(light music) - [Maria] So these amazing communities have been forming over the years where people go to find
support, to give support, to talk about their lived experiences and so we have thousands of people, tens of thousands of people talking about their symptoms
and their experiences. And now that we can analyze
those conversations, turn them into insights, and then do something
with the information, react to the information,
it's so powerful. (soft music) - [Todd] Patients with
rare and chr
onic diseases and their caregivers often find support and solace in online communities where they can openly ask questions and share firsthand experiences. Yet there are patients who feel unheard and drug developers
seeking unfound insights. What would it look like
to break down this wall? To deliver wisdom from these patients to scientists and clinicians, while respecting the privacy
of these communities? I'm Todd Poley, and in this
episode of Vital Science, we sit down with Maria Picone, found
er and CEO of the TREND Community, to discuss how the organization ethically partners with
invite-only online communities and uses machine learning and natural language
processing to turn anecdotes into evidence for rare and
chronic disease patients. - [Gina] Welcome to Vital Science, Maria. We're honored to have
you here with us today. - [Maria] Thanks, Gina,
I'm excited to be here. - [Gina] So I think we have a
really interesting topic today and something that maybe people haven't thought abou
t before, but certainly is really a game changer I think in the field of rare disease, and that's TREND Community and it was born in 2017 by a
personal experience of yours. So I'd love to hear how it started. - [Maria] Sure, so actually
the seed of the idea started in 2012 when our first child was born with a rare disease
called Prader-Willi Syndrome. And prior to that, my
husband, Chris, and I, we had been working in digital
health, data analytics. I'd worked in pharma
and regulatory affairs an
d when our daughter was born, we were pretty well
resourced to be able to go to the medical literature and try to understand what this diagnosis meant for our daughter, for our family. But it wasn't until I joined Facebook and found a group of caregivers
of kids living with PWS that we really had hope
for the first time. And what I was really
struck by in that moment was what appeared to be a pretty big gap between what we'd read about the disease in the medical literature and what appeared to b
e possible for a kid born today with this rare condition. And PWS is mostly
associated with two things. One is it's called hyperphasia which is a chronic insatiable hunger. So even someone with a normal IQ who is living with hyperphagia, they'll forage for food from a trash can or families have to lock up their kitchens to keep their family members safe. The second thing is obesity. So over the years, their metabolism kind of downshifts so it's sort of a perfect storm. So for obvious reasons, ou
r community was fully focused on finding treatments for those things. But we were told that would never be a problem for our daughter. We would never have to
deal with hyperphagia because there were so many promising drugs in clinical development. We can talk about it later. Fast forward, she's 10. It is our problem. One by one, all of those
clinical trials are failing. But at that time, we were sort
of working on the assumption we wouldn't have to deal with that. And so we knew that PWS was ver
y complex and so we thought okay, what can we learn from the conversations that we're hearing in this close community that might give us some
clues about how we might manage the disease so that our daughter could have the best
possible health outcome? And we did that quite successfully. One of the first insights that we gleaned, we realized that parents were talking a lot about issues related to sleep and in fact, they thought that their kids were faking falling asleep. And we can talk more abou
t how we do it, but we used this approach, this social listening approach to connect PWS with another rare disease, narcolepsy. And we started, we formed TREND in 2017 because in 2016, the year before, the FDA passed the 21st Century Cures Act which basically obligated
the pharmaceutical industry to incorporate the patient voice into every stage of drug development. - [Gina] Wonderful,
and before we even dive deeper into how that all comes together and the mechanisms behind it, tell us about you
r daughter. - [Maria] So as I mentioned, my daughter is now 10 years old. She loves dogs and horses. She's in the fifth grade. She's in a mainstream classroom, but she gets a lot of support and one thing that I find
really interesting about her on the rare disease side of things is even though she's lived with Prader-Willi syndrome her whole life, she was actually diagnosed with narcolepsy a couple of years ago. And if you ask her why
she sees so many doctors and takes this medicine or that, she
'll say that she has narcolepsy. So making that connection and finding a treatment for her to treat those symptoms has
been really life changing. - [Gina] And over the last 10 years, how have you come to better
understand Prader-Willi syndrome through your work with
the TREND Community? - [Maria] Yeah, so especially
in the sleep space, we've made a lot of progress. So from those early days where we started to make that connection and we were able to use the data to appeal to the community to say
hey, your kid probably
isn't faking, it has a name, and it's called narcolepsy. And back then we didn't
have the words, right? We were describing the symptoms, but we didn't have the
language to call it narcolepsy. Fast forward, when we do social listening, our community now has those
words in their lexicon which is a really powerful aspect to the process that we use
to create these insights. But then once the
community became informed, then we were able to empower the community members with th
e reports and take them to their neurologists. And ironically we were
all seeing neurologists because our kids are on growth hormone. We're one of the few lucky
rare disease communities that has an approved therapy. Growth hormone was
approved for PWS in 2000. So we were able to take those reports, go to our neurologists, and say, "I think my kid might
be experiencing narcolepsy. Will you do the daytime sleep study?" And as a result of that, many kids including our daughter now have a secondary
diagnosis of narcolepsy. And then through podcasts
like this, and blogs, and articles in the "New York Times" and other media outlets and publishing the data, we attracted a pharmaceutical
company, Harmony Biosciences, who after bringing their drug
to market to treat narcolepsy started working with TREND on a clinical trial for that drug to treat the symptoms of PWS. And so over the last few years, we worked with them to
design that clinical trial. We helped them with the phase two trial. We ult
imately helped with recruitment. We used the social listening, the insights to understand some barriers that they were experiencing
with recruitment and now I'm excited to say
that we are in the process of launching a phase three
clinical trial with Harmony. - [Gina] Wow, that's
incredible, congratulations. - [Maria] Thank you. (light music) - [Todd] The TREND Community
supports individuals with rare and chronic diseases through a system of social listening. Using an app that is
transparently in
stalled in collaboration with online communities, TREND gains permission to access to anonymized conversation data, giving them insights from closed groups that are often not
available in public forums. The team then organizes real
world accelerator sessions to analyze the data
collaboratively with patients, caregivers, key opinion leaders, industry partners, and
patient organizations. This process helps identify unmet needs, fills gaps in medical knowledge, and provide meaningful contributions
to scientific literature. Let's hear from Maria
on how the organization goes about prioritizing
this important work. - [Gina] I know that there
are thousands of rare diseases that need more therapeutic attention. How do you decide which ones to focus on in your
evidence acceleration work? - [Maria] Yeah, so there's two ways. One is a community comes to us, we bootstrapped the company and basically once we realized we had this opportunity back in 2017, we put a call out to any communities that wa
nted to work with us and most of those groups
continue to work with TREND. And so then resource permitting, we'll do this sort of evidence generation
with those communities and as our tech becomes
more and more sophisticated, we're able to do a lot of
things in an automated way which is great because we always wanna be giving back to the communities whether our projects are
revenue generating or not. And then the other way
is that we have a client who comes to us and they have a specific disease
state that they're interested in, in which case either
we will have some data or we'll go out and we'll
try to find a community that would like to partner with us, or generally our pharmaceutical clients are very patient-centric and they'll make warm introductions. - [Gina] It sounds like
you've found several avenues to build relationships with patients. How do you make sure your findings get into the hands of clinicians? - [Maria] So there are a
couple of different ways that we get the data ba
ck to clinicians. In part, it's empowering the
community with the information so that they can go to their clinicians like we did with the neurologist. If the community's lucky enough to have say neurology involved when you're talking about a sleep issue in PWS, then it's really just
inspiring those clinicians to see those patients. And generally doctors like to publish. Researchers like to publish. So we invite KOL clinicians, specialists to be a part of the solution and co-author these publica
tions with us and in doing so, I think
it really helps people to justify investing the
time towards something that may not be talked
about in literature. And then if you don't have the audience, that's when we really start
to lean into the publishing. So in gout, for example, it's about rheumatologists are on board. But how do we get to the
primary care physician so that, and so then we start to think
about where should we publish? Should we do CME? Should we do a podcast, right? Where are the p
eople
that we need to reach? How can we get this
information in front of them? But it's the power of the community. I mean, honestly, I think you'll see more and more of it moving forward. I think even just
telling the gout patient, "Take this data to your
primary care physician." If you're talking about we're working in a new disease state, IgG4-RD. and these patients are seeing all different kinds of specialists. And so again, it's really getting this information in
front of rheumatologists, g
etting it in front of GI doctors. And if you've seen it in one patient, that patient sort of in
the back of your mind. So if you've read something along the way and then a patient comes to you, you're more likely to kind of remember. Yeah, I saw this somewhere. How can I find more resources about it? - [Gina] There's been a lot of backlash against the idea of Doctor Google and seeking disease information online. In light of this, how do we encourage patients to share their voice
within these com
munities and understand the value
of what they find there? - [Maria] I mean, I think the community, I think that people more
and more are, to your point, going straight to the
internet to find everything. And so I think that if
there is a community and if they are creating materials and putting it out there,
and doing the podcasts, and doing the blogs, they're gonna be found. One of our partners, Global
Healthy Living Foundation, was talking about a wonderful idea which is really how can we thin
k about how to influence the SEO, so the search engine optimization? So as we identify these
areas, to Gina's point, about where there might be misinformation or there might be a paucity of
information around something, how do we, as sort of these coalitions, think about how we can influence, come up with strategies
to influence the SEO so that when people do Google it, they're getting good information and they're getting
connected to these resources? I mean, I think the nice thing
about the onl
ine communities, I mean, we all know that
patient organizations play a key role in the
quality of life of a patient or caregiver on their journey. For a lot of these rare diseases if the number's 10,000 plus now, there's not always an organization. So I think those groups on social media sometimes are the only
support that people have. - [Gina] What about the therapeutic areas? Are there some that are well attended or you're seeing
significant activity with? - [Maria] Yeah, we happen to be doing
, so in the rare neurodevelopmental space, a lot of the sleep work. So the PWS sleep work led us
to the narcolepsy community, so we're doing a lot of work in narcolepsy, idiopathic hypersomnia. It also raised awareness
within other communities. We're doing a lot of work in
hypoxic ischemic encephalopathy which is sort of an umbrella, there are a lot of rare epilepsies that sort of live in that community. And then we're also doing a lot of work in autoimmune inflammatory conditions like gout, scl
eroderma, Sjögren's, lupus. And again, the work has
sort of expanded organically because there are a lot
of comorbidities there And so one project and one community leads us to start working with another one. (light music) - [Todd] A goal of the TREND Community is to combat misinformation within rare and chronic
disease communities. Their approach involves refining
data analysis techniques using the NIH's Unified
Medical Language System to ensure precise terminology alignment. This minimizes the
risk of misunderstanding and misinformation when community members
discuss their symptoms. Collaboration is a cornerstone of the organization's approach. TREND partners with patient groups and community administrators,
empowering them to differentiate scientifically
supported information from unverified remedies. For example, TREND worked with the Gout Support Group of America to steer discussions towards
scientifically-validated topics like uric acid monitoring and away from less
substantiated
approaches like apple cider vinegar or cherry juice. But their work doesn't stop at listening. The TREND team actively
disseminates relevant information back into these communities. For example, their gout research revealed that proactive management including uric acid monitoring and consultations with rheumatologists correlated with positive
mental health language. Conversely reactive management such as ER visits was associated with negative mental health language. Findings like these are pres
ented at patient and scientific meetings and published in collaboration
with the community, promoting a feedback loop of informed and accurate information. Let's hear from Maria
on how this information can influence the drug
development process. - [Gina] It's clear that the insights coming out of the TREND Community can really benefit patients,
caregivers, advocates, but you mentioned at the start your work has influenced a therapy that is going through clinical trials. Are you finding these ins
ights to be valuable to other stakeholders like regulatory groups and sponsors too? - [Maria] Oh, absolutely, I mean, I think it's listening, right? So these amazing communities
have been forming over the years where
people go to find support, to give support, to talk
about their lived experiences. And so we have thousands of people, tens of thousands of people talking about their symptoms
and their experiences. And now that we can analyze
those conversations, turn them into insights, and then d
o something
with the information, react to the information,
it's so powerful. And of course, at the end of the day, if you can then couple
that with real stories. So we love to have, whenever
we are doing workshops or we're having talks at conferences, having an actual person
living with a condition, a caregiver, a care partner
telling their stories but then showing that their experiences are also being experienced
by thousands of other people. That's when it becomes really powerful because I th
ink that over the years, all of the different stakeholders
that you've described, pharma, the FDA, other
regulatory agencies, they've developed all of
these platforms to listen. We have patient-focused
drug development meetings, listening sessions, ad
comms, all those things. They're all super powerful, but you're only ever going to get so many people sharing
their personal stories. So by tapping into all of the experiences shared on social media, it just gives another level of perspective to th
ose stories when we hear them. - [Gina] I think we can
all perhaps recognize that there are some
mental health challenges that come with anybody involved
in having a family member or someone they know that is suffering from a rare disease and that TREND has been publishing on the mental health challenges. Is there anything you can share with us around any real world
findings that you're seeing come to light through the
TREND technology and approach? - [Maria] Yeah, I mean, I
think just to set th
e stage, at least 10% of any
dataset that we look at are conversations that are
related to mental health topics and often upwards of 20%. So any community that we go into, there's always a lot of
conversation around mental health. In fact, last year, we spent, our data science team, they invested a lot of
time in actually developing these mental health classification models so we could quickly go
into those conversations, pull out those that are
related to mental health, and then focus on them.
And I think a lot of interesting things have come out of that. I talked about gout and I think not only recognizing that mental health issues
impact the community, but actually that when you treat the mental health issues as well, we're noticing that the patients had better health outcomes generally. I think being able to demonstrate
that is super powerful. Other examples, we recently did a project where we looked at conversations
around taking a medicine, it was for narcolepsy where
patients ha
d to take a medicine before they went to bed and get up in the middle of the night and take a second dose. And what we found was
that patients were not, they were under reporting the challenges that they were having
with that dosing regimen because they were afraid
that this medicine that was life-changing to
them could be taken away. And so I think that is not necessarily obviously
related to mental health, but I think all of these different things that patients are living with and the way that
it impacts how they think about their disease, the decisions that they
have to make every day to ensure that they have quality of life. So that was something that was
really striking to me as well and interesting also while
we have to be careful about not capturing
misinformation as you said, also interesting that people,
when they feel anonymous, are willing to talk about things that they may not feel
comfortable talking about even to their healthcare providers. - [Gina] Yeah, you're in a
uniq
ue position to capture a lot of the things that
people aren't saying out loud, aren't sharing publicly,
pull that together, and then use that as potential data to indicate a problem or an opportunity. - [Maria] Absolutely, yeah,
in this particular case, we were able to do the social listening, identify all of the issues that people were having, quantify them, and then actually this
goes towards understanding what is validating the findings. We then fielded a survey and because we developed these
trusted relationships
with the community, we were able to reach out to the community and ask people to participate in a survey. And within two weeks, we had about 90 people
respond to the survey and the results of the
survey validated the findings that we were seeing on
social media and vice versa. - [Gina] So you mentioned that 10 to 20% of the conversations you
see in patient groups are about mental health and that in your opinion, a similar proportion of
doctors' appointments should be dedic
ated to this topic. Are you seeing any
progress in that direction? Now that you've presented this
data on gout, for example, are doctors asking those questions? - [Maria] That's a good question. I don't think we are quite there yet that I could really speak to that. I think we're making a really hard push in gout to change that narrative. I think the other goal
would be how do we prepare, actually one of our clients
was talking about this, an MSL was saying, "Wouldn't
it be great if you helped t
o create a list of questions
based on our insights that a patient could take to their doctor, give it to the doctor so the doctor could ask
them those questions." I mean, I think part of the problem is especially when you're living with a complex medical condition, there are only so many
minutes in your visit. How do you even ensure that
you're covering all the topics? So I thought that was a great idea too. Not only giving the data, but also empowering the
community with kind of questions that
they can bring to their clinicians. So I don't have a pulse
on how we're moving the needle yet in the clinic or in the doctor's office, but I think next year, we'll hopefully have
some data around that. (light music) - [Todd] Meanwhile the TREND team is hoping to move the needle on mental health offered
outside the doctor's office. The organization has highlighted the mental health burden
experienced by caregivers and individuals with rare conditions, emphasizing the importance
of the mental hea
lth journey and providing support when appropriate. In addition, the TREND team recognizes the natural role of community
members in providing support and aims to empower them
with tools for peer support while safeguarding
their own mental health. In the organization's recent work with myasthenia gravis
on platforms like Reddit, they expanded insights by following breadcrumbs of user activity, allowing them to understand
the patient's journey leading up to the diagnosis. The team realized that pr
e-diagnosis, there are often conversations happening related to mental health with individuals seeking
support for specific issues in relevant subreddits
and social media groups. Through partnerships with psychologists and non-profit organizations, TREND is hoping to answer this call for expanded mental health support for rare and chronic disease
patients and their caregivers. - [Gina] I know your work
is often with professionals and organizations
dedicated to rare disease, but I love the concep
t
of the citizen scientist that you shared in your
#RAREis video a few years back. Can you share more about that for those who may be
unfamiliar with the concept? - [Maria] It was a while ago, but we still name a citizen
scientist every year and I think that it's becoming more and more possible for
individuals to make an impact. And so for us, a citizen scientist is
someone who recognizes that there's an unmet
need in the community and then they empower
themselves to find a solution. And so I ca
n give you some examples. In the Prader-Willi syndrome
example with the sleep, it's not just the AI. The AI is important, but I've told many stories, the people that that
join us on this journey are just as important. And back then or early days, there was an immunologist,
her name is Laura Pullen. She has a son with PWS. Did I say she was an immunologist? So she's a mom, she's an immunologist, and as we started to have this aha moment that the kids were experiencing
these cataplexy symptoms, sh
e was actually the one
that discovered Pitolisant which is the drug that
I was telling you about that Harmony Bioscience
is brought to market and it had just been approved in Europe. And so to me, that's a citizen scientist, someone who has a problem, finds a creative way to collect
some real world evidence, and then pulls together the team that they need to find a solution. And fast forward, we're
actually naming Gary Ho as the community member that started the Gout
Support Group of America and
so having those brave
individuals who have the problem and find the solution, and then in doing so have
such a positive impact on an entire community of people. So yeah, please, I would love
people to visit our website and learn about all of
our citizen scientists. - [Gina] That's really
exciting, I think another aspect that I'm curious about is this
is such a huge collaboration. You've talked about so many
different pieces of groups that take part in this, have a part in helping
it to be succe
ssful. Could you tell us about any collaborations that maybe we haven't touched on? How are you collaborating with groups like Charles River CROs or other key opinion leaders and other sponsors to enable
this to be a successful effort? - [Maria] Yeah, so I mean, one of the things that
I love about what we do is that it really inspires sort of pre-competitive collaboration. I think that everyone who joins one of our real world
evidence accelerators, they probably have some
personal reason for doi
ng it, some motivation for doing that. But I think every single stakeholder shares a desire to do something bigger than whatever it is, than the problem that
they're trying to solve in front of them. I would say on the client side of things, we've done some amazing things in sleep. Pre-competitively, I told you
about Harmony Biosciences. Avadel is another organization that joined the Sleep Real World Evidence Accelerator and we're now partnering with a pediatric
neurologist, Dr. Anne Morse, and
there's lots of
different stakeholders there. There are nonprofit organizations and admins of these groups. But what I think is interesting with that particular group of people is really this idea that
we really need to listen to what people are saying on social media. Dr. Morris actually, we started partnering because she gives a lot of talks and she talks about as a sleep specialist, that she goes to social media
to understand what's happening for the exact reason
that I mentioned before, that
patients aren't always telling their healthcare providers what's happening on the day-to-Day. So that's been a really
exciting collaboration and obviously we've played a role in bringing a couple of drugs
to market which is exciting. In some of the work that we're doing on the autoimmune
inflammatory side of things, it's interesting because as I mentioned, there's so many comorbidities. So we have the Gout
Support Group of America, but we're working with
the Arthritis Foundation, Global Healthy
Living Foundation. As I mentioned, Horizon
Therapeutics has been such a incredible client to work with, supporting all of these
different stakeholders that are kind of coming
into this accelerator. And I think the really interesting thing again is sort of that ability for everyone to take away from the experience something that drives forward
their own initiatives, but then carve out some
time to participate in whatever sort of unmet
need we've all identified. So like I said, with the gout exam
ple, we're working a manuscript
around proactive and reactive management of gout and its impact on mental health. So it's really important to
get that in the literature so that we get it in
front of rheumatologists and they understand how important it is to provide these mental health resources. So to me, it's really incredible to see how you can bring so many people together and get something done so quickly. - [Gina] Yeah, your use of the word accelerator really strikes me and I think it would
be
interesting to put our arms around to what extent has this work
really accelerated treatments, are patients getting relief? And I bet it's quite impactful. I don't know if you've ever measured that, the true acceleration
that if this didn't happen or because of this,
we've moved this forward at a faster rate or? - [Maria] Yeah, so I will say Harmony's drug is in phase
three clinical trials. Avadel's drug recently came to market and the work we did around the need for a once nightly sodium ox
ybate therapy, all of that work that I talked about, we were able to pull it all together and write a letter to the
FDA describing the unmet need and advocating for a once nightly therapy. And shortly after we did that work, that drug was approved for
the treatment of narcolepsy. So I know that it was just
a piece of the puzzle, but we're seeing progress. And then to me the other piece is looking at the shifts in behavior. So we did this work. In gout, we actually looked at recently the last thr
ee
months of conversations compared to the last year and we can see that the conversations around uric acid monitoring are going up and the conversations around cherry juice and apple cider vinegar are going down. And similarly as I mentioned before, in Prader-Willi syndrome,
we did a recent analysis, we're now using that word narcolepsy. We have that in our lexicon. So I think that it demonstrates one, the power of doing social listening and giving back to the communities and co-creating with a
ll
of these stakeholders. But I think it also speaks volumes to those citizen scientists, right? And the people who are really driving these initiatives to change the narrative and we're just giving them
a tool in their toolbox to be able to do that so. Yeah, we have lots of examples and I think AI is only getting
more and more powerful. So I'm hoping we're on really, we're just at the beginning
of something even bigger. - [Gina] Can you expand a little
bit more on your use of AI, and where you'
re at now, and maybe even where you
see that going forward? Or that's too broad of a question? - [Maria] No, no, it's important. So AI, it takes time to train an engine and so I'm really excited
about where we are and though as I alluded to before, our focus for our AI
is really to understand how patients, caregivers, care partners talk about their diseases
in their own words, and that's the gap we're filling and how do we map those to medical and scientific terminologies? And so the way we talk
on
social media is it's complicated and so it's not straightforward. And then in addition to that, where we're talking about things when we may not have the words, so we're talking about our
kids faking falling asleep and we're training the engine
to make that connection between that and cataplexy. So that's how we're using AI and then we're then able
to take the insights and contribute to things like conceptual models for diseases. We're doing a project now to create a new conceptual model
for
Prader-Willi syndrome. So when we started the work with Harmony, it was amazing that we had
a pharmaceutical company who was willing to work with us, but we didn't have good natural history on narcolepsy and PWS. And so at the time we were able to use the social listening data to at least demonstrate
that there was an unmet need and that was part of the justification to the agency to even do a clinical trial around excessive daytime sleepiness, an outcome, an endpoint that had never been studie
d
in Prader-Willi syndrome. And now fast forward, we're actually able to add the social listening
insights to those projects. So we're creating a conceptual model where we're looking at the literature, we're doing the interviews with
the patients and caregivers, but then we're also taking the data that we're getting from social listening and all of that is coming
together to define the disease. - [Gina] Fascinating, so not
that you haven't given us a lot to think about and it sounds like you hav
e
a busy and exciting future, but so curious as to what's on the horizon that maybe you haven't covered yet. I'm sure there's quite a lot of unmet needs for TREND Community. How are you prioritizing
kind of what's next for you? - [Maria] Yeah, when I started, I told you that we founded TREND because we wanted to
tackle all of the things that our community wasn't focused on in hopes that we could improve our daughter's quality of life and we've certainly done that. But then I also mentioned
that
unfortunately, all of the clinical trials for the treatment of hyperphasia
have one by one failed to meet their primary endpoint. And the reason, part of
that reason I believe, is this limited disease understanding and trying to measure something that's really difficult to measure. And so the conceptual model
work that I'm talking about is an attempt to try to broaden
our understanding of PWS and this can certainly be
applied, it's been applied. I talked about SLC6A1. Other communities that are
using the social listening to quickly generate new conceptual models for these diseases. We're doing this work in partnership with Combined Brain is another
organization we work with and then in their most recent guidance, the FDA is now talking about
custom personalized endpoints. One is goal attainment scaling. People can look it up, but they specifically speak
to goal attainment scaling which is a methodology
that basically can measure anything in a way that is personalized to the participant
in the clinical trial. So we're working on a really
important initiative right now, creating those conceptual models in partnership with Combined Brain and then we're working
with a CRO, Ardea Outcomes, a Canadian-based CRO that has really, they've really tackled and perfected the goal attainment scaling methodology. And so we are working on
really sort of to innovate on the process to create
the goal inventories for the gas methodology and then looking for
pharmaceutical partners who would be
willing to
then use the gas methodology in their clinical trials and as an exploratory endpoint so that we can validate
it for PWS or for SLC6A1. So that's where I'm really excited and I'm hoping that we can approve more drugs for rare diseases because I think there are lots of drugs out there that show promise and for some reason we're
just having a really hard time getting them over the finish line. - [Gina] Well, really, I don't know, powerful and inspiring
efforts on your part, Maria. How ca
n our listeners
of Vital Science help? What are you looking for
from the general population to help move this
important movement forward? - [Maria] Yeah, we're
looking for stakeholders from every aspect of the community. We call it community with capital C. If you're a rare disease community that needs help driving
therapeutic progress, contact us. If you are a key opinion leader, if you're a clinician and you have identified an unmet need, either come to TREND or find a community and try to thi
nk about a creative solution to solve for that problem. And I think for pharmaceutical companies, I think that just really
adopting the habit of listening and going and not presuming to know, and being willing to kind of
explore where community is so that you can meet them there. And my hope is that as we
have treatments come online and potentially even cures, the phenotypes of these diseases are going to continue to shift. So we can't just keep relying on old information to develop drugs or to
determine what a community needs. So I'm just hoping that
this will inspire people to kind of think outside the box and to really listen to what patients and caregivers are saying. - [Gina] Very well said, well, thank you for your time today, Maria. It was a pleasure having
you on the podcast. (light music) - [Maria] Thanks, Gina,
it's a pleasure to be here. - [Todd] Maria Picone is the founder and CEO of the TREND Community. Looking ahead to our December
episode of Vital Science, we'll wrap up
the year with our annual Checking Vitals
episode: Disruptors Edition, where we'll review three change makers making bold moves in the
drug development industry. Until then, thanks for listening. Did you know that Charles
River has a sister podcast, Eureka's Sounds of Science? This monthly podcast
shares scientific patient and advocacy perspectives
on trending issues in the drug development industry. You can subscribe to Vital
Science and Sounds of Science on Apple Podcasts, Spotify, Stitcher, or
wherever you get your podcasts.
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