GAO on the Cost of Rare Diseases

Healthcare Economist November 29, 2021
Jason Shafrin

Last month, the Government Accountability Office released a report on the cost of rare disease. The report is interesting, and especially interesting is Appendix 1 which has a detailed list of studies of the cost of rare disease for about 50 rare diseases. Below are some highlights:

Currently, there are no treatments or cures for most rare diseases. Despite federal incentives for development of products to treat them, there are FDA-approved treatments for only about 5 percent of rare diseases…Even when a drug is available for off-label use, NIH officials explained that the drug would only be available to patients with a rare disease if a physician prescribed it (which they might be unwilling to do in the absence...

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