Fighting a Global War on Rare DiseasesMar. 19, 2013
Are we winning the war against rare, or “orphan,” diseases? A casual observer could be forgiven for thinking so, given the drumroll of news. The crescendo came in 2011, when the U.S. Food & Drug Administration (FDA) approved 26 drugs in this category, meaning for illnesses that afflict fewer than 200,000 people. It was the highest number since Congress passed the Orphan Drug Act in 1983, offering incentives to companies that entered the rare-disease field. The same year, the agency granted a record 199 requests from companies seeking an “orphan” designation for their products.
In 2012, there was a stream of orphan-related announcements from Pfizer, GlaxoSmithKline, Sanofi and other industry giants – a break from the past, when orphan diseases were the turf of small companies. Last week, a columnist in this space fretted that the excitement over rare diseases might one day lead to a reduction in research into hard-to-treat ailments such as cancer or Alzheimer’s, which affect many more people.
Yet despite the frenzy – and as the columnist duly noted – we are a long way from winning this war. Around the world, an estimated 60 million people suffer from some 7,000 rare diseases. Fewer than 5 percent of orphan diseases have treatments approved by the FDA.
For the full Xconomy column click here.