology
  241. National Institute of Health
  242. National Multiple Sclerosis Society
  243. National Organization for Rare Disorders 
  244. National University Corporation
  245. Natrogen
  246. Neurimmune Holdings AG 
  247. NeuroSearch A/S
  248. Newron Pharmaceutical
  249. Nippon Shinyaku 
  250. Nogra Pharma Ltd
  251. Novartis
  252. Novo Nordisk
  253. NPS Pharma 
  254. Oncodesign
  255. Oncogenex Pharmaceuticals
  256. Onconova Therapeutics
  257. Online Education Research Center 
  258. Ono Pharmaceuticals
  259. Onyx Pharmaceutical
  260. Opexa Therapeutics
  261. Optimer Pharmaceuticals 
  262. Orion Pharma
  263. Orphan Disease Treatment Institute Co Ltd.
  264. Orphan Europe 
  265. Orphatec Pharmaceuticals
  266. Ortho-Clinical Diagnostic Inc 
  267. Oswald Cruz Foundation
  268. Otsuka Pharmaceuticals
  269. Ovation Pharmaceuticals 
  270. Oxford Biomedica
  271. Oxford GlycoSciences
  272. Oryzon
  273. Pancreatic Cancer Action Network 
  274. PATH Organisation
  275. Patient Advocate Foundation
  276. PDL BioPharma
  277. Peptimmune Inc. 
  278. Pfizer
  279. Pharmacia
  280. Pharmacyclics
  281. PharmaLink AB 
  282. PharmaSwiss S.A
  283. Pink Ribbon Red Ribbon Foundation 
  284. Plexxikon
  285. Premacure AB
  286. ProStrakan Group plc
  287. Protalix
  288. PTC Therapeutics
  289. Pulmonary Fibrosis Foundation
  290. Qiagen
  291. Ranbaxy
  292. Recordati
  293. Repligen Corp
  294. Roche Laboratories
  295. Roche's Translational Clinical Research Center
  296. Royalty Pharma 
  297. Saegis
  298. Sailing Sclerosis Foundation 
  299. Saladax Biomedical 
  300. Samsung Medical Center
  301. San Raffaele University and Research Hospital
  302. Sangamo Biosciences 
  303. Sanofi- Aventis
  304. Sanofi- Synthelabo
  305. Sanquin Blood Supply Foundation
  306. Santen
  307. Santhera Pharmaceuticals 
  308. Schering
  309. Seaside Therapeutics
  310. Seattle Genetics
  311. Sekisui Medical Ltd.
  312. Sepracor Inc
  313. Seragon Pharmaceuticals
  314. Server &Xoma
  315. Shionogi &Co. Ltd
  316. Shire
  317. Siemens Medical Solution
  318. Sigma-Tau Pharmaceuticals 
  319. Sobi
  320. Solasia Pharma Ltd
  321. Spanish National Cancer Research Centre 
  322. Sumitomo Dainippon 
  323. Sunvonion Pharmaceuticals
  324. Swiss Tropical and Public Health Institute
  325. SWOG cancer institute
  326. Synageva Biopharma 
  327. Syntonix Pharmaceuticals Inc
  328. Taiho Pharmaceuticals 
  329. Takeda
  330. Talecris
  331. TargeGen
  332. Teijin Pharma
  333. Teikoku Pharma 
  334. Telethon’s Institute of Genetics and Medicine 
  335. Teva
  336. The Eunice Kennedy Shriver National Institute Of Child Health and Human Development
  337. The European Cystic Fibrosis Society
  338. The KarolinskaInstitute
  339. The National Human Genome Research Institute
  340. The Spanish Federation of Parkinson’s Disease
  341. Threshold Pharmaceuticals 
  342. Tillots Pharma AG 
  343. Tokyo Institute of Technology 
  344. U3-Pharma
  345. UCB
  346. Ultragenyx Pharmaceuticals 
  347. UniQure
  348. University College London 
  349. University of California, San Francisco
  350. University of Cambridge
  351. University of Cape Town
  352. University of Chicago Medical Center
  353. University of Illinois 
  354. University of Liverpool 
  355. University of Manchester 
  356. University of Texas MD Anderson Cancer Centre 
  357. University of Tokyo 
  358. University of Tsukuba 
  359. Upjohn
  360. Valeant Pharmaceuticals 
  361. Valld’Hebron Research Institute
  362. Vanderbilt University 
  363. Veloxis Pharmaceuticals
  364. Veridex LLC
  365. Vernalis Inc. 
  366. Versant
  367. Vertex Pharmaceuticals 
  368. Ventana Medical Systems
  369. ViroPharma
  370. Warner Lambert
  371. Weill Cornell Medical College 
  372. World Health Organization
  373. World Bank
  374. World Federation of Hemophilia
  375. World Intellectual Property Organization
  376. XeneticBioSciences
  377. Xenon
  378. Yamanounchi Pharmaceuticals 
  379. YM Biosciences
  380. Zeltia
  381. ZenyakuKogyu Co. Ltd
  382. Zeria Pharmaceuticals
  383. Zinfandel Pharmaceuticals
  384. Zymenex
  385. Zymo Genetic

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