There's only about a one-in-a-million chance that a newborn infant will be faced with lipoprotein lipase deficiency, or LPLD. But for those rare losers in life's health lottery, the push into orphan diseases has delivered a new therapy: uniCure's Glybera, the first gene therapy to be approved in the West.
As Reuters' Ben Hirchler reports today in an in-depth article, though, Glybera is expected to arrive soon with a price tag of more than a million dollars, eclipsing the likes of Alexion's ($ALXN) Soliris, which costs $440,000 a year. And the 7-figure price, stratospheric even for rare disease drugs, helps illustrate why the orphan field has become so popular in recent years –and why it's growing increasingly controversial among payers.
Up to now there has been little real kickback from payers for a new crop of six-figure orphan drugs. The rare conditions they treated left a handful of big bills that could be spread among a large population. With Genzyme paving the way, developers increasingly focused more resources on more such drugs – a big reason why a quarter of all new drugs approved in the U.S. last year were for rare conditions. And that share of new drug approvals is likely to grow.
Over the past decade, writes Hirschler, the orphan drug market has been growing at a 25.8% pace, compared to 20.1% for the rest of the field.
"More companies are getting into this sector because they've seen the eye-wateringly high prices that can be charged for some of these very rare disease medicines," Karl Claxton, a professor of health economics at the University of York, tells Reuters. "It's unsustainable. Healthcare systems around the world are under increasing financial pressure and all of them are starting to look very carefully at what they get for their money."
With patients clamoring for these drugs, payers have been fighting back. English regulators persuaded Vertex to offer a secret discount off the $297,000 posted price for Kalydeco, a drug that treats a rare form of cystic fibrosis. And with health budgets under siege as prices continue to climb, the resistance is only likely to grow.
- here's the article from Reuters