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Joint Charities’ update following the NICE Scoping workshop (13th January 2017)

19 January 2017

Joint Charities’ update following the NICE Scoping workshop (13th January) which looked at the draft remit and scope for the proposed Technology Appraisal of nusinersen for treating spinal muscular atrophy

Who was there?

Representatives from SMA Support UK, The SMA Trust, Muscular Dystrophy UK, and Genetic Alliance attended a scoping workshop with the National Institute for Health and 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.

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

What is the NICE process?

Possible topics for what is called a ‘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.

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 indication expected in the licence.

Once NICE receives this referral for a technology appraisal, it undertakes a full and detailed assessment of the drug. This 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 finally recommends the drug for use through the NHS, then NHS England is normally expected to fund the treatment within 90 days. 

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

It’s important to understand that a full NICE assessment only takes place if the European Medicines Agency (EMA) grants a licence. Therefore, if the EMA does not approve the drug, then a NICE technology appraisal will not take place.

What was this workshop for?

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

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 workshop?

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

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 the EMA. A NICE representative at the meeting commented “more often this work is timely than not but it does depend on NICE’s workload.”

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.

You can read what Biogen are saying here.

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 and is eligible for treatment under the proposed licence to have access. 

The first step towards possible licensing of nusinsersen in the UK is for it to go through the EMA appraisal process.  We are encouraging people to write to the EMA to speed up their appraisal process. See how you can add your voice here.