Spinal Muscular Atrophy

John Hayes Excerpts

Thursday 25th July 2019 Commons Chamber

[Sir John Hayes (South Holland and The Deepings) (Con)]

There is no fairness to fate, no right to good fortune: new life is a miracle, and great health is a blessing.

I do not normally speak from a text, as you know, Madam Deputy Speaker, having known me since we both entered the House in 1997, but the subject which I wish to address today is technical and medical, and is highly significant, and given that the Health Department is one of the few Departments in which I have not served as a Minister, I thought I had better stick to a script.

Spinal muscular atrophy is a genetically inherited neuromuscular condition which results in the irreversible damage of motor neurons and progressive muscle wasting. It is a complicated and often debilitating disease linked primarily to genetic mutilation in the SMN1—survival motor neuron—gene.

There are three types of SMA. The age of onset and severity of symptoms can vary considerably. Many sufferers will experience extensive disability, and without early intervention numerous lives are damaged and, indeed, cut tragically short.

I know all of this because of my young constituent, 12-year-old Rae White. But there is hope on the horizon for Rae and other sufferers, as the first dedicated treatment, Spinraza, offers hundreds the possibility of an improved quality of life. Clinical trials have already demonstrated the enormously exciting potential benefits of the drug, delivering meaningful improvements to the motor neuron function of patients.

While individuals with SMA have the SMN2 gene, they lack the SMN1 gene, leaving them unable to produce the full-length proteins that work effectively within the body. Spinraza has been developed to address the absence of this survival motor neuron protein. Its synthetic antisense oligonucleotide acts as an enabler, allowing the SMN2 gene to produce full-length protein, thereby alleviating the symptoms of SMA.

Thankfully, the administration of Spinraza is via an already established procedure known as intrathecal injection, an injection into the spinal canal. This procedure is always performed under the direction of a healthcare provider experienced in administering lumbar punctures, with patients sedated or under a general anaesthetic.

The promise of this drug and its potential benefits to those who suffer should not be underestimated. Families of children who have received the drug often report noticeable profound improvements from the very beginning of treatment. During the clinical trials, monitoring committees on rare occasions say it is no longer ethical to treat people with a placebo, and such was the case with this particular drug. It proved so effective in clinical trials on children with SMA type 1 that the trials were stopped early to enable all the children to access the drug as quickly as possible.

Positive signs have also been observed during trials for treatment of other types of SMA, resulting in significant improvements in motor function in children with SMA type 2 and type 3. In April 2019, a three-year study of 28 children aged between two and 15 showed remarkably improved outcomes compared with the natural progression of the condition if left untreated. In particular, a two-year-old child with SMA type 2 gained the ability to walk independently after receiving Spinraza, and two children with SMA type 3 who had lost the ability to walk before taking the drug regained it during the study. One can only imagine the value of the restoration of ability for those children and the joy for those who love them.

Moreover, a clinical trial is ongoing in pre-symptomatic infants with genetically diagnosed SMA who are considered likely to develop SMA type 1 or 2. As of July 2019, children treated with Spinraza continue to achieve motor milestones that are unprecedented in the history of the condition, including 100% of children sitting without support and 88% walking independently. Crucially, trials have shown that the earlier the patients start the treatment, the greater the benefit.

All this considered, it is with much sadness that we must reflect on the less than desirable state of play now. Not all those who suffer from SMA have been able to access this pioneering treatment. Indeed, there has been a great deal of confusion surrounding eligibility. My colleagues, my hon. Friend the Member for North East Somerset (Mr Rees-Mogg) and the hon. Members for Wolverhampton South West (Eleanor Smith) and for Bristol East (Kerry McCarthy), have raised touching testimonies from their own constituents who are facing similar challenges to those of Rae White. I was touched when young Rae’s mother Tanya came to meet me and offered the moving description of her elation at the original announcement by the National Institute for Health and Care Excellence—NICE—and NHS England, promising that Spinraza would be made available to all.

Perhaps it would be helpful at this juncture if I said a word about how new medicines are approved. There are two stages to the approval process. First, the Medicines and Healthcare Products Regulatory Agency—MHRA—tests their quality, safety and efficacy. The quality is tested in respect of a medicine’s purity; the safety in respect of possible side effects; and the efficacy determines whether it does what it is supposed to do. This is a well-established 50-year process that is recognised as robust and reliable. Subsequently, in a much newer process, NICE examines in tightly populated clinical conditions the cost-effectiveness of making the drug available to a wider population of relevant sufferers. This is, in essence, a matter of health economics, with NICE measuring the capacity of the drug in question to add what are called “quality-adjusted life years”.

The problem with that process is that the formula is too crude to effectively distinguish, in what I might call human or social terms, between treating a 12-year-old girl like Rae or a 60-year-old man like me. In practice, efforts end with a balance being struck between efficacy and cost-effectiveness in the form of a managed access agreement. Indeed, in the case of Spinraza, such a managed access agreement was released, revealing that potential patients would have to fulfil a set of access criteria in order to get this life-changing treatment. As we can imagine, that caused panic and pain for those families who, having had their hopes raised, then had them dashed as they realised that many would not in fact be given access to the treatment.

[Kerry McCarthy (Bristol East) (Lab)]

It is just so cruel. There was a party to celebrate the fact that my 18-year-old constituent Jake Ogborne would have access to Spinraza. There was no mention of eligibility criteria, but suddenly he was told that he would not be eligible because he had not been able to walk in the past 12 months. He lost his ability to walk about 18 months or two years ago. I raised this in Prime Minister’s questions. I got a response that basically told me what we already knew and just set out the rules on this. It is immensely cruel for an 18-year-old to be faced with a decision like that.

[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.

[Rachael Maskell (York Central) (Lab/Co-op)]

I am grateful to the right hon. Gentleman for introducing today’s debate. We have had many debates in the House on the procurement of drugs. I have been working with health economists at the University of York who are leading in this field. They very much recommend the model now being adopted by Canada around a national rebate scheme, which takes away some of the tension over cost that we seem to return to time and again. Is not that a way forward that the Government should at least explore?

[Sir John Hayes]

The hon. Lady had the great pleasure of shadowing me when I was at the Department for Transport, and I have had the greater pleasure of listening to her on so many subjects. She speaks with such knowledge, understanding and wisdom. Once again, she has shown all those things today.

The Government and the new Prime Minister must do as much as they can to ensure that those with rare diseases have every chance possible to live the very best lives they can. So, here is exactly what to do: first, as Spinraza has been shown to be both safe and effective, the NHS should provide the treatment for all those who would benefit from it; secondly, in addition to making Spinraza available for all, the Minister should set up a rare drugs fund, similar to the successful cancer fund, to ensure that those battling debilitating degenerative diseases are supported at every stage of their journey; and thirdly, I ask the Minister to implement an immediate review of the criteria used by NICE to determine access to new medicines.

We all want to do the right thing. I described earlier what I said was a crude approach. These things develop; they metamorphose. This is a chance to look again at how we can administer treatment to have the best effect on those in the greatest need. Hegel said:

“Life has a value only when it has something valuable as its object.”

Chamberlain said:

“In great deeds something abides.”

There is no better great deed, no more noble object, no more abiding purpose than the care for those in the greatest need. I ask this Minister to make her abiding object a war on want, a campaign against suffering, a crusade for those in pain—Madam Deputy Speaker, nothing less will do.

[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.