Sydney Perlmutter:

Good day to everyone joining us and welcome to today's Xtalks webinar. Today's talk is entitled The Reimagined Registry: Revolutionizing Patient Care and Research through Real-World Data. My name is Sydney Perlmutter and I'll be your Xtalks host for today. Today's webinar will run for approximately 60 minutes. This presentation includes a Q&A session with our speakers. This webinar is designed to be interactive and webinars work best when you're involved. So please feel free to submit questions and comments for our speakers throughout the presentation using the questions chatbox, and we'll try to attend to your questions during the Q&A session. This chat box is located in the control panel on the right-hand side of your screen. If you require any assistance, please contact me at any time by sending a message using this chat panel. At this time, all participants are in listen only mode.

Sydney Perlmutter:

Please note this event will be recorded and made available for streaming on xtalks.com. At this point, I'd like to thank PicnicHealth who developed the content for this presentation. PicnicHealth is a healthcare technology company that partners directly with patients to build deep real-world data sets. The company leverages state-of-the-art machine learning combined with human curation to put complete medical records into an easy to use online application. The platform gives patients unprecedented access to and control over their medical records, and with their consent, the opportunity to contribute this valuable data to further scientific research.

Sydney Perlmutter:

Now I'd like to introduce our speakers for today's event. Heather Fitzpatrick Medlin is CureDuchenne's Senior Director of Clinical Development. In this role, Heather leads the CD Link program, a decentralized data integrated biobank platform to connect researchers to coded data and biosamples from individuals with Duchenne and Becker muscular dystrophy, as well as the carriers of these mutations. Heather has over 25 years experience in the clinical research industry across all phases and therapeutic areas with a concentration in the late phase medical device and diagnostics projects focused on rare disease. Her prior experience working in the public health arena and hospital settings has enabled her to understand the perspective of patients, healthcare professionals and industry professionals. She completed her master's degree at the University of South Carolina and her undergraduate degree at Davidson College.

Sydney Perlmutter:

Clara Lam received her PhD and MHP from The George Washington University Milken Institute School of Public Health. She continued her studies at the university and pursued her pre-doctoral and post-doctoral fellowships in collaboration with the National Cancer Institute in the Radiation Epidemiology Branch. Clara went on to be a mathematical statistician in the Surveillance Research Program in the Division of Cancer Control and Population Sciences. During the last three years, Clara has taken an opportunity to join AstraZeneca in their US Health Economics and Outcomes Research group. and then became a strategy director. Then finally as an asset lead directing a comprehensive portfolio of epidemiologic and HEOR studies supporting AZ breast assets in the US and globally.

Sydney Perlmutter:

Daniel R. Drozd is the Chief Medical Officer at PicnicHealth. Prior to joining PicnicHealth he was on faculty at the University of Washington in the Department of Allergy & Infectious Diseases where he led research into the use of electronic health record data to power observational research and enhance the understanding of the chronic burden of HIV infection. At PicnicHealth, he oversees scientific collaborations with PicnicHealth's industry and academic partners and works extensively with both the product and commercial teams. Prior to attending medical school, he worked for numerous technology startups as an engineer and at the University of Washington in the Clinical Informatics Research group where he led the development of a large EHR data integration platform used to power HIV real-world research.

Sydney Perlmutter:

And lastly, Evelyn Pyper is a Senior Evidence Strategy Lead at PicnicHealth, a patient-centric real-world data company. Her career in real-world evidence (RWE) spans the public and private sectors as well as regional and global markets. Prior to PicnicHealth, she worked as Associate Director of Market Access at J&J Global Public Health, focused on securing access to HIV treatments in Sub-Saharan Africa and as RWE Manager of a diverse portfolio of partnerships and research projects at Janssen Canada. Evelyn has a bachelor's degree in Health Sciences with a minor in Psychology from McMaster University and a Master of Public Health degree from Queen's University. Evelyn is currently completing her doctorate in evidence-based healthcare at the University of Oxford, with a focus on digital health.

Sydney Perlmutter:

And now without further ado, I'd like to hand the presentation over to our speakers so you all may begin when ready.

Evelyn Pyper:

Great, thank you so much Sydney and welcome everyone. Welcome to our speakers. We are really excited to have you all here live for the third webinar in PicnicHealth RWE ROI series. Some of you may have actually joined us earlier in the year as we discussed the value of real-world data for drug development and regulatory decision making in our first webinar or perhaps in incorporating different data types into clinical trials for our second webinar. But for those who are joining us for the first time today, the goal of the RWE ROI series is really twofold. It's to dive deeper into today's uses or untapped opportunities when it comes to real-world evidence and to convene a diverse group of experts with different perspectives from across the life sciences ecosystem.

Evelyn Pyper:

And today we are very thrilled to have Clara, Heather, and Dan with us to discuss the innovative ways that stakeholders are optimizing registries to generate more robust research insights. So given the topic of this session is the Reimagined Registry, it's probably worth us starting off our conversation with a perspective around what constitutes a traditional registry. While no single definition exists, a patient registry which might also be referred to as a clinical registry, disease registry or outcomes registry, typically refers to an organized system that uses observational study methods in order to collect standardized information about a group of patients in this case. We know that registries are a really key source of real-world data that can meet a variety of needs and answer a wide range of research questions.

Evelyn Pyper:

At the highest level, registries serve an important purpose, which is evaluating and improving outcomes for a population, often that population being defined by a condition, a disease or some exposure. So with that foundational level setting, it's clear that patient registries have an important role to play in healthcare research. However, we know, and this group here knows all too well that there's many challenges with traditional registries that can have broad implications for researchers and participants alike. So starting with you, Clara, what are some of the methodological challenges that you've seen or experienced yourself with traditional registries?

Clara Lam:

Thanks for the question, Evelyn. Thanks so much for the opportunity to be part of this really nice distinguished panel. Thank you all as well for joining. I think registries can be extremely important. They can provide population-based data, longitudinal information. It can be really important for looking at trends over time amongst just understanding the population that we're trying to help and improve their outcomes. But there are issues with that. A lot of it is site-based location. And so there's a lot of restrictions in terms of where the representation might be coming from. And a lot of the time it's actually restricted to academic centers, so we might not get great representation coming from a community setting. They're often specific to different specific sites. So there might be a limit on the scope of data collection that one might actually be able to get.

It's a static data model in many ways as well. It's not necessarily changing with emerging technologies and information that might be coming down in the future. And a lot of the time it's also restricted based on resources, time and budget of the sites themselves. And so I think there are wonderful abstracters out there who have just done so much in collecting this information, but it can be very limited in terms of the resources. And so I think while it's a phenomenal way to collect information, we might want to think about more innovative ways going into the future because I think some of these limitations just cannot be overcome.

Evelyn Pyper:

Thanks so much. That very much resonates. And Dan, would you agree with what Clara said? Would you add anything? And maybe taking a step further beyond methods, do you see any other challenges with traditional registries when it comes to the data that's then available from those sites?

Dr. Dan Drozd:

Absolutely. Thanks for the question, Evelyn. I completely agree with all the things that Clara outlined. I think just to call out very explicitly, registries are incredibly expensive to run, right? They have very high burden both on sites and on patients. And I know we'll dive into both of those topics in a little bit more detail later. But I think it's increasingly difficult to get sites with limited time and budget to run registries compared potentially to observational studies that may be better compensated for sites. I think as Clara mentioned, data elements need to be pre-specified in terms of registries, and there is the potential for a lot of expense and additional time requirements if you need to add an additional data element. Something as relatively simple as adding an additional data element can add months of delay as well as significant cost burden to sponsors.

I think it's just increasingly difficult to get patients to contribute data to registries in an increasingly crowded space and field. So specifically though about data, I think piggybacking off of those, loss to follow-up can be a huge issue for registries. And so that definitely limits the usability and generalizability of the data that's being collected. I think as Clara mentioned, questions of representativeness are key in terms of the data as well. So are the patients that we're capturing data from in the registry, do they look like patients broadly within the larger population? And can we generalize results that are derived from a particular registry to another population of patients if we're only collecting information from large tertiary academic care centers, etc.

And so I think that representativeness becomes a major question, a major challenge in terms of the underlying data as well. I think representativeness, loss to follow-up and long-term follow-up in terms of the data. And then that, as Clara mentioned, the static nature of the data and registries present real challenges to the way that we have done things historically.

Evelyn Pyper:

Thanks, Dan. Taking what you've said in total, it sounds like it's not just an issue of where the data is collected, but also how it's collected and from whom. There's layers to that challenge, which means complex challenges needs complex solutions, I suppose. And before we start talking more solutions oriented, we know that beyond the data is of course patients themselves. And Heather, from your perspective, what are the shortcomings you've seen with traditional registries when it comes to the patient experience? So traditionally, what might some of those barriers be to either initial or ongoing participation in registries?

Heather Fitzpatrick Medlin:

Thank you so much for the question, and thank you for the great conversation so far. I think what we know is that especially in the rare disease space, everyone is looking for an answer. They're spending a lot of time trying to go to different places, collect information from lots of different sources. So there's a burden, especially when you're looking at a pediatric population, we put this on the parent or the caregiver to go to these different sources. One of the biggest challenges we've seen, and I think we see now, is understanding that oftentimes we present the request for this information in English and not everybody speaks English. So making sure we're connecting with the larger community, especially in diseases where we see them more broadly, both geographically, demographically, et cetera.

I think the return on investment, what's in it for me, and making sure we push the message about the fact that they're providing this information, whether it's patient reported data or site or research center provided data, that it may not lead to a treatment right away, but there is that downstream potential for an impact to maybe the next generation or a later change in treatment that could impact the individual participating. I think the ROI, and then what we see is specifically working now in the Duchenne space is that we have a lot of individuals who can't participate in this type of research due to the structure of clinical trials and the exclusion criteria from participating. So talking about loss to follow-up, we lose people sometimes because they have to decline participation or stop participation while proceeding forward with intervention.

Evelyn Pyper:

Thanks Heather. And I'm really glad you mentioned the ROI from a patient perspective, because I do feel like all too often and throughout this series we've considered multiple different perspectives, but we're often thinking about what's the ROI to invest in the infrastructure to do something like this for the people developing it. And often it's an afterthought to think about the actual ROI for the patients themselves and the families of those patients. I'm really glad you mentioned that. And so at this point now, we've discussed the variety of ways that traditional registries really need to evolve and have needed to for some time. And fortunately for those listening, we don't just have to imagine what that evolved registry might look like.

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Evelyn Pyper:

Our panelists here have actual hands-on experience with moving us to that next phase of what registries could be. So as a follow-up question for you, Heather, can you tell us a little bit about CureDuchenne link and what makes that an innovative model?

Heather Fitzpatrick Medlin:

Sure. Coming from experience running, a lot of what we would say are traditional registries, I was excited when I joined CureDuchenne because we've taken not only the component where we collect information directly from what we call our participants, so survey-based information directly from a parent caregiver. But we're also asking for donation of bio samples that we're able to collect aliquot into smaller samples and provide back to researchers. But one thing we were missing, and I think we all talk about this, is the larger set of data that we no longer want to send somebody to abstract or have just limited information coming over, but really understanding that especially in a rare space there's multiple providers, there's often lots of geographies that are covered as parents go to seek care.

So what we've done is partnered to be able to pull that data in to make a large and more robust data source, collecting both retrospectively looking to go back maybe towards the age of diagnosis and then moving forward with no endpoint or upper limit of participation at this point.

Evelyn Pyper:

Thanks so much. And maybe just a side question that comes to mind and might be a question that our audience would have, is typically when folks think of registers, they think of the prospective collection. Do any of the panelists want to speak to what it means to have that retrospective data with a real example or a hypothetical? I think often people think of observational prospective cohorts and not that retrospective lens.

Heather Fitzpatrick Medlin:

I can jump in because we are collecting this retrospective understanding specifically in Duchenne, that the diagnostic journey can be five to seven years. There's a lot of information on that journey that's not presented initially. I did want to speak to the fact that the way we're collecting our data now using PicnicHealth, we're actually able for that ROI question to provide it back to participants so they have a one-stop place where they can access their information, take it to a healthcare provider or just review it themselves. And that's been a really great system, because again, that's not something that has traditionally existed in the registry or even in the medical record space.

Clara Lam:

I'm going to chime in a little bit as well. I think the collection of that retrospective data and in the situation that we're working with for PicnicHealth is we're looking at early breast cancer patients. And breast cancer patients if they receive a diagnosis, it could be a very long time for their treatment journey. And survival in early breast cancer is quite high in the United States, and obviously that's where we want to keep it, but there's a lot of information that we can glean from that retrospective data collection upon their first diagnosis and following them through their patient journey. It's invaluable that we're able to get access to all of that. And then importantly, as Heather was mentioning, being able to provide that to the patient themselves.

I think as we all know, it's just so difficult to keep track of all your medical appointments and all the information that comes along, every scan, every blood test, and having all that in one convenient location that is easily accessible and easily understandable, that's just so invaluable. And I think that's something that is a huge benefit to the patients who are involved in the early breast cancer registry.

Evelyn Pyper:

Thanks so much for sharing, really appreciate that. It seems like it really underscores the point that for these patients, the clock is not starting at the time of diagnosis, at the time of entry into the study, there's a whole wealth of information that is all too often forgotten about or just inaccessible, that can tell a totally different story. And so this starts to introduce some really exciting ideas, I think, and paints a clear picture of the value that innovative registries have for rare disease research. And Clara, building on what you alluded to already, you're someone working in a very different therapeutic area than Heather is. You're working in oncology. And so are these innovative registry approaches relevant to early breast cancer with a variety of other cancers you've worked with, and are there any other types of novel designs or types of data that have you most excited in working in this space?

Clara Lam:

Absolutely. I think you touched upon it already, Evelyn, that everybody wants to do an observational prospective study, but the reality is that those are very difficult to get off the ground. They're expensive, they're time-consuming. They require a lot of personnel and a lot of procedures and logistics to be in place and to be able to have retrospective medical records from patients and following them forward, it really provides a very unique environment that we can look at emerging biomarkers. Every day I feel as though there's a new biomarker that we've discovered, and it might have a key role in whatever cancer we might be looking at. And breast cancer in particular, we know the ones like BRCA for example, and that's something that we all can test for, but there might be more.

Clara Lam:

And so it'd be really fantastic to be able to not only capture the retrospective information for the patient journey, but to follow forward and see what happens with emerging biomarkers. I think another huge innovation that's really helpful is treatment and oncology is just exploding. It changes so rapidly. Every other day in the news you're going to hear about new treatments that are available for a different breast cancer, lung cancer, pancreatic. And so being able to track this forward in time also is really, really important so that we can see, what do the patients get in early breast cancer for their first sign of treatment? When did they have their surgery? What are they getting after? Did they have neoadjuvant treatment?

I think a lot of just being able to have that level of detail and granularity for the treatment journey for these patients, it's so important. I think the other huge component, and this is something that Heather touched upon earlier, but getting those patient reported outcomes, that is something that has become that much more important and become really central to the studies that we do. We want to know, how are the patients doing? What goes into their decision making? How are they discussing this with their caregivers and their patient care team? There's a lot of different important factors that may have prognostic value that we need to include as part of these registries. So to be able to capture the retrospective data but then follow patients forward, it's just invaluable. And that's just something that we really can't do in a traditional setting.

Evelyn Pyper:

Thanks so much. I'm glad you mentioned the patient reported outcome piece at the end. It seems like the traditional registry world, things really fall neatly into categories of researcher driven or patient driven in terms of who birthed it, who's really owning the collection of that data. And it's really exciting to see those boxes be broken down as we think about things that can be equal parts, information coming from patients, they're involved in the process and getting something out of it. Researchers are also really critical in defining what are those key data elements and continuously improving the data model if necessary as new treatments come on the market. I think we'll probably get into this as well a bit later, but just especially the treatment angle, really thinking beyond disease registries to product and drug specific registries where a data model from five years ago might not be relevant for the types of endpoints that are critical.

Evelyn Pyper:

I'm excited to talk more about that a bit later in the session. But for right now, Dan, PicnicHealth has partnerships with both CureDuchenne in DMD as well as AstraZeneca in early breast cancer. These are unique collaborations and different disease areas with very distinct research needs. And so what is it, if you could describe it about PicnicHealth's approach that drives value for researchers and patients across these varying diverse contexts?

Dr. Dan Drozd:

I think it's a really good question. I think the one thing that both of these conditions have in common is they certainly can have complex diagnostic journeys. And really as a patient in the United States in particular, you really are the only through line yourself in your journey through the US healthcare system. And so having data that is siloed in one organization or one institution, really inhibits the ability to understand that comprehensive care journey. I think if you think about what we actually mean by patient-centric research, I think this is a term that obviously gets used really often. I think ISPOR has a nice definition of patient-centric research. And they say that it is the active, meaningful and collaborative interaction between patients and researchers across all stages of research process where research decision-making is guided by patients' contributions as partners recognizing their specific experiences, values and expertise.

I think if we  think about that definition, that definition is so different from the way that we would've conceptualized what research historically has been, right? Historically participants have been subjects, they've been things we study, not active participants in the research process. And so I think one of the things that both of these collaborations have in common is a real desire and motivation to put patients at the center of that process. One, because it's the right thing to do, but also it is the only way that we can actually get at the data that we need in order to move forward treatments and decision-making in both of these conditions. I think that starts obviously with having fully consented patients, it means having a low lift for patients from an onboarding and ongoing participation perspective.

It means meeting patients where they're at. Some combination at times of data that's collective passively and data that we are actively collecting from patients, from a data perspective it really leverages patients individual experiences to compliment the data coming from electronic health records, whether that's codified data coming through in ICD 10 codes or information coming out of narrative text sections of patients records, which are important in different ways in both of these conditions. And then combined, that really gives us a much better 360 degree view of the patient journey. And then just from a technology perspective, I think in order for us to be able to do this at scale, it really does require a sophisticated underlying technology platform that's tuneable and responsive to the specific research needs within a particular condition.

And that it allows those things to change over time, that it becomes relatively straightforward when there's a new biomarker of interest or a new gene mutation of interest, to be able to interrogate the data and get additional information about that without having to go back and do a protocol revision and manually reconstruct a bunch of data, etc. And so I think that's the way that I view our overarching challenge here. And the reason I think that both of these collaborations, while the conditions are obviously in many ways very different, have an underlying through line that has made both these collaborations important and successful.

Evelyn Pyper:

Thanks, Dan. And maybe as a follow-up question for you, for some of our audience members that maybe haven't been to one of these sessions before, can you give even a brief flavor of what those core technology capabilities or platform as PicnicHealth drives this? It can be a fairly non-technical explanation of what that technical component is.

Dr. Dan Drozd:

I think when I think about it, it's having the infrastructure to be able to collect records from anywhere a patient has been seen, that is a foundational part of the electronic health record component. And that is both leveraging electronic means as well as other means of collecting data when necessary. From a data structuring standpoint, it really is the use of advanced machine learning technology to be able to have a scalable process that we then can tune to how much human review of a particular concept or idea we want to put into place. And so there are concepts that are relatively straightforward and we may need less human review. So an individual lab result for example.

And things that definitely need a lot more review, is a patient's cancer progressing? That's something that definitely has a lot more nuance around it. And so I think at its core, it's a technology platform that leverages state-of-the-art machine learning technology to enable us to tune the output of the process to whatever the particular research needs happen to be. And is cognizant of the fact that the needs for data that is being used for a regulatory submission are very different in many ways than data that might be used for an internal study or other purposes.

Evelyn Pyper:

Thanks, Dan. With that in mind, we know that there is value that these registries can bring to life sciences researchers, but what that particular value is is going to probably depend on a variety of factors, including whether or not there's a need to study a particular treatment or disease, what we've spoken to before, the stage of clinical research, what functional groups are involved. And Clara, you have extensive experience working in medical affairs. So in that case, and actually building on what Dan just mentioned in terms of it's really going to depend on what it's being used for. What might success look like for a medical affairs function when investing in a patient registry? What needs to be the output? What do you need to see to call it a success?

Clara Lam:

No, absolutely, and I think I alluded to it earlier, but the ever fast pace of oncology, we need to have the most current data available. Part of the problem with more traditional registries is that there's usually a time lag, there's a data lag, because it just takes time to collect this information. And so unfortunately with some of the registries that are available currently in a traditional setting, there might be a time lag of two to three years. And while it's still extremely irrelevant and very helpful, we are worried about the current treatments today, what is the current standard of care that the patients are dealing with? What are the new options that are being made available to them and what goes into that decision making? We need to have very current up-to-the-minute information about the patients who might be receiving the products that we might be supporting.

And then going into the future, I think the other thing is that we need to have patients who are really representative. Some of the restrictions when it comes to other traditional registries is that side based, it might be a state registry, it might be a local registry, but we really want to be able to capture patients from all over the United States and not just in one or two areas. And I think the other, there's a time element to that too, is that we want to have representative patients that can provide their information quickly and to be able to upload that information in a really timely manner. And so not just the medical records that we're going to be capturing, but also patient reported outcomes or potentially if we want to evolve caregivers somehow and building that into the platform, that's something that we can explore and that might be an option in the future.

But I think just having that more complete picture and we really need a patient population that's representative of folks who are outside of clinical trials. That's a really big component for us, is that clinical trials are only representative of certain subsection of population, and we want to have underrepresented groups, whether it be racial and ethnic minorities, it might be subgroups of different biomarker patients who are not well-represented and registries currently. We want to go after the patients that we don't see anywhere else. And so the novel approach that we have with the platform we're building for the early breast cancer registry with PicnicHealth, that's the patients that we're getting. So that's really phenomenal that we can do that in a really current fashion.

That's exciting for us and that's a sign of success that if this registry can really represent those patients, then we're doing something right.

Evelyn Pyper:

Definitely. Exciting on the PicnicHealth end too, all around huge in terms of what we're able to achieve together in this partnership. And as a follow-up question, some of the attributes you mentioned, you're able to, as a decision maker, you're deciding where to invest in a registry. You can ask the right questions and glean that information up front, but there's probably other attributes that you can only determine what's going to be successful once you're in the data, once you're there. And for the subset of folks that are listening in live who might be also deciding where to invest in a registry like, beyond the let's say representativeness, which you spoke to, are there any other specific registry attributes or signals that you would suggest to folks like yourself who are deciding what's the best approach or where best to invest their time and resources?

Clara Lam:

I have to say with the PicnicHealth team, we've asked a lot of questions repeatedly. I feel very confident in the PicnicHealth team and their level of expertise across all different levels to be able to answer a lot of the questions that we had about different data elements. We would want to capture the cadence of that information, how often we would have the data being delivered, the refresh of that data, how often the army of abstracters are sent out to collect additional information going forward. And two things that are really important is that just because we have a patient enrolled and we start collecting their medical information, that's not the stopping point. We actually keep following them and we keep on getting information at regular intervals. And so not only do we continue adding data to them, we just continue following them forward and have opportunities to interact.

I think the other huge thing, and I can't stress this enough, I'm really excited about this, is the source documentation. Being able to actually go back and look at this information in the event that there's another biomarker that comes up that actually has been collected and we just didn't know how to collect it and we didn't realize its importance. But that information is already in the medical record on some level for some data elements. And so being able to go back and get this information without having to go through the procedures of writing a new protocol or making an amendment or having to fix the static data model to add this in as well, and all the logistics for that, I think the fact that it's very flexible and that it's adaptable, I think that's a huge set of attributes that this registry really provide. It's a lot more straightforward.

And I think in terms of the patient perspective as well, and I say this as a patient myself and just for other things, but I think everyone is really appreciative of the fact that it's simple for us to get this information and we have it in a format that makes sense and is easily used and analyzed, but it makes sense for the patient too. It's a win-win on many different fronts. And so I think this very novel approach of capturing this information, it's really hard to not say yes to and be a part of.

Evelyn Pyper:

Absolutely. And it's wild to think that a three to five years, which might be like an average, let's say study time, to be able to avoid having to spin off a separate study, three to five years in the life of a patient is huge, is massive if that new biomarker becomes known and that information needs to be gleaned for a study. If you just think about timelines, it's massive for researchers, but also even more massive in terms of the life of a patient and saving that time. So Heather, question for you. We know that the integration of different real-world data sources seems to be at the core of your registry's resolution. Can you provide some insight examples about how integrated data translates into better research and ultimately better outcomes for patients in the context that you're working in?

Heather Fitzpatrick Medlin:

For us I think it was really important to understand that for these kids with Duchenne, CureDuchenne's focus is getting that research and getting to the cure. And oftentimes we have information, it's in different places, it's not systematically available, and that's time. We're finding out there's so much active research in the space right now that we're able to provide not only the data that we're collecting from the participants directly, we're able to supplement that with de-identified data from PicnicHealth. And then we have, as I mentioned, the bio samples. We're able to provide researchers, whether they're biotech, pharma or academia with that information on the ready.

It's another disease space where we're seeing so many new and innovative treatments come, and we really want to focus on being able to keep up with that and recognize that over time that the different elements of research are going to change, but we always are going to have a baseline in that data and the samples moving forward.

Evelyn Pyper:

That's great. And for those who are less familiar with the disease space, as a follow-up, are you able to speak any more to the significance of those biosamples? In your average DMD study, are bio-samples a core part of it? Is it pretty unusual to have it collected as part of a study or registry like this?

Heather Fitzpatrick Medlin:

Certainly not unique in the study space, but definitely in a data integrated biobank as we're running, it's uncommon. I wouldn't say we're the only one, but we're one of few. And for us, it's really important that we're able to offer that in a collective place to researchers quickly, because for these boys, time is of the essence.

Evelyn Pyper:

It's clear from our talk so far that registries are not what they used to be. We've talked about the way different types of data being collected, different ways of collecting that data, integration of data types, but also across time retrospective and prospective, and then the entire gamut of patient-centric elements that we can pull into things from patient-centric approaches to getting patients onboarded and aware of the registry in the first place all the way through to giving back data to patients themselves. It really feels like there's this suite of possibilities in this new era of registries. And this is undoubtedly enabled by advancements in data and tech, innovative partnership models like the ones that folks on this call are part of, as well as bringing together the strengths of stakeholders from different aspects of the ecosystem.

Evelyn Pyper:

I'm fairly certain that not one of us on our own could really have the impact that we could have together, particularly when it comes to registries and how many folks are involved. But with that, we've never quite solved the problem. Our work is never quite done. I'm sure all of our panelists have ideas or dreams, top of mind, that continue to move the needle for patient registries. And so as a larger final question before we head into more of the audience Q&A, I'll ask each of our panelists to imagine a world where resources, people, resources, financial resources are unlimited. What would you do with these infinite resources to further optimize the design or deployment of your registry or a registry? Dan, I will start with you.

Dr. Dan Drozd:

All right, I have lots of thoughts and ideas here, but I think maybe what would be more, what I'll do is start by just putting out a couple of north stars that reiterate a few of the things that we've talked about already. I think increasing focus on minimizing burden on patients while at the same time increasing their ability to contribute. We talked a little bit about how there's increasing choice and competition for patients about different sources of data that they can contribute to. I think the extent to which we live in a world where there are silos, no one is going to actively go out and contribute to 10 different disease registries within their condition. And so thinking about ways that we can simplify that process from a patient perspective is top of mind for me. I think it is also key to us recruiting diverse populations of patients and patient retention within registries.

And then in situations where the registry isn't entirely virtual, really a similar frame of mind thinking about sites and interaction at a site level. So what are the things we can do to make the process easier for sites getting from, here's another study that I'm being asked to do, to this is a study that I can show value to patients that can be relatively low impact to my clinic or my setting from a staffing perspective, from a cost perspective. And then also really from a technology burden perspective, I think sites are increasingly being asked to adopt many, many different flavors of e-consent systems or various sorts of things. And so these to me are things that are like north star, top of mind as we think about ways to expand the reach of registries in the ecosystem.

Evelyn Pyper:

I like it. I like it. Thanks Dan. And Heather, how about you, world where there's no constraints to what you can do with your registry?

Heather Fitzpatrick Medlin:

I think the ability to give back to patients or participants in a way that they can see the information more readily, perhaps more graphically, have timelines and being able to really empower them to make the best healthcare decisions with their providers, gives them a bit more understanding. And as I said, we can't dispute the fact that we do a lot of things in English. We need to broaden that. We can't assume people can read and write, so we have to make sure that we're able to provide this information perhaps in an audible way so that information can be shared for those that may not have access to information on the ready. I think that's our goal. And again, looking at how far we've come, it's pretty impressive. And I'm sure if we regroup in two years, we'll be talking about today being the good old days.

Evelyn Pyper:

We should indeed do that, regroup in two years and see if we've achieved any of these goals. That's great.

Heather Fitzpatrick Medlin:

Exactly.

Evelyn Pyper:

And Clara, how about you?

Clara Lam:

I think just to build on everything that Dan and Heather had said, I think if I had a wishlist aside from just capturing all this really wonderful information on the treatment, the journey, the patient perspectives, caregiver perspective, physician perspective, all of that, as a health economics person a little bit on the background, I'd also like to collect information to be able to address the financial toxicity that our patients are dealing with. There is no simple condition in the United States and everything seems to be expensive. So what can we do to help our patients address some of those concerns as well?

So if there were ways for us to be able to capture information on costs, disease progression, just the quality of life, just capturing a lot of this information just on all fronts for economic outcomes research to be able to quantify that and showcase if we can provide better treatment options for our patients earlier, can we delay disease progression? Can we delay the costs and the financial toxicity that comes along with that? I think that's another huge component of information that we don't necessarily have set up in this moment, but I think that could be coupled with the information that we are capturing in the registry would be incredibly valuable and just not available anywhere else. I think if I had my wishlist, I would add economic outcomes as well.

Evelyn Pyper:

That's a great one. Especially it feels like both a probably common researcher wishlist item, but becoming more of a potential reality in the world of value-based healthcare where there'll be certain things that aren't just a wouldn't it be nice if we could capture, it'll be a thou capture this or we're not going to reimburse. And so that just feels like such, again, let's regroup in two years and see how much of this is in fact necessary and not just innovative. Thank you so, so much to our speakers. Oh yeah, Dan, go ahead.

Dr. Dan Drozd:

One other quick follow up to that, which is probably obvious, but I think it's not just patient economic burden, it's caregiver burden as well. And so in many, many conditions that we work in, there's a significant burden on caregivers that is historically under captured. And so I think really reflective of, if part of our aim is to really understand the true impact of disease and the true patient journey, the caregiver aspect is key as well.

Evelyn Pyper:

Definitely. Thank you for adding that. Any other thoughts from the group before we head to audience Q&A? Awesome. Well I think we have some great questions waiting for us, so thanks so much to our panelists and we'll head into the next phase with Sydney.

Sydney Perlmutter:

All right, thank you very much for that insightful presentation. Now I'd like to invite our audience to continue sending in their questions or comments right now using the questions window for the Q&A portion of the webinar. I've already received some great audience questions, so I'll start with those. So our first question is, one of the challenges of collecting RWD is patient and clinician participation. What are the common behavioral science approaches used today to increase participation in our RWD collection?

Heather Fitzpatrick Medlin:

I can start. I think for us it's really, again, not making it something manual when it can be so simple to be able to behind the scenes move this, not to ask the same questions time and time again, to make sure we recognize the value of individuals that are participating is time and being able to not consistently ask the same information or collect the same information and be willing to share. I think that's the other part about running a program like we do, is that we're independent and so we're able to offer this to all researchers and academic individuals to support us or for us to support them. So we're able to make sure everyone gets a chance or a piece of the data if they'd like it.

Clara Lam:

And maybe ust to build off of what Heather said, I think the other thing about patients is that they tend to be a very altruistic population. They understand the condition of their disease and how it impacts them and their family, but they also want to help make sure, at least in the early breast cancer setting, that this doesn't happen to their daughter, their aunts, their sisters, or their friends. And so it's a huge incentive for them to contribute to the research because on some level, even in an aggregate form, they're able to help find, an additional step to finding that cure, an additional step to finding a treatment that works. And so I think that incentive is there as well. I think it's hard to put a value on having all of your medical records in one place, and that's another huge incentive as part of this decentralized registry that this platform that we're building, it just saves so much time and energy on the patient.

They don't have that kind of time and energy. They have other things they need to do. And especially for early breast cancer patients, a lot of the patients that we have are younger women of reproductive age and they have families, they have other responsibilities. And so to take this burden off of them, I think it provides an incentive for them to really participate so then they can really help in terms of the research that we're contributing and that they're participating in, but also to make their lives easier on the day-to-day I think is a huge thing.

Dr. Dan Drozd:

I totally agree with all of that. I think the percentage of patients across conditions that we work in who are motivated really by the ability to give something back, whether it's something that they will directly benefit from or something that might be, as you said, Clara, hoping that their daughters are not diagnosed with this condition, I think does become a particularly important motivator for a large percentage of people. I think the other thing is just being very thoughtful when we are asking patients to do something and provide data. I think we've all taken surveys at some point in time in the past where you get asked 27 questions and you know that you could have gotten the information you actually needed in a couple of questions.

And so I think something that we spend a lot of time thinking about is patient or participant burden in the context of PRO instruments, right? Just very simple things. Let's try to give a questionnaire that requires only 30 seconds of someone's time instead of them needing to sit down and do something for 10 minutes. We obviously see very different responses in terms of follow through and completion of those surveys and those different situations.

Sydney Perlmutter:

Thank you all for those responses. Our next question is, what functionality, if any, does PicnicHealth provide for storing and working with images such as MRI scans, etc?

Dr. Dan Drozd:

I'm happy to take this one. We do as part of our studies, collect raw DICOM images. So these are the actual slices from various radiographic studies, whether those are plain film X-rays or CT scans, MRIs, et cetera. And do have the ability then to de-identify that data and make it the raw imaging data available to research partners when that's relevant for a particular condition. Obviously there are situations where it is more or less relevant. And then consistent with our commitment to share back information with patients, we then make the same imaging available to patients as part of their PicnicHealth timeline and then that data can then be shared with their providers.

So you can imagine how important this is in a patient who has an imaging study, and the provider or center where that study is being done doesn't have prior studies to compare it to. It can be very, very difficult to tell is this lesion in a patient's breast cancer a new lesion or is this an existing lesion? Is it bigger or smaller than was present before? So make that very simple for patients and then make it simple for them as well to share it with their care teams and providers.

Sydney Perlmutter:

Thank you. Another audience member would like to know, for what types of studies or applications are you seeing the greatest potential for impact of these innovative registry approaches?

Heather Fitzpatrick Medlin:

I think from my perspective in the rare space, it's become so critical because there's just so many different types of providers and so many different tests and encounters that they have with physical therapy, occupational therapy, even within reports for the school system. The fact that we're able to aggregate all of that is really critical because it paints that whole picture of what an individual's journey is not just in the healthcare setting, but in all healthcare interactions.

Dr. Dan Drozd:

We can just add settings where the management of the disease is complex and occurs over an extended period of time. So multiple providers, multiple centers, we've all certainly had the experience of changing physicians that we see because our insurance has changed. That's a reality of navigating the healthcare system in the US. And so I think really conditions that are chronic in some sense, that have a prolonged care journey, multiple providers involved either in the path up to diagnosis as we talked about the value of retrospective data earlier in the presentation, but also prospectively as patients are being followed over time across a variety of providers. Those end up being characteristics that I would say where we feel like this approach is particularly important.

Clara Lam:

And I think it also speaks in a way to some measure of precision medicine as well. At least I think in the early breast cancer space, for example, we're following patients for quite a long period of time. And their treatment journey is complicated and the continuity that we can capture by being able to find these patients is not based on a site or based on a particular physician or being able to trace back to their source. I think it's very helpful to be able to basically catalog all that information going forward. And as new treatments become available, new biomarker testing becomes available, to be able to incorporate that I think it really helps understand the ever-changing landscape that we're looking at in cancer care. I don't think this is specific to cancer, I think in general, I think just any condition out there, I think just being able to have the most current information and being able to make it specific to that patient. That's really important.

Sydney Perlmutter:

Thank you all. Our next question. In many cases the research questions that need to be answered using a patient registry are not just disease specific but also highly drug specific. Are there ways that these novel registries can be set up or enrolled to ensure sufficient sample size on the treatment of interest?

Evelyn Pyper:

Maybe I'll start out too just with thought out loud and partial answer to this question, is I often think that we think of disease registries and drug registries as two different things, but it is interesting when you start to dial into what folks mean by a drug specific registries, there's always still going to be that need to compare drug X with a comparator Y on the market, right? It's very rare to have the need to just study one treatment. And so I started to recognize that the real difference is less about are there more than one type of product being studied in patients with the same disease, but more about is there the granularity of data that's important to those treatments available?

And that seems to be, and Clara would love to know your thoughts on this too, the sense that we've gotten from life sciences partners where it's less about excluding patients on other drugs and more about is there at least a depth of data and enough patients on our treatment of interest to make this a worthwhile study type and an investment?

Clara Lam:

No, absolutely. And as a representative of AstraZeneca, obviously we are very keen on understanding what's happening to patients who are on AstraZeneca products. But as a step beyond that, our dedication to really understand breast cancer research is moving forward, and especially in the early breast cancer space, there's a lot of options available. And I think it's very important that we think bigger and not just in the sense of very statistical, you need to have proper sample size to do anything, because last time I checked you cannot do a robust analysis on five patients as much as we seem to think we can. The reality is that the more patients we have, the more represented they are, and the more we can understand with some confidence that the numbers that we're seeing, the results that we're seeing, outcome measures, that they're more reliable and that they really do speak to the population that we're trying to address.

So as much as it's important to understand a specific drug registry, I think the bigger research question that can involve that, is what's happening in our patient population that needs to be addressed on a bigger scale? And then from there you have specific research questions addressing some of those subgroups, and that could be an analysis that can be done.

Sydney Perlmutter:

All right, I think we have time for one last question and that is, what do the panelists think about the application of these approaches to pregnancy registries? There are many challenges with how these are done today, and it's an area of research that could benefit from innovative methods.

Heather Fitzpatrick Medlin:

I'm going to jump in first because that was my first registry I ever worked on, were pregnancy registries, and so they're still very near and dear to my heart. Obviously they've evolved a lot over time and we've watched the expansion by looking at a single product exposure, a class of products, following from a disease perspective. It gets a bit more complicated as we know, because if a baby's born, you have a separate set of medical records that are cleaved off and you want to be able to follow that information. You also have prenatal information that you want to follow with additional testing after birth. But I think, again, this is a place where as we know even women who are pregnant maybe seeing multiple practitioners. It's really important to have all of that. There could be a standing disease that they're seeking treatment for outside of their obstetric care during pregnancy.

I'm always excited to see the pregnancy registry space come to the forefront, but I think this is definitely well suited for the collection of data around that.

Sydney Perlmutter:

All right. Well, thank you very much for those answers. We've reached the end of the question and answer portion of this webinar. If we couldn't attend to your question, the team at PicnicHealth may follow up with you or if you have further questions, feel free to direct them to the email addresses on your screen. Thank you everyone for participating in today's webinar. You'll be receiving a follow-up email from Xtalks with access to the recorded archive for this event. A survey window will be popping up on your screen and your participation is appreciated as it will help us to improve our webinars. Now, please join us in thanking our speakers, Heather Fitzpatrick Medlin, Clara Lam, Dr. Dan Drozd, and Evelyn Pyper. We hope you found this webinar informative. Have a great day everyone.