{"id":235525,"date":"2019-02-04T18:14:06","date_gmt":"2019-02-04T18:14:06","guid":{"rendered":"https:\/\/healthmedicinet.com\/i\/deadlock-must-end-over-cystic-fibrosis-drug-orkambi\/"},"modified":"2019-02-04T18:14:06","modified_gmt":"2019-02-04T18:14:06","slug":"deadlock-must-end-over-cystic-fibrosis-drug-orkambi","status":"publish","type":"post","link":"http:\/\/healthmedicinet.com\/i\/deadlock-must-end-over-cystic-fibrosis-drug-orkambi\/","title":{"rendered":"‘Deadlock must end’ over cystic fibrosis drug Orkambi"},"content":{"rendered":"
\n
Media caption<\/span>Luis tells the Victoria Derbyshire programme: \u201cI wish I could just have a normal life”<\/figcaption><\/figure>\n

The government must use its powers to break a deadlock in making a life-extending drug available to people with cystic fibrosis, campaigners say.<\/p>\n

Vertex, which makes Orkambi, has refused a \u00a3500m offer for the drug over five years – the “largest commitment” the NHS has ever made.<\/p>\n

Those affected want other drug firms to be asked to make a cheaper version.<\/p>\n

The Department of Health said its approach “remains urging Vertex to accept NHS England’s generous offer”.<\/p>\n

Vertex said it was “determined to find a solution that allows the NHS to provide patient access to our precision medicines across the UK with budget certainty, and also allows Vertex to continue its research and focus on a cure for CF [cystic fibrosis] and other serious diseases”. <\/p>\n

It currently charges \u00a3100,000 for a year’s treatment with Orkambi.<\/p>\n

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage and affects around 10,400 people in the UK.<\/p>\n

Only around half of those with the condition live to celebrate their 40th birthday.<\/p>\n

Christina Walker’s son Luis, eight, was born with it.<\/p>\n

She told the BBC’s Victoria Derbyshire programme<\/a> his medical regime takes up to four hours a day.<\/p>\n

“Every day he takes 22 tablets, three nebulisers, two inhalers, nasal sprays and lots of supplements,” she said. “Those are the good days.<\/p>\n

“A bad day will see Luis in hospital. A cough or cold will have turned serious, his lung function will plummet, he’ll be off school for weeks, missing not just classes, but birthdays and time spent with friends – the little things that make being eight years old so much fun.<\/p>\n

“Or it might be like last week when he had a stomach bug and was reduced to skin and bones as his weak body sheds the tiny amount of weight he’d been able to put on previously.”<\/p>\n

Improves lung function<\/h2>\n

The drug Orkambi is licensed to treat cystic fibrosis in patients from two-year-olds to adults, who have a specific genetic mutation known as F508del.<\/p>\n

This causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.<\/p>\n

The drug has been shown in clinical trials to improve lung function and respiratory symptoms in people with cystic fibrosis.<\/p>\n

It is the first of a string of drugs that have been developed, with newer ones expected to be even more effective. <\/p>\n

Ms Walker said for Luis it would be “life-changing”.<\/p>\n

Crown Use<\/h2>\n

The manufacturers Vertex Pharmaceuticals want the NHS to pay \u00a3105,000 per patient per year, and have rejected the NHS’s offer of \u00a3500m over five years.<\/p>\n

Campaigners want the government to set aside Vertex’s patent to allow cheaper versions of Orkambi to be made.<\/p>\n

This is backed by Conservative MP Bill Wiggin, who will ask the government to invoke Crown Use in an adjournment debate in the House of Commons on Monday – which would allow this to happen.<\/p>\n

Vertex said in a statement: “To invoke Crown Use and provide third parties access to Vertex’s intellectual property would seriously undermine our ability to achieve these goals and would significantly weaken incentives for future innovation.”<\/p>\n

Ms Walker – who campaigns on the issue – told Victoria Derbyshire: “[Other] suppliers are making the drug already for \u00a35,000.”<\/p>\n

She added: “We have a chance for [Luis’s] future to be different.”<\/p>\n

The Department of Health and Social Care said in a statement that “it is absolutely right that patients should have access to cost-effective, innovative medicines on the NHS at a price we can afford.<\/p>\n

“Despite being offered in the region of \u00a3500m over five years – the largest ever commitment of its kind in the 70-year history of the NHS – Vertex has refused to accept, putting Orkambi out of reach of patients.<\/p>\n

“We’re aware there may be other avenues open to resolve this issue, but our approach remains urging Vertex to accept NHS England’s generous offer.”<\/p>\n

A spokesperson for NHS England said: “We understand how difficult it must be for families affected by cystic fibrosis.<\/p>\n

“This is why it is so important that as with all companies seeking NHS approval for treatments of this kind, Vertex must fully engage with the NICE [The National Institute for Health and Care Excellence] process.” <\/p>\n

Follow the Victoria Derbyshire programme on <\/i><\/strong>Facebook<\/a> and <\/i><\/strong>Twitter<\/a> – and see more of our stories <\/i><\/strong>here<\/a>. <\/i><\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"

Media captionLuis tells the Victoria Derbyshire programme: \u201cI wish I could just have a normal life” The government must use its powers to break a deadlock in making a life-extending drug available to people with cystic fibrosis, campaigners say. Vertex, which makes Orkambi, has refused a \u00a3500m offer for the drug over five years – Read More<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[],"tags":[],"class_list":["post-235525","post","type-post","status-publish","format-standard","hentry"],"_links":{"self":[{"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/posts\/235525","targetHints":{"allow":["GET"]}}],"collection":[{"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/comments?post=235525"}],"version-history":[{"count":0,"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/posts\/235525\/revisions"}],"wp:attachment":[{"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/media?parent=235525"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/categories?post=235525"},{"taxonomy":"post_tag","embeddable":true,"href":"http:\/\/healthmedicinet.com\/i\/wp-json\/wp\/v2\/tags?post=235525"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}