Report: Orphan drug prices facing increased scrutiny

With the US Food and Drug Administration approving an all-time record number of orphan drugs during 2014, the pricing of these treatments is set to come under increased scrutiny, says an analyst.

According to Adam Dion, industry analyst for research and consulting firm GlobalData, Big Pharma has shifted its focus from large patient populations in established therapeutic areas, such as cardiovascular diseases and diabetes, to developing orphan drugs for rare diseases, which constituted 17 of the 41 new molecular entities approved by the FDA last year.

Mr Dion explains: “The need for product pipeline replenishment, quicker access to commercialization revenues and attractive selling prices may partly explain this new-found infatuation with orphan drugs. Orphan drug developers generally see significant return on investment due to lower clinical trial costs, particularly in Phase III where patient recruitment is much smaller. Approval times are also usually faster, because these treatments tend to receive Priority Review from the FDA.”