Rare Diseases

The Orphan Drug Act of 1983 was created to encourage pharmaceutical companies to develop drugs for so-called rare or ultra-rare diseases. The National Institutes of Health (NIH) defines rare diseases as afflicting as fewer than 200,000 individuals. Generally speaking, it has worked. Many companies are willing to invest millions of dollars needed to develop the drug that has a very small market. The Orphan Drug Act largely made this possible by slashing taxes and changing a few of the regulatory hurdles on clinical trial size. Three broad criticisms on the Act are: Firstly, the FDA is less fussy about the kind of data that it requires to approve a new drug for rare dis...