Earlier this week STAT News published a brief article entitled, “Vertex sinks on reports of deaths of patients taking cystic fibrosis drug” without providing too much context. Given the first line of the article was, “Thirty patients have died after taking a Vertex (VRTX) Pharmaceuticals treatment for cystic fibrosis, according to a government database” I think a lot of folks in our community fell into a dizzying panic since a lot of us, or people we know, have been taking the drug in question, Symdeko.
I also think the rush to panic was amplified because the components in Symdeko make up 2/3 of the Triple Combo.
The government database cited in the article is the FDA’s Adverse Event Reporting System. You can see the data reported on Symdeko HERE.
What doesn’t really materialize in the article was the medical literature to suggest how Sydmeko was a contributing factor to the patient death’s, those patients’ underlying CF related health issues, or if the people with CF who paid the ultimate price were on Symdeko in their final days. The article does, however, note that Vertex doesn’t have any information linking Symdeko to the fatalities in question.
So is the panic a little overblown? It might be.
It goes without saying that nearly all the medications we take have varying degrees of side effects, and choosing to take medications is ultimately up a collective decision between care providers, patients and guardians. If the article in STAT feels threatening, then I think you should feel empowered to bring your concerns to the next clinic appointment and discuss them candidly with your care provider.
I was on Symdeko back when it was still in trials (sadly I’m not eligible user now because of my genetic profile) under the name VX661/VX770, and my individual anecdotal case to this article is that I turned out fine!
In fact, I tweeted that out and I got a real kick out of other people with CF adding their Symdeko experiences to the thread.
I think before we rush to judgement and enter a frenzied panic about the Triple Combo’s review under the FDA, we should all take a deep breath (if you’re able… I know sometimes I’m not) and recognize that the real reason this article caught traction was that it briefly moved the Vertex’s stock price.
The unfortunate reality (as with anything living), is that people with CF die. Our medication arsenal can only do so much. We hope that arsenal gets stronger and stronger, so we push the boundaries of science and technology. For that reason, a lot goes into evaluating the safety and efficacy of a new drug, and two of the key inputs are the risks and the rewards. I think the vast majority of people taking Symdeko would tell you that the benefits of these therapies far outweigh the risks – at least that’s what Symdeko users overwhelmingly tell me.
Weighing the risks against the rewards was the very question I asked myself before I enrolled in the VX661/VX770 clinical trial. It’s the same question I ask myself before enrolling in any clinical trial!
I don’t think this news will negatively affect the Triple Combo’s review and the outcome we’re all hoping for in the coming months.
If you’re a data nerd, and you want to look at some trends in the CF world to see how the 30 fatalities stack up against the overall CF population, I encourage you to look at the 2018 CF Patient Registry when it’s released later this year. (Come on, CFF, let’s open up that data to the cystic fibrosis patient population!!!)
The method that I would use to determine whether those 30 fatalities are statistically significant is track the number of fatalities in the years leading up to Symdeko’s FDA approval (February 2018) for the Symdeko population (DDF508) and then compare the post approval number of fatalities. I think the general feeling is that the number of fatalities year to year in that population will go down given Symdeko’s stated efficacy. Granted that method still lacks the larger context of the individual patient health, but it may provide some much needed relief to the headline (if this headline is still a cause of concern by the time the new registry roles out – I personally don’t think it will be).
The lesson here is that it’s okay to dig deep behind some of these big-time headlines, and it’s okay to talk to your doctor about them. Maybe the panic was initially warranted, but after browsing the FDA’s reporting system (linked above), I think we’ll be okay! Personally, I have enough faith in the FDA to protect our collective health – that is their job after all!
