Rare diseases have gained more attention in recent years, perhaps in part due to the high price tags often associated with approved therapies. But the cost of rare disease drugs is dwarfed by the cost of not having them.
Our recent study shows that a lack of treatment for a rare disease is associated with a 21.2% increase in total costs per patient per year. That places a significant burden on the healthcare system. While rare diseases impact a small population, the cost to society is staggering–we estimate that the societal cost in the U.S. for all 7,000 known rare diseases may be in the range of $7.2-$8.6 trillion per year.
