The FDA is powerless to stop right-to-try profiteers

https://www.statnews.com/2018/06/20/right-to-try-opportunism/

by aaavl2821 • 8 years ago

48 82 8 years ago

dahart2• 8 years ago

“We have to have an incentive,” he said.

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.

emodendroket• 8 years ago

"Rationalize" suggests the people coming up with this stuff believe it.

emiliobumachar• 8 years ago

"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.

XorNot2• 8 years ago

The 50% on marketing number isn't BS: https://www.vox.com/2015/2/11/8018691/big-pharma-research-ad...

Everytime I've gone looking for this, it's just been confirmed over and over.

dahart2• 8 years ago

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?

http://fortune.com/fortune500/list/filtered?sortBy=profits&f...

ekianjo• 8 years ago

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.

f3f3_• 8 years ago

If anything we need more experiments like that.

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?

abowlofpetunias• 8 years ago

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.

Here, you seemed to have missed this part.

mbreese3• 8 years ago

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.

Kuiper• 8 years ago

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.

XorNot2• 8 years ago

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.

gus_massa• 8 years ago

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%.

rhizome4• 8 years ago

something that is not 100% proven but that has some potential is better than nothing

This is not necessarily true, and can certainly be worse than nothing.

snarf21• 8 years ago

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.

amirmc• 8 years ago

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'.

DennisP3• 8 years ago

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.

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...

xevb3k• 8 years ago

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).

DennisP3• 8 years ago

I don't disagree; a better middle ground might be cost plus some reasonable percentage for unapproved treatments.

WC3w6pXxgGd4• 8 years ago

Do it for free

You're aware creating products costs money, right?

bsder• 8 years ago

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.

DennisP3• 8 years ago

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.

WC3w6pXxgGd4• 8 years ago

You should be free to trust whomever you want, be that scientists or bureaucrats in the government.

WC3w6pXxgGd4• 8 years ago

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.

freedomben• 8 years ago

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.

hexane3602• 8 years ago

Unless those other people are directly entangled with your finances and responsible for ensuring the wellbeing of you and others.

WC3w6pXxgGd4• 8 years ago

Agreed 100%.

mbreese3• 8 years ago

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.

peacetreefrog2• 8 years ago

Disagree. People evaluate almost literally every other single product they buy.

anm89• 8 years ago

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.

Thriptic2• 8 years ago

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.

kazinator3• 8 years ago

People shouldn't be charged to serve as guinea pigs. On the contrary, they should be paid.

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.

briandear2• 8 years ago

But that should be their choice to make.

Thriptic2• 8 years ago

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?

Alex3917• 8 years ago

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.

mylons• 8 years ago

I like the idealized version of putting whatever I want in my body before I die in an attempt to extend my life.

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.

RobertRoberts4• 8 years ago

There seems to be an overlapping conflict area between the freedom to be ripped off and the freedom to control my own medical care.

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.

Nursie3• 8 years ago

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).

mannykannot• 8 years ago

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.

https://arstechnica.com/science/2018/06/new-drug-access-law-...

jonhendry183• 8 years ago

" 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.

peacetreefrog2• 8 years ago

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.

mrguyorama• 8 years ago

A dying and constantly miserable human is not a rational actor

openasocket3• 8 years ago

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.

raverbashing• 8 years ago

Let me rewrite the headline: "Redtape fetishists outraged that people now have a choice"

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.

hexane3602• 8 years ago

The article is about "right-to-try profiteers", not just "right-to-try" period.

We can totally discuss that.

That's what this article is trying to discuss.

openasocket3• 8 years ago

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.

briandear2• 8 years ago

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.

jedberg• 8 years ago

A lot of folks commenting in here should look up Thalidomide.

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...

tsomctl• 8 years ago

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.

maym86• 8 years ago

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.

https://en.m.wikipedia.org/wiki/Bad_Pharma

frgtpsswrdlame4• 8 years ago

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.

ada19813• 8 years ago

I’m curious how this law might be leveraged for PTSD and other mental health folks access to MDMA and other psychedelics.

Would this allow for access to scheduled substances?

Could this law be useful in that regard?

narag2• 8 years ago

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.

Most disgusting news I've read this year.

ada19813• 8 years ago

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.

RickJWagner• 8 years ago

An expensive chance is probably better than no chance at all.

The FDA is powerless to stop right-to-try profiteers

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