STAT Plus: Scientists are testing pricey rare-disease drugs as Covid-19 treatments. What if one works?
Some of the existing drugs scientists are testing as Covid-19 treatments have a special status for rare disease treatments, and the price tags to match — prompting early warnings from academics and drug pricing reform advocates that if one is effective, access could be an issue.
At issue are so-called “orphan drugs,” which get special exclusivity perks from the Food and Drug Administration because they treat a rare disease. Drugs get to keep that designation — and the perks — even if they’re later approved for a broader category of patients. That exclusivity often translates into higher prices, especially for rare disease drugs where demand is expected to be low.
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