Jim Joyce, CEO of AEMD, announced a few days ago on Youtube that a FDA IDE studies would be initiated this fall at Beth Israel in Manhattan for its HemoPurifier device in the treatment of HEP C. On 9-11-2012 in a presentation at MD Anderson Hospital in Texas, considered by many to be the premier cancer hospital in the world, he confirmed that AEMD would be submitting a FDA IDE in October.
One expert (purposely not quoted) is anticipating that trials will be abbreviated with perhaps as few as 10 patients to determine safety, and a score of more for P2 trials, before a definitive P3 study that could be completed within a year from now.
The question now simply is: If AEMD can submit a FDA IDE, why not CTSO, and if so, when? Will CTSO submit a FDA IDE after the dosing studies are presumably completed this year?
One of the concerns for CTSO may be what biomarkers to use. Unfortunately, there has been little specificity reported in the literature that would appear to help in defining control and treatment populations in a P3 study.
Although I agree generally with observations that P3 studies in the US for Cytosorb are years away, there is the possibility that the FDA would review and accept European studies to date including dosing studies just as the FDA has accepted preliminary information provided by AEMD.
Concerns that the patient populations in the initial EU Cytosorb trials were not sick enough and concerns that homogeneity of the study populations might be lacking cry out for a scoring system analogous to how acute pancreatitis (BISAP), trauma (ISS), head injury (Glasgow), memory impairment (MIS), or MOF (SOFA) are scored in severity. Many of the parameters, not necessarily standard biomarkers, would be obvious such as b.p., temperature, pulse rate, CVP pressures, white count, base deficit, age > 65, cytokine levels, and MOF indicators like creatinine, spirometry, and hepatic enzymes. So how sepsis should be scored will become an important question in answering similar concerns I suggest for the FDA in approving any final IDE studies for Cytosorb in the States..
In my opinion, SPA (or some designation like the SPA for IDEs) and Fast Track designations would be necessary. Because of the obvious variability in scoring severity that would almost certainly lead to questioning after studies were completed as to efficacy, a SPA-like designation seems mandatory. It takes time to negotiate a SPA.
My working assumption had been that there is no way CTSO could have the funds even through a sale of HemoDefend or sales of Cytosorb in Europe to fund P3 studies in the States initiated within a year. But now in view of the AEMD announcement, I am not so sure.
I would appreciate other IHub members’ remarks about my comments.