Liz - Patient Expert at NICE Committees

Liz Lockley is Mum to 8-year-old twin boys and a 3-year-old daughter. George was diagnosed with SMA Type 2 just before his first birthday.

Here, Liz describes her experiences of being a patient representative at three NICE committee meetings considering whether to recommend access to nusinersen in England:

“Preparing for the meetings, reading the documents and writing my submissions has been challenging, especially dedicating the time it deserved, but I was well supported by the two other patient representatives and other members of the SMA community. The chair of the NICE committee and other staff at NICE were also very supportive in the run up to the meetings.

I found taking part an emotional experience, which at times was both difficult and frustrating. However, it was also extremely satisfying to be able to speak up for George and others affected by SMA.

I was especially pleased to be able to give them a detailed explanation about the true costs of caring for a child with SMA Type 2 as I felt this definitely had an impact on the committee.

However, despite what was said, their decisions seemed to depend on an economic model and appraisal system that appears to be fundamentally flawed when it comes to the treatment for rare conditions like SMA. This is extremely frustrating and upsetting for everyone.

I just hope NICE are now able to make the right decision and approve Nusinersen for everyone with SMA in the UK. We have all waited long enough.”


March 2019