The Canadian Agency for Drugs and Technologies in Health just published a report that recommends only allowing the miracle drug Spinraza to be made available to a very small subset of Canadians who have Spinal Muscular Atrophy. As you can imagine, this is a heart wrenching decision that has filled many Canadian parents and individuals with SMA with fear and sadness. CADTH doesn't make the decision, that's up to each province, but the provinces widely listen to and often accept CADTH's recommendations.
We literally do not know what to say.
If you have been following Emmett's Journey, then you know that he was one of 124 infants worldwide who were part of a blind study and that he joined this study in the fall of 2015. In the care of professionals at BC Children's Hospital, we quickly started to see improvements. This was a blind study, but when improvements begin happening in an 8 month old who had lost all movement in his legs, had minimal arm movement, with very limited movement his head, and was being fed by G-Tube, then you quickly notice. Spinal Muscular Atrophy is a degenerative disease. There may be plateaus but evidence demonstrating improvement is simply not there. We saw and still see improvement so we know that this drug, Spinraza, works.
Emmett's parents were concerned about him participating in a medical study. I've shared before that as grandparents we thought it was a crazy idea. I've also shared that we were wrong and that we're so glad that Alisha and Josh agreed to have Emmett participate. He was one of four kids at BC Children's Hospital who were part of the trial for this drug that came to be known as Spinraza. The three other children, sadly, did not survive. You see, Spinal Muscular Atrophy is a genetic disease that takes the life of more babies than any other genetic disease. It literally shuts down their respiratory system. This is after it has shut down their ability to move their limbs. It is a terrifying disease.
That is why we are terrified by this decision. The drug is already horrendously expensive and that is obviously a challenge to families dealing with SMA. Yet this drug, Spinraza, is the absolute first treatment option available to individuals with SMA. Emmett has SMA Type 1. Some little ones are identified even younger than Emmett - who was officially diagnosed at five months. Some have Type 2 which is a bit later onset. Then there's Type 3 and Type 4. This decision, if the provinces act upon it, bars Type 2, Type 3, Type 4 and even a large number of Type 1 kids (and adults) with SMA from having access to this drug.
Now, because Emmett was and is a part of this study he probably fits all of the criteria that CADTH has set out for access to this drug. That, however, is not the point. Obviously, we want Emmett to improve and that is not in question. But this drug works for all SMA types, so why is it being limited? It is life changing for anyone with SMA and their families. Emmett was not in this trial for himself - he was in it for everyone. Think about it, when Emmett got involved in this it was an unproven drug. No one knew what would happen - although there were indications and a lot of hope that it was positive. But Emmett's parents took a leap of faith - hoping beyond hope for a miracle for their son.
Emmett unknowingly worked very hard to get this drug approved. He has benefitted from this drug, Spinraza, which I absolutely call a miracle drug. That it was approved in the USA, Canada, and several other countries is a miracle. It is available now in the U.S.A. for all types of SMA. Now we need another miracle - a Canadian miracle. We need the experts at CADTH that look at drugs to realize that they need to expand their point of view and recommend full access to this drug. The decision they have made is based upon scientific fact but to the narrowest of definitions. As a result there will be more Canadian babies who die of Spinal Muscular Atrophy. Children and parents who have been waiting years for a treatment will be denied access - their hopes dashed.
To those who question the expense of this drug, please realize that I question the expense too. It's literally beyond belief. Then again, creating new pathways for the brain to be able to speak with muscles that control lungs and muscles is absolutely beyond belief.
Please watch this blog - we hope to be able to share ways in which you can be an advocate for wider access to this drug here in Canada.
https://www.cadth.ca/nusinersen - nusinersen is one of the early names of the drug Spinraza