Replies to post #366197 on NorthWest Biotherapeutics Inc (NWBO)

[biosectinvestor]

Actually, the FDA changed their guidance specifically to address this if you recall, and they did so really, across the board in many ways because of the 20th Century Cures Act, which mandated that they address these kinds of therapies differently and with more flexibility.now companies have proposed using external placebos from the start of their trial, as I recall. I do not think it was the Germans that paved the way here.

I'm a big believer that historical data ought to be what experimental therapeutics work against, I know the regulators don't agree, but in this case, I think the German's convinced the others it was wrong not to dose all patients, and that's what essentially the last third of the patients received.

[Evaluate]

you wrote:

People who are willing to take experimental treatment should get that treatment, the history of their diseases is generally well known and if the experimental drug does just a little better than the SOC and that's caused by a placebo effect, what is the harm in putting the additional drug in the Doctor's tool kit.

Taking into account the incredibly high cost of some of the approved drugs nowadays, I feel that it might not be financially possible to approve drugs if they are only "a little bit better than SOC". If the bar was "set that low" perhaps many, many drugs would qualify as being "just a tiny bit better" than SOC and still cost $200,000 per patient per year? This would not be affordable. Instead, I think that if treatments are going to cost that much (or: that much more than just SOC), then they should provide a substantial improvement. In the case of DCVax-L it is being tested as an "add-on" to SOC, so the cost of getting treated with DCVax-L would be in addition to the existing cost of SOC treatment.
I have the impression that the drug review and approval process in the UK takes this into account with their QALY calculations etc. The quality-adjusted life-year (QALY) is a measure of the value of health outcomes. Since health is a function of length of life and quality of life, the QALY was developed as an attempt to combine the value of these attributes into a single index number. QALYs can then be incorporated with medical costs to arrive at a final common denominator of cost/QALY. This parameter can be used to compare the cost-effectiveness of any treatment.
Nevertheless, QALYs have been criticised on technical and ethical grounds.
Back to DCVax-L: it would not surprise me if the RA's decide to approve DCVax-L only for those prospective patients that have certain attributes that make them more likely to have significant improvement of PFS and/or OS ... and perhaps not approve its use for those patients who may only have relatively low odds of merely some minor improvements of PFS and/or OS.

[GoodGuyBill]

Your view is also consistent with LP's comment around the time this occurred, about DCVax naysayers making good news appear to be bad.

I think the German's convinced the others it was wrong not to dose all patients, and that's what essentially the last third of the patients received. If that's right, how can they deny an approval that's based on comparing the SOC with the experimental therapeutic that's been added to it.