May I add to it?
For the FDA to approve a drug for indicated use, it, of course, insists on proof. Proof of safety, and of efficacy. Efficacy is showing it does, indeed, do what you think it does, in patients. Safety means it doesn’t significantly harm or kill.
I’ll talk Efficacy only. “Proof” of efficacy means showing data from studies. Typically, scientific studies are done, carefully controlled and monitored. It’s a very formal experiment.
Protocols are written, approval sought, patients recruited, med centers chosen...finally, the experiment is run according to the protocol. Data is gathered and analyzed, statistical proof sought and demonstrated. Patients monitored long after initial trial is over. Initial, because very few people are in the study at first. If it all goes well, the study is expanded to a larger population...long, drawn out process.
And expensive!!! Sometimes patients are paid. Dr.s paid. Hospitals reimbursed, for the entire patient stay. Test paid for. Patient monitors hired and paid...huge $$$. Can run to hundreds of millions, just for the clinical trials! Cost to create a drug and get it to market is usually over $1 Billion.
Drug Manufacturers always seek first to compare a new drug to an old one, or other treatment, that has a known efficacy. Then see if the new one has better, worse, or the same outcomes. If no other treatment is available, they use a placebo instead (sometimes they do both). Placebos have no effect.
But if there is a decent chance of serious injury or death from your disease, and the new drug may save you, you sure as heck don’t want the placebo. In fact, it is considered unethical, by most industry people, to even allow it. What doctor would prescribe “nothing” to his mother instead of the drug that could work? What patient would say, “OK” to it?
The “Gold Standard” is a “Double Blind” experiment (trial). Where the patient doesn’t know if they are on either drug or placebo, and the medical workers don’t know, either. Prevents bias. Double Blind is the biggest, most expensive, longest type of clinical trials there are.
Sometimes they allow deviations, ahead of time, after long argument and delays, for certain, exceptional cases. Ahead of time, because drug makers (manufacturers) include the FDA people in the loop before asking for approval to go for the trial. Yes, the manufacturers need FDA approval to run their clinical trials, and that is a battle, always.
Let’s call the new drug “A,” the existing treatment “B,” and the Placebo “Placebo.” Pretty much any controlled A against B trial is better than A against Placebo, as B will at least give some relief to the sick patients. It can also reduce the sample size (number of patients enrolled), and possibly (though discouraged, and somewhat rare) historical data can be used for B, with or without requiring new B proof.
As of today, there is no approved drug treatment (“B”) to treat or mitigate Covid-19. Hence the moon shot effort to find one, the first A, and use it.
Yet A against Placebo is the worst type of study, ethically, you can do. Patients must agree to the trial. Who wants to sign and give approval for a possible placebo when you have Covid-19? For Hydroxychloroquine, that is what Dr. Fauci wants. That is what the FDA insists upon, by default, by internal and external policies and regulations.
The FDA has insisted on these type of trials for many years, for a new drug to be approved, or to approve a new, marketed, use. Long, drawn out, Hydroxychloroquine against Placebo trials. Fauci is dug in, like the FDA always is. They know only one way, and any deviations are head exploding. “Off Label” use makes their heads explode, spin around on their necks, and puke green vomit.
Bad stuff, eh?
Great addition. I was keeping mine as simple as possible. Yours is very informative.