Rare Disease Therapies are No Longer Rare: Can the US Healthcare Reimbursement System Handle Chronic Treatments for Orphan Diseases

Cures to devastating diseases, such as Hepatitis C drugs and gene therapies, are being developed and launched at an unprecedented rate and their one-time financial impact has triggered a new debate on affordability and rising healthcare costs. In parallel the number of approved medicines for rare diseases is taking off exponentially. So much so, that medicines for rare diseases are – arguably – becoming more “common.” Many of these medicines for rare diseases require chronic administration. Are we at an inflection point, whereby chronic therapies are no longer synonymous with common diseases? Are the current systems for chronic therapies appropriate for rare diseases medicines? Do we need to reconfigure the way these therapies are approached by all stakeholders, or can we flex the current systems? Thought leaders will discuss how to collaborate to ensure that the rare disease patients are able to access these needed therapies.