Billion-Dollar Drugs Take Advantage of US Rare-Disease Program

Vertex Pharmaceuticals Inc. has made over $20 billion in worldwide sales from a cystic fibrosis drug approved four years ago that can cost up to $300,000 a year.

With blockbuster sales like that, Vertex wouldn’t appear to need government assistance. But thanks to an obscure program designed to incentivize companies to make drugs for uncommon or neglected diseases, the Food and Drug Administration also awarded Vertex a bonus certificate that it can either use to expedite a future drug approval or sell for around $100 million.

Congress introduced these incentives, known as priority review vouchers, in 2007 to reward companies for developing treatments ...