FDA Approving Drugs at Breakneck Speed, Raising Alarm
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In my own little corner (diabetes) it’s been nice to get monitoring tools earlier than they once would have been approved, and for my medical team to confidently plan for equipment changes based on future approvals.
the approval then means that companies can target people who are desperate with extremely expensive snake oil. The victims of this have no recourse if/when the drug has caused problems.
Doesn’t work = efficacy trials
Is harmful = safety trials
iirc the safety trials come first. A lot of drugs get stalled in efficacy trials because they might only work for some patients and the sample set is small or poorly chosen.
I see no reason to stall drugs that are proven safe, even if they might not be effective for all patients, as long as there is full disclosure and informed consent. Freedom to choose is a good thing.
Being ineffective can be very dangerous as a person taking a drug expecting it to alleviate their condition finds no relief and thus dies from their condition.
You did not fully read and comprehend the parent post.
Phase I determines safety. Phase II determines efficacy. Phase III is larger scale, randomized, and blind to give the FDA more information on the range of potential adverse effects and the effectiveness of the drug. FDA is supposed to care about both. The acceptable safety of a drug is relative to the effectiveness of it (i.e. a drug may be more dangerous or have more adverse effects, but it’s deemed “worth it” due to the effectiveness of the drug while of course maintaining a minimum safety risk for all drugs). In other words, both safety and effectiveness are both supposed to be taken as equal halves of the whole by the FDA.
I do.
For every treatment you are taking, you could have been taking some other treatment that might be more effective.
If we have no idea whether or not a drug is more effective than placebo, we should not be administering it.
However, there are many cases where there is no alternative drug or treatment that has been tested for efficacy, and so you’re stuck with hospice as standard of care.
It’s one thing for a doctor to recommend administration of a medication, it’s another thing entirely to make it unavailable for those that are in a position to make the choice for themselves.
Because they don't want the side effects of proven treatments, and they hope that snake oil will help them.
If people were rational about their health, homeopathic supplements, and other similar bullshit wouldn't be a billion dollar industry.
If you can't prove that your treatment works, you shouldn't be advertising it as a medical treatment. Advertise it as a non-medical non-treatment.
I thought the FDA summed it it up well in the article:
>“We would expect every once in a while that we would be wrong,” with an accelerated approval, she said. “Otherwise, we aren’t taking any chances, we are still making them wait too long to get their hands on this therapy that will extend their lives.”
Vinay Prasad is a cancer expert and an outspoken critic of too fast approvals of drugs. He has done research on it, I could e.g. find this:
https://jamanetwork.com/journals/jamainternalmedicine/fullar...
Interestingly, the paper linked shows that in some respects, fast tracked drugs have a better track reccord. This shouldnt be surprising as the best drugs are fast tracked.
>18 drugs failed to improve overall survival (in 6 of 15 accelerated approvals and in 12 of 21 traditional approvals)
The results for conventional fixed-sample randomized clinical-trial designs suggest that for terminal illnesses with no existing therapies such as pancreatic cancer, the standard threshold of 2.5% is substantially more conservative than the BDA-optimal threshold of 27.9%. However, for relatively less deadly conditions such as prostate cancer, 2.5% is more risk-tolerant or aggressive than the BDA-optimal threshold of 1.2%. We compute BDA-optimal sizes for 25 of the most lethal diseases and show how a BDA-informed approval process can incorporate all stakeholders’ views in a systematic, transparent, internally consistent, and repeatable manner.
Taking enough time to endure a new acne medication or Viagra alternative etc is reasonably safe is very much worth spending some extra time.
Edit: This does seem to be focused on needed medications without close equivalents but that’s a fairly arbitrary line.
It’s not an easy choice, especially when the risks are often hypothetical and the benefits so clear.
It’s interesting looking at lists like this and considering where things broken down. https://en.m.wikipedia.org/wiki/List_of_withdrawn_drugs
If you're looking to treat relatively health people for most of their lives (say a drug that precent type I diabetes), the FDA will scrutinize the hell out of it.
If you're looking to treat someone who is dying (e.g. metastatic cancer), then the FDA is much more lenient in their analysis.
I wish I could buy my own life sustaining hormone that I've been using for 20 years and understand better than most doctors outside endocrinology.
I immediately thought that type of failure should be recoverable without a hospital visit and surgery and tried to figure out why it wasn't.
My mind immediately set to making it so and was held back by the fear I could go to prison for practicing medicine without a license.
"I love you. I wish I could fix you. I could fix you."
"I know you could. I wish you could."
Normally that kind of failure would be quickly recoverable without a hospital visit/surgery, I’m sorry that this one was not.
My chief complaint is that the FDA is overly restrictive. They force manufacturers to make the products expire earlier to head off concerns about them working less efficiently near the end of the cycle. Diabetes is a disease who's treatment is entirely about guesstimating but the FDA does not take that into account when approving devices.
A lot of harm is done to diabetics by denying them better treatments and tools in the name of avoiding extreme highs and lows.
This isn't theoretical--someone already wound up in the hospital "hacking their diabetes". And the FDA came down on the companies involved in manufacturing the devices--even though they had NO involvement.
I'm sympathetic and recognize that modern technology could be doing MUCH more for diabetic patients. However, my measure of importance is how much money is being thrown at the problem. And there simply doesn't appear to be very much money being tossed around for a medical system that will have to go through FDA approval.
This should be obvious.
Because there are people and groups drinking bleach and treating it as a cure-all for diseases. I don't think going back to the old American Wild West of snake oil selling for diseases is a good idea.
It's ok to do those things but not try to care for yourself?
> But even as drugmakers, investors and patients cheer on the agency’s pace, patient-safety advocates argue that speed comes at a price. Studies show medicines approved on a faster time line are more likely to have safety problems emerge after they become broadly available, while other treatments offer fewer benefits than anticipated.
Not sure I understand the concern:
- "studies show medicines approved on a faster time line ...": which study(ies)? And that was the case for previous track records, which says nothing if the FDA is making the same mistakes that they may have done 20 years ago.
- "more likely to have more safety concerns once they are broadly available while other treatments offer fewer benefits than anticipated", wait, do such studies actually have drugs that are virtually identical in all properties and where the only factor that changed is the approval timing? Because if that's not the case, you are just comparing drugs with different profiles, different indications, and different targets, which is pretty much apples and oranges. Good luck making conclusions based on that.
I am not aware of the FDA or any other regulatory body at this time starting to remove regulatory requirements to speed up approvals. The typical things we see nowadays is an exception for "orphan indications" where there is no good treatment available and people are dying without anything to try. It seems fair that they take in account patients' needs when it comes to expediting the last bureaucratic mile to ensure new drugs can save more patients. At the end of the day, patients are also free to refuse newer drugs (and doctors usually tell them when a drug has just been approved and when there is lack of much safety experience beyond clinical trials) so I don't see how you can frame this whole story in a negative light.
_"It’s not just speed. The FDA also is approving more drugs, hitting a record 59 new therapies in 2018. Almost three-fourths received a priority review. That, combined with more efficient data collection, is responsible for the faster FDA action, said Aaron Kesselheim, a professor at Harvard Medical School. Companies are also communicating earlier and more often with the agency, which can head off issues at preliminary stages and help them get products through on the first attempt, he said."_
So as in any field, better communication with stakeholders improved the process and eliminated some issues earlier.
Then, better data collection helps them evaluate studies better. I bet it also reduces the back and forth and the "what does this mean" aspect of the studies after.
And then companies can pay for expedited evaluation.. which if they've been communicating clearly all along and have better data, doesn't seem far fetched.
The next thing you know, someone will suggest clear planning with stakeholders, code reviews, and unit tests will improve code quality and even ship dates! But that may be crazy talk.
Including the most famous one: Epipens
https://slatestarcodex.com/2016/08/29/reverse-voxsplaining-d...
I was at a continuing education course last year. FDA approved a new narcotic very fast. A non narcotic alternative is taking forever to approve.
Its name is Maxigesic. Tylenol and ibuprofen in one pill.
Why?
What's the point of this, compared to just having half a pill of tylenol and half a pill of ibuprofen?
Meanwhile 500mg is, I believe, more than they combine with opiates and acetaminophen does lead to death via organ failure on the order of hundreds of people a year. I was curious why they weighted the acetaminophen proportionally so much higher than the ibuprofen.
Unless they need to be prescribed in ratios that can't be achieved reasonably with the cheap OTC pill dose sizes, it's not clear to my why I'd want a combined bill instead of just taking separate pills.
Taking more than one pill is less convenient, but except for people who have trouble with pills in general I doubt they would want to pay more for that convenience.
Even in the case of fairly high doses, it is not too bad. A few months ago, for example, I somehow managed to get a painful rotator cuff injury while sleeping (no idea how), and my doctor told me to take 1000 mg Tylenol every 8 hours and 800 mg of ibuprofen every 8 hours. At the most common OTC strengths, that would be two Tylenol pills and 4 ibuprofen pills.
It's a damn shame that doctors often fail to tell people about cheaper and more irritating alternatives, but there's nothing wrong with giving people the option to pay more for a better experience.
You often see this in terms of number of times a drug had to be taken per day. There'll be a generic that needs to be taken say three times a day, and a newer drug that only needs to be taken once.
I highly doubt every manufacturer of Ibuprofen or Acetaminophen would decide to throw in the towel if this new drug was released.
The FDA has a massive backlog for generic drugs, while novel drugs (NDA) don’t.
The market is full of drugs that are combinations of OTC medications and usually they are way more expensive. Even if they didn't go nuts with the price, why make a patient pay $0.50 per pill when they could take 2 for $0.04?
As to the question about why it is taking so long at the FDA, I imagine it isn't a very high priority because there are alternatives.
The problem is the lack of transparency into cost which results in some drug companies taking advantage.
Combine two pills that cost $0.10 each into one pill that costs $0.15? Reasonable.
Do the same and charge $5 per pill? Ridiculous. Problem is doctors and patients don’t know it costs $5 and insurers can’t be bothered to push back. Suddenly you’ve increased the cost of therapy by 50x.
The drugs in question could, combined have deleterious effects (say if their extortion mechanisms are the same).
This shouldn’t be a problem because Tylenol + Advil has been done for decades. However, a beauricracy doesn’t see it as a solved problem to fast track unless there is a specific fast track for this exact scenario.
I looked up the "consumer watchdog" that I'm guessing had something to do with this article, and their front page is just an "Impeach Trump" advertisement. See https://www.citizen.org/. Also, if you click their "About Us" page, a few lines down is: "We don’t participate in partisan political activities or endorse any candidates for elected office."
Hmm.