261–270 of 486 posts
What I don't understand is why the FDA doesn't automatically rubber stamp approvals on medicine approved in Canada and the European Union. We all know their medicine is good - so why can't I get it?!
There's a very easy regulatory solution: Roll the drug approval process back to the way things were done prior to 1962. Back then, safety testing was all that was required. Efficacy testing -- which is difficult, expensive, arguably unethical in itself, and in some cases effectively impossible -- was not required. Drugs cost ~20-50x less to bring to market, and were brought to market faster. Indeed the 40s and 50s ar…
I remember Softenon. > it wasn't only because of low-hanging fruit. Yeah, right. They had such advanced cancer medication back then. > Give people with fatal diseases a "right to try" drugs that haven't passed safety testing -- and use that data. That data is practically worthless. I'm in favor of having people try out medication, but it will need to be heavily regulated. And we know what happens to regulation: a par…
This is the reflexive response to every common-sense proposal to roll back the regulatory burden.
I'd raise a couple of points in response:
First, Thalidomide was never approved in the USA -- for safety reasons. The mechanisms that were in place at the time did their job.
Second, the response to Thalidomide was overblown and indeed downright hysterical. "Better to let 100,000 people die of neglect than allow one person to suffer an awful drug reaction" is not rational policy.
> That data is practically worthless.
Enough "practically" worthless data, and you get somewhere. It's a matter of quantity. Obtaining "hiqh quality" data is often unethical in medical practice.
Likely, Moderna doesn't want him to take the drugs because they will probably be ineffective this late in the cancer stage. It doesn't want its treatment to be associated with his death.
[0]https://www.fda.gov/patients/learn-about-expanded-access-and...
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Maybe part of the reason that the Canadian and UK drug regulators are competent, is because we haven't created a sufficiently large system of incentives to undermine them. Maybe if approving a drug in Canada got you access to the entire world, we would have a very different scenario. A rubber stamping scheme removes redundancy from the system. Approving drugs faster sounds like a great idea, but there was a reason so…
There is no reason because it wasn’t a deliberate choice. Different countries created their own standards bodies and didn’t try to coordinate until later. The same thing happened with safety regulations around automobiles and aircraft. Fortunately in those cases, most countries have coordinated and manufacturers can sell the same vehicle in different countries with little extra effort. But for rare diseases with chea…
If you think the UK, European, and Canadian regulators are competent, then you must believe in the possibility of a competent regulator, right? Let's just have one of those, instead of a differently-incompetant regulator who serves as a rubber stamp.
And if we can't, then I'd rather they were too conservative than that they allowed too many drugs on the market.
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> The incentive isn't to cure but to treat. The Market wants (repeat) customers. I am fed up with this argument. Compared to a recurring treatment, a cure will: - destroy the competing recurring treatment - sell particularly well initially, when you still have the patent, potentially at a high price - not stop the influx of repeat customers, since as long as people are alive, they will need medicine, and people who a…
Gilead’s hepatitis cure is a real example of this[1], so it’s not some hypothetical. The sales plummeted and wall street dinged them for it. [1] https://www.investors.com/news/technology/can-gilead-withsta...
> Yee, though, says Gilead isn't filling a hole left from its declining HCV unit. It's merely coming down off a "massive bolus of hundreds of thousands of people who came in during the first two years."
> "The drug is still set to do $8 billion in 2017 and is one of the largest drugs in the world. Not exactly a hole," he said.
The Gilead stock is worth 4x today what it was worth in 2012, before they had the cure. It is better than the average "big-pharma" (ex: Pfizer). The stock plateaued because they couldn't follow up, but before you look at the dip, you should look at the massive raise before it.
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A simple way to stem most snake oil is to put all payments for snake oil treatments into escrow till the treatment is approved. Then the snake oil manufacturer can peddle their unapproved treatment to as many people as they like, but they are losing money on every dose unless the treatment turns out to be safe and effective. Obviously you need a big team of scientists on the approval panel to make sure no snake oil s…
>A simple way to stem most snake oil is to put all payments for snake oil treatments into escrow till the treatment is approved. It should be a escrow release when a patient is cured. Let the science and knowledge speak for itself instead of statistic doing the talking. The problem is these drugs companies are the stealth chemical weapons development program for the military.
This just winds up being cruel. We don't understand a number of diseases, and we don't know how to cure them.
But we can treat symptoms, and sometimes prevent them even if it isn't a cure. It's OK to make Insulin Plus that keeps diabetics alive longer. I have MS, and freaking trust me, I want my non-cure medicine. Without that medicine, my chances of having a very poor quality of life full of discomfort increases pretty dramatically. Lots of things are like this: No real cure, but we make folks lives better by treating symptoms or preventing some damage and things like that.
The only way to not be cruel is to give treatments that work even if they aren't a cure.
There should be a universal law in human rights stating anyone can take anything into their own bodies and governments can never, ever prevent them. Governments/regulators deciding what you can put into your body is beyond ridiculous: applies to all substances and drugs. Inform about the potential risks and effects: sure. Prevent: never.
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I remember at least three instances in my country where the authorities were forced to allow people to use experimental treatments due to media clamor. In all three instances, the result was that the treatments were snake oil and people died that might have lived otherwise. It's easy to say "people who have no other options should get access to experimental treatments" but the problem is that people who have other op…
> It's easy to say "people who have no other options should get access to experimental treatments" but the problem is that people who have other options want those too, because nobody wants to go through chemotherapy or whatever. I reserve for myself the right to try to save my own life, even if that means some people who can't handle that responsibility make things worse for themselves. I can't get from, "but other…
I think the ideal of regulation is to try to remove the guesswork & expertise required for the end user to do due diligence. It's not perfect, but at least some minimum level of regulation, such as mandatory efficacy disclosures, would be needed.
Earlier quoted context omitted.
There's a lot of problems with it, though. Even if advancing medical research was the main goal-- ad hoc use in dying patients provides unclear data. Medical research wants clear inclusion criteria, metrics, reduced statistical noise from similar patients, predeclared outcome measures, and economies of scale in running trials. We already let terminal patients participate in medical research, but only inviting patient…
>There's a lot of problems with it, though. [...] Medical research wants clear inclusion criteria, [...] , but only inviting patients at times and meeting criteria that optimizes the research [...] But terminally-ill patients push for inclusion beyond this: to be dosed when there is not an active trial, [...] Your objections are sensible but that's beyond the scope of this author's argument. I'm just taking author's…
The FDA doesn’t hold back researchers on inclusion/exclusion criteria.
Inclusion for the Moderna trial he linked to:
> Must have primary refractory or acquired secondary resistance to prior immune checkpoint treatments. Primary refractory is defined as prior exposure to anti-programmed death-1 (PD-1)/programmed death ligand-1 (PD-L1) antibody for at least 6 weeks but no more than 6 months with demonstration of progression on 2 separate scans at least 4 weeks apart but no more than 12 weeks apart and progression occurring within 6 months after first dose of anti-PD-1 antibody. Acquired secondary resistance must have confirmed objective response or prolonged stable disease (SD) (>6 months), followed by disease progression in the setting of ongoing treatment and confirmed progression on scans at least 4 weeks apart.
Exclusion criteria:
> Participant has received treatment with prohibited medications (that is, concurrent anticancer therapy including other chemotherapy, radiation [local radiation for palliative care is permitted with approval from the Sponsor]…
From the description of the patient’s treatment course it sounds like he had curative intent radiation (given he subsequently had surgery) and would probably not meet this study’s criteria making the limiting factor Moderna not the FDA.
He also doesn’t mention whether he’s starting immunotherapy/checkpoint inhibitors (or his tumor status) and may be lumping immunotherapy with chemotherapy which is a good new treatment option and would typically be offered if eligible. Definitely better than being enrolled in a dose study and in fact is required by Moderna.