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Orphan Drugs Pipeline Analysis: Wider Incentives Encourage Orphan Drug R&D
Decision Resources, Inc., July 2005, Pages: 107


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This report examines the pipeline of drugs in development under the guidance of orphan drug legislation in the major markets of the United States, Europe, and Japan. Each of these markets has orphan drug legislation with different provisions, and we compare and contrastthese regulatory factors. The contrasting requirements and different time frames in which this legislation has been enacted has also resulted in considerable differences in the numbers of designated and approved orphan drugs within each market.

More than 6,000 rare diseases could potentially be designated as eligible for development of orphan drugs. However, this report focuses on diseases that have attracted the most attention from orphan drug developers—namely, genetic diseases, uncommon autoimmune diseases, and cancer. We also discuss which cancers are potentially eligible for gaining orphan drug designation. Given the large number of orphan drugs targeting various cancers, we devote an entire section of this report solely to cancer therapies. We group other orphan drugs in this report according to their primary mode of action: enzymes and enzyme inhibitors, hormones, gene therapies, gene modulators, monoclonal antibodies, and receptor and ion channel modulators.

We then profile companies that are active in the development of orphan drugs. We separately profile the small number of orphan drug specialist companies and the larger number of biotechnology companies for whom orphan drug development constitutes a significant proportion of their current pipeline. Finally, we discuss how the orphan drug market is likely to evolve over the next ten years by considering the influence of such factors as patent issues, pharmacogenomics, and the reimbursement environment.

Why Buy This Report?
- Learn what the leading players in the orphan drug market are doing via detailed analyses of their pipelines.
- Gain valuable insight regarding emerging companies and the agents in their orphan drug pipelines.
- Explore key trends in the orphan drug market and their potential impact on the commercial outlook for this market.
- Assess marketed and pipeline drugs with respect to development phase and peak-year sales potential.
- Analyze orphan drug alliance activity presented in comprehensive company profiles and evaluate company financial information



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