Yesterday SMA families from all over the country gathered in front of the Manchester office of the National Institute for Health and Care Excellence where a meeting on Spinraza approval was being held. It was a heart-wrenching scene when children and adults with a life-limiting disease all chanted: “What do we want? Spinraza! When do we want it? Now!”
The protest attracted attention of several media outlets as well as ordinary passers-by. We were telling everyone about our lives, about effects of SMA, and about our fight for treatment.
At 12.30pm, selected TreatSMA representatives left the scene to attend a private meeting with NICE top brass, which we had requested in advance. Preset were the Head of the Manchester office Mr Meindert Boysen as well as Programme Director Technology Appraisals Ms Helen Knight and Associate Director Technology Appraisals and HST Ms Sheela Upadhyaya. We were also joined by the representatives of SMA UK.
In an hour-long meeting, the SMA community representatives discussed the challenges with the Spinraza appraisal and as well as the future scenarios. We stressed how beneficial the Spinraza treatment is for all people with SMA, presented new evidence of efficacy, and explained why a broad approval is absolutely necessary. Spinraza works, and we can wait no longer.
Afterwards, the public meeting started. Spinraza cost-effectiveness models were reviewed, and the agency analysts attempted to determine whether Spinraza can be cost effective, and if so then in which patient subpopulations.
Finally, all the external participants as well as the public were asked to leave and the Committee began reviewing, in private, the commercial offer submitted by the manufacturer last month.
The decision is expected to be reached in the next weeks and publicly announced mid-April.
We all did what we could, and we were heard. We showed up many, determined, united. We told the decision makers and the world that hundreds of people in the UK need the life-saving drug Spinraza NOW.
Big thanks to everyone who supported our yesterday’s effort.