Skip to content

Bastard profiteering drug companies!

But 19-month-old Teddi is now a happy, healthy and chatty toddler after becoming the first baby to receive a new £2.8 million cure on the NHS.

An horrendous story – her older sister cannot be cured the same way, it’s too late in the progression of the disease.

But of course, the bastards, £2.8 million?

Around four babies a year in England are born with MLD

The treatment has cost somewhere between $1 and $2 billion to bring to market. Much of that is in the testing process. Well, we can all have arguments about whether the testing process should cost that much and all. But that is about the cost of bringing a new drug or treatment to market.

Whether it’s capitalists in pharma companies or taxpayers doing the work, it’s still a billion and more. So, the cost of a treatment is that £2.8 million or around and about. Where the money comes from doesn’t change that the resources that had to be devoted to getting the treatment into use are the resources that had to be used to get the treatment into use. That’s simply the cost.

Because any protection on the treatment – patents I would assume here – will last about 10 years of the treatment’s life. After that it becomes a generic. So, the first 10 years of treatments must pay the costs of development. The UK is about 5% – -ish, -ish – of the rich world population that will even think of applying this treatment, so there are perhaps 80 kids a year globally who are going to take it. 800 over the decade, $2.8 million = £2 billion and change. Total revenue from something that cost $1 to $2 billion to develop.

Note the important point here. This drug costs society £2 billion – -ish, -ish. Whether this is taxpayer funding through the development process and then free at the point of use. Or capitalists developing and then taxpayers paying through the NHS. Or even capitalists and then out of pocket. The cost of saving these 800 lives is £2 billion.

The allocation of the costs is interesting, the cost itself is just the universe stating a fact.

16 thoughts on “Bastard profiteering drug companies!”

  1. Tim. You’re reminding me of the complaints of those involved in the earthquake in Turkey. Had more resources been available lives would have been saved. Unfortunately you have to draw the line somewhere.

    As you’ve guessed, I always want it to be drawn so I’m above it.

    PS. I did read that Erdogan had ordered that the armed forces couldn’t take immediate action without his ok. Being scared of a coup. Well, I’m selfish too.

  2. That £2.8 mil? How does that compare to insurance payouts for deaths and the like? And, that’s £2.8 mil to extend a life from about 6-8 years, to what? 70 or 80? The pay-off in QALY must be huge.

    Or, what’s the cost of care for MLD from age 3 to death at around 6-8?

  3. It really is time to connect cost of medical intervention directly to those receiving it.

    It is an inherited disease, so evolution being what it is, arranged for it to be fatal so the likelihood of it becoming more widespread in the population is very limited.

    But I suppose a ‘cure’ will certainly help increase the future need for the treatment to give a decent return on that £2.8 billion invested.

    Then people wonder why healthcare is so expensive and there are long waiting lists.

  4. JohnB: the more interesting question (scientific not economic) is why the recessive gene persists enough in the population that two carriers can get together sufficiently often. Like the other more common recessives (CF, Sickle) it probably has some benefit to carriers.

  5. I’m going to be brutal now.

    If the £2.8m hadn’t been spent presumably the child would not have lived.

    In the light of the condition apparently being hereditary what if any restrictions should be placed on her having children in due course (in the unlikely event that the nhs is still going at that time).

  6. @John, there’s a eugenics society for Jews in NYC. Courting couples volunteer to be tested and can then be warned not to procreate if the DNA shouts a warning. I suppose if the like were introduced here there would be immediate claims of racism and Islamophobia.

    (Why you would need testing to tell people to avoid multi-generation first cousin marriages is unclear. Or uncle-niece. Or …)

  7. In Iceland they have a phone app which can tell them if they’re related closely enough to risk getting two copies of a recessive gene. I’m told it’s quite popular among the youth.

  8. Bloke in the Fourth Reich

    Questions in a thread we can answer.

    John: no more restrictions than should be imposed on you as carrier of around 20 recessive lethals.

    Ducky: No, the replacement gene is added to blood stem cells, it does not affect the child’s eggs. Any children would be obligate carriers, or themselves affected if her partner is a carrier.

    Tractor: at this level close to nonexistence mere stochasticity can keep them in existence. With larger genes like FVIII de novo mutations entering the population at a rate in equilibrium with which they are Darwinned out do it to some extent.

    JohnB: given the disease affects perhaps 20 people in the country you’d have to treat a lot of people and they’d have to have a lot of kids, over centuries, for it to make any difference to the population allele frequency.

    Tim: (Direct) development costs for this therapy will have been substantially below $1bn. The proof of concept (if it holds, previous gene therapies have had a habit of becoming ineffective over time, but they have a trick or two for that), will be invaluable and unleash a whole slew of new treatments. Orchard aren’t the only company doing the same thing. Also the treatment cost is never going to fall in the way generics do because it is fundamentally expensive and risky to do.

  9. Development cost sounds a bit on the low side – ZOLGENSMA, invented by Novartis, is a one-off gene therapy treatment for spinal muscular atrophy (SMA), a very rare degenerative disorder. Infants with it usually die within two years.

    Quoted development cost was $8.7b patient population estimated as 250 cases a year worldwide and current quoted price $2.1m per patient

  10. @BiFR – if the per-treatment cost remains high, it follows that the portion of that 2.8 million which pays back investment is lower. What ballpark would you put that difference in?

  11. Timmy, you’ve skipped over an important detail.

    In housing, lefties believe that local authorities should build lots of council flats. They assume that the council’s planning department will automatically grant planning permission to the same council’s housing department. Benefits of doing work in-house, or something.

    By the same logic, the NHS should open its own drug-development labs. It could skip all those expensive phase II and phase III trials, and just experiment directly on patients. Yes many would die, but it’s for the greater glory of our NHS.

    The general idea of “let’s do it in-house” is wildly popular even amongst mild-Lefties, such as BBC employees. It means job security & empire-building.

  12. Bloke in the Fourth Reich


    How do you apportion development costs for something like this?

    The cost is in the proof of concept, lentiviral transformation on top of autologous stem cell transplant (an already established and widely used therapy in its own right). But the transformation took years to get right, probably a lot of it went on in taxpayer-funded labs rather than industry. In terms of development costs it’s not the payload that matters, it’s showing you can get DNA into the cell nuclei and that it works once it is there*.

    This new mechanism, however, could be applied to an almost unlimited range of rare genetic diseases, so its value is immense. However, the clinical research for each individual disease will be of rather limited cost because of the limited patient numbers. Some of these rare disease programmes end up recruiting pretty much the entire known population of patients with the disease being studied. The first gene therapy I worked on was licensed after a trial in (IIRC) just 17 patients – a current programme is going to struggle to reach that number. This rather limits the clinical development costs of any individual therapy to those of manufacturing the treatment (ok, individually not cheap with stem cell transformation) and writing up the very straightforward (due to limited data) submission.

    The clinical POC in any individual indication ultimately needs long-term data which isn’t going to be available. But it seems likely, as with SCT you can pretty much entirely wipe out residual bone marrow so there should be little risk of need for repeat treatments, or untransformed stem cells eventually replacing the transformed ones. The (not inconsiderable) risks of SCT are obviously worth taking with a disease that is always lethal in early childhood.

    *: Same with the mRNA vaccines. While the population wide use of these is highly debatable there’s proof that the idea works at least, and in theory one could adapt this to almost any antigen you want. The covid vaccine’s value to Pfizer is more in establishing it works (for covid to a very limited but not zero degree), thus the potential it opens up. Plus, unless the safety thing rebounds properly (which I think it will, eventually), making it a societally-acceptable technology.

  13. Bloke in the Fourth Reich


    Of course NHS hospitals participate in drug trials all the time. Several of the big CROs rent office and treatment room space at hospitals to facilitate this. That “experimenting directly on patients” is actually the phase II and III trials very typically performed at NHS facilities.

    What the NHS doesn’t do is the preclinical work and early phase work to get to a phase II trial, or the financing of drug development. Though I’m sure some would be able to argue that the government central health care provider should be pouring cash into speculative new treatments, I don’t agree.

Leave a Reply

Your email address will not be published. Required fields are marked *