Spinal Muscular Atrophy

Kerry McCarthy Excerpts

Thursday 25th July 2019 Commons Chamber

[Kerry McCarthy (Bristol East) (Lab)]

It is just so cruel. There was a party to celebrate the fact that my 18-year-old constituent Jake Ogborne would have access to Spinraza. There was no mention of eligibility criteria, but suddenly he was told that he would not be eligible because he had not been able to walk in the past 12 months. He lost his ability to walk about 18 months or two years ago. I raised this in Prime Minister’s questions. I got a response that basically told me what we already knew and just set out the rules on this. It is immensely cruel for an 18-year-old to be faced with a decision like that.

[Sir John Hayes]

Yes. The condition, as the hon. Lady suggests, is often degenerative. In cases such as the one she describes, which, as I mentioned, is not the first time she has raised it, a young person who would normally develop as we all did could be left with arrested development or, even worse, declining capabilities. Indeed, that has happened to my constituent too.

The hon. Lady will know that, following strong advocacy by families of SMA patients, Muscular Dystrophy UK, TreatSMA, Spinal Muscular Atrophy UK and many clinicians, NICE and NHS England made amendments to the managed access agreement. While amendments are far from unwelcome, the disappointing truth is that the new criteria will still exclude some SMA patients desperate for treatment in the way she and others have articulated.

The intensely difficult battle fought by SMA patients has highlighted deeper flaws in the system. Families report feeling that they have been pitted one against another as advocacy groups are forced to decide whether to push for wider accessibility, and as a consequence risk delaying treatment for those eligible, or, alternatively, to take what is on offer and exclude a minority of the SMA family.

Life can be intensely difficult. All our bodies are complicated and vulnerable, intricate and fragile. We are regularly reminded, are we not, that they can go wrong in a multitude of hard-to-understand ways. I have argued many times in this House that a society should be gauged by how it cares for, protects and promotes the interests of its most vulnerable members.

Mental Health Act 1983

Kerry McCarthy Excerpts

Thursday 25th July 2019 Westminster Hall

[Kerry McCarthy (Bristol East) (Lab)]

As always, it is a pleasure to see you in the Chair, Ms Buck. I congratulate my hon. Friend the Member for Bermondsey and Old Southwark (Neil Coyle) on an incredibly powerful speech that was very detailed and knowledgeable about this issue. Obviously, my hon. Friend was speaking from personal experience, which was very brave of him.

It is clear from the findings of the independent review of the Mental Health Act, published last year, that the current legislation needs significant reform. There has been a 47% increase in detentions under the Act over the past decade, while the proportion of people living with a serious mental illness remains relatively unchanged, so clearly there is some imbalance there; it cannot be right that the number of detentions is going up. I echo the points that have been made about the link between this issue and the cuts to, and failings of, other services in the community that might be able to help at an earlier stage. Perhaps that is one reason why people are ending up in the most serious of circumstances; that is, being detained.

Being detained in a secure mental health unit is obviously going to be an intimidating and frightening experience for anyone. However, it is particularly intimidating for people with learning disabilities and autism, especially young people with those conditions, so I will focus on those groups in my speech. I raised this issue at Health questions on Tuesday, and the Minister—not the hon. Member for Thurrock (Jackie Doyle-Price), but another Minister from the Department of Health and Social Care—gave a very thoughtful response.

One of the things that the Minister said was that learning disabilities, autism or other special needs are often not diagnosed until a child is detained in a mental health unit, which again shows the failings. Child and adolescent mental health services are incredibly under-resourced, and schools are really stretched when it comes to education, health and care plans and working with children who show signs of mental health problems. It is so important that we put proper resources in, so that we can give those children the help they need as early as possible. I am sure that the Minister responding to today’s debate, the hon. Member for Thurrock, agrees with that.

Under the Act, people with learning disabilities and autism can be sectioned with no mental health diagnosis if they are displaying challenging behaviour. The independent review found that the Mental Health Act was

“being used inappropriately for people with a learning disability, autism or both, to deal with a crisis that has arisen because of a lack of good community care or placements…It is particularly intimidating for a person with autism, learning disability or both to be removed from a place they are familiar with or from people they know, even if at the time there seemed little alternative…Instead of improving their mental health, the environment (including relationships with staff) has made them worse, not better.”

One of the common characteristics of autism, for example, is being wedded to routine, and being uncomfortable with unfamiliar circumstances and stimuli that those people would perhaps not get in a more protective environment. That can be particularly challenging.

The Equality and Human Rights Commission echoes such concerns, stating:

“The Commission is concerned about the lack of suitable provision for people with learning disabilities and autism, who may be in restrictive institutional settings because there is insufficient community-based support. The Government failed to meet its minimum target to reduce the number of people with learning disabilities and autism detained in inpatient settings, and at the end of March 2019 there were still 2,260 children and adults living in these institutions. On average, they had spent almost 5 and a half years detained away from home. There were more than 2,600 restrictive interventions recorded against inpatients with learning disabilities and autism in a single month, including physical, chemical and mechanical restraint, seclusion and segregation. Of these, 875 interventions were used against children.”

Most of those people should never have had to go into an in-patient unit, let alone be subject to those types of restrictive intervention. They are ending up in such units because of challenging behaviour due to unmet needs in the community. Those needs, as I have said, should be met with vital investment in community mental health services.

From my experience of people who have come to my surgeries, other casework that we have been dealing with and what I have picked up from visiting institutions, it seems that mental health services suit certain people because of the pathways developed in the NHS, but others do not fit into that category. For example, a child with learning disabilities was rejected for anxiety counselling, and they were later diagnosed with autism. To do the standard CAMHS anxiety counselling, someone has to have a certain ability to rationalise their actions and think about how they operate. That cognitive behavioural therapy-type approach was not suitable for that child, but that meant that nothing was available, because that was the pathway for young girls suffering from anxiety.

I had another case where a constituent was told that she was too traumatised by a recent experience to go through the counselling on offer through her GP. She clearly was not in a serious enough condition to be sectioned, however, so it seemed like she was caught between two things. She clearly needed help, but she was deemed to be too serious a case to get the help that was available. We need to look at the people, rather than just trying to fit them into categories and, when they do not fit into categories, rejecting them from the system, almost as though we are saying, “Come back when you are a lot worse, and then perhaps there is something we can do for you.” That has to be the wrong approach.

Going back to children in particular, the UN Committee on the Rights of the Child has expressed specific concerns about the adequacy of therapeutic community-based services for children. Because of that lack of community provision, children are being detained when they should not be. According to NHS Digital, at least 1,777 people aged 17 or under were detained under the Mental Health Act in 2017-18. Of those, more than a thousand were admitted to a non-local bed, in many cases more than 100 miles from home. Some children and young people under care of Bristol, North Somerset and South Gloucestershire CCG had to travel up to 243 miles away from home for mental health care. That cannot be in the child’s best interests and will add to the sense of isolation and anxiety, and it can also prevent their families from being properly involved in their care.

A recent Children Commissioner’s report found that many parents feel they are being shut out of decisions about their children’s care and are not always informed about incidents involving their children. Serious incidents had happened in hospital without families even being told. Some families had even faced gagging orders where they had been prevented from speaking out about their children’s care. The independent review calls for better safeguards to protect children and young people, ensuring that they are treated in hospital only when necessary and that their rights as a child are clearly set out. There is a need to establish clear tests as to whether children and young people are able to consent to hospital admission or treatment and whether they should be involved in decisions made about them. Clear roles need to be established for parents in care planning and treatment.

The review also states that there needs to be more advocacy available for children and young people. That is particularly important where conflicts arise between the clinical team about how to meet the best interests of the young person, including where contact between young people and their parents is seriously restricted—in some cases, the mental health crisis might be the result of chaotic family circumstances at home or ill treatment—or where the wishes of the young person conflict with the wishes of the adult.

Every in-patient child or young person should have a personalised care plan that includes their views and wishes and access to an advocate. They should be regarded as a child in need under the Children Act 1989 so that parents can ask for help from their local authority.

As the British Medical Association lead for mental health, Dr Andrew Molodynski has said:

“Warehousing unwell people in locked wards far from home goes against the very nature of mental health rehab—to help them reintegrate back into society.”

Unnecessary detention of vulnerable patients is not only unhelpful in terms of their recovery, but an ineffective use of taxpayers’ money. Too much money is being given to private firms to look after people with serious mental health problems in units often hundreds of miles away from their homes due to a shortage of NHS beds. That figure was £158 million in 2016-17 and £181 million in 2017-18—an increase of £23 million.

Last year, an extensive study of mental health rehabilitation by the Care Quality Commission found that stays in private beds cost twice as much as in the NHS because they tend to last twice as long. It found that the annual cost of rehab was £535 million and that private beds were on average 30 miles away from patients’ homes, whereas in the NHS they are only nine miles away.

One of the cases that I have been dealing with recently is that of a child who is in a mental health unit 150 miles away from home, but the unit closest to her home is in special measures and not accepting new patients. However, the unit she is in is ranked as outstanding and she seems to be making good progress. Clearly, when parents are faced with such a choice, they want their child to go to the best unit possible, but that can put huge pressures on a family, in particular when they have other children, as well as jobs and lives. They might also not have the money to decamp to somewhere so they can visit their distressed child each day. There is a real lack of support in the system for families placed in such circumstances, with their whole lives disrupted by trying to be in two places at once.

Another problem is that the quality of care in hospitals can be highly variable. The Whorlton Hall case demonstrated just how awful situations can be, and how badly people can be treated in some places. In a recent investigation into children with learning disabilities or autism living in mental health hospitals, some families told the Children’s Commissioner about the excellent support that their children had received—it is important to put on the record that there is good care out there—but others had shocking stories to tell. For example, one family said that their son had not been washed for six months while in hospital. Basic care needs were not being met.

Despite improvements in recent years and the drive towards parity of esteem between physical and mental health services, it has not been realised. The aspiration is noble and shared by both parties, but we need to invest far more in our mental health services for people to get the support they need. I welcome the fact that the need for better community services was highlighted in the recent NHS long-term plan, and I am pleased that the Government have committed more funding to severe mental illness and to greater use of alternative mental health crisis provision, such as crisis cafés. However, an overhaul of the Mental Health Act is clearly needed if that investment is to be used in the best interests of patients by ensuring that their dignity and rights are protected and that they are treated in hospital only as a last resort.

The previous Prime Minister committed to new legislation to bring the Mental Health Act into line with a more modern understanding of how best to treat mental health. She confirmed that a White Paper would be published before the end of the year. The Minister is probably wondering whether she will be in post by the end of today, but even if she is moved on to greater things, I hope she asks the new Prime Minister to give that the same priority as the previous Prime Minister did.

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[Jackie Doyle-Price]

That is an excellent point, and it plays into a general prejudice that people who are in detention are just an inconvenience to be managed. In the context of Sir Simon’s review, the whole ethos that any reform we make should be about empowering patients brings with it obligations to challenge other aspects of the system, not just the care providers. The hon. Lady is right to say that I need to take that up with the Policing Minister, which I will do as part of rolling out our preparations for the White Paper. I understand the hon. Lady’s lack of confidence when I say that the White Paper can be expected before the end of the year, but that is certainly my ambition, notwithstanding the fact that I know she has been waiting rather a long time for another paper that she was promised.

I can reassure the hon. Lady on the extent of the work that Sir Simon has done and the engagement we have had, especially with service users. Rather like the hon. Member for Bermondsey and Old Southwark, those service users shared with us their personal experiences, often reliving significant distress. Given that they have participated and that we have raised their expectations, we would, to be frank, be letting them down if we did not address that. I do not think that would be in any way forgivable, so, as long as it is on my watch, we will be pushing ahead.

As the hon. Member for Bermondsey and Old Southwark said, we are dealing with legislation passed in 1983, so although this appears to be a once-in-a-generation opportunity to reform this legislation, I hope that that is not the case. The situation is probably more symptomatic of the fact that we have not given this matter as much attention as it deserves, but clearly that has changed. The world has changed in terms of how we debate mental health, and that is welcome. Chiming in with the discussion we have had today, I am keen that we take this matter forward with consensus.

I pay tribute to the leadership that Sir Simon has shown in this review. As well as not ducking the controversial aspects of examining the legislation, he has engaged in dialogue and dealt with them in such a way that it is accepted that Members, peers, service users and professionals need to consider them. I am incredibly grateful for what he has done.

We talk more about mental ill health now, but when it comes to severe mental illness—the hon. Member for Bermondsey and Old Southwark spoke about schizophrenia —that is something that reveals incredible prejudice in people. As we have heard, if someone is able to manage their condition, they can live a full and independent life, but the key is being able to manage the condition with the appropriate support. We still have a lot to do in educating the public and society about the real impact of severe mental illness.

There are so many issues, but I will try to address them all in the time I have. The hon. Member for Bristol East (Kerry McCarthy) asked about out-of-area placements for people with autism and learning disabilities. I must say that this is something that really bothers me. Far too many people remain in institutional care and in out-of-area placements, and nowhere is that more true than in the field of learning disabilities and autism.

It is interesting, once we dig under the issue, to see that we have been very successful in getting people with learning disabilities out of in-patient care and into the community. However, that has been matched by a bigger increase in the number of people with autism finding their way into in-patient care. That tells me—I do not think this will be a surprise to anyone in the Chamber—that we are not doing enough to diagnose autism early enough, and as a consequence we are not equipping people with the skills to be able to live independently.

The ultimate result is that we end up putting people in in-patient care. Quite often, those people are forgotten about and it becomes very expensive to keep them there, so not only are we failing people by not having services for them early enough, but we are adding significant cost to the taxpayer and, frankly, doing harm, because the longer those people stay in in-patient care, the more their ability to live independently diminishes. As far as I am concerned, that is a major failing that we need to address.

[Kerry McCarthy]

I thank the Minister for that. There are two aspects that I have come across. First, it is often when a child reaches their teenage years, which we know are difficult for any child with all the turmoil that is going on physically and mentally, that the anxiety takes over and they eventually go into crisis, and things that perhaps should have been picked up sooner are only picked up then. Secondly, that seems to be particularly true for girls. We know that it is more difficult to diagnose girls with autism than to diagnose boys. I was told yesterday, although I do not know whether this figure is true, that one in four girls in mental health units with anorexia is diagnosed with autism. There is a lot of work to be done in the early years, before they reach that crisis point in their teens.

[Jackie Doyle-Price]

I agree with every word that the hon. Lady says. One of our expectations of the new mental health support teams that we are rolling out into schools is that they will be able to work with schools and to spot people who might be in trouble. She is right that, with girls particularly, autism is under-diagnosed, and by the time the challenges start to have an impact on mental health, it is much more difficult to give people appropriate support. Early diagnosis is key.

I am pleased that we are now setting up a unit within the Department of Health and Social Care to look at neurodiverse conditions and what more we can do to improve service provision for them. I am also pleased that NHS England shares my concern about this and that we can expect more work on it, but there remains a lot to do and I do not shirk from admitting that.

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[Jackie Doyle-Price]

I cannot commit to delivering that, but I can commit to looking at it. I hope that is good enough for the hon. Gentleman.

[Kerry McCarthy]

In the case that I was talking about, the problem was not just that the treatment was so specialised that it was that far away, but that that was when the bed became available. It is a bit of a lottery. The one closest to home was not taking in new patients because it was in special measures. There would have been other places closer. If the person does not accept a bed when one becomes available, they may have to look after a distressed child for an awful lot longer.

[Jackie Doyle-Price]

This comes back to housing. One challenge is getting access to a bed, and another is when the person comes to leave, because we need to discharge people into safe living environments. Are there enough supported housing solutions? No, not always, so the bed remains full. I am having conversations with colleagues in local government to see what more we can do to deliver more supported housing so we can get the pathway going. We could fix it by making more beds available, but that is not really the answer. I am concerned that the longer we leave people in in-patient care, the more harm we do. We have to get that movement through the system. Hopefully, if we do that, people will be less likely to grab the first bed because they can be confident that more will become available more frequently. That is where we need to get to. I appreciate that right now it feels desperate.

There is an issue with quality. The Care Quality Commission has a challenge in deciding whether more harm will be done by taking enforcement action on a place rather than working with it to improve. We see that writ large in TV programmes such as “Panorama”. There is a massive disparity in the quality of care. I challenge the CQC to be a lot more aggressive when we see poor standards of care.

The hon. Member for Bristol East mentioned private providers. People have heard of The Priory. They hear that celebrities go there and they think it is a centre of excellence. The truth is that the care there is less than optimal, but someone with a loved one who needs hospital treatment will not know that. We need the CQC to have a lot more teeth in terms of improving what comes out of its inspections. The system generally needs to support it in doing that.

Oral Answers to Questions

Kerry McCarthy Excerpts

Tuesday 23rd July 2019 Commons Chamber

[Stephen Hammond]

I am happy to confirm that.

[Kerry McCarthy (Bristol East) (Lab)]

15. What additional funding he plans to allocate to the NHS to reduce the number of people with autism and learning disabilities held in in-patient settings.

[The Minister for Care (Caroline Dinenage)]

The NHS long-term plan commits to an extra £4.5 billion a year for primary and community health services by 2023-24. Local areas will be expected to use part of this investment to develop specialist services and community crisis care to reduce avoidable admissions and lengths of stay for autistic people and those with learning disabilities.

[Kerry McCarthy]

I do not know whether the Minister has seen the report from the Children’s Commissioner, but it says that the number of children with autism or learning difficulties in mental health hospitals has doubled in recent years. Many are very far from home. Their parents are not consulted properly on their care, and they are in for much longer than they need to be. What proportion of the money that she mentions will go towards trying to ensure that children can have support in their home, so that they are not in this situation?

[Caroline Dinenage]

The hon. Lady has hit on a really important point. The truth is that a lot of children who end up in an in-patient setting are not diagnosed with autism or a learning disability until they are there; they normally come in on a mental health diagnosis. There are a few points to make here. One is on making sure that early intervention is there as early as when a child is in school. That is why the Government have made a commitment to having mental health support available from a very early age, in schools. This is also about making sure that families do not get to crisis point, and that the investment is there in the community, so that children get the support that they need at every possible step.

Oral Answers to Questions

Kerry McCarthy Excerpts

Tuesday 18th June 2019 Commons Chamber

[Jackie Doyle-Price]

I am pleased that my hon. Friend has declared his interest in this matter. He is right that mental wellbeing is about not only clinical interventions but very much the kind of things that he describes—wider social prescribing. We cannot overstate the role of the third sector in giving wraparound support to people going through periods of mental ill health. I am giving clinical commissioning groups the clear message that they need to look at what else they commission in this space, alongside clinical interventions.

[Kerry McCarthy (Bristol East) (Lab)]

In Manchester on Saturday, people were giving away free “Unknown Pleasures” t-shirts, partly to mark the 40th anniversary of one of the greatest albums ever made. But, as anyone who knows the history of Joy Division will know, there is also the important related issue of male suicide and people were being encouraged to donate to charities, particularly those that work with young men at risk of suicide. I was sent one of the t-shirts, Mr Speaker, but I thought you might rule it out of order if I wore it. These charities obviously do great work, but they are trying to fill real gaps in the system. How can we ensure, when we consider long-term health plans and long-term mental health services, that there are not gaps that people fall between?

[Jackie Doyle-Price]

The hon. Lady articulates the issue extremely well. The purpose of local suicide prevention plans is very much to make sure that we have a joined-up approach to combating male suicide and to identify exactly where the gaps in the services are. The £600,000 that we announced yesterday for the sector-led improvement package is to enable local authorities to share expertise and to make sure that, holistically, they provide the leadership to make sure that the gaps are plugged. I am grateful for the hon. Lady’s interest in this matter.

Cystic Fibrosis Drugs: Orkambi

Kerry McCarthy Excerpts

Monday 10th June 2019 Westminster Hall

[Paul Scully]

The hon. Lady has hit on a really crucial point. The underlying thing that I took away from my meeting with the people living with CF this morning was mental health, which ran through all their situations.

I heard from Oli Rayner, who talked about the fact that he is 43 and has dedicated his whole life to staying alive; he had effectively been told that he would not make 10 years. He was then told that he would not make 20, then 30. This is a guy who has now got cystic fibrosis-related diabetes and a number of other conditions. He has had a lung transplant, and he had Orkambi to get him to that stage. The fact is that his lungs are now doing what he wants them to do, without his having to think about it. We can imagine the mental issues that he had before.

Jessica Jones told me that people with CF are very good at living. Yvonne Hughes said she felt broken. One lady, Carly Beale, told me that she had been on the original Orkambi trial. The NHS had not prepared her for when Orkambi stopped at the end of the trial. She had suddenly improved and started to get her life back—perhaps a life that she had not had in the first place—and she was not ready to have it taken away from her. She said that it is almost worse that this drug exists but she cannot get access to it. She said, “I’d rather it didn’t exist than have it dangled in front of me in expectation.”

[Kerry McCarthy (Bristol East) (Lab)]

I am really pleased that there is now cross-party working on this issue. Life expectancy is a very sensitive issue for cystic fibrosis patients and their parents. I am aware that Conservative MPs have been sending round a letter that points out—as if the letter’s recipients ought to be grateful—that life expectancy for CF patients has now increased to 40, although I think it is more like 31. It seems a little insensitive, and I hope that MPs present would report that back.

[Paul Scully]

It is an unfortunate manner of phrasing. There is no doubt about the fact that median life expectancy has increased. For someone born now, the projection is that they are likely to have a median life expectancy of 47. It is clearly an improvement, and hopefully these drugs will carry on extending that. It is a matter of wording, and I do not think any offence was intended.

--- Later in debate ---

[Kerry McCarthy (Bristol East) (Lab)]

Thank you, Mr Bailey; it is a pleasure to see you in the Chair. I thank the hon. Member for Sutton and Cheam (Paul Scully) for introducing the debate on behalf of the Petitions Committee.

As we have heard, Orkambi is a life-changing treatment that stands to benefit 4,000 cystic fibrosis patients in the UK—40% of the total number of people with the condition—and which was licensed almost four years ago. In some ways, the debate has almost moved beyond Orkambi, because other drugs now being developed would help an even greater proportion of patients. Vertex’s ongoing refusal to offer a price for Orkambi that the NHS can afford means that those patients are still unable to benefit from the treatment.

I will point out—this is important to what I will say next—that in 2017 alone, Vertex earned £2.5 billion from the sale of Orkambi, while its chief executive was paid more than £17 million. Pharmaceutical companies such as Vertex play an important role in the development of innovative medicines and money goes into that, so I entirely accept that they should make a profit as a result, but to pursue excessive reward at the cost of patients’ lives is, I believe, morally bankrupt.

I will speak about alternative approaches. A few weeks ago, I had the opportunity to meet an Argentine drug company that has come up with a generic alternative to Orkambi. I also met two parents, one of whom was fortunate enough to be in a position to be able to spend a six-figure sum on Orkambi for his young son, who was, he said, doing well on it. He was clearly in a very privileged position to be able to do that. The other father was not in that position and was looking at the buyers’ club approach that has been mentioned and that featured on “Newsnight” on Tuesday. He said that if he had to sell his house to afford to buy the drugs at the reduced price—around the £18,000-mark—he would do so, because his son is far more important to him than the place he lives.

I was introduced to the Argentine drugs company by a Bristol-based international organisation called Just Treatment. With a range of experts in the field, Just Treatment has made contact with the generic manufacturer of the drugs, which can supply a safe and effective version of Orkambi at a cost for individual CF patients of about £23,000 per patient per year—just 30% of what Vertex charges. If there were a buyers’ club, the price could come down.

The term “buyers’ club” comes from the Hollywood film, “Dallas Buyers Club”, which gave a fictionalised account of the buyers’ clubs that popped up in the ’80s and ’90s because of the lack of access to HIV treatment. The clubs enable patients to understand their right to procure more affordable generic versions of drugs that they would otherwise struggle to access.

In Argentina, a small number of people have CF, and only a couple of hundred would benefit from Orkambi, of whom 120 take the generic alternative and seem to be doing well. The longest that anyone has taken it is 18 months, so although it is early days, treatments seem to be going well. Initial discussions with the generic manufacturer suggest that once 500 patients sign up to receive the drug, the cost would drop to just 20% of that demanded by Vertex.

Clearly, whether it costs £18,000 or £23,000, the drug is still not affordable for most people, which is why I would argue that although that is a step forward for some, the Government need to step in and help patients. The most effective way they can do that is by pursuing a Crown use licence for Orkambi. A few months ago in the House, the hon. Member for North Herefordshire (Bill Wiggin) mentioned that possibility in the context of access to medicines for CF patients. In the legal provisions set out in section 55(1) of the Patents Act 1977, the Government have the power to suspend Vertex’s patent on Orkambi and overturn its monopoly. One of the Government’s primary objections to that route has been the concern that it could take years to develop a generic product, but as I have said, a generic product is now on the market, is being tested and is available for patients in the UK, although only three boxes can be provided at a time and it is still unaffordable at the price that I mentioned.

The Government should also explore the funding of a large-scale clinical trial, which would enable the NHS to use research exemptions in intellectual property rights law and allow it to use the generic version of Orkambi. That approach has been used before, with the HIV prevention medication PrEP, or pre-exposure prophylaxis, and ensured that tens of thousands of patients were able to access that drug while its expensive branded counterpart was still under patent—I would be particularly interested in Minister’s response to that. A trial could be set up in anticipation of a Crown use licence, providing data necessary to support regulatory approval of the generic version of Orkambi.

If the Government are unwilling to pursue either of those routes—I cannot see why they should be reluctant to do so—there is another alternative that goes down the buyers’ club route: providing personalised health budget payments to help patients meet the cost of the generic drug. People are absolutely fed up of the deadlock between Vertex and the NHS, so I urge the Government to look at those more imaginative solutions.

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[Mrs Hodgson]

I agree with my hon. Friend. I hope that the Minister will have some ideas about how this drug company can be held to account and not be allowed to continue in this way. I hope the Minister agrees that the situation should never have been allowed to get to this stage.

Lucaftor has the same active ingredients as Orkambi, and the Argentinian pharmaceutical company Gador is offering a price of £23,000 per patient per year, which drops to £18,000 if patients and their families can get together a group of more than 500 patients to purchase Lucaftor as a collective. That is significantly lower than the £104,000 Vertex wants for Orkambi. I say “want” deliberately—it is not the cost, but what Vertex wants. Of course, for many patients in the UK, Lucaftor will still be way too expensive to access, so it is not a feasible alternative at all. That is why NHS England and Vertex need to come to a conclusion that puts cystic fibrosis patients first, and ensures that they have access to the life-saving drugs they need and deserve.

[Kerry McCarthy]

I thank my hon. Friend for all her support on this issue. I agree that the issue with the Argentinian solution is that in a buyers’ club where people have to pay privately, the drugs will still be out of reach for many people. However, the fact that Gador is offering this drug for so much less than Vertex is charging for a similar product means that the NHS could, if it decided to trial the drug, buy it for 4,000 patients who would benefit from Orkambi. Therefore, no one would have to pay for it privately. The NHS could fund it, but at much less than Vertex is asking for. I ask the Minister: why is that not the solution?

[Mrs Hodgson]

I was going to come on to that, but if a point is worth making once, it is worth making twice. I will make it to the Minister as well, so she will have plenty of time to think about it.

As we all agree, patients and their families should not be put in the position—as some are—of having to pay thousands of pounds for their treatment. Family income should not determine who lives and who dies. That is why the NHS was founded—so that all could have access to the same excellent treatment, regardless of means. That was true 70 years ago when the NHS was formed, and it is still true today.

As the hon. Member for Sutton and Cheam pointed out, our NHS is there for us all and should not be held to ransom by a pharmaceutical company, but neither should access be denied because of unfit processes and systems in the NHS. Over the years, as a shadow public health Minister, I have met many patient groups, including those with cystic fibrosis, who are missing out on life-changing medicines because their condition is not rare enough and is therefore not deemed by NICE to be cost-effective. We need an appraisal process that is fit for purpose and that will capture rare diseases such as cystic fibrosis effectively.

Without drugs such as Orkambi, patients and their families are being harmed physically and psychologically. Every day without the drugs that patients need makes their condition worse and threatens their lives. What steps will the Minister take to ensure that patients with rare diseases have access to the medicines that they need and deserve? It is about access not just to Orkambi, but to other precision medications such as Symkevi and the next generation of cystic fibrosis drugs that could help patients who are suffering.

Vertex recently announced the headline results for its fourth cystic fibrosis medicine, a triple combination therapy that could radically transform the lives of nine in 10 people who live with cystic fibrosis in the UK, delivering unprecedented improvements in acute lung health. That is amazing news, but patients fear that they will never be able to access this ground-breaking drug. I urge Vertex to put patients first and consider the real-life impact of this cost dispute on patients and their families.

Vertex and NHS England must come to an agreement urgently, because patients have already waited far too long. If an arrangement cannot be made soon, will the Minister personally step in and pursue the alternatives that my hon. Friend the Member for Bristol East mentioned, such as a Crown use licence or a clinical trial? Cystic fibrosis patients need urgent access now to the drug that they have been denied for three years. It is time the Government considered all alternatives.

--- Later in debate ---

[Seema Kennedy]

As always, the shadow Minister makes an excellent point. Crown use licensing is not something that any Government would consider lightly. It is very rarely used in health. It has probably not been used—my officials will correct me if I am wrong—since the 1970s.

The ideal thing is to get a deal, and deals have been done with other pharmaceutical companies; that is the point I want to make. As I have said, Vertex is an outlier in this regard, but that does not mean that I do not have an obligation to look at other options. I will do that.

[Kerry McCarthy]

Will the Minister give way?

[Seema Kennedy]

Can I just make a tiny bit of progress first?

[Kerry McCarthy]

It is on this issue.

[Seema Kennedy]

All right, given that we have more than half an hour left.

--- Later in debate ---

[Kerry McCarthy]

I really welcome the fact that the Minister is saying so passionately that she feels she has a moral obligation to act. The question is: when? I say that because Vertex has been in these talks for a very long time now. How much longer will it be given before the Minister decides to look at the other options?

[Seema Kennedy]

Well, I have already said it, so I suppose that means—

[Kerry McCarthy]

When?

[Seema Kennedy]

Now. We will look at other options to consider what other methods we can use. As the hon. Member for Washington and Sunderland West said, Crown use licensing has other risks, so the best option is to get a deal. However, we will look at other methods that might enable families to receive the drugs they need.

Some Members have said that Orkambi is available in other countries and asked, “So why not here?” Although that is true, it is also true that other countries have faced problems in agreeing an acceptable price with Vertex; around 50% of the global cystic fibrosis population is unable to access Orkambi. It is not approved for reimbursement in Spain, which I have touched on, or in Portugal, and it is not used routinely in France. It is also of note that the Canadian equivalent of NICE has rejected the drug, saying that the benefit of Orkambi is small and uncertain.

We do not have sight of specific commercial agreements where Orkambi is approved and we do not have the same population needs, as we know, because of the specific population that the UK has; compared with other countries, the UK has a very high proportion of people with cystic fibrosis. So I am not able to make comparisons with other countries. Cystic fibrosis affects about 10,500 people in the United Kingdom—a far higher figure than in other countries. It represents 12% of the global cystic fibrosis population. The UK is a very important market for Vertex.

I thank all Members who have spoken so passionately here today. In particular, I thank the people who have joined us here in the Gallery; I pay tribute to them for all that they do to support their family members. I also thank those people, such as Lucy Baxter, who work so hard to raise awareness of this issue. I thank members of the Health and Social Care Committee, which is carrying out a very thorough and transparent inquiry into this issue, helping to shine a light on it.

As we have heard in great and moving detail today, cystic fibrosis is a devastating, life-limiting condition, and the bravery of those affected should be an inspiration to us all. Drugs that improve sufferers’ quality of life should be available where appropriate, and I urge Vertex to do everything it can to price its medicines fairly and in a way that reflects the health benefits to patients.

NHS England and NICE will, of course, continue their efforts to reach an agreement with Vertex. Access to treatment for all patients is, and always will be, a priority for this Government. My Department has a moral obligation to look at other options now, and that is what we shall do.

Oral Answers to Questions

Kerry McCarthy Excerpts

Tuesday 7th May 2019 Commons Chamber

[Jackie Doyle-Price]

I thank my hon. Friend for his question. Of course I will be happy to meet him. He is right to identify the fact that people with long-term conditions are more likely to suffer from mental ill health. It is very important that we achieve good care co-ordination so that all those issues can be tackled in the round. We will continue to work to ensure that professionals are made aware of these conditions.

[Kerry McCarthy (Bristol East) (Lab)]

I have spent much of the past week supporting the parents of a child who has a very, very rare genetic condition and who now needs the support of child and adolescent mental health services. It has become very clear to me that CAMHS is set up only to deal with mainstream children who can go through perhaps its anxiety counselling courses and who can process information in a certain way. It does not seem at all geared up to help children who have very complex needs and perhaps learning disabilities. What can we do to make sure that those children who are more vulnerable are not left behind?

[Jackie Doyle-Price]

The hon. Lady is entirely right. I am particularly concerned about the impact on young people going through a period of mental ill health who have neurodiverse conditions and other conditions. It is very important that we tackle the entirety of the individual’s need. Clearly, we need to do more to make sure that all children with whatever conditions can access help when they need it.

Oral Answers to Questions

Kerry McCarthy Excerpts

Tuesday 26th March 2019 Commons Chamber

[Matt Hancock]

I am happy to consider all options that can secure access in a way that provides value for money based on an objective assessment of what is clinically right. That is the basis of our discussions.

[Kerry McCarthy (Bristol East) (Lab)]

I am glad that the Secretary of State is taking a personal interest in this matter. In Thursday’s debate, I mentioned the case of Oli Rayner, who gave evidence to the Health Select Committee. He fell ill in his 30s and was given Orkambi just to make him well enough to undergo a lung transplant operation. Is it not ludicrous to wait until people are virtually at death’s door before being prepared to give them the drug?

[Matt Hancock]

That is one very important consideration. Having met people suffering from cystic fibrosis and heard directly the stories they tell about the impact on their lives and how it potentially shortens their lives, I think it is very important that we find a solution, which is why I was so determined to bring the parties together.

NICE Appraisals: Rare Diseases Treatments

Kerry McCarthy Excerpts

Thursday 21st March 2019 Commons Chamber

[Kerry McCarthy (Bristol East) (Lab)]

I will be brief because I have spoken on this topic many times, specifically about cystic fibrosis, but before I get on to that I congratulate my hon. Friend the Member for Blaydon (Liz Twist) on her speech about PKU. I popped into the PKU drop-in event a few months ago purely by accident when I was doing one of those classic MP things where we are asked to go here, there and everywhere to be photographed holding up a sign or wearing a silly hat. I knew nothing about PKU, but I talked to an 11-year-old boy there about his diet. I am somebody who chooses to have a fairly restrictive diet in that I do not eat meat, dairy or fish, but I look for protein alternatives, and I was struck by the sheer difficulty that the boy has in functioning and by the fact that he is unable to enjoy the normal life of an 11-year-old. My heart went out to him, and I do not need to add any more, because my hon. Friend made such an excellent speech.

We have also had excellent speeches about the difficulties of obtaining Spinraza. I do not claim to be an expert, as some other Members are, but constituents have been in touch with me with similar problems. I would therefore support any efforts to get the drug approved, because that would make a huge difference to their lives.

As I said, I am here to talk about cystic fibrosis, and I have a personal interest in that my 14-year-old niece Maisie has CF. She must be the most mentioned niece in Parliament, because she gets name-checked quite a lot. Before I talk about Maisie, I want to mention the recent evidence to the Health and Social Care Committee from Oli Rayner, who lives with cystic fibrosis. He has just turned 40 and had a lung transport around 18 months ago, so he is at the healthier end of the spectrum, and he talked about being quite lucky until he reached his thirties, when his health started to deteriorate.

However, many cystic fibrosis patients are not as lucky. My constituent Lee Partridge lost both his daughters within eight months when they were both in their late teens, and it is common for people to start to deteriorate when they reach their teens. Luckily, my niece is very much at the healthier end of the spectrum, but that does not influence my judgment; I want to make the case for Orkambi to be available not just for her when it is needed, but for everybody else with the condition.

I have been trying to get my head around quality-adjusted life years, how the calculations are made, whether NHS England is doing the right thing and whether NICE is using the right sorts of calculations compared with other countries. NICE says the calculations factor in societal benefits, but it is not clear how they do that.

The hon. Member for Reigate (Crispin Blunt), who made an excellent speech, spoke about the cost of hospital treatment to society as a whole. If a person has to go into hospital several times a year, there is the cost of care, but there is also the cost of lost work time, the cost of care by family members and even the cost of transportation to hospital if they live in a rural area. There are so many factors. Although NICE says it takes those things into account, I cannot quite see them. Was it Hugh Dalton or Clement Attlee who was called a “desiccated calculating machine”? Anyway, the system seems to be based on desiccated calculations.

It may be incredibly naive of me, but why cannot the system just think of the impact on people’s lives and of the hell they are going through? It is incredibly difficult for teenagers to come to terms with discovering that they have a life-limiting condition, and there is also the issue of the availability of mental health support and counselling.

It is striking that everyone who gave evidence to the Health and Social Care Committee said that the drugs work. There is no question but that this would be of benefit to people. Kalydeco, the first drug, helped only 5% of patients, and it was funded in part because not many people would benefit and, therefore, the costs were lower. We now have Orkambi, and there will soon be a successor that will benefit about 40% of patients. For the future we are looking at triple therapies that will cover about 90% of the cystic fibrosis community. If 90% of people can benefit, of course the overall cost will go up, but the decision should not be made on the basis that we fund one thing because not many people will benefit.

I am concerned that Vertex and other companies would be deterred from exploring further therapies and treatments if they cannot get a commercial deal. The line that jumps out of the Health and Social Care Committee’s letter to Vertex, which it wrote shortly after the meeting, is:

“Vertex appears to have decided on the pricing of its therapies on the basis of the return it wants to make, rather than the value which they bring. NHS England is right to continue to take the wider patient population for whom it is responsible into account.”

The Minister has previously responded to such debates, so can we have an update on where the conversations have got with Vertex, including the Secretary of State’s meeting? There are so many cystic fibrosis patients waiting to hear the outcome. Last time I looked, the petition was up to about 80,000 signatures. Can we try to get some answers soon?

--- Later in debate ---

[Ian Austin (Dudley North) (Ind)]

I thank the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), and the Backbench Business Committee, for arranging this debate. I am here to speak up for people in Dudley with cystic fibrosis, phenylketonuria—or PKU—and spinal muscular atrophy.

I have been campaigning with others to make Orkambi and other new treatments available for people with cystic fibrosis because I was contacted by Carly Jeavons from Dudley, who took part in the clinical trial for Orkambi, and by Samantha Carrier, whose baby daughter Daisy was diagnosed shortly after birth. Samantha has given up her career and now devotes her life to campaigning for access for these life-changing drugs. While listening to this debate, I have been receiving emails, texts and other messages from people around the country—heroic parents of children with cystic fibrosis who work so hard and campaign tirelessly for the treatments that their children need. I know that the Minister has met some of these parents, and I hope he will have some new answers for them today, because they have worked so hard to raise these issues, which really do need to be sorted out.

Before the clinical trial, Carly Jeavons had to take 90 tablets and do two hours of physiotherapy a day. She had a lung function of around 44% and spent two weeks in hospital every three months. She had to choose between the financial hardship of leaving work or her health being made worse as she struggled on at work. She told me:

“Orkambi has changed my life. My health has remained stable. I only need one or two courses of IV’s per year instead of the four previously, hospital visits have been massively reduced and admissions are non-existent.”

Since having Orkambi, she has been able to go on holiday abroad for the first time with her family, and she has got married. She has also started a business and is employing people, so she is making a much bigger contribution to the economy. The Government need to look at the contribution that people who get these drugs can make to the economy, not just at the costs of providing the drugs. I believe that NHS England and NICE are with Vertex this afternoon for yet another meeting about whether these cystic fibrosis drugs can be provided. But this comes three years after NICE appraised Orkambi. I really hope that the Minister will explain how this situation can be resolved and how other situations like it can be avoided, so that patients can get can get access to these drugs.

Let me turn to the issue of treatments for people with PKU. Some of the people who have been leading this campaign are sitting in the Public Gallery, watching this debate. Again, I hope that the Minister will have some good news for them today. As we have heard, PKU is a rare metabolic disease that leaves people unable to metabolise protein properly, leading to a toxic build-up of material that can cause irreversible brain damage. The only existing treatment is a strict diet of extremely low protein, meaning that almost all normal foods are off limits. The diet is lifelong, and sufferers find it stressful and difficult to cope with. I had never heard about this condition until a woman in Dudley called Kirsty Thornton got in touch with me. Since then, I have met the campaigners and taken part in a PKU diet challenge. I have also joined the cross-party parliamentary campaign led by the hon. Member for Blaydon to ensure that people with PKU get access to the treatments and supplements they need.

It is heartbreaking for parents of young kids with PKU who do not understand why they cannot go to their friends’ birthday parties in case they eat the wrong foods that will make them tired, sick and ill for the rest of the day, or for longer. Students say how difficult it is when their friends are going on nights out, or they move to university and the people they share a flat with are ordering in pizzas and so on, or they cannot go out on a date because they do not know what they are going to be able to eat or not eat. This must be really tough for young people.

[Kerry McCarthy]

My hon. Friend will probably be aware that the Government are looking at a new food strategy, and one of the things under consideration is better food labelling. A lot of people think that those who make certain dietary choices do it almost to be trendy, or just because it is the fashionable thing to do, but there are people whose lives are at stake if they cannot get the information on food that they need. Food manufacturers and anyone else involved in the provision of food need to be alert to the fact that it is important that people know what they are going to be eating.

[Ian Austin]

That is a really important point. The PKU campaigners have explained to me that on some occasions when manufacturers have changed the ingredients in food or drinks, that has had a terrible impact because people with PKU have not necessarily known about it, so they have continued to drink or eat things that they have consumed without any problems in the past, but because the ingredients have changed, it has caused them a big problem.

For many people with PKU, taking Kuvan considerably increases the amount of protein that they can safely eat. We are therefore urging the Government, the NHS and BioMarin, which manufactures these supplements, to agree a deal so that people with PKU can enjoy a normal healthy life. I spoke to the National Society for Phenylketonuria this morning, and it told me that the whole PKU community are demoralised. They say that they are working hard but feel that not much progress is being made. What is the Minister going to do today to give these people, some of whom are in the Public Gallery, and others who are sitting at home with their kids watching this debate, to give them hope of this situation being resolved?

My next point is about Spinraza treatment for people with spinal muscular atrophy, or SMA, which affects an estimated 1,300 people across the UK. It can cause irreversible loss of a child’s ability to crawl, walk, breathe and swallow. In the most severe cases, it can cause death. Spinraza is the first possible treatment for those who have SMA types 1, 2 and 3. It can slow its progression and prolong life. From April, this treatment is going to be routinely available in Scotland, and it is already available in 24 other countries in Europe, yet it is still stuck in the NICE process for England, Wales and Northern Ireland.

That is why, in the end, the purpose of this debate is to ask the Government to look carefully at the way that NICE works. New drugs are being developed, and technological changes are happening, so rapidly that the Minister needs to be able to tell us how the way drugs are assessed and licensed, and then approved, will work in future. How is he going to ensure that these ground-breaking drugs are made available to the people who need them, when they need them? In 2016, NICE was not able to recommend the use of Orkambi because of uncertainty around its long-term value, impact and cost-effectiveness. But this drug is available in the USA, across Europe, and, more recently, in Scotland, so when do the Government think patients in England are going to get it? This is really urgent. The system has not worked and patients are being let down.

On PKU, NICE decided to start an appraisal of Kuvan in 2018, but this has since been suspended. NICE is currently reconsidering which appraisal process to use to access Kuvan, and the NHS is considering whether to fund an interim policy for the use of the drug. But, again, this is not enough, and not quick enough. The NICE process sees PKU as rare, but not rare enough. As we have heard, the majority of PKU treatments are assessed by the STA process, which is designed for non-rare treatments. NICE’s approach evaluates the lifelong costs of treatment, meaning that the cost thresholds and the approach to evidence are all designed for more common diseases than PKU. SMA sufferers are waiting for NICE, NHS England and Biogen, which manufactures Spinraza, to come to a deal. I hope we will hear better news on all those things from the Government than we have in the past.

It is my job to listen to people in Dudley who are living with cystic fibrosis, PKU, SMA and other rare diseases, to come down to London and speak up for them in Parliament and to demand, as we are doing this afternoon, that the Government ensure that they get access to the treatment they need and deserve.

Orkambi

Kerry McCarthy Excerpts

Monday 4th February 2019 Commons Chamber

[Kerry McCarthy (Bristol East) (Lab)]

As some of my colleagues will know, I have a 14-year-old niece with cystic fibrosis, as well as many constituents who have it. I obviously know people within the community, too. Is the hon. Gentleman aware that in 2017 Vertex earned £2.5 billion from the sale of Orkambi and the chief exec was paid more than $17 million? I think that the Department of Health and Social Care probably has to go some way towards meeting Vertex on this, but it seems to me that there is an awful lot of money sloshing around and both sides are in a position where they could compromise.

[Bill Wiggin]

First, let me say how sorry I am to hear about the hon. Lady’s niece. We should take this very seriously. The figures that I have are even worse than the ones that she has laid out.

The price remains inaccessibly high, and this is entirely due to the powerful patent laws that allow pharmaceutical companies to monopolise drug production. Vertex expects to retain monopoly intellectual property protection on its cystic fibrosis drugs well into the 2030s. Analysts conservatively estimate that it will generate profits of $13 billion on Orkambi and another related drug, Kalydeco, alone. This could be used to fund further research and development—to reward its shareholders for its brilliant breakthrough and perhaps to encourage it to do more. But no, Vertex has spent $500 million on buying back its own shares. Well, that should certainly boost executive remuneration.

I am aware that provisions exist under the Patents Act 1977 for the Government to take independent action against Vertex. Crown use licensing is a powerful legal tool that can be used to safeguard public health. It can ensure the availability of fairly priced medicines in a competitive pharmaceutical market. Section 55(1) of the Act states that the Government can be granted non-authorised use of patents

“for the services of the Crown”.

That can be granted at all stages of manufacture, use, importation, sale and retention of a product. This is a legal opportunity to break the lethal deadlock that eats away at the youngest sufferers who stand to gain the most from this medicine. Crown use licensing has been used by the UK Government before, to great effect. They can suspend a patent and thereby force down the high price of particular pharmaceutical or medical equipment. For example, in 1991 the Government authorised the supply of machines known as lithotriptors for treating kidney stones. More recently, breast cancer patients have lobbied the Scottish Government to implement a Crown use licence on the drug Pertuzumab. Crown use licensing could similarly be used to overturn the patent monopoly on Orkambi by Vertex.

Oral Answers to Questions

Kerry McCarthy Excerpts

Tuesday 27th November 2018 Commons Chamber

[Matt Hancock]

I can recommend to anybody spending the night with my hon. Friend in Derriford Hospital, where we learnt a huge amount. The team there were absolutely amazing and it was a brilliant experience. I also learnt a lot about the capital bid, which I have been keeping my eye on very closely. My hon. Friend should hear shortly.

[Kerry McCarthy (Bristol East) (Lab)]

T2. My niece Maisie turned 14 last week, and she has cystic fibrosis. It is not enough for the Secretary of State to say that letters are being sent back and forth between the drugs company and NICE. Will he get the relevant parties in a room, with him chairing the meeting, so that teenagers such as Maisie can have the drugs that will certainly change their lives, if not save them?

[Matt Hancock]

I have a huge amount of sympathy for the hon. Lady’s point. We did act to ensure that the parties came together. The offer has been made and the response from the company has frankly not been good enough. It needs to come to the table; the ball is in its court.