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FDA Worried Drug Was Risky; Now Reports of Deaths Spark Concern (cnn.com)
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.
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?
"File to fit, pound to insert, paint to match" - Aircraft Maintenance 101
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.
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.
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...
Not every disease can be neatly solved and not every precaution is warranted. For America, deadly government-imposed regulations are a worse evil than patient choice (hopefully made with their physician) both by the philosophy and by the statistics. It shouldn't be surprising in the light of the Socialist Calculation Problem (from an information theory perspective) that freedom works better than being "managed" like livestock.
My God, it's Full of Source!
OUTSIDE_IP=$(dig +short my.ip @outsideip.net)
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.
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.
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?
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.
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.