Our previous post looked at drugs that were discovered by accident or used for uses beyond their initial intentions. These drugs went on to make large impacts to millions of people around the world, and we can feel good about that.
But what if a drug made an impact in fewer people’s lives, say 1000? Would they still be embraced? If you are an empathetic and compassionate person you would probably say yes. However, the fact of the matter is that drugs cost money and their development is largely dependent on funding and demand. As a result, a drug that can benefit millions of people will likely see the market before a drug that can benefit a hundred or a thousand.
Orphan drugs are drugs that treat rare medical conditions (also known as orphan diseases). Orphan diseases are often caused by genetic mutations and some examples include: hemophilia, cystic fibrosis, celiac disease and “Lou Gehrig’s disease” (amyotrophic lateral sclerosis or ALS). Other less recognizable orphan diseases include cat eye syndrome and Nelson syndrome. There are over 5,000 rare diseases with new rare diseases discovered every year1.
Funding and Incentives for Development
Since the drug industry operates in the free market (i.e. in a market based on supply and demand), support for the development of orphan drugs is necessary, unless we depend solely on philanthropy and charity. Orphan drugs often receive government promotion and funding in the form financial assistance, tax incentives, and enhanced rights over patents and marketing.
Legal action has also taken place to promote the development of orphan drugs. The Orphan Drug Act was passed in the United States in 1983 and allows pharmaceutical companies to develop and market orphan drugs without competition for seven years.
Hope for the Future
Overall, the Orphan Drug Act has been successful in encouraging ‘the adoption’ of orphan drug development2. Since 1983 the FDA has approved more than 300 orphan drugs and the rate of approval continues to rise. In 2011, ten (10) new orphan drugs were approved by the FDA.