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March 2015
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Report Description
The service oriented and dynamic nature of the healthcare industry has led to the discovery of a plethora of treatment options for a wide range of ailments.However, there are certain diseases, termed as rare, that continue to remain untreated and neglected. Rare / orphan diseases, as the name suggests, are those diseases which are relatively infrequent in occurrence. Many orphan diseases are life-threatening and progressive in nature. Absence of potential treatment regimes catering to these diseases is a big unmet need in the market.
The prevalence and adversity of these ailments has gained significant attention in the global healthcare industry. Stakeholders have realized the necessity and importance of urgently addressing the widening concerns and the need for discovering treatment options for several rare diseases. There is a tremendous scope offered by the innovation driven research being executed by various organizations in an effort to bridge gaps in the current market landscape.
The enactment of the Orphan Drugs Act in the US and EU emerged as ground breaking and provided the necessary support to guide research focused on rare diseases worldwide.The orphan designated drugs are granted important incentives which include market exclusivity and fees reductions. This regulatory assistance has provided a strong impetus to the pharmaceutical companies and led to their active participation in the market. Collaborative agreements and partnerships, amongst the pharmaceutical firms and other active organizations (such as EURORDIS, NORD and Global Genes), are also playing a key role in addressing the needs of this market.
The wide gap between the demand and supply of drugs is likely to provide a major boost to the market in the coming decade. Several drugs for various rare diseases are currently under development and, once approved, are likely to be readily accepted by the untreated patient population.
Scope of the Report
The ‘Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas’ report is a comprehensive study of the market of drugs being developed by the top pharmaceutical companies for rare diseases. The focus of this study is primarily to uncover several initiatives currently underway to harness the potential presented in different therapeutic areas. One of the basic building blocks for analysing the market was to prepare an extensive pipeline of drugs for rare diseases. This effort encompassed reviewing several databases, company websites and other information available in the public domain.
We confined the focus of our study to 50 large pharmaceutical companies which invest a proportion of their R&D spend on drugs for rare diseases. These pharmaceutical companies were profiled to get an in-depth view of the work being done by them in this domain. The profiles include a general overview of the firms, financial performance over the last five to six years, and the current marketed and clinical pipeline products specific to rare diseases.
With a robust pipeline in place, we then analysed where the industry’s current vision lies and highlighted a number of efforts which have been taken by pharmaceutical companies, healthcare organizations and other industry stakeholders. Specifically, our market potential analysis outlines key patient demographics and underscores potential areas anticipated to emerge as major revenue generators for the pharmaceutical companies. In addition to the above, we have also provided our own independent perspective on the key drivers behind the growth of this market, and the associated challenges.
Contents
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.
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
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, %)
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
The following companiesand organizations have been mentioned in the report.