skip to Main Content
The SMA Community And Clinicians React To Draft NICE Guidance

The SMA Community and clinicians react to draft NICE guidance

The last couple of days brought understandable stress to many of us. On 15th May, jubilant NICE and NHS England announced that “all relevant SMA patients (SMA 1,2,3a and 3b), including adults and siblings who are yet to show symptoms, will be able to benefit from this treatment.” We all celebrated, and tears of joy flowed by gallons.

However, the celebrations turned up sour when it was disclosed that a large part of the population – an estimated 15% – will be permanently excluded from accessing the only effective treatment for their disease. What is more, in preparing the draft guidance NICE and NHS totally diregarded the opinions expressed by TreatSMA, SMA UK and MD UK during a closed-door meeting that took place on 30th May.

At TreatSMA, this didn’t stop us.


We carefully looked into the consequences of formally appealing the draft guidance.

In case we do not appeal, it means we roll over and let the NHS dictate the terms which are detrimental to many people with SMA. It would mean that at least the majority of people will get access, whilst the remaining ones will have to wait for a new drug to be developed, approved and got through the NICE procedures.

We frankly do not think this is acceptable. TreatSMA has always been about getting treatment for all. From the day we started, we never stopped. We negotiated the start of EAP. We advocated for Spinraza to be appraised via STA route to make sure that no people are excluded. Now, we are aware that any appeal would halt the process and those who can start now would not be able to until the appeal is resolved, a process which usually takes 4-5 months.

We are between a rock and a hard place.

We then went on to contact NICE, NHS England, clinicians, other SMA organisations, and Biogen to work out a satisfactory solution. This week, as a result of a number of meetings, emails and phone calls we came out with an alternate proposal: children with SMA type 3 no longer able to walk must be included in the MAA now; adults with type 3 no longer able to walk must have the guidance reviewed before a year from now to allow the consideration of new clinical evidence that may (and will) be published by then; and those in whom scoliosis or contractures are of concern must be allowed a referral to a national panel composed of neuromuscular experts knowledgeable of SMA.

This is not an ideal solution, but one that gets us much closer to having treatment for all.

A word on hospital capacity

We are acutely aware that there is currently very little in terms of physical and organisational capacity to administer Spinraza on a large scale. This is a sad fact, unfortunately. Spinal fusions, contractures and other bits need to be assessed and expertise shared and trained. Paediatric centres will find it easier to do, but adult hospitals already struggle with capacity everywhere. We have seen the onboarding process in other countries and we assess that it will take at least until 2020 before all the people with SMA will start on treatment. Not because the consultants don’t want to, but because of hospital capacity limitations.

As a reassurance, clinicians will try to prioritise the most urgent cases.

Two letters sent today

TreatSMA co-signed two letters which were sent to NICE and NHS bosses today.

UK clinicians, led by Professor Francesco Muntoni, today sent a letter that outlines the above position and presents a strong argument for the above solution. It is a very balanced letter that presents the clinicians’ perspective in objective medical terms. Note that the names of the addressees and signatories have been redacted in this web version.

Read clinicians’ letter

SMA UK, TreatSMA and MD UK sent a letter that stresses our strong disappointment with the way the MAA and guidance have been drafted.

Read patient organisations’ letter

It is a great thing that the clinicians and patient community are so aligned.

What’s next?

We will carefully consider NICE and NHS responses to the two letters. Based on their willingness to consider our and clinicians’ point of view, we will take a decision on whether we shall appeal the guidance this coming Monday or not.

We stand for treatment for all.

Back To Top