Garth Sundem  00:00

Welcome to this episode of the Medical Affairs Professional Society Podcast Series: “Elevate”. I’m your host, Garth Sundem, Communications Director at MAPS. And today we’re speaking about the opportunities of real-world evidence with Kirstan Summers, Director of Client Solutions and Strategy at H1. This episode is sponsored by H1, whose platform helps life science companies, hospitals, academic medical centers, and health systems, connect with providers, find clinical research, locate industry experts, and benchmark their organizations. So, KIRSTAN maybe you could get us started by refreshing our audience’s memory of what we mean by real world evidence. Maybe we’ll start with the historical take, and then we’ll move to the present. But what is real world evidence as we understand it?

Kirstan Summers   00:57

Yeah, it’s a great question. And I think it’s changed over time, you know, real world that the real-world evidence of 10 years ago or even 15 years ago is very, very different than the real-world evidence of today. And it even varies based on the disease ontology that you’re focused on, right, real world evidence in oncology, for instance, has become not just a method to identify safety around certain therapies, but also, it’s become a means of diagnostic identification of appropriate patients, for more precision medicine. So, you know, I would say that real world evidence has developed very differently, not just as something that supports a more traditional randomized controlled trial, but also now as a new means for drug label extensions and approval. You know, I think it’s really interesting, just a couple of weeks ago, the US FDA gave a draft guidance around the use of real-world evidence. And this is all part of a larger drive to revolutionize healthcare delivery, drug development, speed of which, you know, paid therapies that are important to patients get from pipeline, you know, into the hands of the people that that really can benefit from them. And so, the interesting thing about the new guidance is that it actually gives specific direction around registries, fitness for regular regulatory decision making, based on attributes that would support collection of relevant and reliable data. And so, you know, the big deal here, I think, is that the FDA review of it really draws attention to the fact that they’re becoming open to registry data as part of submission packages and part of packages that provide for label extensions and things like that. It’s, it’s a huge deal. I think that will impact the speed of the approval process, and hopefully, you know, solve for some of the clinical inertia that that, that doesn’t make access to patients move as quickly as we’d like it to.

Garth Sundem  03:19

Okay, so there’s a lot to unpack there.

Kirstan Summers   03:22

It’s a really fun topic. It’s interesting, right? And you know, one other thing I’ll mention is our brand new FDA commissioner, Rob Califf is also a top expert. And these types of trials of real-world evidence is he’s a huge proponent of it, so I’m excited to see kind of where that goes with.

Garth Sundem  03:40

That’s interesting. Is he a HEOR guy or is he uh,

Kirstan Summers   03:45

He’s, he’s one of those people. He’s one of those. He’s a cardiologist by training. And he but he is an expert in health economics and outcomes research in, in drug development, also in regulatory decision making. He was, he was a commissioner already. And they brought him the Biden administration just brought him back. But the big thing about him is that he has driven a lot of the innovation around, he’s one of the godfathers really, I would call him of innovation in real world evidence and big data in healthcare. He actually went in and worked at Google verily, for a while, before he was pulled into this. So, he’s a data guy too. It’s pretty interesting.

Garth Sundem  04:29

Okay, so real world evidence used to be used for safety monitoring, you know, drug would be approved and watching the real world to see if bad things were happening. And now there are all these other uses. One thing you mentioned that I thought was really interesting is that the new FDA guidance looks at the appropriateness of registries. So, I’m sorry, are they somehow scoring the different massive data sets that are out there like giving the seer database, some sort of appropriateness, and giving different databases. So, and what I’m wondering for our audience is, should they be looking to certain sources of data and prioritizing those over others?

Kirstan Summers   05:12

Yeah, you know, because not all datasets are created equal. Not all, one of the things about big data is that there’s so much of it. The health systems and institutions don’t always collect it the same way. So that’s a tricky piece of it. The other part of it is that you know, when it when you’re looking at partnership, on real world evidence type work, certain institutions, just as though just as they’re known for certain disease area expertise, they are also known for certain areas of disease area expertise, also in real world evidence, which is a different kind of view. And, you know, the, the guidance really provides important information for like around supplemental data that needs to be included and looked at like rigor around medical claims data, electronic health records, digital health technologies that are being utilized for patient monitoring and engagement, and other, you know, connectivity with other registries and things like that. So where, you know, sometimes you’ve got, you’ve got institutions now that are data sharing across multiple institutions based on certain patient types and combining data and mining it to be able to identify certain, certain signals that you might want to see that can help inform patient management, patient health, all that sort of stuff.

Garth Sundem  06:44

Okay, well, let’s go to these data sources. So, if we take data as kind of a starting point, how does that inform our action? So, if we look at something like claims registry or other data source, what then can Medical Affairs teams do from this starting point?

Kirstan Summers   07:07

Yeah, I think it’s, it’s, I think data and looking at data is a really big and important clue. And it’s a big and important piece of rigor that you need before you decide who you’re going to approach where, when, and how for different types of real-world work. And that can be you know, we understand through diagnostic claims data, the institutions, even the people who are seeing lots and lots of certain patient type or certain patient demographic, we can also understand by other data points, such as publications around on this type of expertise. So, therapeutic area, clinical expertise is one thing and clinical development, clinical trial development expertise is, you know, a piece of knowledge as well. But identifying correct investigators and people to partner with understand very well and have demonstrated interest in these real-world type studies, is also a key and important piece of determining which institution you might partner with, which investigator you might approach. And, you know, often and really finding synergies between their goals and their patient management and overall patient health of their population. And what you’re trying to accomplish organizationally, just like you would really be choosy about who you partner with on a randomized control trial, you have to be really choosy the same thing the same way about, about real-world evidence. And then you even go broader sometimes that too, because often you’re looking at partnering with health systems, not just not just an institution, because health systems have lots and lots of data. But we also can understand easily to about health systems, the patient populations that they have. If for instance, it’s important to your organization to understand through means of real world data, certain patient groups, maybe it’s based on based on race or you wanting to look at a diverse patient population that’s underserved, you would be able to through claims data through understanding of, of the patient mix of a certain in either institution or health system, you might understand better how to how to position your work in a way that’s going to give you and that institution meaningful outputs.

Garth Sundem  09:48

Okay, so that’s interesting. So real world evidence within claims data or wherever you find it can be used to identify who and how to run a subsequent RWE study, can it also help Medical Affairs teams identify which studies to run?

Kirstan Summers   10:10

Yeah, I would say that, you know, that’s a tricky one, because identifying which studies to run is something that has to, has to fill, there’s usually a data gap that has to be filled, right. And so, organizations from a Medical Affairs perspective, and a lot of times, there’s health economics and outcomes research colleagues from within your organization that get involved in that too, to identify, Okay, we’ve had a randomized control trial, we have this evidence in this data now, which, you know, which data gaps are really are we trying to seek or fill by way of real-world evidence? Do we, do we, are we conducting real world evidence simply to be supportive of the data, the safety and efficacy data and our randomized control trial? Or are we running this real-world evidence research so that we can better understand specific disease manifestations and a particular patient population? Because we don’t, we didn’t look at that in random in our RCT. And also, we know there’s an unmet medical need there, and we want to learn more. So, I would say that it’s sort of an organizational identification first. And then secondly, where’s the best place to partner with to fill that data gap?

Garth Sundem  11:32

Okay, cool. So, the organization is identifying the gaps. Our RWE is teaching or giving us the opportunity to fill it.

Kirstan Summers   11:41

Absolutely. And, you know, the thing is, too, is because organizations partner a lot with a lot of different scientific experts to help them understand to where their data gaps are, where there’s need, all of that sort of stuff. When they’re conducting data gap analyses, it’s oftentimes beneficial to have the right types of thought leadership that you’re engaging with, for advisory boards to help inform these types of questions. So, you know, one of the things I think that is, and I’m going I’m going way back, like strategy around that kind of stuff. But what’s really I think, important for organizations to always include when they’re considering advisory boards, when they’re even further back in, in drug development, working with people who understand registries, real world evidence, and how to appropriately leverage data in ways that can help support regulatory submission packages, and stuff like that is critically important. It’s not just working with the people who are the known people to design clinical trials, or the known people who are known are experts in treatment of this particular disease, it’s not enough anymore, you have to be designing your clinical trials in a way that is either producing data sets that are appealing to payers and health systems, health systems, decision makers, and also even the FDA and building these things in as part of your submission packages.

Garth Sundem  13:19

Well, so you’re talking about the submission package and even label change. You know, it’s always been that, you know, you needed the RCT to get that, to get that label change or to get the, you know, submission package is that is that changing is RWE now a more important part of the label.

Kirstan Summers   13:38

Oh, it’s becoming, you know, oncology is actually a really great example of how our web has become a critical piece. And it’s, it’s starting to happen more in, in, in chronic conditions. But I would say with, you know, with oncology, it’s become this huge enabler to measure the benefits and risks associated with not only treatments and development, but understanding and utilizing data mining around genomic profiling, as a standard of care has made real world evidence sort of this tool for oncology for precision medicine. And so, they’re using really population level high quality, real world data to inform target identification, optimize their study designs, and deliver like in real time, insights and signals that help inform clinical practice. And this happening in a much more efficient way than with traditional label extension and approvals, by way of RCT.

Garth Sundem  14:45

Is there a danger with RWE in and this is a loaded phrase in moving fast and breaking things? Is there, is there, is there a danger in the speed of RWE?

Kirstan Summers   15:01

You know, it’s I think that that’s, that is, that’s a super loaded question. And it’s not a question that I think is my question to answer. I think that’s a question that the scientific community is working really, really hard to answer. The other piece of it, though, I think that’s when it’s done, right. And when there with, with the availability of so much data, in with now you’ve got all of these people, scientific experts, regulatory decision makers coming together and really saying, Okay, we know this is important, we really want to increase speed. And actually, we believe that in some ways, real world evidence could be the future of how we develop medications and things like that, I think there’s some there’s always going to be there’s always going to be risky, wonky things with speedy AI, but there’s always risky, wonky things, even with randomized controlled trials, right, because we learn things later. All the time. And in clinical development, it’s, it’s a, it’s a, because the by nature research, is the re searching of things that we know, we test theories, right continuously. So the cool thing I think about real world evidence is that you’re you have a way of continuously identifying a lot of other things related to certain populations, certain disease ontologies, that perhaps you wouldn’t have even caught in a signal in a randomized controlled trial in a way that you can with real world evidence, but I, you know, again, I’m not a real world evidence, you know, triallist by any stretch of the imagination, so that would be a question to me, like, is there risk of speed? I think some of the people would say, I think there’s a risk of not speeding up. Yeah, because what we have with RCTs is, you know, just because innovative medicines come to market doesn’t mean patients have access to them. You know, people right now, randomized controlled trials are being developed. for phase two, they won’t be in the hands of patients for another, you know, eight to 10 years. And then the other piece of it is once they’re approved, clinical inertia is a big, big problem here in the United States, right? Like, it’s, you know, clinical inertia contributes to inadequate management and control of chronic disease care. It’s a leading cause of potentially preventable disability, adverse events and even death. Right. So, and this is usually due to a physician or institutions. This is this is controversial, but it’s usually due to a physician or institutions overestimation of the quality of care that they’re delivering reluctance to intensify, or trying new therapies, because people are funny about being the first. We saw this with COVID vaccines, it’s been like a great business case that’s rolled out before our eyes. And then also, the most important piece of it is lack of education and awareness. And for the physicians and the practicing clinician, and, you know, the payers and the health systems who put together pathways and protocols and things like that. So, um, you know, I think there’s, I think there’s two sides to that, you know, speed equals access to a lot of patients who need therapy. But I think it’s to be I think it’s to be determined and not for me to decide if that’s if that’s a danger from a from a pharmacologic or patient safety perspective. That was a long-winded answer, sorry. I feel like I was going in circles for that one.

Garth Sundem   18:58

I can see why oncology is leading the way because you need access now to things that could work better. You know, I also would love to do a whole nother episode on clinical inertia, because I think that is a fascinating phenomenon. But I wanted to circle back and ask if things circle back in that does RWE now is it influencing the choice and design of clinical trials? So, are we going back? Okay, it used to be clinical trials, and then you monitor it with RWE, right, is RWE now feeding back into the design of clinical trials?

Kirstan Summers   19:41

Yeah, absolutely. Because it’s become, it’s become really, really difficult because we’ve got so many. There’s so many drugs on the market, right? There’s so many and there’s so much complex treatment pathways and protocols that are there needed to be developed in order to support precision medicine, that the randomized controlled trial doesn’t really allow for true precision medicine, because you’re really you’re looking at however we define standard of care, which is usually a significant volume of pharmacologic intervention, right? plus whatever the new one thing is. And with a lot of these disease areas, and you mentioned oncology, that’s really, really hard. Because there’s a tremendous amount of, of work that’s being done to really be able to identify all of these different tumor types, all of these different things happening, that when if you if you were to develop an RCT for each and everything you would have, it would be you couldn’t afford to do it, right, because you’d have to have so many different ones and all sorts of ways, shapes and forms. So really the traditional randomized controlled trial, I think, while still in, it’s still an important piece of development, I don’t know. We can all speculate on what the how those, how these things come together in the future. Whereas right now, you know, you have your RCT, but perhaps all at the same time, you’re collecting and observing all of this data for potential label extensions to look at other patient populations. You’ve established, you know, the safety and efficacy here and gotten approval based on that. But what we’re seeing is more openness of regulators to view real-world, real-world data as part of, you know, expanding access to different patient types on an already kind of approved drug.

Garth Sundem   21:46

Well, and that brings us back to where we started, which is that RWE is now being recognized as a very valid source of, you know, more formalized information about emerging treatments. So, let’s, let’s leave it at that. Thanks.

Kirstan Summers   22:04

Are we done already?

Garth Sundem   22:06

We’re done already! Oh, my goodness gracious. But you got to come back because this is fun. So, thanks for joining us today to learn more about how your organization can partner with H1. To unlock key insights and opportunities for industry engagement. To create a healthier future, visit H1.co. MAPS Members don’t forget to subscribe. And we hope you enjoyed this episode of the Medical Affairs Professional Society Podcast Series: “Elevate”