Using Real-World Data to Understand Patient Journeys in Dermatology
Post-approval safety studies have traditionally been cumbersome and expensive, and safety study sites are likely to mirror those used in clinical trials, leading organizations to potentially miss safety signals in populations beyond those that typically participate at trial sites. Here we speak with Stefan C. Weiss, MD, Managing Director, Dermatology at OM1 about a transformation in the way we conduct safety studies — relying on EMR patient data overlain with technology to create a more accurate and equitable view of post-market safety.
Download accompanying article: “A New Approach for Post-Marketing Safety Studies,” by Stefan Weiss.
Following is an automated transcription provided by otter.ai. Please excuse inaccuracies.
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 talking about using real world data to understand patient journeys in dermatology with Stefan C. Weiss, MD, Managing Director of Dermatology at OM1. This episode is sponsored by OM1. So, Stefan, first of all, thank you very much for joining us.
Stefan C. Weiss 00:29
Thank you, Garth, for having me.
Garth Sundem 00:31
And I think we are used to from Medical Affairs looking out into this world of post-marketing safety studies. They can be big, they can be cumbersome. How do they work now? And what is their importance?
Stefan C. Weiss 00:48
Right. So I think they’re still big, and they’re still cumbersome. And, and I think that that’s where they can change. And like everything. As we’ve seen in this world, technology is allowing us to do things better, faster and more efficiently. And the goal is to convert these types of safety studies in a similar fashion. And so it’s important to understand why they’re big and cumbersome. And then why we should be able to do them in a better way, I think,
Garth Sundem 01:18
Okay, well, we’ll catch me up. So I mean, clinical trials, you have all these sites around the country, and people, you know, come in physically, and they are usually given some treatment. And then safety studies, did they just continue with these same sites, and people keep coming in for checkups? And as you know, they’re asked about adverse events, or how do they physically work now?
Stefan C. Weiss 01:42
Right, so so partially, that is correct. And so I think it’s always important to understand the communication of safety in the information that drives that communication is achieved in two ways. So one is, as you were saying, clinical trial patients are continuously monitored long after the clinical trial has completed, and long after the drug is approved. So that we have three, five and 10 year safety and efficacy data on these individuals that aren’t lost to follow up. Yeah. Okay. Other aspects of it is the post marketing safety commitments that the FDA may put on top of a therapeutic following its approval. And so these are the post approval safety studies that are required by the Agency for a new therapeutic, those are done historically, not dissimilar from the way you described a clinical trial, where you’re going to enroll a bunch of sites around the country, and have people that are being prescribed a certain therapeutic, or have a certain disease, if you’re looking to therapeutics more broadly within that class, and followed again, for some prescribed period of time as defined by the agency.
Garth Sundem 02:50
Okay, and I’m sorry for these background questions. But how do you get those people to come in once the drug is approved? And, you know, you have a safety commitment from the FDA? What what is their incentive to come back in for a safety monitoring, don’t they just get the drug drug approved, and then they they’re administered the drug.
Stefan C. Weiss 03:08
So so they administer the drug, and the safety studies for post marketing safety commitments are done on routine clinical care. So if you’re a patient with a particular disease, and you’re prescribed drug X, and that drug is under the as a newly approved drug with a post marketing safety commitment, the provider who gave it to you would enroll you in this study to follow you long-term.
Garth Sundem 03:27
Okay, so great. One example we brought up when we were chatting about what we were going to talk about is JAK inhibitors and dermatology, I know dermatology is your specialty. Is this. Is this an example of how safety studies are cumbersome and expensive? Or is this a story of how they should be run? I can’t wait to hear the story.
Stefan C. Weiss 03:50
Right? So So classically, they’re still being done in the larger, cumbersome way. And again, getting back to a point we started talking about a little earlier. It’s really based on history. So it wasn’t that long ago, where you walked into a physician’s office. And there were these things called manila folders. And we refer to them as charts. And they had little letters on the side for your last name. And if you’re an office that had been in existence for 30 years, you could imagine the chart rooms that existed. Today, we have EMRs propagating throughout the medical system, which allows us to collect all of this information in a data driven fashion. And because of that, we can now look to surveil safety, efficacy, etc, by using the data that’s coming from the EMR. And so the goal would be to take the safety studies, no longer have them in their cumbersome fashion, and use data to do this more efficiently. The other aspect that you run into when you use the classic methodology of enrolling sites to bring in patients versus data surveillance is you don’t necessarily Get an appropriate context of the population. So you can imagine the sites that you’re going to go to for a post marketing safety study are not going to be that dissimilar from the sites you’re going to use for clinical trial enrollment pre approval. That’s not necessarily capturing the large population of patients who may be exposed to a drug. And so for instance, I live in the state of North Carolina, you could well envision that Charlotte, Greensboro week for Winston Salem area in Raleigh, Durham would be sites that you would have both clinical trials and post marketing safety studies. But there’s a large population of patients in eastern North Carolina, that look different than the patients who may be in one of the three major metropolitan areas. There are older, more retired populations, both in the mountains on the coast, that may be different than the younger, more affluent population in the three urban centers. And so if we don’t conduct safety studies with a broader and catchment, we’re not necessarily going to understand the impact of any particular therapeutic on individuals that represent a more diverse but representative population of the users of the therapeutic.
Garth Sundem 06:12
Okay, you know, talk with folks about distributed clinical trials, is this a distributed safety study?
Stefan C. Weiss 06:18
You know, we sort of represent the same thing exactly.
Garth Sundem 06:22
OK, the same thing. Okay. Okay. Well, you you We missed the story of JAK inhibitors. So what what what happened and what’s the learning?
Stefan C. Weiss 06:33
Right, so So JAK inhibitors sort of represent any other immunologic therapeutic that we’ve brought into the world of dermatology, rheumatology and inflammatory bowel disease over the last decade, were trying to enroll patients to follow their long term safety. In the case of JAK inhibitors specifically, we understand there are certain safety signals that we have to be respectful of. And we want to ensure that those are not occurring at a rate higher than they did within the clinical trial populations. But in order to do that, we need to have a diverse population of individuals who we can follow over a long period of time. Unfortunately, what ends up happening is you end up having number of patients who are enrolled in the classic model of safety studies lost to follow up or changing providers. Because again, when you go from insurance, company A to Insurance Company B because your employer in the enrollment period has changed, you may now need to see a different provider. And if your provider is not part of the safety study population of sites, you are now out of that study, we’ve lost that data. By following you in the background, we can continue to maintain that relationship, ie distributed clinical trial where the relationship is with the patient, more so than the site. And that data continues to be collected. So we understand what the implications are. And that’s going to be critical for the use of JAK inhibitors in dermatology, where they’re coming in now a second line agents, specifically in atopic dermatitis against a more well established therapeutic, that has a more favorable safety profile by clinical trial design, but may not be much different, as we understand it over the long term. But we need to have that long term data to really understand what the implications of that therapy are.
Garth Sundem 08:19
Okay, so that’s interesting. The data is attached to individual patients, not necessarily to the site, or the provider. So if the site of the provider changes, you can still follow safety in that patient. This sounds like a lot of data. Big data requires technology. So how do we then follow all of this data to keep track of the safety signals that we need to ensure that usage in the real world population adverse events isn’t exceeding what we saw in the clinical trials.
Stefan C. Weiss 09:03
Right. And so so to your point to to collect and curate all of that data, we start by tokenizing the individuals and so that keeps everybody de identified which is important from the standpoint of healthcare privacy, and start with the EMR data where we get the information on the individual patient that that individual has disease, x in his being prescribed treatment, why? And then we link that data deterministically to other sources of data. So it could be hospitalizations, it could be pharmacy data, it could be social determinants of health data, it could be death data in certain instances, because there you’re going to begin to follow that patient journey in a more robust manner. So we may not be collecting in a, let’s say, a dermatology office looking at a JAK inhibitor for safety. Someone’s hemoglobin A1C because they’re diabetic. And we may not be checking blood pressure on any regular basis within the dermatology visit. But, but those are important factors to look at as we try to understand the safety of any particular therapeutic. So by linking that individual’s medical record that we’re collecting, as part of the safety study on the dermatology side, with the other data points that come from other visits that are within the real world data cloud that we’ve linked to within the company’s database, it allows us to paint a more thorough picture of that individual. Understand, should there be a hospitalization for a race event? Should there be an elevation in blood pressure, should there be a change in hemoglobin, a whimsy, whatever the safety metric is what we’re most concerned about, we’re able to follow passively, and not lose that information. Again, when the patient may leave one particular provider or site due to another.
Garth Sundem 10:48
This sounds like it just got much more complex, you know, I was thinking safety study. So you have patient x taking drug A, or you have a patient taking a drug. And if they have an adverse event, then that becomes part of the safety profile the drug, but you’re saying that, you may have to disentangle that a little further, saying that if if a patient is taking a drug, and has an adverse event, like high blood pressure, maybe there’s something else causing that, you know, is that due to the drug, are you disentangling an adverse event from causality of the drug using this data? Or, or? Or is it still just as simple as you know, a patient taking a drug across the population? How many of these adverse events are there?
Stefan C. Weiss 11:39
Right, it’s still more the latter, whereas they’re not going to be able to link specifically the causality. But what the FDA is interested in, is there an elevation in signal? Are we seeing more mais events in patients that are on a particular therapeutic than in those with a similar disease on a different therapeutic? So what do we understand about the relationship between a specific patient population on drug A, versus a similar population on everything but drug A, in that way, we’re able to follow that information. The other aspect of it is in think about this yourself, when you see a particular physician, be at a dermatologist, orthopedist, etc, you’re really only focused on the reason you’re in that office, you’re not likely to discuss the other aspects of your medical issues with a provider that’s not relevant to that. So you’re not always going to capture these events in the normal course of conversation with the provider of interest, that’s the site provider for this study. Whereas if we look at your data in the aggregate will begin to understand how this is impacting you in areas that may not be relevant to you. In that initial conversation with again, let’s use your dermatologist in this situation.
Garth Sundem 12:58
Okay, so we talked a little bit about, it seems like there’s a lot of promise and using a big data approach to safety studies, we talked about one of the promises, and that would be a more representative population. You know, rather than just looking at similar sites to your clinical trial, it also seems like this could be faster, cheaper, easier. You know, what do you see is the value of transitioning to a more big data technology based approach.
Stefan C. Weiss 13:23
I think that you summarize the two big reasons. One, it’s faster, cheaper, better, and two, which everybody wants, especially in today’s day and age. And two, you really want to capture the diversity of patients that are using a therapeutic to understand are their specific safety signals in individuals who may not have been appropriately captured within the clinical trial. And so an example is a recent therapeutic that was approved in psoriatic arthritis was 96 or 97%. Caucasian in the clinic. But the population itself is roughly 60 to 70%, Caucasian that suffers from the disease. So there’s an entire population of individuals who were not studied in the clinical trial, may very well received the therapeutic as part of their routine clinical care. So what I want to understand as a provider is if I put this patient on a drug, would they get the same response as the individuals who were studied in the clinical trial, but more importantly, would they have the same favorable safety profile that was elicited within the clinical trial?
Garth Sundem 14:31
That sounds like a lot of data. And I’m glad that you are working with the technology to make sense of it all. Okay, so let’s leave it there for today. Stefan. Thanks for joining us. To learn more about how you can partner with OM1, visit OM1.com. With specialization in chronic conditions, OM1 is reimagining real world data and evidence. MAPS members, don’t forget to subscribe and we hope you enjoyed this episode of the Medical Affairs Professional Society podcast series: “Elevate”.