I think the answer is that many can’t afford million-dollar drugs and must forgo treatment. Examples of expected million-dollar (more or less) drugs are the upcoming ASOs for Huntington’s disease and for SCA3, based on the recent market. Even with stellar insurance, the copayments (i.e., from a few thousand to a few tens of thousands of USD, maybe more) and other incidental costs (e.g., insurance generally does not cover the cost of administering drugs into the brain or spinal canal) keep the drugs out of reach for many, perhaps most.
Let’s look at the example of the most expensive drug ever produced (though not for the U.S.): Glybera to treat LPLD. Only 31 people were treated with it, and worldwide, the number of people with the disease is about 7,500 (SCA is 50 times as prevalent), meaning that fewer than 0.5% of people with the disease were treated. Yet it was apparently financially viable to develop and sell the drug. The drug company (uniQure) is still around, in fact with the most promising SCA3 drug to date in their pipeline.
Simple math indicates that 31 people paying USD 1.2 million yields about the same as 7,440 paying USD 5,000.
It’s a catch-22
The goal of the U.S. Orphan Drug Act of 1983 was to financially motivate companies to develop rare-disease drugs that would otherwise not be financially viable, but it does not consider consumer drug pricing. In fact, in the U.S., drug prices are unregulated. The year 1983 was years before drugs started being developed for genetic diseases, of which there are thousands (of diseases).
A pharmaceutical company that successfully brings a million-dollar orphan drug to market can bring in billions of dollars per year.
The consumer cost of the kind of drugs developed that the Orphan Drug Act has encouraged is now exceeding USD 1 million annually per person treated. There might be few enough drugs now that most of the cost is willingly paid by insurance providers and possibly charities (i.e., private donations)—or in the case of Medicare, not allowed to not pay (but it’s not that simple; a 5% copayment on USD 1 million is still USD 50,000 and out of reach for most). As more drugs become available, the system is destined to break. It is unsustainable.
Perhaps some will realize that the time to treat a genetic [brain] disease is not after birth and switch focus to the only truly effective approach of genetic [brain] disease prevention at conception, for subsequent generations. At the least, hopefully ataxians will understand that these expensive drugs aren’t disease cures, just disease modifiers.
Ataxians will need to come face to face with million-dollar drugs not being covered because they are symptomatic, and yet many won’t understand because of decades of misleading distortions about their condition and its modifiability via support groups and cheerleading neurologists. If they want the million-dollar drugs drugs to be effective, they’ll need to treat their asymptomatic children, not themselves. They’ll need to finally face the fact that drugs based on ASO and RNAi do not aspire to cure disease but only reduce its impact on those currently asymptomatic. For example, I assume that READISCA is all about identifying those with a genetic defect before they develop symptoms, i.e., identifying those with SCA1 and SCA3 who are candidates for ASO and RNAi.
Oh, what a big market you have
If the rare-disease drug market is 20% of the drug market, and if the size of the U.S. drug market in 2018 is USD 500 billion annually, then the rare-disease drug market in the U.S. is about USD 100 billion annually. True, a much smaller fraction of that is in the super-expensive realm. It appears to me that public insurance (e.g., Medicare, i.e., the U.S. government) and private insurance companies are somehow footing most of that enormous bill for now. But what about in upcoming decades as that total grows by a factor of 10? Maybe the insurance providers can miraculously absorb USD 100 billion, but USD 1 trillion (a million millions)? Impossible. Eventually there’s going to be pushback, maybe even a system collapse and/or overhaul.
The amount of money we are talking about is in the same ballpark as U.S. student loan debt, which is about USD 1.5 trillion total, and about USD 40 thousand is the average owed.
Crazily rich ataxians
In 2015 or so, we started exceeding USD 1 million annually for gene-based drugs. We can attempt to look at how this is currently handled to see what we can possibly expect for SCA.
First, it’s worth wondering if any patients forgo insurance and pay full price out of pocket. Perhaps, though I doubt it. For an individual to be willing to pay USD 1 million annually for something without incurring debt or destitution, they’d need something like USD 25 – 100 million in the bank. The prevalence of people with that much money in the U.S. is about 25 per 100,000, or about five times as prevalent as SCA itself. In the U.S., we might expect there to be up to four people (5 per 400,000,000) “superrich” with SCA. They probably didn’t get superrich by paying full price for everything, but who knows? And I think in practice the number of crazily rich ataxians is zero. Furthermore, to sink that much money into a mere disease modifier would be, simply, insane.
My assumption is that no patient pays full price. Thus, when I see on the web: “One of the reasons they charge such high prices is so they can help people who can’t afford it”—I assume this means nobody pays full price. In any case, it’s certainly nearly nobody.
We seem to be highly dependent on the drug companies “teaching” insurance providers (especially Medicare in the U.S. and the NHS in the U.K. [example]) that they must cover their drug. What happens if there’s contention of any kind, I don’t know. This ensures that drug companies get their money for their orphan drugs, but I can’t imagine why private insurance providers would agree to pay, as that doesn’t fit their business model of denying claims.
I’d expect covering drug costs to get more difficult as more million-dollar+ drugs come into existence for more diseases—i.e., as rare becomes less rare, and insurance providers truly can’t afford covering not-so-rare drugs without going bankrupt.
The devil is in the details
Here’s another example, from 2016/17. An ASO called Spinraza for SMA (spinal muscular atrophy, about twice as prevalent as all SCAs combined) became available at the cost is USD 750,000 for the first year, then USD 375,000 annually, for one’s lifetime.
Continuing with the example, here is a large glop of financial assistance details, perhaps similar to what we’ll encounter for SCA.
Related
2022-11-23. “FDA approves world’s most expensive drug [Hemgenix = etranacogene dezaparvovec], a hemophilia B treatment [from uniQure] expected to cost $3.5M“
2019-12-07. The New York Times: “Two New Drugs Help Relieve Sickle-Cell Disease. But Who Will Pay?”
2019-08-23. The New York Times: “Life-Changing Drugs Almost Nobody Can Afford”
2019-08-13. The New York Times: “This Drug Will Save Children’s Lives. It Costs $2 Million.”
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