Firm must come clean over wonder drug, says minister
A MINISTER has urged a US firm to provide vital new facts about its cystic fibrosis wonder drugs, so sufferers can soon have them on the NHS.
Vertex has stalled over a £108million-a-year NHS offer for its lifesaving medicines such as Orkambi for almost 18 months.
The National Institute of Health and Care Excellence (NICE) refused to recommend Orkambi to the NHS in 2016, because of its cost and lack of clinical data.
NICE now wants to reassess Orkambi and Vertex’s newer version Symkevi, but is desperate for fresh information from the firm.
Health minister Jo Churchill told Parliament yesterday that if Vertex supplied all its data proving Orkambi and Symkevi’s effectiveness, NICE could recommend the drugs to the NHS.
Taking either of the drugs would immediately help around 5,000 sufferers of the genetic condition – half our CF population – with the right gene mutation to take them.
Ms Churchill told MPs: “NICE is in the process of inviting the company [Vertex] to participate in this appraisal.
“If the company participates, NICE expects to be able to come to a decision on the use of Orkambi in the autumn of 2020.
“The Secretary of State for Health wrote to Vertex in August to consider what the barriers are to an agreement and how the situation can be resolved.”
But confusingly, Vertex CEO Dr Jeff Leiden told Daily Express columnist and CF sufferer Carlie Pleasant, 29, this week that his firm had given NICE all its data.
He told the mother of one: “We have submitted all the evidence we have to the Government on all our medicines.”
This was denied last night by NICE, which told this newspaper: “We have asked Vertex to participate in the evaluation of Orkambi. However there has not been a positive response from the company.
“If the company decides to participate, we will do our utmost to publish guidance in the autumn of 2020. NICE also stands ready to receive an evidence submission for Symkevi, for which we invited Vertex to provide evidence in May 2018.
“Again, we will do our utmost to publish guidance for this product in 2020, if the company decides to participate.”
The institute warned: “We operate a large work programme and we cannot continue to hold slots for these evaluations for much longer.”
Since 2016, NHS England has offered Vertex a £108million-a-year deal covering all its current CF drugs – Kalydeco, Orkambi and Symkevi. Vertex has refused to supply extra data on Orkambi or any for Symkevi until NICE changes the way it assesses drugs, following the price row.
The Daily Express has been crusading for NHS England to strike a deal with Vertex for its CF lifesaving drugs.
While Orkambi has been unavailable in the UK, it is feared more than 263 young sufferers who could have taken it have died needlessly.
Carlie, of Sittingbourne, Kent, tackled Dr Leiden for 30 minutes this week over the UK’s agonising drugs stalemate. During the emotional meeting, Dr Leiden denied-Vertex had been inactive and stressed he was always striving to secure a deal.
He told Carlie: “I can only imagine how frustrating and frankly frightening this [situation] must be for you.
“I do have a full appreciation of how difficult it is, how frightening it is, and the tremendous hope [these medicines] bring.
“I personally have a commitment to bring these medicines to every patient around the world.
“The Government is considering our proposals and we are waiting to hear back from them.”
If Health Secretary Matt Hancock shakes hands on a new deal soon, the drugs could potentially become available within a week of their agreement.
Campaigner Alison Dillon, the mother of CF-suffering TV actor Oli, 21, said: “CF sufferers do not have the luxury of time. They need these drugs now.
“Even one week’s delay can cause more damage to a CF sufferer’s lungs and mean more lives lost.”