MPs are due to hear evidence relating to the cystic fibrosis drug Orkambi as campaigners warn that families “are at their wits’ end”.
A disagreement between NHS England and US firm Vertex Pharmaceuticals over the price of the drug shows no signs of being resolved more than two years after negotiations started.
The Health and Social Care Committee (HSCC) is holding an evidence session on Thursday to hear from those involved in the talks in the hope of breaking the deadlock.
The NHS has offered £500 million over five years for access to Orkambi and other cystic fibrosis drugs made by Vertex, with the promise of renewals in the future.
But this has been rejected by Boston-based Vertex, which has secured deals with other countries for access to Orkambi and has criticised the way new drugs are appraised in the UK.
Orkambi is a game-changing drug for cystic fibrosis, which affects more than 10,000 people in the UK.
It is suitable for about half of people with the condition. Other drugs being developed by Vertex are showing astonishing results in clinical trials and could potentially treat most people with cystic fibrosis.
Orkambi has a list price of £104,000 per patient per year and the drug must be taken for life.
Vertex is understood to have offered to reduce this list price, though the National Institute for Health and Care Excellence (Nice) has said the US company would need to drop the list price by 90% in order for it to be approved on the NHS.
Vertex has said this is unacceptable and has also failed to reach agreement with NHS England, which intervened in the hope of brokering a compromise.
Last summer, Vertex called on Prime Minister Theresa May to take action and said NHS England was being “outrageous”.
For its part, NHS England has said it hopes “Vertex chooses to act responsibly” and accept its deal.
The HSCC will hear from Professor Stephen Powis, national medical director of NHS England, Sir Andrew Dillon, chief executive of Nice, Dr Jeff Leiden, president of Vertex, and Stuart Arbuckle, executive vice president of Vertex.
Orkambi is available in other countries including Germany, Italy, Sweden and the US. Patients in Germany have had access to the drug since 2015.
The Cystic Fibrosis Trust has submitted evidence to the HSCC, saying that an immediate resolution is needed and interim access to the drug must be secured for patients.
It also said the pricing dispute between NHS England and Vertex was anticipated in 2015 when the Cystic Fibrosis Trust warned that Orkambi would not pass Nice’s appraisal process due to price.
Every day this continues to drag on is another day too long.Nick Medhurst, Cystic Fibrosis Trust
It said Nice and NHS England have not managed the challenge relating to price “without causing considerable harm through delay of over three years”.
Nick Medhurst, head of policy and public affairs at the Cystic Fibrosis Trust, told the Press Association the charity hoped the HSCC hearings would hold Vertex, NHS England and Nice to account.
But he added: “We are acutely aware that it’s not a decision-making forum and there is important work still to be done in finding a deal to get access to Vertex’s medicines.
“We understand the company will meet with senior officials from NHS England and possibly government, but every day this continues to drag on is another day too long.
“Other countries around the world have found a way which gives us hope that a compromise can be found.
“Cystic fibrosis is a progressive disease and every exacerbation of ill-health is something that may have been avoided with treatment that is available.”
He said the charity was hearing from families “decrying” the ongoing stalemate, adding: “They are at their wits’ end”.
Each day that passes without a deal means the risk of more damage to the lives and prospects of thousands of children and adults with cystic fibrosis across the UKDavid Ramsden, Cystic Fibrosis Trust
David Ramsden, chief executive of the charity, said: “All three parties – Nice, NHS England and Vertex – must reach a compromise and end the agonising three-year wait for these life-saving medicines.
“Each day that passes without a deal means the risk of more damage to the lives and prospects of thousands of children and adults with cystic fibrosis across the UK. This tragic situation must end.”
Christina Walker, the mother of eight-year-old Luis, from Horam, East Sussex, said the behaviour of Vertex should be called to account.
She said: “Vertex’s exclusive pricing strategy has effectively withheld life-changing medicines from patients like my son for more than three years.
“It’s all about corporate profits. They blame the appraisal system as a distraction from excessive pricing, while not engaging with it in good faith.
“The price of Orkambi is particularly prohibitive in England because we have 12% of the global cystic fibrosis community.
“NHS England would be the highest single payer by volume so the cost just can’t be supported and, more importantly, isn’t justified.”