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Drug Dev Res. 2014 Jun;75(4):231-4. doi: 10.1002/ddr.21176. Epub 2014 May 14.

The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.

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Department of Pharmacology, All India Institute of Medical Sciences, Bhopal, India.


Strategy, Management and Health Policy Large pharmaceutical companies have traditionally focused on the development of blockbuster drugs that target disease states with large patient populations. However, with large-scale patent expirations and competition from generics and biosimilars, anemic pipelines, escalating clinical trial costs, and global health-care reform, the blockbuster model has become less viable. Orphan drug initiatives and the incentives accompanied by these have fostered renewed research efforts in the area of rare diseases and have led to the approval of more than 400 orphan products. Despite targeting much smaller patient populations, the revenue-generating potential of orphan drugs has been shown to be huge, with a greater return on investment than non-orphan drugs. The success of these "niche buster" therapeutics has led to a renewed interest from "Big Pharma" in the rare disease landscape. This article reviews the key drivers for orphan drug research and development, their profitability, and issues surrounding the emergence of large pharmaceutical firms into the orphan drug space.


orphan drugs; rare diseases

[Indexed for MEDLINE]

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