NorthWest Biotherapeutics Inc (NWBO)

[sentiment_stocks]

These days, however, the FDA seems to be leaning away from randomized trials, especially for rare diseases, which GBM is considered to be. Not only do they consider them to be infeasible, they are moving towards thinking them unethical.

For instance, in the September NEJM, in this rather convoluted passage, these FDA authors...Fashoyin-Aje, et al (including Richard Pazdur)... indicate that randomized trials for rare diseases are not feasible. They further indicate that overall response rate and response durability endpoints will serve as verification of clinical benefit. I'd think it obvious that overall survival would serve as an even better verification of clinical benefit than ORR and RD.

With the exception of rare diseases, for which the FDA has accepted the further characterization of the overall response rate and response durability as verification of clinical benefit because of the infeasibility of conducting randomized trials, oncology drug sponsors have typically conducted a single-group study to support AA and subsequently conducted a randomized, controlled trial evaluating a long-term outcome such as survival to verify clinical benefit.

https://www.nejm.org/doi/full/10.1056/NEJMp2208954

And for those who can't access the full text at this time, ae austerer provided the full text to this article in this link:

https://investorshub.advfn.com/boards/read_msg.aspx?message_id=170040350

So while you think using ECAs in a randomized P3 trial is not going to fly, it would seem there some at the FDA signaling otherwise.

As for the SAP... it's not the same, you are correct. What is the same is that the UK agreed to the same methodology that was used to pre-specify the external controls in the DCVax-L P3 SAP to identify those for the PIP external control arm.