Today NICE released its final appraisal document (the appraisal committee’s final draft guidance about using a treatment on the NHS ) and the draft managed access agreement (MAA) relating to Spinraza. You can see all the documents on the NICE website.
A managed access agreement is an agreement between NHS England and NICE that allows patients to receive new treatments while long-term data on them is still being gathered and before final funding decisions are taken. We feel that the draft MAA requires some further clarity and development and jointly with SMA UK and Treat SMA have already raised concerns that as it is currently written there are potential restrictions to access that we do not consider to be in line with the announcement NICE made last month. NICE and NHS England have committed to respond to our concerns at the earliest possible time. We will continue to update the SMA community as we receive the clarification that we are seeking.