By Angi Robinson, executive director, pediatrics and rare diseases, Premier Research
The year 2015 was a productive one for introduction of drugs that target rare diseases. U.S. regulators approved 21 new orphan drugs, a 40 percent increase from the previous year. European regulators approved a record 18 orphan compounds, a small increase over 2014.
Any progress is a good thing, but these advances pale when weighed against the enormous unmet need for rare-disease treatments. Worldwide, an estimated 350 million people suffer from rare disease, a list of afflictions that numbers more than 7,000 and grows year by year. Rare disease advocacy group Global Genes says about 30 million Americans — nearly one in 10 — live with a rare condition. In Europe, the percentage is about the same. Rare disease, thus, is largely a misnomer: While no single condition affects a lot of people, the sheer number of diseases makes for significant medical and societal impact.