Batten Disease

Part of the debate – in the House of Commons at 4:10 pm on 22 July 2019.

Alert me about debates like this

Photo of Dr Caroline Johnson Dr Caroline Johnson Conservative, Sleaford and North Hykeham 4:10, 22 July 2019

I have looked after a number of children with Batten disease in my career, and no one should underestimate the horrific nature of this condition with which a child develops apparently normally and then gets the horrific diagnosis that they will suffer neurodegeneration. I completely respect the importance of NICE being independent, and in general I do not get involved in these debates, but I believe I should do so in this one, because I actually think that NICE has this wrong. This drug does not make a little bit of difference—it does not have the effect of making someone die a couple of weeks later; it makes a phenomenal difference to the quality of life for these children. Yes, the trials have been short so far, but over a reasonable period it makes a massive difference, and I think we should do everything we can. I have heard the Minister say that she will ask the chief executive of NHS England to get BioMarin back round the table. How long will she give him to achieve that, and if he does not succeed, what will she herself do to ensure that these children get these drugs as soon as possible?