It's been awhile since I saw the statistics, but when I did, roughly 30% of Phase 1 drugs were reaching approval. I really wish that all trials were initially designated as Phase 1/2 and if efficacy was demonstrated in the Phase 2 portion the trial sponsor would have options that included FDA participation in the Pivotal trials.
Essentially the drug developer could turn the pivotal trial over to the FDA completely, and the FDA would get a substantial royalty stream on approval. The could also share the costs, and different royalty streams would apply. The point is, over time this would fund the FDA to at least the point of it's expenses, and potentially much more, but it would also permit tiny biotech to bring products to market without BP, so when BP did get involved, they'd pay more for what they were getting.
This certainly would require seed money from the Govt, but in a matter of years it should be self sustaining. More importantly, with the FDA participating in the trial, they would see what's happening, and they could approve a drug far earlier in the process as they could see the benefits for themselves. Companies would with their Phase 1/2 data show the FDA that the pivotal trial is worth pursuing, but once convinced, the FDA would have a hand in the pivotal development, the company could determine what degree the FDA would be involved financially, but as long as it had even the smallest position, the FDA would be involved in the pivotal trial. I realize this in some ways makes the FDA the fox guarding the hen house, but I believe the FDA staff approving the drug could be others than those working in the clinic, so it would be up to those in the clinic to convince the others that it was worthy of approval.
I cannot say that Dr. Padzur changed dramatically, but I believe his wife was struck with cancer and it has made a change in some of his thinking. I really believe everything would change dramatically if some reasonable price could be established for drugs in pivotal trials under the right to try provisions. If drug makers could profit even slightly by this program, I believe a lot more experimental drugs would be used. Every use of the drug should be recorded, but I also favor all newly approved drugs going into a Phase 4 where all use is reported. Off label use would certainly be permitted under right to use, or Phase 4, but the outcome would be clear in the database.
I know my ideas aren't likely to occur, but you never know who might agree to see them and actually is in a position to do something.
Gary
