In the draft guidance issued today, the National Institute of Health and Care Excellence (NICE) decided NOT to recommend the use of nusinersen (Spinraza®) as an NHS treatment for spinal muscular atrophy in England and Wales.
This is a black day for more than a thousand people affected by spinal muscular atrophy in our country. Nusinersen (Spinraza®) is the only approved treatment that brings about a meaningful change in the lives of those affected by SMA. Thousands of children globally owe their lives to this treatment. Thousands more – children and adults alike – have regained their muscle function when the disease course was reversed with Spinraza treatment.
Nusinersen therapy has been approved and is available to SMA patients across most of the developed world as well as in a number of developing countries. To-date, more than 5,000 people with this debilitating rare disease had their lives saved and disease course reversed. Currently, twenty-two countries of the European Union provide Spinraza. Regretfully, England and Wales is poised to become the only country in Europe where the health authorities have refused to offer this life-saving treatment even to small babies who die within the first years of life if left untreated.
The draft appraisal along with accompanying documents is available on the NICE website.
Representing the voice of the UK SMA Community, we at TreatSMA have continuously advocated that the treatment must be made available for all those with SMA regardless of age, genetic makeup, disease “type” or other criteria. In our contacts with the decision-makers we challenged the prevailing notion that Spinraza should be appraised only as a highly specialised therapy for those with SMA type 1 alone. We stressed that it should be reviewed as a treatment for all with SMA. We have been successful: the drug was appraised as a treatment across the entire spectrum of spinal muscular atrophy.
We have then spared no effort to influence the process, so that effective treatments for SMA are available to everyone who needs them. Thanks to the work of countless people – parents of SMA children, those with SMA, and medical experts – we submitted the Community’s views on the treatment to NICE; they can be read on the NICE website. We also submitted reliable real-world evidence on long-term effects of nusinersen treatment in many categories of patients. This data, gathered by prominent clinicians and SMA experts worldwide and often published in respectable academic journals, indicated that the treatment is exceptionally effective across a wide spectrum of SMA.
We are bitterly disappointed that the NICE Appraisal Committee has failed to give due weight to the studies and instead appears to have relied in their analysis on the few randomised controlled trials of a limited duration which were all conducted years ago.
What’s next?
As an official Consultee in the nusinersen appraisal process, we have been requested to comment on the draft guidance. We are currently working on the document that will outline our objections. We intend to challenge the choice of evidence as well as several other points in the document that we have found contentious.
Nevertheless, in line with our earlier discussions, NICE has left open a possibility for NHS to provided nusinersen under a separate agreement with the manufacturer – a so-called Managed Access Agreement. We expect Biogen to propose an MAA to NHS within the next weeks.
Judging from past cases, it may take long time – months or years – before an MAA is finalised. We intend to press all the stakeholders so that the Agreement is signed without delay. As always, we will continue advocating that no person with SMA is excluded from treatment irrespective of age, SMA type, functional ability or other such arbitrary criteria.
What is important is the fact that the treatment was viewed as effective by clinicians.
This is not a disaster but a warning that we must stand firm. TreatSMA will be at the forefront of the process, as always. We will leave no stone unturned to move things forward, and we will keep you posted on the progress.
What you can do
- Read the draft appraisal document yourself. It is available here.
Tell us now how you feel. Vent out your anger and frustration. Cry. We all cried when we learned about the outcome. There is a perfectly valid reason. Share your feelings in Comments below or on the social media under our post. It is crucial. We at TreatSMA will need to read them all and convey their content to the decision makers. Let them understand how we the SMA Community see their decision.
If you want to share your thoughts anonymously and/or in more detail, click here for the appraisal feedback form.
Or email us, but then we won’t be able share your message with anybody.
- Write to your MP. Ask him or her to press the Health Secretary on why SMA is being left untreated in our country. Find current contact details of your MP on this page. Do not use letter templates – it is always better to write a personal email straight from your heart. About you, about your child. Thank you to copy treatsma@treatsma.uk on your MP correspondence.
- If you have contacts in the media or with celebrities and opinion leaders, help us to tell the world. Speak to them or put us in touch with them please.
- Wherever you are, pass on this message:
- There is a treatment to SMA
- Without this treatment deterioration is inevitable.
- There is NO ALTERNATIVE.
- Why we can’t have is because the NHS and Biogen cannot agree on the costs, more than any other issue.
- We need the government, the NHS and Biogen to sit down and negotiate an agreement which has the SMA Community at its heart.
If we do not step up, nobody else will. It is our lives. It is our children’s lives. It is not just about SMA – it is about everybody’s right to treatment.
Frequently Asked Questions
What is EAP?
EAP, or Expanded Access Programme, is the compassionate programme under which, Biogen, the company behind Spinraza, made the drug available free of cost to patients with the most severe form of SMA – SMA type 1. The programme was launched in a number of countries, including in the UK.
Despite the extremely promising results that Spinraza had shown in clinical trials and access to the drug free of cost, UK hospitals were extremely slow to initiate the EAP, citing lack of funds or manpower to administer the treatment. As a result, at a time when many European countries had begun to provide Spinraza to all of their SMA type 1 children, in the UK we were still seeing children with SMA type 1 dying before having the chance to start treatment.
From its beginnings TreatSMA campaigned to make the nusinersen treatment available to all babies and children with SMA type 1 across the country. The efforts bore success. More and more hospitals in the UK decided to introduce EAP, and the current progress can be seen on the SMA treatment Map.
Currently, more than 70 UK children are treated with nusinersen under EAP.
What about children currently receiving Spinraza treatment under EAP?
The decisions does not affect them. NICE proposes that they continue being treated under the current arrangements. Spinraza used in EAP is not funded by NHS, so those enrolled in EAP may receive the treatment until they or their NHS clinician consider it appropriate to stop.
The decision to stop should be made jointly by the clinician and the child or young person, and the child’s or young person’s parents or carers. It would be unethical to withdraw treatment from patients who have already started a treatment plan on nusinersen, even if the final guidance is a negative one.
Is the decision by NICE related to the recent safety warning on the side effect hydrocephalus?
The Appraisal Committee evaluates cost-effectiveness and budget impact rather than pharmacological properties. Also, the Appraisal Committee meeting was held before this new warning was issued in EU.
If you are concerned about hydrocephalus, Biogen has issued a community statement which you can read here.
What is the Managed Access Agreement (MAA)?
NICE have advised that the financial risk to the NHS can be reduced with the introduction of a Managed Access Agreement (MAA). Such agreements are normally negotiated between the NHS and drug suppliers. As NICE have stated, an MAA for Spinraza would need the support of NHS England, patients, carers and clinicians, and that the drug would need to have the plausible potential to be cost effective. The scheme would potentially then be handled on a case-by-case basis and a trust-by-trust basis.
The process may be allowed to start immediately after the final NICE recommendation in order to reduce the delay to treatment.
What are STA and HST?
NICE has two principal routes to assess health technologies, including new drugs. The STA route has been designed to assess treatments for use in broad populations. Apart from the standard clinical efficacy analysis, treatments appraised under STA have to pass a rigorous value-for-money test. NICE’s recommendations for STA stipulate that a treatment that brings a patient from severe disability and certain death to full health should cost no more than £30,000 per patient per year. Treatments that bring about less improvement must cost proportionately less.
The other route, HST, was introduced recently for highly specialised life-saving interventions that are carried out in a handful of patients only (usually, no more than 100 patients per year); mostly in ultra-rare indications. This route allows for a significantly higher treatment per-patient cost.
TreatSMA has sought to convince the decision makers that nusinersen has been proven clinically effective across its entire label and therefore must be assessed and made available to all people with SMA. Our efforts bore fruit and NICE agreed to appraise nusinersen as a treatment for everyone. Despite the likely negative recommendation, this choice of route also opened the door to early negotiation of a Managed Access Agreement that would cover all.
