When Dying Patients Want Unproven Drugs

Re:When Dying Patients Want Unproven Drugs by A_D_E_P_T on Monday June 19, 2023 @07:02PM (#36397514)

RCTs were not required and were rarely performed prior to 1962, and yet that period from roughly 1940 to 1960 is still today widely known as "the Golden Age of drug development." How did they know, back then, that new drugs work? Simple: They have sufficiently powerful (in a statistical sense) effects in a patient population. Very powerful effects require a very small population -- this applies to ALL drugs that are curative -- whereas moderate effects would require a large population. Reasonably sized safety trials and extensive postmarketing surveillance are entirely sufficient.

> How would you weed out the snake oil?

The current paradigm is "better 10,000 people die of neglect than 1 person die of snake oil or quackery." I'm okay with a little bit of snake oil if it means that more drugs are being introduced more quickly, and I trust practitioners and patients to, more often than not, determine the therapeutic regimens that are right for them.

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Re:When Dying Patients Want Unproven Drugs by youainti on Tuesday June 20, 2023 @01:34AM (#36400121)

Those were also small molecule drugs which are relatively simple to produce compared to modern large molecule drugs (biologics such as modern insulin, mrna vaccines, humira, many cancer drugs, etc.)

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Re:When Dying Patients Want Unproven Drugs by AstralStorm on Tuesday June 20, 2023 @01:59AM (#36400270)

You might be surprised that the modern processes for true biologicals like peptides and mRNA are actually simpler than the chemical processes for classic drugs. E.g. solid state peptide synthesis. Once you can make a 10 peptide one, you can make a 1000 unit one in an identical way with more iteration.

Compare that to establishing a bespoke chemical process for each drug, or messing with purification of natural biologicals, or fusing antibodies to protein or peptides to grow inside a whole cell.

They should be actually cheap. They aren't because new.

Humira is quite a bit harder to manufacture cleanly than a peptide or mRNA, and still harder than a plain solid state synthesized and folded protein. It requires a lot of post translational modification, which means cellular machinery, which means a whole bacterial cell you now have to grow, feed and remove from the product.

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Re:When Dying Patients Want Unproven Drugs by random78965 on Tuesday June 20, 2023 @03:09PM (#36408981)

I see! My intuition was that it would be hard to evaluate efficacy without RCTs - I assumed low effect sizes, which I guess is not necessarily true for many drugs.

Agreed that there should be a faster way to trial drugs and get them to market. Though I've heard that the "slowness" of the FDA is actually overstated; not sure how true that claim is.

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