This is another example of how the FDA sucks: it sucks for terminal cancer patients like me (https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-ce...), who are denied potentially life-saving treatments due to the FDA's slowness and intransigence, and it sucks for the millions of mental health patients who are likely to be aided by psychedelic medicine. The FDA's paternalism is incredible. It's grossly inserting itself between clinicians and patients, and preventing the use of a drug that's been known for decades.
In "Is the FDA Too Conservative or Too Aggressive?: A Bayesian Decision Analysis of Clinical Trial Design," the authors find that:
Implicit in the drug-approval process is a host of decisions---target patient population, control group, primary endpoint, sample size, follow-up period, etc.---all of which determine the trade-off between Type I and Type II error. We explore the application of Bayesian decision analysis (BDA) to minimize the expected cost of drug approval, where the relative costs of the two types of errors are calibrated using U.S. Burden of Disease Study 2010 data. 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 23.9% to 27.8%. 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% to 1.5%. 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.
PTSD is not generally fatal, apart from suicide risk, but denying treatment due to arbitrary hypothetical dreamed up by the committee members is a travesty.
When you take into account drug overdose + suicides + issues caused by morbid obesity + propensity to engage in high risk impulsivity, PTSD (and especially CPTSD) are much closer to generally fatal than anyone should be comfortable with.
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[ 2.7 ms ] story [ 21.0 ms ] threadIn "Is the FDA Too Conservative or Too Aggressive?: A Bayesian Decision Analysis of Clinical Trial Design," the authors find that:
Implicit in the drug-approval process is a host of decisions---target patient population, control group, primary endpoint, sample size, follow-up period, etc.---all of which determine the trade-off between Type I and Type II error. We explore the application of Bayesian decision analysis (BDA) to minimize the expected cost of drug approval, where the relative costs of the two types of errors are calibrated using U.S. Burden of Disease Study 2010 data. 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 23.9% to 27.8%. 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% to 1.5%. 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.
PTSD is not generally fatal, apart from suicide risk, but denying treatment due to arbitrary hypothetical dreamed up by the committee members is a travesty.
[0]: https://news.ycombinator.com/item?id=40558487 [1]: https://news.ycombinator.com/item?id=40580334 [2]: https://news.ycombinator.com/item?id=40580525