Patient empowerment, digital innovation, and rare diseases

How is clinical research becoming more patient-focused and more convenient for patients to participate in clinical trials? Why is a decentralized approach especially important concerning rare diseases? And how will digital innovation advance the way clinical research is conducted? We will learn those answers and more in this episode with Scott Schliebner, an innovative life sciences executive with 30 years of experience across the biopharma, CRO, medtech, and non-profit sectors. With a strategic and consultative approach to building and growing life science businesses, Scott has developed relationships, partnerships and collaborations that have driven commercial success. His vast experience includes leveraging real-world data and real-world evidence (RWE/RWD), leading technological innovation, and driving patient-focused paradigms to accelerate clinical drug development. Scott is an active board member, advisor, and mentor and his passions lie with infusing data and innovation into life sciences organizations—especially where rare diseases are concerned. He is currently the leading executive at Rare Clinical. -------------------------------------------------------- Episode Transcript: 00;00;00;07 - 00;00;24;21  How is clinical research becoming more patient focused and more convenient for patients to participate in? Why is a decentralized approach especially important when researching rare diseases? And what is the most likely future for how clinical research is conducted? We'll get the answers to all that and more on Research in Action.     00;00;24;23 - 00;00;48;24  Hello and welcome back to Research in Action, brought to you by Oracle. I'm Mike Stiles and our guest today is Scott Schliebner. Scott is a leader and innovative life sciences executive with 30 years experience across biopharma, CROs, medtech, and nonprofit. He's developed relationships, partnerships and collaborations that have driven commercial success with a strategic and consultative approach to building and growing life science businesses.     00;00;48;27 - 00;01;16;06  Scott got a lot of experience, including leveraging real-world data and real-world evidence, leading technological innovation and driving patient focused paradigms to accelerate clinical drug development. And he's an active board member, advisor and mentor, and he's all about infusing data and innovation into life sciences organizations, especially where rare disease is are concerned. And last but certainly not least, Scott is the leading executive at Rare Clinical.     00;01;16;09 - 00;01;35;01  Scott, we're glad to have you with us. Thanks for letting me grill you with all these questions. Thank you, Mike. My pleasure to be here with you. Well, let's start at the beginning. A fine place to start. What got you into the field of clinical research and drug discovery and why this special focus on rare diseases? Yeah, great.     00;01;35;01 - 00;02;04;13  It's a great place to start. I think, like a lot of my colleagues in this clinical research, clinical drug development profession, a lot of us sort of find our way into this field as there aren't necessarily a lot of like formal training programs or pathways necessarily. So for me, I was in graduate school, I was doing some more like I would call more basic science, more basic research that I found my one day struggling to.     00;02;04;16 - 00;02;22;00  As I was writing a grant for a professor, I found myself struggling to justify why, why this was really important. I kept saying to myself, Yeah, this doesn't really seem very applied. Is this really make a big difference? I, I can't convince myself this is critical. How am I going to convince a funder of our grant that this is really important?     00;02;22;00 - 00;02;44;17  And it kind of was a little bit of a light bulb moment for me that made me realize while I loved the field of research, I needed to be doing something that was more applied and could have a little bit more of a direct impact upon people. So it sort of led me to the clinical drug development space and clinical trials, and I got started back.     00;02;44;17 - 00;03;12;21  It's been a couple of decades now as I've been around for a little while, but it got started in a sort of like a biotech clinical research setting, helping to design and manage clinical trials and have been sort of engaged and passionate about this industry ever said. So it's been it's been a fun ride. But again, like a lot of people in this space, I think I stumbled into clinical research, maybe not accidentally, but, but, but there's not an obvious clear entry point for some of us.     00;03;12;23 - 00;03;33;25  Yeah. So I get that you, you got into the bio research space and drug development and those kind of things develop that interest. And I get that you wanted to make a real impact that you could feel like you were making a difference. Is that where the focus on rare diseases came into play or when did that? Yeah, thanks for following up on that part of the question.     00;03;33;25 - 00;04;12;02  I think that, yeah, after having been in the industry for a little while, you know, about, I don't know, this was probably like 12 years ago or something. Rare diseases at that time were really still a little bit. They weren't certainly a hot and sexy topic like they are today in 2023. But I came across some patients, I came across some patient groups, and I also came across a couple of clinical trials and I realized that what we were trying to do and what was required really to function and develop drugs in the space of rare diseases really required, honestly, a completely different, really way of operating a completely different paradigm than what we     00;04;12;02 - 00;04;48;16  were doing in most of clinical drug development. And with, you know, with our biopharma industry being pretty risk averse. That's a theme I think you'll hear come up probably a lot today. In our conversation. There hadn't been a lot of appetite or initiative around trying different approaches or looking at things differently. And these rare disease studies for sort of a countless sort of logistical and medical and scientific reasons really require a very different approach of, you know, you're talking about small populations that are geographically dispersed.     00;04;48;16 - 00;05;23;21  You're talking about patients that may have they may have to go through a diagnostic odyssey. A lot of people don't know about these disease states. There's a host of challenges that kind of come together and create a scenario that is even more complicated than your average challenging clinical trial. So also, when you look at the fact that there's something like 10,000 individual rare diseases individually, they're all rare little sub populations, but taken together they make up about 10% of the U.S. population and about 10% of the global population.     00;05;23;21 - 00;05;50;24  So it's it's a big area of unmet medical need. When you look at it from a big picture perspective, when you drill down into individual disease states, individual patient populations, you notice that these patients and families don't have any therapies, they don't have any treatments, they don't have a lot of hope sometimes. And clinical trials. And this world is really their only source of hope at times.     00;05;50;24 - 00;06;10;22  It's less of an experiment and more of a care or treatment option for rare disease patients. And so I found myself really immersed and passionate about this area and felt like it was a space that really needed new approaches. And I've been happy to kind of delve into that and try to make a difference there. And what is the state of that research like?     00;06;10;24 - 00;06;39;24  Is there reason for people with rare diseases to have hope? For instance, there are people in my family who have ankylosing spondylitis, which is a relatively rare form of arthritis. Is it appropriate for them to have hope that in their lifetime something's going to happen? Or are these populations so small and the research to develop drugs for it's so difficult that, you know, we're looking at 50, 60 years in the future before we make any progress.     00;06;39;25 - 00;06;55;20   Yeah, it's a great question. I mean, there really is a really broad spectrum here when we talk about rare diseases. We have such a such a large number of them. I think that the short answer is there is hope. And in a lot of cases that hope is in front of us or is on the very near horizon.     00;06;55;22 - 00;07;18;09  There certainly are other scenarios where another disease states where it's going to take a while and that hope is a little further out to be seen. But the good news is that we've well, there's been a lot of mobilization, there's been a lot of innovation and a lot of attention devoted to rare diseases over the last decade, 15 years, we've seen a lot of drug approvals.     00;07;18;11 - 00;07;39;24  We've seen a lot of companies, we've seen a lot of investment in biopharma biotech firms come into the rare disease space, whether they are small little biotech startups or whether they're the big pharma of the world. Everyone sees this as an opportunity to help develop drugs and help people. And in an area that really needs as much help as we can provide.     00;07;39;24 - 00;08;15;29  So there's a lot of hope. Some of these disease states are a little more clear than others. We understand the biology and the genetics, and maybe we can develop targeted therapies that help these patients some of these other more obscure, ultra rare or nano rare diseases. We're still learning who the patients are and how do we diagnose them and before we can develop a drug and show that it works and that it's safe in those populations, we need to first even understand a little bit about the natural history of some of these diseases and how they progressed kind of on their own and what kind of end points we would want to choose and some     00;08;15;29 - 00;08;37;27  things like that. But the bottom line is that there's a lot of hope, there's a lot of progress, there's a lot of activity, there's a lot of investment. I think there's a fair amount of awareness. We've seen a lot of progress here with people, with people and organizations and industry really getting into the space. Of course. With that said, there's a lot more that we can be doing.     00;08;37;27 - 00;09;02;21  There are a lot of disease states that really need some more attention and more funding and more research. But from where I sit, we've made some great strides and I hope to kind of keep accelerating that progress. Well, science is kind of inherently a social enterprise, but despite that, scientists and clinicians seem to work mostly behind closed doors, maybe even a little too far removed from the people they're actually working to help.     00;09;02;23 - 00;09;24;27  The pandemic changed a lot of things, but for one thing, Big pharma got kind of pushed out of its risk averse comfort zone because they had to speed the science and adopt more openness. So what do you think COVID did to innovation in the clinical research space? What changes are permanent and which ones aren't? Yeah, this is a fantastic topic.     00;09;24;27 - 00;09;57;11  We could we could spend a lot of time on those, I think. Well, necessity being the mother of invention, I think that COVID presented a lot of unique challenges and picking on my risk averse colleagues who may not want to necessarily try something new or go out on a limb with some sort of more risky, unproven approach. COVID forced us to reconsider how we were doing things, and it forced us to keep clinical trials going in a in a manner and keep them operating.     00;09;57;11 - 00;10;32;15    When we couldn't go to clinics or go to hospitals or when we had to social distance. And it forced us to really rethink a paradigm that had not really changed in many decades. So if you rewind a little bit to pre-COVID, there was several movements out there around creating more patient focused approaches. So this idea that you mentioned science being inherently a social enterprise, I envision a lot of clinical protocols and clinical trials being they're often developed in a little bit of a bubble.     00;10;32;18 - 00;11;01;01  Sometimes I'll joke and say in a conference room in New Jersey, right, these clinical trials come to life and are sketched out and designed in a little bit of an insulated bubble of sorts that don't really take into account the perspective and the input and the needs and the voice of the end users, namely the patients themselves. So similarly to the fact that, you know, you may have an iPhone sitting there on your desk next to you.     00;11;01;04 - 00;11;26;27  Apple, of course, didn't design a camera to put on their phone and say, let's see if people want to use a camera. The camera was designed, of course, by consumer demand and designed for the people using it. Ironically, even though science and even though clinical drug development is completely dependent upon patients participating in clinical trials, we rely on them and their data to move things forward.     00;11;26;29 - 00;12;06;22  They're very rarely considered actually, in the design process. Right. That's the irony, is that we rely on them. We must have their participation, but they're kind of an afterthought historically when it comes to designing a clinical trial and thinking about how to implement it. So that being sort of the baseline of how we've operated COVID hits and all of a sudden our world is interrupted and some of these novel approaches that had been being developed, these mobile health platforms, early COVID, we were talking about how can we make clinical trials, quote unquote virtual or hybrid with some of the language we were using.     00;12;06;24 - 00;12;35;18  How do we instead of requiring patients to maybe travel long distances to a clinical site or an academic medical center? Sometimes it could be a weekly visit for 52 weeks. A lot of times that's not going down to your neighborhood primary care physician. It might be driving into Manhattan and going to Memorial Sloan-Kettering every Friday afternoon and taking time off of work and away from your family and your children to participate in the clinical trial.     00;12;35;21 - 00;13;02;09  The bottom line, clinical trials were really not designed for patients, often not realistic and often not feasible. So when we ran into COVID and the challenges that kind of shutting down hospitals sort of created, it forced us to adopt this what we ended up naming decentralized clinical trial paradigm, and it forced us to think through, well, how do we bring clinical trials to patients themselves?     00;13;02;11 - 00;13;25;15  So this was a long, long overdue need that was out there. Again, we've been operating in a little bit of an archaic fashion for quite a while, putting out studies that were not realistic for patients. But when the world around us sort of crashed down and we needed a new approach, we adopted this more patient focused paradigm simply out of need, I think.     00;13;25;18 - 00;14;05;21  So there's been some traction with this. Decentralized clinical trials have become a common term in this industry. We have the Decentralized Trials and Research Alliance that was formed in 2020. There's a lot of companies on investment that have come up to develop this new paradigm of, instead of these critical end users, patients, instead of having them have to travel long distances and inconvenience themselves and for a scenario to be very hard for them, let's create something that revolves around them, that's create a patient centered, patient focused approach where we bring trials to patients in their homes.     00;14;05;21 - 00;14;28;07  And so we do this via some tools like apps on our phone and a mobile health platforms. We do this, of course, now via telehealth and things like that that have become much more routine and regular. We're able to collect data remotely. We're able to push out sort of questionnaires and quality of life and clinical outcome assessments to patients wherever they are.     00;14;28;11 - 00;14;53;23  We're able to send nurses to their home to help them administer drug or check on how they're doing or evaluate their symptoms. So this transition that COVID has sort of forced us into, again, necessity being the mother of invention has forced a more patient focused approach that I personally think we've been really long overdue. So I think of that as a little bit of a silver lining of the pandemic.     00;14;53;25 - 00;15;24;08  There's been some good progress, I think, made as a result of that. But these technologies that you're talking about that are used to monitor these patients in these trials, like maybe wearable technologies or whatever, what's the reliability level of that? Does a lot of this rely on the patient just reporting accurately what's going on? Well, as we've come out of the pandemic now, the interesting sort of dynamic is does this new model really stick around when it's not as needed as it was during COVID?     00;15;24;11 - 00;16;06;16  Do we continue to move forward in this new innovative approach, or do we revert back to the way it was? And along with that, are these sort of challenges maybe, and complications that come along with this decentralized approach, like remote data capture, lots of data sources coming from various areas, various directions, wearables, as you've said. And there's also this kind of push also to not only engage patients where they are, but also to collect data in a little bit of a real world setting in this, you know, real world evidence, real world data, you know, kind of concept around, you know, we're conducting clinical trials that are controlled and we're we're looking at specific variables     00;16;06;16 - 00;16;25;12  and we're looking to evaluate safety and efficacy. But at some point, if this new therapy is going to be approved and patients are are using this in their daily regular life, how it's better for us to also know what that's going to be like, like to study that real world experience now even in the context of a clinical trial.     00;16;25;12 - 00;16;44;27  And so you are even seeing regulators like the FDA encourage the collection of more real world data for that to be used to supplement more controlled clinical trial data. So you're having a little bit of a mixture here and a little bit of an evolution, I would say, in terms of the data and evidence that we're bringing in.     00;16;44;29 - 00;17;12;08  But your point, there's there's lots of challenges with that as well. Now we have a lot of different technology platforms. We have a lot of data sources that need to interface and communicate and integrate. There can be complexities with having lots of different technology platforms for clinical sites and patients to use. You mentioned the reliability component. Are we using instruments that are validated and have been used over and over and we can trust?     00;17;12;10 - 00;17;39;17  So with this new approach of collecting more data, collecting data from different sources, collecting real world data, we also have to make sure that we're integrating it well, that we're not overly burdening both the clinical sites and the patients. But the trend that we're seeing is that each year now, clinical trials become a little bit more complex. They're collecting more and more data each year.     00;17;39;20 - 00;18;10;11  Data points in these clinical protocols are becoming more extensive. Right? So, you know, that's more data that becomes a little bit more of a burden for a lot of people. And it also takes us back to this, this mantra of, okay, let's remember the patients again, because if you're going back to my example, if you're a scientist or a drug developer in a conference room in New Jersey, you may very well want to collect all kinds of interesting data points that you think are going to be informative to you.     00;18;10;11 - 00;18;41;03  And if everybody sort of pours in all their sort of data requests, you can see this sort of ballooning, mushrooming clinical trial becoming overly burdensome. And let's not forget that each of those measurements require something of a patient. So there needs to be some kind of balance here between gathering more data, real world evidence controlled clinical trial data, but also remembering that, you know, the patients are producing that data and let's not make things overly complicated here.     00;18;41;03 - 00;18;59;00  So there's a little bit of a dynamic there that I feel like we're right in the midst of it. I'm interested to see how this kind of plays out a little bit. Well, yeah, And that's being seen everywhere. There is a ton of data. Companies are drowning in data. Some of them know what to do with it, how to handle and process it, process it.     00;18;59;00 - 00;19;22;20  Some don't, but it is crucial. There are challenges and benefits to being data focused, especially where sensitive data is concerned. Things like evolving standards. I mean, is too much data a problem? Are these supposedly connected platforms causing redundancies and other headaches? Yeah, I mean, I think so. Probably depends on who you ask, right? I'm sure a lot of people would say more data, the better.     00;19;22;20 - 00;19;45;16  Let's get everything we can. It'll inform us better on a patient experience or on the effects of a drug. At the same time, though, right, there's logistical challenges. Where are these pipes of data running into in the while? It's happening across all kinds of industries in the clinical research, clinical drug development space, you also have the added piece of patient privacy.     00;19;45;19 - 00;20;11;07  And in this country, HIPA and the need to, you know, keep patients data safe, private, de-identified as well. So when you're implementing different platforms that are collecting data at a clinical site, but maybe also at a patient's home and also maybe a wearable as a patient is, you know, walking around and just their daily life and collecting data via that.     00;20;11;09 - 00;20;34;22  Where do these all come into? How are they getting analyzed? Is it secure? We can very easily and very quickly make things more complicated than we needed to. I personally think we should be mindful of not collecting every possible data point we might have an interest in. We might not even know what we want to do with that data today, but that might be nice to have down the road.     00;20;34;24 - 00;20;56;18  I do realize that's great for science and discovery and exploration, but again, in the context of a clinical trial, you know, the environment we're in right now with clinical trials is that more than 60% of all clinical trials are behind schedule due to enrollment. And even when patients do enroll, we also have a lot of patients dropping out and not staying on studies.     00;20;56;18 - 00;21;27;09  So the retention of patients is also a problem. And so while we balance collecting more data, while we balance more complicated, complex clinical protocols as we're increasing the burden at times on patients and I use this patient word a lot, but really we're talking about people, right? We're talking about people like like Mike and I who have jobs, who have lives, who have hobbies, who have families, work people, and we have other commitments in our lives.     00;21;27;09 - 00;21;50;24  And we can't just drop everything to participate in a clinical trial. So the the concept of if you build it, they will come doesn't really apply here really anymore. And we need to be thinking about building things that will appeal to people and be realistic and feasible for patients, both in terms of time, commitment and travel and there might be you know, there might be emotional issues.     00;21;50;24 - 00;22;14;12  There's all kinds of domains of burden that we've learned. And this balance of like, wow, we can collect data from anything anywhere, any time, that's great, but let's maybe we need to real that and a little bit here and balance that out with what can a human being really handle And are we overly burdening patients because we're finding that people are just declining to participate, which is actually.     00;22;14;14 - 00;22;40;16  So this idea of more data better can actually be slowing down drug development in a way because clinical trials are delayed now due to enrollment. So there's an interesting dynamic there that I think progress in this area, real world evidence, remote data capture, lots of data sources, is also creating a more complex environment that I think is giving giving some patients pause to like before and roll in that study.     00;22;40;16 - 00;23;09;24  I'm going to I'm going to think twice. So really interesting thing that's really very fluid and dynamic. I think this is kind of changing on a monthly basis these days. Well, let's turn back to rare diseases, because I think we can all agree plenty of innovation is needed to make a dent in those. But what kind of innovations and as I hear you talk, it's like, okay, there's challenges to this patient centered research, decentralized research as it is now.     00;23;09;24 - 00;23;37;12  You're talking about rare diseases where the population is small to begin with, it seems. I mean, what are the most effective things we could be doing? Is decentralized trials the answer? Yeah. Well, I think, you know, in that rare space, you're right, clinical trials are challenging. Clinical trials are behind schedule. Clinical trials might be overly complex. And I think that happens in worlds where we could be dealing with a fairly common, you know, diabetes or arthritis study.     00;23;37;14 - 00;24;03;10  When you overlay those challenges on a disease area and on a patient population, that is rare or ultra rare, you know, it becomes magnified, it becomes extra challenging. You know, some of those challenges include things like, well, by the nature of the name rare diseases, there's very few patients with that disease. Sometimes those patients and some of these disease states are very, very small.     00;24;03;12 - 00;24;25;08 And people are, of course, geographically spread out. So if you wanted to learn more about a disease, study it, run a clinical trial, what have you. You know, you're not just opening up a clinical center in New York City and enrolling all those patients. You might be needing to go to multiple countries around the globe just to be able to find a couple dozen patients.       00;24;25;10 - 00;24;50;26  So you've got a dispersed and few and far between patient issue is challenging to come to come up with. And again, by the nature of, you know, the definition, there are physicians and our principal investigators in clinical research sometimes don't see a lot of these patients. So for those of you listening that have that know a little bit about the rare disease space, you'll know that that's the symbol of rare diseases is the zebra.     00;24;51;02 - 00;25;13;17  And the reason for the zebra symbol is, you know, back in the fifties, there were some physicians that were training medical students, and they used to use the phrase that, you know, trying to teach their medical students that, you know, keep things simple, Don't overly think it don't overly complicated. If you think you're sort of stumbling upon a diagnosis, you're probably right.     00;25;13;17 - 00;25;34;13  So the phrase was, you know, if you hear hoof beats, it's probably a horse. Right. And the rare, rare disease community, of course, sort of shudders at that idea and says, well, hold on a second here. You know, if you hear hoof beats, don't forget that 10% of those diseases could be a zebra. Right. And the zebra or the rare disease has been one to not forget about.     00;25;34;13 - 00;25;55;17  So we we also have challenges with physicians that may come across a case or a patient like that once a year or once every couple of years. So there might be a limited knowledge about the disease and the care of those patients. It also makes those patients being diagnosed initially really, really challenging. I mentioned earlier the diagnostic odyssey.     00;25;55;19 - 00;26;19;18  It's not uncommon for a rare disease, patient or family to see seven, eight, nine, ten physician ones go to all kinds of different hospitals and institutions and have a variety of sort of tests done to figure out even what they have. Right. They're craving a diagnosis so they can figure out what's going on with them so that then they can start to think about how do I get this treated.     00;26;19;18 - 00;26;44;22  So there's there's challenges around diagnosis, There's challenges around how do you develop a drug in this new rare disease where clinical trials never been done before? What what are the end points? What kind of benefit do we need to see if it's a genetic or life threatening disease? Does that change our, you know, the the efficacy safety sort of ratio we're willing to accept?     00;26;44;22 - 00;27;10;07  Right. So whole bunch of challenges there in that space. Again, though, I think there's been amazing progress. We've even seen things like the FDA and other regulators be more flexible and encourage innovation. They realize it's hard in this area and, you know, there aren't that many patients and conducting clinical trials and finding those patients and collecting data is extra challenging and we're not going to get that much of it.     00;27;10;07 - 00;27;53;14  So I've seen the FDA be very flexible and responsive and kind of partnering and collaborating with industry to help because we all want to kind of move these therapies forward and help these patients. So lots and lots of progress, but extra challenges. And I think it really creates an environment here where the rare disease space has really been the tip of the spear, both with decentralized clinical trials, with maybe some of these remote data capture paradigms, because again, necessity being the mother of invention, just to just to manage to do this in the rare disease space does require us to think differently and approach things differently and be creative and I think the     00;27;53;14 - 00;28;13;25  success we're seeing in this space, I'm hoping that other parts of our industry see that and also can adopt a little bit of innovation in their own little areas as well. Well, I'm always looking for good stories. Do you have some examples of where patient driven data really changed the trajectory of drug discovery for a specific rare disease?     00;28;13;27 - 00;28;49;06  Well, there's been a lot of good examples of this patient focused approaches that we're talking about, this idea of get outside of that conference room bubble, incorporate the patient voice and perspective into your drug development just to the point of. So if you're developing a, you know, a drug and you have a clinical trial, you're looking at endpoints, you're looking to write to measure the effect of your drug on, say, some kind of performance factor, You know, when you look at how we're going to develop that, the FDA may want a certain endpoint that is validated and it's the gold standard.     00;28;49;06 - 00;29;21;15  And they're looking for industry to kind of measure that and hopefully exceed that in their trial. The drug developers and industry, you know, they're often thinking about what's more what's realistic. Does it take us five years to achieve that endpoint? Is there something that's sub or is there a surrogate, is there a biomarker? And then interesting, when you talk to patients, you know, you hear this a lot of diseases, well, we'll use what's called the six minute walk test, which has been talked about ad nauseum as a measure of a patient's sort of performance.     00;29;21;15 - 00;29;44;20  The how far can you walk in the six minute space? And if you have, you know, any kind of respiratory or cardiac condition or maybe even something neuromuscular going on, it really limits your ability to kind of perform and walk in that that test that's been used as a standard for a time that's very controversial, that, you know, the FDA is required at a Times or industry has wanted to pursue it.     00;29;44;23 - 00;30;06;14  And then when you actually sometimes talk to patients themselves and say, you know, they'll look at the endpoint in a trial as measuring and say, well, that's not really relevant to my life. You know, if I'm able to walk across my kitchen and reach my arms up and get something out of the cupboard, that would be a dramatic, meaningful benefit to me, right?     00;30;06;20 - 00;30;27;22  Just the ability to do that on a day to day basis would change my life. And is there a way to measure something there that's maybe a little bit more relevant for patients, but also balances out what our scientists and our regulators and our industry professionals want, I think is so there's often a dynamic there around what are we going to measure?     00;30;27;22 - 00;30;53;07  There might even you might even argue there's a there's another arm in there that's maybe our our payers, right. Are insurance companies, what are they wanting to see, What's the benefit there? Because this drug will cost something. So it can't just be safe and we think it works. Is it working enough? Is the cost benefit enough? So there creates multiple kind of perspectives on how do we measure the effectiveness and and from what from whose perspective are we measuring it.     00;30;53;07 - 00;31;18;16  So that's pretty interesting. You're seeing more and more, you know, even in FDA new drug review meetings, you're seeing patients come up to podiums and share their experience on the trial, share their experience on the difference this drug made. There's been a lot of, you know, in the muscular dystrophy space, it's a really challenging disease, and drug development has been exceedingly difficult.     00;31;18;16 - 00;31;39;16    So it's been a lot of interesting stories there around not only the six minute walk test, but also just hearing patient voices, hearing the impact a drug has had on a patient or a family or a child. And there's often some competing data here around, well, the seemed to work for patients, but it didn't hit the primary endpoint that we wanted it to.     00;31;39;16 - 00;31;59;15    Yet there's some benefit here and we end up in some kind of gray area. And it's been an interesting kind of discussion and dialog we see playing out in those areas. So yeah, kind of competing priorities in a way. How do we find like what works for everyone? Now I'm going to ask you to put on your Nostradamus hat and predict the future.     00;31;59;23 - 00;32;22;05  Thinking about how things are trending now, what role will patients have in the next ten years of drug discovery? Will it be a story of, Hey, we found better ways to do decentralized trials and involve patients and that? Or does it shift to, hey, most of this stuff is done in silico with AI and that's how we do our research.     00;32;22;07 - 00;32;48;27  Yeah, fascinating. Yeah, I think the patient focus, the emphasis on being patient focused and considering patients, I feel like, you know, ten, 12 years ago was a really novel concept. I think we've made incredible strides in that area to where that's not a foreign idea anymore. You still may have some some people in the industry questioning the value of that, or can patients really inform their disease?     00;32;48;29 - 00;33;10;17  You know, a lot of us think the patients are truly the experts in their space. And there's been a lot, I think, to be there's advance things that informed us a lot by including that patient voice, patient perspective, bringing patients and patient panels to the to have a seat in that New Jersey conference room I referenced. Right. To actually provide their input early.     00;33;10;17 - 00;33;34;19  We're starting to see that more and more. And I think that's having impact and progress on making sure that we're creating things that are feasible for patients. So, you know, my crystal ball here on my desk sees us continuing to do more and more of that. I think there's a nice trend. I think rare disease, again, has been a space where we've kind of led the charge with that out of necessity.     00;33;34;21 - 00;33;56;02  But I think you'll see that extend into most other therapeutic areas. It's logical, right? It's good science. Consider your end user. Consider the people you're trying to help. Make sure what we're building or delivering or or trying to execute works for the people. You know, it's our Apple. Our iPhone works for the people who are actually using it right?     00;33;56;04 - 00;34;21;25  So I think we're going to see more and more of that, the decentralized clinical trial sort of paradigm that popped up as a necessity as a solution during COVID, you know, really has been like the hot sort of term and topic in our industry for the last two years. Again, if you picture if you if you zoom out, you see an industry that is risk averse and has been conducting clinical trials the same way for several decades.     00;34;21;28 - 00;34;45;25  We have a pandemic, we adjust, we adopt some new technologies. You know, from where I sit, I see this pendulum swinging back toward the middle a little bit. I feel like decentralized clinical trials are being looked at, maybe with a little more scrutiny and skepticism. You know, was this something that we just had to do short term? Is there really value in doing things that way?     00;34;45;25 - 00;35;19;25  Is there really are a slide back to your earlier point, Mike, Are we are we making things more complicated at times? Right. When you start to think about a patient focused approach where we collect data remotely, where we have nurses visit a patient's home, where we have things on telehealth, maybe we go into a clinical site as well, kind of introduces a few more different variables, and it makes not only the data more complicated, but if you're a patient, you might suddenly have six, seven, eight touch touchpoints of Who do I contact?     00;35;19;25 - 00;35;40;10  Is that the is it the help desk for the app or is it my nursing group or is it the clinical site or is it the sponsor or the CRO? So we're I think we've made some things a little bit more complicated and I think some of the risk averse folks in our industry are sort of looking at decentralized clinical trials saying, I don't know about this.     00;35;40;10 - 00;35;58;15  I wonder if maybe we're going to this pendulum will swing back and maybe all clinical trials will have maybe a little bit of a DCT component in them, maybe a little bit of remote data capture, maybe a little bit of consent, maybe a little bit of telehealth, and maybe that'll be just kind of the way we do things.     00;35;58;17 - 00;36;15;28    And we won't be looking at a regular clinical trial or a full DC trial. It'll be a little bit maybe about a mixture would be my thought so, But I do think we're going to continue to emphasize patients more and more. I think there's value in that. I think it's important. I think it's the right way to operate and the right thing to do.     00;36;16;01 - 00;36;33;16  Again, I think in any kind of industry, right, you want to be paying attention to your to your customers, right. And thinking about what they need and what's realistic for them. And I think our industry has a long way to go still in that area. So but again, with my crystal ball, I'm encouraged. I'm excited about where we're headed.     00;36;33;19 - 00;36;54;21    Well, getting back to the technology, especially how researchers figure out what tools to use, making decisions like legacy on premises systems versus cloud, what do you see? Are there mostly hybrid strategies out there, or is everything moving to the cloud? What are the pros and cons of each? Well, I'm the strongest technical person when it comes to things like that.     00;36;54;21 - 00;37;15;18  I do tend to focus on how we can collect data with patients, and I do a lot with wearables and some of these mobile platforms. I mean, it feels like, you know, the people I talk to, it feels like the legacy and on premises systems are, you know, are going away. It feels like everyone's in the cloud. Granted, I don't know, Mike, I live in Seattle.     00;37;15;18 - 00;37;37;16    I have Microsoft and Amazon down the street from me, so maybe it's just a little bit of a a bias with where I am geographically, but it feels like the cloud is where everything is going. I'm sure there are some folks listening who know a little bit more about the details than me, but that's my take. Yeah, well, you have been a longtime partner of Oracle, and in fact, you were at the Oracle Health Conference in September.     00;37;37;18 - 00;38;07;16  You're often participating in Oracle Life Sciences webinars. And I guess this goes back to the question of, you know, what is the industry's role in this thing? What has it been like working with Oracle and what role do you see companies like that playing in clinical research and drug discovery? Yeah, well, it's been a great partnership working with Oracle, both working with folks like yourself and your colleagues, but also leveraging a lot of the good technology solutions that Oracle developed.     00;38;07;19 - 00;38;38;17    I think that, you know, not to be a broken record, but again, risk averse drug development industry here. I think that a lot of innovation can occur from organizations like Oracle all pushing forward new technology, new concepts, new approaches. I'm a strong believer that the innovation in the space occurs from, you know, technology companies, service providers, bringing new solutions to biopharma sponsors.     00;38;38;17 - 00;39;05;18  So Oracle's been a group like that that has moved forward with different electronic data capture systems, you know, sort of the regulatory safety database solutions, things like CTMS's, the platforms that are being created are making us more efficient, they're making us more effective When it comes to the trials themselves. They're allowing data to be captured and integrated better from lots of different sources.     00;39;05;21 - 00;39;29;15  We talked about those challenges to that. But I think the direction is is a really good positive one. So Oracle and, you know, organizations like Oracle, I think are the ones really driving that. They're the ones to be to present new bright, shiny objects, new innovative technology and new solutions to pharma, and to say this will help move things along faster for you.     00;39;29;15 - 00;39;52;05     And with the you know, what the pressures around, you know, right now, the pressures around fundraising, the pressures around firms, maybe like prior noticing different sort of compounds in their pipeline, the challenges in the industry right now, I think everyone's looking for better, smarter, faster solutions. And that's where I think some technology and some innovation can come in.     00;39;52;05 - 00;40;15;01   And I think that's where Oracle will continue to make a really, really strong difference. Well, embrace my own shortcomings as a host. I never assume I've properly picked my guests brains. So what are you thinking about most these days? What's the big question or challenge you think we're facing? Or maybe there's just something you think it's important for our brilliant listeners to know.     00;40;15;04 - 00;40;35;25  And oh boy, we've covered some really interesting topics around, you know, data being captured. Is there too much data out there? How do we integrate data from all these all these different sources? I feel like that is a that is a topic that won't be going away anytime soon. Right? That is something that I think we're going to struggle with for a while of.     00;40;35;28 - 00;41;16;22  We want to capture data from all these new sources. How do we manage it, integrate it, validated, keep it secure. And then, of course, when you have this group of data, we're seeing, of course, artificial intelligence and machine learning and all kinds of derivatives of that occurring with really every data set we have. So that's I think the other big piece is how do we how do we as we collect more and more data, what kind of systems do we have built that are going to help us learn from that and inform us and hopefully within the context of clinical trials and drug development, all of the data we're collecting should allow us to design     00;41;16;22 - 00;41;44;25  the next trial better for the next one to be a little more smarter, to be a little more efficient or a little faster applying this. Maybe upstream into some of the drug discovery pre-clinical stages when we're really looking at new drug candidates, what might work? I think being able to leverage that data and be smart and inform ourselves to be able to accelerate drug to drug discovery in drug development.     00;41;44;28 - 00;42;12;15    I'm hoping that that's where we see great progress, because there are patients out there that really can't wait. And their only hope is that new therapy coming from a clinical trial. And right now, our process just takes a long time. So to that point, do you think do you think A.I. is overhyped? Like if I'm someone with a rare disease and I'm hearing about A.I. and I think, Oh, this is it, this is going to get the therapy or the drug treatment I need in the next year.     00;42;12;16 - 00;42;34;01  Yeah, I think I think that you should be realistic, right? I think that, you know, the AML is going to be those are going to be sort of tools and approaches that are going to be a big part of, like you said, probably most industries and our daily life everywhere. I don't know if that's going to be, you know, a magic bullet that is suddenly going to, like, revolutionize drug development overnight and move things along in a year.     00;42;34;09 - 00;42;53;17    I would temper expectations probably a little bit, but I'd be hopeful that this will help us and this will help us move faster and be smarter. But yeah, maybe that's not a maybe that's not a silver bullet that fixes everything. I think that's another tool in our arsenal to kind of move things forward faster. Well, Scott, thanks again for your time today.     00;42;53;19 - 00;43;15;07  If anyone wants to learn more about you or what you've talked about or a rare clinical, what's the best way for them to do that? Yeah, absolutely. It's been my pleasure to be here today. I've really enjoyed it. Really enjoyed the topics in the conversation. You know, my email address, I guess will put an email out. There is a Scott Schliebner – I’ll spell out Schliebner     00;43;15;12 - 00;43;40;19  @ msn.com. You can also find me on LinkedIn. Scott should leave her and be happy to engage, support, help you out there in any way that I can. Great. We appreciate that. If you are interested in how Oracle can accelerate your research and data needs, all you have to do is check out Oracle dot com. And join us again next time for research and action.