Eli5 how do human pharmaceutical trials work?


I’m wondering because pharmaceutical ads can list some pretty major side effects, let’s say like kidney failure or “even death”. Does that mean that in the human trials a significant enough percentage of the test population died? Seems kinda messed up to pay people to test medications if they can end up getting seriously hurt..

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3 Answers

Anonymous 0 Comments

A big reason they always say “possible side effects” is because it is usually not possible to directly and unconditionally say that a drug is the cause of an effect. If you have a trial in which there are a thousand people participating over five years, chances are at least some of them are going to die. Some from heart attacks, some from strokes, some from car accidents. Figuring out if this drug makes one of those things more likely can take a long time.

But keep in mind that we don’t just start clinical trials with no idea what will happen: these only occur after other extensive testing, and these days most of the effects of a drug can be predicted simply by analyzing its chemical structure.

Anonymous 0 Comments

I can tell you about a trial that I was involved in where my company was testing a drug in humans.

The first thing we did was test the heck out of the drug in mice and monkeys. Once we we fairly certain it could treat the deadly disease and was relatively safe, we got government permission to set up a trial.

We found people who had the deadly illness, and who had failed to respond to any other drugs, and who were a few weeks from death.

The doctors asked those people: “would you be willing to try a new drug? It might kill you faster. It might buy you some time. We don’t know. But whatever information we gain might someday help save the lives of people with your illness.”

Some people signed up. They weren’t paid. They did it out of altruism or out of hope that we would give them a miracle cure.

We tried out the drug, first at super low doses just to make sure it wasn’t too toxic. Then we tried to figure out what dose might be the most effective dose.

We tested their blood and urine. We asked them lots of questions about every possible side effect that they might feel. We measured carefully. If someone seemed to be having a side effect that could kill them faster, we would have to take them off the drug as soon as we realized.

Eventually, we got the dose right, and then somebody who was at death’s door got healthier and lived for another year more than they should have.

We made the drug better and better. As we got more confident that our drug was at least as good as the other drugs, we asked more people to do the trials. At this point we were constantly doing math to try to keep the risk to our drug trial participants as low as possible. But they knew there was a risk that participating in our drug test could cause them to live less long. We obviously hoped that we would help them to live longer, but we couldn’t be sure. We would watch the numbers every week and test everyone’s blood and urine frequently to try to make sure that if someone reacted badly, we could take them off the drug as fast as possible.

We tried different versions of the drug to compare to each other. If one version was significantly better than the other, we might be obligated to stop the trial and give everyone the better version as soon as the math had proved it was better.

Eventually the drug was able to cure the disease 90% of the time and it was approved.

Anonymous 0 Comments

No humans are given drugs until *well* along in the process. We don’t just give random chemicals to people to see what happens. Drugs are developed with at least some known characteristics based on their molecular structures and are extensively tested on cells in labs and then on animals before any human.

Drug warning labels legally have to list every medical condition that occurred in any person participating in the trial even if there was no link to the drug being tested. In other words, if I was diagnosed with lymphoma while participating in a trial for an asthma medication, they still have to list “lymphoma” as a possible side effect even if there’s no evidence that it was caused by the drug.

And deaths are taken extremely seriously. Any deaths during a trial must be reported and investigated. If even a single death is linked to the drug being tested, it can lead to the abandonment of the drug entirely. Even a single death during a drug trial typically makes the news, so no, there’s not some cemetery somewhere filled with the bodies of people who died because of a drug during the drug trial because that’s extremely rare.