Some food for thought:
Dr. Nicole Verdun mentioned regulatory flexibility in relation to rare diseases in the Alliance for Regenerative Medicine interview. The guidance below on benefit-risk assessment at the FDA for drug approval was updated in October 2023:
https://www.fda.gov/media/152544/download
See p.12 for a reference to the term “regulatory flexibility” in relation to rare diseases - you can see that what Dr. Verdun was thinking of was likely this larger question or context of the Impact of Uncertainty on Benefit-Risk Assessment on pp.11-12.
See also an FDA zoom “Townhall” (as part of a series) on Cell Therapy Cell Therapy Chemistry, Manufacturing and Controls, in which Matthew Klinker, Head of Branch 2 of the Cell Therapy area who reports to Dr. Verdun, was asked a number of very specific questions starting from the 24 minute mark. At the 24 minute mark, he said “We know it’s hard” in relation to potency assays.
Finally, from the 21 minute mark, this video on the BMT CTN website by Dr. Levine on Transforming GVHD treatment talks about the use of biomarkers in the treatment. Their treasure trove of biomarker data from hospitals around the world that have been gathered by the MAGIC Consortium would be excellent input for the adult trial. (The conflict of interest declaration by Dr. Levine is also interesting as far as his biomarker tool which is patented is concerned - he received research support from both Novartis and Incyte. I think of this as extremely interesting. He would be fully aware of the limitations of Rux and despite his (previous?) support from the two companies, he has decided to go ahead with the adult trial for MSB.)
https://youtu.be/n0kgwt6062k?si=zgtcT2t1tkdRTtHG
The ball is of course in the company’s court to come up with data which satisfy the FDA but all this is I think, very informative as to the macro backdrop.
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