FDA Worried Drug Was Risky; Now Reports of Deaths Spark Concern (cnn.com) 183
Blake Ellis and Melanie Hicken, writing for CNN: Two years ago, Brendan Tyne pleaded with the Food and Drug Administration to approve a drug that he was hopeful could finally bring his mother some peace. She could no longer move without assistance and had fallen victim to the debilitating and frightening psychosis that haunts many people with Parkinson's disease. "She thinks there are people in the house and animals are trying to get her," he told an FDA advisory committee. He believed that a new medication called Nuplazid, made by San Diego-based Acadia Pharmaceuticals, was the answer.
Nuplazid's review was being expedited because it had been designated a "breakthrough therapy" -- meaning that it demonstrated "substantial improvement" in patients with serious or life-threatening diseases compared to treatments already on the market. Congress created this designation in 2012 in an effort to speed up the FDA's approval process, which has long been criticized for being too slow. Around 200 drugs have been granted this designation since its creation. [...] The committee voted 12-2 and recommended that the FDA approve Nuplazid for the treatment of Parkinson's disease psychosis based on a six-week study of about 200 patients. It hit the market in June 2016. As caregivers and family members rushed to get their loved ones on it, sales climbed to roughly $125 million in 2017
[...] In November, an analysis released by a nonprofit health care organization, the Institute for Safe Medication Practices, warned that 244 deaths had been reported to the FDA between the drug's launch and March 2017. [...] Since the institute released its analysis, FDA data shows that the number of reported deaths has risen to more than 700. As of last June, Nuplazid was the only medication listed as "suspect" in at least 500 of the death reports.
Nuplazid's review was being expedited because it had been designated a "breakthrough therapy" -- meaning that it demonstrated "substantial improvement" in patients with serious or life-threatening diseases compared to treatments already on the market. Congress created this designation in 2012 in an effort to speed up the FDA's approval process, which has long been criticized for being too slow. Around 200 drugs have been granted this designation since its creation. [...] The committee voted 12-2 and recommended that the FDA approve Nuplazid for the treatment of Parkinson's disease psychosis based on a six-week study of about 200 patients. It hit the market in June 2016. As caregivers and family members rushed to get their loved ones on it, sales climbed to roughly $125 million in 2017
[...] In November, an analysis released by a nonprofit health care organization, the Institute for Safe Medication Practices, warned that 244 deaths had been reported to the FDA between the drug's launch and March 2017. [...] Since the institute released its analysis, FDA data shows that the number of reported deaths has risen to more than 700. As of last June, Nuplazid was the only medication listed as "suspect" in at least 500 of the death reports.
Ah yes.. The reason the FDA does reviews (Score:5, Insightful)
It's a catch 22. The FDA usual process is slow and plodding but results in medications and medical procedures which are generally safe and effective by reducing as much risk as possible. However it takes a LONG time to perform all the necessary studies and clinical trials and critically ill patients die while they wait.
The catch is that if you are trying to get approval for a novel medication that saves lives of the critically ill, how do you justify the delay needed to do all the safety and effectiveness studies? People will die if you don't try, but you might also kill and/or cure. What to do?
Re:Ah yes.. The reason the FDA does reviews (Score:5, Insightful)
People keep telling me that we need to test different drugs differently because it's expensive and takes forever, and some drugs are obviously less-dangerous. I usually point out that tilting a single methyl group in a molecule to one side or the other determines if it's a harmless nasal decongestant or high-potency methamphetamine.
For these complex cases, you can counsel the patient into a high-risk experimental drug program. High-risk programs would be highly-irregular and so would put a lot of warning in front of the patient. Your first-pass should tell you if there are really bad risks (this one had a concern about twice as much death happening in the experimental group as in the placebo group).
It's kind of crap, but it's better than putting the drug into the "normal drug with scary warning labels like every other drug for this condition" class by flatly approving it for normal dispensation in normal pharmacies. The real problem is that psychotic patients aren't capable of really considering all of the risks (as if anyone really is), so you're still facing an ethics crisis.
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More comprehensive analysis of results, more comprehensive analysis of the pathways, more comprehensive analysis of what is going on in live patients and more comprehensive analysis of any deaths should improve things, but you can't do everything to the hilt. You need to balance any increase in these so as to get the best payoff (best outcomes for survivors, fewest deaths) for the same budget.
Unfortunately, experts are considered heretics at the moment, and science is held as blasphemy by some. Added to tha
Re:Ah yes.. The reason the FDA does reviews (Score:5, Insightful)
It's not even legal in America to take action that could hurt shareholders, ....
Please don't say this. It is not correct. Not even close. I have read the court decision that this fable is based on, and that is not what it says.
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But you are arguing in favor of a longer and more detailed study. And while you do that study, people who might benefit from the drug, if it works, are suffering and dying.
OTOH, it might not work. Whoops!
There's no obvious way forwards when you're working in ignorance. I favor letting people into an experimental program with blatant warnings, and rules preventing any sponsor from getting more than cost recovery (and, of course, data). This would *NOT* be a double blind study, so it's less than perfectly
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OTOH, it might not work. Whoops!
The shelves are filled with things that might not work. We expect the FDA to actually stop things that kill people. 700 deaths attributed to the drug is a bit of an extreme side effect, wouldn't you say?
I favor letting people into an experimental program with blatant warnings,
Of course. This is not the same as FDA approval for use in treating a specific illness. A six-week, 200 participant study might be justification for that experimental program, but it shouldn't be enough for release of a drug for general use.
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OTOH, it might not work. Whoops!
The shelves are filled with things that might not work. We expect the FDA to actually stop things that kill people. 700 deaths attributed to the drug is a bit of an extreme side effect, wouldn't you say?
It depends on how many people received the drug and recovered. 700 deaths out of 7000 patients is bad. 700 deaths out of 70k patients is less bad, especially if the remaining 69300 patients experienced relief from dementia.
Honestly, if my options are dementia or a pill that might kill me with a 50:50 chance of relief from the dementia, I'm going to take the pill.
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It depends on how many people received the drug and recovered.
You might pick 700 people losing their loved ones so yours would be better. Would you be happy to be in the 700 so someone elses loved one would be better? That's a rhetorical question, since it all depends on how you value life.
Honestly, if my options are dementia or a pill that might kill me with a 50:50 chance of relief from the dementia, I'm going to take the pill.
Those weren't the options you were given. You were told your options were 50:50 "relief from dementia" and "no change". Sure, anyone would pick that. Would you be so quick to pick "relief or die"? Wouldn't it be nice to know the true options? Would you be informed enough to ask "ho
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I've no problems with people trying things out, as long as there is a good flow of information back from patients to doctors and researchers when things either succeed or fail, where this includes genetic information and (in the case of failures) biopsies that can be tested using AMS to find out what was going on, and for patients where it's too early to really tell there's monitoring.
You won't be able to catch everything in time, and as I've noted elsewhere, there's a balance between cost and effectiveness
Re:Ah yes.. The reason the FDA does reviews (Score:5, Funny)
How hard is it to tilt this methyl group?
(Asking for a friend.)
I mean, think of all the dangerous meth that could be converted into a harmless nasal decongestant. And btw can you tilt it either way?
Re:Ah yes.. The reason the FDA does reviews (Score:5, Interesting)
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I remember seeing an article somewhere about converting meth back into a decongestant.
That article was satire, written by an accomplished chemical engineer who knew how to write it so it looked completely technical.
For better or worse, converting meth into pseudoephedrine is not realistically possible.
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>For better or worse, converting meth into pseudoephedrine is not realistically possible.
But is really is easier to get meth than pseudoephedrine in the United States.
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In Oregon you need a doctor's prescription. Of course that costs a lot more than a bit of meth.
So Oregonians stock up when they are in another state.
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I can get meth 24x7 delivered to my house, I can't get (pseudo)ephedrine without waiting in line during hours that are convenient to the pharmacist after driving to their place and then I have to show ID and get all nasty looks if more than one member of my family has had a cold this month.
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But if that really is the boat you are in, a light dose of meth will clear your sinuses and really doesn't carry any (physical) risks that don't also go on the label of the sudaphed. Either one will keep you up all night if you aren't careful in how you time it. There is a nighttime formula for sudafed but it falls right back in th
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There are 24 hour Walgreens and CVS about 30 minutes from my house, but the pharmacy isn't open 24 hours, only the convenience store part. If pseudo-ephedrine was still available OTC I could buy it whenever I wanted (in fact there's a 24 hour grocery store about 10 minutes from the house where I could pick it up). I bet in Chicago there are true 24 hour CVS locations but in this tiny metro area of 2 million people there are none outside of maybe a hospital and they're not going to be selling me my decongest
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There are no shortage of things like this that have fallen victim to the war on drugs. Cocaine in very light doses (natural coca leaf tea vs concentrated powder extract) is much more like mild coffee with far
Re: Ah yes.. The reason the FDA does reviews (Score:2)
Uhh.. except cocain is much more habituating and people tend to keep amping up the dose to dangerous levels, while high dose caffeine isn't much fun.
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Yeah, but if you do this you might as well not approve it all. Insurance companies won't pay for what will always in a case like this be a brand new and extremely expensive medication.
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Since when do insurance companies pay for new drugs?
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The way I see it. If a drug is really that dangerous/experimental than they folks that are desperate enough to use it are essentially doing a trial for the drug company and should get it at low or no cost
If its a new drug for a chronic condition where alternatives exist (say a new high blood pressure med for instance), than until that med completes exhaustive trials (and perhaps until it shows benefit over the dozens of cheap alternatives already on the market), insurance companies s
Game theory (Score:2)
It's a catch 22. The FDA usual process is slow and plodding but results in medications and medical procedures which are generally safe and effective by reducing as much risk as possible. However it takes a LONG time to perform all the necessary studies and clinical trials and critically ill patients die while they wait.
The catch is that if you are trying to get approval for a novel medication that saves lives of the critically ill, how do you justify the delay needed to do all the safety and effectiveness studies? People will die if you don't try, but you might also kill and/or cure. What to do?
What you should do is put all the responsibility for making a mistake on the bureaucrats responsible for safety protocols, and all the costs associated with those safety protocols should be borne by the drug manufacturers.
In a game-theory sense, that gives you the safest drugs possible within the system.
Then you mandate that no one can go outside this system - no one can decide for themselves whether to take a risk on a non-tested procedure or drug, even if their disease is known to be terminal or completel
How to make drugs risk free! (Score:5, Insightful)
It's a catch 22. The FDA usual process is slow and plodding but results in medications and medical procedures which are generally safe and effective by reducing as much risk as possible. However it takes a LONG time to perform all the necessary studies and clinical trials and critically ill patients die while they wait. The catch is that if you are trying to get approval for a novel medication that saves lives of the critically ill, how do you justify the delay needed to do all the safety and effectiveness studies? People will die if you don't try, but you might also kill and/or cure. What to do?
What you should do is put all the responsibility for making a mistake on the bureaucrats responsible for safety protocols, and all the costs associated with those safety protocols should be borne by the drug manufacturers.
You do realize that higher the penalty you put on making a mistake, the result is that the people responsible for safety protocols will become more risk adverse, and no drug will ever get approved, right?
That's the only way to get 100% certainty of never making a mistake in approving a drug: making sure you never approve a drug.
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Following your argument, no-one would ever get in a car and drive it down the street.
Pharma companies make their money from introducing new drugs. They are not going to stop that just because the risks are higher. Instead, they will find ways to manage the risk.
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Following your argument, no-one would ever get in a car and drive it down the street.
If you want to be risk free, that's correct.
Demanding that you must be risk free in all activities means never doing any activities.
Pharma companies make their money from introducing new drugs. They are not going to stop that just because the risks are higher. Instead, they will find ways to manage the risk.
The post I was responding to was talking about approval of drugs for use. That's not up to the pharmaceutical companies.
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There are definitely things companies stop because the risks are higher, some risks must be accepted and priced into what you do, some you "manage" by finding less risky answers or insurance (such as testing). If the FDA and legal immunity it grants drug companies went away tomorrow they'd likely INCREASE testing and safety protoco
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What people forget is that the FDA testing isn't to make sure drugs are safe, it is make sure drugmakers are safe from lawsuits. Take away that level of regulation and you also remove the protection. D
Nobody is perfect (Score:2)
What you should do is put all the responsibility for making a mistake on the bureaucrats responsible for safety protocols, and all the costs associated with those safety protocols should be borne by the drug manufacturers.
If you make individuals responsible for mistakes then nobody would be willing to do that job. That's why corporations exist and why governments are usually shielded from liability except in extreme circumstances. Would you take full liability for the actions of your employer even for actions you do not control? Everybody makes mistakes sometimes. Don't make perfect the enemy of good. The system cannot work if individuals have unlimited personal liability.
As for the costs, most of the costs of the studi
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Actually, dangerous stuff will take care of itself. Without FDA evaulation the drug companies lose automatic legal immunity for anything they disclosed to the FDA and those 700 dead people would come with heavy lawsuits for drug makers which make skimping on testing way more expensive tha
Whoosh... (Score:2)
DAFUQ?!?!?!
You just spent an entire post advocating a literally stifling bureaucracy in charge of a huge portion of health care, and now you say that won't work?
Whoosh...
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It's difficult to answer this one, but the best I can come up with is that they need to understand what it is the drugs actually do better. What they are supposed to do is easy, what they do in the target area - hmmm, I might possibly believe them. But that's not where the metabolic chain begins or ends and that's where the problem lies.
Isotopes that can be used as tracers might help, particularly in animal studies. If you can use MRI, positron emission detection, etc, to see where things end up, then you c
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It's a catch 22. The FDA usual process is slow and plodding but results in medications and medical procedures which are generally safe and effective by reducing as much risk as possible. However it takes a LONG time to perform all the necessary studies and clinical trials and critically ill patients die while they wait.
The catch is that if you are trying to get approval for a novel medication that saves lives of the critically ill, how do you justify the delay needed to do all the safety and effectiveness studies? People will die if you don't try, but you might also kill and/or cure. What to do?
What's worse is, if you read the article, the company that makes this drug wants to expand its use to other forms of dementia, not just Parkinson's related. For a drug like this, which was approved after only a few small trials with very questionable results (even the author of the latest trial has reservations), it should be limited only to patients who have tried all other available medications. But of course, the company expects $250 million in profits this year, and if a couple people have to die fast
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It is not a catch 22 but the problem is the populations severe lack of understanding of science and medicine.
We have some people who "Put their faith behind science" and others who distrust it as just silly guessing by people who think they are more important then you are.
The problem is the Body is complicated. There are things that will not kill us right away, but will kill us more slowly. The things that may kill us slowly, may stop the thing that is killing us right away. So this would get approved. Whi
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I think the issue here is the improper use of antidotal evidence and it's prevalence in society today. We are barraged with "It worked for me" advertisements and claims all the time and most people I've run into don't understand how to weigh such evidence. How do you think vaccines get such a bad rap in some circles and how people can persist in the "DTaP causes SIDS" misinformation?
Actual training in the scientific method is sorely lacking these days....
The needs of many outweigh the few (Score:3)
The catch is that if you are trying to get approval for a novel medication that saves lives of the critically ill, how do you justify the delay needed to do all the safety and effectiveness studies?
We need to understand the effects of a range of dosages and what sort of patients are helped and which are not. Yes some patients will be harmed while we take the time to study safety and effectiveness but MORE patients will be harmed if we don't take the time to study safety and effectiveness. Merely knowing that a drug can help some patients isn't sufficient. We need to know what the range of safe and effective dosages are, what patient populations are helped, what the range of expected outcomes are, a
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Approval doesn't have to be all or nothing.
You can have a shorter initial approval, that, once complete, allows the drug to be used by "they'll die anyway if we don't" patients.
Additional scrutiny (and data from the above class of patients) can then be used to solidify the initial verdict.
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Um... Yea, the FDA does that.
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However it takes a LONG time to perform all the necessary studies and clinical trials and critically ill patients die while they wait.
Before the thalidomide incident, the FDA only required safety testing, and thus Americans were protected from thalidomide.
Meanwhile in Europe their regulations only required efficacy testing, and thus Europeans were hammered hard by thalidomide.
After the thalidomide incident, the FDA demanded adding efficacy testing, and thus Americans have since seen longer delays and more expensive drugs hurting every American.
Solution: We dont need or want the federal government demanding efficacy. Thats for your
Re: Ah yes.. The reason the FDA does reviews (Score:5, Insightful)
Solution: We dont need or want the federal government demanding efficacy. Thats for your fucking doctor to decide, not some professional bureaucrat asshole
Yeah you do. There are multiple problems with these two sentences:
1. You're OK with "some professional bureaucrat asshole" deciding whether something is safe, but not whether it's effective? Why? Do you, by any chance sell homeopathy? "Buy my shit; it doesn't to dick, but it's completely safe!"
2. "Your doctor" is no more able to asses the efficacy of an untested treatment than is your garbage man. In order to know whether or not a medication is effective we need large scale studies, not clinical anecdotes. Your doctor isn't doing any large scale studies; the "professional science assholes" at the FDA are.
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2. "Your doctor" is no more able to asses the efficacy of an untested treatment than is your garbage man. In order to know whether or not a medication is effective we need large scale studies, not clinical anecdotes. Your doctor isn't doing any large scale studies; the "professional science assholes" at the FDA are.
Just to clarify a bit, the FDA doesn't conduct studies, they review and evaluate study data. Studies are usually conducted at a health care facility of some kind, so it is possible, though highly unlikely, that "your doctor" is involved in a few drug studies. Your primary physician almost certainly isn't part of any studies, but specialists at larger hospitals often have patients participating. Overall management of a study is usually, but not always, done jointly between the pharmaceutical company sponsori
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Doesn't the FDA request and fund studies? If not, how do these studies on the new drugs get started? Please don't say the drug companies fund all of them!!
The FDA doesn't fund studies on new drugs. However it DOES determine what studies they will require for licensing a new drug, then after the manufacturer has completed those studies, the FDA reviews them before it issues a license for a drug to be used in specific ways.
HOWEVER, the FDA does order and pay for studies for existing licensed drugs when possible and significant adverse reactions for said drug are found.
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Doesn't the FDA request and fund studies? If not, how do these studies on the new drugs get started? Please don't say the drug companies fund all of them!!
For the most part, yes, the drug companies fund them. It's part of the cost of research for developing new drugs. The FDA then reviews all of the data from the studies and decides whether or not to approve the sale of the new drug.
Yes, a company could try submitting falsified data to the FDA in order to get approval, but the penalties for doing so are quite harsh if they get caught.
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Oh come on, the FDA does more than that. Be honest about this.
They DO finance their own studies, funded by the fees the pharmaceutical companies pay for licensing. They also are charged with actually reviewing all the studies the makers do seeking approval and over seeing that the studies are properly done. The FDA does a lot of it's own drug monitoring and reporting of possible adverse events.
That's just the "drug" part of their mandate and activity. There is the "food" part too..
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The catch is that if you are trying to get approval for a novel medication that saves lives of the critically ill, how do you justify the delay needed to do all the safety and effectiveness studies?
I think the information in just the summary is sufficient to do that. A six-week study with 200 people is now enough data to start selling a drug? (I'm sure some smart statistician can tell us the percentages for potential undetected serious complications from a long-term drug in a study with 100 test and 100 control subjects over just six weeks.) A connection with 700 people dead from taking the drug should be enough.
We have a history of other drugs that have been through full testing that are now pulled
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I know the math, I'm willing to accept the risk, and the risk is reasonab
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How about people have a brain and it's their life?
What are they supposed to use their brain to do if they aren't told that the chances of dying as a side effect are relatively high? You can't make an informed decision without data.
I used to take a medication that was later found to kill ~1 in 100,000. Given the benefit and risk of that drug, I would have elected to continue treatment with that drug.
That's nice. Wouldn't it have been better if you knew before you made the choice what the chances were? Should others be restricted in knowing the danger because you are a risk taker?
Inform me and allow me to make the choice.
What value is the information that this certain drug didn't kill anyone in a group of 100 test subjects after taking it for just six weeks? How do
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Yes, I DID think that through. It is STILL true.
I never ONCE suggested any of the things you attributed to me; you pulled ALL of that straight out of your own mental fog.
mnem
I know, it's so much easier to talk out your arse when you post as AC; Ifni forbid you should take the slightest amount of responsibility for what you say.
Re: Ah yes.. The reason the FDA does reviews (Score:2)
Giving more power to big pharma doesn't sound like a better alternative. At least politicians can be removed. Large abusive corps don't go away as easily.
Re: Ah yes.. The reason the FDA does reviews (Score:2)
Oh, FFS... this isn't conspiracy theories, you bonehead... it's just following the money.
aka conspiracy theories.
"Urmaghurd, China made a bundle off the steel from the WTC! China did 9/11!!! Follow the money!!1!1!!!1!!"
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HAHA Or maybe he'll try to claim some power company would skimp out on lining wells, knowingly contaminating the groundwater with deadly hexavalent chromium and then engage in a conspiracy to "disclose" it to the
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This evil and corrupt agency, the puppet of Big Pharma, makes it illegal for terminally ill people to try potentially life-saving treatments (for their own "safety," of course). It also tells us that lots of over the counter drugs are good but too many eggs will kill you.
The FDA is evil and stupid, and it should be abolished.
To what end?
IF you recall the time BEFORE the FDA, you will remember the so called "snake oil salesmen" who sold their bottles of often harmful and addictive "elixirs" which cured no one beyond the placebo effect.
I for one do not wish to return to the wild wild west medical practices.
Worth the risk (Score:5, Insightful)
Like surgery and other non-drug therapies, sometimes the risk of death - even if very high - is a good gamble vs. the near-certainty of a poor quality of life.
The key is knowing the risks and taking marketing out of the equation so patients, doctors, and caretakers/family members can make a truly informed, sober choice.
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A broken clock is right twice a day, but that doesn't mean you should rely on it to decide when to do things.
Three out of four studios for the drug didn't show any benefit, but four out of four showed increased risk of complications including death. It sounds like they kept testing until they found a statistical anomaly that gave them the answer they were looking for. I seriously doubt hey told patients about the studies that dind't show improvement, so you can forget informed choice.
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Wrong. A stopped clock is right twice a day.
Re:Worth the risk (Score:5, Insightful)
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IIUC Parkinson's is normally terminal...if you don't die of something else fairly quickly. My wife's father died of it, though it did take him several years...but during the last one he was rather incoherent and disoriented. During the early years it was extremely frustrating for him, as one activity after another became impossible.
OTOH, they decided that his case of Parkinsonism was caused by chemical poisoning from his years of developing his own photos. Perhaps other causes would evolve differently.
What's worse? (Score:4, Insightful)
Paranoid delusions which keep an elderly person miserable, disabled, and disconnected disabled from everyone (even families)?
Or a small chance a Parkinsons sufferer might die or have a radical improvement?
You can't have it both, sign a waiver before treatment and move on. Save and enjoy the few who are able to make it. Mourn and cherish the memories of those who didn't.
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This system is ripe for abuse because you are targeting people who are both absolutely desperate and lack the training to make a fully informed decision. You can use plain English like "Might kill you and cause horrible suffering for weeks" but they will latch onto that hope, no matter how false, and try it anyway. It isn't a rational decision, it is an emotional one. There is a reason why we have a faceless government entity evaluate the merits of a drug as opposed to a free market customer driven demand a
Who's being targetted? (Score:2)
The patients aren't the prescribers. Or are you saying that Doctors merely surrender to the expectations of their patients. Admitted the crazy American system of allowing Big Pharma to advertise their drugs directly to the public can make a physicians job harder in refusing what isn't good for them, but in the end the professional responsibility remains.
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Yes, you can have both. Drugs aren't random and the outcome is not the will of God. All the drug companies need is more data and better data, and all doctors need is better access to the tools needed to identify which drugs are likely to have harmful side-effects. We won't reach truly personalized medicine, but the better we can approximate it, the fewer needless deaths we'll get.
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Why not take money out of the university system?
Give universities a fixed fund for the year, then let them divide that between departments, then let departments divide that between researchers, then let researchers divide that between all the projects they want to do that year.
The way it traditionally was and still is in some countries.
This has the advantage that funding is then not a popularity contest, the market can't block research that is inimical to any given market, and politics is largely eliminated
Worry is for Children (Score:2, Insightful)
Adults understand that life is about risk management. Even if all 700 deaths are attributable to the drug (highly unlikely) many people will take that risk to avoid living with Parkinson's/psychosis.
One can be too cautious or not cautious enough. The FDA is far too cautious, to the extent that current estimates are that the FDA regulations have resulted in as many avoidable deaths as two Nazi Holocausts: http://www.ruwart.com/FDA/prot... [ruwart.com]
Not every disease can be neatly solved and not every precaution is
Re:Worry is for Children (Score:5, Funny)
Adults understand that life is about risk management.
If only that were true.
Deliberate studies are a Good Thing (Score:5, Insightful)
One can be too cautious or not cautious enough. The FDA is far too cautious, to the extent that current estimates are that the FDA regulations have resulted in as many avoidable deaths as two Nazi Holocausts:
What a bunch of bullshit. The FDA has saved orders of magnitude more people than have died and I think your equating their actions to the holocaust is entirely inappropriate and false. Here is the cold hard fact. Clinical trials only work because some people are sacrificed to save more people in the long run. Yes people will die who theoretically might have been saved but doing so actually costs MORE lives in the long run. There is absolutely no way around this. This process usually takes lots of time and money to do correctly and it is inevitable that some people will suffer and die during the process. While tragic it would be a far greater tragedy to short cut the process to save a few at the expense of many.
Not every disease can be neatly solved and not every precaution is warranted.
Quackery is a real thing. Drug companies can/will/have sold snake oil in a heartbeat if it means more profit. While there are ways to improve speed to market for drugs we cut corners at our peril.
Re:Deliberate studies are a Good Thing (Score:5, Interesting)
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Clinical trials only work because some people are sacrificed to save more people in the long run.
Indeed. I have a friend who recently passed away from a terminal disease. She would have (and did try) nearly anything to get into a trial that might have helped her live a few more months. When your time is short, as her oncologist put it, the survival instinct is strong. If you know you are going to die for sure in a matter of weeks, your tolerance of risk increases substantially and you will most definitely try things that might only maybe have a chance of helping.
What's the efficacy? (Score:3)
I'm sure many Parkinson's patients would prefer a small chance of death for a severe reduction in symptoms. It's a very painful and debilitating disease. I'm not saying that deaths are a good thing, or that it shouldn't be investigated carefully... but sometimes quality of life is more important than not dying.
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This is very true. My mother always regretted having treatment for cancer when in her '80s. The treatment indirectly destroyed her quality of life and when the cancer returned, she opted for palliative care instead of treatment, knowing that death was inevitable.
The people taking the drug are very sick anyway (Score:5, Insightful)
Having read the article, the summary misses a crucial point: this drug is being given to people who are very, very sick. The drug manufacturer is in close contact with the families of the people taking this drug. Given that the people taking the drug literally have zero other options, it's honestly not surprising that there is a huge number of people dying whilst on this drug.
The key question is: would they have died anyway?
Yes, the FDA was concerned about rushing this drug to market. Yes, a significant number of the population dies when taking the drug. But there is also zero evidence that the drug caused the deaths.
It's incredibly sad when a loved one dies. Seeing my dad slip away (eg. getting lost in a supermarket he had gone to for 40+ years, talk about me as if I was someone who he had just met) was heartbreaking. I can get a sense of the pain of families who though they were doing the best by getting their loved on on this drug. But life is finite and new treatments carry risk. We learn as much as we can from this and move forwards.
And cry. But that's normal, too.
Clinical trials are how we learn (Score:4, Insightful)
The key question is: would they have died anyway?
The only way to know that is with a properly conducted clinical study. Preferably of the double blind variety if possible. Which makes the whole argument about rushing the drug to market before we know if/when/why/how it works idiotic. We do studies the way we do them for very good reasons which we learned the hard way. It's a tragedy that anyone has to suffer or die but those deaths become wasteful if we do not learn anything from them. The greater tragedy is to have more people die because we lacked the patience and fortitude to see the studies through and look for the evidence.
My mother just died from ALS. Horrible way to die. There are some experimental treatments but she understood that the odds were against her so she enrolled in some studies to help other people. She didn't want to die or suffer but the only way we will ever cure a horrible disease like ALS is if some people willingly sacrifice themselves for the greater good.
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While there do need to be double blind studies, there also needs to be a "fast usage" track for drugs like this. It shouldn't be a track that leads directly to general approval, and it should include some features that discourage the manufacturers from pushing it.
My preference is a requirement of thorough monitoring of the patients and forbidding the manufacturer from making a profit on drugs used in the study. And guaranteed open publication of the results. This is clearly inferior to a double blind stu
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While there do need to be double blind studies, there also needs to be a "fast usage" track for drugs like this
The double blind studies ARE the fast usage track.
The doctor is supposed to ask terminally ill / patients with extreme symptoms if they want to participate in a study about unproven but promising drug. The next-of-kin decide if the patient is deemed unable to decide for himself. The drug is promising because of in-vitro / animal studies. There is a chance they will get placebo - neither the patient nor the doctor will know. The "promising" drug is generally free for such patients, and some additional monito
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They'd do it because it could yield data on whether or not they should go to the time, effort, and expense of doing the double blind study that would justify FDA approval.
Double blind studies are expensive to set up, and it would be quite useful to be able to get a better idea ahead of time as to whether one is worthwhile.
The Post Is Incomplete (Score:5, Informative)
The original poster provides information from the CNN article that shows deaths. It does not report that (1) the patient population is old, thus prone to death, and (2) that two follow-on studies found no difference in death rate between the drug and placebo. FDA is monitoring the reports of deaths, but unless someone does the science to find out whether the deaths of these old people are unusual you risk denying the patient population that benefits from the drug the relief from their disease.
It is good that the medical community is being made aware of the adverse event reports. Doctors and patients (or their guardians) should know this information in making a personal decision.This is not a drug category with good options as shown by the breakthrough drug designation.
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The original poster provides information from the CNN article that shows deaths. It does not report that (1) the patient population is old, thus prone to death, and (2) that two follow-on studies found no difference in death rate between the drug and placebo. FDA is monitoring the reports of deaths, but unless someone does the science to find out whether the deaths of these old people are unusual you risk denying the patient population that benefits from the drug the relief from their disease.
It is good that the medical community is being made aware of the adverse event reports. Doctors and patients (or their guardians) should know this information in making a personal decision.This is not a drug category with good options as shown by the breakthrough drug designation.
CNN reporting a half truth / incomplete story for sensationalist purposes? Unbelievable. If they keep that up someday they will be known as fake news. Did they link Trump to it yet?
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CNN reporting a half truth / incomplete story for sensationalist purposes? Unbelievable. If they keep that up someday they will be known as fake news. Did they link Trump to it yet?
Give me one for profit network that is not willing to report "a half truth / incomplete story" or even a straight out lie "for sensationalist purposes."
Honest TV reporting died in the 80's when network executives stopped treating journalism as a "loss leader" and turned news it into another "entertainment" revenue stream
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Give me one for profit network that is not willing to report "a half truth / incomplete story" or even a straight out lie "for sensationalist purposes."
Honest TV reporting died in the 80's when network executives stopped treating journalism as a "loss leader" and turned news it into another "entertainment" revenue stream
I don't disagree. That's why I always consider the source when I read the headline and also why I don't watch TV news. I only read my news, and even then I try and get it from multiple sources to try and triangulate the truth. This only gets harder when people purposely distort / conflate things such as mixing and matching all immigrants with just legal immigrants with just illegal immigrants depending on which data set best supports their position at the moment.
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Actually, it DOES point out that the target group is people with advanced Parkensons and that there is likely to be a large number of deaths due to that. It also DOES point out that other studies showed that the death rate is the same as those on placebo. You may have stopped reading too soon.
It also points out that some patients saw a lot of improvement and others saw none.
Just let people use pot! (Score:2)
Just let people use pot!
SAEs... (Score:5, Informative)
As a former Serious Adverse Event Co-ordinator for an ethical review board for medical studies (IRB) let me explain what is going on here.
Firstly, the study population will have advanced Parkinson's with psychosis. This is a cohort that is very ill and has a high probability of adverse outcomes.
Secondly, the risk a drug can present is set against the benefit it might provide. So for example if a pain killer offers very mild pain relief, but you're just as likely to have blood clots if you take it, the risks outweigh the benefits. Similarly, if you have a medication that is a breakthrough medication with a high degree of success at attenuating psychosis in people with Parkinsons, a higher level of risk would be considered acceptable.
The clearest example of this is the various chemotherapy drugs. They're absolutely toxic - poisons. They're just not as bad as dying.
So here's where it gets complicated. And it's why I am no longer working in the medical ethics field.
As a Serious Adverse Event co-ordinator, if something bad (the "adverse event") happens to a patient taking a study medication or using a study device, the Principal Investigator has to submit an SAE form to their IRB (Independent Review Board.) So I come in at 9am and find a pile of letters reporting various negative outcomes. I then have to sort them into two piles based on some criteria.
The first pile is ignored. Here's what goes in that pile: Any outcome that was described as a possibility in the Informed Consent document. If the Informed Consent the patient or their representative signed when they joined the study said, "risks include bone necrosis, blindness and death" then if any of those things happen, they don't get reported to the FDA. Instead, I send the PI a form letter saying the Adverse Event was not notifiable.
The next level of filtering is that I then examine the adverse event itself, to make a judgment call of whether the event was "Unlikely" "Possibly" or "Definitely" related to the study medication or device. If it was unlikely or possibly related, the form letter is sent to the PI and the FDA isn't notified.
Only if the outcome ISN'T described in the Informed Consent, AND I determine the event was "definitely" related to the study medication does the FDA ever get to know of the event.
The disheartening thing about this is, and I'll give a real but anonymized example: one study I was SAE for, I would get many, many reports of bone necrosis of the jaw for a cancer medication. People's jaw bones were dying, and they would lose all their teeth in their lower jaw. They often would get infections, and in a few cases they would die. This was all described in the Informed Consent. For this one particular drug, the adverse event happened a lot - at least 30-40% of the cohort was experiencing this. The drug was lifesaving, so a high level of SAEs could be tolerated, BUT there was an existing medication on the market that had this adverse event occur at a much lower level - around 10-15%.
And there was no mechanism for me to give this information to the FDA. There was no way for them to know what the real stats were. Their information collection system was designed to ignore vast swathes of negative outcomes.
The pharma companies know how the system works, so they obviously will try to list any inhibiting adverse events in the documentation. If you read the documentation for approved and released drugs, the same things happen. Everyday drugs have listed side effects that include death all the time. Now you know why.
So, obviously, I don't work in that field any more. It was soul destroying.
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The pharmacos made it this way. Only national scale adverse events cause changes to the legislation that do not favor the pharmacos.
The situation is highly predictable. People aren't watching.
The IRBs have to follow the rules too. They do their best to make sure the Informed Consent ethically and honestly describes the study, the process and the possible/probable negative outcomes.
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Sometimes they are worse than dying. That was the decision my mother made when her cancer returned -- she opted for palliative care the second time around, instead of the chemo.
Your points are valid. Many treatments have side effects and risks. There is a need for a rational assessment of the risk versus the benefit.
Re:SAEs... (Score:5, Interesting)
The Pharma companies created the system, as they're the only ones interested in the legislation. The public who are interested are simply a couple of determined individuals with no money and the huge emotional burden of a personal loss. They are ineffective.
Here's a secret: Every IRB is required to have a board of medical professionals, and a lay person. The lay person must come from outside the medical profession.
YOU can be on an ethical review board.
Also, it pays quite well.
Wrong model for approval (Score:2)
A bette
Disease is life-threatening, so... (Score:2)
"substantial improvement" in patients with serious or life-threatening diseases compared to treatments already on the market.
Reports of deaths by those on the drug does not necessarily imply a causal relationship.
Their condition is life-threatening, therefore, you administer the drug. If the drug doesn't work then possibly they die. If the drug kills them, then they die.
So how to make sure they responsibly analyze the report and find the reason for complications was caused by the drug, and
The try before approval program is valueable (Score:2)
These people used these drugs being aware of the dangers and lack of review, because they were their last hope. They were used on people as a last ditch effort for people who are already very ill. Overall, allowing people to try more dangerous but promising drugs will save lives by helping getting drugs that do work available faster. Since these people were already in a very critical state its absurd to blame a drug that was their last hope, because they had lost their quality of life already. To move thing
Problem solved (Score:2)
Problem was Parkinson disease. Death removed Parkinson, the cure is efficient
Seriously speaking, if I was trapped in Parkinson, a risky cure with possible death outcome would seem better to me than status quo.
Good question. No. (Score:2)
The FDA receives notifications of patient deaths all the time. The patient was taking a drug for hair loss when he walked in front of a bus and got killed? It may be reported.
I know people who work for the FDA and deal with reports like these. It is a complex and never certain statistical task trying to interpret all this data.
Re: Society killed these people (Score:3)
The truth is every one of them could be having quality of life improvements from Marijuana usage
Don't forget whisky usage. Works just as well.
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Frankly I don't agree with the FDA regulating consumption, use, and access to drugs no matter how safe or dangerous. Study them, publish and inform people, control safe manufacture and production, accurate labeling and advertising, that sort of thing.
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YES! The FDA was ostensibly created to ensure the purity and accurate labeling of products and foods being sold. That is a good, worthy, and defensible job. It would be within their general remit to publish studies of effectiveness of various materials for treating various conditions.
When they go beyond the jobs listed in the previous paragraph they step beyond being definitely justifiable and worthy, and enter a different realm of activity, which is much less laudable.
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Actually, no. Whiskey doesn't work as well. Whiskey tends to foster inflamation, and inflamation is a general system toxin. Being toxic is, of course, it's purpose as it's designed to kill off bacteria, but when it becomes systematic is almost always does more harm than good.
That said, I also use Whiskey for mild pain, when I've taken as many aspirin or ibuprofen as I feel secure in taking. It's quite effective against pain, though when taken in doses strong enough to quiet severe pain it's likely to en
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Fast Tracking has been pushed by the last 4 administrations. [latimes.com] #FTFY #Troll