Vertex Pharmaceuticals was already playing hardball with the cost-effectiveness watchdogs in England as price negotiations for its drug Orkambi hit another impasse last week. But CEO Jeffrey Leiden took it to a new level Friday with a letter to Prime Minister Theresa May.
In a big departure from the usual back-and-forth with the National Institute for Health and Clinical Excellence, Leiden argued that rejecting Vertex’s pricing offer for the cystic fibrosis drug threatened the entire U.K. biopharma ecosystem.
After years of pricing proposals to U.K.'s cost-effectiveness agency, Leiden said the company’s latest offer for its suite of CF drugs was the “most innovative and best” it has made. Rebuffing it ultimately devalues patients, the CEO said.
And in a direct political threat to May, Leiden said his company is now questioning its own commitment to the U.K. It maintains an international HQ in London and a R&D center near Oxford.
NHS England had already thrown down the gauntlet as negotiations collapsed last week. "If Vertex really believe they are offering a reasonable deal they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair," NHS England said in a statement.
Vertex hasn’t taken the agency up on that. In response, Vertex director of external communications Heather Nichols said NHS specifically asked the company to sign a "mutual confidentiality agreement." The agreement prohibits Vertex from disclosing details of the offer.
For its part, NICE has determined the price is “unsupportable," according to NHS. NHS England pointed out that NICE is a “long established, internationally respected independent body" whose entire mission is weighing the costs and benefits of treatments. Orkambi costs £104,000 per patient per year in the U.K. before discounts, according to NICE documents.
This isn’t the first time that NICE has run afoul of biopharma companies. The agency has been notoriously tough on cancer treatments, particularly as prices have rocketed upward over the past decade. Roche, for instance, had a long-running spat with the agency over pricing for its breast cancer drug Perjeta, only recently resolved with a deal. CEO Severin Schwab famously called one agency move “stupid” and “arbitrary.”
Vertex, meanwhile, claims it has made the "best offer in the world" in the U.K., reflecting its goal to "provide access to all of our medicines for all cystic fibrosis patients as soon as possible." The company says the proposal includes Kalydeco, Orkambi, Symdeko and pipeline meds for CF. The offer provides "budget certainty" for NHS and "rapid access" for patients, according to the company.
Vertex has some broader criticisms, saying that NICE needs to revamp its assessment processes to keep up with new science. The agency has shown "no flexibility" in the negotiations and is adhering to "arbitrary value thresholds," Leiden wrote. By doing so, he argues the agency is "placing a lower value on the life of a CF patient" in the U.K. than in other countries around the world.
And Leiden told May that future biotech investment in the U.K. "is at significant risk" if NICE”s system lags. Further, he said Vertex is questioning the U.K. biotech ecosystem "as it is unable to value life changing medicines for the patients that need them."
RELATED: NICE sticks to its guns and turns down Vertex's Orkambi even as patients plead
Vertex called on the government to step in and said it stands ready to meet to strike a deal. If May were to intervene, however, that would undermine the work at NICE and NHS England.
Patient advocates aren't fond of the public fight. In a statement, Cystic Fibrosis Trust chief executive David Ramsden said his group is "appalled and deeply frustrated" to hear about developments in the negotiations. In a related blog post, Ramsden called on the sides to make their arguments in the negotiating room instead of in the public.
"Vertex and NHS England must get back around the table quickly and ensure these medicines are made available to people with cystic fibrosis," Ramsden said in a statement.
RELATED: Vertex cancels plans for CF triple combo testing in France, citing failed Orkambi negotiations
It's not the only time Vertex has faced resistance securing coverage for its pricey CF meds. The company in February canceled plans to test a new triple combo CF drug in France because Orkambi reimbursement negotiations in the country hadn't gone anywhere. The trials were later resumed, Nichols said on Monday.
In the U.S., Vertex recently blasted cost watchdog ICER over its analysis of CF drug costs. After ICER said discounts of up to 77% are warranted on Vertex drugs, the company called ICER's evaluation process a "sham." Vertex argued ICER's findings were "agenda-driven and pre-ordained.” Payers have granted broad access to Kalydeco, Orkambi and Symdeco, according to Vertex, demonstrating that they have “universally recognized" value for the meds.
RELATED: ‘Sham’ or public interest? ICER suggests 70%-plus discounts on Vertex’s cystic fibrosis drugs
ICER found the drugs offer significant benefits, but come with costs "far in excess of those needed to reach commonly cited cost-effectiveness thresholds," according to an official.