The FDA is a corrupt organization., page-46

It's pilot data from an emergency program.

So it appears we have been using Ryoncil in Adult salvage patients and Si is indicating a positive response.

What I do not understand is what possible signal/data revealed in Adults will lead to approval in pediatrics? @stanjupiter

My hallucination is that we commence a trial in Grade 4 adults, use ORR at 28 days or less- use early ORR trial data to get AA in kids. Continue adult trial to survival at 90/120 days .

FDA control - placebo - its salvage therapy , nothing left to throw at it

What's crazy is that Ryoncil is unapproved but being used in both kids and adults with FDA permission- what's going on in the USA?

I'm still punching


Reg

Emergency Exemption from Prospective IRB Approval

Emergency use is defined as the use of an investigational drug or biological product with a human subject in a life-threatening situation in which no standard acceptable treatment is available and in which there is not sufficient time to obtain IRB approval [21 CFR 56.102(d)]. The emergency use provision in the FDA regulations [21 CFR 56.104(c)] is an exemption from prior review and approval by the IRB. The exemption, which may not be used unless all of the conditions described in 21 CFR 56.102(d) exist, allows for one emergency use of a test article without prospective IRB review. FDA regulations require that any subsequent use of the investigational product at the institution have prospective IRB review and approval. FDA acknowledges, however, that it would be inappropriate to deny emergency treatment to a second individual if the only obstacle is that the IRB has not had sufficient time to convene a meeting to review the issue.

Life-threatening, for the purposes of section 56.102(d), includes the scope of both life-threatening and severely debilitating, as defined below.

• Life-threatening means diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted and diseases or conditions with potentially fatal outcomes, where the end point of clinical trial analysis is survival. The criteria for life-threatening do not require the condition to be immediately life-threatening or to immediately result in death. Rather, the subjects must be in a life-threatening situation requiring intervention before review at a convened meeting of the IRB is feasible.