How can real world data be used to guide treatment decision making?

As a myeloma doctor. Doctor Banerjee uses examples of real world data from multiple myeloma. However, this information on real world data can apply to all blood cancers.

How can real world data be used to guide treatment decision making?

Real world data can be very helpful to guide treatment decision making in terms of for drugs that are already approved, how can we use them better. So several examples of this that I can think of for patient with myeloma.

Many of you know the drug bortezomib or Velcade which can cause neuropathy. So that's a good example that real world data shows that once per week dosing is works just as well as twice per week the dosing with fewer side effects.

Another example from my research that I've done actually with SWA, which is the cooperative group that was trial data, but we've been looking at in the real world setting as well, is that dexamethasone dosing.

So patients who have lower dexamethasone dosing do just as fine. That would be a good example where, is bortezomib a new drug in myeloma. Nope. Is dexamethasone a new drug in myeloma. No. But what real world data is doing is saying, look, now that we know that these drugs exist, and now that we know that there's heterogeneity in practice because some doctors use twice weekly, some used once weekly for dexamethasone, some are on 40mg weekly, some 20mg weekly.

We actually kind of embrace the heterogeneity in myeloma treatment. To actually identify this is the best path based on all these different treatment paradigms that are out there. This one is associated with the best outcomes and the fewer side effects. And so that's an example of real world data sharing, real world evidence that I would then take back to my patient in the next clinic room and say, well, we now know that dexamethasone 40mg forever is not necessary. So let's lower your dexamethasone or let's make your bortezomib lower, or let's stop that bortezomib. Stories like that.

Can real world data be used to lead to cures?

First part of the question is, you know, how do you define cure? It sounds really simple. That'll cure me so the myeloma is gone, I would argue that it would mean the myeloma is gone, but you're also able to live your day to day life without thinking about the myeloma in terms of toxicities from the myeloma itself, side effects from the myeloma, or side effects from the treatment that you've received for the myeloma.

So I think one good example of where real world data are very, very helpful is in terms of improving how we deliver care to minimize side effects. You're right that we're not going to... retrospective real world data, looking back is not going to discovered a cure for myeloma necessarily, but it'll mean that when we get to that cure with DNA based phase one studies of new drugs, we're able to do it in a better way for more patients with fewer side effects.

A good example of that would be the drug bortezomib that many of you know as, as Velcade. So Velcade was FDA approved almost over 25 years ago I think now. It has a lot of history for it. It is a proteasome inhibitor, is a medication that works quite well against myeloma. It has been shown to improve outcomes in myeloma, not cure, but dramatically improve length of remission in several randomized trials. It's big side effect is neuropathy.

So a good example of how real world data I think right here right now is changing how we care for our patients is historically bortezomib, this drug velcade, was dosed twice per week Monday, Thursday, Monday, Thursday or Tuesday, Friday, Tuesday, Friday.

That's just how the trials were designed. You know, again, 25 years ago, over two decades ago.

And it's kind of stuck that way in all the trials since then. So all these new trials, looking at all these cutting edge new therapies, you know, for example, venetoclax which is a precision medicine drug, and the Bellini study, the bortezomib that the new wonder drug was combined with was still given twice per week, even though we found with real world data that twice weekly velcade twice weekly bortezomib has higher risk of neuropathy, a higher risk of tingling in the fingers and toes that can be quite painful, quite detrimental to quality of life compared to once weekly bortezomib.

So myself and my colleague Doctor Gurbakhash Kaur from UT Southwestern have really been working on this, using actually real world data, where in two forms one, we actually surveyed physicians about how they dose bortezomib and what they see is happening. And physicians overwhelmingly prefer once per week bortezomib. And then we actually use real world data from a health source called Flatiron that looked at over 2500 patients and looked at them.

And there we found that once weekly, bortezomib patients who received once weekly bortezomib did equally as well as twice weekly bortezomib and required fewer medications for neuropathy, fewer pain medications, fewer visits to neurology, and so forth.

And that would be a good example where real world data is very meaningfully improving outcomes for our patients because we're taking those data to say, look, once weekly dosing does just as well for patients.

Fewer side effects. This should be the standard of care, not just for, you know, patients who I see in practice, but for future clinical trials as well. But we're taking this to the FDA. We're taking this the drug companies, we're saying, look, look at our data here. We understand that 20 years ago, the trials used to use twice per weekly bortezomib.

But with this real world data, we know that once weekly bortezomib is better for our patients. Our loyalty as physicians is not to the trial protocol 20 years ago, it's to our patients. And so once weekly bortezomib is the way to go. So I think that's a long winded answer of saying that.

Do I expect the real world data to lead to the cure for myeloma? No, I don't want to overstate that. I think we absolutely need prospective clinical trials, new substances, new investigational antibodies and bispecific therapies and Car-T and so forth are all wonderful tools to have. But all those tools are given with our traditional therapies. And how do we optimize how our traditional therapies are given? Real world data.