13 ms·
The FDA is powerless to stop right-to-try profiteers
- dahart 8y 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 8y ago"Rationalize" suggests the people coming up with this stuff believe it.
- emiliobumachar 8y 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.
- XorNot 8y agoThe 50% on marketing number isn't BS: https://www.vox.com/2015/2/11/8018691/big-pharma-research-advertising 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.
- hackerman12345 8y agoIt'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.
- rhizome 8y agoInsurance 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. Ergo hookers and blow? I don't buy it.
- dahart 8y agoNot 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&first500 http://fortune.com/fortune500/list/filtered?sortBy=profits&f...
- ekianjo 8y 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_ 8y 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 8y 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.
- mbreese 8y agoIt 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 8y agoThe 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.
- DennisP 8y agoLast 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-Winnable/dp/0374536481 https://www.amazon.com/Death-Cancer-Pioneering-Oncologist-Wi...
- xevb3k 8y 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).
- DennisP 8y agoI don't disagree; a better middle ground might be cost plus some reasonable percentage for unapproved treatments.
- filoeleven 8y agoIsn’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?
- danielnesbitt 8y agoThey 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.
- WC3w6pXxgGd 8y agoAnd 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 8y agoI 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.
- hexane360 8y agoUnless those other people are directly entangled with your finances and responsible for ensuring the wellbeing of you and others.
- WC3w6pXxgGd 8y agoAgreed 100%.
- mbreese 8y agoExperiments 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.
- peacetreefrog 8y agoDisagree. People evaluate almost literally every other single product they buy.
- jadell 8y agoAlmost 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?
- anm89 8y agoThose 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.
- Thriptic 8y agoI 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.
- kazinator 8y agoPeople 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.
- briandear 8y agoBut that should be their choice to make.
- kazinator 8y agoIs 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?
- peacetreefrog 8y agoIt'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 8y agoA dying and constantly miserable human is not a rational actor
- openasocket 8y agoEspecially 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 8y agoLet 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.
- hexane360 8y agoThe 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.
- openasocket 8y agoI 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.
- briandear 8y agoCould 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 8y agoA 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_crisis https://en.wikipedia.org/wiki/Thalidomide#Birth_defects_cris...
- tsomctl 8y agoAccording 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 8y agoRead 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 https://en.m.wikipedia.org/wiki/Bad_Pharma
- frgtpsswrdlame 8y 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.
- ada1981 8y agoI’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?
- narag 8y agoOnly 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.
- ada1981 8y agoMDMA 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.
- narag 8y agoThat'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.
- ada1981 8y agoMDMA 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. The war on drugs is a relatively new obsession.
- RickJWagner 8y agoAn expensive chance is probably better than no chance at all.