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Vertex needs to halve the price of cystic fibrosis drugs, says US price group

Three Vertex Pharmaceuticals Inc. medicines carry substantial benefits for cystic fibrosis patients but too large a price tag, according to the U.S. price group the Institute for Clinical and Economic Review.

Both the therapy Kalydeco alone and a combination of Symdeko and Orkambi far exceed cost-effectiveness ranges used by the institute, even when factoring in the special and costlier circumstances surrounding rare diseases.

While the institute, or ICER, has no jurisdiction over pricing, its assessments increasingly carry weight with drugmakers and investors. Sanofi and Regeneron Pharmaceuticals Inc. recently worked with the group to configure a new, lower price range for their cholesterol medicine Praluent.

Kalydeco has a wholesale price of $426.72 per dose and patients generally take two doses a day — racking up an annual cost of nearly $310,000.

Symdeko has a list price of $400.08 and an estimated annual cost of more than $282,000, while Orkambi lists for $186.78 and comes out to almost $265,000 a year.

Depending on the severity of the disease which causes fluid buildup in the lungs, often leading to infections and early death the therapies could add up to more than $7 million over the course of a patients' lifetime.

All three are part of a newer class of cystic fibrosis drugs known as CFTR modulator therapies. While these medicines can significantly improve patients' health when used along with routine care, their prices need to be reduced by half even when the highest cost-effectiveness range is assumed, ICER said.

That would make the per-dose prices $215.09 for Kalydeco, $202 for Orkambi and $188.53 for Symdeko for certain patients, while Orkambi could be $229.94 and Symdeko could be $226.39 for more severe patients.

The draft assessment is open for public comment through April 12, followed by a public meeting of a regional panel, the Midwest Comparative Effectiveness Public Advisory Council, on May 17.