? On EAP’s and trial drugs

[JAM]

Do only some drugs that are currently in trials go to EAP? If so, why do only some and not all drugs in trials do it? How/why is it decided?

I guess I have the same ? Regarding AA, why don’t all drugs that are going into a Ph 3 trial, or raising capital for Ph 3 go for AA (like Cline is doing)?

*If this is in wrong thread feel free to move.

 

[Nikki J]

EAPs cost money. Historically they were funded by a combination of philanthropy and the company providing the drug. Not all companies can do this especially if they are a little start up. Philanthropy is also necessary to cover costs in the clinic. The biogen eap for qalsody was combination funded like that.

Now there is some government funding for eaps but it needs to be applied for and approval is not automatic and funds are not unlimited

Preparing an application for the fda is a huge project- thousands of pages if the fda agrees to consider. Even asking for accelerated approval is a lot of prep work. And if you have enough positive results for phase 3 you very well could also know they are not strong enough to go for aa. I think going for aa is very much the exception rather than the rule.

 

[lgelb]

Accelerated Approval is only granted to therapies that can demonstrate benefit via an already-approved or newly-approvable surrogate endpoint (like NfL) ahead of demonstrating clinical benefit.

It's the FDA that decides whether a particular surrogate is better integrated into traditional or AA pathways. There's a table for that.

And since most surrogate endpoints are not well-validated, it's not a given that the surrogate and clinical measures synch up. For example, a recent study reported that only 40% of cancer drugs granted AA demonstrated a clinical benefit in confirmatory trials after more than five years of follow-up.

There are several FDA programs that work together: Breakthrough Therapy designation, which houses Fast Track, kind of an umbrella for frequent early-stage regulatory consultation; Priority Review (up to 4 months saved on the usual review process); Accelerated Approval if it applies; rolling consideration of the NDA so earlier and more helpful feedback can help shape the submission.

 

[JAM]

Ty for that explanation Nikki. I am most surprised/impressed (?) that Clene has done EAP for CMN-AU8, considering it being a new, smaller company- that’s what prompted my question to begin with

 

[JAM]

Ty Laurie,
One other ? Re AA, while under AA do PAL’s pay out of pocket for the drug? I assume yes since not FDA approved so not covered by insurance?

 

[lgelb]

Interestingly, this has come up with Qalsody and though commercial plans have the option not to cover it, CMS recently issued a regulation that Medicare Advantage plans have to. Regular Medicare will as well.

So the answer is, it depends. But based on the CMS decision, anyone whose plan tries not to reimburse Qalsody should appeal and cite CMS. I suspect you can be successful.

 

[Loriaus]

I am looking for any information on the CNM dash AU8 as being a positive drug? I started this drug through a expanded access program about 6 weeks ago just wondering if anybody else is on that and has shown improvement? With their NFL numbers? Any advice any hope would be appreciated thank you

 

[JAM]

Hi L,
I am interested in your question as well, as I believe (just my feeling) that Clene is specifically doing the EAP for the NFL data to provide the FDA in hopes of getting AA for this drug. I pray this helps, but I am aware they have been declined 2x in the past already based on preexisting data.
Wouldn’t it be a beautiful New Year for everyone if there was some success!

 

[lgelb]

Yes, there has been confirmation that is what they are doing.