Epidermolysis Bullosa: Drug Repurposing Trials

Wera Hobhouse Excerpts

Tuesday 9th May 2023 Commons Chamber

[Gareth Bacon]

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.

[Wera Hobhouse (Bath) (LD)]

Does the hon. Gentleman agree that the problem with skin conditions, particularly rare conditions, is that people are also embarrassed and want to hide them, which adds insult to injury?

[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.