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.
And as troubling as these figures are, more sobering still is the fact that half of these diseases affect children. Yet for all the need, there are only about 400 approved orphan therapies. That means 95 percent of rare diseases go untreated, a fact that reflects the time and expense involved in developing these drugs and proving their efficacy.
Since passage of the U.S. Orphan Drug Act in 1983, regulators have provided extensive guidance and resources to support rare disease research. Receiving orphan designation qualifies drug companies for development incentives that include: