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Parents of children with cystic fibrosis face 'postcode lottery' if Scotland gets go-ahead for life-changing drug

The i logo The i 09/08/2019
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Parents of children with cystic fibrosis in England say they would be forced to move to Scotland if medical chiefs there give the go-ahead on Monday to life-transforming drugs.

One mother of a nine-year-old boy with cystic fibrosis said sufferers would be in a “postcode lottery of their lives” if the Scottish Medicines Consortium (SMC) approves universal use of Orkambi and Symkevi, two treatments that have provided radical improvements for cystic fibrosis patients.

Cystic fibrosis is a genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus.

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The drugs are not available in England because the NHS and Vertex, the pharmaceutical company which manufactures them, are in a long-running battle over funding. The list price is £104,000 per patient per year, although the company says it has offered a discount of “tens of thousands of pounds” to the NHS in England. While the exact details are shrouded in secrecy, NHS England says the costs are still too high.

Life-changing drugs

Families have been campaigning for the life-changing treatment

More than 100 MPs from all parties have written to the Health Secretary, Matt Hancock, and Dr Jeffrey Leiden, the chief executive of Vertex, urging them to break the deadlock.

If the SMC grants universal access approval in Scotland for the two drugs – a decision thought to be on a knife-edge – it would benefit around 400 patients. It is thought that several thousand cystic fibrosis patients in England, which has the second highest number of cases in the world, have the genotypes which are suitable for Orkambi or Symkevi.

While health has been devolved in Scotland for 20 years, any disparity in cystic fibrosis treatments would be one of the starkest examples of a “health border” with England, exacerbated by the re-emergence of SNP calls for a second independence referendum because of Brexit.

Case study — ‘We’ll move if drugs get green light’

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Christina Walker, who lives in East Sussex, said her family will consider moving to Scotland if her nine-year-old son, Luis, could be prescribed new drugs Orkambi and Symkevi

Christina said a green light in Scotland on Monday would force her family to relocate north so she could get potentially life-extending treatment for her nine-year-old son, Luis, even though it would mean they would be moving away from her recently widowed father. She said other families were considering the same action.

She said: “To know there are drugs available that could extend and improve his life, yet Luis can’t access them, is torture in the extreme and I can’t allow that to be the case. It’s life and death for Luis and thousands others.

“If there’s a deal in Scotland and people from around the UK can access the drugs by moving there, I will be forced to do so. This will tear me apart because Luis and I are very close to my 86-year old Dad, and we recently lost my Mum to cancer. But how could I not do everything in my power to give my son a chance at a longer life? On Monday people with cystic fibrosis will find out if they’re in a postcode lottery for their lives.”

An NHS England spokesman said: “NHS has made two of the most generous offers of its kind to this company and intensive work continues on a daily basis, but the quickest way for patients to get access to Orkambi is still for Vertex to accept our offer and engage with Nice.

a close up of a newspaper © Provided by Johnston Publishing Ltd “Vertex is an extreme outlier in both pricing and behaviour. The pharmaceutical trade body said the offer represents the sort of flexibility industry wants and warned companies they cannot just pick any price they like for a new medicine.”

A spokesman for the Department of Health and Social Care said: “We know how terrible cystic fibrosis can be for those affected and their families – we are in constant discussions with Vertex in order to get them to engage constructively.”

Vertex said in a statement that the company “continues to meet regularly with NHS England to discuss access to our potentially life-changing cystic fibrosis medicines”, adding: “We remain highly committed to the ongoing negotiations and our intensive work continues on a daily basis.

“In the absence of an access agreement, Vertex will continue to provide free medicines to patients who are the most seriously ill, based on objective clinical criteria.

“Since its inception, these medicines have supported more than 800 of the sickest CF patients in England and over 1,000 patients across the UK.”

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