I may be wrong, but I believe that the company can define the size and duration of Phase 2 Trials they'd like to run, as long as the trial can't be used for approval. If a trial could possibly used for FDA approval, the FDA would establish the criteria, but Phase 2 Trials in general are intended to learn more about the drug, and I believe the trial can properly be included in the Clinical Trials database as defined by the company. I believe the IRB will have far more to say about precisely how the trial is to be run.
In the case of the Safety Trial, blood draws were required every 12 hours. If in fact these draws showed nothing to be concerned about, I would hope that in a small efficacy trial, blood draws, if needed at all, would be limited to perhaps once a week. In a really small trial, I believe the trial could be open, no control group, and perhaps just weekly observations of how the cream was working on a particular application, like acne. Photo's could easily document progress, and the entire duration might be just one to two months of treatment. If we had 20 patients in the trial, each would represent 5%, so a substantial improvement noted in 15 of the patients would suggest 75% efficacy. Such a trial wouldn't cost that much, or take that much time, but it would be up to the IRB to agree to such terms. If the IRB believed that daily blood tests and observations were required, the cost of the trial could go up dramatically.
In the case of the cream, I'm uncertain if Mediq or OWCP is the sponsor of the trial. It's not impossible that Mediq is paying for this effort in exchange for higher royalty percentages than if OWCP were paying for it.
I believe it's up to OWCP to tell us more. A quarterly report is due no later than the middle of August, they could wait that long, but hopefully they won't. I don't believe that anything can really start until the safety trial ends, and hopefully that will happen very soon. I would hope they announce the completion of the safety trial, and that they discuss whether any concerns must be considered in the use of the cream, and the design of follow on trials.
For instance, the safety trials required two blood draws a day. If the results of the blood draws showed nothing to be concerned about, I would hope that a Phase 2 Trial could be developed that had few, if any blood draws, greatly reducing the cost and ease of being a patient in the trial.
To me, personally applying the cream a specified number of times a day with weekly visits with a clinician, probably a dermatologist, should be all that's needed. I believe in a few weeks of observations, a dermatologist should easily be able to say a condition is improving in a quantifiable way. This is far less than the FDA would require for drug approval, but all that should be needed to market the cream and indicate it's been found effective in Phase 2 Trials. I don't know the IRB would accept this as rigorous enough, and that's the key to how such trials are designed. The safety trial to my knowledge is being done at Sheba Hospital, the Phase 2 as I'm suggesting it could be done by many dermatologists from their offices everywhere, as long as they were trained to make clinical observations. I'm not saying it will be that simple, just that it could be.
Gary
