Magazine Article | January 1, 2017

Orphan Drug Incentives & Innovations On The Rise

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.

To continue reading this story and receive uninterrupted access to LSL Online and its monthly magazine subscribe to Life Science Leader today!
Already a subscriber? Log in now.