NICE Releases Nusinersen Final Appraisal Determination (FAD), Draft MAA, and Committee Papers

03 June 2019

Today, NICE released: its Final Appraisal Determination (FAD) which will form the guidance to the NHS; the draft Managed Access Agreement (MAA); and committee papers that summarise the information it considered in the process of getting to this stage. You can access these documents here, on the left-hand side dated 3rd June.

We note that the draft Managed Access Agreement is only a draft and as such still requires some further clarity and development. Jointly with MDUK and TreatSMA we have already raised concerns that as it is currently written, it appears to exclude some people from access for whom the treatment would be potentially beneficial, and clinically safe. We do not consider this to be in line with the announcement NICE made last month that, "This is one of the most comprehensive deals in the world, meaning 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".

NICE and NHS England have committed to respond to our concerns at the earliest possible time.