BETA - How can real world evidence lead to FDA label changes?

How can real-world data lead to FDA label changes? So this is the holy grail of what I want to see with my research career, is that using some of these projects that are very near and dear to my heart to actually change how the FDA approves things. That's a much higher bar to set, right? Because the FDA, obviously, they are very interested in patient safety. They are very interested in well-run clinical trials that answer that question. So do I expect real-world data to change FDA practices tomorrow? I don't think so. But as I alluded to earlier, one good example is the question of bortezomib, the drug Valkade, where again, twice weekly bortezomib, Valkade was used in clinical trials and it continues to be used in clinical trials just by inertia, just because the old study did it, therefore this study does it, therefore this study does it, even though that means more neuropathy for patients, even though that means more time in clinic, it's like a lose, lose, lose situation for patients. And so if the statistically pure way to fix this would be to do a randomized trial of once weekly versus twice weekly dosing and prove in a randomized study that twice weekly bortezomib is worse, I wouldn't feel comfortable with that study. My patient would not feel comfortable with that study because I already know that twice weekly bortezomib is worse. And so we are actually going and we're going to be in the coming year working with the FDA hopefully to show that maybe this is a good decision part. We have many, many and the biggest real world data and co-first author on those published two months ago in Blood Cancer Journal, a big journal, open access, Dr. Fiki Hoff is the first author on it, Dr. Kaur from UT Health is the senior author on it. You're welcome to look at it. Real world data showing that once weekly bortezomib is a standard of care used by most physicians and it works just as well as twice weekly with fewer side effects. We're going to take it up to the FDA to say hopefully for future trials the FDA should allow future trials to use once weekly bortezomib and then if the FDA allows it in the trial that's what they will allow in the label and the actual package insert once it's approved. I will say for rarer disorders, so very rare disorders like POMs or amyloidosis, we still tend to prefer randomized studies and oncology we often aren't able to do randomized studies, we are, but in some other domains like very rare, you know I can think of an example of very rare neurologic disorders where sometimes it's not ethical to give someone, you know to do a randomized trial where someone gets nothing or it's not even practical to do that right. Every patient coming to you they're so rare we have to put them onto something and so the FDA has said that real world control arms, so looking historically at patients who were treated before the drug became available and how they did is a reasonable strategy to pursue. Is it the best kind of data? No. But is you know are some data better than no data? Yes. So I think that not so much in oncology there are scenarios where the FDA may consider real world data or what's called a synthetic control arm where it's actually designed from real world data kind of designed to kind of match the patients who were on the trial. For multiple myeloma and for cancers I don't see that being something that the FDA or any of us will adopt tomorrow, however like I said in terms of changing how an existing drug is used that's where real world data may be very helpful.