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Translarna (Duchenne Muscular Dystrophy)

Volume 596: debated on Tuesday 2 June 2015

2. When he expects NHS England to reach a decision on access to Translarna for the treatment of Duchenne muscular dystrophy; and if he will make a statement. (900001)

11. When he expects NHS England to reach a decision on access to Translarna for the treatment of Duchenne muscular dystrophy; and if he will make a statement. (900011)

NHS England is considering the interim commissioning position for Translarna as part of its wider prioritisation process for funding in 2015-16 and expects to come to a decision by the end of this month. Translarna has also been referred for evaluation by the National Institute for Health and Care Excellence’s highly specialised technologies programme. Draft NICE guidance will be available later this year, with final guidance expected in February 2016.

I thank the Minister for that response and welcome him to his place. Yesterday my constituent Jules Geary came to see me regarding her son Jagger, who suffers from Duchenne muscular dystrophy. Jagger had been approved for Translarna treatment but then suddenly found that it had been withdrawn at the last moment. Like many other boys, he is now waiting, not knowing when a treatment that will prolong his mobility will be forthcoming. Will my hon. Friend meet me, Jules and Muscular Dystrophy UK to discuss how this process can be streamlined so that other children do not have to wait this long?

Muscular dystrophy is a terrible, debilitating illness and my sympathies go out to Jagger and his family. My hon. Friend will be aware that families and their representatives will be going to Downing Street on 10 June to make their representations on this matter. The Minister for Life Sciences has introduced an accelerated access review precisely because of the concerns that my hon. Friend has raised, and I know that he will welcome representations once it has been completed.

Is the Minister aware of the case of my constituent, little George Pegg? At one time he could not walk, but this drug has made his life 100% better and he can now walk. Why are we dithering? This has been going on for at least a year, so why don’t you get off that backside of yours and get it approved?

Order. May I just exhort Members to have some regard to considerations of taste? This is a new Minister. I call Minister Gummer.

I thank the hon. Gentleman for his question. In relation to posteriors, it is good to see his in its rightful place. I have heard of his constituent’s case, which is as distressing as that of Jagger and of all those suffering from Duchenne muscular dystrophy. It is a terrible disease that causes lasting pain to the sufferers and their families. That is precisely why we are pushing hard for a decision from NHS England by the end of this month—it could not have come as quick as he had hoped—and for interim NICE guidance by the end of this year. I am pushing officials to move as quickly as they can on this.

The reality is that NHS England has failed to respond to letters or to turn up for meetings, and it has behaved in an utterly unaccountable manner in regard not only to Translarna but to Vimizim, which is used to treat Morquio syndrome. We have still not had confirmation that an interim decision will be made on 25 June, but we are now being told that there will be a decision from NICE on 5 June. Will Ministers finally get a grip on this and give the families affected by these various conditions some sense of when they might get the treatment that could improve their quality of life?

I am sorry to hear that the hon. Gentleman has had that experience with NHS England. My hon. Friend the Minister for Life Sciences will want to speak to him about that; if it is the case, it is clearly unacceptable. As the hon. Gentleman will have heard from my previous answer, we are hoping to get quick decisions from NHS England on the interim commissioning guidance this month, and I am pushing hard for a decision from NICE as soon as possible this year, so that we can get interim guidance from it.