And they're slow to avoid approving dangerous treatments, but that's absurd since the patients are already dying. The risk of not approving needs to be taken into account.
https://www.fda.gov/patients/learn-about-expanded-access-and...
There isn't really a single baseline untreated group. For a comparison between groups to be valid, all groups must be obtained by unbiased random sampling of the same population. In a clinical trial, that population is the patients served by the participating clinical center. Patient characteristics differ by time and place.
You can try to retrospectively construct a control group using a case control study design, but then you're getting to pick what control group to use, so the results are less reliable (more opportunity for human bias).
Unless a treatment is both miraculous in effect and works for everyone, it's hard to figure out if it works.
Yup. It's worth noting that "all or none" evidence is still considered category 1 evidence on many scales. (If you treat a group where all would be expected to die, and some survive... or a group where many would be expected to die, and all survive). It's only valid for the most dramatic effects, but you don't need randomization. During a safety trial you might come up with "all or none" evidence if your effect is strong enough.
But otherwise, you're going to need to compare the treatment to something else. There's no ability to magically draw the exact same population from some earlier trial.
In the best case, you end up bankrupt. In the worst case, you end up bankrupt and dead.
This is only a slightly more extreme version of the Sackler problem.
Deregulation sounds great if you believe everyone is logical and has accurate information to make decisions for themselves.
I'm sure there must be a much better solution than what we've got for the people who are dying.
But I doubt the answer is to just let drug companies sell anything to anyone and make Medicare pay for it.
We pay people to figure out that this is happening (the police), then we prosecute the doctor and if that is what happened we hit the doctor with the full force of law, and they never walk as a free person ever. We also do the same thing with those who bribed the doctor and they also never walk as a free person again.
What you describe already crosses into criminal conduct. We do not need FDA approval process to prosecute it. In fact I’m not sure how the FDA approval of the drug prevents it in your opinion.
Does it? How many people from Purdue Pharma went to prison exactly?
There are no consequences for the behavior you describe regardless of whether it's technically illegal.
Have they told the patient that they are dying?
Treatments should have solid evidence they improve overall survival when compared against the best treatments available today, and unfortunately too many studies either aren't aiming at revealing that info, and/or are comparing against inferior treatment options.
You might as well be saying "what happens if some improbable risk of X exists in this very unusual context". That's existence, especially in complex and fraught situations. You can't completely regulate it away and especially not if the regulations themselves can cause much greater harm in the much, much more probable situation of patients who are already dying being willing, as human beings with autonomy, to try something personally risky.
Deregulation shouldn't and doesn't depend on the world being one of everyone being logical and always having accurate information. Those two conditions don't exist in the human world at all times, period. Furthermore, the exact same problem applies to regulators and legislators as well, whose poorly reasoned decisions can cause broad harm too. Making a lack of perfect information and attendant risk into the key basis for onerous regulations is bad reasoning with sometimes grotesque consequences, particularly in situations where the regulations are known to cause suffering to people in extremes (as is the case with denying risky treatments to those who are in any case at death's door..
Also, very basic but obvious: anyone in the extreme situation of having a doctor suggest a very experimental treatment can go and get a second opinion from another doctor.
It's a largely different clinical and treatment situation from specific cases in which people are genuinely, terminally ill and a genuine though dangerous treatment option exists that might save their very lives. The Sackler case is a valid and powerful criticism of medical/pharma dishonesty, but it's extremely unfair to desperate patients that it be used to prevent them from having the autonomy over their own bodies and literal chance at life that they might legally be allowed to pursue.
Also worth noting that even in the market for prescription opioids, the Sackler case has more recently been used to wrongfully prevent patients who are in deep pain from obtaining a drug that provides needed relief despite its addiction dangers. So even here, obsessions about malpractice are hurting legitimate use.
If it's experimental it shouldn't be expensive. In fact it probably should be free until it's approved as effective. So with that out of the way, what are your other objections? (I am genuinely interested)
That was like Robert Stack, in Joe Versus the Volcano[0].
Most competent doctors will explain WHY they cannot prescribe something, and it's usually more specific such as "your liver is failing and this drug will accelerate that process, perhaps we can find something else".
But the pharmaceutical companies really want to prove that their drugs work. If their drug doesn't work, nothing is lost or gained by having people try it.
If the drug does work, but the study of it is confounded by giving it to people in a haphazard way, such that we don't know if it works--- more people suffer.
It sucks, but most things don't work. Occasionally people are screwed by not being able to get into a trial for something that might have saved them or lengthened their life. But much more often they're spared false hope and suffering from side effects, and we end up with trials that we can trust.
The incentives of the FDA are unfortunate. If they don't approve a drug that would have saved 100k people there is no bad press for those 100k deaths. But if they approve a drug that kills 1k people there is a lot of bad press.
So they have strong incentives to not approve anything unless absolutely needed.
Aka one hell of a tough call
The solution for terminal cancer patients is to let them use any experimental services they please.
Next.