Press Releases

Global Orphan Drug Market to Gain Almost $68 Billion by 2019; Favorable Legislation Spurring R&D

September 22, 2015

Wellesley, Mass., September 22, 2015 – Growing competition, new technologies, improved gene therapies, and favorable legislation and incentives have led the global orphan drug market to experience a significant increase in the number of approved orphan drugs and their market availability.BCC Research reveals in its new report that approval of biological orphan drugs for multiple indications has been a growth factor, along with successful product launches of non-biological drugs.

The global orphan drug market, which totaled $117.3 billion in 2013, is projected to reach $123 billion and $191 billion in 2014 and 2019, respectively, reflecting a compound annual growth rate (CAGR) of 9.2% from 2014-2019. The biological orphan drug market should total $126.1 billion in 2019, up from a projected $83 billion in 2014, while the non-biological orphan drug market should reach $64.7 billion in 2019, up from a projected $39.9 billion in 2019. The biological and non-biological markets are anticipated to achieve five-year CAGRs of 8.7% and 10.1% respectively.

Orphan drugs are pharmaceutical or biological agents used to treat or prevent rare medical conditions. These drugs have paved the way for the treatment of the many unmet clinical and therapeutic needs of patients with orphan and rare diseases such as Gaucher’s disease, Pompe disease, cystic fibrosis, and myelogenous leukemia.

Successful legislation has contributed mainly to the growth of the orphan drug market, which is dominated by the U.S. and Europe. The significant increase in the number of orphan drug approvals, collaborations and successful launches during the study period was overwhelmingly driven by the Orphan Drug Act. The availability of infrastructure and technological advancements also helped spur momentum in both regions, as well as worldwide.

“Pharmaceutical giants and biotech firms are trying to increase their global presence through strategic alliances, collaborative developments and marketing agreements. This strategy proved sound with the emergence of markets in India and New Zealand, for example,” says BCC research analyst Shalini S. Dewan. “Increasing global awareness of orphan diseases and associated treatment options, along with the greater availability of approved orphan drugs in emerging markets through strategic alliances, will continue to be major boosts to emerging market growth.”

Global Markets for Orphan Drugs(PHM038E) examines orphan drugs in pharmaceutical markets worldwide. The report analyzes each market and its applications, regulatory environment, technology, market projections and market shares, with forecasted trends and revenue through 2019.

Editors and reporters who wish to speak with the analyst should contact Steven Cumming at

Global Markets for Orphan Drugs( PHM038E )
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