Still: even so I - as a German not being too much bothered by FDA decissions - agree with all your arguments, John, it would have been wise to claim your COI with your long lasting friendship with Vinay Prasad. You, as well as Vinay, often asked for it for others and even so this is not a financial COI, it still is one.
I also am offended at the indemnification of the 'drug' companies re vaccines...if we are taking in the wild west approach. One would think safe and effective would negate the need for such blanket protections.
My guess about cardiac function not chosen as the primary outcome is probably it takes much longer to see clinically meaningful change and would need very large sample sizes. So could be a feasibility issues. And usually PO is approved in talks with FDA.
The point of having regulation of pharmaceuticals is to try to ensure that marketed products have demonstrated safety AND efficacy.
This is already in distinction to unregulated “nutra-ceuticals”, which to me appear to fall into the “safe but useless” category almost without exception.
So maybe this company should’ve just marketed this as a “DMD supplement” and gone to town with it.
I guess the issue here is that the company has failed to demonstrate either safety or efficacy with their 20-pt study. So in some ways it’s even worse than your typical “turmeric enhancer”.
On the one hand, I get that you’re not going to have 1000 pt RCT for such rare conditions. OTOH, it would be irresponsible for a regulator to endorse a product that gives nothing more than false hope (based on available data to date).
I got paywalled by the WSJ but was able to read enough to see the author’s bias. We are all biased to a degree. I have to hope that Vinay’s role will help reduce what I used to say during the opiate epidemic: FDA approval = business deal made.
PS - for some reason I don’t see Vinay as a Fouchi in any way shape or form.
Here’s the problem with trying to hold the same RCT standard for rare diseases. Some of these diseases have fewer than 100 patients annually and demanding large randomized trials is simply not feasible. Demonstrating zero flexibility simply takes investments away and leaves these diseases unaddressed. I don’t have a problem with someone taking a stand on RcT across the board - you set the standard and live with the outcomes - but doing a whole road show about approving on “plausible benefits” and “small steps forward” and then demanding full randomized trials shows lack of basic integrity where actions don’t match words. I would not trust Dr. Prasad to be in any leadership role - let alone leading an agency like CBER. Sorry, I just don’t agree with the author.
An argument could be made that the FDA doesn’t care about these vulnerable patients lives- bc they will die soon anyway (thus give them all the experimental treatments). They need to be protected even more.
Having read the WSJ for 60 years I find Ms Finley's article like something out of the National Enquirer. I suppose there is an extreme libertarian position that would abolish the FDA and all government regulations but as a libertarian myself I would like to see objective scientific evaluation of treatments and drugs before I consume them. Thank you for taking the time to evaluate this article.
Did you not do the basic research to find out that the BLA was based on the OLE study at 3 years and the cMRIs collected during that time? No PUL data was included in the BLA, and the BLA wasn't based on the primary endpoint of HOPE-2. Research Soslow et al 2023 to learn about the natural history they're comparing against, and why the scientists previously in charge of the FDA thought it compelling enough to submit a BLA based on cardiomyopathy and not skeletal muscle.
Sensible Medicine is a media to promote specific doctors/politics or medical practices? I do not know Dr. Prasad and am looking for medicine here—not politics. Thank you TiredMedStudent, for a comment based on medical perspectives and not promoting politics.
Maybe you need a little more context before making a comment. Dr Prasad actually is very thoughtful and nuanced… im a medical provider but by no means an expert in reading studies… I read the WSJ article and I was inclined to side with the WSJ based on the article (even though I like Dr Prasad)… so I’m glad there was a balanced review of the decision that addressed some of the info in the WSJ article. Dr Mandrola is a lot more credible in reviewing this situation than a WSJ reporter. I think the back and forth conversation is super enlightening and I’m continuing to learn. Thanks Dr Mandrola. This isn’t political- it’s the art of debate of ideas.
Completely agree that the study is insufficient to prove that the therapy works. However, I think Dr. Mandrola oversimplifies the 2 possible functions of the FDA/how drugs could enter market. I don’t think it needs to be as black and white as “completely free market vs FDA controlled”. I could imagine a world where the FDA exists and still selectively provides its stamp of approval, but companies are still free to market their products.
In other words, the FDA would still provide recommendations, it just would not restrict entry into the market. I like this solution because most people would still need and want to go through their doctors to acquire therapy recommendations and oversight, and these doctors would likely still look to the FDA for approval and review of the data. However, it allows the desperate parents whose son is dying to gamble on a therapy.
I agree. But I think people should pay for most medicines themselves. When they spend their own money, the arguments for denying access are weak. Insurance complicates the picture because we need safeguards when spending others' money.
Fair point, but also in an insurance system where others are paying in and subsidizing treatments I think it is fair to not greenlight a drug that shows little gain and comes with a substantial cost (from my Google, the drug would cost $1million per year). If parents want to pay for a drug 100% out of pocket that is another story, but it's also the FDAs job to help regulate safety. This drug has questionable safety, and imagine it gets let into market and then people are hurt by the drug. There would be lots of blow back on the FDA for approving a drug without proper safety data.
If we’re being honest, insurance companies don’t cover lots of treatments as it is. I would doubt that in this hypothetical any insurance companies would cover a treatment that didn’t receive FDA approval.
Your point about insurance is dead on. I'm all for allowing people more access to try experimental treatments for these types of diseases. I am not fine with those experimental treatments being covered by insurance without strong data on efficacy AND safety.
Vinay Prasad is well-versed in Institutional knowledge, ethical, and highly intelligent. He is a perfect choice to lead the FDA. The Wallstreet Journal, on the other hand, is such a hack newspaper, it can’t even get the lede right.
Imagine a government department, especially the FDA, actually asking for medical treatments to be safe and effective. Vat a Country! as a Russian comedian used to say back in the 1970's in his shtick. I agree with Dr. Prasad - it's high time government was more accountable and less gravy-train.
Still: even so I - as a German not being too much bothered by FDA decissions - agree with all your arguments, John, it would have been wise to claim your COI with your long lasting friendship with Vinay Prasad. You, as well as Vinay, often asked for it for others and even so this is not a financial COI, it still is one.
https://open.substack.com/pub/kevinbass/p/trumps-fda-picks-now-under-coordinated?r=kia1s&utm_campaign=post&utm_medium=web&showWelcomeOnShare=false
I also am offended at the indemnification of the 'drug' companies re vaccines...if we are taking in the wild west approach. One would think safe and effective would negate the need for such blanket protections.
My guess about cardiac function not chosen as the primary outcome is probably it takes much longer to see clinically meaningful change and would need very large sample sizes. So could be a feasibility issues. And usually PO is approved in talks with FDA.
The point of having regulation of pharmaceuticals is to try to ensure that marketed products have demonstrated safety AND efficacy.
This is already in distinction to unregulated “nutra-ceuticals”, which to me appear to fall into the “safe but useless” category almost without exception.
So maybe this company should’ve just marketed this as a “DMD supplement” and gone to town with it.
I guess the issue here is that the company has failed to demonstrate either safety or efficacy with their 20-pt study. So in some ways it’s even worse than your typical “turmeric enhancer”.
On the one hand, I get that you’re not going to have 1000 pt RCT for such rare conditions. OTOH, it would be irresponsible for a regulator to endorse a product that gives nothing more than false hope (based on available data to date).
ONE percent improvement in ejection fraction?
A statistician might be impressed. And certainly Capricor investors. No clinician would be. And yes, I am one.
This is pharma financial skullduggery. They’ll trot out a couple of ‘concerned parents’ to cry the blues and write letters and such.
Sorry. The drug is a BUST. The end.
I got paywalled by the WSJ but was able to read enough to see the author’s bias. We are all biased to a degree. I have to hope that Vinay’s role will help reduce what I used to say during the opiate epidemic: FDA approval = business deal made.
PS - for some reason I don’t see Vinay as a Fouchi in any way shape or form.
Agree. It’s also nice to see a rebuttal of someone I respect… I like having my biases challenged.
Indeed. I try to look at all sides as well. Sometimes I even watch MSM! 😜
Here’s the problem with trying to hold the same RCT standard for rare diseases. Some of these diseases have fewer than 100 patients annually and demanding large randomized trials is simply not feasible. Demonstrating zero flexibility simply takes investments away and leaves these diseases unaddressed. I don’t have a problem with someone taking a stand on RcT across the board - you set the standard and live with the outcomes - but doing a whole road show about approving on “plausible benefits” and “small steps forward” and then demanding full randomized trials shows lack of basic integrity where actions don’t match words. I would not trust Dr. Prasad to be in any leadership role - let alone leading an agency like CBER. Sorry, I just don’t agree with the author.
An argument could be made that the FDA doesn’t care about these vulnerable patients lives- bc they will die soon anyway (thus give them all the experimental treatments). They need to be protected even more.
It’s a cr*p drug. Go back and make it a better one.
1% improved EF. Functionally irrelevant.
Having read the WSJ for 60 years I find Ms Finley's article like something out of the National Enquirer. I suppose there is an extreme libertarian position that would abolish the FDA and all government regulations but as a libertarian myself I would like to see objective scientific evaluation of treatments and drugs before I consume them. Thank you for taking the time to evaluate this article.
Did you not do the basic research to find out that the BLA was based on the OLE study at 3 years and the cMRIs collected during that time? No PUL data was included in the BLA, and the BLA wasn't based on the primary endpoint of HOPE-2. Research Soslow et al 2023 to learn about the natural history they're comparing against, and why the scientists previously in charge of the FDA thought it compelling enough to submit a BLA based on cardiomyopathy and not skeletal muscle.
Sensible Medicine is a media to promote specific doctors/politics or medical practices? I do not know Dr. Prasad and am looking for medicine here—not politics. Thank you TiredMedStudent, for a comment based on medical perspectives and not promoting politics.
Maybe you need a little more context before making a comment. Dr Prasad actually is very thoughtful and nuanced… im a medical provider but by no means an expert in reading studies… I read the WSJ article and I was inclined to side with the WSJ based on the article (even though I like Dr Prasad)… so I’m glad there was a balanced review of the decision that addressed some of the info in the WSJ article. Dr Mandrola is a lot more credible in reviewing this situation than a WSJ reporter. I think the back and forth conversation is super enlightening and I’m continuing to learn. Thanks Dr Mandrola. This isn’t political- it’s the art of debate of ideas.
Multiply this by 1000. Everyday, reporters who are not experts interpret information way above their pay scale, and influence naive readers.
Completely agree that the study is insufficient to prove that the therapy works. However, I think Dr. Mandrola oversimplifies the 2 possible functions of the FDA/how drugs could enter market. I don’t think it needs to be as black and white as “completely free market vs FDA controlled”. I could imagine a world where the FDA exists and still selectively provides its stamp of approval, but companies are still free to market their products.
In other words, the FDA would still provide recommendations, it just would not restrict entry into the market. I like this solution because most people would still need and want to go through their doctors to acquire therapy recommendations and oversight, and these doctors would likely still look to the FDA for approval and review of the data. However, it allows the desperate parents whose son is dying to gamble on a therapy.
I agree. But I think people should pay for most medicines themselves. When they spend their own money, the arguments for denying access are weak. Insurance complicates the picture because we need safeguards when spending others' money.
Fair point, but also in an insurance system where others are paying in and subsidizing treatments I think it is fair to not greenlight a drug that shows little gain and comes with a substantial cost (from my Google, the drug would cost $1million per year). If parents want to pay for a drug 100% out of pocket that is another story, but it's also the FDAs job to help regulate safety. This drug has questionable safety, and imagine it gets let into market and then people are hurt by the drug. There would be lots of blow back on the FDA for approving a drug without proper safety data.
Especially a drug that’s harming vulnerable patients
If we’re being honest, insurance companies don’t cover lots of treatments as it is. I would doubt that in this hypothetical any insurance companies would cover a treatment that didn’t receive FDA approval.
Your point about insurance is dead on. I'm all for allowing people more access to try experimental treatments for these types of diseases. I am not fine with those experimental treatments being covered by insurance without strong data on efficacy AND safety.
Here's Finley's subhead: " A top FDA appointee who doesn’t think patients can be trusted to make their own healthcare decisions."
I didn't make it much further. What a ridiculous smear on Vinay.
Vinay Prasad is well-versed in Institutional knowledge, ethical, and highly intelligent. He is a perfect choice to lead the FDA. The Wallstreet Journal, on the other hand, is such a hack newspaper, it can’t even get the lede right.
Isn’t Makary over the FDA and Vinay over the vaccine studies?
Dr. Prasad is FDA Chief Medical and Scientific Officer and Director of the Center for Biologics Evaluation and Research (CBER)
Thanks! I see Makary is the commissioner. His title makes more sense than just over vaccine testing.
Imagine a government department, especially the FDA, actually asking for medical treatments to be safe and effective. Vat a Country! as a Russian comedian used to say back in the 1970's in his shtick. I agree with Dr. Prasad - it's high time government was more accountable and less gravy-train.