The problem is the approval of drugs that don’t actually work, and so are actually harmful.
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.
There is obviously a tradeoff between risk and speed. The article provides evidence that speed is increasing. I don't think the article provides any compelling argument that approvals are too fast. Do you?
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.”
> There is obviously a tradeoff between risk and speed. The article provides evidence that speed is increasing. I don't think the article provides any compelling argument that approvals are too fast. Do you?
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:
Is the FDA Too Conservative or Too Aggressive?: A Bayesian Decision Analysis of Clinical Trial Design
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.
Im not familiar with his work, but from what you posted, it seems Prasad takes issue with the use of surrogate end points in trials, not fast track approvals. I tend to agree that surrogates and bio markers are a huge problem, but think they are a necessary evil. Drug development would screech to a halt if developers had to wait another 4-5 years for survival data. What we need is more Pase 4 postmarket trials with direct endpoints.
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)
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.
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.
But you also want to consider alternatives. If you have something which is only effective for a small percentage of people even though it's really dangerous, that may still be worth it as something to use for people for which existing treatments were ineffective and the remaining alternative is certain death. Saving 15% of those people can be better than none.
> 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.
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.
This perspective is very frustrating to me. Why in the world would a sane person take a drug with unproven efficacy when others with proven efficacy are available?
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.
> This perspective is very frustrating to me. Why in the world would a sane person take a drug with unproven efficacy when others with proven efficacy are available?
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.
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.
While I agree with you... if you take the side of the Medical and Insurance industry, it works great. Money is extracted before they died with a high margin process. The incentive is perverse, but it's real and effective.
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.