Batten Disease: Access to Drugs

Debate between Andrew Griffiths and Jacob Rees-Mogg

Tuesday 16th July 2019 Commons Chamber

[Mr Jacob Rees-Mogg (North East Somerset) (Con)]

Mr Speaker, may I begin by thanking you for allowing me this Adjournment debate and for your personal encouragement to me to bring it forward? My gratitude goes beyond that; I also thank you for the way you so encourage this House to hold the Government—the Executive: those who rule us—to account.

I am also grateful to the hon. Member for Newcastle upon Tyne North (Catherine McKinnell) and the terrific campaign she has been running in relation to Batten disease; she has done more than almost anybody else to highlight it. I note that two of my hon. Friends on the Front Bench, my hon. Friends the Members for Macclesfield (David Rutley) and for Pudsey (Stuart Andrew), have a great interest and a constituency concern in this issue, although they are not allowed to intervene on me for obvious reasons. I want to put their concern on the record. I know that others too might wish to intervene on this very important subject.

This evening’s debate is about my constituent, Max. Max is a little boy; he is eight years old. He is a lively boy, and those of us who have children know what eight-year-olds are like—what a joy they are and how wonderful their spirit is. But Max has this horrible disease. Batten disease is perhaps the cruellest disease that one can imagine as a parent. We all see our children grow; we see them learn to walk and then to talk, and to run and to do all the things that children do. Batten disease means that they then go backwards. It tends to hit at about two—on a child who has shown no signs until then. The talking stops and the walking becomes more difficult. The average life expectancy of a child with Batten disease is between six and 12.

The blow to parents, and to grandparents and families, that this is and must be, is so hard to bear and so difficult; it is so sad for them to see a child who they would hope would be going on into adulthood instead declining, and declining steadily. It is a neurodegenerative disease. To put it in layman’s terms, it is essentially dementia of the young: all that we see of Alzheimer’s in people in their 70s, 80s and 90s is instead happening to a child.

But there is a drug that delays this. It is not a cure and it does not reverse the disease, but it seems to stop its progression—nobody knows for how long. It is called Brineura and it has been shown to be effective on a number of children who have taken it. So far in this country there are only two children for whom it would be suitable who are not receiving it, one of whom is Max. The others are receiving it as part of a trial that has been successful and is still being funded by the drug company—but that might not continue for ever, so there is an argument for them as well. I mentioned earlier the enormous contribution that the hon. Member for Newcastle upon Tyne North has made, and I am very glad to see her in her place; without her, I do not think this really important matter would have achieved the publicity that it has received.

This drug Brineura has been given a quality-adjusted life years rating by the National Institute for Health and Care Excellence of 30, which is the highest rating that it gives. That means that the drug is thought to provide 30 extra years of life of good quality. That is a stunning achievement for any drug, and it has been given the highest rating and the highest amount of funding, but unfortunately that amount of funding is not enough. The pricing cannot be agreed between NHS England, NICE and BioMarin, the manufacturer.

BioMarin is a drug company that needs to make a return on the amount of money it has spent. To be fair to the company, it spent $696 million last year on research and development and made a pre-tax loss of $142 million, so it is not an enormously profitable, rapacious company that is being difficult. One might think, however, that having lost $142 million, it might quite welcome a little bit of income from the national health service. If I were one of its shareholders, I might suggest that it would be a good idea to do something with the national health service so that the company could get some income back on its $696 million of research and development expenditure in 2018. Without an agreement between the buyers and sellers, Max will not receive the drug and his standard of life will decline month by month.

[Andrew Griffiths (Burton) (Con)]

My hon. Friend will know about my constituent, Michal Luc, who is in exactly the same situation. We talk to parents who see their children degenerating and dying before their eyes. Does he agree that they cannot understand how we can argue over money when their children’s lives are disappearing before their very eyes?

[Mr Rees-Mogg]

I completely agree with my hon. Friend.

Generally, I recognise the need for public expenditure constraint. Money always has to come from somewhere; it has to be either taxed or borrowed. However, in a country that spends over £800 billion a year, and £120 billion or whatever it is a year on the national health service, can we not find just over £6 million a year for this small number of children who have a terrible disease that can be held at bay?