Science? We don't need no stinkin' science!
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Will more harm be done to the patients undergoing this therapy given the expanded approval? Or is this just a matter of the therapy not delivering (statistically significant) enough benefits?
Is there a reason the FDA rules allow one director to override objections from three reviewers and two other directors? Would you rather that there be absolute rules on the FDA goes by majorities among the reviewers and directors?
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Will more harm be done to the patients undergoing this therapy given the expanded approval? Or is this just a matter of the therapy not delivering (statistically significant) enough benefits?
Is there a reason the FDA rules allow one director to override objections from three reviewers and two other directors? Would you rather that there be absolute rules on the FDA goes by majorities among the reviewers and directors?
@Axtremus said in Science? We don't need no stinkin' science!:
Will more harm be done to the patients undergoing this therapy given the expanded approval?
I dunno. Will patients not benefit from other therapies that might be withheld?
Or is this just a matter of the therapy not delivering (statistically significant) enough benefits?
In the distant past, hydroxychloroquine and other medications in the distant past were shown to not deliver statistically significant benefit.
Is there a reason the FDA rules allow one director to override objections from three reviewers and two other directors? Would you rather that there be absolute rules on the FDA goes by majorities among the reviewers and directors?
Yeah, I'm not down with one person permitting the approval of the drug because of the feelz.
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@Axtremus said in Science? We don't need no stinkin' science!:
Will more harm be done to the patients undergoing this therapy given the expanded approval?
I dunno. Will patients not benefit from other therapies that might be withheld?
Or is this just a matter of the therapy not delivering (statistically significant) enough benefits?
In the distant past, hydroxychloroquine and other medications in the distant past were shown to not deliver statistically significant benefit.
Is there a reason the FDA rules allow one director to override objections from three reviewers and two other directors? Would you rather that there be absolute rules on the FDA goes by majorities among the reviewers and directors?
Yeah, I'm not down with one person permitting the approval of the drug because of the feelz.
@George-K said in Science? We don't need no stinkin' science!:
@Axtremus said in Science? We don't need no stinkin' science!:
Will more harm be done to the patients undergoing this therapy given the expanded approval?
I dunno. Will patients not benefit from other therapies that might be withheld?
FDA approval is not a zero-sum game, the FDA can approve unlimited number of drugs and therapies. Actual prescription and payment for drugs/therapies may be a zero-sum game. But that comes down to the physician prescribing the drugs/prescriptions, whether the physician is knowledgeable enough to prescribe the more beneficial ones over the less beneficial ones.
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@George-K said in Science? We don't need no stinkin' science!:
@Axtremus said in Science? We don't need no stinkin' science!:
Will more harm be done to the patients undergoing this therapy given the expanded approval?
I dunno. Will patients not benefit from other therapies that might be withheld?
FDA approval is not a zero-sum game, the FDA can approve unlimited number of drugs and therapies. Actual prescription and payment for drugs/therapies may be a zero-sum game. But that comes down to the physician prescribing the drugs/prescriptions, whether the physician is knowledgeable enough to prescribe the more beneficial ones over the less beneficial ones.
@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."
"Now look here, you Baltic gas passer... " - Mik, 6/14/08
The saying, "Lite is just one damn thing after another," is a gross understatement. The damn things overlap.
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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.
“I am fond of pigs. Dogs look up to us. Cats look down on us. Pigs treat us as equals.” ~Winston S. Churchill
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@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."
@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.
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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.
@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?
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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.
"You never know what worse luck your bad luck has saved you from."
-Cormac McCarthy -
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.
@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.
"You never know what worse luck your bad luck has saved you from."
-Cormac McCarthy -
@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?
@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.
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@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.
@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.
Not trivial at all. This is a tempest in a teapot. IMO there was good reason for the approval.
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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.
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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 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.
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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 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.
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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?
