NICE Scoping Workshop – Joint charities update

Joint charities update following the NICE Scoping workshop to look at the draft remit and scope for the proposed Technology Appraisal, nusinersen for treating spinal muscular atrophy.

Representatives from MDUK, The SMA Trust, SMA Support UK and Genetic Alliance attended a meeting with the National Institute of Health and Care Excellence (NICE) on 13th January, along with two parents from the campaign group TreatSMA, clinical representatives and representatives from Biogen.

Who was there?

Representatives from MDUK, The SMA Trust, SMA Support UK and Genetic Alliance attended a scoping workshop with the National Institute of Care Excellence (NICE) on 13th January, along with two parents from the campaign group TreatSMA, clinical representatives and representatives from Biogen.

This was to discuss information that NICE had already collected  through its draft remit and scoping work.

The full list of stakeholder organisations invited to participate can be found here.

What is the NICE process?

Possible Topics for Technology Appraisal are filtered through a Topic Selection process to ensure it’s a correct topic for NICE to look at. Topics usually reach the scoping stage around the time the company plan to apply for a licence for its drug in the UK or Europe.

NICE had established nusinersen as a correct topic and prepared a draft ‘scope’ – a document that describes how NICE would look at the new drug, and what questions it would ask and how, if the drug were to be referred  to NICE for a full appraisal. See draft scope link

At this stage, NICE collects information from stakeholder groups about the condition and the treatment so that it can make a preliminary report to NHS England and the Department of Health about whether an appraisal should go ahead and what questions need answering if there is to be a full appraisal.  It is then up to Government Ministers to decide whether or not to make a referral to NICE to fully appraise the product for the specific licencing purpose or indication it expects the company to request. This NICE review is known as a ‘technology appraisal’.

Once NICE receives this referral for a technology appraisal, it undertakes a full and detailed assessment of the drug, which considers all aspects of the drug and condition, including how well the drug works, who it is for, any side effects, how it can be used, how the condition affects people and other areas including the cost of the treatment. Evidence is also taken from expert healthcare professionals, and patients and families on the impact of the condition.

If NICE recommends the drug for use through the NHS, then NHS England is normally expected to fund the treatment within 90 days.

However, a full NICE assessment would not take place unless the European Medicines Agency (EMA) grants a licence. Therefore, if the EMA does not approve the drug, then a NICE review (known as a ‘technology appraisal’) will not take place.

What was this meeting for?

The meeting on 13th January was a chance for NICE to talk to patient representatives, clinicians and the company about this potential new drug, to gather more information and to refine the scope document. This s so that NICE is ready if the drug if/when it does come to appraise it, and is making sure it asks the right question, in the right way.

At the workshop NICE gathered further details on:

  • SMA, classification of different types and impact on individuals and families
  • the treatment nusinersen – how it is delivered and the results of the clinical trials on children to date
  • the potential impact of observed improvements on patients’ quality of life
  • the likely uptake and delivery of the treatment if it were to be licensed – in terms of actual numbers and how many Centres might be involved

What was said at this meeting?

Patient representatives responded to NICE’s further questions which included answering that, in their opinion:

  • The topic nusinersen for treating spinal muscular atrophy should be assessed as a Highly Specialised Technology (HST) rather than as a Single Technology Appraisal (STA)
  • If it is ultimately licenced, nusinersen should be made available to those affected by SMA Types 1,2 and 3

What happens now?

NICE will now report to NHS England and the Department of Health. NICE anticipate that they will hear back from the Minister in April – May 2017 as to whether or not the topic will be referred to NICE to fully assess.

At this point NICE will write to Biogen and all the consultees, including all the Patient organisations, to advise them of this decision and what the timeline for the assessment will be.  NICE’s report from today’s meeting will not be released until this point and no further information may be shared until then.

If NICE does receive this referral from the Minister, they are asked to publish guidance within six months of the product receiving its licence from EMA.  NICE representative at the meeting commented ‘more often this work is timely than not but it does depend on NICE’s workload.’.

The full NICE approval process (including possible appeals) takes an absolute minimum of 43 weeks, and this process can be much longer.

What about the rest of the UK?

In Scotland, the process for assessing new medicines is usually conducted by the Scottish Medicines Consortium, following a licence by the EMA.

Wales, Northern Ireland and the Isle of Man will often follow the guidance issued by NICE,

What can we all do now?

We will publish the decision and timeline on our websites as soon as we have this information from NICE. This will include information about how and when you can let NICE hear your views.

We will continue to work with the expert centres and Biogen to collect evidence to inform the NICE appraisal

If/when  NICE does fully appraise nusinersen, it will be vital that Biogen presents a value proposition / pricing framework that makes it possible for the drug to be made available to everyone in the NHS. At the moment we don’t know what this will look like. Biogen (in a meeting with SMA Europe) advised:

“The healthcare systems and pathways to access in the European countries are very different than the US. However, with the recent approval by the FDA and access within the US, we feel it is important to share the principles on which Biogen articulates its approach to prices of our medicines.  Consistent with the pricing of the overall Biogen portfolio of therapies, we strive to achieve an appropriate balance among three key Biogen pricing principles – clinical value the therapy provides to patients and families, the impact this new treatment has on the healthcare system, and finally our ability to fulfill our long-term commitment to patients and advancing science through the funding of research and development. We will be using these principles to determine the European price.

We want to assure you that we are committed to providing access to patients who may benefit from nusinersen. We have begun working, where feasible, with health-care systems and governments to discuss solutions to achieve nusinersen’s access as quickly as possible, should nusinersen be approved.”

As soon as we know if a full technology appraisal is going to take place we will be asking for your help to urge Biogen to offer a pricing framework that makes it possible for the drug to be made available to everyone in the NHS who has SMA that is eligible for treatment under any licence granted to have access.

It is vital that the EMA grants the drug a licence: that way, NICE and organisations in other parts of the UK are able to begin reviewing the drug for use on the NHS.

Get involved in our campaign to bring Spinraza to the UK by contacting your local MP.


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