The Orphan Drug Act was first signed into law in 1983. The Act provided a variety of incentives for pharmaceutical companies to develop medications specifically for rare diseases, including tax breaks and seven years of market exclusivity. In 1983, there were only a handful of drugs for rare conditions but thanks to the Orphan Drug Act, most pharmaceutical companies have developed and/or are developing orphan drugs for rare diseases.

Badhiu Basu, PhD, owner of the Converge Advisory Group talked about the Orphan Drug Act and what it has (and has not) accomplished.

“If you look at the number of designations that the FDA is given its increased from 150 to 200, twelve years ago,” noted Dr Basu, adding, “It peaked at 480 I believe in 2017. So each month, there's 25 to 30 designations that are given out to various companies of various diseases.”

That is the good news. Unfortunately, the drugs being developed are still only a fraction of the rare diseases in need of treatment. “Our database has about 750 unique diseases. Now, those numbers are big but when we put it in proportion to 9,000 diseases it's only a fraction that's getting developed or getting marketed. So, we're talking 8 – 12% of potential amount of diseases that are actually being developed,” state Dr Basu, adding, “So part of my drive towards this sector is to start talking to companies and stakeholders about how we can do things on a completely different scale.”

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