[HN Gopher] Embracing Bayesian methods in clinical trials
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Embracing Bayesian methods in clinical trials
Author : nextos
Score : 68 points
Date : 2026-03-24 03:08 UTC (3 days ago)
(HTM) web link (jamanetwork.com)
(TXT) w3m dump (jamanetwork.com)
| doctorpangloss wrote:
| > Although an adaptive design guidance finalized in 2019 left the
| door open for bayesian trials, their use in drug development has
| to date been limited,4 such as in Ebola and SARS-CoV-2 epidemics
| and in pediatric and rare disease trials.
|
| the reason it has been limited to those cases is
|
| drug development, today, is constrained by commercialization.
|
| all four categories listed - ebola, sars-cov-2, pediatric and
| rare disease drugs - each for their own reason, have low
| commercialization risk, so if they are scientifically robust
| solutions, there is ROI.
|
| most drugs in development today are new indications for existing
| molecules, generally in oncology, also because these are the most
| favorable conditions for commercialization.
|
| commercialization, commercialization, commercialization. people
| don't want innovations in drug approval, nobody is clamoring for
| that. they want innovations in commercialization.
|
| i don't _know_ if bayesian methods will make trials a _lot_
| cheaper. and that 's their problem! there are already a lot of
| very smart people working on this issue, and they have litigated
| to death all the objective facts.
| cogman10 wrote:
| There's a weird thing that happens with cancer drugs that I
| just experienced.
|
| When they are working with a pharmacy and your insurance, the
| price they'll charge you is $10,000 for enough pills to last a
| month. But when your insurance says "We won't cover that" all
| the sudden you find out the company has a backdoor
| subsidization program which will fully cover the cost of the
| drug for reasons I can't really fathom (good will?)
|
| What's even more uncomfortable is my insurance (aetna) also
| mandates that I get my medicine from their subsidiary, CVS.
|
| I really don't like this sort of thing. The price of everything
| in medicine feels distorted in unbelievable ways. Like famously
| a $0.25 acetaminophen pill that somehow magically costs $10 in
| the hospital. I guess there's some nice individual packaging.
|
| I'm less and less convinced that it's even a cost sort of thing
| with these pharmaceutical companies. Like, sure they'll love to
| reduce that as much as possible. But the price itself seems
| entirely fictitious and based on what they can commonly get
| insurers to pay, and not anything related to the actual R&D of
| the drug.
| ggm wrote:
| The mutability of the price emphasis the cost/price disjoin.
|
| Probably you are a soft recruit to "they got better' numbers
| for post trial marketing but even then, it's like a Ford is
| $30,000 or $3 depending how easily it can be sold.
| doctorpangloss wrote:
| > I really don't like this sort of thing. The price of
| everything in medicine feels distorted in unbelievable ways.
| Like famously a $0.25 acetaminophen pill that somehow
| magically costs $10 in the hospital. I guess there's some
| nice individual packaging.
|
| okay well, let's say you were to model "what explains the
| price of something" and you had two factors in your model,
| "markets" and "politics." most good models will have a
| residual which means "everything else."
|
| when i say "low risk" to commercialization, i could mean a
| lot of things. it depends on what the drug category is.
|
| if you develop the only cure for an otherwise fatal,
| pediatric, congenital disease, the parents will be willing to
| pay an unlimited amount of money for it. so there's no market
| price for it. so we know that, no matter what, a correct
| model for that is, 0% market. this is really, extremely
| difficult for most people who look at this issue, because it
| renders their opinions about what should you pay for this
| drug basically moot. they'll all have different answers. so
| the numbers of our independent variable, "price people are
| willing to pay" will be uncorrelated with the price people
| actually pay. and this is one of those instances where the
| absence of correlation means no causation. instead of looking
| cogently at this situation and saying, "well, okay, maybe i
| should temper my outrage about drug pricing in this actually
| very important scenario" they just get more outraged.
|
| so what factors are left: political and everything else?
| well, the drug is effective, it's a cure, this describes a
| lot of drugs. that's what we are talking about, effective
| drugs. the FDA process has ALSO attacked the importance of
| market pricing from this angle too, the clinical trials
| process here and how it has been standardized around the
| world basically works. so the residual, if it's about the
| efficacy of the drug, actually does NOT explain the price
| very much at all. this is counterintuitive!
|
| this is why a CURE for a disease is not 10x more expensive
| than something that already exists and manages a disease.
| so...
|
| what's left? my guess would be, the price would be explained
| by, okay, a lot of it will be political. like 90% political.
| this is what i mean by "low risk of commercialization."
| nobody is going to win an election being like, your kid
| should die.
|
| are you getting it now? you are getting hung up on prices.
| you don't know the first thing about prices. you think the
| journey to understanding prices has to do with "$0.25
| acetaminophen pill that somehow magically costs $10 in the
| hospital" and hospital charge lists or whatever, which is
| 200% wrong, that's a huge red herring.
|
| prices in categories that are low commercialization risk are
| almost always explained by a political process. which is to
| say, all the innovations today in commercialization are
| political. that's bad. but that should also illuminate why,
| if I were running the FDA, that's where my focus would be -
| besides, at the rest of the HHS, 10x the basic science R&D
| budget.
| selimthegrim wrote:
| Isn't the usual answer here to try and segment the market
| and even if your initial guess is horribly wrong price
| discovery will follow?
| nextos wrote:
| > the reason it has been limited to those cases is drug
| development, today, is constrained by commercialization.
|
| That's a good observation, but I think it's an incomplete
| picture. Another important constraint is often regulatory
| inertia and historical baggage.
|
| The UK pioneered small classical and adaptive trials using
| Bayesian methods, and there were some promising results. A lot
| of modern Bayesian methodology was, in fact, developed at the
| MRC BSU Cambridge with this goal in mind. For example, the
| probabilistic programming language BUGS (1989).
|
| Given that most drugs fail, the industry is highly incentivized
| to use Bayesian methods to fail faster. These models allow for
| more rapid dose-finding and the ability to distinguish
| promising leads using interim data, which is vital given the
| massive cost of any trial, especially late-stage failures.
|
| But for Bayesian methods to make a dent, they'd need to be
| applied to a large number of trials, and change doesn't happen
| overnight. Lots of big pharma players, e.g. GSK, are becoming
| interested in moving to Bayesian methods in order to leverage
| prior information and work better within small-data regimes.
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