[HN Gopher] First malaria treatment for babies approved for use
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       First malaria treatment for babies approved for use
        
       Author : toomuchtodo
       Score  : 113 points
       Date   : 2025-07-08 15:16 UTC (4 days ago)
        
 (HTM) web link (www.bbc.com)
 (TXT) w3m dump (www.bbc.com)
        
       | toomuchtodo wrote:
       | https://www.reuters.com/business/healthcare-pharmaceuticals/... |
       | https://archive.today/Gsw4p
        
       | frogarden wrote:
       | Good news! How do you safely develop medications for babies?
        
         | lamuswawir wrote:
         | This particular one is mostly about dosing, the available
         | medicines were weight based, with lowest dosages in the 5-15kg
         | range. This brings dosages lower allowing more precise dosing
         | for the lighter babies.
         | 
         | Edit: it's a very welcome addition. Limits side effects.
        
       | zkmon wrote:
       | Approved for use means approved for testing on populations.
        
         | squigz wrote:
         | Isn't that how medicine works?
        
           | zkmon wrote:
           | Only for lab mice. Humans require making it clear that they
           | are not being used as lab mice. But often, you see report
           | saying that "After seeing the results for x years of use by
           | populations, we found that it has y side effect which was not
           | known earlier". A doctor literally said this to me last week.
        
             | Calavar wrote:
             | What alternative process do you propose that will discover
             | all side effects, including those with well under 1%
             | occurence, without human use?
        
             | squigz wrote:
             | > "After seeing the results for x years of use by
             | populations, we found that it has y side effect which was
             | not known earlier"
             | 
             | I would think that any reasonably intelligent adult could
             | think about things for a few moments and come to the
             | realization that... yeah, that's how things work. This is
             | how we progress and learn. We develop, test, use, and learn
             | about things - and if we learn that hey, turns out, this is
             | bad over a long period of time... we change things.
             | 
             | And this of course doesn't apply to just medicine - it's
             | just technological progress.
        
             | wizzwizz4 wrote:
             | What's the alternative? "Oh, sorry, we would _like_ to
             | treat your baby 's malaria, but that would disregard the
             | baby's inherent dignity: we don't yet know the full side-
             | effect profile up to 3 sigmas."
             | 
             | Most indignities are lesser than dying of malaria.
        
         | 1over137 wrote:
         | As opposed to what?
        
           | parpfish wrote:
           | Probably as opposed to "approved for general use in the
           | population because we've passed all of our tests", which is
           | what I'd assume "approved for use" means
        
         | rsynnott wrote:
         | Are you taking the stance that no drug should ever be released
         | on the basis that it is impossible for trials to cover
         | literally the entire population? Like, what are you looking for
         | here?
        
       | _heimdall wrote:
       | Unless I'm reading the original study [1] wrong, I'm surprised
       | the study only used a population size of 28.
       | 
       | They did do a 12 month check-in which is good, but why such a
       | small group of study participants, especially when malaria is so
       | widespread?
       | 
       | [1]
       | https://clinicaltrials.gov/study/NCT04300309?term=CALINA&ran...
        
         | _heimdall wrote:
         | Also they apparently didn't use a control group, the study was
         | terminated early, and after the 43 day test window 9 of the 28
         | participants are listed as having the adverse event of malaria.
         | 
         | I'm particularly confused by that last one. How is malaria
         | considered an adverse event when testing an anti-malaria
         | treatment? Other data in the study shows that 1 participant had
         | malaria again with matching DNA, meaning the original infection
         | likely came back. 6 others were reported as getting malaria
         | again but with different DNA. So what does it mean to have 9
         | with the adverse event of malaria?
        
           | refulgentis wrote:
           | Think you're over-parsing a bit: adverse events ~= something
           | we should mention, not Bad Things The Treatment Did. "What
           | does it mean?" is a qualitative question, not a quantitative
           | one. Taking that question more colloquially, in this case,
           | I'd say it means "the vaccine did not prevent infections in 9
           | of 28 cases and / or they had an infection before the vaccine
           | was taken"
           | 
           | I'm sure you mean well -- since your post, the thread got
           | cluttered up with outright know-nothing-type comments. I am
           | being very literal in order to help teach people in despair
           | what we expect when we read these.
        
             | _heimdall wrote:
             | I did mean well, thanks for assuming good intent here. I
             | still don't understand how 9 of 28 could have the adverse
             | event of malaria, though, given that other data in the
             | results show that 1 participant had what seemed to be a
             | reinfection with a genetic match and 6 others were infected
             | again with a parasite that didn't match their original
             | infection's genetic makeup. That is 7 individuals, where do
             | the other 2 come from?
             | 
             | My uncertainty is that I may be misunderstanding the
             | meaning of malaria as an adverse event entirely here - I
             | don't get how reinfection would be an adverse event rather
             | than a potential failure of treatment.
        
         | freeone3000 wrote:
         | Statistical power. Malaria doesn't go away on its own. They
         | know the treatment should be overwhelmingly effective if dosed
         | correctly, it's merely a measure of determining dosage vs
         | negative effects.
        
           | 3eb7988a1663 wrote:
           | The famous comparison: you do not need a large N to test the
           | effectiveness of parachutes.
        
           | _heimdall wrote:
           | Right, in this case my concern really wouldn't be with
           | efficacy as much as safety. When the test group is only 28
           | participants how can we assume that we would have found most
           | of the safety concerns? Is the assumption being made that the
           | only concerning factor is age and that there are no other
           | contribution factors that could lead to negative outcomes?
        
         | BugsJustFindMe wrote:
         | Ethics dictate always using the smallest viable cohort to show
         | that giving the treatment regimen (in this case dosage for a
         | drug known to be effective) isn't obviously worse than the
         | thing it's supposed to treat, even if we're already pretty
         | sure. This also keeps the costs of running the studies down so
         | we can effectively conduct more studies for more treatments.
         | 
         | We also already have data about its use in babies over 11 lbs,
         | and this is just going even smaller to 4.4 lbs, so a strong
         | baseline has already been demonstrated.
        
           | verisimi wrote:
           | a small study.... that then gets approved and released to
           | everyone!
        
             | refulgentis wrote:
             | A little dull assertion can't hurt you, individually, and I
             | bet it feels fun.
             | 
             | I assume you're well-aware the process for something like
             | this doesn't fit in a sentence.
             | 
             | Additionally, there's context in the comment you're
             | replying to, this isn't the only study.
        
           | _heimdall wrote:
           | What do you mean "ethics dictates?" We define ethics and they
           | generally reflect the current culture, ethics aren't
           | universal and can't dictate anything.
           | 
           | The scientific method, though, would dictate that a cohort
           | size should be large enough to show a high probability of
           | safety and efficacy, assuming that is what is being tested.
           | It would also dictate that a control group would be needed to
           | compare against the test group.
           | 
           | I totally understand the ethical concerns of potentially
           | allowing children to be harmed while part of a control group,
           | but when the test is being done specifically because there is
           | currently no treatment the only change is that they would
           | pick a group of untreated children that are a valid control
           | group for the study. Either way those children wouldn't be
           | treated and there really isn't an ethical issue to deal with.
        
             | roywiggins wrote:
             | There is a treatment, but it's done ad-hoc:
             | 
             | > Until now there have been no approved malaria drugs
             | specifically for babies.
             | 
             | > Instead they have been treated with versions formulated
             | for older children which presents a risk of overdose.
        
               | _heimdall wrote:
               | The claim still holds then that there are no approved
               | treatments for malaria in this age range. If we were
               | already treating patients off book, and that were the
               | reason we couldn't have a control group, then there is no
               | real reason to do the study at all.
        
         | dev_l1x_be wrote:
         | It is pretty common to have a very small (and usually not
         | exactly random) sample size.
        
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