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Aw, that poor pill has no parents…

medicationNo, no, that’s not what is meant by an “orphan drug.” Orphan drugs are used to treat orphan diseases (ones that affect fewer than 200,000 people in the United States). Orphan diseases - usually genetic in origin and often chronically debilitating - are so rare that a typical general practitioner in a busy urban area would see only one case in a year. Multiple myeloma, sickle cell anemia and cystic fibrosis are a few of the more well-known conditions that fall under the umbrella of orphan diseases.

Because of the relatively low number of patients affected by these rare diseases, there has never been much financial incentive for pharmaceutical companies to develop medications to treat them. In an effort to combat that, the government passed the Orphan Drug Act in 1983 to stimulate the research, development, and approval of products that treat rare diseases. In return, pharmaceutical companies receive tax incentives for any clinical research undertaken. They also get market exclusivity for seven years, which can prove to be a double-edged sword. If Company X accidentally finds that a pill they’ve been developing to treat acid reflux disease also happens to relieves the symptoms of Crohn’s Disease, consumers wouldn’t be able to buy it until the Crohn’s drug currently on the market that is produced by Company Y has exhausted its seven-year contract.

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