400 Orphan Drugs In Development

A report from PhRMA looks at rare diseases and orphan drugs.

An orphan drug is a pharmaceutical agent developed to treat a disease that affects less than 200,000 people in the US.

While individually this may not seem like a big market, it is estimated that 25-30M Americans suffer from a rare disease.  And, developing therapies for these unique conditions can allow for price premiums. 

At the end of the document, they have some FAQs and suggest some websites.  Here’s one FAQ:

How can you find out about clinical research on rare diseases?

There’s a web site that was just set up a few years ago by the federal government. It’s called www.clinicaltrials.gov.  It’s important to remember “.gov” because there are some commercial sites that have similar names. Every research project receiving any money from the U.S. government must be listed on this site. It’s a requirement. You can type in the disease name and find all sorts of information about the studies, where they’re being conducted, what is needed to be eligible, and who to contact to learn more about participating. If you don’t have a computer, ask your local librarian to help you search on that web site.