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Rob Freishtat

Jul 22, 2026

3:43
So I'm just kind of curious, what did you learn about trial design that the traditional pharma models often overlook? Can you provide us some insights there?
3:52
Yeah.
3:53
So, you know, trial design is an interesting, you know, interesting historical subject.
4:00
You know, the classic randomized double-blind placebo-controlled trial dates back to the World War II era and really hasn't changed much since.
4:12
And when you think about it, you know, it's obviously... are very much used to it, and people go to it over and over and over again, the larger question is, is it the best choice out there? And the answer is, in most cases, no.
4:32
You know, that design is very rigid.
4:35
It doesn't allow for any adjustments on the fly.

12 MINS LATER

16:58
And I'm curious if you can help us, what caused this shift? What were the practical aspects that made this possible?
8:49
But what does it look like when an organization, I guess, rejects that trade-off and builds what they're trying to do around both?
8:58
I, I mean, I don't see them as at odds with each other, right? First, I think you need to define what those things mean, starting with patient access.
9:09
In many ways, patient access goes hand in hand with financial viability, right? The more patients that are using your drug, the more revenue you're generating from that drug.
9:21
Where it gets complicated is how much you're being reimbursed for a patient to have access and all the ex-US issues around different pricing models, different authorizations, et cetera.
9:31
I don't think they're mutually exclusive.
9:33
I think they are tied together, and you have to consider them together.
9:37
At the end of the day, patient access is what's going to position you for success and for any sponsor for success, and especially in the rare space where there are limited numbers of patients.
12:46
So what is the most important thing the broader industry should be pulling from this rare disease or ultra-rare disease playbook as we move forward with other spaces where we're kind of implementing this concept of personalized medicine that we see in that rare space?

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