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.
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.
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?
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.
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.
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
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.
> "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.
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.
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...
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.
a small study.... that then gets approved and released to everyone!
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.
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.
The famous comparison: you do not need a large N to test the effectiveness of parachutes.
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?
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.
https://www.reuters.com/business/healthcare-pharmaceuticals/... | https://archive.today/Gsw4p
Good news! How do you safely develop medications for babies?
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.
Approved for use means approved for testing on populations.
As opposed to what?
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
Isn't that how medicine works?
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.
What alternative process do you propose that will discover all side effects, including those with well under 1% occurence, without human use?
> "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.
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.