The designation, which is made by the Food and Drug Administration, allows drugmakers to claim seven years of market exclusivity.
Orphan drugs for rare diseases have helped or saved hundreds of thousands of patients like 2-year-old Luke Whitbeck, but families and insurers are picking up the astronomical cost.
Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives but nearly a third of those products aren’t new or were repurposed multiple times, an investigation shows.
Follow the twists and turns of the orphan drug industry over the past three decades.
Check out all the drugs the FDA has approved to treat rare diseases. You can search by brand name, or by disease, and see familiar names that were first sold on the mass market or all the drugs that won FDA approval to treat more than one rare disease.
The former Congressman championed the Orphan Drug Act decades ago but now he fears it’s being manipulated to make money.
A voucher awarded to companies that find treatments for rare childhood diseases can be sold to the highest bidder — and then used to speed up approvals for much more common drugs.