Is the orphan drug sector sustainable?

In Advocacy, Clinical Development, Market Access, Partnering & Investment, Regulation & Government, Supply Chain & Manufacturing by Freya Smale

Although the orphan drug sector had a very productive 2014 with the largest percentage increase in orphan drug designation requests, in orphan drugs designated and in the highest number of orphan drugs approved by far, it was also the most tumultuous. There’s the 340B Rule, orphan drug prices scrutinized by NICE, talk about revising ODA to reduce incentives… Is the orphan drug sector sustainable?

As we were getting various perspectives on this, we thought, why not ask Henri Termeer, the former Chairman, President & CEO of Genzyme, and who is known as the guy who figured out how to build a great business by making drugs for rare diseases, according to an interview by Luke Timmerman of Xconomy.

Is the orphan drug sector sustainable? “It has to be”, Henri says. He goes on to talk about the various stakeholders and talks about the importance of academic medical centers who understand the science but are also closer to the patients. They can serve as the bridge between the scientist in the lab and the patients who need the drugs.

Below is a short clip of Henri on cam. We will be showing this in full at the 5th annual World Orphan Drug Congress USA in DC and will allow participants to send us their questions for Henri which we will present and publish after the conference.

View the clip here! 

Haven’t registered? Register now at to book your delegate ticket.