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Cystic Fibrosis Drugs: Orkambi
Exerpts for David Hanson
Monday 10 June 2019

(5 months, 4 weeks ago)

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Westminster Hall
Department of Health and Social Care
Jim Shannon - Hansard
10 Jun 2019, 6:18 p.m.

I thank my hon. Friend for those words. This is not an easy subject for the Minister to respond to, but it is one that has captured the interest of us all on behalf of our constituents, and we need the Government to grasp that and move it forward to the next place. We look for that.

If the Republic of Ireland is able to come to some arrangement with Vertex, if the Scottish Parliament is able to do similar and if, according to the background information, it is possible to go to Argentina and buy a year’s course of drugs for one patient at £23,000, compared with £104,000 for a year’s supply here, that tells me that something can be done if we had the willpower to do it, as my hon. Friend the Member for East Londonderry (Mr Campbell) has said. We can look around at our UK neighbours and look toward Scotland, whose Government has reached an agreement with Vertex.

The permanent secretary went on to say:

“In the absence of this positive NICE determination, the Health and Social Care Board…may take into account guidance produced by other appropriate HTA”—

health technology assessment—

“bodies based in other UK countries such as the Scottish Medicines Consortium…when making decisions about access to new drugs.”

I say to the Minister that I have made a comment about the Republic of Ireland, but I also make a comment about Scotland, because I think that the process enables us to use what Scotland has done as an example for us elsewhere.

The permanent secretary continued:

“The Department is aware that Vertex have re-applied to the SMC for consideration of approval for Orkambi, and that in the meantime there is currently limited access to the drug in Scotland via their PACS”—

peer approved clinical system—

“Tier 2 scheme, which is broadly analogous to our Individual Funding Request Process.

If Orkambi is approved by the SMC, details of the funding models in place, which are currently bound by commercial confidentiality, will be shared with the other UK countries. This will allow for us to have full access to the evidence and costs associated with this therapy and will inform any further decisions on access.”

The hon. Member for Colne Valley (Thelma Walker) referred to the destruction of some medications. Whatever the reason for that was—whether they had run out of time or whatever—I think it is disgraceful that people have destroyed some medicines rather than letting them be used by the general public, by those who need them. If that is not unacceptable in this day and age, I do not know what is. It is absolutely disgraceful; it really annoys me.

It is simply terrible that we are in a position where our hand are tied. I say again to the Government: look to your Scottish counterparts. I firmly believe that we can and must do more from this place and that that must start with acknowledging that the NICE guidelines do not currently take into account the differences, when it comes to pricing, between treatments for rare diseases and a new antibiotic strain. We need a new form of assessment for rare diseases and I would like to see that taking place as soon as possible to ensure that the mummy of my two-year-old constituent, who is asking me for this drug in order to give her child as normal a life as possible, can look forward to securing the best for her child. That is what every Member has said here today on behalf of their constituents.

Again according to the background information that I have, in May 2019 the Government said in response to a parliamentary question that discussions between Vertex, NHS England and NICE were ongoing. You know something? They have been ongoing for more than a year. Let’s get them sorted. Time is passing fast. I am joining colleagues in asking the Department to make the administrative changes necessary to end the Orkambi stalemate with NICE and to put in place a body designed specifically to address rare disease patients and their needs. We acknowledge that NICE does a tremendous job in ensuring that safe, cost-effective medicines are available on prescription, but we need a different set-up for those whose illnesses are very different and for the sake of my constituents and those represented by other MPs who have spoken today. I am asking that those decisions be taken and the changes made to enable Richard Pengelly, the permanent secretary at the Department of Health in Northern Ireland, to do what he knows he needs to do and allow the prescription of Orkambi to those whose lives would be radically altered and enhanced by it. It would give them life-changing opportunities. As others have said, give those children a chance.

David Hanson Portrait David Hanson (in the Chair) - Hansard

The previous Chair set a time limit on speeches of six minutes, which has been successful, in that all right hon. and hon. Members have been able to take part in the debate, but it leaves us with a maximum of one hour and 10 minutes for the Front-Bench speakers to respond. Front-Bench speakers, who have more than the normal 10 minutes, can be more flexible and take interventions should they so wish.

The other point I wish to make is that because the winding-up speeches have started early, some Members who have spoken in the debate are not present for these speeches and they should be. If you are watching on television, please return to the Chamber now. This is an important debate; you have contributed to it and you should be present for the Front-Bench responses.

Marion Fellows Portrait Marion Fellows (Motherwell and Wishaw) (SNP) - Hansard
10 Jun 2019, 6:23 p.m.

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.