NorthWest Biotherapeutics Inc (NWBO)

[pgsd]

The FDA's position is very supportive of adaptive trial designs for rare diseases (GBM is an Orphan disease).

You might want to take note of the following including FDA guidance below. Note GBM is an orphan disease

Trial Design and Statistical Considerations in
Rare Disease Clinical Trials
[FDA - September 6, 2019)

Conclusions:
To overcome significant challenges in designing and conducting adequate and well controlled rare disease trials, we support innovative trial designs and analyses provided they are well thought through, justified, and able to

“distinguish the effect of a drug from other influences,
such as spontaneous change in the course of the disease,
placebo effect, or biased observation.”

Adaptive Designs and Orphan Diseases: Building a Bridge of Innovation

For example, the 2010 FDA guidance on adaptive study designs classified designs into “generally well-understood adaptive designs with valid approaches to implementation” or “adaptive study designs whose properties are less well understood”

https://www.worldwide.com/blog/2018/04/adaptive-designs-orphan-diseases-building-bridge-innovation/

GBM is the most aggressive and lethal form of brain cancer, and is an “orphan disease.”

https://nwbio.com/nw-bio-confirms-phase-iii-trial-of-dcvax-l-for-gbm-brain-cancer-is-ongoing/
Link to 'Trial Design and Statistical Considerations in
Rare Disease Clinical Trials' FDA

https://www.fda.gov/media/131882/download