Orphans are not alone

At Adelphi Values PROVE we recognise that value demonstration for orphan/ ultra-orphan drugs in rare diseases requires a different focus to support successful market access.

Despite high costs of treatment, these debilitating, high-burden high unmet-need conditions have often flown under the radar of payers based on the small patient populations and relatively small overall budget impact.

With over 500 million people around the world afflicted by one of over 7,000 rare diseases, this area of research is growing rapidly and it is important for biotech and pharma alike to understand how this landscape is evolving.

Based on our extensive experience in this area, the team at Adelphi Values PROVE has developed a thought leadership article to help our clients understand this ever-changing landscape, published in the June 2019 issue of Pharma Times. “There is increasing payer scrutiny over pricing and a greater challenge in value demonstration” says a Senior Director at Adelphi Values PROVE. “Coupled with the scientific advances in understanding the genetic basis and pathophysiology of rare diseases, this makes for a very interesting and timely area for discussion, along with the confusion for health systems over which incentives can create the right balance for this market.”

The full article is available at the following link:

https://www.adelphivalues.com/content/uploads/2019/06/OrphansAreNotAlone.pdf