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