What are challenges associated with using real world data?

What challenges are associated with using real world data?

When people hear the word research, right, they tend to assume that it's an investigational drug. They tend to think when they hear the word research, they think, oh, it's a new drug. We're testing it. We don't know if it works or not. We don't know if it's safe or not. We don't know if that actually makes sense for patients or not. That indeed is research. But research is so much more than that.

That particular bucket of investigational trials, new trials, that is an important part of research and most work happening in cancer centers does focus on that kind of research. What we call phase one trials, phase two trials, phase three trials. Those are all prospective, meaning that we follow patients, we enroll them in the trial and follow them prospectively.

Not all prospective trials are interventional or pharmacologic. For example, you know, my, when I was a fellow, when I was a trainee, the big project that got me interested in myeloma to begin with was Digital Life Coaching during transplant for myeloma. That’s a good example of research where we actually, you know, randomly assigned patients receiving transplant to either get usual care with some handouts about how to stay well and how to feel well and rebuild our lives after transplant. Or we did that. Plus we gave them access to a life coach by phone or text who could help them with sleeping, with moving, with feeling just less stressed about returning to work after transplant.

That’s research too. Even though there are no drugs involved, nothing is changing about the transplant itself or the myeloma itself. That absolutely is prospective research. You know, I have colleagues, for example, Dr. Urvi Shah at Memorial Sloan-Kettering in New York who’s doing prospective research around gut microbiome, seeing how people’s gut bacteria changes over time and what that means for them in terms of myeloma precursor conditions.

So not all research needs to involve new drugs. Also, there can be trials, prospective trials of research, of drugs that are already approved using them in different ways. A good example of that with Car-T therapy, Car-T therapy is approved in the US. As of the day that I’m filming this April 5th. You know, the FDA has just approved one kind of Car-T for two prior lines and not four prior lines of therapy.

So patients earlier in the myeloma journey, that trial that led to that approval was an example of research, even though the drug is already approved, it’s not approved in that line of therapy. That’s also prospective research. Then, if you go big picture separately from prospective research, there are other kinds of research as well.

So there’s retrospective research. So, for example, that I alluded to earlier, if you go back and look at people’s medical records and they say look, for everyone diagnosed with myeloma in the year 2021, how did they do? Or if we look at their dexamethasone dosing or look at their acyclovir dosing, what changed about it? And that’s retrospective research, meaning that we already have a question in mind. We’re not going to enroll patients in a trial. We’re going to look back at what happened to these patients, you know, over the last two years, three years, etcetera. And make conclusions based on that retrospective research is not as strong as prospective research, obviously. Ideally, we should run trials for the big questions in myeloma. But as we’ll talk about, there are some cases about real world data, which often is retrospective by definition, is very useful in that regard.

And I would say there’s a third bucket of research, which is, you know, health services research or cross-sectional research. So I have also done work with HealthTree and with other organizations for survey based research. Survey research is kind of in the middle somewhere where it’s not really retrospective, because you are asking a patient to donate their time and physically answer these survey questions in front of you.

It’s not always prospective. We’re not always following them over time. Right now, for example, with HealthTree we have a survey ongoing of eye health with myeloma? Alluding to my earlier retrospective question with dexamethasone and eye complications of myeloma. This is a one time survey. Could a survey be research? Absolutely. Doesn’t have to be, right. If you’re surveyed about how was your experience with your doctor for a, you know, quality survey and what could be better? That’s also a survey, but that’s not research. When I say research, I mean that we’re using it to improve care or improve outcomes for our patient. So if I’m using that survey not just to make myself feel better or not just to make my practice better, but actually to make myeloma care paradigms better for patients, then it’s research.