We came across an interesting blog post by the National Organization of Rare Disorders (NORD), a nonprofit organization dedicated to helping people with “orphan” diseases, or diseases that affect fewer than 200,000 people. Together, there are nearly 7,000 orphan diseases that impact nearly 30 million Americans who NORD supports through education, research, and advocacy.
Peter Saltonstall’s post, “The Economics of Living With Specific Rare Disorders: What We Really Don’t Know,” explores how NORD leaders are trying to understand the economic costs of not being able to manage rare diseases effectively. “At the most simplistic level,” Saltonstall explains, “if we really did have some idea about the annual costs of caring for a patient with rare disease X, then it would help us to understand a ‘reasonable’ price to pay for a new form of treatment that was able to eliminate many of the current, existing costs.”
Gaining a fuller picture of the economics of rare disease goes back to NORD’s mission to develop “a social, political, and financial culture of innovation that supports both the basic and translational research necessary to create diagnostic tests and therapies for all rare disorders.” Also interested in tapping into innovation around rare disease research, Charles River’s Executive Director of US Toxicology Steven Bulera recently discussed similarities between orphan drugs and personalized medicine and whether the strategies used in the orphan drug development process can benefit the development of personalized medicine. Check out his recent Eureka post here.