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Vertex told to reach UK agreement in ‘final’ Orkambi submission or have prices published

by Natalie Morrison
LONDON, 9 Nov (APM) - The UK government has issued Vertex with an ultimatum on its cystic fibrosis drug Orkambi - to reach an agreement with NICE and the NHS by the end of the month or to see its proposed prices published.
The call follows a two-and-a-half year battle for reimbursement of the cystic fibrosis transmembrane conductance regulator (CFTR ) modulator in the UK.
Pressure to reveal the price has been a huge issue for NICE and the NHS throughout repeated rejections, during which they have pushed Vertex to unveil the “unsupportable” asking price.
On Wednesday, the Health and Social Committee’s Chair Dr Sarah Wollaston sent the government’s latest demands to Vertex’s chief executive officer Dr Jeffrey Leiden. The letter was published on Parliament’s website on Friday.
The committee calls for a “final submission” from Vertex to NICE for drug Orkambi (lumacaftor+ivacaftor) by 30 November which reveals details of the drug’s price, and says the committee will publish the documents it receives unless an agreement between the company, NHS England and NICE is reached before this date.
If this happens, it would be the first time any indication of the drug’s proposed UK pricing has been revealed since NICE first denied reimbursement in March 2016 (APMHE 47016), when it said Orkambi would cost £104,000 per patient for every year of treatment.
“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,” the NHS said in a statement after the latest rejection this year (APMHE 58826).
Vertex, meanwhile, said it had made “the best offer in the world” for the NHS, and accused it of placing a lower value on the life of UK cystic fibrosis patients than that given by other countries to their patients.
Wollaston said Vertex’s submission should include full details of all formal offers made to NHS England, including the price and overall money sought from the NHS over a five, 10 and 15-year timeframe.
Besides revealing the UK price, the letter also calls for Vertex to give details on its pricing elsewhere.
Wollaston asks for “evidence in support of your public statement that you have offered the NHS in England the lowest price for your drugs of any country in the world”.
“This should take the form of the specific pricing and commercial arrangements you have entered into in each country where Vertex sells its medicines,” she said.
Wollaston also called on Vertex to come forward with any further formal offers to NICE, either to reduce the price or to provide further evidence of greater clinical benefit.
It seems Vertex has little choice but to respond to the request.
Wollaston warned: “As previously indicated, the committee would expect to publish any documents received, and it will use the formal powers granted to it by the House of Commons to require the provision of those documents if they are not provided in response to this request.”
“The committee expects to receive the documents it has requested by Friday 30 November. Should an agreement between Vertex, NICE and NHS England have been reached by that date for reimbursement at a level the NHS would recognise as cost effective, I would not expect the committee to publish any documents it has received or to progress its inquiry any further at the present time.”
Parliament’s website also published letters in similar terms to NICE and NHS England from Wollaston to request the provision of relevant documentation which they hold.
This includes a letter to NICE’s Sir Andrew Dillon asking for the “unredacted assessment of the clinical and cost-effectiveness of the drugs presented to the NICE appraisal committee, and the committee’s findings”, as well as details of the difference between Vertex’s submitted price and a “cost-effective price”.

Hope

Parliament’s website adds that the Health and Social Care Committee’s hope is that negotiations are successful and a deal can be reached so that Orkambi can be made available to NHS patients.
“It looks forward to hearing that agreement has been reached by the end of the month, in which case it has indicated that it would not be likely to progress its inquiry any further at the present time,” it said. “If no agreement is reached by that date, it will consider further how to proceed.”
In an emailed statement in response to APM’s questions on the letter, Vertex confirmed it had received the notification from the Health and Social Care Committee.
“Vertex welcomes the committee's interest in access to cystic fibrosis medicines in England and we look forward to supporting its inquiry into this important issue,” it said.
It did not, however, respond specifically to questions over whether Vertex feels it has indeed been issued an ultimatum, or on other matters surrounding the situation.

Government intervention at last

The letter could be seen by some as the government finally intervening in this situation, after years of back and forth between Vertex, the NHS and NICE.
Last month, Nick Medhurst, head of policy and public affairs for the UK’s Cystic Fibrosis Trust, told APM the government's failure to intervene previously was tantamount to 'passively' accepting that UK patients will not have access to the most innovative drugs (APMHE 60381).
The UK has the world's second largest cystic fibrosis population, with around 11,000 patients.
The latest action over Orkambi follows shortly after European approval for Symkevi (tezacaftor+ivacaftor). There have been suggestions that lack of Orkambi reimbursement in the UK could affect Vertex's drive to gain reimbursement for Symkevi on the NHS. In August, NICE suspended a review of Symkevi after Vertex reportedly failed to provide evidence for the appraisal (APMHE 59374). According to media reports, the company said it would not file the data until NICE made changes to its assessment procedures.
However, Vertex has said it is in renewed discussions with the NHS over Symkevi (APMHE 60444).
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