Update: Brainstorm has now abandoned their Right-to-Try approach. Who’s next?
That didn’t take long. That didn’t take long at all. The federal “Right to Try” bill was just signed the other week, and we already have a company that’s willing – no, eager – to try it out. I will now cruelly caricature some of the the bill’s advocates for a moment. They’re imagining cures, live-saving medications, finally freed from the sticky masses of red tape and lethal inertia that is keeping them from desperate patients. Desperate patients who are free actors, informed citizens and consumers, and certainly capable of making medical decisions on their own behalf and spending their money and time exactly as they see fit to spend it.
Actually, that may not be as much of a caricature as I thought, especially the last part. And I would be lying if I said that I didn’t have some sympathy with the people-can-make-their-own-decisions argument. On that ever-present other hand, though, it’s one thing to say that patients should be able to choose among a wide variety of therapies, some of them unproven, with a knowledge of the risks that they are taking. Objections can be raised (for example, that’s it’s very difficult, if not impossible, to be fully aware of those risks with drugs that are still in development). But even stipulating the above, it’s quite another thing to not just open up patients to the whole pool of possible treatments, but to outright encourage people to pour mud and slop into it in hopes that some patients will choose the slop. Which is what I fear we may have done.
I say this because a small company (Brainstorm Cell Therapeutics) says that they would like to make their investigational cell therapy for ALS available under Right to Try provisions. According to that Bloomberg piece, they’re looking at charging in the low hundreds of thousands of dollars (which, to be sure, is probably what their treatment would go for were it ever to actually get regulatory approval and reach the market). No insurance company will pay for this, as you would imagine – they’re only interested in paying for things that have been shown to work. So those informed citizens and consumers that I mentioned above are now free to give these folks six figures for a treatment whose statistical evidence for efficacy is not at all impressive and whose risks are still mostly being discovered. How many will? Bloomberg:
If it decides to proceed, Brainstorm — a company with no drug on the market yet and no revenue — would introduce a profit motive into an effort many expected to be altruistic, adding more controversy to an already contentious debate. Small drugmakers where much of the innovation in medicine originates can’t afford to provide their compounds for free, and terminally ill patients with no other options may be eager to pay for access. There would be little protection for patients already grappling with a tumultuous time in their lives, adding financial risk to the known medical gamble.
Who expected altruism? Not me. In fact, I expect worse than this. Brainstorm, at least, appears to be a company that is actually trying to develop a therapy. They’re not getting very far, but they’ve generated some data by their own efforts. I’m sure that they hoped for better numbers, but that’s drug development, and it’s especially drug development with as unexplored a technique as neuronal cell therapy against as tough a disease as ALS. My guess is that they’re having trouble raising more money by conventional means – i.e., among competent investors who have reviewed their slide deck – so they’re going this route instead, for both money and publicity. This won’t help their clinical efforts at all, honestly – my guess is that many of the patients showing up are well outside the cutoffs for inclusion in a trial, and indeed, the Right to Try law specifies that they have to be.
Can’t the prospective customers be as competent as those investors? In theory, yes. But in practice, said investors spend all their time looking at biomedical opportunities and evaluating them, and have (one hopes) achieved a level of expertise that will take some time and effort to recapitulate. It can be done, though. But the other big factor is that these investors have only their money in the game, not their health and their lives. It is very difficult to make rational homo economicus decisions when you’re evaluating something that could – might – maybe – do something about the disease that is visibly and inexorably killing you or your close relative. This is the factor that, more than anything else, makes buying health care different from any other purchase that we make.
Running behind it, though, is another: the fact that health care varies far more than most other goods that we have available. Want a new car? Some of them are more expensive than others, have different amenities and options, or can do slightly different things. But they’re all new cars: you can buy one in the reasonable assurance that it will not start shedding important parts half a block down the lot, or that (in spite of your best efforts) that it will simply refuse, for reasons unknown, to take you from point A to point B but will dump you instead at point C while you beat your hands helplessly against the steering wheel. Cars don’t do that.
But drugs do. And investigational drugs do that even more than most. That’s what the investigation is for, to figure out if they (1) can take you to point B at all and (2) to see how many times they do other stuff to you along the way. We call those “efficacy” and “safety” in the biz, and we spend huge piles of our own money trying to establish those two. But hey, you can bypass that and take therapies for which neither of those have quite been proven, and Brainstorm will charge you hundreds of thousands of dollars for the privilege. Let’s see who steps right up.
And let’s see if my nastier, more cynical Derek-Against-Humanity prediction comes true: that the next wave will not be just people who have seized on this plan opportunistically, but who have targeted it right from the start. Who will pick out terrible diseases for which there are no therapies at all, the better to insure a supply of completely desperate patients and families, and provide them with utterly useless therapies at stiff, stiff prices. Utterly harmless therapies, though – don’t want to get sued while you’re raking in the bucks. I’m talking polysaccharide therapy for pancreatic cancer, antioxidant phytonutrients for Alzheimer’s: in other words, corn starch and grape juice. What the hell. The customers are going to die, anyway. Why should they die with their money as well?