Spinal Muscular Atrophy

Seema Kennedy Excerpts

Thursday 25th July 2019 Commons Chamber

[The Parliamentary Under-Secretary of State for Health and Social Care (Seema Kennedy)]

What a pleasure it is to speak in the final parliamentary debate of what has been an historic and tumultuous term for all of us.

I congratulate my right hon. Friend the Member for South Holland and The Deepings (Sir John Hayes) on securing the debate on such a timely day, because we have had news on Spinraza in the last 24 hours. He outlined very movingly the symptoms of spinal muscular atrophy, which has been raised before in this House. The hon. Members for Bristol East (Kerry McCarthy), for York Central (Rachael Maskell) and for North Tyneside (Mary Glindon) are particular champions for sufferers.

SMA is a terrible disease that can have a terrible impact on the children who suffer from it, their families and the wider community. We in the Department know how important it is for them to be able to access effective treatment. It is a rare disease. It affects about 100 babies born each year. It is estimated that it affects between 1,200 and 2,500 children and adults in the UK. Until recently, there were no licensed treatments for it, so the condition has been managed using nutritional support, physiotherapy and social care.

Spinraza, as my right hon. Friend outlined, has been licensed across Europe for the treatment of SMA since 2017 and is very effective. However, it is extremely expensive and has been undergoing assessment by NICE to determine whether it would be an effective use of NHS resources. I say to him that QALYs do take account of wider costs—not just physical costs but social care costs. As far as I am aware, there is currently no difference in the quantitative evidence. He gave the examples of a man in his mid years—at 60—and a child.

I will go on to talk more broadly about the NICE process, but I am really pleased that only yesterday, NICE published its final guidance, meaning that this debate is very timely. The guidance recommends the use of Spinraza for many patients with SMA through a managed access agreement, which has been negotiated between NICE, NHS England and the manufacturer, Biogen. This makes Spinraza available to the NHS at a discounted price. Importantly, it also sets out arrangements for collecting evidence on the long-term impact of Spinraza, because at the moment the evidence is quite immature. The managed access agreement is one of the most comprehensive deals in the entire world and it brings Spinraza to one of the widest cohorts of eligible patients in any country. It was signed and supported by Spinal Muscular Atrophy UK, Muscular Dystrophy UK and TreatSMA. I put on the record my thanks and my tribute to all those organisations for the support they have given patients and families with the illness. That very welcome development has involved hard work and flexibility from all parties to reach this point; if only all parties in all the debates I have to speak in on this matter were as flexible.

[Sir John Hayes]

The Minister makes a good point about the managed access agreement and the amendments to it that she described. But the real issue here is whether Spinraza would benefit every single sufferer. If it would, it should be universally available.

[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.