NICE, The National Institute for Health and Care Excellence has confirmed it has started the appraisal of Spinraza following a frustrating delay, which has been partially down to uncertainty about how the treatment will be reviewed for funding by the NHS. The treatment will now be being assessed for all types of SMA, with Spinraza the first and currently only treatment for people with SMA.
NICE is now looking at the treatment via its Single Technology Appraisal (STA) route. This is designed to assess drugs for both clinical- and cost-effectiveness and is usually used for more common conditions. The other avenue, the Highly Specialised Technology (HST) route is more suited to rare disease drugs, accounting for small populations and high costs per treatment, but would have limited the scope for potential patient access, meaning only SMA Type 1 patients would have benefitted. Given the confirmed STA route, there is concern that this route is not set up to assess rare disease drugs, like Spinraza.
However, there is some promising news. The pharmaceutical company, Biogen, has announced that they are in discussions with NICE and NHS England to put an interim access scheme in place. This scheme, known as a Managed Access Agreement (MAA), could give more people with SMA access to the life-changing treatment while further data and evidence are gathered over several years.
Time is key and it is crucial Spinraza is not subjected to any delays during the NICE assessment process, which is scheduled for completion on 21st November. Please help us push for the MAA to be implemented as soon as possible by writing to your MP and asking for their support.
Given the urgent need to ensure that Spinraza is made available and pre-empting the potential economic factors of evaluation via the STA route, we will continue to work with NICE, Biogen the manufacturer, partners Muscular Dystrophy UK and other SMA charities to call for patient access, as quickly as possible, during the NICE evaluation and on a longer term basis.
Please note, Biogen has also advised that although they will continue to support children currently receiving the treatment through the Expanded Access Programme (EAP), they may close the programme to those newly diagnosed.
We would expect guidance produced to be followed in Wales and Northern Ireland. Scotland has a standalone system, the Scottish Medicines Consortium (SMC). The SMC is currently carrying out a consultation re: Spinraza funding, for which we are currently writing a submission.