Below is an answer to “”What’s the difference between clinical...

Below is an answer to “”What’s the difference between clinical trials and registration trials, the latter has not been used before in any announcements, and it is available on appliedclinical trials online .com:

“While clinical trials focus on efficacy—the extent to which medical interventions achieve health improvements under ideal circumstances—registries provide strong evidence for the extent to which medical interventions achieve health improvements in real practice settings.”

It is an answer to Whytee’s comment that it is all about a randomised, controlled trial. No, it is not. Instead, it is about real world evidence, something increasingly leaned upon by the FDA in the last few years in approvals, that will support the adult label extension if and when the potency work is finished. Remember that the BLA is still an application for approval of the children’s aGVHD indication and so this work is still targeted to that BLA. The trial design for the label extension is the extra regulatory step or procedure discussed in the Type A meeting.

It is clear that the last few bullet points in the announcement are the same as what the company discussed in the latest annual results CC and confirmed in the Type A meeting. Whereas the company had likely hoped that the trial design for the label extension would be signed off in the meeting as well, the FDA wanted to see it first finish the final potency work to demonstrate the standardisation of the trial product and the inventory which will go into the trial.

In the fourth last bullet point in the announcement, the company expresses confidence that in doing so, it will be able to provide the link between the trial product and the inventory. Note this is the concept of “similar enough” introduced by Dr. Krause. It’s specifically applicable to and essential for donor stem cell products. We have gone past the proof of efficacy or efficaciousness, and the FDA is focused on the need for the BLA applicant to show that it is going to be able to sell and/or use in future trials a consistent, commercial product. This then lays the foundation for the next step, which is the label extension for not only the adult indication, but other things like IBD and lung ie. ARDS (note not limited to Covid related anymore).

To me, this is progress, not gilding the lily, that represents the last step in the children’s aGVHD BLA approval plus the adult label extension.

Patience is still needed though, panic not so much.