Epidermolysis Bullosa: Drug Repurposing Trials

James Sunderland Excerpts

Tuesday 9th May 2023 Commons Chamber

[Gareth Bacon]

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.

[James Sunderland (Bracknell) (Con)]

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?

[Gareth Bacon]

I am extremely grateful to my hon. Friend for his intervention and for hosting a reception for EB and for DEBRA here in June, I believe.

[James Sunderland]

It is on 19 June.

[Gareth Bacon]

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?

[The Minister for Health and Secondary Care (Will Quince)]

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.

[James Sunderland]

Since the Minister is clearly on a roll, if he is available will he commit to come to the DEBRA reception here on 19 June?

[Will Quince]

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.