Duchenne Muscular Dystrophy

Maya Ellis Excerpts

Thursday 12th June 2025 Commons Chamber

[Chris McDonald]

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.

[Maya Ellis (Ribble Valley) (Lab)]

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?

[Chris McDonald]

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.