The FDA is powerless to stop right-to-try profiteers
statnews.com
statnews.com
This strongly disincentivizes companies from leveraging the compassionate use pipeline, and patients are left to die and suffer as a result.
Also as far as profiteering goes, there is no way that an insurance company would reimburse for any untested therapy which by default would keep costs down, and patients would still be required to get a prescription from their physician who would hopefully review the trial data. The idea that this would be used as an end run around FDA regulation for huge profits is moronic.
Here is a problem: if a pharma company can simply make money from someone's desperation, they have a reduced incentive to actually make the drug work.
The situation is reduced to a simple scam: the business model of selling some desperate people the promise of a cure which is just snake oil.
We always have to consider the effect of how the flow of money in a situation creates incentives that have unintended consequences.
If that's not okay, why is okay in the case of a special class of patients? Because they will be dead soon anyway, so who cares?
The drug approval and regulation mechanism is what limits that sort of abuse. Remove it out of the way and the abuse will creep in.
The drug approval and regulation mechanism is what limits that sort of abuse. Remove it out of the way and the abuse will creep in.
Uhhh, I think you skipped over at least eight steps in your logic there.
Also, the whole point is that these people are not guinea pigs. They are allowed access to promising therapies in late stage clinical trials in the hopes that they will help them, not just random investigational compounds. Let's remember that these requests would require physician approval.
The result of blocking this is to say that dying patients should just die, tough shit, but hey at least they will die with more money right?
I mean that's wrong because the article directly states that they're charging lots of money for this therapy so that they have an incentive. If 'the real money' was in insurance at scale, why does NurOwn require an additional monetary incentive beyond free test cases? Obviously they care about being able to bill individual patients. Especially in right-to-try cases because the type of person willing to pay to try an experimental therapy is often the type of person who would hand you everything they own.
>The result of blocking this is to say that dying patients should just die, tough shit, but hey at least they will die with more money right?
Most of these people are going to die anyway and many will now die experiencing side effects they had no way of knowing about. They'll also leave their families nothing. Marketizing healthcare has already raced us to an ugly bottom. This law creates a new and even lower bottom to how pharmaceutical companies are able to take advantage of some of the most desperate people our society has. That's not a good thing.
I agree. But the problem with this new policy is that it incentivizes scammers which are new actors who do not care about trying to get legit patients at scale.
I'm not positive, but I think this only requires drugs to have passed Phase I clinical trials. And that, according to the article, NurOwn's Phase II trials showed it was unable to slow the progression of ALS compared to a placebo.
Small-scale quackery scams can be highly profitable even if there's never any move to large scale production. It doesn't cost much to run a clinic giving coffee enemas, but you can charge an arm and a leg. At that rate a few patients a year can keep you riding in Ferraris.
"hey at least they will die with more money right?"
Their kids might appreciate not being left homeless by an unethical fraud.
These drugs would be similar to or worse than drugs available on the Cancer Drugs Fund in England. We found that they i) are very expensive and ii) had no menaingful benefit. https://www.bmj.com/content/357/bmj.j2097
On top of that you've got to factor in unpleasant side effects.
There are better ways to die than spending all your money on meds that make you feel terrible and don't provide any benefit.
The FDA doesn't prevent you from taking whatever drugs you want, they (previously) just prevented companies from selling and marketing unapproved drugs as treatments.
The law has a misleading name, patients were already allowed to take any of these drugs.
There are definitely snake oil salesmen out there, and I do believe there is a place for the FDA to get in their way as much as possible.
I'd rather have more control over my life than less, even if it means there's risk. At least it's my choice which risk, not someone else’s.
Especially where, for instance, there are sick kids involved. You see campaigns in newspapers and on social media, for donations so that some poor, dying child with untreatable cancer can be flown to Texas or Mexico, and they just have to have $150K for the treatment and drugs they need. They aren't medically trained and they can't spot the scam - hell, they don't want to spot the scam, they want their child to live, and who can blame them? They've already taken out as much debt as they can afford. Their families and close friends have probably done the same, and they're campaigning for the general public to chip in too, for a treatment that's effectively a fraud. It's heartbreaking.
This is where we need someone to step in and prevent the behaviour.
Education is probably not any sort of cure for this - the scams are often run by educated, authoritative people who offer a fake lifeline to parents who are almost beyond hope and who are certainly not rationally evaluating the options.
There is a role for authorities here.
Yes but you're speaking as if the FDA removes control over your life in a way that a snake oil salesman doesn't. They both remove control, one explicitly and the other through trickery. In that case since one is ostensibly designed to protect you and one is explicitly designed to harm you I'm going to side with giving the FDA discretion.
If you want the FDA to protect you, fine, then the laws should allow you to get protection. But they shouldn't be designed to "force" me to accept protection... that sounds like the local mafia.
We're talking about the terminally ill here. Most of the time they have no future.
>The government has real "power" over you, a con-artist only get's you once.
Again, this is the terminally ill. In this case the con-artist only needs to get you once and he's going to take everything you own.
>If you want the FDA to protect you, fine, then the laws should allow you to get protection. But they shouldn't be designed to "force" me to accept protection... that sounds like the local mafia.
It's about preventing new bottoms in society. Let's say we allow the poor to sell their kidneys. We could say: 'well it's purely optional, if you don't like it don't sell your kidney' but we're really ignoring all the externalities of creating a new market. Since those willing to breach this new bottom now have more cash, maybe payday loans will require kidneys as collateral, maybe rates will go up for those who don't want to sell their kidney. In that case you have definitely negatively affected all sorts of people who are making the choice not to sell their kidney. The collective decision of society, to allow this new bottom has transformed the circumstances of everyone in society, not just those who go to the new bottom. That's why we must look at that bottom and collectively evaluate whether the costs are worth it.
To bring it back to right-to-try, it seems that 'early access treatment' is probably going to be plagued by some of the same problems we see currently in 'early access gaming' - it's expensive and glitch-ridden, the landscape can change without notice and developers feel no rush to actually finish the game since they're getting paid anyways. Does that seem like a bottom we want to allow in healthcare? I say no.
I appreciate the need for give-and-take and the pull between parties to ultimately end up finding the best result. Sometimes things have to be tried before they can be fixed. I think forward progress only happens by taking risks, and this seems like a pretty mild one in the grand scheme of life.
I have an uncle that spends over a 1k _per pill_ to help him with his disease, I think he's already being ripped off by someone. Maybe this law change will give him an alternative previously unavailable. Or possibly add competition so the prices for his medication go down.
I agree wholeheartedly that if there is a genuine treatment in development, and if someone is on the path to imminent death, and there's the possibility it might cure them, or significantly enhance the length and quality of the life they have left, then denying them access to it is unforgivably cruel.
But I'm not sure how you filter out the charlatans that have fraudulent products in perpetual 'trials', who want to take people for everything they have (and far more).
https://arstechnica.com/science/2018/06/new-drug-access-law-...
Wrong. The patients just get second mortgages and/or crowdfund it. And people contribute because they don't know they're paying for the person to be subjected to useless quackery.
In decades past, researchers could come up with ideas and use them on patients who had no other options, and often saved lives that way. The field advanced quickly. Now progress has slowed and clinical practice lags well behind the state of the art in research, largely due to FDA restrictions.
So I suspect the book's author is happy about this law. Here's the book: https://www.amazon.com/Death-Cancer-Pioneering-Oncologist-Wi...
The problem isn’t that they get to try an unproven treatment to see if it works. The problem is that they get to charge desperate people huge amounts of money for it.
Do it for free, as a way to validate a drug and advance research and this wouldn’t be a problem (or at least it’d be a completely different problem).
The rule prevents negative outcomes for risky non-qualified patients from harming the trial, but the converse is also true. Positive outcomes for patients outside the regulated trial will not help gain approval at Phase II or Phase III.
On a board like hn, there's a tendency to look at all data as useful data, but this isn't true in the case of a clinical trial.
On further thinking, I'm not even sure what happens in the case of a patient receiving a pre-approval drug outside of the trial. Handling their data would likely need to meet the same standards required in trial (consent tracking, electronic signatures, etc) without any of the machinery of the trial to help. It could well be the case that nothing is collected if the cost to keep it and use it are too high.
IIRC, some of this was handled by FDA (or could have been), if they were part of the process by granting an unapproved treatment waiver. This way some amount of 'informal' data could be collected and used to guide others, even if the treatment was outside of a formal trial. Now, without FDA having a role, I fear that none of this data will be captured (or at least not in a useable form).
You're aware creating products costs money, right?
Your argument sadly does not add much to the discussion. If we were debating whether to allow people to take these unapproved pills or not you would be right. But we are talking about allowing companies to market and charge for totally untested and unapproved (stated) cures.
If these companies would need to test their treatments to prove that they are really working, why then should people not only bear the risk of (till then unknown) side effects, but also pay hefty sums for that "right"?
And Steve Jobs might be alive if he listened to actual science.
In decades past, there wasn't anything effective for cancer. Now there is, and the bar should be much higher.
We are winning the war against some cancers. It's just that we've picked off the easy stuff, and now we're left with the hard stuff.
There are still lots of people dying of cancer, and lots of new treatment ideas that we could be trying on those patients, but haven't been due to government restrictions.
It's a false dichotomy to say our only choices are the FDA or snake oil. In oncology at least, we used to be better at this.
Approved by most of the agencies in Europe, but denied by the FDA, it turned out it was really really bad.
So yes, the FDA does slow things down, and yes, people have a right to try what they want, but just keep in mind that the FDA does serve a purpose.
[0] https://en.wikipedia.org/wiki/Thalidomide#Birth_defects_cris...
There already is an enormous incentive for therapies that work. Whether you agree or not, you kinda have to admire the leaps of logic that allow people to rationalize needing to make huge profits on unproven trials from day 1, for an industry that has some of the highest profit margins of any.
Big Pharma profitability generally is pretty good but not stellar, unless you only look at profitable drugs and ignore unprofitable drugs.
Everytime I've gone looking for this, it's just been confirmed over and over.
Ergo hookers and blow? I don't buy it.
http://fortune.com/fortune500/list/filtered?sortBy=profits&f...
Every therapy was unproven at a certain point in time. It can only become proven with time and tests.
Now, I agree that an experimental drug should be sold at a discounted price, or even free. We can totally discuss that.
But blocking the last possibility of hope of a dying person is just being petty.
>We can totally discuss that.
That's what this article is trying to discuss.
As for price, it should be free. The reward for making an unproven drug that works should be fast-tracking them through the FDA approval process.
Also, how does the market work if the customers die (because the treatment is a sham) before they can warn other potential customers? What stops me from peddling snake oil to every dying person, knowing that other dying people will never hear about how it doesn't work?
Would this allow for access to scheduled substances?
Could this law be useful in that regard?
Most disgusting news I've read this year.
Previous trials with the VA found 86% of soldiers with PTSD no longer had it after a couple sessions.
So, in this case illegal drugs are. It’s the only way they would ever get unscheduled, as schedule 1 means no known medical use.
There was a study cited where they accidentally used Meth vs MDMA as evidence to schedule it and then after it came out the study made a mistake, the legislation still stood.
But keep in mind that most other drugs were used medicinally before they were scheduled.
Cocaine, Pot, LSD, Mushrooms, etc were all legal before they were illegal.
The war on drugs is a relatively new obsession.
I'm not saying the FDA has no purpose but it would be really hard to convince me that giving additional options to people with terminal illnesses is a bad thing.
If anything we need more experiments like that. If you have no treatments left to try and you are dying, being able to try something that is not 100% proven but that has some potential is better than nothing. It's not like selling snake oil or something, most therapies have clear identified pathways and targets based on actual fundamental research.
Companies are hardly going to make tons of money on unapproved therapies anyway, because the word will get out if x patients tried it and had no benefits.
How does reducing the number to the wealthy help here? In Germany at least, your insurance will surely not pay for this.
Margins are so high in this sector, why should they prey on the hopeless to turn a bit more profit, when they'll make huge ones anyways once the drug is finalized?
Here, you seemed to have missed this part.
There's also the fact that even when a drug does successfully pass through the FDA testing process, there can be a period of up to a year between the drug being successful in Phase 3 trials and actually getting approved for prescription. If you're a terminally ill patient, a 12 month waiting period between "proven in phase 3 trial" and "actually finally getting the FDA's rubber stamp" can be a long time.
Which is what this article is about: you can have no choices, but here, pay us a ton of money for UnprovenDrug(TM)! It might work according to nobody but us.
You're making the classic misstep of assuming a Pascal's wager type situation - as though there's only ever 1 unproven option out there so you might as well, when in reality there's many and getting scammed out of all your money on one means you can't try others which actually might work.
Most groundbreaking new treatment don't cure you forever, they only prolong your life for 6 month or 1 year. (After more test and improvement, they extend the time more, hopefully many years.) So don't expect too mush from an unproven treatment.
So a more realistic alternative is
A) No unproven treatment, and try to have a good pain relieve treatment and good emotional support for the family
B) Spend all your money in an unproven treatment and left a broken family, and
- 1%? chance of 6 more months of life
- 50%? chance of no side effects
- 49%? chance of nasty side effect of the treatment that makes your small time on Earth worse
For a real example, there is a somewhat popular but ineffective treatment for some types of breast cancer that include bone marrow transplant, and living with a transplanted bone marrow is a problem. https://www.nejm.org/doi/full/10.1056/NEJMbkrev58584
> It has been estimated that more than 30,000 women in the United States with breast cancer received bone marrow transplants between 1985 and 1998, at the cost of millions of dollars, considerable adverse effects, and treatment-related mortality rates of 3 to 15%.
This is not necessarily true, and can certainly be worse than nothing.
I also think the "word will get out" argument doesn't really wash. If every other option says you'll die, a miracle is worth a shot.
That seems wildly optimistic to me. Companies, especially pharma, will always find ways to 'make tons of money'. The second part of the sentence assumes a level of disclosure (from the companies) and rationality (on the part of average people), that I don't believe exists in the world. There are plenty of examples to back that up, especially people's willingness to believe self-serving 'facts'.
Drug manufacturers have finally raced to the bottom. The explicit purpose of charging high costs for experimental drugs is to entirely empty the bank accounts of the most desperate people without even having to tell them a potential rate of success. This is sick and preys on people in the most desperate situation one is likely to face in life.
People waste billions on quack medicine such as Traditional Chinese Medicine but now we are concerned about the finances of dying people? And yes, TCM is quackery; any form of “medicine” that prescribes black rhino horn as a cure for impotence is pure nonsense. Is statnews writing strongly worded opinions about that? Or are they just interested in picking on big pharma— that, despite its warts, actually does make drugs that have benefited society in countless ways.