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Bon Kwi Kwi's avatar

Malory and Prasad are heroes for standing against Pharma’s mighty Wurlitzer machine.

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Nora M. Shamoon, MS's avatar

"...how about biotech companies complain less about 'changing standards' at the FDA and instead produce more than just bare-minimum science that checks boxes but actually provides no answers about whether their drugs work?"

Yes. Not sure what can be learned from a study if patients are cherry-picked, previously received chemotherapy (pre-treatment) prior to the drug(s) in question (not to mention were well enough to tolerate chemotherapy, another filter perhaps), and there is no placebo control arm. I think open-label should also be considered a less-than-ideal design. Double-blinding helps when possible/ethical. At minimum, when rating "tumor response," the investigator should be blinded. But in this case, it wouldn't matter since there's no true control group.

To that end they mentioned, "the clinical trial will evaluate complete response (CR) rate and overall response rate (ORR) as its two primary efficacy endpoints as assessed by independent review." It’s still non-randomized and open-label. The only "independent" variable is that the reviewer didn't physically administer the treatment.

They seem to argue that it costs more up front to do the proper study and takes too long, and then get upset when their drug is not (acceleratedly) approved with weak evidence. What did they expect? It would likely lead to a stronger likelihood of approval if FDA was presented with data from a well-designed study: control vs. treatment arms, properly powered, showing statistical significance of clinically meaningful endpoints, (overall survival primarily, tumor response(s) secondarily, although I saw CERPASS has a primary endpoint based on response rates) and significant follow ups that show duration of response.

I wouldn't care if my tumor "shrank" on the scan if I still felt like shit and had recurrence in the following years. And there would be significant financial toxicity after paying for these six-figure treatments. Even if CMS covered the majority (only after approval), it could still cost tens of thousands.

It's ridiculous and the wrong way to convince any good scientist, especially a sound regulatory body. I am very confused about why they are attacking the FDA, what benefit is there other than clicks and praise (and financial incentives?) from pharma? Otherwise, we are just hoping it works based on weaker evidence.

Should our FDA be attempted to be coerced (I'm not sure how else to see these hits pieces) and subjected to deeply hurtful accusations by WSJ and others every time they don't immediately rubber stamp a product with weak evidence? Should we all just take drugs that cost a lot and may not help reduce or stop disease and so be left with the negative side-effects any drug undoubtable has? Should GDP on health care spending in the US just keep increasing but our life expectancy/quality not be improved? Should we keep bowing to pharma and making their CEOs/stockholders beyond rich while people with serious illnesses are given false hope?

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