NICE Appraisals: Rare Diseases Treatments

Neil O'Brien Excerpts

Thursday 21st March 2019 Commons Chamber

[Lyn Brown (West Ham) (Lab)]

I am sure hon. Members will not be surprised to hear me talk again about baby Maryam, my seven-month-old constituent with spinal muscular atrophy type 1. Without treatment, Maryam will have only a few more months to live. She is waiting for NICE to announce a decision to allow access to Spinraza, the drug that she and other babies with the condition so desperately need.

I start by thanking my hon. Friend the Member for Blaydon (Liz Twist) for securing this important debate; the Daily Mirror, especially Pippa Crerar, for telling the stories of SMA families; the Newham Recorder, the campaigning newspaper, for its continued coverage and encouragement; and the amazing members of the local community in West Ham for their support. Councillor Mas Patel has been brilliant, as has Kevin White and many others who have taken the plight of this little baby into their hearts.

Most of all, I pay tribute to Shakil, Maryam’s father, and all Maryam’s family who have steadfastly campaigned despite knowing that, when we finally get access to Spinraza in England, it could so easily be too late for Maryam.

As we know, SMA1 is an awful disease. It is a progressive muscle-wasting condition, and for a baby it causes difficulties with breathing, swallowing and gaining weight. Babies with SMA1 may not even be able to cry aloud. Spinraza is an effective treatment for SMA. With this treatment, babies and children are living longer; they are even crawling and walking. I am told that by last August, when NICE first met to consider funding Spinraza on the NHS, none of the babies who had been treated with it in the UK had died as a direct result of their SMA. The drug was so effective that the trials stopped early, yet NICE refused to approve Spinraza at that August meeting, or at the second meeting in October or in November.

In November, the expanded access programme, which allowed treatment with Spinraza while negotiations were ongoing, was closed—Biogen, the drug company that developed Spinraza, closed it. So when Maryam was diagnosed in December last year, she had no access to this drug. Even if it could have been afforded privately and even if my community in West Ham had fundraised for it—I can tell Members that an awful lot of people have wanted to do just that—she could not have got it. We are not talking about huge numbers; we are talking about fewer than 100 babies a year. Treating this condition should not break our bank—it could not, surely.

NICE met again two weeks ago—we held a vigil while it was making its decision—but I am told it could be three or more weeks yet before that decision is known. This is callous. Making families, making Maryam and making the other babies wait this long is callous. The NICE approvals process for Spinraza has now taken 14 months. That is longer than many of the babies with SMA1 can be expected to live without the treatment. At the same time, agreements have been reached on Spinraza in the United States and in 25 European countries, including Scotland, Germany, Italy, Lithuania and Romania.

NICE has failed Maryam and all the other babies with SMA, but others are culpable, too. The closure of the access programme no doubt saved Biogen some money, but I am sure that it was closed to increase pressure on NICE as well. I cannot say that that is definitely true, but what I will say is that if it is true, the suffering of tiny babies, and the pain and suffering of their families, too, has been used in the service and pursuit of profits. I now believe that our whole system of for-profit medical research combined with public healthcare encourages this kind of brinksmanship, it encourages delaying tactics and it encourages the exploitation of sick children and their families.

Let us also consider the actions or inaction of the Government. I am sure hon. Members will recall how in the spring statement the Chancellor talked of a

“proud, successful, outward-looking nation, with no limit to our ambition and no boundaries to what we can achieve.”—[Official Report, 13 March 2019; Vol. 656, c. 352.]

He bragged about

“the single largest cash commitment ever made by a peacetime British Government”.—[Official Report, 13 March 2019; Vol. 656, c. 346.]

This was for the NHS. Surely we can afford to help these babies, too. Currently, we are the fifth richest country in the world, yet we are withholding vital medical treatment from babies. This is medicine that 25 other European countries, including some much poorer than we are, are willing to provide. What does that say about us? What does that say about our values? What kind of a country have we become?

I have asked the Prime Minister to act—twice. I have asked the Health Secretary to act. As far as I can see, nothing has happened. I hope that Conservative Members will forgive me when I say that I thought the Prime Minister’s response to me last week was cold; it was more about defending an outdated process than about understanding and empathising with the desperate plight of Maryam and her family. Let us remember that 25 European countries already have access to this drug, We do not, yet it seems to be nothing to do with the Prime Minister.

[Neil O’Brien (Harborough) (Con)]

The hon. Lady is making a good and powerful speech. Two of my constituents, Mark and Panna Wilson, are in a situation similar to that of her constituents: their son Aadi is only three years old and is regressing every day because he, too, has SMA, which is a terrible condition. They told me that

“without access to new treatments, each and every day is a step, not towards an exciting future, but towards a painful and dark place.”

The hon. Lady is right, and I hope that we will find a new route to the approval of Spinraza in this country. The route it is currently going through is intended for much more generic medicines and it has not been put through the route for rare medicines. We need either to put it through the route for rare medicines or to find or invent a new mechanism so that we can get treatment for these people. As she points out, this is a rich country: we must be able to save these babies and toddlers.

[Lyn Brown]

The hon. Gentleman is absolutely right. This is a political issue—what we are discussing today is political. It is about which of our constituents get access to which medicines, and when; who our public healthcare service helps with our public money; and who we allow to die without help.

Members on both sides of the Chamber have recently urged the Government to use their Crown use licensing powers in the case of Orkambi. Those powers can get around medicine patents when pharmaceutical companies are simply refusing to deal honestly with the NHS, and they are an option. There are always options for the Government if they wish to use them, but they seem to be content with voicing frustration. They do not do anything—they will not even threaten to do anything that might help. If the Patents Act 2004 is not working, as Ministers have said, we need to do something to replace it. Maryam’s family and others want action now. They need Spinraza now; when will they get it?