I Approve of Orkambi, But Not of the Price

Orkambi—the new Cystic Fibrosis drug from Vertex Pharmaceuticals—just received FDA approval. I was hoping that it would, because it’s a fine drug. That’s my review of it: it’s fine. It does some things very well—my lung function is currently hovering around 77%, which matches my adulthood high from 2009—while other things—my digestion, diabetes and sinuses—haven’t responded as positively. If you’d like, I wrote a whole bunch of words about my experience here, here and here. Most of that still applies, though I’ve since switched digestive enzymes and added a long acting insulin to my routine, both of which have been positive changes. With my new enzymes, I’m able to get away with around 240,000 units of lipase per meal, which is down from the all-time high I hit during the study—300,000, a number that’s probably unsafe–but still above my pre-study number of 200,000 units.

The point of this is that Orkambi is fine. It’s not a miracle drug and it has yet to replace any of my other drugs. In fact, since joining the Orkambi trial—I’ve been in the open label study for a little over a year now—I’ve actually increased the amount of insulin, enzymes and omeprazole I’m taking. But I cough less and bring up less mucus, so that’s one in the win column.

It is not, however, 259,000 in the win column.

According to Reuters, that’s the wholesale price of Orkambi: $259,000 a year. Holy shit. I understand this is a niche product and I understand a lot of money went into development, but my statement stands: holy shit.

Let’s do a thought experiment:

My pulmozyme costs about $19,000-20,000 a year. It thins my mucus and makes it easier for me to cough it up. If you offered me either $20,000 or a year’s supply of pulmozyme, I would take the pulmozyme, no questions asked. In fact, I once put a month’s supply of pulmozyme–$1900–on my credit card because my insurance wouldn’t pay for it. It probably took me years to pay that off, but I don’t regret it.

My digestive enzymes cost about $50,000 a year. They make it so I can digest food. If you offered me either $50,000 or a year supply of digestive enzymes, I would—without any hesitation—take the enzymes. They are a “rob a bank” drug, in that I would go straight up John Dillinger to get them. Without them, I’m as good as dead.

Since starting Orkambi, my lung function has returned to my adulthood peak (about 77% capacity). This is big, because usually these things roll downhill. However, I only know this because I’ve seen my clinic numbers: I do not feel any better. My face is fatter, but my weight was stable before I went in the study anyway. So if you offered me a year supply of Orkambi or $259,000, I would take the money. $259,000 could buy me a small house and/or one of those flaming Mad Max guitars, either of which I’d give up a little lung function for. I know what it’s like to live with CF; I do not know what it’s like to live with $259,000.

And I will never find out, because I’ll never get that money. Assuming my insurance pays for it, I’ll probably stay on Orkambi and I guess that’s my problem with it. It’s a drug that I don’t think is worth what they’re looking to charge, but I won’t stop taking it.

For me, CF comes with a functional superstition. I’m scared to change any part of my routine because I’m scared it might kill me. Sure, I could stop taking Orkambi and I might be able to take fewer enzymes. Maybe stopping Orkambi would let me lower my insulin dose and bring my testosterone back into the normal range. But maybe then my lungs will crap out and I won’t be able to work anymore and they’ll throw me in debtor’s prison. And I’m coming at this from a priveleged position: I was stable before I went into the study. I can only imagine what the idea of Orkambi looks like when you’re sleeping on death’s doorstep. I bet $259,000/year looks like a bargain then. But how far away from that doorstep does Orkambi get you? $259,000/year would look like a bargain to me if Orkambi replaced any or all of my current routine. I’d say I’m worth that. But I’m not sure I’m worth $259,000 + $100,000 for everything else.

Insurance wise anyway. When it comes to salary, I’m worth at least double that.

Am I wrong about Orkambi? (I hope I am) Tell me in the comments.