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dekhn 3 days ago

Yes, the most common example would be clinical trials for drugs and other medical treatments- often have thousands of patients (with recruitment being the limiting factor). There are tons of ways that studies can go wrong, for example when patients don't take the treatment and lie (this is common) or have other lifestyle factors that influence the results, which can't be easily smoothed out with slightly larger N.

I don't know how to fix the nutritionist studies- I'm still pretty skeptical that you could ever control enough variables to make any sort of conclusion around things with tiny effect sizes. This isn't like nutritional diseases we've seen in the past, for example if you look at a disease like pellagra (not getting enough niacin), literally tens of thousands of people died over a few years (beri beri, rickets, scurvy are three other examples; these discoveries were tightly coupled to the discovery of essential nutrients, now called vitamins).

atombender 3 days ago | parent [-]

From my reading, that's not generally true. It all depends on the methodology. Safety or feasibility studies can use very small sample sizes. I've been reading safety studies on monoclonal antibodies like Cimzia, for example:

https://pubmed.ncbi.nlm.nih.gov/29030361/ (N=16)

https://pubmed.ncbi.nlm.nih.gov/28814432/ (N=17)

https://ard.bmj.com/content/83/Suppl_1/1145.2 (N=21)

Of course, these are not nutritional studies.

dekhn 3 days ago | parent [-]

You should try reading the FDA approval for the drug; it was already approved before these publications (which aren't so much clinical trials as just medical research). The FDA approval has a whole paragraph about how the effect size was too small to demonstrate statistical significance, and the trials had n=300.

It's also indicated for use in a disease we don't understand, for people who didn't respond to all the previously approved drugs. Not a good example at all.

atombender 3 days ago | parent [-]

I'm not comparing these to the FDA approvals process, but to your claim that trials use thousands of patients. These three studies are ascertaining the pregnancy safety of a drug, irrespective of whether we understand the disease or what the response rate is.

Cimzia has been well-studied, and we understand why it works on autoimmune diseases like inflammatory arthritis. It has 6 FDA approvals for different indications, so your description of the drug itself is incorrect.