Cystic Fibrosis Drugs: Orkambi
Jim Shannon Excerpts
Monday 10th June 2019

(1 year ago)

Westminster Hall
Read Full debate
Department of Health and Social Care
Clive Lewis Portrait Clive Lewis (Norwich South) (Lab) - Hansard
10 Jun 2019, 6:10 p.m.

It is a pleasure to serve under your chairmanship, Mr Hanson. I add my thanks to the hon. Member for Sutton and Cheam (Paul Scully) for securing this debate.

I will focus on two key things: asking the Minister a series of questions on potential remedies for this situation, but also using my time to speak about Rachael and Ethan, a mother and child in my constituency who are forced to live with the realities of cystic fibrosis every day, and the adverse impact that the actions of the company Vertex are having on their lives. Ethan is 11 years old. He has a big smile and loves pizza, trampolining and spending time with his friends, but sadly that is where the similarity to other 11-year-olds ends, since every day his life and that of his mum Rachael are dominated by the strain of dealing with cystic fibrosis.

To put this in context, because his lung function has declined to just 54%, in the past four weeks alone Ethan has coped with two separate full-day hospital admissions, 14 days of intravenous antibiotics, which are administered by his mum twice a day at home and take 45 minutes each time, and two sessions with a psychologist to help him to overcome needle phobia brought about by years of blood tests. All that is on top of his regular daily cocktail of medication and a physio session of a minimum of 22 minutes every day. Yet none of those treatments are designed to cure Ethan. They are simply designed to treat his symptoms—symptoms that are expected to get worse the older he gets. They attempt to slow down the irreversible lung damage that will slowly cause him to suffocate. He knows that. In 2017, half of all people who died with cystic fibrosis were under the age of 31, as has already been stated. It is a statistic that his mum Rachael is only too aware of as she spends the majority of her time caring for her son.

We know that Vertex drugs could change the lives of cystic fibrosis sufferers and their families, since they fix the underlying genetic mutations that cause the condition, but, as we have heard today, Vertex is more interested in 45% investor returns than in 20%. That seems to be its priority. Although the chronic underfunding of the NHS is a contributing factor in this story, it is by no means the only reason why a deal has not been reached. The unforgivable actions of Vertex Pharmaceuticals, which admittedly does important work in research and development relating to the treatment of cystic fibrosis, mean that it continues to put patients’ lives at risk as it seeks to extract the highest possible price from our NHS. When Alexander Fleming created penicillin, he had it publicly patented so that it was accessible to all, and it became a revolution in modern medicine. Should we not be legislating for pharmaceutical companies to do something similar and put patients, not profits, at the centre of their development?

I have a number of questions for the Minister. How do we approach this matter systematically, so that we are not back here time and time again, as we have heard? The reality is that we have a socialised—dare I say socialist—healthcare system, which treats people’s health on a collective basis, based not on their ability to pay, but on their need. That is quite revolutionary. However, that healthcare system operates in an international pharmaceutical industry based on rapacious profiteering. How do we square that circle? I suggest the answer lies at the European level, not the US level. Ultimately, the EU’s being one of the biggest healthcare markets in the world gives the European Union immense clout in imposing its will on international pharmaceutical companies; that is one of the reasons I want to stay in it.

My other question is a philosophical one: what is a fair price for years of investment and research? Who determines what is a fair price—our collective democracies, which enable these companies to exist, or a handful of corporate executives whose primary motivation is to maximise profit? Who determines that price? At the moment, I do not think that the balance is correct.

Finally, has the Minister considered the use of compulsory licensing, allowing a UK company or other company to make cheap generic copies? That is allowed under World Trade Organisation rules; the definition of an emergency is up to the host country that needs to use it, and from initial research it is something that could be used and has been used before. I know that is not something she would want to do initially, but ultimately, would she consider it if Vertex refuses to move?

I will leave hon. Members with a quote from Ethan, who had his 11th birthday on 5 June. He said:

“I want to live a long life, because then I get to see some things and do things, so please fund The Triple”.

Jim Shannon Portrait Jim Shannon (Strangford) (DUP) - Hansard
10 Jun 2019, 6:15 p.m.

I congratulate the hon. Member for Sutton and Cheam (Paul Scully) on setting the scene so well for us, and all the right hon. and hon. Members who have made such fantastic contributions on a subject in which we all have a deep interest.

This is an issue that I have spoken on many times in this Chamber and indeed outside it. I have received emails from constituents with photos of their children, begging me to do something to give these precious little ones a better quality of life. If ever we needed to be reminded of the importance of this for the children, as every hon. Member has said, that is such a reminder.

Let us be clear: cystic fibrosis is not only, tragically, a life-limiting disease, but a disease that massively impacts on the quality of life and the life experiences of the sufferers and their families, because the families live the children’s tragedy as well. Cystic fibrosis is one of the UK’s most common life-threatening inherited diseases. It is caused by a defective gene carried by one person in 25, usually without their knowing it. That is more than 2 million people in the UK, and if two carriers have a baby, the child has a one in four chance of having cystic fibrosis.

Around 10,400 people in the UK have cystic fibrosis; that is one in every 2,500 babies born. It affects some 100,000 people in the world. According to the most recent report from the UK Cystic Fibrosis Registry, based on people with CF who were recorded as alive from 2013 to 2017, half of people born with cystic fibrosis in 2017 were expected to live to at least 47, but the 132 people with CF who died in 2017 had a median age of 31. That is a massive difference and we cannot ignore it.

Parents are begging me, begging us, begging this House to ensure that those years are of the best possible quality. After numerous trials, some carried out with Northern Ireland constituents, Orkambi seems to be a drug that does exactly that for many people—enabling the best possible quality of life. My most recent correspondence from Richard Pengelly, the permanent secretary for health in Northern Ireland, outlined clearly that he does not have the power to do what we all need him to do and what he wants to do:

“Let me say that I share your disappointment that the progress in making this drug more widely available has not advanced as we had hoped. At the heart of this matter is the inability of the manufacturer Vertex to come to agreement with the relevant UK Health Technology Assessment bodies.”

Mr Gregory Campbell (East Londonderry) (DUP) - Hansard
10 Jun 2019, 6:18 p.m.

When we have the most senior civil servant in Northern Ireland, along with NHS England and virtually everyone else who has any dealings with the issue, saying, “Look, we need action, we are powerless to move,” does that not throw the ball firmly back into the Government’s court to resolve the matter with the company?

Jim Shannon Portrait 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 (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.

Break in Debate

Seema Kennedy Hansard
10 Jun 2019, 6:45 p.m.

I will talk about the interim measures, but I think the more important thing that we need to grip is having a permanent solution for everybody living with cystic fibrosis.

Throughout the negotiations, which are rightly being led by NHS England, the Government have been crystal clear that Vertex must re-engage with the NICE process. I know that hon. Members have questions about that process, and I will try to address some of the points that have been raised. The hon. Member for York Central (Rachael Maskell) raised more detailed points for me to consider; if I do not address them, I will write to her with more detail, but there are some points about the NICE process that I will address later in my remarks.

Jim Shannon Portrait Jim Shannon - Hansard
10 Jun 2019, 6:46 p.m.

Would it be possible for the Minister to give her Department’s and her own opinion on the buyers’ group that is seeking to buy similar drugs from Argentina for cystic fibrosis? Have the Government given that possibility any consideration?

Seema Kennedy Hansard
10 Jun 2019, 6:47 p.m.

I shall turn later in my speech to that specific avenue that some sufferers have gone down.