Spinal Muscular Atrophy

Mary Glindon Excerpts

Thursday 25th July 2019 Commons Chamber

[Sir John Hayes]

Yes. The condition, as the hon. Lady suggests, is often degenerative. In cases such as the one she describes, which, as I mentioned, is not the first time she has raised it, a young person who would normally develop as we all did could be left with arrested development or, even worse, declining capabilities. Indeed, that has happened to my constituent too.

The hon. Lady will know that, following strong advocacy by families of SMA patients, Muscular Dystrophy UK, TreatSMA, Spinal Muscular Atrophy UK and many clinicians, NICE and NHS England made amendments to the managed access agreement. While amendments are far from unwelcome, the disappointing truth is that the new criteria will still exclude some SMA patients desperate for treatment in the way she and others have articulated.

The intensely difficult battle fought by SMA patients has highlighted deeper flaws in the system. Families report feeling that they have been pitted one against another as advocacy groups are forced to decide whether to push for wider accessibility, and as a consequence risk delaying treatment for those eligible, or, alternatively, to take what is on offer and exclude a minority of the SMA family.

Life can be intensely difficult. All our bodies are complicated and vulnerable, intricate and fragile. We are regularly reminded, are we not, that they can go wrong in a multitude of hard-to-understand ways. I have argued many times in this House that a society should be gauged by how it cares for, protects and promotes the interests of its most vulnerable members.

[Mary Glindon (North Tyneside) (Lab)]

I congratulate the right hon. Gentleman on securing this important debate. I, too, have a young constituent, Sam McKie, who has SMA type 2. I hope the right hon. Gentleman agrees that the issue is that, whatever has happened so far, we need to know that NICE will look at the fact that the drug can halt further deterioration. As he says, we are all weak mortals, but if that deterioration is halted, people can have some quality of life, whatever type of SMA they have.

[Sir John Hayes]

Exactly. That is why, in the exciting conclusion to this speech, I shall make demanding suggestions. I think they are demanding because of the demands of those who need this drug, not because of any particular interest I might have in this matter beyond a passion to ensure that my constituent and others like her get what they need so desperately.

--- Later in debate ---

[Seema Kennedy]

I know that there is an issue with some people suffering from SMA type 3, but I am not the person who would make the decision on whether Spinraza was effective or not; that is why NICE is there.

Children with the most severe form of SMA type 1 are already benefiting from treatment with Spinraza. Following yesterday’s announcement, eligible patients with types 2 and 3 will begin treatment as soon as possible within the next three months.

NICE has concluded that there is not sufficient evidence at this stage for the managed access agreement to cover some patients with SMA type 3 or any patients with type 4; that is why we are still carrying on collecting evidence. I realise how dreadfully painful that is for those not able to access Spinraza. The majority of patients will be eligible under the managed access agreement.

The parties to the agreement have agreed to keep the eligibility criteria for treatment under review during the five-year term of the agreement, so those criteria may be further extended in future if more evidence of benefit emerges over time. NHS England and I have acknowledged that the inclusion criteria associated with the managed access agreement could have been more clearly communicated when it was announced.

I turn to the NICE process altogether. This is an important system. It makes independent, authoritative, evidence-based decisions, which is essential so that taxpayers can be assured that the price we pay for new medicines reflects the value that they bring. It also helps ensure rapid access to effective new treatments for NHS patients. It has been going for 20 years and is internationally respected. There are both established and new pharmaceutical companies developing medicines for rare diseases, which takes an awfully long time, based here in the UK, and medicines can be brought to the market through the NHS very quickly. The Department is keen to press on with that.

We have a UK rare diseases strategy, which was set out in the NHS long-term plan. Genomics is a particularly important area, in which we want to innovate so that we have more comprehensive and precise diagnoses and allow patients to access the right drugs. We are committed to that. The NICE process has recommended more than 80% of the medicines appraised and 75% of medicines for rare diseases for some or all of the eligible patient population, but of course the processes must evolve. They have to keep going, taking into consideration developments in science, healthcare and the life sciences sector. That is why NICE keeps its methods and processes updated through periodic review, which includes extensive engagement with stakeholders, including patient representatives, drug manufacturers and clinicians.

[Mary Glindon]

Has the Department given any consideration to the MAP BioPharma report released earlier this year, which looked at how the appraisal process could make a fairer playing field for rare diseases?

[Seema Kennedy]

Yes, the Department has looked at that report. NICE recently initiated a review, and I assure Members—as I did when I answered the urgent question on Tuesday—that it will be wide-ranging and carried out with extensive engagement with stakeholders. I shall be keeping a close eye on it if I remain in this job—and even if I do not.

This has been an important debate. I thank my right hon. Friend the Member for South Holland and The Deepings, who always speaks so passionately on behalf of people who are sick and on behalf of the most vulnerable—those children who have no voice for themselves. I reassure Members that access to effective new treatments will always be a priority for the Government.

I thank you, Madam Deputy Speaker, for your service to us in the Chair, and wish all right hon. and hon. Members and all who work here in the Palace of Westminster a very revivifying recess.

Question put and agreed to.