BB, while I've heard of what an effort it is I've never been involved in any way. Frankly I think it's ridiculous. I believe all that paperwork is to take the place of the trust that should be established between the regulators and the drug developers. I really believe the regulators should be more involved in the trial process, so greater trust is established there and the regulators trust the results they're seeing.
Companies who no doubt sent a ton of paperwork have clearly been known to hide flaws in their drugs. I believe that possibility would be eliminated if all approvals were followed with Phase 4's in which every use of the newly approved product was reported, and all problems noted. When the regulators were satisfied that no problem exists with the product they could remove this requirement.
A further benefit of such a trial would be the fact that off label use would be documented and others looking for possibilities could find the off label uses, and note if benefits seemed to have occurred. Let's say DCVax-L were approved for GBM but someone tried it for breast cancer, it would be so noted, and if the patient appeared to improve after using the vaccine, that would also be noted.
We all know that one or a few uses don't come close to a trial, but as anecdotal evidence builds, it's very possible that even insurance companies will go for it's use. In my treatment for leukemia, many of the drugs I used have not been specifically approved for the form I was fighting, but all have been accepted for insurance coverage. I believe this is becoming more accepted all the time, especially for a variety of cancers.
Perhaps the greatest beauty of our vaccine is the fact that it has practically no negative side effects, that's something that can not be said for many products in oncology. That, and the fact that all evidence to date indicates that it doesn't diminish the effects of other products, it either enhances them or has no effect. I suspect that this will result in a lot of off label use. At first it may only be from wealthier patients in search of benefit, but as anecdotal evidence increases, insurance and perhaps fund me campaigns will get the vaccine to more patients. We know it won't be cheap, but no cancer treatment is. I believe that if our vaccine is ultimately leading to cures of even a small percentage of those who would not be cured otherwise, it could actually save insurance companies money.
I believe the key to off label use is Doctors who believe in the possibility that something will work. Doctors in places like City of Hope, where I was treated, will fight to use the products that they believe will benefit their patients, and in some cases I don't doubt that the organization will cover the cost if insurance won't. I know at COH they make personalized drugs all the time, I suspect much of it is covered by donations that permit them to do the research they do routinely there.
Gary
AI
