Amid pricing spat with U.K. government, Vertex declines to submit data on new CF drug to NICE

The battle between Vertex Pharmaceuticals and U.K. health officials over cystic fibrosis drug pricing has reached an impasse after NHS England’s "final" offer in July. Now, Vertex is withholding data on new drug Symkevi that patients are anxiously awaiting until the NHS fixes what the company calls a flawed appraisal system.

In a statement, Vertex director of external communications Heather Nichols said the company supports "the need for robust and fair medicines appraisal in England." But, she said, Vertex believes "NICE’s single technology appraisal has not kept pace with changes in medicine and has significant limitations in how it captures and values the full benefits of precision medicines such as tezacaftor/ivacaftor." 

As such, Vertex hasn't submitted data on the forthcoming CF drug to the watchdog. Nichols said the company "would be happy to re-engage with NICE in the context of an appropriate appraisal process, which takes into account the benefits our cystic fibrosis medicines provide to people with this devastating disease."

The development, which sparked disappointment from patient advocates, comes after the sides recently reached a standstill in negotiations over funding for Vertex’s suite of CF drugs, including the already approved Orkambi and others in development. Symkevi, for its part, won a European panel's backing for approval late last month. The drug is called Symdeko in the U.S. and won FDA approval in February.

Vertex and U.K. health officials have been trying for years to reach a deal for Orkambi, which secured European approval in 2015. Last month, after those talks failed to yield a deal, Vertex CEO Jeffrey Leiden took his complaints to the very top. He reached out to U.K. Prime Minister Theresa May to say that England’s decision to reject Vertex’s pricing offer devalued patient lives and threatened the U.K. biopharma ecosystem. The company, which maintains an international HQ in London and a R&D center near Oxford, would have to reconsider its commitment to the U.K.,  he wrote.  

A week later, NHS England made a "final" offer that would have provided Vertex revenues of £500 million over 5 years and £1 billion over 10 years for its CF drugs, the agency's "largest ever financial commitment" in its 70 year history. Still, Vertex said the offer "fails to adequately reflect the value of our current and future medicines and the number of patients that will be treated with these medicines."

Now, Nichols said the company "remains committed to working with all stakeholders to ensure all eligible patients with CF in England get rapid access to all our current and future medicines." Leiden contacted U.K. officials "weeks ago" to try and resume talks, according to Nichols, but the company hasn’t heard back. 

Meanwhile, patients and advocates have been urging the sides to reach a deal. After the latest development, Cystic Fibrosis Trust chief David Ramsden said in a statement it’s "awful news ... and it is a situation that I find deeply concerning." 

"Every day that people in the U.K. are unable to access new precision medicines is a day too long and it means people who could see their lives prolonged are dying," he added. 

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