I Stopped Taking Evrysdi

When SMA treatments first became available, I honestly wasn’t sure how I felt. At the time it was just Spinraza which I never tried due to other medical issues and a spinal injection wasn’t worth the risk to me.

But then a few years later, Evrysdi was FDA approved and this intrigued me considering I’d be taking the medication by mouth and in the comfort of my own home.

Trying this new drug

Previously I wrote about my beginning journey with Evrysdi and how I immediately took the opportunity to get sent the trial package while I worked on getting long-term insurance approval. Well, that process turned into a battle involving multiple appeals and my doctor doing peer-to-peer phone calls.

Struggling to get insurance approval

The problem is insurance companies are not properly educated by drug manufacturers on the importance of the medication, why it needs to be taken, and how it can positively affect a patient. So, when insurances see a prior-auth for a large amount of money, they deny it because they actually don’t understand the medication.

On top of that, clinical trials for medications are often not appropriately designed to help the person who will be taking the drug. Instead, they are designed to help pharmaceutical companies get easy FDA approval by creating large enough test groups that will hopefully show the medication is working.

Adults with SMA denied Evrysdi

A lot of people (and by people I mean drug manufacturers) will say this is untrue, but we can see the impact of a poorly designed trial based on who is getting denied continuous treatment after their original approval period expires. The answer: adults with SMA.

Older people with SMA are being denied Evrysdi because insurances are saying there isn’t evidence that the medication is working. In my recent battle for re-approval, my doctor had to perform physical tests like can I rip a folded piece of paper, how long can I sit on my own without support, and, my favorite, how many steps can I take.

Clinical trials lumped together separate groups

Tests like this aren’t relevant for older SMA patients who have been dealing with the side effects of our disability for over twenty years. However, the reason they are required for approval goes back to poorly designed clinical trials.

One of the test groups for Evrysdi included young children all the way to adults in their mid-twenties. Of course, younger patients are going to see significant improvement because they’ve had SMA for much less time and therefore fewer side effects. Adults, on the other hand, are taking the medication to stabilize and not see further muscle deterioration.

Adults and children with SMA see different results

Insurances unfortunately don’t see the importance of the medication for adults because when the trial data was published, all ages were grouped together. Younger SMA patients skewed the results and now insurances think I should be able to get out of my wheelchair and walk after never doing that in my life.

We can blame insurances for their obvious ignorance, but this really falls on the makers of Evrysdi. They need to design more accurate trials and, if they don’t, explain the different benefits of taking the drug for each age group.

Didn't want to stop, but had no choice

So, at the beginning of this year I found myself fighting with my insurance after taking Evrysdi for over 18 months, a medication I once called liquid gold. But ultimately I had to stop.

Not necessarily because I wanted to, but because our pharmaceutical and insurance system is broken. I think we all know that, though.