A years-long cystic fibrosis drug pricing standoff has finally yielded a deal, and now thousands of patients in the U.K. are set to access key medicines from Vertex Pharmaceuticals.
Vertex and NHS England have struck a deal requiring the drugmaker to submit its suite of medicines to England’s cost watchdog NICE for a full review. During and after that process, patients will be able to access the drugs under a "flexible commercial mechanism" the company offered up, the NHS says. Financial terms of the deal were confidential.
More than 10,000 people in the U.K. have cystic fibrosis, according to Vertex, and more than 8,000 patients are in England. Of those, about 5,000 can benefit from Vertex’s currently approved drugs. The U.K. has the second-highest prevalence of CF worldwide, according to NHS.
In a note to clients, Jefferies analyst Michael Yee wrote the deal opens up $500 million in sales per year. Earlier this week, Spain and Australia reached their own access agreements with the drugmaker, he pointed out.
The NHS agreement covers Vertex's Orkambi, Symkevi and Kalydeco. Officials expect the meds to be available to patients within 30 days.
It’s a “very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting,” Cystic Fibrosis Trust chief executive David Ramsden said in a statement.
The deal, which NHS chief exec Simon Stevens called “good for our patients and fair to British taxpayers,” shows that drugmakers must work “constructively” with the country’s health systems, he said.
But the agreement comes after years of deadlock. Orkambi won its European approval in 2015, and patients and lawmakers have been pushing the sides to reach an access agreement after multiple rounds of failed talks.
Tensions rose last summer when Vertex CEO Jeffrey Leiden reached out to then-Prime Minister Theresa May to say that the country’s stance in the negotiations devalues patients and threatens the country's biopharma industry. During the negotiations, Vertex said it had made its best pricing offer worldwide to England, while British authorities said they had offered to pay more for the CF drugs than for any other medicines in NHS history.
“It has at times been frustrating, and for patients distressing,” Stevens said in a letter to Sarah Wollaston, chair of a British health committee. “But I am in no doubt that the independent, fair, flexible, and objective approach we take to drug price appraisal and negotiation in this country—borne out of more than two decades of internationally respected experience and expertise—is something that we squander at our peril.
“In the round, both patients and taxpayers benefit,” he added.
The deal comes right after Vertex this week won an early U.S. approval for triple combo CF med Trikafta, which could one day treat 90% of CF patients, CEO Jeffrey Leiden said. Ramsden said his group will continue to push for patient access to the best available options.