snip ODAC Minutes - Europe vs. US approvals

[Guest guest]

DR. PAZDUR: I just wanted to kind of emphasize, from Hilde's talk, several

important points.

.... If you notice, they use the word " preliminary evidence of a

benefit-risk. " [in the European Union]

.... Nowhere is this issue of surrogacy, a surrogate reasonably likely to

predict. And I think that when one takes a look at the time and energy that

we spend here in the United States debating, well, is this effect on PFS

[Progression Free Survival] going to reasonable predict a value, an

improvement in overall survival, that kind of lexicon doesn't necessarily go

on in the EU, because that issue is not there.

.... It's a risk-benefit relationship. And here, again, in dealing with them

and in our discussions with them, they have had *significant issues also

with single-arm trials,* because of the issue of the lack of clarity and

ability to really evaluate a risk-benefit decision in a single-arm trial.

....Obviously, you don't have a comparator, and, hence, if you have an 80

percent AE, adverse event, you have to assume that it's due to the drug. You

don't have a control arm really to compare it to. "

DR. WILSON: I think you said it, and that is that maybe functionally, it's

not as different as the language, but they're talking about preliminary

evidence that could be applied to a surrogacy, as well.

DR. PAZDUR: Correct.

DR. WILSON: So maybe you can comment Is there a -- I don't want to use the

word " higher bar, " but is there a bar that is above a surrogate marker that

is more commonly applied in these applications to the EMA versus here?

I would gather not, because I think a lot of the same studies are used for

both FDA and EMA. But perhaps you could comment with regard to the points

that Dr. Pazdur made.

MS. BOONE: I fully agree with the comments from Dr. Pazdur that, indeed, our

framework [EU] speaks about an initial positive benefit-risk and it doesn't

specifically refer to the use of surrogate endpoints. It's one of the tools

that we have available and that we will use, but it's not exclusively based

on the use of surrogate endpoints, as such.

DR. WILSON: Dr. Pazdur, though, wouldn't it be reasonable to say that we

always -- in evaluating drugs here, we always look at the risk, as well, and

the risk is always part of it.

DR. PAZDUR: I'm just bringing this out as a difference in the way that the

things are written. Actual application, I'm not quite sure how differences

are interpreted, and you'll see a great deal of similarity between the

actions. Obviously, a lot of people like to point out differences that may

exist between approvals in the EU and the United States. But if one takes a

look at the number of concordant approvals and disapprovals or

non-approvals, it far exceeds the few differences that we have. "

All the best,

~ Karl

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