How many rare diseases have treatments?
Most rare diseases do not have specific FDA-approved treatments. Many are treated “off-label”, which means that products approved by FDA for other diseases or conditions are used. Increasingly, insurance companies, Medicare, and Medicaid are refusing to cover off-label uses since they are investigational. Since the Orphan Drug Act went into effect in 1983, more than 1,800 products have been designated “orphans” by FDA (which means they are being developed for the treatment of rare diseases). Nearly 330 of those have been approved by FDA for marketing. In comparison, during the decade before 1983, only 10 products were developed by industry for diseases considered rare. FDA has estimated that 11 to 12 million Americans now have treatments for their rare diseases as a result of the Orphan Drug Act. However, that still leaves millions of Americans with diseases for which there is no FDA-approved treatment. In addition, there are many rare diseases on which no research is being done. It ca
