UK urges Vertex to accept 'generous' Orkambi funding offer

Vertex remains at loggerheads with NHS England over funding for Orkambi, but said it would consider the body’s most recent offer of a £500 million, five-year funding deal.

The two sides have struggled to come to an agreement ever since NICE rejected the cystic fibrosis drug on cost-effective grounds two years ago, but recent weeks have seen a flurry of activity.

Earlier this month the biotech firm said it had made the "best offer in the world", with NICE England responding that price – which remains confidential – was “unsupportable”.

NHS England made a counter-offer two days ago that would be worth £500 million over five years, with an option to extend for a further five years, and health minister James O’Shaughnessy said it was “vital that Vertex accepts this generous offer without delay so NHS patients can benefit”.

NHS England said its ‘final proposal’ would guarantee immediate access for cystic fibrosis patients to Vertex’s Orkambi (ivacaftor+lumacaftor), Kalydeco (ivacaftor) and Symdeko (tezacaftor/ivacaftor and ivacaftor).

National director of specialised commission John Stewart said in a letter to the Cystic Fibrosis Trust: "We very much hope that Vertex chooses to act responsibly and accept this deal and take advantage of the significant flexibilities the NHS is offering.

"In the event that this company again chooses not to, their products will now default to NICE to consider independently in the usual way."

So far Vertex has responded cautiously to the new offer, saying in a statement that it hopes the latest offer “signals a willingness to re- engage in discussions”.

"While we are evaluating the proposal, we share the CF community’s concerns that [it] fails to adequately reflect the value of our current and future medicines – and the number of patients that will be treated with these medicines – and stand ready to meet to find a productive path forward,” the company said.

NHS England’s latest offer comes less than a week after Vertex CEO Jeff Leiden wrote to UK Prime Minister Theresa May to express his disappointment about his company’s offer had been rejected. He said that doing so devalued patient lives and threatened the entire UK pharma ecosystem and he had asked her to personally intervene.

The NHS in England currently spends no more than £150 million on standard CF care, for around 4,000 patients. Orkambi costs £104,000 per patient per year in the UK before discounts, according to documents from UK health officials, which would make over £830 million a year to treat the same number of patients.