Parents of Jarrow youngster feeling frustrated as row continues over life-saving drug

Emma and Chris Corr have been campaigning, alongside thousands of other parents whose children have been diagnosed with cystic fibrosis, to have Orkambi available on the NHS.

The couple - whose three-year-old daughter Harriet has cystic fibrosis - had been hopeful a deal would be reached between drugs company Vertex and NHS England.

A counter-proposal by the NHS of £500m over five years has been put forward, believed to be the largest financial commitment in its 70-year history.

But with Vertex charging £104,000 per patient, per year for Orkambi, Emma s concerned about the offer.

Each day, the youngster, who was diagnosed with the condition at just five days old, depends on a variety of drugs to help keep her alive and also requires regular physiotherapy.

The company says it appreciates the offer and hopes it “signals a willingness to re-engage in discussions” to reach the collective goal of providing access to medications.

A spokesman said: “While we are evaluating the proposal, we share the CF community’s concerns that this proposal 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.”

However, in a letter released by NHS England, it is hopeful Vertex will accept the deal and “take advantages of the significant flexibilities the NHS is offering.”

If accepted, it would guarantee immediate and expanded access for patients with cystic fibrosis to the two licensed medicines, Kalydeco and Orkambi. It would also provide immediate access for Symdeco from the date it is licensed for use in the UK.

But it added: “In the event that this company again chooses not to, their products will now default to NICE (National Institute for Clinical Excellence) to consider independently in the usual way.”

Mrs Corr, from Wansbeck Road, Jarrow, said: “NHS England have put forward an offer for something in the tune of £500m to cover existing medications and the medications they have got coming. They have said it is the biggest financial commitment to anything they have made before.

“On the outside, it’s like wow, that’s amazing but Vertex charges around £100,00 per patient. We have other countries paying £100,00 per patient, the whole thing is a mess.

“There was a debate in parliament, again the MPs were saying all the right things, but we have heard all this for the past three years, but nothing is happening.

“The NHS said this is their final offer which is extremely worrying. The whole thing is horrendous, it’s like our children are being used as bargaining chips.

“There are no options available to us, to raise this kind of money ourselves each year is not sustainable. And what is the other option, to uproot my family to go and live in Ireland where the drug is available? I pray they can come to some sort of deal.”

Vertex CEO, Dr Jeff Leiden, has said he is “ready to meet” with NHS England CEO Simon Stevens, “at any time, any place” so a conclusion can be reached.