Spinal Muscular Atrophy — House of Commons, 2019-07-25
Topic: Health
John Henry Hayes (Conservative, Centre-right): 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.