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 salesmen are faking trial data to get their payday, but that seems solvable.
Even simpler is to prohibit payments for these experimental treatments, but let people who whould die anyway participate in them as long as it is free for them. In return the drug companies could use the outcomes as data points towards their aproval applications.
Why would a drug manufacturer do that? Why would they offeir their advanced but as of yet unaproved drugs for free? It is true it would cost them the manufacturing cost of the drug, but what they get in return is data on how efficient/safe the drug is. Assuming the drug works well they could use that data to prove their case and get approval. And if it is safe and has the desired effect they get their aproval and they could then charge for their drug as usual.
This sets the right incentives for the drug company. If they know they are selling snake oil they would be crazy to offer it for free, especially because all it would do is to prove that it does not work. If they think it can work they gamble with their own money. Which is good because the drug company knows the most about their own drug. The patient in general is not knowledgeable enough to evaluate the chances of an experimental therapy, but the drug company is.
Hell I'd even approve my tax money to go to those companies which end up saving lives as reward.
That would never happen for good reason. Drug companies would have every incentive to bias the results towards approval by giving the drugs to detain patients. I’m not exaggerating when I say this might be the worst idea I’ve read in this entire thread.
What do you mean “detain” patients? Remember we are talking about people who have a terminal illness with no known cure. How can drug companies “bias the results”? Obviously I’m not saying we take anything they say as gospel, but in short order these patients are expected to die. If a drug managed to cure them that would be quite hard to falsify don’t you think?
> I’m not exaggerating when I say this might be the worst idea I’ve read in this entire thread.
Well, thank you. I aim to please. Are you sure that you understood the idea well? If you are, could you please explain with more words what is the misbehaviour you are worried the drug companies would commit?
I meant to write "certain".
> How can drug companies “bias the results”?
Choosing appropriate patients will lead to longer survival rate unrelated to the drug in question. The whole point of double-blind studies is to avoid this.
> Well, thank you. I aim to please. Are you sure that you understood the idea well? If you are, could you please explain with more words what is the misbehaviour you are worried the drug companies would commit?
I did understood you. You, however, don't seem to understand statistics or economics. Your idea directly incentivizes drug companies to choose patients more likely to make their drugs look better. It is a truly horrible idea.
Yes. So? The whole patient group in question is destined to die fast. The best we currently can offer them is aleviate their discomfort and help them come to terms with their demise. If the drug company can identify a subgroup who they can possibly save and actually help those that is winning.
> You, however, don't seem to understand statistics or economics.
And you are abrasive and rude. These exclamations don’t add much of anything to the conversation. If there is something particular I am overlooking please adress that instead of speculating about the areas you think I am deficient in. Thank you.
You are aproaching it as if the question we are asking is if this is the best study design. But that is not the question we are asking. There is a patient who we believe is about to die soon. In comes a drug manufacturer and says to the patient: “you do not have to die. I think I have the cure for you”. The patient wants the drug the drug manufacturer is offering. The question is if we are to interject and stop the drug manufacturer giving the patient the drug, and if so under what circumstances.
I am saying: “yes, we should stop the drug manufacturer if the drug is not proven to be effective yet, and he is asking for money for it. There is too much of a chance that the drug manufacturer is a snake oil salesman in disguise and only wants to extract the wealth from the patient and the patient’s family. But if he is honest they can give the drug for free.”
In turn you are saying. “We should stop the drug manufacturer giving the unproven drug to the dying patient even if they are giving it for free, because it is possible that they cherry picked this patient. So though luck dear patient, you must die in the name of scientific rigour. I hope you understand.”
Do I understand you right?
This is already largely possible in most states and called "right to try".
> Do I understand you right?
No you seem not to be. Giving away drugs is fine and largely already possible. Choosing patients and then pretending that the data gathered has any reasonable statistical validity when considering general approval of a drug is totally crazy. I think there is essentially zero chance the FDA would ever go for an idea with such overt conflicts of interest so I'm happy your idea will never turn into reality.
The article’s author is convinced it is not covering them. Do you know otherwise? Perhaps you should let them know. You might save a life.
> Choosing patients and then pretending that the data gathered has any reasonable statistical validity when considering general approval of a drug is totally crazy.
Would like to mention that it is your adition that the drug manufacturer chooses the patients. It was not part of my original comment. And it is not core to the idea.
One can simply imagine a protocol where the manufacturer declares their criteria (what condition, what severity[1]) and how many doses they are able to provide and those “places” get filled up with volunteers. And this of course can be made even more buletproof.
1: and before you point it out, of course if the manufacturer is only willing to test their drug on not-so-bad-but-dying people then they get approval to be used on the same selection.
Experimental treatments being available to people with no options (there must be no applicable option with a proven probability of success or this becomes very questionable) could save drug companies millions-billions in the off chance that it proves to be incompatible with human testing or fails to produce the expected effects in humans.
I would take this up a notch or two for patients in a short-range situation that is certainly fatal, opening the door for them to try treatments that have just barely crossed en-vidrio and seem to have acceptable toxicity in mouse models. This would essentially be donating one’s body to science but in a much more useful form, with the side benefit of a slim chance of life extension.
This could benefit humanity by significantly reducing time to market for some drugs and cutting costs on eventual dead-ends.
If everyone you give it to dies from reactions, or the expected effects seem to be happening on a physiological level, you don’t know much about efficacy but you do know a lot more about the eventual prospects for an actual clinical trial. It’s basically an extension of en-vidrio testing.
The HepC drug Sovaldi brought in around $50B-100B in sales, but probably costs less than $50 to administer. Under your scheme it would make economic sense for a company to kill 1,000,000,000 people testing variants until they chance upon the right one because they incur no costs for administering deadly experiments.
The problem with your scheme is that human lives are valuable. A unsafe drug that causes harm or death is immensely destructive. If we want to be completely reductive, the actuarial value of a human life is around $10,000,000. A unsafe drug could kill the person resulting in a $10M loss to society. Therefore, the company should be required to escrow $10M per person until the treatment is done, paid out proportionally to any potential death or harm in excess of the gold standard treatment. Only then would the incentives be reasonably aligned.
This is obviously a very reductive treatment, but it helps illustrate the problems with unrestricted testing.
I was only talking about testing on people who we know are going to die fast because of their condition, and we do not have a way to save them in any other way. And naturally only with their consent. People who have a ticking clock and therefore willing to try anything.
This is the context the article in question talks about.
You have enough stories like this (plus there will inevitably be some mistakes by the FDA that will be shown to be proof of government perfidy) and the conspiracy theories will have a field day and fuel a black market.
Creating an equitable drug approval process is a true dilemma.
The ethical tradeoff between definitely saving a life now or probably saving many lives later is an age old problem, but one which society hasn't properly converted into laws and processes in medicine.
I doubt any company anywhere (legitimate or not) would provide drugs under such conditions. It would make much more sense to just focus on getting approval instead.
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.
Lets not forget the expansion of the DSM which pretty much incorporates every human being as having a mental health issue or sexual deviancy fetish. There is no normal in the DSM, so would some medical experts start dialling back on diagnosing new conditions to pad out their careers and income?
There is a huge difference between being down after a loved one's death and finding yourself feeling that way for no reason or not coping 2 years later.
Considering that - there is no way to answer the last question. That's just a "what if" argument and fails to take into consideration that there is a shortage of mental health experts, and there is absolutely no need for someone to do that sort of thing.
And no, no everyone has a "sexual deviancy fetish", just for the record.
As far as 'treating symptoms'... I have MS. I take medication that slows the disease. If I had fatigue or pain, they would try to treat that too. If it causes depression - which it can - they'd try to treat that. No matter what treatments I get for symptoms, I still have the disease. There is no actual cure.
Define normal? Likewise considering the all encompassing vagaries of some words, as noted in legislation, and the lack of detail that exists in descriptions of diagnosis and symptoms, whilst this makes it easier to generate AI's to replace some experts, its not really helping the patient is it?
>And no, no everyone has a "sexual deviancy fetish", just for the record.
Define normal.
> There is no actual cure.
You are uniquely placed to actually find a cure, because I'm reminded of how tolerant (lab) animals are when level 1 of maslows heirachy of needs exists, and I'm also reminded that (lab) animals can not convey information that an owner of a pet or intelligently constructed lab experiment could elucidate.
The phrase "Dont take no for answer" springs to mind, and Thomas Pynchon "If they can get you asking the wrong questions, they don't have to worry about answers.".
Survival of the fittest comes in all shapes and form. I know that copious amounts of histidine a precursor to histamine also causes remyelination of the nervous system, so maybe you have an amino acid lead that might be worth investigating.