Why There Was Virtually No Research into Cures for Rare Diseases before 1983

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There were virtually no pharmaceutical companies researching cures for rare diseases—also known as “orphan” diseases—before 1983, but that has since changed.

Here’s why, says Patrick Girondi:

“In 1983 the orphan disease act was introduced. Marlene Haffner, who at the time was called the godmother of the orphan disease act, she’s a wonderful woman. Even she said there's no money going into curing diseases because there's no money in it, because there's too few of them.

And they came up with these rules and regulations, stimulations. They gave 50% tax credit to people who would invest in clinical trials for orphan diseases. Today it's 25%. They would give all sorts of stimulation through finance etc. to companies who would dedicate themselves to curing rare diseases. It was a wonderful legislation, and there's 6000 rare diseases.

I believe that the 1983 Act needs to be revisited to be quite honest, because I think there needs to be some kind of separation: you have a disease like sickle cell disease, where 100,000 patients can't be treated and a disease like leukocyte adhesion deficiency where you only have like 100 patients. You have to have different rules.

But it was a wonderful piece of legislation. They were called rare diseases…They were orphaned by big pharma, supposedly, and that's why today they call them orphan diseases as well as rare diseases.

…Now there's hundreds of companies going after these rare diseases. How many of them are successful? It's not easy to get into clinical trials. We're one of the few companies of our size that ever got into clinical trials.”