Seeing What’s Often Ignored.

Quick Facts

• The definition of a rare disease varies by country, but for the U.S. specifically, it is classified as a medical condition that impacts below 200,000 people (Cleveland Clinic).

• Over 10,000 rare diseases affect over 25 million people per year in the US alone. Roughly half of this population are children, and many of these illnesses are life-threatening (FDA).

• Due to the small margin of people living with these conditions, very few patients are available for testing, and only a small fraction of the total population will purchase treatment drugs. This lack of profit discourages companies from both producing existing medicines and carrying out new research (FDA).

• The government has attempted to combat this through the use of the Orphan Drug Act, which incentivizes the development of rare disease treatment drugs (FDA). The legislature was successful in some aspects; in the 40 years after it was signed into law, 882 new FDA-approved treatment drugs were procured (NIH). But looking at the whole, the fact remains that 95% of all known rare diseases have no FDA-approved treatment — demonstrating that its effects are largely insufficient (NIH).

• Rare disease nonprofits are conducting research and offering support to those who are influenced by these disorders, but unfortunately, they are often underfunded (Beacon).