Well, the issue you allude to is, of course, not unique to DCVax-L.
The same phenomenon applies to all the ICI's!
They do best in melanoma. But in their largest numerical market,NSCLC, they have the same (or smaller) minority of long surviving outliers. Along with a large percentage who derive little if any survival advantage.
Plus a small but significant percentage, who do worse than the previous SOC regime.
(Hyperprogression is real..)
They have, I would suggest, even greater variance of response. But that did not prevent NICE giving several green lights for various indications.
Therefore, I don't really see 'variance of response' as a real world impediment at all.
It's pretty much the nature of the game with immunotherapies in general.
(In an ideal world, you would have identified and validated predictive biomarkers telling you which patients will benefit with a high degree of probability.)
But, again in the case of the ICI's, no real solid predictive biomarker has been identified.
PD-L1 status is an indicator, but an imperfect one.
And understandably, companies are perhaps less than enthusiastic at the possibility of their treatment being restricted to just a fraction of patients being eligible.
NICE also undertake partition state analysis, involving a calculation of the duration of progression free survival. And we know that in the trial most of the 3yr survivors had reached that milestone without disease progression, and without debilitating treatment side-effects, thus preserving Q of L.
And even if NICE wants more real world evidence before finally deciding whether to fully ongoing fund a treatment, then they may recommend interim funding by the Cancer Drug Fund.
The CDF was still in existence last time I checked!
And Brain Cancer is one of those conditions with high public awareness. Just the sort of thing that the MHRA might make into a well-publicised marquee approval.
An MHRA that wants to do something to prove its autonomy, and responsiveness.
And because of the small numbers (but growing), it's a lot cheaper and 'sexier' than doing something about chronic disease (like Type 2 diabetes for example).
And that's why Funding pages for brain cancer patients do well.
And here is a current example (recently referenced on IHub) of a patient and family seeking DCVax-L treatment (under UK Specials).
And just the fact that the UK authorities have appeared quite happy for some time now, for patients to be treated with DCVax-L under UK Specials, tells me something.
If L achieves approval, I can assure you that 'take-up' won't be much of an issue.