Gene therapies are being priced according to the annual costs of treatments they replace, not the cost of producing the gene therapy product or the value of the cure to the patients.
For example, Hemgenix is a gene therapy for hemophilia. It is a single-dose product priced at $3.5 million [1]. It's not CRISPR-based, so it's not an apples-to-apples comparison with the sickle-cell treatment discussed in the article. But I know from officials at the World Federation of Hemophilia that the direct costs of production and administration for Hemgenix are around $50,000. That's two orders of magnitude less than the list price.
The $3.5 million figure was likely arrived at because the existing products for hemophilia cost north of 350k annually, and Hemgenix is estimated to replace them for 8+ years.
> But I know from officials at the World Federation of Hemophilia that the direct costs of production and administration for Hemgenix are around $50,000. That's two orders of magnitude less than the list price.
That doesn't account for the R&D effort which has to be recouped during the runtime of the patent. Depending on whom you ask, that can reach into billions of dollars [1], and there's just about 200k people diagnosed with some form of hemophilia of which only a fraction has the funds or the insurance to obtain that kind of treatment in the first place (both the current and the new one), so these few patients have to account for the R&D cost of the medication, the R&D cost of failed candidate substances, the R&D for ongoing other medication and profits. For "orphan diseases", these economies are a serious problem.
Pharmaceutical development is incredibly expensive; to make it worse a lot of governments have cut back drastically on fundamental R&D grants for universities and so private companies with their profit interests stepped in.
Noted and agreed on R&D costs (especially failed candidates).
> there's just about 200k people diagnosed with some form of hemophilia of which only a fraction has the funds or the insurance to obtain that kind of treatment in the first place (both the current and the new one)
This was actually the crux of the discussion where I learned about the $50k figure. There are a lot of hemophiliacs in countries that spend, on average, just a few hundred dollars per capita each year on health care. Even if Hemgenix went royalty-free right away and no one ever turned a dollar of profit on it, gene-therapy cures are still inaccessible to the bulk of people the World Federation of Hemophilia represents.
$3.5 million would be like landing on the moon, and $0.05 million would be like a few orbits at ISS height, but they're both astronomical. They're equally out of reach for most people with severe hemophilia.
Given the resources consumed by sickle cell patients, who often require multiple lengthy hospitalizations I bet it would be close to breakeven even at this price
In fact this is exactly how they set the price for these curative treatments. Gargantuan sums but hypothetically on average cheaper than the overall lifetime costs of living with the disease. Lots of recent debates on this in the world of Hepatitis C.
pricing like that only lasts as long as the patent, the argument being that without the opportunity for that pricing, the research for the drug would not be worthwhile in the first place.
I'm not arguing this is true, but I wouldn't accept a counterargument based on "but it's people, it's people, think of the children"
- Most formal drug research, today, is already not worthwhile. The norm is that you spend several billion dollars and recover nothing.
- Most achievements that were worthwhile in retrospect were not worthwhile prospectively. Therefore it is not obvious that things need to be worthwhile prospectively in order to be done. Going into a career as a rock star is, objectively, stupid. But we have a huge supply of wannabe rock stars anyway.
A more charitable interpretation might be that getting under the cut off is why it's one of the first treatments available. More treatments will become available as the overall costs start beating out the costs of living with $disease.
The recent curative hepatitis treatments have paved the way for this. There are a variety of different approaches including statewide cost sharing funds that pool the cost across all the insurers in the state.
IIRC, single-payer health care systems like the NHS do functionally the exact same thing, which allows them to negotiate significant discounts. I remember reading about a gene therapy for a type of leukodystropphy that was in the millions of dollars, but got negotiated down. [1]
As with everything though, prices for experimental procedures and devices are vastly higher than the asymptotic cost of production. There's nothing about a CRISPR process that cannot in principle be automated, it's just that right now it involves an army of lab techs with pipettes.
The Human Genome Project to read a full copy of the base pair sequence in one human started out with about 3 Billion dollars in initial funding, and it took a couple of decades to get complete coverage.
However, the technology, techniques and equipment developed to automate the process have massively cut costs and increased speed over that time.
> A new speed record in DNA sequencing may soon help families more quickly find answers to difficult and life-altering questions.
In just 7 hours, 18 minutes, a team of researchers at Stanford Medicine went from collecting a blood sample to offering a disease diagnosis. This unprecedented turnaround time is the result of ultra-rapid DNA sequencing technology paired with massive cloud storage and computing.
Doesn't 23andme only do localized sequencing for particular SNPs that are relevant for genealogical investigation? I didn't think they did full-genome sequencing.
they used to offer full data download but it looks like they disabled that. Nebula Genomics is offering to do it for $500 with black Friday sales, which is close.
They still do (update: not they are not). It is not and was not a full genome sequencing, but rather a SNP genotyping. Current 23andme raw report (using their v5 chip) contains around 24K SNPs.
We don't have a lot of experience with taking cells out of the human body, processing them, and then putting them back in. A significant amount of the cost, above and beyond the R&D required to develop the treatment and the cost of actually performing the cure, comes from extreme quality control measures that reduce the chances of infection and other problems. Also, you have to basically run a full, certified lab and house it with people qualified to perform the cure.
Everything about this is bespoke (each treatment is based on the individual cells of a patient) and artisanal (the process requires a highly skilled individual to complete), and run by a guild (only so many people are allowed to run gene therapy trials).
On the other hand, since this is a cure, and probably reduces long-term medical care costs, it could very well actually be "cheaper" in the long run, which incentivizes the government to treat as many people as possible.
That's a complicated topic, but the short version is cost of this treatment needs to cover the R&D costs to develop it, plus the costs for all the other failed experiments that didn't work out.
Plus gene therapy is in its infancy, so everything here is going to be novel and expensive. Novel treatments generally have a limited audience with even fewer practitioners, so you can't spread the R&D costs across many patients.
Prices will go down with time as patents expire and competitors emerge and this line of treatment (perhaps) becomes mainline, but that's on a ~20 year timeframe.
Because right now, it's a very bespoke process. Very low supply, with a lot demand.
As more people become proficient at this treatment, the cost will come down. But to truly get it down to reasonable amount will require automation, and we're a long way off from automating crispr.
I assume it's a long and labour-intensive process?