RE: FDA changes in trial design27 Feb 2026 11:27
I'm not sure what you mean Ice. For some time now oncology trials can lead to provisional approval based on a pivotal Phase 2 trial but need a Phase 3 trial to confirm that approval. To speed things up, as cancer is time critical for patients, surrogate end points that are indicative and achieved more swiftly are being used.
A summary of the new FDA guidance:
"While US law has been in place since 1997 stating that approval can be based on a single adequate and well‑controlled study when supported by confirmatory evidence, the FDA’s standard has been a reliance on two pivotal trials. Under the new guidance, the single pivotal trial will still need to be combined with confirmatory evidence, and the approval process will put a stronger focus on controls, endpoints, effect size, and statistical protocols. The paper says the move will “substantially reduce costs for sponsors” and “speed drugs to market”. Estimates suggest that the cost of a single pivotal study may range from $30m to $150m. Makary and Prasad add that as a result of reducing capital costs for drug developers, it should reduce “justification of lofty and rising drug prices for everyday Americans”. Prasad and Makary acknowledge that this guidance may be met with criticism that the FDA “relaxing its standards”, but the pair disagree. [continues]"
https://www.clinicaltrialsarena.com/news/fda-to-allow-one-pivotal-trial-instead-of-two-for-drug-approval/
Full guidance paper (requires free account): https://www.nejm.org/doi/full/10.1056/NEJMsb2517623
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You wrote: "once you prove the delivery works in humans, every new payload isn’t a brand new science experiment, starting from scratch like 30 years ago under the old FDA process of evidence and more evidence through trials. If FDA is happy with one strong pivotal backed by mechanism and biomarker data, that massively suits a platform model. Validate the engine once, then you’re just swapping warheads."
Every pipeline drug is novel and unique. Are you saying that every pipeline drug can be approved after one strong pivotal trial—what I wrote earlier—or that, because AVA6000 gets approved for a particular indication or two (or three), all drugs using the pre|CISION dipeptide should be approved ...for all indications? ...without their own pivotal trials?
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