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Still no breakthrough on Orkambi talks, MPs want answers

As other price deals go through, Orkambi stalled again


A breakthrough on access to Vertex’s cystic fibrosis drug Orkambi in England looked to be round the corner earlier this year – but two more months of silence has prompted calls for an update from MPs and campaigners.

The deadlock over access to Orkambi has now reached its three year milestone, thanks to a clash between NICE and NHS England and Vertex over the price of the drug.

Campaigners say many patients who could have benefited from the drug have died during the period, and are urging the two sides to reach a deal to allow immediate access for eligible patients.

The parliamentary health select committee called a special hearing on the Orkambi row in March, with Vertex’s CEO Jeffery Leiden flying in from the US especially to give evidence to the committee, and defend his company’s stance.

NHS England and NICE also gave their side of the story at the hearing, after which the two sides resumed talks with the hopes of securing an agreement at last.

However after those meeting in late March, neither side has reported any progress, with campaigners and families with children with CF fearing the impasse could drag on and on.

The chair of the health select committee, Dr Sarah Wollaston MP, was critical of Vertex of at the time of the hearings, but stopped short of taking sides, and instead urged both it and NHS England to forge an agreement.

Dr Wollaston yesterday announced that she had written to NHS England, NICE and Vertex seeking an urgent update on the talks.

Meanwhile patient organisation the Cystic Fibrosis Trust has maintained pressure for political action as well.

Last week campaigners delivered 65 roses – which is how some children pronounce ‘cystic fibrosis’ – along with a letter from the charity inviting Prime Minister Theresa May to attend a Parliamentary Debate on 10 June.

Health select committee chair Dr Sarah Wollaston said: "The best interests of patients should be paramount in the arrangements for access to Orkambi, and the other treatments in the pipeline.

“We continue to urge all parties to the negotiations to keep at the forefront of their minds the needs of cystic fibrosis patients, and to do all they can to ensure that these therapies can be provided to them at a price which does not disadvantage the wellbeing of the many others who also rely on the NHS for their medical care."

Vertex says it remains committed to finding a solution, and said in a statement: "We continue to actively meet with NHS England in the hope of achieving this shared goal through an agreement that is acceptable to all parties.  We are working on a response that will be sent to the Committee in due course."

The lack of progress on Orkambi is in stark contrast to other new, high cost treatments, with NHS England announcing several major new pricing and access deals in recent weeks. These have included those with Biogen on rare disease treatment Spinraza and Roche’s multiple sclerosis treatment Ocrevus.

Article by
Andrew McConaghie

23rd May 2019

From: Regulatory



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