Episode 3 - The Far-Reaching Burden of Rare Diseases

In addition to the substantial clinical challenges that are associated with managing rare diseases such as hemophilia and SLE, optimally treating those patients are often tied to heavy economic burden that may often exceed $100,000 per year per patient. And while the total healthcare budget impact is minimal, managed care authorities are tasked with designing protocols that ensure the appropriate use of medications, deriving the highest value from the high cost therapies. While guidelines provide useful reference points in approaching rare conditions, they do not necessarily define the process of identifying the right patient and channeling them towards the right clinical care strategy, while minimizing waste and managing disparities in expectations.