Rich pickings from orphan drugs

Big Pharma is raking in billions from orphan drugs while India's policies on rare diseases is way behind in protecting patients

- 

SOME TERMS tend to be misleading, like when they confuse function with cause. Like orphan drugs. The name has a forlorn ring to it, and might lead the uninitiated to believe that these are medicines that are abandoned/neglected and have no protection either from the government or the market. But this is the reverse of reality. Orphan drugs, which treat rare and neglected diseases, are the new blockbuster medicines earning their companies billions in profits thanks to generous incentives in the rich world that have facilitated research and manufacture of such treatments.

Till the 1980s, there were just a handful of drugs to treat rare diseases, termed orphan diseases by several countries. Today, close to 900 such drugs have been approved in the US, a stunning jump in recent years. What is significant is that last year 52 per cent of the drugs approved by the US Food and Drug Administration (FDA) were treatments for rare diseases, a trend since 2020. Yet, for millions of patients, there is little access to these vital medicines because of a plethora of challenges, not least the cost.