Replies to post #719544 on NorthWest Biotherapeutics Inc (NWBO)

[sentiment_stocks]

Interesting… from your link:

The Alliance for Regenerative Medicine (ARM) explained in their comment that use of external controls may not be feasible for some rare diseases.

“Some of the guidance on the use of retrospective natural history studies as a control arm could be challenging for treatments for diseases with small populations, as is the case for many of the diseases that cell and gene therapies would be able to treat,” they explained. “These historical control groups are critical for evaluating gene and cell therapy products in patient populations that heretofore had no effective therapies and would now have the potential to receive a product that would prevent disease progression or provide a cure rather than symptomatic treatment benefits.”

And while that may be the case for some rare diseases, the SAP from the DCVax trial for the very rare disease of GBM was able to assemble a fairly large external control arm using concurrent historical data.

Still, the fact that the DCVax-L ECA did not offer patient level data could represent a problem, but yet what I presented is that there is another guidance that indicates it might not be.