Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas - Key Players are Roche, BMS, Sanofi, GSK, Boehringer Ingelheim, Celgene, Novartis and Pfizer

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Jul 09, 2015, 11:50 ET

DUBLIN, July 07, 2015 /PRNewswire/ --

Research and Markets (http://www.researchandmarkets.com/research/j4xcgn/rare_diseases) has announced the addition of the "Rare Diseases: The Pharmaceutical Landscape and Opportunity Areas" report to their offering.

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.

Key Highlights:

- With over 7000 rare diseases reported in the world, lack of awareness, dispersed target population and inefficient reporting of these diseases limits the pace of development in this industry. However, the industry-academia coalition has helped in filling some of these gaps. An active participation by pharma firms through various initiatives, such as the Rare Disease Day, has helped to address the existing lacunae in the demand supply gap for these drugs.
- The market is characterized by the presence of several pipeline drugs under development for the treatment of rare diseases. Of these, 30% drugs are in phase III of clinical development whereas 38% of drugs are under development in phase II clinical trials.Phase I drugs account for 20% to the total market.
- Companies such as Roche, BMS, Sanofi, GSK, Boehringer Ingelheim, Celgene, Novartis and Pfizer are some of the key players in the market. However, the market remains opportunistic for newer players to step in and benefit from the vast potential in different therapeutic areas.
- With a share of more than 50%, oncology remains the key focus area of pharmaceutical firms. Inflammatory disorders and neural disorders are some other well-known areas being targeted.
- On an average, 13-14 drugs are being developed by each pharmaceutical company covered in our analysis. However, there is a stark variation amongst the companies. Some of the bigger pharma companies, such as Roche and BMS, have a much wider portfolio.

Key Topics Covered:

1. Preface

2. Introduction

3. Rare Disease: Pipleine Analysis

4. Rare Diseases: Market Potential

5. Rare Diseases: Company Profiles

6. Conclusion

7. Appendix: List of Companies - Click the link below to view the 385 companies that feature in this extensive report

For more information visit http://www.researchandmarkets.com/research/j4xcgn/rare_diseases