Successful hospitals hire teams of medical coders. Why you ask ? @DrDiGiorgio
My own photo finish 27 years ago as I anchored our mile relay. I never heard how close he was, with roar of the crowd. My teammates yelled for me to lean. I won by 2 hundredths of a second. Never quit.
The media bias and mayor's public statement on the Gracie Mansion story is a case study in misleading narrative formation.
1/4
Liar. The only person who ever said control arm was burr holes was the Uniqure CEO, which the journalists printed without seeking confirmation about that from anyone in the FDA.
HHS 2 days later said this was a distortion of the conversation.
Incredibly malignant people.
While my heart aches for Iranians, I have zero sympathy for the regime leaders
Watch the below video and remember what happened on Oct 7
Never forget
And never forget the tens of thousands of Iranians killed in January
More people need to wake up to this.
The arbitrage for biotech is not “does the science work” it’s based on whether or not regulators can withstand the public pressure from investors/ rare disease advocacy groups /
hostile media that aren’t observers, but actual players in the field that are looking to get you fired.
Of course you can’t say that definitively. Generate some evidence that isn’t founded on a not reality historical control and let’s talk.
Otherwise you are asking society to pay $3million/therapy to drill into the brain of desperate ppl based on hopes and prayers not data.
This has significant downsides.
For any HD patients, please read about the sarepta / elevedys story. Yes it is possible to make a bad thing worse.
I have seen some scummy people in my life, but the people on this site pushing UniQure are some of the scummiest I have ever seen. They literally have no shame, although this seems to describe much of biotech. Pushing treatments that don't work, exploiting vulnerable people.
Everyone understands that even extremely low IQ regulators will be unable stop HAART for HIV, Harvoni for Hep C , CAR-T, etc.
The ecosystem wants low IQ / spineless regulators in power to approve slop that doesn’t work.
So the whole debate about threatening true innovation is just a smokescreen.
Yes, the ideological tribe that’s the problem is the one that said:
1. Locking down for infinity (hyperbole) was bad
2. Printing money to allow lockdowns would be bad
3. Masking 2 year olds is ineffective
4. Infinity boosters is the only way for society to suppress and survive COVID
5. Keeping kids remote for > 1 year was best for them
Most recent edition:
Vinay is divisive and chaotic and tribal ! because he
1. tried to pull a gene therapy that didn’t work and was killing young boys
2. Told a company with a gene therapy product with extremely patchy data that it works (oh btw it requires drilling into the brain to deliver it) to come up with better data if they wanted approval.
The real epistemological problem here is Mandrola commenting on a truncated y-axis.
Ok then.
Political tribalism is ignoring the science.
In this case, ideologically motivated to do what?
Which specific decision do you think Vinay ignored the science and made a political decision ? Sarepta? Uniqure? Moderna flu?
I looked under the covers at the actual science behind the decisions, and Vinay was correct based on the evidentiary standards that based on your feed you appear to support.
You either haven’t looked at the Science or are making a political judgement.
Trial after trial shows minimal meaningful decline - except for the company’s external historical control at 1 year.
The entire valuation of this company at this point is based on the company’s unnatural external historical control. (And their ability to control who is head of CBER)
Great job legacy science reporters. Keep speaking truth to power.
Prasad is a moron for saying we shouldn’t approve a non-working drug that requires drilling into your brain to deliver said drug.
I am also a moron for saying Prasad was correct.
Pharmabro in a nutshell.
Agree here with most of what he’s saying, but the story should be the ridiculous decisions with regards to evidentiary standards made under the prior FDA.
Inexplicable/harmful decisions made related to Duchenne’s is a good example - there is little doubt woodcock was wrong to approve exondys, elevidys killed some boys (a Peter marks decision to expand a non working therapy with serious side effects beyond the kids in the trials)
Adu also doesn’t comment on the troubling playbook I write about - to 90% of objective ppl looking at this , this is the FDA closing a loophole opened up inappropriately to give dangerous therapies to desperate patients that don’t work and make biotech loads of money.
Incorrect. I do refer to this in article
This thread demonstrates some major gaps in how to evaluate evidence.
You don’t do a sham control for safety and dose finding.
If you can’t interpret the Uniqure slides below, and identify multiple red flags about efficacy of AMT-130, whether you know it or not, you are a stock pumper not someone who impartially evaluates evidence.
Some points for those observing who don’t own stock in the company. (There is no reason other than having a equity position in the company that I can think of for a random dude/dudette sitting at a computer analyzing this)
1. The historical control arm is a suspiciously straight line even though the natural history is actually not like this.
(Don’t believe me? Look at the control arm in the RCT that are doing better than everyone at the end of year 1. )
2. About power - the sample needed to show a difference relates to the size of the effect you are looking for. If the difference is as large as they say, the same 10 person in each arm trial will show a significant gap at the 1 year mark. And for a therapy that literally requires drilling into your brain , if it takes a 100 patients to show benefit, that means a large portion of those 100 are not seeing benefit, but are all still having their brain drilled into.
Even if this is approved, which appears much more likely now that uniqure and its media partners got their way (will
Any other regulator dare to ask Uniqure for additional evidence??), any HD patients should take these serious concerns into account. If I was a HD patient I would 100% not allow them to do this to me based on the data presented because I have no idea if there’s a benefit to having my brain drilled into.
(And before you say it the surrogate data in this space is weak. Need more
Work to validate nfl as a therapeutic target. Clearly mhttt shows you can’t rely on markers of disease progression )
I hold no position in uniqure and never have.
"The law of diminishing returns reminds us: biology isn’t linear.
Pushing LDL from 70 to 30 mg/dL isn’t equivalent to dropping from 130 to 70 — the absolute payoff shrinks as you approach the floor, and the floor doesn’t even get you close to zero risk.
Plaque quantification may create the illusion of precision, but the current state of cardiovascular therapy means that most of the benefit accrues to those selling CAT scans and plaque quantification software, not patients."
I think the FDA shouldn't exist outside of controlling antibiotics, antivirals, & antifungals, but if we are going to have it, it should be a serious regulator that doesn't just rubberstamp industry wants. Vinay was so. His replacement likely won't be.
https://x.com/anish_koka/status/2030313761333031352
From Uniqure’s slide deck.
There's statistically significant separation at one year comparing to historical controls.
That result is directly contradicted by the randomized data at one year.
The sham control arm in the RCT is actually the best performing at 1 year.
I’m not against making all sorts evidentiary leaps for rare disease terminal patients, Uniqure’s data is just crap.
There only source of value is an FDA that will give them approval.
2 options : either this group can’t read graphs, or are unwilling… but please @AppleHelix go on about Mandrola and the y-axis.
These are the statistical geniuses who care so much about rare disease patients.
Then you need a good clinically validated surrogate.
If you don’t have that, you’re just drilling into people’s brains with no real idea about what you’re doing and giving Uniqure $3million each time you do so.
We’ve been down this road before - remember Elevidys killed young men
The 800-pound gorilla
HHS Appointee Study (2023): A study in Health Affairs tracked appointees across the Department of HHS (includes FDA) between 2004-2020. It found that 32% of government appointees exited directly into private industry at the end of tenure
#revolvingdoor
Again.
Complete bull 💩 .
You want to use historically matched controls to allow a drug that REQUIRES BURR HOLES and a deep brain injection.
The drug failed in an RCT. Apparently it’s also a 10hr procedure.
Yes, a working FDA should ask for better data.
“Instead, I would challenge you to embrace uncertainty, even so far as to Believe things you don’t really Know. Until eventually, with time and patience, you realize how much you really do Know – Data be damned!”
Again the hill these knuckleheads chose to defend was Sarepta and Uniqure.
Thats how you know they’re lying about it just being a vinay personality problem.
This was so good.
Consider giving it a read even if you're not interested in cardiology.
There are many homologs of this story in Oncology.
I have been in medicine for 39 years, first in academia, now in private practice. I cannot name another physician with Dr. Prasad's combination of intelligence, integrity, understanding of study methodology, and ability to accurately interpret study results. Sigh. It's hard to have hope.
Not only did the journalist decide to reveal identifying information, someone higher up then ok’d that story not being paywalled to make sure as wide an audience would know.
AI Plaque Quantification: The Billing Code from US @MedicareGov Arrived Before the Evidence Did
https://x.com/i/status/2030273007566889234 via @anish_koka
How @VPrasadMDMPH came to Washington & @US_FDA & why it was always going to end this way
https://substack.com/@anishkokamd/note/p-190196571?r=tduh3 via @anish_koka
Am I getting the journalistic ethics part wrong? Very troubling to this non-journalist.
@neerajadeshp @emilyakopp @AlexBerenson
Wow. My head has been spinning with many of these same thoughts, and Anish pulled them together remarkably quickly. Impressive
He wanted to do good, but could not. The system would not allow him.
Another model of transparency at work:
Hospital care is nearly 1/3 of overall spend on healthcare in America. At a recent @SenFinance hearing Dr G. Keith Smith, founder of the @SurgeryCenterOK testified that his surgery center “recently performed a tonsillectomy on a child for $3,875 after the family had been quoted $72,000 by a Dallas-area hospital. [The OSC’s] prices are generally half of what Medicare pays hospitals, and less than Medicaid payments to hospitals for the same procedure.”
How does the OSC do it? With publicly posted, transparent prices and without conventional insurance. When employers steer their employees in the direction of the OSC, they save tens of thousands of dollars by flying their employees to Oklahoma and paying directly for the procedures performed there.
@RogerMarshallMD (R-KS) and @Hickenlooper (D-CO) intro the Patients Deserve Price Tags Act. I brought up this bill at the onset of our discussion with @cherrigregg and @Avi_WA . The bill requires hospitals, surgery centers, imaging centers, and labs to report their negotiated rates and cash prices publicly. More than that, the bill would prohibit third-party insurers from concealing from insured employers the prices they are paying. Transparency for employers would be a huge boon to the 60% of Americans under 65 who have insurance provided through employers. The bill would also require that patients receive an itemized bill for services rendered.
We clearly need this fix, because the Executive order we have alone on Price Transparency is not enough. Per @PtRightsAdvoc only 21% of hospitals are fully compliant. What are they hiding? Maybe the fact that they are able to provide care for less.
Here is the whole hearing https://www.aging.senate.gov/hearings/modernizing-health-care-how-shoppable-services-improve-outcomes-and-lower-costs
There is so much inside baseball happening within these FDA trials that creates a ripe environment for distortion, allegations of ideology and politics, or other ad hominem attacks. Moreover , those suffering from untreatable chronic disease can easily be co-opted to advocate for companies developing treatments that could have promise but haven’t passed a reasonable bar for efficacy and safety. Someone or some organization has to stand in the breach and demand this standard. Whither critical appraisal?
x.com/i/article/203031182839…
R to @anish_koka: substack.com/@anishkokamd/no…
Better things to do than spend Saturday morning writing this, but important since there aren't enough actual journalists who can do their job. (link in reply)
The Prasad - FDA timeline
This is an incredible effort. It is many fold better than anything you could read in a top medical journal.
Not just for cardiologists either. A good read for economists as well.
Caveat Emptor, Caveat Lector.
Wrapping gossamer-thin evidence in an aura of AI might be a successful reimbursement strategy, but it does not make it authoritative or convincing!
@drjohnm @venkmurthy @anish_koka
👇🏻