The Economics of Disposability
I’m halfway through a spreadsheet of gene therapy trial data—Zolgensma, hemophilia, spinal muscular atrophy protocols, cost analyses from three biotech firms, and a patient forum thread that’s been updating since 2018—when I notice something that makes the whole thing click into place.
Zolgensma costs $2.125 million per treatment. One dose. One person. That’s what a hospital spends on insulin for 300 diabetics that year. Nobody’s upset about the comparison. That’s just… the number. The way prices are numbers. The way oxygen has a cost.
The hemophilia trial protocol is thirteen pages. Inclusion criteria run like an exclusion machine: age 12-65, no advanced liver disease, no active infection, no HIV, stable inhibitor history, no bleeding disorder with “secondary complications.” I’m reading the fine print. “No patients with organ dysfunction that would preclude intensive monitoring.” Translation: if you’re already breaking, we can’t fix you and measure it. This is selection posing as science. It’s not cruel to exclude them. It’s just that 67-year-olds with cirrhosis fuck up the efficacy numbers, so they don’t get in.
A parent post from a CMT gene therapy forum: “We’re so grateful. She’s been degenerating for 3 years. This is our only shot.” The post has 47 likes. Gratitude for access to a maybe. The trial doesn’t have to work. The desperation does. They’ll believe it worked because they have to believe it worked—their daughter is in it.
Deeper in the protocol, one footnote: liver function monitoring advised given history of hepatotoxicity in animal models. We don’t actually know what happens to your liver long-term. We’re doing this in humans first because the math on the people who might die is acceptable. That’s not a problem with the trial. That’s the trial.
Investor newsletter headline: “GENE THERAPY APPROACHING INFLECTION POINT. Insurers will absorb $2M treatments if efficacy data holds. Early movers will own the market.” They’re not wrong. If Zolgensma works for 80% of SMA kids, insurance will pay it. Not because kids deserve it. Because 40 years of disability payments cost more. The business logic is cleaner than the moral logic. That’s the only way this scales.
Then I read a Reddit thread. Parent asking: should I put my sick kid on the experimental trial waitlist, or save the money for the kidney transplant that’s definitely coming in five years? Doctor responds: “Put him on the list. If the gene therapy works, you won’t need the transplant money. If it doesn’t, you were going to spend it anyway.”
That’s when it inverts.
The trials aren’t gatekeeping hope. They’re the only hope. The cost isn’t a problem with the system. The exclusion isn’t a failure. They’re what makes the system work at all. Nobody funds this if they have to cure everyone cheaply. No insurer absorbs $2M treatments because it’s the right thing to do—they do it because leaving people broken costs more. The cruelty of the exclusion is the engine. Scale it up, make it accessible, and you break the thing that keeps the lights on.
I’ve been blaming the wrong thing.