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Off-Label Pharmaceutical Use

According to NIH, there are almost 7,000 identified distinct rare diseases, the vast majority of which are genetic in nature.  The Orphan Drug Act of 1983 has been a landmark success at making the development of orphan products a profitable venture, thereby driving industry to invest in research and development for rare disease treatments.  To date, some 2,200 investigational therapies have been designated “orphan drugs” by FDA’s Office of Orphan Products Development, and 360 of these have gone on through the review process to be approved for marketing.

The approval of 360 drugs is a huge step in the right direction for the rare disease community.  However, these drugs are indicated for only about 200 rare diseases, leaving approximately 6,800 diseases without FDA-approved treatment.  In other words, providers must treat over 90% of rare disease patients “off-label,” using other drugs and methods to provide some level of care.

People with rare diseases often have difficulty being reimbursed for the care they receive because that care is “off-label” and may not be covered by health insurance.  This financial burden can be an enormous strain on patients and families, especially when the cost of off-label treatments runs very high.

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