This is a summary of a session a the recent Partnering for Cures conference. It illustrates the importance of creating a registry and collecting natural histories from patients, two things the association is working hard on setting up right now.
In 1983, the year the Orphan Drug Act became law, there were just two drugs approved for rare diseases. In 2011, 26 drugs were approved to treat rare conditions. In the 30 years since the Orphan Drug Act created incentives for drug developers who pursue therapies for conditions that affect fewer than 200,000 people, more than 360 drugs have been licensed for 449 rare conditions. Some command high prices, with more than 10 drugs for rare disease topping $200,000 per patient annually.