UPDATED: NICE sticks to its guns and turns down Vertex's Orkambi even as patients plead

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England’s drug cost watchdog today gave its near-final denial to Vertex’s cystic fibrosis combo Orkambi, saying the drug’s price was just too high to be a “cost-effective use of NHS resources.” That verdict came despite patient advocates decrying the agency’s recommendation and pressuring both the body and the Boston-based Vertex ($VRTX) drugmaker to find common ground on a price.  

The  National Institute for Health and Care Excellence reiterated its earlier stance that while use of Orkambi reduced hospitalizations for CF patients, Orkambi--which costs about  £104,000 ($147,000) per patient--hadn’t separated itself enough from existing treatments, only showing “modest” lung function benefits.

“We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high,” Carole Longson, director of the NICE Centre for Health Technology Evaluation, said in the announcement of the decision. “We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it.”

In its own statement today Vertex said: "Today’s recommendation is an expected part of the complex reimbursement process in England. We are pleased that NICE has recognized the clinical benefits of ORKAMBI and remain confident we will be able to work with all parties involved to make ORKAMBI available to eligible patients in England, just as we did with KALYDECO."

The Cystic Fibrosis Trust Friday pointed out that since that initial thumbs-down in March, new data have shown that Vertex’s drug slows the rate of decline in lung function for CF patients by nearly half and can possibly add years to patients’ lives. It pointed to a petition that has so far garnered 20,000 signatures in its efforts to get the groups to have “urgent discussions” and come to an agreement that would ensure patient access.

Cystic Fibrosis Trust Chief Executive Ed Owen in a statement Friday said the decision by NICE "demonstrates the weakness of the current system, and NICE has been unable to confidently predict the long-term benefits of the drug,” Owen said.

NICE acknowledge that the Cystic Fibrosis Trust suggested additional real-world data could be collected, which would demonstrate that Orkambi does offer significant benefits in the long-term. But it said the committee concluded that that could only be accomplished if Vertex cut the price and also pointed out that Vertex did not suggest that route. It did say the company could appeal before the decision becomes final in July or could return later with a different cost proposal.

The CF advocy group said nearly 2,800 people in England could benefit if an agreement is reached. NICE put that number at 2,750. At the $147,000 per patient price that Vertex has offered, that could amount to more than $404 million in sales. That is more than Orkambi brought in in the U.S. last year, where its retail price is $259,000. Vertex reeled in $220 million in Orkambi sales last year, $30 million short of analyst expectations. That compared with a Street-beating $630 million haul for Kalydeco, its predecessor, whose patient pool is 5 times smaller.

- here’s the NICE announcement

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