Dr. Holly Fernandez Lynch

Eventually, the ideal is that they would be the first drug that a patient tried, but you would design your study completely different depending on what scientific question you're asking.

Yeah, so this is a really important question to understand how drugs get regulated in the U.S.

Imagine you have a drug that's never been approved for any use before.

What you need to demonstrate to FDA is that it's safe for its intended use,

that it's effective for its intended use, and that you can manufacture it appropriately and consistently so you're getting the same drug every single time.

So that's a pretty basic question, right?

Does this drug work for people who have this disease?

It's not required.

for FDA approval that you show that your drug is better than another drug that's already been approved or is available to patients.

That's right, although unsurprisingly, it's a little bit of an oversimplification, and I'll just give you an example, right?

Imagine that there are several other drugs that are already on the market, and it would be unsafe to ask the people who are in your study to stop taking those existing drugs in order to take your new drug.

you're not going to be able to get that study off the ground for ethical reasons.

But from a regulatory perspective, really, it's just a very straightforward question.

Does this drug work better than nothing, right?

That's the typical design.

And so you might see, right, a study that's evaluating a product against a placebo.

But more often, right, what you're seeing is a drug being studied against something

what's called the standard of care.

So what would a doctor typically provide a patient who has this disorder, right?

You're gonna typically be testing your new drugs against that baseline for very pragmatic reasons.