Rare Disease Drugs Market Competitive AnalysisSize and Trends

The global rare disease drugs market was not considered lucrative until recently, owing to various uncertainties associated with these diseases. Small percentage of affected population provided limited opportunity for study. Moreover, diseases were poorly understood, owing to huge differentiation and heterogeneity among the group of diseases which discouraged manufacturers to address unmet needs in this space. However, introduction of some highly priced orphan drugs e.g. spinal muscular atrophy drug Spinraza priced at US$ 125,000 per vial, and high unmet needs has drawn attention of manufacturers towards rare disease drugs market.

The total revenue of top 10 global rare disease drugs market for 2016 accounted for US$ 62.5 billion.

Global rare disease drugs market is impacted by various key trends which are driven by supply as well as demand side. Wide gap between market needs and potential market offerings is the main driving force behind an increasing interest of the manufacturers towards providing therapies in this segment of the healthcare industry. According to the U.S. Food & Drug Administration (FDA), in 2016, 582 requests for orphan drug designation were filed by the biopharma companies. Furthermore, the sales of orphan drug diseases has been on a rise due to availability of highly effective drug therapies. Moreover, potential drug therapies are present in pipeline for various cancers and rare diseases such as Niraparib by Tesaro, Inc. in phase I for the treatment of ovarian cancer, Emicizumab by Roche in phase III for the treatment of hemophilia, and LentiGlobin by Bluebird Bio in different phases for the treatment of sickle cell anemia and beta-thalassemia major. Positive results from these clinical trials are expected to change the outlook of the global rare disease drugs market over the foreseeable future.