A Corporate View of Rare Drugs and the Story of Miglustat - A Perspective

Natasha D'Souza*

Abstract

To be rare is a gift. It makes you stand out from the crowd, defining you with qualities that most people don’t seem to have. But in the medical world, it may not be a boon after all. To have a rare disease means to have unique health issues, often a lifetime of medication which is moreover difficult to find and at times not being able to live a normal life. The toughest challenge here is your disease being recognized enough to have a decent population base which prompts pharmaceuticals players to invest research in a drug thereby putting the product on pharmacy shelves. In addition to this, rare drugs are often viewed as an opportunity to make the drug a cash cow due to fewer players in the market thus making affordability a prime concern to patients.