Motion made, and Question proposed, That this House do now adjourn.—(Julie Marson.)
Epidermolysis bullosa is dreadful. It is often referred to as butterfly skin. It is a little known and rare genetic skin blistering condition, which causes skin to blister and tear at the slightest touch. BE—I will be kind to myself and refer to it in the rest of my speech as BE—causes excruciating, lifelong pain, and can have a devastating impact on the physical and mental wellbeing of patients. It can also greatly affect patients’ families because of the constant treatment needed. In many cases, this means several hours of bandaging and unbandaging the most affected parts of the body daily. In its most severe form, EB also affects internal organs and eyes, causes severe problems when it comes to eating and drinking, and requires extensive healthcare—and there is no cure. It is believed that EB affects around 5,000 people in the UK, although there is limited information available from the NHS. Although that is a relatively small number, the severity of the condition is such that it has a deep impact.
I was asked to secure this Adjournment debate by some of my constituents, DEBRA, which is a UK-wide charity supporting people with EB and their care givers, healthcare professionals and researchers who work with EB. The charity exists to improve quality of life for people living with EB, and to fund pioneering research to find effective treatments. The charity works in partnership with the NHS to deliver EB healthcare services. There are four centres of excellence delivering specialist care in the UK, along with additional hospital care and clinics at other locations. The charity has a proud record of funding EB research in the UK and internationally, funding the first clinical trials in gene therapy. Its current focus is on fulfilling its mission to help people with EB live a life free of pain, a point I will return to later in my speech.
Many colleagues may be familiar with DEBRA from some of its network of more than 100 charity shops, which are the main source of the charity’s income. I was able to visit one of the DEBRA shops, located in Locksbottom in my Orpington constituency, a few months ago, and I met not only the volunteers there, but a local constituent called Wendy, who suffers with EB. This particular lady has been suffering from EB since birth, and she told me of her experience and the experience of others who have an even more severe type of the condition.
As a child and even as a young lady, Wendy did not know that she had EB because doctors had not diagnosed the condition, which, particularly in the early years, is difficult to spot. She did, however, suffer constantly from blisters on her feet and her legs. Wendy told me about how, while she was growing up in constant pain, she would try to hide her wounds from friends and classmates, and would avoid wearing skirts to avoid causing unpleasant reactions from others. Yet as terrible as this sounds, Wendy told me her case was not uncommon and not as severe as it was for other patients.
I commend the hon. Gentleman for bringing this debate forward. He raises an issue that affects not only his constituents, but mine and those of others across Northern Ireland, so I commend him. Does he not agree that this dreadful disease, which is named the butterfly disease because it is unusual, has the capacity to limit life and the quality of life, and that the funding for the treatments he refers to, which can provide relief to constituents in all of our constituencies—in his, mine and those of others in this House—is vital? Would he urge the Minister, who is a very compassionate man and understands the issue very well, to give even the smallest bit of funding towards research to try to find a cure for the disease and to help the lives of those who have it?
I thank the hon. Gentleman for his kind words and the way he expressed them, and I agree entirely with those sentiments. Without giving out too many spoilers at this stage, there will be a request for a small amount of funding towards the end of my speech.
What I learned during the visit I referred to was truly moving, and I am particularly grateful to have met Wendy. I also thank DEBRA’s director of research, Dr Sagair Hussain, and the excellent staff at the charity shop in my constituency, for inviting me to visit them and learn more about how they help individuals who live with this painful condition. In the spirit of thanking people, I also thank the Minister for his interest in this subject and for being here this evening to respond to the debate, and the Minister for Social Care for recently answering a written parliamentary question that I tabled about EB.
I stress that we cannot merely wait for a cure for this condition. We need to make a difference for patients who are suffering today and those who will be living with the condition for the foreseeable future. All EB patients are crying out for better therapeutic treatments, which have the potential vastly to improve their lives. DEBRA has set an objective of securing two to three treatments from drugs that are already licensed for other conditions, to radically improve the quality of life experienced by people with EB. In reply to my recent written question, the Minister for Social Care said that medicines that are potential candidates for repurposing in this way should be put forward for consideration for support from the Medicines Repurposing Programme. I am grateful for her guidance, and officials from the MRP have been in touch with DEBRA since to talk about the programme’s work. That is excellent news.
In addition, I was delighted to hear that some innovative treatments for EB are either in trials or are being considered by the National Institute for Health and Care Excellence. Specifically, NHS England is working with NICE on the evaluation of two products for EB: birch bark extract for skin wounds, and a gene therapy with a name that I find particularly difficult to pronounce, although I will give it a shot—beremagene geperpavec. I have almost certainly mispronounced that, but it is still encouraging news. However, my understanding is that those two treatments will be available only to a fraction of the total number of people suffering from EB. That is why the repurposing process for more mainstream therapeutics is so important.
DEBRA has identified six anti-inflammatory drugs that could help with EB. Several of those are already available for people with more common skin conditions such as eczema and psoriasis, but for people with EB they could be nothing short of life-changing. They have the potential to transform thousands of lives by improving wound healing, reducing pain, and lowering the burden on the family members and carers of those with EB.
I agree with the hon. Lady. That was very much the story for Wendy, the lady I met in the shop, and she was not alone in that. It is particularly true when people are young and have EB but doctors are unable to diagnose it at that stage. They do not know why they have open and weeping sores. These things sometimes attract a smell as well, and as a result people are ashamed of their condition. It has a bad social stigma and is bad for their sense of morale.
The drugs would also have a significant economic benefit. For example, research by an expert dermatology professor at King’s College London found that, when used for EB, one of the drugs has been reported to reduce daily bandaging time from three hours to one by reducing the severity of the wounds, and to reduce skin itch by 60%. That in turn would save time and money for the NHS, and reduce stress on the family unit supporting the patient. Studies by the London School of Economics in 2016 and 2022 reported that EB has a wider economic impact, as parents and family members are currently obliged to reduce labour market participation due to the informal care of their loved one. The same study also revealed a higher prevalence of psychological and psychiatric symptoms among those with EB—that refers back to the point made by the hon. Member for Bath (Wera Hobhouse)—indicating a further tranche of support costs that could be reduced if treatments were improved. The most recent LSE study, published in September 2022, said that the annual cost per patient with dystrophic EB—the most severe form of the condition—is about £45,800, depending on the level of disability. That takes into account direct and indirect costs for patients and care givers. So the benefits are hugely significant, but, to enter the MRP process, the treatments in question will need to go through research trials to prove their efficacy in treating EB. To pay for that, DEBRA is seeking just £10 million from the Department of Health and Social Care, the NHS and the devolved Administrations to go with a further £5 million from its own fundraising campaign. That relatively small amount of money would do so much to address the misery caused by this awful condition.
I know a bit about EB. The headquarters of DEBRA are in Bracknell, and I have visited them on a number of occasions. DEBRA’s work is incredible; I am full of admiration for what it does. Having come across people suffering from EB, which is a terrible, dreadful, debilitating disease, I can say with complete authority that the money we are asking for today is an absolute drop in the ocean in terms of the UK’s overall health budget. Actually, we need to be throwing the kitchen sink at this and doing what we can to repurpose these drugs to give the patients and sufferers—these fantastic people—a better quality of life. I urge the Minister please to do whatever he can to ensure that £10 million is just the start. Does my hon. Friend agree that we need to do everything possible for these sufferers?
You heard it here first—19 June. I will attend, and I very much hope that hon. Members will join us in the Terrace Pavilion.
As I conclude my remarks, I have three requests of the Minister. First, will he agree in principle to the Government supporting this request for funding? Secondly, I understand that the MRP process tends to focus on generic drugs, but most of the treatments identified as candidates by DEBRA are not generics. Will he therefore confirm that the MRP will consider non-generic drugs for potential use to treat EB? I have a list for him, in case he needs to see it. Finally, will he agree to meet me and representatives of DEBRA to discuss these proposals for drug repurposing and the many other ways in which we can support patients with EB and alleviate their often devastating symptoms?
I congratulate my hon. Friend the Member for Orpington (Gareth Bacon) on securing this important debate, which has been helpful to highlight this serious condition and the efforts to find treatments. I thank him for sharing the experience of his constituent, Wendy.
I very much recognise the challenges faced by people and families affected by epidermolysis bullosa, which as he rightly pointed out is a rare disease. It is estimated to be diagnosed in one in 17,000 babies born in the UK. Rare diseases are defined as those affecting fewer than one in 2,000 people and, although they are individually rare, sadly, these conditions are collectively all too common. One in 17 people will be affected by a rare disease at some point in their lifetime. In the UK, that amounts to more than 3.5 million people. It is therefore vital that these people have access to the right care, the right treatments and the right support.
I turn to the rare disease strategy. The “UK Rare Diseases Framework”, published in 2021, embodies our commitment to securing a better future for all people living with rare diseases. It sets out our vision on how to improve the lives of people with rare diseases through four vital priorities. They are helping patients to get a final diagnosis faster; increasing awareness among healthcare professionals; better co-ordination of care; and improving access to specialist care, treatments and drugs. To deliver on the Government’s ambition, all four nations have published rare diseases action plans, which set out our tailored approaches to deliver the aims of the framework in ways that are most effective for each nation’s populations and healthcare systems. In England, we published the second rare diseases action plan on 28 February, in which we set out 13 new actions to drive improvements across the health system.
My hon. Friend rightly pointed to research funding. The UK is internationally recognised for our leadership in research, the excellence of our scientific institutions and our fantastic healthcare system. We must continue to utilise those resources to benefit those affected by rare conditions. The Government are committed to increase spending on research to £22 billion by 2026-27, moving towards our target of investing 2.4% of GDP in research and development by 2027. Alongside industry and medical research charities, the Government primarily fund research into rare conditions such as EB via UK Research and Innovation and the National Institute for Health and Care Research.
Through the NIHR, the Department of Health and Social Care invests over £1 billion a year to fund, enable and deliver world-leading health and social care research. Pioneering research is a cross-cutting theme of the rare diseases framework. We recently announced significant new funding of over £12 million via the Medical Research Council and NIHR for a rare disease research platform. The hon. Member for Strangford (Jim Shannon) rightly referred to the importance of Government-backed research funding. Since 2019, the NIHR has funded three studies specifically into EB, with a total award value of over £4 million, and we have supported the delivery of more than 25 studies.
Research is vital—that has been well articulated today—but treatment is, too. I agree with my hon. Friend the Member for Bracknell (James Sunderland) that therapeutic treatments are also a part of the solution and are absolutely key to improving the quality of life for EB patients. As my hon. Friend the Member for Orpington said, unfortunately there is no cure for EB. However, as he also rightly pointed out and I am very pleased to say, the National Institute for Health and Care Excellence is currently evaluating two treatments: birch bark extract, as he pointed out, for treating skin wounds; and the gene therapy—I am afraid I am will not make a much better job of pronouncing this than him—beremagene geperpavec. I apologise to all medics and research professionals for that pronunciation; I am not a medic. If either of those treatments is given a positive recommendation by NICE, NHS England will ensure that service provision is in place to deliver it into the hands of those affected by EB.
My hon. Friend also rightly pointed out the drug repurposing process, which could be absolutely life-changing for people with EB. There is substantial interest in repurposing existing medicines to treat rare diseases, as repurposing is often quicker and less costly than developing new medicines. Our medicines repurposing programme identifies and progresses opportunities to use existing medicines in new ways that are not included in the current licence, with the aim of improving clinical outcomes, patient experience and, also importantly, value for money. As one approach to identifying candidate medicines for repurposing—my hon. Friend touched on this—the NIHR has an innovation observatory, which searches for suitable clinical trials nearing completion. That routine scanning has identified a French trial investigating the use of—I again apologise for my pronunciation, Madam Deputy Speaker—ixekizumab, a licenced treatment for other conditions such as psoriasis, for simplex generalised severe EB. The medicines repurposing programme is monitoring the study and will use the results when available to assess whether the drug is a suitable candidate for the programme.
My hon. Friend mentioned the MRP and evidence of efficacy. I want to clarify that for a medicine to enter the MRP programme there has to be some evidence of efficacy and safety, but—this is the important point—conclusive proof of efficacy and safety is not required. Projects that need a further clinical trial are potentially eligible to enter the programme, at which point we would liaise with the NIHR about trial funding.
My hon. Friend’s first specific ask was on funding. This is always the difficult bit, because it would be easy to say yes. He makes a compelling case, but I do not think he would expect me—nor would it be appropriate—to commit to funding at the Dispatch Box, though his point was very well made. I will look into it carefully and discuss it with officials, and I will be happy to meet him.
I re-emphasise that the Government very much encourage healthcare professionals, voluntary sector organisations—some of which my hon. Friend mentioned—and companies to propose candidate medicines for the medicines repurposing programme. The details and eligibility criteria are available on the NHS England website. As I said, I would be happy to meet my hon. Friend to discuss that further, which was his second request. Further to that, my understanding is that the medicines repurposing programme has invited the EB charities DEBRA and Cure EB to meetings to discuss specifically the repurposing of medicines for EB. The timing of this debate could not be more spot on, as I believe the DEBRA meeting is scheduled for tomorrow. Those meetings are happening, and I would happy to meet my hon. Friend. He certainly has his finger on the pulse.
If I am available I would be happy to do that. I thank my hon. Friend for his support for that charity, which is based in his constituency but works nationwide. The support that constituency Members of Parliament provide to charities through this place should not be underestimated. If I am able to attend, I will.
When we spoke earlier, I was quite sure that the Minister is truly a compassionate man. He has given us the answers that we wish to hear, and I thank him for that. He talks about the possible cures for EB. Will that information be shared with all devolved Administrations? I think he said that it would be, but I wanted to check.
Absolutely. We work on clinical and medical research across our United Kingdom, and rightly so. We work very closely across all four nations.
I am conscious of time, and I want to pick up on the final question from my hon. Friend the Member for Orpington about generic versus branded medicine and the MRP. Branded medicines are potentially eligible for the medicines repurposing programme. I understand that the eligibility criteria state that the programme can support generic, biosimilar and branded medicine. I would be happy to look at my hon. Friend’s list.
In closing, I give my thanks again to my hon. Friend for securing today’s important debate, and to all Members who have contributed. I also pay tribute to the whole EB community and charities such DEBRA and Cure EB, which I know work tirelessly to improve the lives of people affected by the condition. They are fortunate to have my hon. Friend in this place as their champion.
Question put and agreed to.