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FDA helping drug companies develop treatments for orphan diseases

Orphan. It’s such a lonely word. And when coupled with the word “disease,” it’s downright scary. The truth is I had never heard of the term “orphan disease” before being diagnosed with a blood disorder 10 years ago, but I quickly became familiar with it. The term meant there weren’t a lot of other people whose platelets were being gobbled up by their own body, and it meant there wasn’t tons of research being done on a treatment or cure. I also learned the term applied to around 7,000 (!) diseases in total, which meant I actually wasn't without company.

Because of my personal stake in the issue, I always perk up when I come across articles on orphan diseases and potential treatments - and I took great interest in a Wall Street Journal piece yesterday. The article talks about the efforts being made by FDA officials to get more drug companies to develop drugs for these rarer diseases and to take advantage of financial incentives made available through the Orphan Drug Act. The Office of Orphan Product Development's first step: two workshops with "on-the-spot regulatory advice" for interested companies.

Office director Timothy Coté, MD, told the Journal that we're "barely scratching the surface" when it comes to orphan treatments; so far, there are only 350 approved drugs for about 150 diseases. But perhaps these efforts will help boost those numbers.

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