A few days ago, I asked for anyone who might know a local doctor who would be willing to take over the management of the investigational drug Francesca is on.
We learned that the company that bought the medicine was ending the Expanded Access Program (which has allowed access to the medicine after the clinical trial ended) and then suggested a couple ways our community might still get to access the drug.
Thankfully no one responded to my post and I didn’t waste anyone’s time because since then we’re learning that the company isn’t actually approving any of the requests they initially suggested patients make as alternate paths. Because of course they aren’t.
So then, why even suggest it in the first place, one might ask? But that’s really only the tip of the iceberg when it comes to the questions I have. And we have asked questions. Lots of them. But we don’t get a clear answer, if we get any response at all.
We’ve heard from the clinic in New Jersey where we were going for our appointments to let us know there will be no more traveling.
We’ve heard from the pharmacy who schedules and delivers our drug to tell us there would be no more deliveries but we could expect to hear more information about sending any unused medication back at the end of the month.
Ha, fat chance.
But that won’t be a problem for us. While some of our community has recently received refills on their meds, we are down to just a week or two left. There will be none to send back by the end of the month. Not that I would.
But we’ve heard nothing from the company who is making the decision to take this medicine away from our kids. No explanation. No clear plan for trying to get approval from the FDA. Nothing.
Now back to the medicine. This is where I get really angry. In what medical scenario is it safe to just stop a medicine cold turkey, without decreasing the dose at least once? Granted, these are small doses – and if I remember right, with this medicine the dose does go down as they get bigger…Francesca is only on 2.8 ml. Still, something about stopping it one day, and hoping for the best doesn’t sit well with me.
Soon Francesca will run out of medicine. And that will be it. She won’t have a doctor following her to ask her about any symptoms we observe like they did while she was on the medication. There won’t be any labs to see how her body may react to the shock of not having a daily substance it has become accustom to for the last almost three years.
And here’s the thing, am I worried? Yup. But am I worried for others in our community more than I am for Francesca? Absolutely.
I’ve been honest since the beginning that this wasn’t a miracle drug for us, but we didn’t need a miracle. We have definitely seen improvements in her math and reading scores and her memory but I also credit that to her committed teachers and IEP.
And we know from her neuro psych testing that deficits still exist. Not to mention, her hand tremors started while she was taking the medication. So yes, I’m definitely worried about what could get worse or regress once she stops the medicine. And there could have been other impacts the medicine was keeping at bay that she’ll no longer be protected from. The unknown that lies ahead is unsettling.
But I’m more worried for those who saw the huge improvements on this drug. Tremor improvements, seizure control, significant memory improvements. Huge quality of life changes. All at risk. It just doesn’t feel fair or safe.
When it all feels like too much I try to remind myself that I always knew this was how it could end. This medicine was never a sure thing. But that’s not what really bothers me. I’m equal parts frustrated at the FDA for not approving the medicine and at Big Pharma for caring more about their lined pockets than the safety and wellbeing of a rare disease community. I do wonder how some of these people sleep at night.
This is what happens when the FDA and big Pharma can’t figure out how to set their own personal agendas aside – our kids suffer. But ultimately how much they will suffer remains to be seen.
I am so angry. I am terrified. I am weary.
Francesca gave the last 5+ years of her life to this clinical trial. And some gave longer. And for what? To have the medicine taken away with less than two weeks notice and no safe dose reduction plan and follow up protocol?
Without an actual call and explanation from the company making the decision?
Complete insanity.
And while I might not feel okay, I know we will be okay. Because I believe in a God who goes before us, who is working even when we can’t see it and who will not let us down.
Medicine or no medicine Francesca and our entire community will be okay. And I’m praying that something bigger and better than we could have ever imagined is around the corner.
And in the meantime, we’ll still be here advocating for our community, holding events like Glow to raise awareness and funds and supporting the work of the Galactosemia Foundation, as a board member and as a mom. And those dual roles have had me walking a fine line the last couple weeks, which I will continue to navigate carefully.
So while I am sad and angry I am also doing my best to be respectful. We are rare but we are mighty and we deserve a treatment. And I will not be silent. Whether a government entity like the FDA needs to hear it or big Pharma like Cycle. You can bet I won’t back down when it comes to our kids and our community.
“We are children that need to be loved
We were willin’, we came when you called
But man, you fooled us.
Enough is enough…
It’s the start of us, waking up, come on… Are you ready? I’ll be ready.”
-P!nk and An Exhausted, Pissed Off Mom With a Cause