Angus Russell, Shire’s longtime CFO—now CEO—likens the failing US automobile industry to pharma. “All the problems with the automobile industry were a long time coming,” he says. “It’s just that the SUVs and the relatively cheap price of gas created a bubble, just like a booming economy and blockbusters.”

But now that the bubble has burst, pharma can expect what Russell predicted a decade ago: a more competitive industry, with greater focus on pharmacoeconomic data, ROI, and less tolerance for me-too drugs. “The economic miracle is over—there will be a struggle to survive in this industry,” he says.

Russell, a speaker at the Reuters healthcare conference today, breakfasted with Pharm Exec at the W Hotel, and offered a preview before tomorrow’s main event, the business update for its Human Genetic Therapies (HGT) business. The meeting will take place in HGT’s new Lexington, MA, headquarters, and with more than 1,000 employees (up from 300 in 2005), it will also provide a visual reminder of how this business is a growth engine for Shire. (more…)

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.