Replies to post #708612 on NorthWest Biotherapeutics Inc (NWBO)

[dennisdave]

Has been quoted and cited many times. From FDA guidance on external control arms:

Nothing in there that underlines your claim that the FDA rejects ECA if there is no patient level data. As a matter of fact the guideline does say this

"This guidance does not address other types of external controls, such as using summary-level estimates instead of patient-level data.

You just referring to a guideline or the FDA, just as Doclogic does, makes you an idiot. Put your money where your mouth is and start QUATION the FDA. Pock pock Pock pockPock pockPock pockPock pock chicken. from bcplatforms.com

BC Platforms’ Global Data Partner Network provides patient level, longitudinal, and geographically diverse real world data (RWD) for use as external control arms (ECAs) in randomised human clinical trials. Through the use of RWD, fewer patients are required to be directly enrolled in randomised clinical trials, enabling broader patient inclusion and shaving significant time off the clinical development process — saving cost and resource — while facilitating improved access to new drugs, in new markets, for the global patient population.

. There is an industry around summary-level estimates that should not exist according to you

The sponsor can know results of efficacy IAs prior to datalock.

Not if the sponsor plays by FDA rules which what NWBO did, you liar. You have zero knowledge of FDA regulation and are as dumb as a monkey's ass. You keep lying about this and have no evidence what so ever

The designed in OS was between the 233 that were randomized to -L and 99 that were randomized to placebo. That failed badly.

No they didn't they improved mOS x3 vs SOC

You continue to argue it would make trials impossible to stick with this,

I didn't argue this you arrogant lying ass, I literally quoted The American Brain Tumor Association saying that, but you ignored that as you ignore all logi.

You twist lie cheat. It is not possible to have a normal conversation with you since you keep ignoring reliable resources and MDs, refuse to quote the FDA and twist everything that anyone says.

[biosectinvestor]

The guidance it has been quoted to you is not fixed, it does not have the power of regulation, it is purely guidance and FDA does things not consistent with Guidance all the time as it explores new ways of doing things.

The relevant issue there, in what you repeatedly claim is fatal, is BIAS, and if you address bias by other means, the regulator very likely will take those efforts completely under consideration and I expect the company was in complete consultation as they did take such steps. I would not expect your formalism to be their primary concern.

In the JAMA paper ancillary docs they provide details about the external control trial arm and how it was developed. A completely blinded, expertized epidemiological and statistical consulting firm was hired on a completely blinded basis. They know GBM, they know the parameters of this trial and all the trials that were in the industry. No additional information was provided to them and they have provided affidavits to that effect with their report. They remained blinded to the trial as though it had not begun and their advice was prospective in the development of their ECA tool. Then they did statistical analysis once the trial was unblinded to validate their work. They went far beyond the normal statistical analysis that would be presented to do that analysis and present proof that their method was valid.

They were the ones that created and measured that ECA and did external additional statistical analysis to validate it in ways far beyond the basic techniques of traditional analysis.

The FDA is charged with getting cures to market, faster than they have traditionally. That is the purpose of their reforms going on still, and the 20th Century Cures Act.

You repeat notions as if they are stuck in the 1950s and make them seem like contemporary regulation, and I get it. Competitors want their rivals to be stuck in the 1950s or at least for it to seem like they will be, while they sell their outdated drugs. That is the nature of the marketplace.

But the reality is, the market and the regulator are not going back and they are not going to waste the efforts and sacrifices of patients on the alter of formalism when the results are learned and powerfully validated right before them and patients and doctors are crying out for some more effort to advance a stagnant field of medicine in brain tumors with drugs that technically could already be available to patients and extending their lives and improving their quality of life substantially. They have no guaranty that other promising trials, as we have seen before will result in the same results provided here if outstanding survival data, both from the phase 3 and also compassionate use and side trials like the combination trial and trial with poly-ICLC.

It would be way out of the range of norms for that to happen, though I do believe they will kick the tires and possibly make some noise to satisfy the formalist/traditionalists who are just voices for their own treatments yet to be proven. That’s part of the dance they all take to affirm the old guard while moving the science and technique of accelerating the science forward.

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Guidance:

The guidance docs are often a work in progress and the FDA approves drugs with virtually zero proof of benefit quite frequently and to their shame, because there is a need and they know not much else is out there, for diseases like cancers, and especially rare diseases.

This one has incredible, credible proof from major researchers that are highly respected at top research universities worldwide. Looks like the UK will also validate it.

Guidance documents represent the Agency's current thinking on a particular subject. They do not create or confer any rights for or on any person and do not operate to bind FDA or the public. An alternative approach may be used if such approach satisfies the requirements of the applicable statute, regulations, or both.

https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs#:~:text=Guidance%20documents%20represent%20the%20Agency's,statute%2C%20regulations%2C%20or%20both.