A lovely story. In that sense of being loverly and explanatory. For in reality this is an appalling and vile little tale.
So, there’s a genetic disease - failure perhaps - which kills little kiddies. They’re fine up to toddler stage then gradually decline into a vegetative state and then die by about 7 or 8. The problem is something to do with myelin, nerve coverings, brain cells and so on.
Simply vile. And as far as is observed cases run in families - well, obviously, genetics and so on. But a couple who have one seem likely to find that some to all have it. It’s less of that dice throw that the next will have it than in many other such genetic bloopers. And, of course, that means that having had one diagnosed means that the toddler little brother or sister is highly likely to also be so. Or, having had one child that vegetated to death by year three of school that the seemingly healthy toddler will also do so.
Quite apart from anything else this is probably a pain we’d like to alleviate those parents of. As well as the children of course although as should be obvious they’re going to be less aware of what is happening.
Sure, sure, we all know that life begins in nappies and, if we live long and hearty, ends in them too but imposing upon parents only a three or four year gap before it happens again and that white coffin has to be ordered? We really would like to alleviate this if we could.
Well, we can. And that’s where the problem is.
In a medical triumph, the US Food and Drug Administration on Monday approved a gene therapy that appears to trounce a rare, tragic disease that progressively steals children's ability to talk, move, and think, leading to a vegetative state and death. For those who begin to slip away in infancy, many die by age 5. But, with the new therapy, 37 children in an initial trial were all still alive at age 6. Most could still talk, walk on their own, and perform normally on IQ tests, which was unseen in untreated children. Some of the earliest children treated have now been followed for up to 12 years—and they continue to do well.
But, the triumph turned bittersweet today, Wednesday, as the company behind the therapy, Lenmeldy, set the price for the US market at $4.25 million, making it the most expensive drug in the world. The price is $310,000 higher than what experts calculated to be the maximum fair price for the lifesaving drug; the nonprofit Institute for Clinical and Economic Review, or ICER, gave a range last October of between $2.29 million to $3.94 million.
No, that’s not the problem. A difference of $300k isn’t the problem in the slightest. It’s the $4 million that is. For it is caused by this:
occurs in about 40 children in the US each year
That’s where the problem is. And the problem is nothing to do with capitalism, patents on drugs, greedy bastards or anything like that. It’s just an inescapable part of the costs of drug development.
Capitalism and patents might make the problem clearer, but they don’t change the nature of the problem.
So, our current method of drug development is that research work happens in research places. Often these are government supported, universities, or more direct grants. But that’s not where the vast costs occur. Those are in the proof - the running of the clinical trials that show that it actually works.
A useful estimate of those costs is some $2 billion. OK, we can cut that for our little economic model by ignoring opportunity costs - what someone else might have done with the money if it wasn’t this. But then, as we all know, if you’re not including opportunity costs then you’re not doing economics. Or we can insist upon not including interest costs. Or perhaps we insist that successful drug development should only pay the costs of the development of that drug, not also make a contribution to the costs of those that don’t work. Sure, we can do all of these things and make our costing less and less accurate - but we would still be talking of $800 million to $1 billion.
Which is, you know, a lorra money. Which has to be earned back somehow. Which is why patents on new drugs and treatments. So that the developers can make back their money and then, when the patent expires so the drug becomes cheaper and huzzah.
But it’s still true that this capitalist and market based system doesn’t change our basic problem here. It costs a $billion - even if everything were govt paid - to develop the new treatment. And is that worth it? If society pays that in taxes, government allocates the money perfectly (Ha!) and the drug is then issued free of charge to all who need it - there’s still that $billion spent upon getting to that point. And is that worth it?
Wayull. It’s obviously true that $4 million on rice beans and oral rehydration therapy will save more lives in Haiti than treating one child in the US. Rather more so for $1 billion. So, there’s that opportunity cost - and no, insisting that we all spend everything on dealing with such problems isn’t going to work either. Largely because it’s not going to happen, so there.
There’s one further little wrinkle here. Maybe we should just leave the kids to die?
Stat notes that Orchard has previously abandoned four therapies for other rare genetic conditions because of the difficulty in meeting regulatory standards for essentially custom therapies and questions about whether health plans will pay the steep, multimillion-dollar prices.
Well, we are. These other kids, with these other rare genetic conditions, are being left to die. The cure is there even if not proven to the required standards. And it won’t be applied because it costs too much to prove it to the required standards.
Which is what the complete bugger is here.
Nope, it’s nothing at all to do with capitalism nor profit, not even patents. For that $billion cost is the same whatever financing system we use. The problem we face is it worth it - is it righteous and just - that we spend a $billion on 40 kids a year? We can even take the money out of it - as some would insist - and just talk of real resources. That much attention and time of lab space, reagents, skilled researchers and so on who could be doing something else (again, there are always opportunity costs).
Nope, I dunno either. A complete bugger the entire question. I can think of mild guides. Now that we’ve got this solution we probably should go spend the money on the treatments for this one. For no better reason than I’m not willing to stand in front of those parents and tell’em, well, no, we ain’t spending that. We might want to think about reducing the costs of proving a new medicine. It’s long been said that the FDA imposes far too much cost. We could certainly cut that in half, near certainly much more. But that just changes where the line is, not the existence of such a line - die/don’t die on the grounds of expense.
But the actual solution to the whole problem? It’s not just that I don’t know nor does anyone else. Because, sadly, it’s a right bugger of a problem.
Anyone? Bueller?
We're looking at around 25 million USD per child, every year I think, until that child grows old and dies from something else. Or is it 25 million USD one off. Either way, such a sum could save far more residents of Haiti or the DRC or failed asylum claimants from appalling lives.
The best outcome is one where the residents of CA or TX or wherever can voluntarily donate to those drug costs if they think those American lives are worth it. If you personally think an American child's life is worth a significant multiple of a life in DRC, you'd be mistaken imv, but you should be permitted to hand over your cash according to your belief.
In other words keep government out of it: don't rook the many for the benefit of the few, set the people free to donate to FundRareDrug CIC voluntarily if they want to.
I know bugger all about the testing regime for new medical treatments, but it seems obvious that it's main purpose is to ensure that the treatment isn't actually worse than the disease. If the treatment is going to be applied in millions of cases a year, it's arguably worth spending billions to ensure this is the case. But for diseases that affect a relatively tiny number (and where the disease itself is pretty ghastly), it looks as though a less strict process would produce a net benefit.