Tappy,
The orphan drug designation may lead to a smaller Pivotal trial, but if we're speaking about our FDA, from initiation of Phase 1 trials to completion of a Pivotal trial can hardly be done in under about 5 years. I would suspect that gaining approval in Israel, or somewhere else could be much quicker, and then documented presented to the FDA could result in a fairly fast Pivotal trial here.
The thing that takes so much time with our FDA is their review process, they will spend half a year working on the design of a trial, then you submit an IND, and they get 90 days as I remember it to accept it, then you have an IRB who'll usually spend another 3 to 6 months to design the ultimate trial protocol before you can even start the trial, which is only Phase 1. After you have Phase 1 results, normally in about a year or more, you determine where to go next. If the Phase 1 is designated Phase 1/2, you can determine the most effective dosing level in the Phase 1 portion, then dose more patients in the Phase 2 portion and data from that may be sufficient to skip a larger Phase 2 trial and go immediately to Phase 3. The point is, from initial discussions with the FDA until you've actually finished this, 2 to 3 years is probably pretty fast.
The Phase 3, or possibly a registrational Phase 2 should be designed with consultation with the FDA, again probably no less than 6 months, filing an IND, again 90 days, IRB review 3 to 6 months, so it's again about a year before the trial can even start. Most Phase 3 trials take a few years and even with orphan drug designation I doubt it can be done in under 18 months. Then you must prepare an NDA, which normally takes months, and after submission the FDA will have 6 months to review it. Occasionally the FDA will judge the product in slightly under 6 months, but normally you get the decision at precisely the 6 month point, and if they ask a question, or request something further, when you provide the information, they get 6 months more to consider it.
In short, what people believe should take a couple years actually takes 5 to 10 years in nearly all cases.
If I understand what OWCP is intending for MM, I believe it's largely what will be in at least one form of the sublingual tablet. If I'm correct about that, and the tablet is available, outside the trial, some patients with MM will no doubt purchase the pills and we may get anecdotal evidence of its efficacy well before trial results are available.
IMHO the company should initiate trials elsewhere, probably in Israel, and demonstrate efficacy there. Then with some efficacy demonstrated, the trial process for approval here could be expedited, but more importantly, if the sublingual tablets were available here, patients could see benefits before FDA approval was ever granted, and with sufficient anecdotal evidence, Insurance companies could be forced to pay for the tablets.
I don't remember the specifics, but I did read about a Court in one State ruling that an Insurance Co. had to pay for a cannabis based treatment that was proven effective in the patient. So the State can rule that Insurance must pay for something which the Federal Govt. says is illegal. It's time we got our head on straight.
Gary
The orphan drug designation may lead to a smaller Pivotal trial, but if we're speaking about our FDA, from initiation of Phase 1 trials to completion of a Pivotal trial can hardly be done in under about 5 years. I would suspect that gaining approval in Israel, or somewhere else could be much quicker, and then documented presented to the FDA could result in a fairly fast Pivotal trial here.
The thing that takes so much time with our FDA is their review process, they will spend half a year working on the design of a trial, then you submit an IND, and they get 90 days as I remember it to accept it, then you have an IRB who'll usually spend another 3 to 6 months to design the ultimate trial protocol before you can even start the trial, which is only Phase 1. After you have Phase 1 results, normally in about a year or more, you determine where to go next. If the Phase 1 is designated Phase 1/2, you can determine the most effective dosing level in the Phase 1 portion, then dose more patients in the Phase 2 portion and data from that may be sufficient to skip a larger Phase 2 trial and go immediately to Phase 3. The point is, from initial discussions with the FDA until you've actually finished this, 2 to 3 years is probably pretty fast.
The Phase 3, or possibly a registrational Phase 2 should be designed with consultation with the FDA, again probably no less than 6 months, filing an IND, again 90 days, IRB review 3 to 6 months, so it's again about a year before the trial can even start. Most Phase 3 trials take a few years and even with orphan drug designation I doubt it can be done in under 18 months. Then you must prepare an NDA, which normally takes months, and after submission the FDA will have 6 months to review it. Occasionally the FDA will judge the product in slightly under 6 months, but normally you get the decision at precisely the 6 month point, and if they ask a question, or request something further, when you provide the information, they get 6 months more to consider it.
In short, what people believe should take a couple years actually takes 5 to 10 years in nearly all cases.
If I understand what OWCP is intending for MM, I believe it's largely what will be in at least one form of the sublingual tablet. If I'm correct about that, and the tablet is available, outside the trial, some patients with MM will no doubt purchase the pills and we may get anecdotal evidence of its efficacy well before trial results are available.
IMHO the company should initiate trials elsewhere, probably in Israel, and demonstrate efficacy there. Then with some efficacy demonstrated, the trial process for approval here could be expedited, but more importantly, if the sublingual tablets were available here, patients could see benefits before FDA approval was ever granted, and with sufficient anecdotal evidence, Insurance companies could be forced to pay for the tablets.
I don't remember the specifics, but I did read about a Court in one State ruling that an Insurance Co. had to pay for a cannabis based treatment that was proven effective in the patient. So the State can rule that Insurance must pay for something which the Federal Govt. says is illegal. It's time we got our head on straight.
Gary
