Replies to post #770276 on NorthWest Biotherapeutics Inc (NWBO)

[Galzus Research]

DCVax-L’s statistical analysis plan (SAP) — using RWD-based ECA — has already been peer-reviewed, reviewed by the MHRA, and discussed in CHM. The MHRA has also completed site inspections, issued RFIs, received responses, and granted full GMP + procurement licensing to Advent.

While it's true that the SAP was approved, it is not accurate to say that the ECA was based on real-world data or included any.

All of the external control patient data were collected from other clinical trials. Hence, not fitting the definition of "real world" laid out in the very documents you cite.

This documentation's timing is coincidental, not illustrative. It has nothing to do with the DCVax trial, because the DCVax trial did not use real-world data. It certainly did not use real-world data in a prospective way from the outset, which is what the guidance is intending to guide on.

[Doc logic]

dennisdave,

Because what you describe is a typical cover move it certainly reaches high up into being a likely possibility. I believe May 10th 2022 was a similar catch 22 scenario where FDA guidance had not been changed in time to allow a change on the clinical trials site to OS from PFS without announcing the change and statistical analysis plan to peers first as part of garnering peer support for the change. This is actually the process necessary for following the exceptions to adequate and well controlled studies which requires an acceptance by peers of the need for the change and adoption of the new methods utilized to adequately and properly measure treatment effect. Best wishes.

[CaptainObvious]

So, will that be one of the new angles of attack, once approval is gained? Evil, like rust, never sleeps.

[attilathehunt]

So why launch a consultation now?
Because this is not a scientific need. It’s regulatory cover. The MHRA is likely preparing to approve a treatment without a traditional randomized control arm — and wants procedural insulation from criticism. They’re building a paper trail that says: “We asked the scientific community first.”

And most likely, in the future, they will be many other submissions with RWE and thus want to establish a CYA process.

I wonder where the clock stands on this? Is it on? Or is it off?

If what you state is true, then August 30% / September 70% is the likely decision period. Good thing I am patient...I hope my patience outlast my aging mind and body!

The Happenings....See you in September!!!

Have a good time....But remember. There is danger (fudsters) in the summer moon above...

[Lykiri]

So why launch a consultation now?
Because this is not a scientific need. It’s regulatory cover. The MHRA is likely preparing to approve a treatment without a traditional randomized control arm — and wants procedural insulation from criticism. They’re building a paper trail that says: “We asked the scientific community first.”

I don’t believe the MHRA is waiting for the outcome of its May 2025 consultation on the draft guideline ‘MHRA draft guideline on the use of external control arms based on real-world data to support regulatory decisions’ before approving DCVax-L.
On 31 December 2024, they approved tarlatamab (Imdylltra) for adult patients with extensive-stage small cell lung cancer (SCLC) after at least two prior lines of therapy, based on a Phase 2, open-label, dose-ranging study (DeLLphi-301) with no control arm. The approval was supported by a 40% objective response rate in the 10 mg group. No formal RWD framework or consultation preceded that decision.

https://www.gov.uk/government/news/tarlatamab-approved-to-treat-adult-patients-with-small-cell-lung-cancer