Vertex Strikes Spanish Payment-For-Results CF Deal
Company & NHS England Hope To Announce New Deal Soon
Executive Summary
Vertex has struck a reimbursement deal in Spain for Orkambi and hopes that it will also make progress in England where a new proposal has been made by the company.
After three years of negotiations, the Spanish government and
Vertex Pharmaceuticals Inc. have agreed a payment-by-results reimbursement deal for the cystic fibrosis treatments, Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor). The company says it is also hoping to finally negotiate a deal in England based on newly tabled proposals.
"We are facing a drug that generates clinical uncertainty, so the payment is conditional on the patient responding to the treatment" – Spanish health minister
Under the Spanish deal, announced on 21 October, 554 patients – 441 adults and 113 children – are expected to receive treatment, according to the Spanish health ministry.
Children aged six to 11 with the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene will be able to access Orkambi. Patients aged 12 and older who have either have two copies of the F508del mutation, or one copy of the F508del mutation and a copy of one of other 14 mutations in which the CFTR protein shows residual activity, will be able to access Symkevi.
Both drugs will be dispensed in hospitals after patients have been diagnosed through a genetic mutation test, and will be available from 1 November.
According to the health ministry, the deal is a “mixed model” involving payment for results and a maximum ceiling for expenditure. “We are faced with a drug that generates clinical uncertainty, so the payment is conditional on the patient responding to the treatment," said health minister María Luisa Carcedo.
The agreement was made possible “after intense negotiations with the pharmaceutical laboratory that markets them and which began in June 2016,” the minister added.
Patient organizations welcomed the news. “After almost four years of waiting for an agreement to be reached, today is a very important day for us, and for me, the happiest of my presidency,” said Blanca Ruiz, president of the Spanish Cystic Fibrosis Federation (FEFQ).
Experience gained in resolving the funding issue should also help speed up access to other medications due to be approved for cystic fibrosis, added Juan Da Silva, vice-president of FEFQ. Vertex’s Trikafta (elexacaftor/ivacaftor/tezacaftor), which is expected to meet the needs of 90% of the general cystic fibrosis population, was approved in the US this week. (Also see "Vertex Trikafta Approved As First Triple Combo For Cystic Fibrosis With $311,503 Price Tag" - Scrip, 21 Oct, 2019.). The company has filed the triple combination with the EMA.
Further details about the agreement were confidential, said Vertex. However, it told the Pink Sheet that that the company and the Spanish authorities had “worked in a collaborative and flexible way to find a solution that meets mutual requirements… Our goal has always been to provide access to our cystic fibrosis medicines for all eligible patients.”
Kalydeco (ivacaftor) is already reimbursed in Spain for the majority of eligible patients.
English Deal Soon?
Meanwhile, Vertex is hoping that a deal will be agreed over the next few weeks in England, where cystic fibrosis patients still have no access to Orkambi despite protracted talks. The company’s CEO Jeff Leiden met health secretary Matt Hancock on 8 October. Vertex “brought another solution to the table that would provide immediate access for patients to CFTR modulators and we are committed to trying to negotiate this to a formal agreement within weeks,” said the company.
At a Health and Social Care Committee meeting on 22 October, NHS England chief executive Simon Stevens was also cautiously hopeful that a deal might soon be announced. “I am increasingly confident we will be able to reach an agreement between NHS England and Vertex,” said Stevens. He hinted the two parties were as close as they have ever been to agreeing a deal, commenting that they are on “the verge of an agreement.” However, he added the caveat that Vertex must accept that NICE's endorsement is necessary. “Until the deal is concluded, it is not concluded,” he declared.
The issue of access to Orkambi is controversial in England and has even been debated in parliament. Some patients are accessing a generic version of the drug produced in Argentina through a buyers club. (Also see "UK Buyers Club To Get Lower Cost Versions Of Vertex’s CF Drugs " - Pink Sheet, 6 Jun, 2019.)
It is unclear if the deal proposal will resemble the five-year deal struck in September with the Scottish government for the funding of Orkambi and Symkevi. (Also see "RWD Key To Scottish Orkambi Deal" - Pink Sheet, 12 Sep, 2019.) Under this agreement, the company offered a further discount and agreed to gather more real world evidence on the effects of the drugs. A new access agreement will be discussed at the end of the of the five-year period.
Vertex announced a reimbursement deal for the two drugs in Australia on 20 October. The agreement expands access to Orkambi to children with CF aged 2 to 5 who have two copies of the F508del mutation in the CFTR gene. Patients over the age of six are already able to access the drug.
This article was updated on 23 October to include comments from Simon Stevens.