What does "orphan disease" mean?

I am researching primary ciliary dyskinesia (PCD) and I see it referred to as an "orphan disease" what does that mean?

2 Answers

  • Anonymous
    1 decade ago
    Favorite Answer

    Orphan disease: A disease which has not been "adopted" by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it.

    An orphan disease may be:

    1. A rare disease. According to US criteria, an orphan disease is one that affects fewer than 200,000 people. (There are more than 5,000 such rare disorders.)

    2. A common disease that has been ignored (such as tuberculosis, cholera, typhoid, and malaria) because it is far more prevalent in developing countries than in the developed world.

    The US Orphan Drug Act of 1983 offered tax incentives on clinical trials and 7 years of marketing exclusivity for drugs developed for conditions that occur only rarely in the US. Since then, more than 200 orphan drugs have been approved by the US Food and Drug Administration (FDA) and are on the market. Similar legislation has been adopted in Japan and Australia.

    In the year 2000, the European Union adopted "orphan medicinal products" legislation modeled on the US law, but including tropical diseases and other disorders prevalent only in the developing world. The EU law provides for 10 years of marketing exclusivity, but no tax incentives (because there is no centralized EU taxation system).

    Source(s): google
  • Anonymous
    1 decade ago

    According to these sites, an "orphan disease" is a disease that occurs so rarely that it draws little attention to the public, and consequently, the creation of treatment for it doesn't receive enough funding.




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