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comment by thundara

You're presenting a lot of this data from the point of view of your conclusion, rather than taking into account each study individually. It seems to have given you the completely wrong impression of a lot of what you're talking about.

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Let's start with one of these: proof-of-efficacy

Before the Kefauver Harris Amendment, companies did not have to prove their drug actually worked before selling it to patients. Which means that all you had to do was have a few trials that showed your drug didn't kill people and you'd have an opening to make hundreds of millions of dollars.

Doctors simply don't have the time or expertise to evaluate the data on every drug put before. So if a drug is FDA-approved, and a company comes to them with a marketing report claiming 20% benefit over competitors, they won't double-check the methodology of the trial, the patient enrollment, the statistical tests. If the last set of treatments didn't work, they'll hop on the new drug until either (1) they see for themselves that it didn't work, or (2) an independent organization runs its own trial or checks patient records after the fact.

So if that drug turns out not to cure disease X, you've exposed patients to unnecessary risk (and yes, all interventions carry risk), because the regulatory bodies would rather look in the rear view mirror to catch an accident.

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Take the conclusion of your figure: it's harder to get a drug approved when you require proof-of-efficacy

The other outcome of the Kefauver Harris Amendment was a study to characterize the efficacy of all past drugs: DESI. They found that 3 in 10 drugs of those drugs didn't do anything.

Say you had stage 4 melanoma, the doctors give you 6 months to live, and you're already looking at drugs that have a 10% chance of delaying your death. If you're going to pick one, do you want a 3 in 10 chance of picking the wrong one because the FDA was only there to make sure it didn't also give you a heart attack?

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There's trade-offs with every one of these regulatory decisions, but let's consider another example: off-label prescriptions

This is a controversial solution to a problem of binaries. If a drug is good, a company can go through a full set of trials and regulatory approvals. But if that costs hundreds of millions of dollars, with a 1:3 chance of success, and the new market is smaller than cost * risk, they won't do it. So off-labels are a patch to the problem by saying: they won't run they trials, they can't market the drug. If a doctor doesn't have better options, they can prescribe the drug, knowing that it carries the lower grade label and increased risk.

Some would say that's counterproductive, others would say a stratified landscape is better than a binary one. Your own link describes it as a polarizing term. It's a tough choice, but not really a reason against the FDA as a whole.

And this isn't even starting to take into account fast-tracking drugs, FDA underfunding over the decades, fewer low-hanging fruit, etc.

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    "No FDA official has ever been pubicly criticized for refusing to allow the marketing of a drug. Many, however, have paid the price of public criticism, sometimes accompanied by an innuendo of corruptability, for approving a product that could cause harm."

I beg to differ...





wasoxygen  ·  2801 days ago  ·  link  ·  

    You're presenting a lot of this data from the point of view of your conclusion

I agree that I am giving one side of the argument; I think it is a side that gets less attention and should be included in a balanced discussion. Regulation has benefits and costs; alternatives to regulation have benefits and costs.

When you say "There's trade-offs with every one of these regulatory decisions" you have expressed 90% of what I want to say. Trade-offs are almost inevitable in complex environments, and it's hard to make fair evaluations. I think there is a common tendency to over-value intentions rather than results, and to overlook large but inconspicuous effects.

Mainly I find it fascinating to look into these subjects and learn what snake oil actually was, and that the FDA permits prescription-based homeopathic remedies for grave illnesses.

Thank you for sharing your thoughts; in particular I had not heard of the Drug Efficacy Study Implementation.