Science? We don't need no stinkin' science!

[George K]

@Axtremus said in Science? We don't need no stinkin' science!:

FDA approval is not a zero-sum game

You missed my point. It's not a question of zero-sum, it's a question of "Since you're already on Drug A, perhaps we should hold off on trying Drug B until we see how it works."

[Mik]

I think we'd need to know what those secondary endpoints were. Perhaps I will run this by my BIL who was the chief statistical analyst for a large pharma firm.

[Axtremus]

@George-K said in Science? We don't need no stinkin' science!:

@Axtremus said in Science? We don't need no stinkin' science!:

FDA approval is not a zero-sum game

You missed my point. It's not a question of zero-sum, it's a question of "Since you're already on Drug A, perhaps we should hold off on trying Drug B until we see how it works."

I addressed that point, essentially saying that’s on the prescribing physician, not on the FDA.

[Axtremus]

@Mik said in Science? We don't need no stinkin' science!:

I think we'd need to know what those secondary endpoints were. Perhaps I will run this by my BIL who was the chief statistical analyst for a large pharma firm.

Hmmm …. big pharma guy may have a bias for the FDA to approve stuff more liberally; can we trust him?

[jon-nyc]

I saw it yesterday and was heartened by the news. I’ve been in meetings with him where he said he’d issue guidance on clinical trial endpoints just to be later scuttled by his staff for rather dubious reasons. I could give details if anyone is interested.

I don’t know the details of the Duchenne drug but, knowing him, there’s no way it’s as simple as ‘feels’. There’s probably a small signal and knowing it will help at least some boys destined to become cripples to stave it off a bit is not a bad thing IMO.

There’s some new legislation in the senate being pushed by Braun (R-IN) and Gilibrand (D-NY) that would allow ultra-accelerated approvals (post P1!) for certain ultra-rare conditions providing the patients become part of a registry so they can figure out quickly if it’s working.

[jon-nyc]

@Mik said in Science? We don't need no stinkin' science!:

I think we'd need to know what those secondary endpoints were. Perhaps I will run this by my BIL who was the chief statistical analyst for a large pharma firm.

Primary endpoint was a 18 point motor skill test. Secondary was the ability to stand and walk. Non trivial if it’s your son.

Also the expanded approval is provisional and they’re requiring a confirmatory study.

More here. https://endpts.com/fda-widens-sareptas-duchenne-gene-therapy-to-older-boys-in-significant-expansion-of-use/

[Mik]

@Axtremus said in Science? We don't need no stinkin' science!:

@Mik said in Science? We don't need no stinkin' science!:

I think we'd need to know what those secondary endpoints were. Perhaps I will run this by my BIL who was the chief statistical analyst for a large pharma firm.

Hmmm …. big pharma guy may have a bias for the FDA to approve stuff more liberally; can we trust him?

You have no idea of the ethical standards our pharma firms adhere to. If you did you'd be impressed.

[Mik]

@jon-nyc said in Science? We don't need no stinkin' science!:

@Mik said in Science? We don't need no stinkin' science!:

I think we'd need to know what those secondary endpoints were. Perhaps I will run this by my BIL who was the chief statistical analyst for a large pharma firm.

Primary endpoint was a 18 point motor skill test. Secondary was the ability to stand and walk. Non trivial if it’s your son.

Also the expanded approval is provisional and they’re requiring a confirmatory study.

More here. https://endpts.com/fda-widens-sareptas-duchenne-gene-therapy-to-older-boys-in-significant-expansion-of-use/

Not trivial at all. This is a tempest in a teapot. IMO there was good reason for the approval.

[Mik]

There comes a point where you have to delve deeper into what 'do no harm' really means. Perhaps allow no harm might be included. In the cases of hopeless diseases with assured catastrophic outcomes it is hard to imagine you could do much harm with a drug that had made it through phase three trials, even if the benefit is less than what you hoped.

[George K]

@jon-nyc said in Science? We don't need no stinkin' science!:

I saw it yesterday and was heartened by the news. I’ve been in meetings with him where he said he’d issue guidance on clinical trial endpoints just to be later scuttled by his staff for rather dubious reasons. I could give details if anyone is interested.

I don’t know the details of the Duchenne drug but, knowing him, there’s no way it’s as simple as ‘feels’. There’s probably a small signal and knowing it will help at least some boys destined to become cripples to stave it off a bit is not a bad thing IMO.

There’s some new legislation in the senate being pushed by Braun (R-IN) and Gilibrand (D-NY) that would allow ultra-accelerated approvals (post P1!) for certain ultra-rare conditions providing the patients become part of a registry so they can figure out quickly if it’s working.

Interesting take, thanks.

[Jolly]

@jon-nyc said in Science? We don't need no stinkin' science!:

I saw it yesterday and was heartened by the news. I’ve been in meetings with him where he said he’d issue guidance on clinical trial endpoints just to be later scuttled by his staff for rather dubious reasons. I could give details if anyone is interested.

I don’t know the details of the Duchenne drug but, knowing him, there’s no way it’s as simple as ‘feels’. There’s probably a small signal and knowing it will help at least some boys destined to become cripples to stave it off a bit is not a bad thing IMO.

There’s some new legislation in the senate being pushed by Braun (R-IN) and Gilibrand (D-NY) that would allow ultra-accelerated approvals (post P1!) for certain ultra-rare conditions providing the patients become part of a registry so they can figure out quickly if it’s working.

All for it. The FDA had become a bureaucratic bunch of assholes, at least at the far end of the stream that I traipsed through.

Case in point...Europe had HIV and Hepatitis C tests on the market from major manufacturers (Abbott, etc.) months and even years before we did.

The fast-tracking of drugs and testing during COVID showed us that on some things, we were waaay too cautious.

[Axtremus]

OK, now that it looks like @jon-nyc’s post turned the tide of opinions, let’s take a look at the opposing side.

What are the reasons and motivations of the three reviewers and two FDA directors to have opposed approval? Any conflict of interest or ideological bias that we need to look into? What makes “notable pharmaceutical industry expert and commentator, Derek Lowe” admonish the approval? Does Derek Lowe know what he’s talking about? Do those three reviewers and two FDA directors know what they are doing?

Is it good that FDA rules allow for a single director to override three reviewers and two other directors now?