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President Trump will likely nominate Dr. Scott Gottlieb as head of the FDA. Though he is presently a resident fellow at the conservative American Enterprise Institute and a partner at a large venture capital fund, he used to be an FDA deputy commissioner known for advocating dramatic reforms in the process to approve new medical products.
According to his statements as well as comments to people familiar with his thinking on the FDA, Gottlieb intends to shoot for the rapid approval of complex generics, ushering in a wave of less expensive rivals to some of the biggest blockbusters on the market. He's also likely to spur the FDA to follow the course laid out by agency cancer czar Richard Pazdur in speeding new approvals, possibly setting up a special unit aimed at orphan drugs to hasten OKs with smaller, better designed clinical trials. Other potential reforms include the possible quick adoption of new devices that could be used to improve the kind of medtech Apple, Verily and others have been working on.
Gottlieb is viewed very favorably within the pharmaceutical industry as a regulatory reformer but not destroyer. If nominated, he will have been chosen over another high-profile name on the short list: Jim O'Neill.
The close associate of Peter Thiel, O'Neill famously suggested that drugs should be approved based on safety alone, letting consumers sort out what works. That left many fearing that Trump intended to toss out the regulatory framework for new drug approvals, raising fears that his idea of competition would allow de facto placebos to compete for market share.
(Score: 2) by c0lo on Sunday March 12 2017, @04:28PM (7 children)
So, Gottlieb says "stopping some trial after 3 deaths is an overreaction" and you ask me to trust him he'll rollback only some regulation which, presumably, are unnecessary, right?
Just what regulation do you think can be safely rolled-back?
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In constructing your answer, on top of "elixir sulfanilamide", consider:
- homeopathy - pure non-toxic water. You don't die because of it, you die because the original illness is not addressed. In the same category, snake-oil from various snakes pushed by noisy markedroids, swamping out the signal of a good cure;
- Thalidomide [wikipedia.org] - non-toxic on short time span, on medium-long term causes malformed children with 40-50% chances of survival. After studies of efficacy/safety, it still have (FDA-)approved uses [wikipedia.org].
https://www.youtube.com/watch?v=aoFiw2jMy-0 https://soylentnews.org/~MichaelDavidCrawford
(Score: 1) by khallow on Sunday March 12 2017, @04:50PM (6 children)
So, Gottlieb says "stopping some trial after 3 deaths is an overreaction" and you ask me to trust him he'll rollback only some regulation which, presumably, are unnecessary, right?
Just what regulation do you think can be safely rolled-back?
First of all, why should the trial have been halted? Gottlieb made an important point [time.com] at the time that the deaths could have been due to MS complications rather than the drug:
"Just seems like an overreaction to place a clinical hold" on the trial, he wrote. An FDA scientist rejected his analysis and replied that the complication "seems very clearly a drug-related event."
Even if the latter was true, that still means that the medication could be adjusted to attempt to reduce such drug-related events.
Second, a significant part of the problem is that pre-clinical trials (testing before testing on humans occurs) are notoriously ineffective at weeding out drugs that have problems in clinical (human) trials. We need some serious innovation in coming with with better pre-clinical trials. FDA is a solid roadblock to that.
(Score: 2) by c0lo on Sunday March 12 2017, @05:00PM (5 children)
Exactly how FDA roadblocks pre-clinical trials?
https://www.youtube.com/watch?v=aoFiw2jMy-0 https://soylentnews.org/~MichaelDavidCrawford
(Score: 1) by khallow on Sunday March 12 2017, @06:24PM (4 children)
Exactly how FDA roadblocks pre-clinical trials?
Let me first demonstrate that this is a huge problem [sciencemag.org] (speaking of :
So how is everyone doing in the clinic? Sheesh, you had to ask. The overall Likelihood of Approval” (LOA) from Phase I is 9.6 per cent. So no, if you were hoping for an upturn in the more recent numbers, it doesn’t seem to be there. Somewhere around ten per cent success has been the estimate for some time now [sciencemag.org], and here it is again [sciencemag.org]. The worst therapeutic area is oncology, with a 5.6% LOA. That reflects some of its traditional features: lower barrier to entry, which brings in a lot of marginal ideas, difficult diseases to target once you’re in the clinic, and a corresponding willingness to try some fairly out-there stuff.
These numbers shouldn’t be too surprising to folks in the industry itself. What I’ve found while writing this blog, though, is that people who don’t know drug discovery tend to be very surprised indeed. It bears repeating: ninety per cent of all drugs that go into the clinic fail. And ninety-five per cent of all cancer drugs. That’s the central problem of the whole industry, right there. If you can make those numbers look better, hundreds of millions of grateful patients (and untold billions of dollars in revenue) are waiting for you.
The point here is that approval of pharmaceuticals, medical technologies, and other treatments is a sort of inverted pyramid system where expense increases substantially with each step forward. When you get to the point of FDA rejection (one of the very last steps before marketing), it tends to be a huge financial disaster at present. It should be a huge warning sign to us that we're seeing a 90% failure rate (which gets worse as one goes into certain areas like cancer treatments with particularly bad animal models) from human testing (with apparently still a 40% failure rate going into the last and by far most expensive phase of testing before FDA consideration). So right here, the formal testing regime currently approved by the FDA has a huge, rear-loaded failure mechanism and introduces considerable delay in the system.
It appears that final stages of human testing are for the foreseeable future unavoidable [sciencemag.org], due to human biology being ridiculously hard and conflicts of interest often being less than ideal, meaning we will have considerable regulation, delay, and expense even under the best of circumstances. But what we have is this lengthy, expensive, risky, remarkably unreliable process for doing anything to improve peoples' lives and health. That needs to change. The FDA has shown little interest in doing so.
(Score: 2) by c0lo on Sunday March 12 2017, @10:56PM (3 children)
Ok, I got the numbers. What I don't get is: what you see as "FDA roadblocks pre-clinical trials" which need to be removed?
The only way that I see the higher chances of a new drug to be proven effective and safe with reduced costs is to have the drugs failing the process as soon as possible thus removing extra cost in advanced steps.
Do you see some other way here?
https://www.youtube.com/watch?v=aoFiw2jMy-0 https://soylentnews.org/~MichaelDavidCrawford
(Score: 1) by khallow on Monday March 13 2017, @06:42AM (2 children)
(Score: 2) by c0lo on Monday March 13 2017, @07:26AM (1 child)
Those hoops, added or removed?
If removed: how this is going to drive down the costs if the drug turns out to be toxic? Given that human testing is much expensive than testing on model animals, I believe its their interest of doing the best the pharmas can in pre-clinical trials, when the costs are lower.
If added: how's this removing roadblocks from pre-clinical trials?
I'll let aside we are barely at the level of cloning organs (from stem cells), much less at the level of cloning humans without brains.
What stops them from using any line of immortal cells [wikipedia.org]? At least the toxicity can be (partially) evaluated on a cell closer to human than any model animal.
https://www.youtube.com/watch?v=aoFiw2jMy-0 https://soylentnews.org/~MichaelDavidCrawford
(Score: 1) by khallow on Monday March 13 2017, @02:59PM
If removed: how this is going to drive down the costs if the drug turns out to be toxic?
It allows the drug company to solve that problem as they see fit before it gets to human testing.
What stops them from using any line of immortal cells?
As I recall, for a time, certain lines were banned from federal funding by the Bush administration. And the higher level Department of Health and Human Resources was for a time the means by which a ban on the funding of anything derived from human fetuses was enforced. There is a history of interference here.