Our Response to NICE's Consultation Paper

08 September 2018

On 5th September we submitted our response to NICE’s consultation paper which had been released on 14th August with the devastating news that they had not recommended nusinersen for funding by the NHS. You can read our response in which we put forward further evidence to challenge this decision and in support of access to nusinersen for all, hereWe know many of you will also have responded.

The paper encourages, and we have supported, the interim possibility of a Managed Access Agreement (MAA). This would be a 3 – 5-year scheme agreed between Biogen, NICE and NHS England that would enable access to treatment and the opportunity to collect further evidence of nusinersen’s clinical benefits and impact on quality of life. In our response we have urged that this should enable access for all with SMA Types 1, 2 or 3 where there is agreement between the family/adult and clinician that it is a clinically safe option. We have also urged that there is a seamless transition from Biogen’s Expanded Access Programme (EAP) for those with SMA Type 1 that closes on 1st November to a new agreement, so that there is no delay in treatment of any infant.

We will continue to push for these outcomes when we take part in any further discussions between NICE, NHS England, Biogen clinicians and patient groups over the next weeks.