German cost watchdogs look askance at uniQure's $1.4M gene therapy

German cost-effectiveness watchdogs aren't among pharma's favorite people. Since lawmakers instituted a tough price-setting process, state officials have questioned the benefits of one new drug after another--to the point where some drugmakers decided not to launch there at all.

But a debate now playing out there is different. The first of its kind, actually; the Federal Joint Committee is assessing uniQure's ($QURE) Glybera, the first-ever gene therapy approved in Europe, and now the world's most expensive drug at €1.1 million ($1.4 million). And the questions raised in Germany offer a preview of reimbursement conversations in other countries, about this and other uber-pricey gene therapies.

So far, officials are skeptical. The "added value" of Glybera, used to treat a rare metabolic disease that triggers pancreatitis, is "classified as non-quantifiable," the committee said in a Thursday statement. "No scientifically sound statement on the extent of additional benefit is possible based on the data submitted," Germany's Federal Joint Committee said in a statement.

The group asked for more data on safety and efficacy to power a new assessment due next June.

uniQure CEO Jörn Aldag

As Bloomberg notes, German insurers set drug prices based on input from government assessors--and this equivocal decision could interfere with UniQure's ability to collect full price for its med. But UniQure and its marketing partner Chiesi hope to use math to prove their case: CEO Jörn Aldag points out that Glybera is given once, and studies show it's effective for 6 years. That's $170,000 per year, less than orphan drugs such as Alexion's ($ALXN) Soliris, which tops $300,000.

Makers of potential gene therapies--and payers, of course--have been mulling over a variety of payment models to make the ultra-expensive treatments affordable, including spreading the cost out over time and making payments contingent on results. In the meantime, though, the early entrants face an uphill battle. 

As a gene therapy, however, Glybera isn't 100% representative. UniQure's attempts to win regulatory approval repeatedly faltered on lack of evidence. And now, European regulators are digging back in, worried that the drug doesn't remain effective as long as claimed.

Aldag is philosophical about the current hurdles. "It is often hard to be the first," Aldag told Bloomberg. "This is where people are learning. Not only are we learning, but so are the regulatory agencies."

- see the Joint Committee's statement
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Special Report: Top 10 most expensive drugs of 2013 | The 25 most influential people in biopharma today - 2013 - Jürgen Windeler, IQWiG