Will orphan drugs find a home in the world of pharmaceutical R&D?

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Screen Shot 2014-08-21 at 2.20.41 PMYesterday, the FDA gave the thumbs up to Sanofi s Cerdelga, the first oral treatment for a rare condition called Gaucher s disease. Cerdelga is classified as an orphan drug specialized therapies designed to treat rare diseases in small numbers of patients.

Gaucher s is a serious genetic defect where the body is unable to produce the enzyme glucocerebrosidase, resulting in fatty deposits in certain organs. In its most severe form, spleen removal and bone marrow transplants may be required.

This provides a good opportunity to examine the changing landscape in pharmaceutical research, which is quite different than it was only ten years ago.

We spoke with pharmaceutical uber blogger, Derek Lowe about Cerdelga and the relatively new focus on orphan drugs.

He says, drug companies have a bit of a quandary on their hands. [If this] is going to be just as hard to develop as a drug that has a huge possible number of patients, why should we go after this?'

Yet, they are doing just this. According to ACSH s Dr. Josh Bloom, in 2013 there were 27 new drug approvals. Nine (33 percent) were orphan drugs designated for patient populations of 200,000 or less. By comparison, in 2003, orphan drugs constituted three out of 21 (14 percent) of newly approved drugs, and four of 21 (19 percent) in 2008.

The very high price for these drugs is always a thorny issue. Lowe says, In theory- a price is what people will pay, and the company is saying, If insurance companies will pay $300,000 for the IV, they'll pay 300K for the pill'- which seems to be just as good... and not everyone is happy about this.

Yet, the economic impact on our health care system is not as much as you d expect. Dr. Bloom explains, Since these drugs, even though they are extremely expensive, are used only in very small patient populations, the total cost is actually small compared to that of typical new drugs, which are both expensive and much more widely used.

We encourage you to listen to Ana Dolaskie s interview with Dr. Lowe here.