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.
It's not just marketing in that graph, it's sales & marketing. Insurance companies don't make getting drugs covered and to patients easy. Newly created drugs are pretty useless if neither doctor nor patient has heard of them.
Insurance companies don't make getting drugs covered and to patients easy. Newly created drugs are pretty useless if neither doctor nor patient has heard of them.
Not stellar? Across multiple searches I see companies like Johnson & Johnson, Gilead Sciences, Novartis, Procter & Gamble, and Pfizer on the top 10/25/50 lists, with profit margins in the double digit percentages. What is stellar in your book if not billions and billions in profit?
> The idea that BrainStorm could make a profit from NurOwn before the treatment is proven effective and approved by the FDA is a bad look for the company and the entire pharmaceutical industry. It reeks of opportunism, even when couched in compassionate rhetoric.
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?
>There’s little evidence that ALS patients, especially those with advanced disease, will benefit from NurOwn. In BrainStorm’s Phase 2 study, NurOwn was unable to slow the progression of ALS compared with a placebo. The company turned to a responder analysis to eke out a signal of efficacy, which it’s now trying to confirm in the Phase 3 study.
It used to be the FDAs job to monitor and report if unapproved therapies (which could be used with a waiver) worked or not. Now, there’s no one to provide that role, so the best you’ll get is anecdotal evidence. Removing the FDA from this process doesn’t help.
The author of the article also seems to imply that FDA approval is the only process by which a treatment could be "proven effective." There are many treatments that are already available in Europe but spend years waiting for FDA approval.
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.
The FDA allows "compassionate use" exceptions for this type of thing and generally grants them. The point of the approval process (and the entire FDA) is to prevent charlatans peddling dangerous and ineffective therapies and drugs to desperate people for profit.
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.
> being able to try something that is not 100% proven but that has some potential is better than nothing
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%.
Okay, so instead of charing someone desperate $300K for this unapproved treatment, why not just relax the rules for clinical trials for end of life treatments? This still matches the intent of compassionate care, still collects structured data that can be explored and gives people the right to try anything.
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.
> 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.
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'.
Last year I read a book by one of the top oncologists in the U.S., who's been in the field for five decades and held senior positions at leading institutions. He did not have good things to say about the FDA.
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.
> BrainStorm CEO Chaim Lebovits told Bloomberg he’s considering charging $300,000 for each NurOwn treatment
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).
Isn’t that akin to making people pay to participate in a study? Shouldn’t their payment consist of all the information a company receives from essentially testing their new treatment on a human?
They are not participating in the study though, at least not the regulated portion of the trial.
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.
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 are aware that you want people to pay for the 'right' to be guinea pigs?
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"?
> 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.
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.
One of the author's points was that we should trust the scientists more than we trust the bureaucrats at the FDA. It used to be the scientists making these treatment decisions, now it's largely not.
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.
And this is bad? The healthcare industry desperately needs experimentation and cheaper options. If a product works, people will continue to buy it. Welcome to how markets work.
I agree, but there's an even more compelling argument in my opinion. Government (which is basically an abstraction for "another person that isn't me") has no right to tell me what I can and can't put into my own body.
Experiments aren’t cheap. Patients aren’t in a good position to evaluate if a product is working or not. This is especially true when they are emotionally or personally involved in the outcome - everyone believes they are an outlier.
Almost every other product they buy is not a matter of life or death where they are personally emotionally invested in the outcome.
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?
Those gosh darn profiteers providing optional life saving therapies for patients. Drat them!
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.
I totally believe right to try should be a thing. Who is the FDA or anyone else to tell dying patients that they can't put something in their body to try to save their life? The reason this is necessary is because under the previous compassionate use policy, pharma conpanies are required to report any adverse events from these patients using these products to FDA which could result in a halt of their independent clinical trials running in parallel. Because the patients utilizing compassionate use many be in any stage of disease with multiple comorbidities, it makes root cause analysis of adverse events much more difficult than in a clinical trial, and presents a much higher risk to developers.
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.
Is that so? In that case, why should only the special class of the terminally ill have the privilege of this choice? Everyone should have it. Just do away with drug trials and approvals in favor of a situation whereby anyone can advertise any substance whatsoever as being a cure for any ailment. The free market will determine what drugs work, no problem!
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 players who are initially in that game might not be lying; they may offer full disclosure about the treatments so everyone is informed about what their chances are and the risks and so on. But a "can of worms" is open: all sorts of scum will notice the money-making opportunity and want a piece of the action.
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.
What are you talking about? Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients who can't pay. That requires FDA approval. Historically iirc compassionate use distributions were free, and it is likely that will continue to be the case because the amount of money companies can make off of individual patients is rounding error relative to bringing a product to market and getting widespread approval for reimbursement.
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?
>Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients.
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.
> Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients who can't pay.
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.
> 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
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.
"Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is"
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.
> They are allowed access to promising therapies in late stage clinical trials in the hopes that they will help them
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.
As someone who successfully tested and completed stem cell therapy to prevent an amputation, I don't care who gets paid. Fuck you to saying I can't try something when it's my bone getting cut off.
> Who is the FDA or anyone else to tell dying patients that they can't put something in their body to try to save their life?
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.
The converse to this, though, is that there are people who do get duped, and they get ripped off for hundreds of thousands of dollars.
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 a partial cure for this, as well as the law pursuing thieves. But there will always be scams no matter how many laws you pass or protections put on people.
Yes there will always be scams. That doesn't mean we should leave the field wide open and encourage scams, or create a legal landscape in which it is difficult to detect and close down such things.
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.
>I'd rather have more control over my life than less,
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.
I've been tricked before, and I've been cornered by the government into an action I didn't want. Which one do you think I am able to avoid in the future? The government has real "power" over you, a con-artist only get's you once.
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.
>Which one do you think I am able to avoid in the future?
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 think it's a pendalum swinging issue, the medical industry has economical (insurance/govt healthcare) and political strangle hold on our society. The constitution almost had a clause protecting us from the medical industry.
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'm not sure I have as much faith in humanity as you do. We already have examples of long-running medical scams that take the dying for all they're worth, take the kind-hearted who donate to their causes for all they're worth, and often leave families in debt, all for a treatment that is little more than a tissue of lies.
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).
The FDA already had a mechanism for this purpose, as did 38 states. After the passing of this bill, Senator Ron Johnson, one of the bill's sponsors, took upon himself the privilege of explaining how it should be implemented to FDA Commissioner Scott Gottlieb (a Trump appointee), apparently because Gottlieb expressed excessive concern for patients' well-being. This raises the possibility that the bill's authors were coy about their intentions, perhaps to avoid them being scrutinized prior to its passage, and I hope the FDA will interpret it as written, and consistently with its broader responsibilities.
" no way that an insurance company would reimburse for any untested therapy which by default would keep costs down"
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.
It's "right-to-try" not "have-to-try". Articles like this make it sound like patients are powerless and have to take and pay for whatever treatments these companies offer.
Especially when the drug seller and the patient have asymmetrical information. As far as I can tell, there are no requirements for the drug maker to report the effectiveness of the drug on those that they sell it to.
I think we should also discuss that there doesn't seem to be any requirements on the people selling these unproven drugs. Like monitoring the efficacy and making those results public. Basically just an expanded phase II trial.
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.
Could the lede be any more biased or willfully ignorant as to how long FDA processes take? A terminally ill patient doesn’t have much need for money if they are dead, so what business is it of anyone’s if they choose to spend all of their money of a last ditch hope.
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.
According to your link, Thalidomide was introduced in 1957, and banned in Canada and most European countries by the early 60's. I really, really hope that the FDA and EMA have improved their methodology in the past 50 years.
Read bad pharma by Been Goldacre. It's a detailed outline of many issues with the industry. The extent that pharmaceutical companies go to make profit, mislead doctors and harm patients is crazy. This law will definitely be abused for profit.
>BrainStorm CEO Chaim Lebovits told Bloomberg he’s considering charging $300,000 for each NurOwn treatment offered through a new pathway set up as a result of the right-to-try law.
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.
Only if they can charge $300,000 for the privilege. Anyway it seems that the drug must have passed a first phase of testing. Are illegal drugs allowed to be tested to begin with? I don't think so.
MDMA is in Phase III clinical trials for PTSD and showing very promising results. It was given a special designation by the FDA as a breakthrough treatment to speed up the process.
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.
That's interesting, but wasn't MDMA a prescription drug before it became popular as a recreational drug? I mean, not the only drug that's legal if prescribed by a doctor and illegal otherwise, like morphine etc.
MDMA was not a prescription, it just wasn’t a scheduled compound. It was originally used by therapists however before it spread into the mainstream and then it was scheduled despite the recommendation that it be scheduled to allow for 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.
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[ 4.3 ms ] story [ 173 ms ] threadThere 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...
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'.
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.
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?
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.
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.
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.
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.
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...
https://en.m.wikipedia.org/wiki/Bad_Pharma
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.
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.