Your questions on Spinraza answered

Published Date
13/02/2019
Author
Laura Burge
Category
Care & Support

We have heard today that, subject to sign-off, Spinraza will be available on the NHS in Scotland for children and adults with SMA Types 1, 2 and 3 from April 2019.

Spinraza is set to be one of the first drug to be approved through a new decision-making process, known as an ultra-orphan pathway, introduced by the Scottish Government. Muscular Dystrophy UK and SMA UK welcome the announcement, which gives SMA families in Scotland hope for the future. It could also mean that more patients with muscle-wasting conditions are able to access new treatments more quickly when they emerge in the future.

The charities will also keep campaigning on behalf of families in the rest of the UK, where the treatment is not available on the NHS. Now more than ever, we need to see leadership and urgent action from NICE, Biogen and NHS England, who must sit down together to find a solution before any more lives are unnecessarily cut short.

You can read more about SMA and the support and information Muscular Dystrophy UK offers for those living with the condition here.

QUESTIONS AND ANSWERS

Spinraza is the first treatment for SMA. Developed by pharmaceutical company Biogen, clinical trials have shown significant improvement in children’s motor function; for the first time, we have seen children with SMA Type 1 crawl and even walk. Families of children who have received the drug often report noticeable improvements from the start of treatment. The drug proved so effective in clinical trials with children with SMA Type 1 that in August 2016 they were stopped early so all the children could access the drug.

The drug also has promise for the other types of SMA; motor function for children with SMA Type 2 and 3 was significantly improved in trials, although data so far refers only to children.

While the drug was highly effective in most treated children, there were some who did not respond to the treatment. Some patients may also be too weak to receive the injection through the spinal canal.

England, Wales and Northern Ireland: Spinraza is not available on the NHS in England, Wales or Northern Ireland for all types of SMA. Some children in these countries with SMA Type 1 can access treatment through a compassionate scheme – known as an Expanded Access Programme – introduced by pharmaceutical company Biogen in 2016. However, this closed to new patients on 1 November 2018. Infants already on the scheme continue to receive Spinraza.

Scotland: Infants with SMA Type 1 in Scotland have access to Spinraza on the NHS after it was approved by the Scottish Medicines Consortium in May 2018. Before this, infants with SMA Type 1 received Spinraza through the Expanded Access Programme. Currently, children and adults with other types of SMA do not receive Spinraza, although applications can be made on a case-by-case basis.

The new pathway was prompted by recommendations made in 2016 in Dr Brian Montgomery’s Review of Access to New Medicines. Dr Montgomery’s report highlighted that there was a low approval rate for medicines for very rare conditions. To resolve this, the report recommended the development of an alternative assessment pathway for ultra-orphan medicines.

Drugs must meet the following criteria to go through this pathway:

  • The condition has a prevalence of 1 in 50,000 or less in Scotland
  • The medicine is granted orphan designation by the European Medicines Agency (EMA)
  • The condition is chronic and severely disabling
  • The condition requires highly specialised management

If a medicine meets the definition of an ultra-orphan medicine and the Scottish Medicines Consortium (SMC) considers it clinically effective, then it will be made available on the NHS in Scotland for at least three years while further information on its effectiveness is gathered. Following this period, the SMC would review the evidence and make a final decision on its availability on the NHS in Scotland.

Medicines like Spinraza that fall under the new definition and which have been recently reviewed by SMC but not recommended for routine use will be admitted to the new pathway.

The Scottish Medicines Consortium has now granted Spinraza ultra-orphan designation. Subject to successful sign-off, it is expected to be routinely available for SMA Types 1, 2 and 3 from April 2019. It is anticipated that this will be for a period of three years, to collect data on the drug’s efficacy, with a view to making a final decision on routine availability.

Spinraza is currently being appraised by NICE for use on the NHS in England (Wales and Northern Ireland typically follow this guidance). NICE gave the drug a negative opinion for all types of SMA in its initial guidance, which was published on 14 August. NICE also held a committee meeting on 23 October and has said since then that discussions are ongoing with the drug manufacturer, Biogen. NICE is due to make a decision over the coming months. Meanwhile, also in August, Biogen announced it would bring its compassionate scheme to a close on 1 November to new patients. While this does not affect those who currently receive the treatment on the programme, anyone diagnosed with SMA Type 1 after this date does not have access.

Spinraza is available for children and some adults with all types of SMA in 24 European countries. Negotiations are under way in other countries. More widely, Spinraza was approved in the United States in December 2016, and has gained additional approvals in Korea, Canada, Japan, Brazil and Australia since then.

Muscular Dystrophy UK and Spinal Muscular Atrophy UK welcome this positive news from Scotland. We have long been calling on the Scottish Government to implement the new assessment and approval pathway for ultra-orphan medicines. Our hope is that the new pathway will mean that more patients with muscle-wasting conditions are able to access new treatments.

However, while it is exciting news for patients in Scotland, the introduction of this new pathway highlights failings with the appraisal process elsewhere. In the rest of the UK, the process needs to change to adapt to the growing number of breakthroughs in research we are seeing. We are calling on NICE to approve the treatment for use on the NHS in England for infants with SMA Type 1 and would like to see a temporary arrangement put in place for those with other types of SMA so more data can be gathered on its efficacy.

SMA is passed from parents to their children through faulty SMN1 genes:

  • People who have inherited two faulty copies of the SMN1 gene (one from each parent) have SMA
  • People who have inherited one faulty copy and one healthy copy of the SMN1 gene (one from each parent) are carriers of SMA. Carriers usually do not have SMA or any symptoms of SMA
  • People who have inherited two healthy copies of the SMN1 gene (one from each parent) do not have SMA and are not carriers.

When two SMA carriers have a child together, for each pregnancy there is a:

  • 1 in 4 (25%) chance that the child will inherit both faulty copies of the SMN1 gene and will develop SMA
  • 1 in 2 (50%) chance that the child will inherit one faulty copy and one healthy copy of the SMN1 gene and will be a carrier
  • 1 in 4 (25%) chance that the child will inherit two healthy copies of the SMN1 gene and will not be a carrier or have SMA

Spinraza is administered through an injection into the spinal canal in an established procedure known as an intrathecal injection. This delivers medication through the lower back via a lumbar puncture and directly into the central nervous system. This procedure is performed under the direction of healthcare providers experienced in administering lumbar punctures and requires special precautions – either sedation or general anaesthetic.

The list price of Spinraza is £75,000 per vial (excluding VAT; British National Formulary, accessed June 2018). At list price the total annual treatment cost is £450,000 for the first year and £225,000 for subsequent years. Biogen proposed a commercial arrangement – effectively a negotiation over the cost – which would apply if the technology had been recommended.

Two trials, called ENDEAR and CHERISH, were carried out by Biogen. There are also three ongoing studies: NURTURE, SHINE and EMBRACE.

In August 2016, the drug proved so effective in clinical trials with children with SMA Type 1 that they were stopped early so that all the children on the trial could access the drug. Biogen then opened its Expanded Access Programme for Spinraza, offering the drug free to all children with SMA Type 1 who showed signs of SMA before six months of age. Clinical data indicates that Spinraza has shown, in some cases, to improve survival and motor function.

If you have the condition, or know someone who has the condition, you can contact your local MP and ask them to put pressure on NICE, Biogen and NHS England. Please email Jonathan Kingsley at: j.kingsley@musculardystrophyuk.org to find out more.

We are also encouraging MPs to write to NICE, Biogen and NHS England about Spinraza. If you are an MP, you can download our template letter here.

We would encourage you to speak to your consultant for any queries on accessing Spinraza.

You may come across different acronyms when hearing people talk about Spinraza. Below is a guide to what these mean.

AAP: Accelerated Access Programme. A government scheme to fast-track breakthrough treatments announced in November 2017. We are waiting to hear if rare disease drugs will be eligible for it.

EAP: Expanded Access Programme. A special scheme currently providing access to Spinraza for those with SMA Type 1 from the pharmaceutical company Biogen. This was closed on 1 November 2018.

EMA: European Medicines Agency. Pharmaceutical treatments typically need to get a licence for marketing a drug from the EMA before they are assessed by NICE. The EMA approved Spinraza in 2017.

HST: Highly Specialised Technology route. One of the two routes, along with STA, through which NICE assess treatments in England.

MAA: Managed Access Agreement. This is an agreement to provide treatment on the NHS while more data is gathered on its efficacy, and we want to see one put in place for Spinraza in England, Wales and Northern Ireland.

NICE: National Institute for Health and Care Excellence. NICE is in charge of recommending whether treatments should be funded on the NHS.

PACE: Patient and Clinical Experts. A special meeting held by the SMC to garner views.

SMA: Spinal muscular atrophy.

SMC: Scottish Medicines Consortium. The SMC is in charge of recommending treatments for provision on the NHS in Scotland.

STA: Single Technology Appraisal route. The main route through which NICE assesses treatments in England, including Spinraza.

August 2016: The drug proves so effective in clinical trials with children with SMA Type 1 that they are stopped early so that all the children can access the drug. Biogen then opens its Expanded Access Programme (EAP) for Spinraza, offering the drug free to all children with SMA Type 1 who showed signs of SMA before six months of age in the UK.

21 April 2017: The drug is recommended by the European Medicines Agency (EMA) to be licensed as a treatment for SMA Types 1, 2, 3, 4.

1 June 2017: The European Commission finalises the recommendation by the European Medicines Agency (EMA).

 August 2017: NHS England agrees to support delivery costs for Spinraza, but only for children with two copies of the SMN2 gene (around 80% of children), as these were the only children involved in the clinical trials.

November 2017: The Department of Health announces a new, fast-track route into the NHS for ‘breakthrough’ medicines and technologies, known as the Accelerated Access Pathway (AAP). Five drugs a year will benefit – but no criteria is given and the scope of the scheme is unclear.

November 2017: The Scottish Medicines Consortium (SMC) starts its appraisal of Spinraza.

January 2018: NICE announces it will begin its appraisal of Spinraza for SMA Types 1, 2, 3 and 4 through its Single Technology Appraisal (STA) route. Discussions of an interim scheme, known as a managed access agreement (MAA), also get under way between manufacturers Biogen, NHS England, and NICE.

13 March 2018: Families and charities make submissions and give evidence on the benefits of Spinraza at a Patient and Clinical Experts (PACE) meeting held by the Scottish Medicines Consortium (SMC).

3 April 2018: The Scottish Medicines Consortium (SMC) holds a second meeting for final submissions from charities and families on why Spinraza should be approved in Scotland.

7 May 2018: The Scottish Medicines Consortium (SMC)’s appraisal of Spinraza is published. It recommends it for use on the NHS in Scotland for children with Type 1 SMA – but not Types 2 and 3.

14 August 2018: NICE publishes its appraisal consultation document for Spinraza, setting out provisional recommendations for not approving the treatment for use on the NHS in England. This was originally expected to be released in June.

22 August 2018: Biogen announces it will be stopping the Expanded Access Programme from 1 November for all babies who are diagnosed after this date. Biogen says babies and children currently on this scheme will not be affected.

8 October 2018: The Scottish Government launches the ultra-orphan pathway for rare treatments.

1 November 2018: Biogen’s Expanded Access Programme is closed to new patients.

13 February 2019: Announcement is made that Spinraza is due to be made available on the NHS in Scotland for children and adults with SMA Types 1, 2 and 3, having been assessed through an ultra-orphan pathway.

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