Last night was the Muscular Dystrophy Cross Party Group Meeting at the Scottish Parliament. We were joined by Aileen Campbell, Minister for Health & Sport and Prof Alison Strath, Principle Pharmaceutical Officer for the Scottish Government. Spinraza was, as expected discussed.
In summary, I shared by disappointment with the announcement; the errors in documenting the cost-effectiveness - therefore it not being possible to compare like with like between the types; the feeling that what we had discussed at the PACE had not being accurately conveyed to the SMC; and the need for the adult voice to be heard. These concerns were echoed by another member of the meeting who had gone through the same process with Translarna (a treatment for duchenne) 2 years ago and she was dismayed to hear that the process had not improved despite the publication of the Montgomery Review.
The Minister and Prof Strath listened and genuinely seemed interested, taking on board all that was said. I can provide an update on what was said in reply.
The Montgomery Review on Access to New Medicines, published in December 2016, talked of new medicines for ultra-rare diseases being considered differently under a new appraisal processes. This process was supposed to be implemented by Spring 2018. It was confirmed last night byt Prof Strath that this process is still under consideration with no known timeline being confirmed. Biogen are keen for Spinraza to be reassessed after this new process has been implemented. This may therefore take some time.
It is hoped that in June of this the new Peer Approved Clinical System (PACS) Tier 2 will start, allowing for non-routine access to medicines on an individual basis. While each health board will operate PACS Tier 2 for their own area, a new National Appeal Panel will be introduced, for individual requests for access to medicines not approved by the Scottish Medicines Consortium. Prof Strath seemed to think that this was a more likely path for SMA types 2 and 3 in the interim.
Given the time frame and the urgent need for Spinraza to be approved for those with types 2 and 3 - to potentially halt further muscle weakness - Jackie Baillie, who chairs the CPG meeting,- supports the petition that I have lodged with Parliament pushing for an early reassessment and will help as she can. This petition should soon be available for circulation and allow us to promote awareness re SMA and Spinraza, but also show the strength of our voice.
The Minister and Prof Strath will feed back to the SMC about our concerns re erroneous data re cost effectiveness which is the most pressing of my concerns given the decision was based on cost alone (Prof Strath confirmed this). Prof Strath is also going to speak to me directly about the PACE and the Minister promises to continue the conversation.
The long and short of it, our disappointment has been shared, there is no quick answer but there is hope, and the conversation continues.