Welcome back to plenary session. On today's plenary session, I'm going to talk about this paper.

This paper by the USFDA author's response rate event-free survival and overall survival in newly

diagnosed acute myeloid leukemia, the USFDA's trial level and patient level analysis.

This is for the oncology people out there. This is about surrogate validation. And if you're

in oncology and you're in research, you won't want to miss this. So let's get started. First, I think we all need a little bit of background.

Okay, we need a little bit of background before we can even approach this paper, and that

background is surrogate. What is a surrogate? What is a clinical endpoint? How do you do

validation studies? Let me give you the rough lay of the land. A surrogate. A surrogate, as my good friend Adam Seifu from the University of Chicago, says, is an end point the patient didn't know was important until the doctor told them it was. So your LDL cholesterol, your hemoglobin A1C. Now, we care about LDL cholesterol because we think drugs that lower LDL cholesterol also make us better off.

But if you didn't know about LDL cholesterol, you wouldn't necessarily care about that.

Patients care a great deal about it, but because they were taught by a doctor to care about it.

Clinical endpoints are things that patients intrinsically care about. Living longer overall survival.

I think it's the most important endpoint. It's the most important,

one, because it's objective, it's readily measurable. It's the most important because I think that's

what a lot of people with chronic or life-limiting diseases care about living longer, maybe even

that cure fraction, which is sort of a subset of an overall survival, that there's a fraction of

people who, after a treatment course is finished, have survival comparable to age, sex-match

controls. That's the Easton and Russell, 63 definition. I think people care about survival

and cure. That's really important. Patients also care about health-related quality of life.

They care about how they feel, living longer or living better. Those are the two things patients care about.

The problem with living better is it's very difficult to measure.

You know, there are a number of scales that have been quote unquote validated.

You know, validated means sort of validated against other sorts of benchmarks.

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