$MESO Started Phase 2/3 Covid ARDS trial 3 days ago with 6 locations now expanded to 14!, page-65

The trial will only be stopped early, if it is deemed unethical to proceed with a placebo cohort. Due to overwhelming survival benefit in the remestemcel cohort. This is a big call to make as essentially any future trials regarding remestemcel efficacy for ARDS with a placebo cohort, would not pass an ethics committee. The P value may have to be P<0.01, ie less than a 1% chance the increased survival in the remestemcel group is due to random chance for the trial to be stopped early. If there is a survival benefit with a p value less than 0.05 (ie less than a 5% chance survival benefit is due to random chance), but greater than 0.01. The trial may continue, but remestemcel approval for on label use would likely be expedited by the FDA and immediate off label use expanded. The examiner for the trial would at this stage monitor results on a daily basis, and if the p value of the survival benefit with remestemcel went below a certain trigger level (I have used P<0.01), then the trial would immediately be stopped due to overwhelming efficacy.

The off label and on label use in the USA, has implications for subsidised use. Insurance companies will not fund off label, use but will fund on label use in the majority of cases.

From a medical perspective. I would like to see the FDA rapid approve remestemcel for use in paediatric graft versus host disease. As if remestemcel is shown to be safe to use in the paediatric population, it could possibly be of benefit in this Kawasaki like illness.This illness is probably secondary to covid 19 and likely has an autoimmune trigger.