3 ms·

> 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 compellin

by hannob 7y ago

> 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: https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2463590 https://jamanetwork.com/journals/jamainternalmedicine/fullar...

s1artibartfast 7y ago
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)
robbseaton 7y ago
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. https://www.nber.org/papers/w21499 https://www.nber.org/papers/w21499

by hannob 7y ago

s1artibartfast 7y ago

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)

robbseaton 7y ago

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. https://www.nber.org/papers/w21499 https://www.nber.org/papers/w21499