Orphan Drug Economics: What’s The Right Price For Rare Disease Drugs?

The economics of orphan drugs have been pretty straightforward: Transformative treatments for rare diseases deserve premium prices. And so far, payors – at least the larger ones – haven’t really balked at those price tags. But stakeholders are starting to ask whether the orphan drug pricing benchmark set by Ceredase 20 years ago is right for all rare disease drugs. Industry needs to have good answers.