[todayiamme]

You seem to have assumed something over here. That,

a) A Start-Up has that type of money b) They'll be willing to invest in something so risky.

As others have pointed out there are StartUps with the money, but somehow we still don't hear about orphan drugs with potential to make it burst onto the scene. Why not? I think that the second factor is more at play over here. Ultimately it comes down to the cost vs. benefit analysis.

Imagine this you are a VC with a $100 mil. to burn will you support a long and twisted development process of something that might not even work at the end?

You might argue that the benefit is that you might become the up and coming Google of pharma, but try telling that to any smart VC.

This is why we need something more than a profit based industry for something that forms the basis of our society. On one hand people talk about freedom, and especially how it's related to the freedom of the markets, and on the other you trap them with the very failings of their bodies.

Perhaps, I am wrong, but this is something that we all need to think about. What do we value more as a species money or wealth? I wish I knew the answer.

[rsheridan6]

>As others have pointed out there are StartUps with the money, but somehow we still don't hear about orphan drugs with potential to make it burst onto the scene.

Imatinib. It was developed as a cure for CML, a disease which only affects a few thousand people in the US. That's just the most notable. From wikipedia's article about orphan drugs,

"In the USA, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization."

>Imagine this you are a VC with a $100 mil. to burn will you support a long and twisted development process of something that might not even work at the end?

The fact that pharmaceutical startups exist (google pharmaceutical startup or something like that and you'll find plenty of them) show that VCs, or whoever it is who invests in them, have different ideas about risk than you do.

[todayiamme]

>"In the USA, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization."

Thanks. I realize my mistake now. I meant it as in how many new companies try something as risky as making an orphan drug? I shouldn't have leaped in with a bad example without checking it more thoroughly first.

Sorry.

>The fact that pharmaceutical startups exist (google pharmaceutical startup or something like that and you'll find plenty of them) show that VCs, or whoever it is who invests in them, have different ideas about risk than you do.

My point over here was that they fund them, but they might not fund something as risky as stem cell therapy. As far as orphan drugs and StartUps go; is there an data freely available online on the composition of companies that market orphan drugs?

Perhaps that would be more revealing?

(I found this site from the wiki article http://www.urchpublishing.com/publications/discovery__rd/orp... , but it's paid)

[rsheridan6]

Here's a company that was started in 2006 with the intention of treating rare diseases: http://www.atonrx.com/background.php

They were just bought out for $318M. Not bad.

Treatments for rare diseases actually have fewer regulatory hurdles because of the Orphan Drug Act, which basically says that there's a lower standard of evidence for drugs that treat rare diseases.

>My point over here was that they fund them, but they might not fund something as risky as stem cell therapy.

Maybe not, but maybe that's a good thing, for reasons carbocation stated.

>is there an data freely available online on the composition of companies that market orphan drugs?

There's a list of drugs here (well, do a search on an empty string and you get a list) and the companies who make them, but it's not exactly predigested.

http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index....