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.

Comments

Ah yes.. The reason the FDA does reviews

[bobbied]

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

[bluefoxlucid]

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.

Re: Ah yes.. The reason the FDA does reviews

[c6gunner]

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.

Worth the risk

[Anonymous Coward]

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.