Speaker 1: Welcome to Farmer Talk Radio. This podcast is focused on
Speaker 1: enverching clinical trials with patient reported outcomes, discussing best practices,
Speaker 1: potential pitfalls, and new approaches from the twenty twenty five
Speaker 1: Chief Medical Officer summ at three sixty. For more information,
Speaker 1: I'm on the CMO Summit. Editorials, podcasts or webcasts, please
Speaker 1: visit CMO three sixty dot org. Thank you enjoy the podcast.
Speaker 2: So we'll start with some introductions and then get into
Speaker 2: the heart of the discussion. I'm Barry Tico, I'm chief
Speaker 2: Medical officer at Stoke Therapeutics, and we'll just go down
Speaker 2: the line here. Everybody introduce themselves in their affiliations.
Speaker 3: Hello everyone, My name is well Sam samble CeON, founder
Speaker 3: of delve Health. We focus on patient engagement pros wearables
Speaker 3: will probably have one of the more prominent set of
Speaker 3: wearables for collecting patient outcomes.
Speaker 2: Hi.
Speaker 4: I'm Dave Vessels. I'm the chief medical officer of Signant Health.
Speaker 4: We are a clinical endpoints company, so we focus on
Speaker 4: patient reported outcomes. Clinical reported outcomes. We have about two
Speaker 4: thousand employees, fifty of them are scientists and physicians, and
Speaker 4: we will we help our clients to select the right
Speaker 4: patient reported outcomes to implement them and then also to
Speaker 4: make sure during the trial that the quality of the
Speaker 4: data that you collect is of the highest standard that.
Speaker 5: It can be.
Speaker 6: Hi, good morning. My name is Natalie Agafonov. I'm chief
Speaker 6: medical officer of company and Drivon. We are clinical stage
Speaker 6: company phase two move into phase three dvincient therapy using
Speaker 6: bond marrow derived missing humans themselves. Our focus is a
Speaker 6: rare pediatric indications such as hyperplastic left heart syndrome and
Speaker 6: also we're working with Alzheimer disease. Over twenty years in
Speaker 6: clinical research, I develop really deep passion for the patient
Speaker 6: reported outcome, for the patient voice to shape up clinical
Speaker 6: outcome assessment. Very excited to have this conversation.
Speaker 5: Thanks.
Speaker 7: Hi. I'm Jason Sager.
Speaker 8: I am the chief medical officer of Accent Therapeutics. We're
Speaker 8: a small molecule clinical stage oncology company running two phase
Speaker 8: one for a modifying protein enzymes inhibition and really looking
Speaker 8: forward to deciding how to incorporate this in oncology because
Speaker 8: it's always been a challenge to look at pros and
Speaker 8: something that I think is getting more and more important
Speaker 8: as we move forward.
Speaker 2: Thank you everyone, So as Jason mentioned, PRO is becoming
Speaker 2: more and more commonplace. And let me just get a
Speaker 2: hands here of how many people have used the clinical
Speaker 2: trial PRO in one of the article trials that they've designed. Yeah,
Speaker 2: pretty much almost everyone.
Speaker 7: I expected.
Speaker 2: Now the question how many of used the PRO as
Speaker 2: a primary endpoint.
Speaker 7: In one of your trials?
Speaker 2: Okay, so it's already pretty good that they're that many, because,
Speaker 2: as we'll talk about, there are quite a few challenges
Speaker 2: in you using pros in general, but as primary endpoints
Speaker 2: they are especially challenging. But they're going to become more
Speaker 2: and more needed, both from the regulatory perspective, where right
Speaker 2: now probably about half of all trials have some PRO
Speaker 2: and in Europe about half of all labels include some
Speaker 2: PRO information. FDA is a little bit more reticent, so
Speaker 2: recently only about twenty percent of labels actually are including
Speaker 2: PRO information, but that's likely to change. We heard from
Speaker 2: ken Getz this morning about how trials are becoming complicated
Speaker 2: and their.
Speaker 7: More and more endpoints.
Speaker 2: Well, one of the main contributors to the additional endpoints
Speaker 2: is likely many of you know, are the pros that
Speaker 2: are being collected, but they're not even being collected for
Speaker 2: regulatory purposes. They're they're becoming, I think, even more required
Speaker 2: for payers, and that's part of why. So I'll tell
Speaker 2: you we have a Phase three program for genetic form
Speaker 2: of epilepsy called drave syndrome, and we have made our
Speaker 2: trial quite complicated because we're including additional endpoints just for
Speaker 2: the payers. One of them is a collection of the
Speaker 2: impact of the disease on the family and the parents
Speaker 2: work time, employment time. So I can tell you that
Speaker 2: there is no regulatory agency that cares about that, but
Speaker 2: the payers want to know how much time can this
Speaker 2: person be full time employed and pay premiums? And we've
Speaker 2: had to incorporate video is, we've had to incorporate EEGs,
Speaker 2: there are quite a few other patient reported outcomes that
Speaker 2: we've had to incorporate, especially for payers. So what I
Speaker 2: like to do is have the panel members talk briefly
Speaker 2: about one or two p r ohs that they've they've
Speaker 2: focused on. And I know we're going to hear about wearables,
Speaker 2: We're gonna hear about others that can give some advice
Speaker 2: to this audience as to how best to incorporate p
Speaker 2: rros into your clinical protocols.
Speaker 3: Yes, I mean so pros and co as our core
Speaker 3: capability for what we do. So we do we work
Speaker 3: across multiple disease dates. On cology Alzheimer probably two of
Speaker 3: the most sophisticated ones that we see. Compliance by far
Speaker 3: is probably one of the biggest issues that everybody deals with,
Speaker 3: especially if you're you know, well, really depends on how
Speaker 3: often you want to collect that that diaries and the
Speaker 3: p r os.
Speaker 7: And one of the most.
Speaker 3: Complex aspects that we see often is how many diaries
Speaker 3: are you actually trying to collect on a daily basis
Speaker 3: or on a weekly basis, you know, take bristle mile
Speaker 3: stool for example, right, you're trying to get compliance how
Speaker 3: many times the patient goes to the bathroom, especially in
Speaker 3: an oncology trial. So some of those aspects are complex,
Speaker 3: and one of the ways that we look at it
Speaker 3: is more of every patient is different, right, every environment
Speaker 3: and disease state is different. And even are you really
Speaker 3: trying to push out three pros a day for oncology patients?
Speaker 3: Cancer patients that go in for a treatment and go
Speaker 3: home and they're really sick, and yet we're complaining that
Speaker 3: compliance is really bad. So I think what we've seen
Speaker 3: is what models can we incorporate to help ease the
Speaker 3: patient burden, but also ease the site burden as well.
Speaker 3: And that can be from whether it's from a protocol design,
Speaker 3: whether it's from you know, patient engagement rules, et cetera.
Speaker 4: So from our side, it really depends on the therapeutic area.
Speaker 4: So if you look at at oncology for example, so
Speaker 4: we see that, I'm gonna let me just step that back.
Speaker 4: So one of the principles that I brought in earlier
Speaker 4: in my career that I still believe is that you
Speaker 4: need to make sure that you know what you're going
Speaker 4: to measure and why you're measuring it. We spoke a
Speaker 4: lot about the the earlier today about the complexity of
Speaker 4: clinical trials. Your phase three trial is not the time
Speaker 4: to test e pro You have to do it much
Speaker 4: earlier because you don't want to learn anything new in
Speaker 4: your phase three trials for that. For the rest of
Speaker 4: it's it depends so much about the patient situation, It
Speaker 4: depends about the environment that the patient is in, and
Speaker 4: then also it depends on the disease area for that.
Speaker 4: So it's it's something that we're going to have to
Speaker 4: bring out more and more in the future. But let's
Speaker 4: make sure that you just understand why you're doing it.
Speaker 6: I actually have the same idea to initiate thinking about
Speaker 6: the PRO as soon as possible and don't use phase
Speaker 6: three as a validation study for the PURO. I think
Speaker 6: we always have to be mindful that perro a very
Speaker 6: important tool to understand patient voice, to understand fit for purpose. Also,
Speaker 6: I think the biggest challenge is sometimes when you in
Speaker 6: phase two to understand this disease area, and even with
Speaker 6: the available tools, you can actually develop your PRO and
Speaker 6: validate during the phase two and incorporate and phase three,
Speaker 6: or in my experience, sometimes when you get to phase
Speaker 6: three and you have this aha moment, it's a little
Speaker 6: bit too late, and then you have to overcome this
Speaker 6: regulatory hurtle and be creative how to overcome that.
Speaker 8: Yeah, I mean in oncology, and I'm glad it's come
Speaker 8: up already from my perspective.
Speaker 7: You know, really things.
Speaker 8: Are we're moving away as we develop these molecular targeted
Speaker 8: precision medicines, especially the oral ones that are going to
Speaker 8: be used on a day by day basis for months
Speaker 8: at a time. You really need to move away from
Speaker 8: the model of the traditional dose limiting toxicity where you
Speaker 8: see in a cute dose it's reported to the doctor.
Speaker 8: You get into like sort of how do those patients
Speaker 8: feel on a chronic basis thing, and then there are
Speaker 8: those and you already mentioned diarrhea. There's only a handful
Speaker 8: of things, but fatigue, nausea, these things are not reported
Speaker 8: well and they're not reported well in the paper versions
Speaker 8: of the pros because of just you know, a lack
Speaker 8: of timing and you know, if you only get to
Speaker 8: see the patients once every three weeks, you're not going
Speaker 8: to find those things out. And so getting a tool
Speaker 8: into the patient's hands that you could be handheld on
Speaker 8: a handheld device and making it so that they benefit
Speaker 8: from using it with a report that comes to them
Speaker 8: that they can then bring to the doctor to actually
Speaker 8: ease the burden of that communication is something that we're
Speaker 8: looking into and working with a company called represent with
Speaker 8: a puro in there and to see if we can
Speaker 8: actually optimize that patient reporting from the get go. Just
Speaker 8: so you know, in oncology we now have to deal
Speaker 8: with project optimists and it's a huge question of how
Speaker 8: are you going to choose that second dose? And I
Speaker 8: won't get into that but I would love to see
Speaker 8: pros start to enable some of that decision process in
Speaker 8: a smart way.
Speaker 4: Yeah, it's interesting. We actually just published a paper on
Speaker 4: that exact idea. So and what we did is we
Speaker 4: postialized and this was together with industry we published it,
Speaker 4: and we postialize that that you actually miss a lot
Speaker 4: of the adverss event if you don't do it as
Speaker 4: part of a ePRO, and your dough selection becomes a
Speaker 4: problem later on. And if you look at the phase
Speaker 4: three trials, the majority if your phase three trials, you
Speaker 4: start it off and then you have to reduce the
Speaker 4: dose afterwards, and that's a huge cost burden to the
Speaker 4: trial that you have to put through, administrative burden for
Speaker 4: protocol amendments and safety reports, all these kind of things.
Speaker 4: So yeah, it's definitely there, and we bring your own
Speaker 4: devices the way that you can design e pros. It
Speaker 4: makes it really very easy for the patient to collect
Speaker 4: that data.
Speaker 2: So this is an important discussion the difference between paper
Speaker 2: and the e pros. And I'll tell you running an
Speaker 2: epilepsy trial, we collect daily seizure record from the families
Speaker 2: and this is a method that's been used now for
Speaker 2: decades we're still using paper. I just got a copy
Speaker 2: of the binder that the families are going to take
Speaker 2: paper home with and record every day, and it really
Speaker 2: disappoints me that that's actually the way we're going. But
Speaker 2: I tell you that we spent months and months trying
Speaker 2: to figure out how do we use an electronic diary.
Speaker 2: Do we give the family a device, do we make
Speaker 2: the family use their own device, what's the cost, the
Speaker 2: time for startup, and then all the regulatory requirements because
Speaker 2: that's an issue around the differences between what the FDA wants,
Speaker 2: EMA wants in terms of collected collection device. And then
Speaker 2: on top of it, one of the regulatory agencies wanted
Speaker 2: a paper copy to verify what we're collecting on the
Speaker 2: phones from the family. So after all that, we ended
Speaker 2: up with paper. But I'd like to hear a little
Speaker 2: bit from the panel on thoughts about trying to move
Speaker 2: us more into the electronic age as we go forward
Speaker 2: with our trials.
Speaker 3: Yeah, I've seen I think the first time that I
Speaker 3: saw a paper diary was back in twenty ten and
Speaker 3: it was an Alzheimer's study and it was interesting because
Speaker 3: I was managing a study for a larger company and
Speaker 3: as you talked to the investigator, they're talking about you know,
Speaker 3: they were actually explaining the situation.
Speaker 7: They're like, hey, I'm sitting here with this patient.
Speaker 3: And it was a patient and their caregiver and asked
Speaker 3: me like, hey, how was your pain like two days
Speaker 3: ago or something like that, and he's like it was
Speaker 3: a seven and then the caregivers like, are you sure
Speaker 3: it sounded more like a nine? Right, So it was
Speaker 3: like it was one of those initially interaction and then
Speaker 3: twenty sixteen, actually I started del because my mom had
Speaker 3: cancer and she was part of a study.
Speaker 7: And you know, like we go to Mayo Clinic.
Speaker 3: And they're like, oh my god, I didn't fill out
Speaker 3: all these pros. Right, So you said there in the
Speaker 3: parking lot filling out all the pros. But now, like
Speaker 3: we pass forward to kind of where we are today,
Speaker 3: we want more of that real time aspect what really
Speaker 3: happened to you. And that's the reason why a lot
Speaker 3: of prs are like a diary needs to be responded
Speaker 3: to within twenty four hours, maybe forty eight hours of max.
Speaker 7: Right of course, depending on the diary.
Speaker 3: And I think we're really seeing that, we're seeing the change,
Speaker 3: we're seeing the results, and what we're seeing from our
Speaker 3: perspective is how do we design the pros and make
Speaker 3: sure that if we're doing pr if we're doing BYOD,
Speaker 3: for example, we need to make sure that every single
Speaker 3: device that a patient's gonna pull up is going to
Speaker 3: look exactly the same in every single device. And that's
Speaker 3: you know, that's one of the you know, probably the
Speaker 3: biggest things that we've seen from regulatory agencies. So how
Speaker 3: do we make sure that we identify the pros design
Speaker 3: and make sure that they look exactly the same. But
Speaker 3: patient engagement also like a huge component as part of this.
Speaker 3: If we're really worried about compliance, we got to think
Speaker 3: that it's not just like a website that you're going
Speaker 3: to give to somebody, right, how are you going to
Speaker 3: nudge their make sure that they're aware, hey, you've got
Speaker 3: a pr O that's ready for you, or how do
Speaker 3: you nudge maybe like a caregiver and maybe that network
Speaker 3: of people around them. So technology is here, I think
Speaker 3: it's how can we really utilize it better in today's
Speaker 3: agent world?
Speaker 4: Ye, So we've been working with the Epilepsy Consortium on
Speaker 4: exactly developing that epilepsy diary. So we understand the challenges
Speaker 4: of it, but I mean there's and it sounds obvious
Speaker 4: coming from a ePRO, so it's a vendor. But there's
Speaker 4: already there's a lot of studies out there. There's a
Speaker 4: study that's quite old, I think it's more than fifteen
Speaker 4: years old, where it was quite eloquently designed. It was
Speaker 4: a paper diary. It was giving two hundred patients and
Speaker 4: they were told to complete it. But that wasn't the
Speaker 4: real study. The real study was there was a chip
Speaker 4: at each page and you can actually see when they
Speaker 4: open it and complete it. And something like eight percent
Speaker 4: of the patients finished the diary before the month, so
Speaker 4: they were just proactively writing out what they're going to do.
Speaker 4: The majority of the patients actually did it in the
Speaker 4: parking lot while they were waiting to come back, so
Speaker 4: you could see it all those dates times. So the
Speaker 4: challenge with paper, unfortunately is that we see that people
Speaker 4: don't I mean, people have busy lives, they forget to
Speaker 4: do this, and so that's one part of it. The
Speaker 4: other thing that we also see is that if you
Speaker 4: use electronic diaries, for example, you can identify abnormal patterns
Speaker 4: on when the data gets completed. And you can identify
Speaker 4: some sites that completes the data at very different times
Speaker 4: than the rest of their sites as well, and it's
Speaker 4: something that we don't talk a lot about, but it
Speaker 4: is out there. Clinical trials is expensive, there's a lot
Speaker 4: of money in there, and there is still fraud that's
Speaker 4: happening there. But basically coming back to the epilepsy part,
Speaker 4: so our approach is to work worth a consult with
Speaker 4: the epilepsy consort, Tium or the epilepsy group and just
Speaker 4: work through all these processes with regards to timelines, setups
Speaker 4: and so on. AI has really made a difference in
Speaker 4: that and where we see the biggest the biggest difference
Speaker 4: is actually in the user testing because you can now
Speaker 4: do in a few minutes, you can do ten thousand
Speaker 4: tests and then you can identify exactly where the problems
Speaker 4: are and fix it very accurately. So it improves your
Speaker 4: quality and you don't have to get any programming down
Speaker 4: to one day or something like that. You just need
Speaker 4: to get it off your critical path and that's already
Speaker 4: been done. So there's a huge movement with that in
Speaker 4: the last year and you'll see more and more of that.
Speaker 6: Yeah, it's very challenging and and as you, I would
Speaker 6: just want to emphasize the al Zeimer disease especially. It's
Speaker 6: very surprising to me that not only patients but also
Speaker 6: caregivers have difficulties to work with electronic data. But I
Speaker 6: know we have issues and problems. I want to bring
Speaker 6: fresh idea. If it's if I made that we have
Speaker 6: AI people in a room. A couple of days ago,
Speaker 6: I just visited MIT Museum and there is an amazing
Speaker 6: tool predict whether or not you have opportunity to have
Speaker 6: Parkinson disease based on how you type, how many letters
Speaker 6: you miss. I think in the future, it just brought
Speaker 6: just idea came to my mind. We can actually use
Speaker 6: difficulties for patient to complete data electronically and predict their
Speaker 6: severity based on how patient type. So it's just an idea,
Speaker 6: let's do it.
Speaker 8: So the way I've been trying to get at this
Speaker 8: is when we had discussions because obviously your safety in
Speaker 8: phase one for oncology is the primary goal, and yet
Speaker 8: it's very tightly regulated, and so that's where you know
Speaker 8: there's a lot of questions, Well, what if the pro
Speaker 8: database doesn't match your hospital database, what's the after you're
Speaker 8: going to say to us, and all these sorts of things.
Speaker 8: At the end of the day, what it came down
Speaker 8: for us is to basically slip it into an exploratory
Speaker 8: endpoint in phase one. I think in sort of in
Speaker 8: honor of don't wait until phase three, let's learn about
Speaker 8: what the signal is, but let's do it in a
Speaker 8: way that's more flexible, where we have the opportunity to
Speaker 8: do an pro such that once it's validated and you
Speaker 8: start to get that data, you can then escalate it
Speaker 8: as you go through so that by the time you're
Speaker 8: in phase three, you can make it a primary endpoint
Speaker 8: or something that is akin to something that's going to
Speaker 8: get into your label.
Speaker 7: One of ken.
Speaker 2: Gets his favorites the exploratory endpoints, right. It's complexifying exactly,
Speaker 2: and well, I'd like to turn a little bit two
Speaker 2: wearables now and just where you as panel members see
Speaker 2: from those of you who've had experience with wearables, how
Speaker 2: are we going to be able to incorporate those? Are
Speaker 2: those going to be used more and more or are
Speaker 2: they too complex for us to utilize at this point,
Speaker 2: especially when you consider the number of data points that
Speaker 2: was talked about before. One wearable one heart monitor can
Speaker 2: generate five million data points in a day. So when
Speaker 2: you're thinking about data complexity and the data we need
Speaker 2: to collect, how are we going to start using wearables
Speaker 2: efficiently in our studies?
Speaker 3: So we live and breede wearables almost every single day,
Speaker 3: you know, we've got we have had studies that do
Speaker 3: as much as five wearables on the same patient, and
Speaker 3: we've had as little as one. I think the time
Speaker 3: is coming for wearables. We're definitely seeing that wearable data
Speaker 3: can enhance pr O data. Right, it's all part of
Speaker 3: patient outcomes. Right, you've got the pr O data here's
Speaker 3: how I feel. Then you've got the wearable data that
Speaker 3: tells us here's what really happened at home. There's been
Speaker 3: a couple of studies, probably a few studies that have
Speaker 3: used wearables as a primary endpoint. I don't think it's
Speaker 3: not I don't think it is for every study, at
Speaker 3: least not yet, but they're here. If you're considering it,
Speaker 3: I'll probably go back to the same mentality as use
Speaker 3: it on a phase one or a phase two, so
Speaker 3: that way you understand how wearables work in your clinical
Speaker 3: trial and think at the end of mind, Right, what
Speaker 3: exactly are you trying to understand is it their movement,
Speaker 3: is it their sleep patterns or you know, And you
Speaker 3: have to think about it as patterns over time rather
Speaker 3: than just one data point for a day.
Speaker 7: Right.
Speaker 3: So it's a different mentality, different mind shift from the
Speaker 3: PR data. I think it's but I think it's coming.
Speaker 3: We're seeing definitely a lot more interests in almost every
Speaker 3: single disease state.
Speaker 4: Yeah, I think that's very very valid. We're learning a
Speaker 4: lot st all about wearables. Were currently just starting a
Speaker 4: study with Lead University where we are looking at wearables
Speaker 4: e pros and then also some of the hard biomarkers
Speaker 4: genetic testing, et cetera in elderly patients with cordiovascular disease
Speaker 4: and diabetes. And there's some glucose monitors, etc. And then
Speaker 4: heart monitors. It's a lot of data and again you
Speaker 4: need to understand why are you doing that? And then
Speaker 4: secondly you just have to think about the practical part
Speaker 4: of it is how are these patients going to remember
Speaker 4: to charge the device, how are they going to wear
Speaker 4: the device every day? How are they going to forget
Speaker 4: about the device? And all those questions. You have to
Speaker 4: sit down and really think about it before you come
Speaker 4: up with yeah, I'm going to implement this on my
Speaker 4: study or not. It's otherwise it just becomes another shiny
Speaker 4: tool and then we end up with massive amount of
Speaker 4: data that we don't know what to do with.
Speaker 6: So I see this as a future, and especially working
Speaker 6: with pediatric rare diseases. One all of our pros basically
Speaker 6: come from parents the infants.
Speaker 7: We inject.
Speaker 6: Cells during the second stage sogery when the infants are
Speaker 6: four month old, and using right vigicle ejection fraction as
Speaker 6: a primary endpoint. But there are biases and evaluation based
Speaker 6: on evaluator assessment, etc. So and the way parents describe milestones.
Speaker 7: Et cetera.
Speaker 6: This is all we have right now. So I'm actually
Speaker 6: right now designing new clinical trial thinking about how we
Speaker 6: can use wearables in this tiny patient population, how they move,
Speaker 6: how they breathe, what they do. But again, I think
Speaker 6: more work needs to be done because I'm always nervous
Speaker 6: when I get a lot of data. It's not just
Speaker 6: getting data, it's exactly what question you ask, what data
Speaker 6: do you want to receive, and how you're going to
Speaker 6: analyze it and why you're using this. But I see
Speaker 6: this as a big future, especially for rare disease indications
Speaker 6: patient cannot express themselves.
Speaker 8: I'm interested in actually surveying the audience on this because
Speaker 8: how you know that like almost everyone raise their hand
Speaker 8: when you ask about the pros, But how about for wearables?
Speaker 8: Anyone integrate that into your protocols? Okay, and then keep
Speaker 8: your hands up if it was in an oncology protocol. Yeah,
Speaker 8: that's what I kind of thought. So no respondents, just
Speaker 8: a bed a couple, Yeah, maybe one in the back.
Speaker 7: I'll talk to you later.
Speaker 8: But you know, I think that it's been a challenge,
Speaker 8: and especially for the elderly population, all the technical aspects
Speaker 8: that you mentioned, and I just haven't seen it get
Speaker 8: there yet. I'm hoping that kind of maybe the phone
Speaker 8: itself becomes the wearable, that that's the thing that's most
Speaker 8: attached to them, that they're keeping charge for whatever reason,
Speaker 8: and therefore we can get more information out of it.
Speaker 4: Yeah, And actually that's one of the points that we're
Speaker 4: going to measure in the study with leads, is whether
Speaker 4: you have a smartphone or not, whether that has an
Speaker 4: outcome on your and positive or negative outcome on your life.
Speaker 4: As an endpoint around that, so yeah, great.
Speaker 5: Thanks.
Speaker 2: Well, we have some time for some questions from the audience,
Speaker 2: so if you want to step up to the mic
Speaker 2: any of you see someone walking up here, we'll take
Speaker 2: your question first.
Speaker 9: People are CMO I want to treat so thank you,
Speaker 9: Thank you very much for these I would say, very
Speaker 9: inspiring panel discussion. I was convinced before the penal discussion
Speaker 9: of the importance of PIERO, but even more convinced. And
Speaker 9: I do agree that understanding patient perspective is challenging, and
Speaker 9: I do agree that Piers can really help. Now my
Speaker 9: question is, oh, my next challenge is a following, how
Speaker 9: can we do even better than understanding but considering the
Speaker 9: patient perspective into the clinical trial design? And I would say,
Speaker 9: not on the secondarian points, but really in the heart
Speaker 9: of the design of the new studies. I think this
Speaker 9: is probably the next challenge. I'd like to get your
Speaker 9: opinion on this one.
Speaker 4: You want to do Yeah, yeah, I can. I can
Speaker 4: try that one. I think it's so important, especially on
Speaker 4: rare diseases. Actually to really make your selection of what
Speaker 4: clendros or E pros or pros you're going to choose,
Speaker 4: spend a lot of time on focusing on that because
Speaker 4: these patients have such tough diseases and such tough lives
Speaker 4: and there the care takers are exhausted all these kind
Speaker 4: of things, you have to be very practical in doing so. Again,
Speaker 4: it sounds repetitive and boring, but it's it really depends
Speaker 4: on your disease. It really depends on the situation and
Speaker 4: what do you want to get from it. So that's
Speaker 4: why I would say spend as much time thinking about
Speaker 4: it and finding out why you want a certain pro
Speaker 4: as implementing it.
Speaker 3: Sor And when I think we've done in the past,
Speaker 3: we've done you know, smaller like patient surveys and we're
Speaker 3: actually we contractor with a third party say like hey,
Speaker 3: can you interview maybe like five or ten patient population
Speaker 3: from within the specific disease and kind of walk them
Speaker 3: through what we're going to do and give us that feedback.
Speaker 3: And that's been like amazingly helpful. So if this is
Speaker 3: something that really kind of top of mind, something i'd recommend.
Speaker 2: Yeah, I recommend it too as well, you know the
Speaker 2: question of back here.
Speaker 10: Yeah, thank you guys so much for the information.
Speaker 7: I appreciate it.
Speaker 10: And your collective experience is where have you seen the
Speaker 10: best practices of real world evidence being leveraged to show
Speaker 10: investors the value of the data that you're getting as
Speaker 10: it's happening before it goes to market. You know, one
Speaker 10: of the things that is key now with capital strap
Speaker 10: markets is figuring out how you can say their phase
Speaker 10: one top line is going to show value for the
Speaker 10: future when it could get to a registrational pathway. Are
Speaker 10: there ways that you all have seen railword evidence being
Speaker 10: brought into that into maybe combination studies or different programs
Speaker 10: where you've had access to the information to help assist
Speaker 10: with your protocols.
Speaker 8: Maybe I can't, so I'll answer very quickly and then share.
Speaker 8: You know, recently Astrozenica presented their part one selective inhibitor. Now,
Speaker 8: obviously they had the wherewithal to be able to do
Speaker 8: three doses and so, but you know, they had some
Speaker 8: really nice data where they showed that the efficacy of
Speaker 8: the top two doses were the same, but you had
Speaker 8: a lot less nausea with the second lower dose. Unfortunately,
Speaker 8: for biotech where we are limited to two doses, I
Speaker 8: think it's still going to remain a challenge. But that's
Speaker 8: where I've seen this data lay out in a way
Speaker 8: that I think would be very powerful.
Speaker 6: Thanks for the question. I think actually real world davidence
Speaker 6: is becoming a part of our lives right now. You
Speaker 6: can use syneatic arms using real patient data on publicly
Speaker 6: available domain. For example, there is a single Vincicola trial
Speaker 6: with twenty years worth of data an Alzheimer's disease near abnormalities.
Speaker 6: You can use this data as a synthetic arm to
Speaker 6: kind of compliment your clinical trials. So you can do
Speaker 6: open label trial and complimenting this with the synthetic arm
Speaker 6: and present to the a FD. And they're really really
Speaker 6: accepting this idea right now. It's it's really hot right now.
Speaker 2: Time for one more question here, sorry, thank you very much.
Speaker 5: So Anil Dargent, Dunny Edward Genomics and we're running an
Speaker 5: LS study with a weariable remote monitoring as well as
Speaker 5: pros in there. Just a question for the audience what
Speaker 5: kind of correlations have you seen between pros, whether it's
Speaker 5: ePRO or paper versus what has been collected by remote
Speaker 5: monitoring or wearables? Is it one to one when they
Speaker 5: say the patients say I walked two times a day,
Speaker 5: did the wearable also show that same data? Or is
Speaker 5: it not correlated.
Speaker 3: I don't know that there's ever really a true one
Speaker 3: to one, to be honest with you, because when we
Speaker 3: look at this Steffa, so correlation between pros and wearables
Speaker 3: is definitely core part of this, right, But as you
Speaker 3: look at the wearable data, we need to look at
Speaker 3: trends where it happened, right. So for example, if you've
Speaker 3: got the pro that asked, you know, how many miles
Speaker 3: did you walk yesterday?
Speaker 7: And they said one? Did you really? You know? So
Speaker 7: that's kind of what the wearable would really show.
Speaker 4: Yeah, I think it's it's important to understand what the
Speaker 4: patient's going to gain from that answering that question. So
Speaker 4: one of the things that we focus on a lot
Speaker 4: is training and PA see mitigation training as well, and
Speaker 4: this falls into that part. If the patient's going to
Speaker 4: gain something by lying, then they'll lie thinking that is it?
Speaker 7: So thank you very good.
Speaker 2: Well we're all set here, so i'd like to think
Speaker 2: again the panel and thank you all for your attention.
Speaker 2: Thank you, thank it.
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