“Salami-slicing” orphan drugs

Ultra-orphan? It’s a phrase I never heard before, but increasingly, companies are taking it on and finding it a useful way to describe the very smallest patient groups.

 ”Language is very important, and you have to understand how it affects perception,” says Maria Hardin, vice president of patient services for NORD (National Organization for Rare Diseases). “You have to think of the 30 million patients with rare diseases as a whole. We don’t want to salami slice it any more than we already have.” 

Hardin presented today at CBI’s Pre-Approval Access conference, here at the Hilton Baltimore, and said she is concerned that classifying some drugs as “ultra-orphan” and others just “orphan” will artificially divide services and access to life-saving drugs. “We refuse to use that word,” she says.

Instead, says Hardin, the word “ultra-orphan” has been put into play by pharma companies that specialize in treating rare diseases. But sources say that this sub-classification has at least one utilitarian purpose.

“How do you explain to a regulatory agency that your trial only has 30 patients in it–and half of those are on placebo control?” asks Mark DeRosch, senior director of regulatory affairs for Vertex Pharmaceuticals. DeRosch plays a part in the development of the company’s promising cystic fibrosis candidate, VX-770.