No #scandal here, say rare disease #drugmakers, as #pricing scrutiny lands on their doorstep

Rare disease drugmakers get that their typically high prices are under the lens these days—just like everyone else’s. But they don’t all necessarily agree that the scrutiny is fair.

Shire CEO Flemming Ornskov, for one, recently told a group at the Convergence Forum that although he understands why the field has landed in the pricing spotlight, he doesn’t think it belongs there.

As scandal after scandal (after scandal)—and a healthy dose of backlash from politicians—have turned the country’s attention toward drug costs, list prices that would have flown just a couple years back have become unacceptable in the public eye. Rare-disease drugmakers’ go-to pricing justifications—that their meds are innovative and used by few patients, meaning they’re not too big a hit on payers—are no longer getting it done.
Want proof? Just check out the reaction to Biogen’s spinal muscular atrophy drug Spinraza, which set off a firestorm of critical comments and calls for action from lawmakers after the Massachusetts drugmaker unveiled a sticker price equating to $750,000 for the first year of treatment and $375,000 after that. To say it’s way more attention than Alexion got when it set Soliris’ price upward of $500,000 per year would be an understatement.

“The sticker-shock presented in the media could turn Spinraza into the Sovaldi of rare disease drugs,” Leerink Partners analyst Geoffrey Porges wrote to investors at the time, referring to the Gilead Sciences hep C drug that set off a firestorm with its $1,000-per-pill list price.

It could be “the straw that breaks the camel’s back in terms of the U.S. market’s tolerance for rare disease drug pricing,” Porges wrote.

The uproar hasn’t stopped some wannabe rare-disease drugmakers from aiming high, pricing-wise, though it has in some cases changed the way they’re doing it. Israel’s Gamida Cell, for its part, thinks Nicord—its candidate stem cell treatment for blood cancer patients—can “support a justification for a higher price,” CEO Yael Margolin said in a recent interview.

But it’s the med’s cost-saving potential that will ultimately “dictate the pricing,” she said, noting that the company would be looking at pharmacoeconomic parameters as secondary endpoints in its recently begun phase 3 study.

Fellow Israeli biotech VBL Therapeutics, which is slated to participate in Israel’s MIXiii BIOMED Conference taking place later this month in Tel Aviv, is also hoping trial results can help it make its case for cancer-fighting gene therapy VBL-111, but it views overall survival as the key data.

“It will not be a very cheap drug,” Erez Feige, the company’s VP of business operations, said in an interview, adding that “we believe it should be in the range of” Merck’s Keytruda, which rings up at $150,000 per year. But “if the drug is really improving overall survival,” he said, “there’s justification to pay for that.”