Duchenne Muscular Dystrophy

Motion made, and Question proposed, That this House do now adjourn.—(Kate Dearden.)

17:00:00

I thank the Minister for what I think is her triple shift at the Dispatch Box today. A few months ago, I met three really great young lads—Benjamin, Eli and Jack. We had a great conversation, and they had me laughing and joking along with them for a very long time. I slightly disappointed Eli when I first opened my mouth because, listening to me, he recognised where I was from, and he asked if I could introduce him to Sam Fender. The north-east is quite small, but I do not know everyone, and I could see the disappointment on his face. One of the things we talked about was what they want to do in the future. Eli is considering being a musician, and Jack said he would like to do something involving public speaking or, who knows, even be a politician. It was really great to see that level of ambition, but for me it was tinged with sadness; I knew that they were unlikely to realise their ambitions, because Benjamin, Jack and Eli are unlikely to see their 30th birthdays. Benjamin, Jack and Eli all have Duchenne muscular dystrophy. It is a disease that affects only boys, and about 100 baby boys are born with it every year. Diagnosis is normally at the age of three or four, and at that moment the parents find out that their son has a life-limiting condition that cannot be treated, and that he will probably need a wheelchair by the age of 12 and a ventilator by the age of 20. There is no cure for Duchenne muscular dystrophy, but there is hope now with a new drug called givinostat. Givinostat was developed by an Italian company, Italfarmaco, and it is an example of the brilliance of our UK life sciences sector that it decided to develop the drug here in the UK. As it has been trialled here, Italfarmaco has given the national health service a unique option to prescribe givinostat through an early-access programme entirely free of charge. The NHS can offer this drug to boys in the UK completely free of charge, and the early-access programme has been available since November last year. Members might think that such an opportunity would be seized quickly by the NHS, but sadly it has not been. Many families have had their hopes raised, only for them to be dashed when they find that they cannot access the treatment. Patients, families and charities are now calling for urgent access to givinostat, as are many Members of this House. My constituent Tracy is one of the many who have been fighting for their child’s right to live, and for the treatment that has been proven to slow down the progression of Duchenne. Her son, Tom, is 14 years old, and she tells me that he is deteriorating. Givinostat has market authorisation for all boys with Duchenne over the age of six. It has been proven to slow its progression in clinical trials. It is not a cure—the families realise it is not a cure—but it could help boys and young men to keep walking and use their muscles for longer.

Like my hon. Friend, I have a constituent, Jamie Tierney, who, sadly, suffers with Duchenne muscular dystrophy. Recently, he has been able to begin treatment, as have other patients in Scotland, but it took a lot of work and in some cases intervention by lawyers to get that. Jamie’s family tell me that “Time is muscle”. Does my hon. Friend agree that the sooner we make givinostat available across all parts of the UK, the better it will be for those people?

Yes, I do agree with that very timely intervention. In fact, the slogan of Duchenne UK is “Time is muscle”, and it is absolutely right that every day and every week makes a difference to these boys.

I congratulate the hon. Gentleman on bringing forward this debate. He and I spoke about it the other day. I have some constituents who have had a brave few years with Duchenne muscular dystrophy. The problem is that the opportunity of this drug has never been there for them, but it needs to be. Up until now in Northern Ireland, Duchenne treatment has included corticosteroids, physical therapy and other supportive therapies, while access to newer treatments, such as vamorolone or givinostat, are being worked on—we in Northern Ireland have not had access to them either. It seems to be almost a postcode lottery. Does he agree that these drugs could give those young boys a life-changing opportunity that they would never have otherwise, and that, with great respect to the Minister, the Government need to move and ensure that they all get these drugs?

I thank the hon. Member for that intervention. I am very pleased that he has intervened and I think the comment about a postcode lottery is exactly right. I noted, when I looked at the figures, that currently the Belfast health and social care trust is not offering the drug to lads in Northern Ireland, because it is claiming that it will cost £309,000 for 13 patients. That is different from the rest of the country by an order of magnitude, so I would be grateful if the Minister looked very carefully at the situation in Northern Ireland.

I thank the hon. Gentleman for securing this debate. I have been engaging with the Oxford NHS trust, which is not yet providing givinostat to boys in Oxfordshire. Its own business case suggests that it would cost less than £2,000 per boy and £66,000 in total for the 35 boys. Clearly, as a country, we should be able to afford such a sum. Does he agree that a hospital that wants to be world-leading should be providing it? Does he further agree that this is exactly the sort of thing we need Government intervention on, so that these boys can get treatment as soon as possible?

I do agree. I recognise the figures from the Oxford university hospitals NHS foundation trust. It is quoting around £2,000 per lad treated, which I understand is very similar to other areas of the country. I see that as quite a small amount of money for the 35 lives that could be saved. Despite, as we have heard, givinostat being available for more than seven months, there has been a very inconsistent approach across the UK. A very small number of ambulant people living with DMD have so far received the treatment, and no non-ambulant boys have been treated with it yet.

I, too, thank my hon. Friend for securing this important debate. I met a family in my constituency whose son is suffering with Duchenne. He is at that key stage right now where he still has his mobility, but he is starting to lose it. Does my hon. Friend agree that, as the campaign says, time is muscle? If we act now, we can prevent further deterioration of his mobility.

Yes, my hon. Friend is absolutely right about that. Time is muscle, as she says. Sadly, because the drug is currently only available, in the areas where it is available, to lads who are ambulant, the waiting time has meant that some lads out there who would have qualified for the drug now no longer do so. That is, frankly, heartbreaking. Of course, the use of one’s legs is not the only thing people are concerned about. We need to be able to use our arms to be able to dress ourselves, feed ourselves and brush our teeth, and givinostat could help with that. It is very disheartening that while coping with all of this, families, parents and carers and so on are having to fight for access to the drug on a trust by trust basis. They have done that with the support of all the Members in the Chamber tonight. I am very grateful for the fact that they have turned up, because I know families are watching. I also want to mention some Members who have approached me who would have loved to have been here but cannot be due to other commitments in the House. My hon. Friend the Member for Newcastle upon Tyne East and Wallsend (Mary Glindon) has campaigned on this subject for many years and chaired the all-party parliamentary group. A constituent of my hon. Friend the Member for Basingstoke (Luke Murphy), who has the disease, visited Parliament on Monday and was so pleased to see on the Annunciator that we were having this debate. My hon. Friends the Members for Bury North (Mr Frith) and for Bury South (Christian Wakeford) worked together to ensure that Manchester university NHS foundation trust does now provide the drug to their constituents. William from Codicote, a constituent of my hon. Friend the Member for Stevenage (Kevin Bonavia), is living with Duchenne. The hon. Member for Chester South and Eddisbury (Aphra Brandreth) has been supporting Mr and Mrs Binns, whose son Jack has Duchenne. They do not yet have access to givinostat and are trying to get it. I have also had representations from my hon. Friend the Member for Beckenham and Penge (Liam Conlon), the hon. Member for Westmorland and Lonsdale (Tim Farron) and my hon. Friend the Member for Altrincham and Sale West (Mr Rand). We can see that there is broad support across all parties in the House, with Members working with their constituents to try to secure this drug which is free of charge—free of charge—to the NHS. We have heard that provision is very patchy across England. There is some central co-ordination in Scotland, but no lad in Northern Ireland can access the treatment.

I thank my hon. Friend for securing this debate. It is important to acknowledge quality of life with this drug, but we also have to acknowledge that many people with Duchenne are living with 24/7 care needs, including my constituent, Dylan Phillips, who lives at the excellent care home in Glenbervie. Does my hon. Friend agree that those living with Duchenne in Scotland should be protected as they would be in England under continuing healthcare schemes, rather than having to bear the cost of their social care and living on only £35.90 a week with universal credit and adult disability payment, as they do not qualify for continuing healthcare in Scotland?

The important thing for us to consider here is that with only 1,100 or 1,200 lads and young men in the country living with the disease, and only 500 boys eligible for the treatment, we could at least expect some consistency in approach across the whole of the United Kingdom.

To build on that point, I recently met the parents of a young man in his 20s who absolutely reinforced the need to get givinostat funded properly across the country. I was also struck by how the lives of both parents—they are teachers—and their other child were deeply affected by having to support their son and brother. Does my hon. Friend support my plea to the Minister to reassure my constituents that adult social care services will continue to support people like them, so that their lives can be enriched?

It is really important that we remember the parents, families and carers—the big support network around these boys. It seems to be such a small thing that we need to do from the point of view of the NHS. There are some very good examples: Leicester royal infirmary is leading the way as the first hospital to dose a patient. However, as we have heard, some large specialist children’s hospitals in many areas have been slow to commit, and only a few have actually started dosing patients, although some are still working to make givinostat available. The barriers that we hear about are a lack of capacity and resource constraints. Clinics say that they need small amounts of additional consultant time for pharmacy support and extra blood tests, but really, in the context of the number of blood tests that are carried out in the NHS on a daily basis, this number is really quite small. However, some trusts still insist on telling families that they cannot deliver the treatment because of that. Given that some trusts can and some cannot, I would like to hear from the Minister what we might do to even out the service across the country. Some trusts have expressed concern that after starting patients on givinostat, the National Institute for Health and Care Excellence might subsequently not approve it at its upcoming meeting in July, and trusts would have to withdraw the treatment. Well, they would not need to do so: Italfarmaco, the previously mentioned pharmacy company, has made it clear that in the event of a negative decision from NICE, it will continue to provide givinostat for those already enrolled on the early-access programme for as long as it is deemed clinically beneficial to the patient. Continuity of supply letters have been signed between hospitals taking part in the early-access programme and the company to ensure that this is in line with NHS England guidance. There are two reasons now to accelerate the roll-out before NICE’s decision in July. The first is that every day and every week makes a difference to these young lads; the second is that every lad who gets on that programme before July will be guaranteed this treatment for the rest of their lives, if it is not approved by NICE. When I met Benjamin, Jack and Eli, I had already disappointed Eli by not being able to introduce him to Sam Fender, so I thought I would try to redeem myself. I said to them, “Look, I’ve come to see you, but clearly, when you came to Parliament, you didn’t want to meet the Member for Stockton North. Who would you most like to meet?”. They all said they would like to meet the Secretary of State for Health, not primarily because he could help them with their disease, but apparently they like him—he is a very popular Member of Parliament. I said, “I’ll see if I can sort that out,” and I did manage to sort it out. The Secretary of State very generously gave a lot of his time—I think his private office thought he had vanished off the face of the earth, because he had a great time chatting to Benjamin, Jack and Eli. I know that his intervention really cheered them up, but it also gave them hope for their futures. I want sincerely to thank the Secretary of State for Health for his generosity in sharing his time on that day and for the difference that he made to those boys. I turn to my requests of the Minister. In the short term, what these families need is for hospitals with specialist neuromuscular services across the whole of the United Kingdom—in England, Wales, Scotland and Northern Ireland—to implement the early-access programme swiftly, and for it to be rolled out to non-ambulant patients, too. The free availability of the drug from the manufacturer means that cost alone is not the barrier here; the barrier is bureaucracy. My simple ask to the Minister is to act with the urgency needed to roll out the medicine across the country as quickly as possible. Every day and every week matters—the lads with Duchenne do not have time to wait.

17:14:00

It is a real pleasure to respond to this moving debate. I know that many people with Duchenne muscular dystrophy and their families will have wanted to tune in to hear what was said this afternoon. I thank Members who have contributed in different ways to the debate. In particular, I thank my hon. Friend the Member for Stockton North (Chris McDonald), who secured this debate and advocated so powerfully for these families. He really brought to life the experiences of Benjamin, Jack and Eli, and I commend him for doing so. I first acknowledge the profound impact that this debilitating disease has on those living with it, and their families, and the urgent need for new and effective treatments. As has been said, my right hon. Friend the Secretary of State for Health and Social Care heard at first hand from people affected by this condition earlier this year, when he attended an event hosted by Duchenne UK. He met many young patients and listened to what they said about the challenges that they face. As we all know, meeting families and individuals from our constituencies leaves a long-lasting effect on us, and it is important that we continue to meet them. Timely and equitable access to innovative medicines for the treatment of DMD and other rare diseases mentioned today is of the utmost importance. The National Institute for Health and Care Excellence is the independent body responsible for assessing whether new licensed medicines can be recommended for routine use in the NHS, based on a thorough assessment of their clinical effectiveness and cost-effectiveness. Through this process, many thousands of patients, including those with rare diseases, have been able to benefit from effective new treatments at prices that represent value to the NHS. NICE has been able to recommend two medicines for the treatment of DMD: ataluren, recommended in 2023, and vamorolone, which was recommended in January this year and is now available on the NHS to around 1,700 eligible patients, in line with NICE’s recommendations. As my hon. Friend has said, NICE is appraising givinostat, and the first NICE committee meeting is scheduled for July this year. If the medicine is recommended, the NHS in England will be legally required to fund it. I am aware that a small number of patients in the UK have been receiving treatment with this drug through a company-led early access programme, established by the pharmaceutical company Italfarmaco, as we have heard. It is important to note that participation in these programmes is decided at NHS trust level, and although the drug is free to patients taking part in it and to the NHS, NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP. NHS England has published guidance on free-of-charge medicine schemes, such as the givinostat EAP, providing advice on financial, administrative and clinical risks. NHS England cannot, however, centrally direct NHS trusts to participate in company sponsored EAPs like this one, or in any other private activity. To issue any form of national direction around participation in EAPs would both pre-empt and undermine the role of NICE, whose purpose is to advise the NHS on whether particular treatments should be made routinely available on the NHS. Even when there is an agreement that a company will continue to provide a drug free of charge in the event of a negative decision by NICE, participating trusts remain liable to cover the significant costs of delivering that service, including the cost of the clinical resources and staff time needed. That would be outside their funding allocation and in addition to paying for any subsequent NICE-recommended treatments that they would be mandated to fund.

I thank the Minister for her response, to which I am listening very carefully. I appreciate her point that it would be inappropriate for the Department of Health and Social Care to direct what trusts should do, but what we have heard from the trusts is that they would like to issue this drug, but have certain issues and problems. Perhaps it might simply be a matter of the Department giving help and support, and facilitating information-sharing between trusts that have made this work and those that have not. Perhaps it could be more encouragement than direction.

I thank my hon. Friend for that constructive suggestion. We need to wait to see how the NICE recommendation goes in July. With this disease and so many others, it is important to share learning and information, and trusts should be encouraged and supported in doing so. We will work with him on that constructive recommendation. I understand my hon. Friend’s concerns about the fact that non-ambulant patients are not yet able to access givinostat, but it is important to note that the eligibility criteria for participation in the early access programmes have been determined by the pharmaceutical company. NHS trusts that decide to participate in the EAP must only provide treatment in line with the criteria, which state that patients must be ambulant. A clinical trial is being carried out by the pharmaceutical company to evaluate the safety and tolerability of the drug in non-ambulant patients, and to further explore the efficacy of the drug in this population. I know that for the patients and families affected, it will be disappointing to hear that there is no access to the drug for ambulant patients before a NICE decision, or for non-ambulant patients prior to clinical trials being concluded. I want to assure my hon. Friend that we have arrangements in place to support rapid access to new medicines.

Will the Minister give way?

I will not; I want to finish in the time available, and I think Members want to hear the full response. Outside of company-led EAPs, there are established routes for patients to get access to new, innovative medicines prior to them being licensed. The early access to medicines scheme, or EAMS, helps give people in the UK with life-threatening or seriously debilitating conditions early access to new medicines that are not yet licensed where there is a clear unmet medical need. EAMS is supported by key partners, including the MHRA, NICE and NHS England, and is a key part of this Government’s commitment to accelerating patient access to innovative, life-changing treatments, in support of the UK’s position as a global leader in life sciences. In fact, since the scheme launched in 2014, over 50 medicines, including for this disease, have benefited from being accessed early through EAMS. The innovative medicines fund has also made available £340 million of ringfenced funding for the NHS to fund early access to medicines that NICE has recommended with managed access. Through this process, licensed treatments that demonstrate substantial clinical promise but still have significant uncertainty around their clinical and cost-effectiveness can be funded. Further evidence is then collected on the drug for a defined period of time. That is considered by NICE in determining whether the drug can be recommended for routine NHS funding. The Secretary of State has been clear that if givinostat is recommended by NICE in draft guidance, NHS England should aim to work with the pharmaceutical company to provide early funding through the innovative medicines fund. This could potentially speed up access by up to five months, and the treatment could be funded as soon as this summer, if recommended. This scope of this debate is wider than just access to new medicines. It is important to note that while rare diseases are individually rare, they are collectively common. One in 17 people will be affected by a rare condition over their lifetime. The UK rare diseases framework outlines four priorities, based on engagement with the rare disease community. They are: helping patients to get a final diagnosis faster, increasing awareness of rare diseases among healthcare professionals, better co-ordination of care, and improving access to specialist care, treatments and drugs. In England, we publish a rare diseases action plan annually. These detail the specific steps we are taking to meet the shared priorities of the framework. I am pleased to highlight the 2025 England action plan, which was published in February this year on Rare Disease Day. One such action is reviewing the effectiveness of early access schemes, such as the early access to medicines scheme, the innovative licensing and access pathway, and the innovative medicines fund. They are all designed to help make innovative treatments available earlier to patients who need them. We are specifically considering how well they support access to treatment for people living with rare diseases like Duchenne. NHS England, NICE and MHRA will meet annually to continue to discuss progress on these schemes. These meetings will include representatives from patient advocacy groups and from industry, and clinical researchers, and the next one will happen in the summer. Managing a complex rare condition can be challenging, and it often means interacting with many different specialists and providers of health and social care. It can mean travelling across the country to access highly specialist care from experts. All of that can add up to a significant emotional and physical burden, and it can deepen inequalities. Co-ordination of care can minimise this burden on patients and their carers, and it can ensure that healthcare professionals work together to provide the best possible care, as we have discussed this evening. In the 2025 action plan, we have introduced a new action to incentivise providers to run clinics for multi-system disorders, in order to reduce the number of appointments and improve co-ordination of care for families. The NHS is also working to include the definition of “co-ordination of care” that is set out in the CONCORD—co-ordinated care of rare diseases—study in all new and revised NHS service specifications for patients with rare diseases. I recognise how hard it is when patients want access to these new treatments. I also recognise the distress and worry it causes, not only to patients, but their families and friends. Hon. Members have articulated that well on behalf of their constituents this evening. The Government are committed to providing access to the most innovative medicines, but it has to be at a price that provides value for the NHS, and it has to be clinically safe and effective. That is why we are working hard with industry, NICE and MHRA to make that happen. I know that my hon. Friend the Member for Stockton North will continue to work with the Government and providers to make that happen. I am grateful for the opportunity to respond to this debate on such an important issue. Question put and agreed to.

17:25:00