Cystic Fibrosis Drugs: Orkambi

Marion Fellows Excerpts

Monday 10th June 2019 Westminster Hall

[Marion Fellows (Motherwell and Wishaw) (SNP)]

It is a pleasure to serve under your chairmanship, Mr Hanson. I thank the hon. Member for Sutton and Cheam (Paul Scully) for setting out in his opening speech facts that a lot of us did not know and facts that some of us did. It was a very useful start to this wide-ranging and well attended debate. I will just ask this question, though: how many times do we have to debate this very serious subject?

Again, I have to declare a personal interest. I am a cystic fibrosis carrier. My late husband was, too. My children are carriers, and my granddaughter, Saoirse Grace, has cystic fibrosis—mutations F508del and D1152H. I do not understand to any great degree what the last part of that actually means, but I do know that she is not the worst sufferer of cystic fibrosis. She has the best kind, if you like. She is pancreatic sufficient, and for that we are always grateful. Saoirse will not directly benefit from Orkambi, but along with 90% of people with cystic fibrosis in the UK, she will benefit from the triple therapy coming down the line. Those therapies will deliver unprecedented improvements in acute lung health and reductions in pulmonary exacerbations—a key driver of decline.

Approximately 900 people in Scotland live with cystic fibrosis. NHS Scotland estimates that one in 24 Scots has a CFTR mutation, which, if carried by both parents, would lead to a child being born with cystic fibrosis. England has the highest prevalence of cystic fibrosis in the world, with 1% of the world’s population but 12% of the world’s CF population.

There should be no postcode lottery for treatment. The UK Government should follow the Scottish Government’s lead on cystic fibrosis treatment. For people in England to receive Orkambi, NHS England would need to pay Vertex £500 million over five years and £l billion over the next 10 years. However, in a debate on CF and Orkambi last year, it was stated that the costs of Orkambi can be offset by reduced hospital admissions, and other benefits should also offset the cost, as has been stated in the Chamber today. Vertex has stated that its offer to NHS England for the provision of its CF drugs represents the lowest price for Vertex’s portfolio of CF drugs in any country in the world. I point out that I am not here to make a case for Vertex; I am here to make a case for people with CF who need this drug.

The Scottish Government recently announced that NHS Scotland and Vertex Pharmaceuticals had reached a deal to provide interim access to Orkambi and Symkevi, through a system known as peer approved clinical system tier 2, while the Scottish Medicines Consortium reviews the relevant evidence for Orkambi. PACS tier 2, introduced by the Scottish Government in June 2018, involves an individual application for patient access to drugs not recommended by the SMC and not routinely available on the NHS.

The Scottish National party believes that the UK Government need to do more to facilitate interim access to Orkambi and other drugs to treat cystic fibrosis and to get NICE to re-evaluate making the drug more widely available for people living with the illness. In England, NICE said Orkambi was too expensive for the NHS in 2016, and since then Vertex and NICE have been unable to reach an agreement that will bring these drugs to patients. The UK Government are showing a lack of flexibility by only urging Vertex Pharmaceuticals to fully re-engage with the NICE appraisal process and to accept the offer that the NHS made in July 2018. Vertex has said that the methods used by the Scottish Government to obtain the drugs reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases such as cystic fibrosis, and that it is hopeful that, through that process, all eligible patients in Scotland could have access to its medicines soon. If Orkambi and Symkevi are accepted by the SMC, which makes decisions independently of the Scottish Government, eligible patients in Scotland could have access to these precision CF medicines in 2019.

In the interim, Vertex and the Scottish Government have agreed a confidential discount that would be applied to approved PACS tier 2 applications. The Scottish Government also asked Vertex to provide access to the medicines at a discounted price to the list price while they finished the contract negotiations, and Vertex said, “Of course.” Vertex stated that it would be willing to do exactly the same thing in England.

There is agreement across the Chamber that Orkambi, Symkevi and Vertex’s triple therapy should be available UK-wide. I have no desire to get into the argument between NICE and Vertex in England. I want England to do what Scotland has done. I want children across the UK to get the benefits of these drugs. As has been said, this problem will exist whatever view we hold of Government at the UK level. However, political will must be exercised in the process of getting those with cystic fibrosis the drugs that will improve their lives and futures.

Finally—I will not take too long, Mr Hanson—I give my heartfelt thanks to all the parents and guardians of CF sufferers, and to those affected by cystic fibrosis, for their continuous campaigning and awareness-raising. It is awful that they have to keep doing that. I ask the Minister, please do not let their work be in vain.