Replies to post #766944 on NorthWest Biotherapeutics Inc (NWBO)

[Galzus Research]

Doc, what biomarkers do you think would make for a relevant go/no-go here? Certainly none that have come up to date that are remotely predictive for DCVax.

If this is what it seems like, I’d imagine the “case by case” review would amount to “hey, this patient has GBM and likely dismal outcomes.” The cancer itself in this case would be the biomarker.

Hopefully this is all the sign of real movement from the regulators.

[exwannabe]

This is not “approval” it is conditional access. There is a big difference because of case by case review. Those who are considered to be best suited candidates will receive a chance for greater access. This does look like an expansion of indications that can apply for funding but each indication will likely be reviewed for certain biomarkers that indicate a greater probability of successful outcome so that funding produces the most benefit per £ spent. Kind of a rational way to approach healthcare and yes this is a big deal; ). Best wishes.

No, it is neither.

What it says is that the NHE may elect to fund approved drugs on a patient by patient basis outside of any funding provided by reason of a NICE opinion, CDF or IDF.

They make clear that this path is an exception as otherwise they are "line jumping" (my term) the others in the system who only get funded per the general funds.

And it would not fund off-label (unless cheaper than the alternative).

[beartrap12]

Yes, I understand, Doc. GGB and I meant, by saying "this is approval", that MHRA would not have put DCVax-L /Murcidencel on their list of high-cost drugs if they didn't intend to approve it. In fact, I assumed it had to be listed as conditional and limited to compassionate care cases because we are not yet approved.
But to be fair, we should have said, "this means approval".. IMO
Thanks for the correction on word usage.

[Arby2000]

Doc, could this mean that it is available for IFR for the broader indication and MHRA MAA approval will commission it for n/rGBM? 

[GoodGuyBill]

The debate over whether DCVax-L’s inclusion on the NHS High Cost Drug (HCD) list constitutes “conditional access,” “conditional approval,” or “approval, though limited via IFR” is, at its core, a matter of semantics.

The reality is simple: DCVax-L has been approved, albeit with access restricted via Individual Funding Requests (IFR). This description most accurately reflects the situation—it acknowledges that the NHS has formally validated DCVax-L’s safety, efficacy, and therapeutic value in treating newly diagnosed and recurrent brain cancers, and possibly even all tumor-based cancers, while also recognizing that this approval, at the moment, comes with case-by-case limitations under IFR.

Regardless of how one chooses to phrase it, one fact remains undeniable, unassailable, and beyond reproach—by adding DCVax-L to the HCD list, NHS England has cemented its legitimacy as a valuable cancer treatment.

As for the persistent naysayers—those who have knowingly or unknowingly spread FUD about trial data, accused NWBO of cherry-picking results, or claimed the trial was a failure—their arguments have now been effectively discredited. The evidence speaks for itself, and their narrative no longer holds weight.

[EMHLondonUK]

Yes to clarify one more thing: the NHS is *not* the FDA. The MHRA is the closest equivalent of the FDA. This part of the NHS is closest to Medicare’s CMMS This institution decides what part of the England National Health Sevice will allow be used to sometimes treat and reimbursed by government funds.

Now you say, why would NHS England decide to pay for and use something that MHRA hasn’t approved? Well,
1. To provide access to experimental treatments that are essential to treating effectively life limiting disease where there are few good other treatments (EAMS)
2. To front run the MHRA when their process is just slow approving something that other regulators have already signed off on in other countries
3. To allow pharma companies to offer free of charge medicines in pre-commissioning before MHRA or NICE decisions are complete (FOC)

My guess is we got in on EAMS originally (this was the old entry thaf was deleted that was GBM-only). My guess is that they have enough confidence that uk clinicians are or will be asking to use the treatment - and use it in cancers beyond GBM - that they updated the entry, changed to the new INN name, and updated indications all cancers.

I’ve never seen the direct interplay between this body and MHRA. It’s even more uncertain now given the turmoil in NHS England with the PM’s restructuring. However, speculating, I can’t imagine they’d do all the work on the HCL entries to have the MHRA just reject the MAA in the coming days/weeks/months.