Speaker 1: Well, good morning everyone. My name is Sabina Kanin and
Speaker 1: I'm a patient and a caregiver as well. And I
Speaker 1: can't tell you how honored I am to be sitting
Speaker 1: here and sharing the stage with Jane Gershkowitz of Amikis Therapeutics.
Speaker 1: She truly is a hero of mine. We've known each
Speaker 1: other for close to seventeen.
Speaker 2: Years, yeah, at least.
Speaker 1: Around seventeen years, so it's an honor to have her
Speaker 1: here and to hear her words of wisdom. How she's
Speaker 1: been able to really embed the role of patient advocacy
Speaker 1: within the corporate DNA.
Speaker 2: So Jane, welcome, Thank.
Speaker 3: You, Sabina, and yes, full disclosure, we have known each
Speaker 3: other for a very long time. But when I was
Speaker 3: asked to do this session with you all this morning,
Speaker 3: Kate said, gee, what do you think about having a
Speaker 3: patient advocate interview you, you.
Speaker 2: Know, to do the chat. I said, I think that'd
Speaker 2: be great.
Speaker 3: She said, yeah, We've got this woman who's worked with
Speaker 3: us before.
Speaker 2: She's a great patient advocate. Her name is Sabina.
Speaker 3: I was like, yeah, I've known Sabina and her family
Speaker 3: from very early days at Amicus, so it's equally an
Speaker 3: honor for me to be here and to be interviewed
Speaker 3: by a colleague and a friend.
Speaker 2: Thank you, Jane.
Speaker 1: Okay, well let's get started, because I know you do
Speaker 1: have a wealth of information to.
Speaker 2: Share and a pressure.
Speaker 1: Well we know that if you're all attending this conference,
Speaker 1: you care about patient engagement, right everyone here. You wouldn't
Speaker 1: be here if you didn't. But however, we all face
Speaker 1: different obstacles. No matter what position you are in your organization,
Speaker 1: you're gonna you're gonna face different obstacles about really kind
Speaker 1: of selling the importance of patient advocacy within within the organization.
Speaker 1: So how do you communicate the real value of patient
Speaker 1: advocacy and secure leadership buy in and their resources.
Speaker 3: Yeah, so absolutely, there are a lot of challenges that
Speaker 3: we all face in our organizations, and it also depends
Speaker 3: upon where we are in the development of our organizations.
Speaker 3: So very young companies today are sometimes bringing in lead
Speaker 3: patient advocates.
Speaker 2: From the get go.
Speaker 3: Sometimes they're among the very first few people hired. And
Speaker 3: then you have other companies where this has really been
Speaker 3: part of their DNA And I'll just quickly say that
Speaker 3: at Amicus Therapeutics that's really been the case. In January
Speaker 3: of two thousand and five, when our former president CEO,
Speaker 3: John Crowley came on board as the CEO. Then he
Speaker 3: had already been focused on the patient story because he
Speaker 3: was not only an entrepreneur in biotech, but he was
Speaker 3: a parent and patient advocate for two of his three
Speaker 3: children who live with a rare disease, and so he
Speaker 3: had already experienced that in a couple other companies when
Speaker 3: which he started in, which then eventually had been acquired
Speaker 3: into a larger rare disease company.
Speaker 2: And he knew the value of that.
Speaker 3: So it was very different at Amicus when I came
Speaker 3: there almost nineteen years ago.
Speaker 2: We weren't necessarily pushing.
Speaker 3: The rock up the hill with the leadership in terms
Speaker 3: of the CEO, but I will say that we were
Speaker 3: pushing the rock up the hill with many of our colleagues,
Speaker 3: in particular those in clinical operations. In clinical research, and
Speaker 3: back then the reason was when you held a lunch
Speaker 3: and learn, which we always did, and we can.
Speaker 2: Talk about that as well.
Speaker 3: There were actually people in clinical who would say, well,
Speaker 3: I can't go to that lunch and learn, and they
Speaker 3: would literally come to the buffet table, take their food
Speaker 3: and go back to their desks and you had external
Speaker 3: guests coming taking their time to speak to share their
Speaker 3: perspective not only about clinical research, but about everything. And
Speaker 3: my message for all of you is, I understand, and
Speaker 3: I haven't been to this particular conference in years, but
Speaker 3: I have spoken at it in the past. It's patients
Speaker 3: as partners throughout everything, not just in clinical research. And
Speaker 3: that is also the distinction I believe between advocacy and engagement.
Speaker 2: And we can talk about that later, but the.
Speaker 3: Point is that the reason given was, but what if
Speaker 3: that person speaking at our lunch and learned decides to
Speaker 3: enroll in one of our clinical studies in the future,
Speaker 3: they're deidentified.
Speaker 2: I'm like, huh what? Because back then that's how a
Speaker 2: lot of people thought. And what's really important is.
Speaker 3: That we have all of the firewalls and the measures
Speaker 3: in place to protect people who do participate in our
Speaker 3: clinical research studies. And that should not in any way
Speaker 3: make anybody think that you can't sit down just like
Speaker 3: this to learn their experience, their lived experience, their perspective,
Speaker 3: their insights. It doesn't mean you can't participate and share
Speaker 3: information to the advocacy team that coordinates the patient advisory boards.
Speaker 2: The medical Advisory boards which are.
Speaker 3: Equal, and Sabina actually knows about that as well. So
Speaker 3: it's really important to show the value, and the value
Speaker 3: is by bringing in your colleagues across the different functions
Speaker 3: of the company and for those who are hesitant people
Speaker 3: in advocacy because they know as best they can without
Speaker 3: living with a disease themselves, they understand the patient perspective
Speaker 3: and they know the kinds of questions that everybody else
Speaker 3: should be asking, the kinds of information people should want
Speaker 3: to know if they're in clinops, if they're in marketing,
Speaker 3: if they're in medical affairs, if they're in legal, if
Speaker 3: they're in CMC. And so that's why I believe that
Speaker 3: advocacy is really the central node as opposed to engagement,
Speaker 3: because the patient advocacy team is the one that is
Speaker 3: to developing those relationships which we just heard are so
Speaker 3: important as just described in the National nem and pict
Speaker 3: Disease Foundation, which I've known and work with for many years,
Speaker 3: to understand and to have those relationships with patients and
Speaker 3: patient organizations is.
Speaker 2: The role of advocacy to then.
Speaker 3: Be your central node, to then work with clinical, to
Speaker 3: then work with everybody else. But it's just like medical affairs,
Speaker 3: there's only one function that owns the physician relationships and
Speaker 3: the strategies for the physician and kol relationships, and then
Speaker 3: you work cross functionally. And it's the same thing with
Speaker 3: patient advocacy. So I hope that answers in.
Speaker 1: A roundabuve now it does, and it actually kind of
Speaker 1: a nice little segue and maybe build upon for my
Speaker 1: next question. You know, we all know it's kind of
Speaker 1: a harsh reality that departments work in silos. It's I mean, yeah,
Speaker 1: you have clinopsy, medical affairs, you have all these different
Speaker 1: different departments within the organization and they're all trying to
Speaker 1: reach their own their own goals. How do you And
Speaker 1: I know emmic Is is unique in the like in
Speaker 1: the fact that you have a way to really kind
Speaker 1: of break down those silos when it comes to patient advocacy.
Speaker 1: So what are some of the ways that you do that.
Speaker 3: Well, I hope we're not unique, because I really believe
Speaker 3: this should be the way of the world, and that
Speaker 3: is you make sure you're at.
Speaker 2: The table with every cross functional meeting. That's it. And
Speaker 2: so if a company has.
Speaker 3: Brand meetings, matrix meetings, core mean, you know, everybody's got
Speaker 3: their lingo, right. But if you've got somebody from every
Speaker 3: major function that's going to be touching the program and
Speaker 3: they're at that table, then you're not as siloed. The
Speaker 3: silos still happen because everybody has their timelines and their
Speaker 3: list of things they need to do, and often it's
Speaker 3: just heads down and let's.
Speaker 2: Get through that checklist. And I think that that also.
Speaker 3: Speaks to what if he said at the beginning, which
Speaker 3: is you have to put the patient first. If you're
Speaker 3: not putting the patient first, and you're looking at a
Speaker 3: gant chart and you don't see a patient advisory board
Speaker 3: meeting up there, but you see the medical ad board
Speaker 3: and you don't see whatever it is, you're like, wait
Speaker 3: a minute, guys, stop, you can't do that if advocacy
Speaker 3: is not in the room. And so we really have
Speaker 3: put into the charters. And that's the other way that
Speaker 3: you can embed this, if you will, in the DNA,
Speaker 3: is that when these committees, these cross functional teams are
Speaker 3: created and then two years later, recreated, in three years
Speaker 3: after that, recreated again, and they change the names, but
Speaker 3: the functions are similar. You've got those charters and you
Speaker 3: need to make sure that advocacy is there.
Speaker 2: And I have a favorite quote.
Speaker 3: I'm not a big quoter because I like them, but
Speaker 3: I always remember them, but Shirley Chisholm. And I hope
Speaker 3: everybody here knows who Shirley Chisholm was. She was the
Speaker 3: first African American woman to serve in the US House
Speaker 3: of Representatives, and she was the first African American to
Speaker 3: run for President of the United States. And something that,
Speaker 3: as Chisholm said, has always stuck with me. If you're
Speaker 3: not given a seat at the table, bring a folding chair.
Speaker 2: That's it. And I and I you know, and I
Speaker 2: push that.
Speaker 3: And fortunately, I've been in the c suite for many
Speaker 3: years at Amicus, report directly to the CEO. And even
Speaker 3: still there can be a challenge of silos, particularly when
Speaker 3: people come from other companies and other corporate cultures and
Speaker 3: other experiences where maybe things weren't done quite the same
Speaker 3: and they don't always give themselves time to sit back,
Speaker 3: observe and learn context, and so they kind of push
Speaker 3: ahead and you're like, Okay, chill foot on the brake,
Speaker 3: and this is how we do it. And you see
Speaker 3: the difference in how people not only react but in
Speaker 3: how they appreciate what they can accomplish because they truly
Speaker 3: see that it's it's holding hands all around.
Speaker 1: No, I appreciate that, and I appreciate you pointing out
Speaker 1: that you know you are in the C suite, and
Speaker 1: I'm so happy to hear that there are more chief
Speaker 1: Patient officers out there, and I know there's several here
Speaker 1: within our audience. It's a good step forward, but you
Speaker 1: need to be handling it right and really keep pushing
Speaker 1: because there's a lot of obstacles in your way.
Speaker 2: So thank you for that.
Speaker 1: Okay.
Speaker 2: So we understand the.
Speaker 1: Importance of science and hard data in clinical trials. How
Speaker 1: do you balance scientific rigor and the real world needs
Speaker 1: and experiences of patients.
Speaker 3: It's a really good question and something that I have
Speaker 3: said for many years is data does not come from
Speaker 3: clinical studies alone. And so at Amicus, in our patient
Speaker 3: advocacy department, which is global. So we have a group
Speaker 3: based in Princeton, we have a group based in our
Speaker 3: international headquarters outside London. We've got somebody in Japan. We
Speaker 3: conduct a lot of research. Most recently we have done
Speaker 3: really a string of research projects on the lived experience
Speaker 3: and focusing in fabric disease, which is a rare lysosomal disorder,
Speaker 3: on the experience of females with FABRAE only because over
Speaker 3: the years excellent condition not thought to really affect females.
Speaker 2: They're just carriers and we're.
Speaker 3: Not going to go beyond that, and you will see
Speaker 3: that in other EXCELLNK diseases, particularly rare diseases as well.
Speaker 3: We wanted to make sure that we were understanding the
Speaker 3: experience of females, girls and women living with fabra so
Speaker 3: that we could learn where their gaps are in service
Speaker 3: in knowledge, et cetera, and where together we could co
Speaker 3: create and fill those gaps for the community.
Speaker 2: So we've done. We have many, many.
Speaker 3: Posters that have been presented. We have manuscripts in other areas.
Speaker 3: We just completed a piece that we did in conjunction
Speaker 3: with our market access and that was published January twod
Speaker 3: in the Journal of Health, Economic Outcomes and Research, and
Speaker 3: it's on the lived experience of people with late onset
Speaker 3: Pompeage disease. So it's both the emotional and the physical experience.
Speaker 3: So this is what I mean when I say that
Speaker 3: advocacy goes beyond the clinical studies or that patient engagement,
Speaker 3: and what we do goes beyond the clinical studies is
Speaker 3: to understand what that lived experience is and to do
Speaker 3: rigorous IRB approved research that that data is published or
Speaker 3: those manuscripts are published alongside the scientific and the clinical.
Speaker 2: Because that really helps medical.
Speaker 3: Affairs share their story with physicians and with KOLs, and
Speaker 3: it also helps your salespeople as well. So it's again
Speaker 3: it's all around disease awareness. It's not around product advocacy
Speaker 3: is above product you collaborate.
Speaker 2: With and you helped to in warm product development.
Speaker 3: But this is where the lived experience and the true
Speaker 3: connection I think happens with the patient community.
Speaker 2: Yeah.
Speaker 1: I think that's going to be a common thread that
Speaker 1: we're going to hear throughout this conference, is just looking
Speaker 1: at the patient and the caregiver as a whole. Is
Speaker 1: again our earlier presenter had touched upon, it's so important
Speaker 1: because it's not just it's not just lab numbers. It's
Speaker 1: there's so much more to the lived experience and it
Speaker 1: really speaks into and it really it does go from
Speaker 1: clinical clinical trial design award.
Speaker 3: Yeah, because and again it is important for everyone to
Speaker 3: know their numbers, right, we all need to know our numbers,
Speaker 3: whatever they may be, but we need to know what
Speaker 3: they mean, and we need to be able to have
Speaker 3: cogent conversations with our healthcare providers about those and sometimes
Speaker 3: for a whole list of really, you know, obvious reasons.
Speaker 3: Physicians don't always have the time, and so in advocacy,
Speaker 3: whether you're in industry, wherever you might be an advocacy,
Speaker 3: and particularly in the patient organizations, we are in a
Speaker 3: role to help people know what are the things.
Speaker 2: To ask about their numbers.
Speaker 3: It's not about medical advice, it's just what do you
Speaker 3: need to know or what can help you to better
Speaker 3: understand your numbers so you can have that conversation with
Speaker 3: your healthcare provider and you can have shared decision making,
Speaker 3: you can look at appropriate treatments and really be a
Speaker 3: partner in your own management. And I think that that's
Speaker 3: the piece that's really important too. And we hear from
Speaker 3: patients through our advisory boards and our patient champions and
Speaker 3: others that there still is this gap in resources to
Speaker 3: help them have those best conversations. But we also want
Speaker 3: to tell the physicians, here's something that you can initiate
Speaker 3: with your patients and your patient families to make the
Speaker 3: most of their visits.
Speaker 1: Hey, Jane, going back to one of the points you
Speaker 1: made earlier about we know that John Crowley was amazing
Speaker 1: started with the heart of the patient is so important
Speaker 1: right from the beginning. But what about if your a
Speaker 1: new employee coming into Amicus, So tell me some of
Speaker 1: your strategies there.
Speaker 3: So I'm really proud of where we are today.
Speaker 2: It took a while to get there.
Speaker 3: So together with our chief compliance officer and our Chief
Speaker 3: people officer, we've really said, look, guys, you've got to
Speaker 3: have advocacy in the beginning, because so many times in
Speaker 3: doing interview panels, people say we came because of your
Speaker 3: commitment to the patient community, to advocacy, et cetera. And frankly,
Speaker 3: we have had many people over the years work at
Speaker 3: Amicus who themselves or their families live with a rare
Speaker 3: disease and that's part of the attraction.
Speaker 2: But the onboarding process.
Speaker 3: If you will, we have a separate on our patient
Speaker 3: advocacy onboarding session that we do for every new employee.
Speaker 2: So that's separate.
Speaker 3: From the onboarding they get with how to do your
Speaker 3: expense reports, which, when you think about it, you know,
Speaker 3: when a new person comes into a company. It's a
Speaker 3: lot of the administrative logistical obviously, the compliance training, the
Speaker 3: master control, all that stuff, But you want to make
Speaker 3: sure that they're not around for two months before they
Speaker 3: have the opportunity to attend to lunch and learn they
Speaker 3: may have just missed one in the you know, in
Speaker 3: the rotation. Oops, And so we do that. Okay, that's
Speaker 3: one thing. So that's at the beginning. And again because
Speaker 3: we have advocacy at every cross functional team meeting, wherever
Speaker 3: that person might be, they're hearing it. If they're more
Speaker 3: on the support side, legal finance, etc.
Speaker 2: They're getting it.
Speaker 3: But also what we did over the last handful of
Speaker 3: years is in our performance management we have mission focused
Speaker 3: behaviors and the first one is patient first thinking. So
Speaker 3: in their goals and objectives in the mid year and
Speaker 3: year end, people have to show how they have met
Speaker 3: that mission focused behavior of patient first thinking. And we
Speaker 3: all know how the scales work and if they've met it,
Speaker 3: they've met it.
Speaker 2: But if they say they've done more, or their manager
Speaker 2: says they've done more, they need to explain it.
Speaker 3: And people's bonuses are tied to their mission focused behaviors.
Speaker 3: So a certain amount of your bonus is your performance, right,
Speaker 3: It's what you do, but it's how you do it
Speaker 3: where you can score extra points. So that's one thing
Speaker 3: and we're really pleased about that, and we've been able
Speaker 3: to even fine tune it over the last couple of years,
Speaker 3: so people know that this is a real thing. It's
Speaker 3: right up there with compliance and performance and.
Speaker 2: Innovation, etc.
Speaker 3: And this year, twenty twenty five is the first official year,
Speaker 3: if you will, of our PAIR program. It's called Patient
Speaker 3: Advocates in Residence. At some larger companies there may be preceptorships,
Speaker 3: there may be rotations where people can go in and
Speaker 3: out of different functions to learn more about the different
Speaker 3: aspects of the business, and advocacy is sometimes included there.
Speaker 3: We're too small a company to do that. All of
Speaker 3: our individual functional teams are too lean to let somebody
Speaker 3: go for four or six months into another department. So
Speaker 3: last year we completed the pilot year of PAIR and
Speaker 3: we had I think eleven people and they self nominated,
Speaker 3: and it was to understand more about what patient first
Speaker 3: thinking is and then to think about how they can
Speaker 3: integrate it into their own personal and professional development and
Speaker 3: into their functions so that they become the advocate in residence.
Speaker 3: They're they're almost like an ambassador because they're starting to
Speaker 3: think more like an advocate in a formal way. And
Speaker 3: then each person or people could team up did projects.
Speaker 3: So one of the projects, for example, was done by
Speaker 3: legal and it was something we already had started, but
Speaker 3: it was to move it even farther, and that is
Speaker 3: to put legal documents, particularly consulting agreements and things of
Speaker 3: that nature, in lay language, just making you know we
Speaker 3: were there.
Speaker 2: They took us father.
Speaker 3: There was a range of other things, and it was
Speaker 3: also that people are starting to think of about their
Speaker 3: own volunteerism outside of the company. And one of the
Speaker 3: people is now a member of her county's United Wayboard
Speaker 3: of Directors.
Speaker 2: So that's one thing.
Speaker 3: And now this year, yes it's still a self nomination,
Speaker 3: but the manager also has to endorse that nomination. And
Speaker 3: it's basically a six month program. There is a workshop
Speaker 3: once a month. We have external guests who helped to
Speaker 3: explain about what patient for us, thinking for them mean
Speaker 3: what they want industry to think of.
Speaker 2: And then we have internal We have.
Speaker 3: Case studies presented by internal colleagues who have demonstrated very
Speaker 3: strong patient first thinking, and so we've had people from marketing,
Speaker 3: we've had people from medical affairs, and these are people
Speaker 3: who sort of either innately or they this is just
Speaker 3: how they operate or it's something that they have built
Speaker 3: upon during their time at the company. And so we're
Speaker 3: really excited about that as well. And then people at
Speaker 3: the end of the year get to present their projects
Speaker 3: to the whole company and that type of thing.
Speaker 1: Now I love to hear how there's just there's simple
Speaker 1: ways that you can incorporate the patient voice and caregiver
Speaker 1: voice always to more intricate programs such as that. So
Speaker 1: it's interesting because I know with lunch and learns you've
Speaker 1: had some great experiences. Just real quickly, there was a
Speaker 1: story that you told us when we were chatting earlier
Speaker 1: about a lunch and learn where you brought at a
Speaker 1: patient in.
Speaker 2: Because I believe the gentleman and nobody had no it
Speaker 2: wasn't a lunch no.
Speaker 3: But I want to share this because it really is
Speaker 3: was a It was formative to me when I moved
Speaker 3: into rare diseases in the nineteen nineties or whatever it was.
Speaker 2: It was.
Speaker 3: It was a leap of faith and took me totally
Speaker 3: out of my comfort zone. I became the executive director
Speaker 3: of National TAYSACS and Ally Diseases Association, and I hadn't
Speaker 3: been in rare diseases before, and.
Speaker 2: We won't go there.
Speaker 3: But I was attending a conference for people with late
Speaker 3: onset tas acts, which is similar to late onset POMPEII,
Speaker 3: it's similar to some of the muscular dystrophees. It's a
Speaker 3: progressive neuromuscular disorder and very small community obviously. And we
Speaker 3: were in a hotel room much smaller than this in Philadelphia,
Speaker 3: and they were waiting for the research session to begin,
Speaker 3: and there was somebody who was going to be speaking
Speaker 3: and sharing a basic researcher but sharing his latest information
Speaker 3: and results of looking at upstream and downstream effects of proteins.
Speaker 3: And you had people lined up somewhere in wheelchairs, somewhere
Speaker 3: had their walkers, somebody had their service dog. Their family
Speaker 3: and friends were seated behind them. There were people from industry,
Speaker 3: other physicians. So this doctor came up, nice introduction, and
Speaker 3: he looks at the room and he sees the people
Speaker 3: living with this disease in front of him, and he's
Speaker 3: just looking like this, and he says not and then
Speaker 3: he like deer in the headlights and he said, I'm
Speaker 3: so sorry.
Speaker 2: I'm so sorry.
Speaker 3: I've been working on this disease for almost thirty years
Speaker 3: and I have never met anybody living with late onset
Speaker 3: TA sex disease. And I sat in that room and
Speaker 3: I was like, I won't tell you what I really thought.
Speaker 3: I was like, if I have anything to say, either
Speaker 3: at the organization.
Speaker 2: Or wherever, that's never going to happen again.
Speaker 3: And so I think back to the lunch and learns
Speaker 3: at and Amicus in the early days, when you did
Speaker 3: have a couple of people say I can't go because
Speaker 3: what if that person, you know, three years from that
Speaker 3: goes into one of our clinical studies.
Speaker 2: I'm like, you don't get it. We need to understand it.
Speaker 3: And those with the Lunch and Learns in particular, scientists
Speaker 3: love them because they're looking at diseases on us literally
Speaker 3: on a microscopic level. They may have cells from somebody's
Speaker 3: fibroblast and they're looking at it and they're thinking about
Speaker 3: it and whatever, you know, the scientists do.
Speaker 2: I was an English major, so that's as far as
Speaker 2: I go.
Speaker 3: But to then have the opportunity to meet someone to
Speaker 3: listen to their experience, to ask them questions, to give
Speaker 3: them a tour of the lab. You know they say
Speaker 3: when you give, you get more than the person you're
Speaker 3: giving to. The scientists really really experience that, and so
Speaker 3: that's something that has always stuck with me. And again
Speaker 3: that's where I feel it's advocacy, not only engagement. To me,
Speaker 3: advocacy is linear and goes across everything, and patient engagement
Speaker 3: is more vertical, and you have it at different and
Speaker 3: important time points throughout, not just the drug development or
Speaker 3: the approval, but throughout. And that also counts when you're
Speaker 3: talking about a clinical development program.
Speaker 2: That doesn't make the grade.
Speaker 3: And I'm looking at you because I know Nieman Pick
Speaker 3: and you know, thank goodness for Garrett, but look at
Speaker 3: all the other programs that didn't make it. We've had
Speaker 3: programs that didn't make it. I'm sure just about everybody
Speaker 3: here has.
Speaker 2: Had an experience.
Speaker 3: And what you do when something doesn't make it is
Speaker 3: as important reputationally as to when it does.
Speaker 2: And that's where advocacy comes in. Wow. Well that's a
Speaker 2: great point to end on.
Speaker 1: So thank you so much, Jane. Again, always an honor,
Speaker 1: and especially to share a stage like this to hear
Speaker 1: your words.
Speaker 2: So thank you so much. Thanks everybody, thank you,