I don't know if the U.S. FDA and Israeli authorities are in agreement with what the FDA here has been saying. I'm not saying they're already doing what they say, but as I understand it, the entire trial structure may be modified with the FDA and trial sponsor agreeing on the changes.
If I understand what's happening, a trial sponsor would reach an agreement with the FDA to initiate a trial, no doubt still escalating the dose until either MTD is seen, or a dose was determined to be optimum for further studies. During this period, mods could be made to the trial to for instance mitigate things like headaches, diarrhea, etc that could cause MTD to be prematurely called without these trial modifications. At that point, instead of initiating Phase 2, with FDA concurrence the trial could just evolve into what would typically be considered a Phase 2. Again as more information came in, working with the FDA it could become a Pivotal Trial, normally referred to as Phase 3, but in this case, it would evolve into it, and everyone involved could be shown in consideration of the effectiveness of the drug. As the trial evolved, it might be decided to go a variety of ways, perhaps target a variety of diseases, rather than just one, but it would all be considered one trial. If the FDA was satisfied with the data for one direction the trial had taken, approval would be granted while other directions continued in working to expand the drug label.
I cannot say this will truly occur, but if it does, I believe it can greatly simplify the process, reduce cost and time to approvals primarily because the FDA will essentially remain engaged in the trial process rather then involvement only at certain pre established times.
Gary
If I understand what's happening, a trial sponsor would reach an agreement with the FDA to initiate a trial, no doubt still escalating the dose until either MTD is seen, or a dose was determined to be optimum for further studies. During this period, mods could be made to the trial to for instance mitigate things like headaches, diarrhea, etc that could cause MTD to be prematurely called without these trial modifications. At that point, instead of initiating Phase 2, with FDA concurrence the trial could just evolve into what would typically be considered a Phase 2. Again as more information came in, working with the FDA it could become a Pivotal Trial, normally referred to as Phase 3, but in this case, it would evolve into it, and everyone involved could be shown in consideration of the effectiveness of the drug. As the trial evolved, it might be decided to go a variety of ways, perhaps target a variety of diseases, rather than just one, but it would all be considered one trial. If the FDA was satisfied with the data for one direction the trial had taken, approval would be granted while other directions continued in working to expand the drug label.
I cannot say this will truly occur, but if it does, I believe it can greatly simplify the process, reduce cost and time to approvals primarily because the FDA will essentially remain engaged in the trial process rather then involvement only at certain pre established times.
Gary
