While historically on the margins of R&D spend, orphan drug development increasingly looks like a strategy whose time has arrived for delivering value in drug development. The blockbuster model is wearing thin — the pressure for lower cost drugs with better outcomes flies in the face of the “one-size-fits-all” approach to drug development where outcomes across a diverse population can be challenging to prove. It’s time to diversify the strategic approach.

The benefits of shifting focus to orphan drugs are many — including a streamlined approval process, patent extension and exclusivity, tax credits, and smaller clinical trials based on narrow indications. But as competition moves into these niche spaces, everyone will be looking for an opportunity to get an orphan drug designation (“ODD”). This will likely lead to additional scrutiny and will intensify the need to demonstrate the economic and clinical value of your product as it applies to a particular population before approval.