FDA

“So I think if anything, the FDA has become an incredible collaborator to industry.”

“FDA risk or regulatory risk is really just another way of saying scientific risk, of saying we're going to conduct an experiment, and we are not quite sure what's going to happen.”

“Ironically, I think, you know, most people think about the FDA or CLIA being your big, ah, concern. I think reimbursement's probably the first place to start, because I wouldn't want to sort of be designing a test without having the confidence that I'll get re…”

“We actually did a hearing on my subcommittee to force the FDA, the regulator in this case, to tell us, is an iPad a medical device?”

“It was only relatively recently that the FDA Allowed clinical data to be entered directly into computers as opposed to put on paper because there was a reluctance to trust the quality and integrity of that data, which I think we would all agree is kind of sill…”

“Presumably will be an FDA that'll be more likely to approve experimentation and special trials to get beneficial drugs and technologies out into the market sooner.”

“There's no way right now that any drug is going to be approved for treatment of healthy people.”

“You probably couldn't even get it through FDA if you didn't have an indication, like staying healthy longer was not an indication.”

“The second one that I think had a negative effect was the Vioxx situation in 2005, and, you know, there was just a small increase in deaths with this drug, but there were so many lawsuits over it, That people at the FDA would get called on the carpet for appro…”

“If you wanted to make an artificial heart, what the clinicians did in 1967 is they said, well, what object in your house has a good flex life like a heart? And they came up with the idea of a lady's girdle. So they looked at what that was made out of, and It's…”

“The regulatory risk thing is being largely addressed by this new Office of Digital Health and the FDA, and they're really doing, seem much more forward thinking about it.”

“We'll see more generics approved this year than in any year in history previously.”

“I mean, one example we launched in the U S a digital medicine. I mean, with paratherapeutics, this is the first digital app with an FDA label that's being used for opioid addiction and other psychiatric illnesses. And it is literally an app That has run clinic…”

“Since the creation of the FDA, there's only been about 1500 new molecular entities ever found.”

“In a world where things have gone past clinical trials, but really now the new barrier is not getting past the FDA, the new barrier is reimbursement.”

“So first, our special guest is Amy Abernethy, who has been named the new principal deputy commissioner of the FDA, the U.S. Food and Drug Administration, pending ethics clearance.”

“When I'm at FDA, I will have a trans-FDA remit, so I will need to spend my time and energy working on the things that the FDA needs me to do, and I'm looking forward to learning more and more about that every day. But I know that I'll at least spend some of my…”

“To some extent, I think they like new technologies more than they like the old ones. The old ones tend to fail because they're so incremental that they're no longer compared to the placebo, they're compared to whatever they're in increment over, whatever thera…”

“This past year we've seen FDA and CBER leaders drum up plans to make a big push on assessing new cell and gene therapies, as well as introducing new guidance on accelerated approvals for these new areas of product development.”

“In fact, the way the majority of our applications still come in is through PDFs or sort of essentially large digital representations of what used to come in on trucks.”

“And one of the things the FDA does in concert with CDC and now really through international database is have, is maintain a database of all the different genomes so that we can also track back and do this more quickly.”

“Can we come up with, essentially, preconditions for software updates? So that if there are strong quality controls in the way software is developed, well understood product performance as an expect, in terms of the expectation updates, can you now have algorit…”

“One of the things we've been contemplating at FDA is, like, ultimately, could you imagine the application on your phone that allows you to scan peaches and understand did the peaches have a full supply chain that we can monitor?”

“We've seen drugs approved based on phase one data.”

“Historically, the way we've often thought about effectiveness is sort of as a fixed book of work, and I think that what you're going to see Over time is we're going to talk more and more about longitudinal performance, and this is a perfect example of that.”

“Even information that is de-identified from a HIPAA perspective actually still is probably re-identifiable even in our context.”

“So practically speaking, we can help reduce the number of opioid tablets, for example, a patient has access to after back surgery or knee surgery in order to reduce the chance that this particular person has access and becomes addicted in the first place.”

“Practically speaking, if the kit was going to be approved for commercial purposes with claims and labeling, et cetera, that's when it starts to get into the FDA perspective. It gets really murky when we live in this landscape of the internet without claims.”

“In 2018, we had something north of 50 drug shortages, but the little known secret is that we helped to avert over a 160 drug shortages.”

“Last year we ultimately developed agreements with USDA so that the parts of the cell cultured food, food activity that's got to do with cell culture, for example, and that, that part of the equation ultimately became The FDA's responsibility, and then as we mo…”

“And a drug or biologic will go through a series of clinical studies, typically phase one through three, although these days those lines are blurring for exactly what drugs, followed then by a new drug application or biologics application of BLA to the FDA.”

“And it's true that we update the rules every year, but we do so by hand.”

“So it may not necessarily be a software product that's acting As a diagnostic or treatment activity, but it's a software product that's intended to support life sciences more globally, whether that's to make clinical trials more efficient, to match patients to…”

“If there's a great drug that ah, that nobody ever gets, you know, nobody ever knows about it. Let's say there was a cure to cancer, but the FDA didn't approve it. There's no outcry because, ah, no one ever knew about it. But on the other hand, if the FDA let s…”

“Not only are we responsible for regulating about you know, 20% of international GDP as I mentioned, that sort of was across a broad number of products,”

“And I would argue that in a time of rapidly emerging biology, when we've got more and more scientific innovations and potential products coming to bear, The need to make sure that we have an objective way of assessing safety and effectiveness and providing con…”

“And so right now we've got an experiment going on where we're looking at machine learning based prediction of which trucks we should inspect on the border.”

“In my CIO role, I just recently started pushing on the fact that I really think we need a chief privacy role at FDA, which we don't currently have.”

“It's because media Only covers it when a drug doesn't work and doesn't cover it when a drug does work, but it's been delayed.”

“And you could have avoided like a million dead. That million dead is on the FDA's head, like, because challenge trials could have been done, you know?”

“Fundamentally, they're miscalibrated on terms of risk, right? They want to minimize PR risk for themselves, the agency, as opposed to allowing people to take a risk on their own, right?”

“Basically, if you have a drug and it was held up by 10 years, and then once it was approved, it reduced morbidity and mortality by X percentage. That means FDA's delay of 10 years multiplied by that is how many lives it cost. Relative to just taking the risk a…”

“Once we can get a zone outside the FDA, once we can exit the FDA, people don't understand how bad the FDA is. Your entire life, it's like tens of trillions of dollars of values being held back by FDA. It's like incalculable how bad they are as an agency.”

“The FDA is looking to the skies and taking a page out of the FAA's approach to aviation safety, rigorous yet adaptive. Recently launching its own new office for therapeutic products and piloting Operation Warp Speed for rare disease to create more transparent …”

“The breaking news is that the first CRISPR therapy has been approved. This is a therapy for sickle cell anemia and beta thalassemia that was developed by a biopharmaceutical company called Vertex Pharmaceuticals, working with another company called CRISPR Ther…”

“Ironically, nothing that the FDA has done would have helped the phlegmide crisis because you don't test on pregnant women and so on”

“If you don't let the drug through, we're actually killing people right now by not getting these advances through, and I think human beings aren't very good at sort of holding that in their head.”

“If you had a drug that was as good as the best drug, but cheaper, That still can't get that won't go by the FDA because they actually has to be better than the last drug, even if it's not better scientifically, but it's better in other ways.”

“It's no more legal to field a new AI design drug without FDA approval than it is a standard design drug.”

“I think that this is a interesting, like, weird misapplication of good intentions in a policy way that's probably going to Kill more people than ever get saved by every kind of health, FDA, et cetera, policy that we have”

“We are the only industry that actually has an existing regulatory framework for approving products, especially in the clinical setting.”

“There will be, yeah. So that's something that Dr. McCary, the FDA commissioner is working on. And there'll be also you know, changes in, in like how Medicare pays, Medicare, CMS, Medicare, and Medicaid pay for Leucovorin. So it's a cross-agency collaboration f…”

“Personnel costs are not a giant aspect of America's budget, and arguably we need a lot more personnel in order to, you know, process drug discoveries and make the FDA much more efficient, Process high-skilled immigrant visas and have a much more functional gov…”

“Since the birth of this industry, we only had increasing regulation over time. I think there was like only one time in history of biotech where we made it easier to develop and approve new drugs. And that was during the ACE crisis where AIDS patients were just…”

“Since then FDA started to require not just safety, but also efficacy in the approval process.”

“There's a very interesting drug that I think is going to become far more popular soon, which is already FDA approved called Sunosi, S-U-N-O-S-I, which is approved for excessive daytime sleepiness.”

“As you mentioned, CAR-T, CAR-T was also approved in the same way as we believe Intesmiran will be, which is as a process BLA not a product BLA.”