WEBVTT 1 00:00:00.080 --> 00:00:02.919 Well, good morning everyone. My name is Sabina Kanin and 2 00:00:03.120 --> 00:00:05.759 I'm a patient and a caregiver as well. And I 3 00:00:05.799 --> 00:00:08.199 can't tell you how honored I am to be sitting 4 00:00:08.240 --> 00:00:12.439 here and sharing the stage with Jane Gershkowitz of Amikis Therapeutics. 5 00:00:13.080 --> 00:00:15.240 She truly is a hero of mine. We've known each 6 00:00:15.279 --> 00:00:18.280 other for close to seventeen. 7 00:00:17.920 --> 00:00:20.000 Years, yeah, at least. 8 00:00:19.839 --> 00:00:22.280 Around seventeen years, so it's an honor to have her 9 00:00:22.320 --> 00:00:25.480 here and to hear her words of wisdom. How she's 10 00:00:25.519 --> 00:00:29.839 been able to really embed the role of patient advocacy 11 00:00:29.920 --> 00:00:31.359 within the corporate DNA. 12 00:00:32.159 --> 00:00:34.439 So Jane, welcome, Thank. 13 00:00:34.280 --> 00:00:37.280 You, Sabina, and yes, full disclosure, we have known each 14 00:00:37.280 --> 00:00:39.920 other for a very long time. But when I was 15 00:00:40.039 --> 00:00:43.359 asked to do this session with you all this morning, 16 00:00:45.079 --> 00:00:48.719 Kate said, gee, what do you think about having a 17 00:00:48.880 --> 00:00:51.200 patient advocate interview you, you. 18 00:00:51.159 --> 00:00:52.719 Know, to do the chat. I said, I think that'd 19 00:00:52.719 --> 00:00:53.119 be great. 20 00:00:53.520 --> 00:00:56.159 She said, yeah, We've got this woman who's worked with 21 00:00:56.280 --> 00:00:56.920 us before. 22 00:00:56.960 --> 00:00:59.280 She's a great patient advocate. Her name is Sabina. 23 00:00:59.359 --> 00:01:03.039 I was like, yeah, I've known Sabina and her family 24 00:01:04.439 --> 00:01:08.519 from very early days at Amicus, so it's equally an 25 00:01:08.560 --> 00:01:12.280 honor for me to be here and to be interviewed 26 00:01:12.359 --> 00:01:14.599 by a colleague and a friend. 27 00:01:15.480 --> 00:01:16.439 Thank you, Jane. 28 00:01:16.799 --> 00:01:18.840 Okay, well let's get started, because I know you do 29 00:01:18.920 --> 00:01:20.200 have a wealth of information to. 30 00:01:20.200 --> 00:01:22.079 Share and a pressure. 31 00:01:25.079 --> 00:01:27.719 Well we know that if you're all attending this conference, 32 00:01:27.760 --> 00:01:31.000 you care about patient engagement, right everyone here. You wouldn't 33 00:01:31.000 --> 00:01:34.680 be here if you didn't. But however, we all face 34 00:01:35.120 --> 00:01:38.200 different obstacles. No matter what position you are in your organization, 35 00:01:38.239 --> 00:01:42.120 you're gonna you're gonna face different obstacles about really kind 36 00:01:42.159 --> 00:01:47.319 of selling the importance of patient advocacy within within the organization. 37 00:01:47.959 --> 00:01:51.560 So how do you communicate the real value of patient 38 00:01:51.959 --> 00:01:57.640 advocacy and secure leadership buy in and their resources. 39 00:01:58.000 --> 00:02:01.000 Yeah, so absolutely, there are a lot of challenges that 40 00:02:01.079 --> 00:02:06.159 we all face in our organizations, and it also depends 41 00:02:06.280 --> 00:02:10.400 upon where we are in the development of our organizations. 42 00:02:10.479 --> 00:02:14.879 So very young companies today are sometimes bringing in lead 43 00:02:14.960 --> 00:02:16.759 patient advocates. 44 00:02:16.159 --> 00:02:16.800 From the get go. 45 00:02:16.960 --> 00:02:20.800 Sometimes they're among the very first few people hired. And 46 00:02:20.840 --> 00:02:23.639 then you have other companies where this has really been 47 00:02:23.719 --> 00:02:25.800 part of their DNA And I'll just quickly say that 48 00:02:25.879 --> 00:02:30.080 at Amicus Therapeutics that's really been the case. In January 49 00:02:30.120 --> 00:02:33.680 of two thousand and five, when our former president CEO, 50 00:02:34.240 --> 00:02:38.080 John Crowley came on board as the CEO. Then he 51 00:02:38.199 --> 00:02:42.639 had already been focused on the patient story because he 52 00:02:42.759 --> 00:02:47.280 was not only an entrepreneur in biotech, but he was 53 00:02:47.400 --> 00:02:50.319 a parent and patient advocate for two of his three 54 00:02:50.400 --> 00:02:54.560 children who live with a rare disease, and so he 55 00:02:54.639 --> 00:02:57.719 had already experienced that in a couple other companies when 56 00:02:57.800 --> 00:03:01.319 which he started in, which then eventually had been acquired 57 00:03:01.759 --> 00:03:04.199 into a larger rare disease company. 58 00:03:03.919 --> 00:03:05.919 And he knew the value of that. 59 00:03:06.759 --> 00:03:09.919 So it was very different at Amicus when I came 60 00:03:09.919 --> 00:03:11.520 there almost nineteen years ago. 61 00:03:12.479 --> 00:03:14.759 We weren't necessarily pushing. 62 00:03:14.400 --> 00:03:17.680 The rock up the hill with the leadership in terms 63 00:03:17.719 --> 00:03:20.400 of the CEO, but I will say that we were 64 00:03:20.439 --> 00:03:23.000 pushing the rock up the hill with many of our colleagues, 65 00:03:23.039 --> 00:03:27.319 in particular those in clinical operations. In clinical research, and 66 00:03:27.400 --> 00:03:30.599 back then the reason was when you held a lunch 67 00:03:30.639 --> 00:03:33.159 and learn, which we always did, and we can. 68 00:03:33.280 --> 00:03:34.560 Talk about that as well. 69 00:03:35.280 --> 00:03:37.599 There were actually people in clinical who would say, well, 70 00:03:37.759 --> 00:03:39.319 I can't go to that lunch and learn, and they 71 00:03:39.360 --> 00:03:42.360 would literally come to the buffet table, take their food 72 00:03:42.479 --> 00:03:44.840 and go back to their desks and you had external 73 00:03:44.919 --> 00:03:48.919 guests coming taking their time to speak to share their 74 00:03:48.960 --> 00:03:53.479 perspective not only about clinical research, but about everything. And 75 00:03:53.560 --> 00:03:56.000 my message for all of you is, I understand, and 76 00:03:56.039 --> 00:03:58.719 I haven't been to this particular conference in years, but 77 00:03:58.759 --> 00:04:01.599 I have spoken at it in the past. It's patients 78 00:04:01.599 --> 00:04:06.319 as partners throughout everything, not just in clinical research. And 79 00:04:06.360 --> 00:04:09.960 that is also the distinction I believe between advocacy and engagement. 80 00:04:10.120 --> 00:04:12.840 And we can talk about that later, but the. 81 00:04:12.879 --> 00:04:17.000 Point is that the reason given was, but what if 82 00:04:17.079 --> 00:04:20.319 that person speaking at our lunch and learned decides to 83 00:04:20.480 --> 00:04:23.040 enroll in one of our clinical studies in the future, 84 00:04:23.839 --> 00:04:24.920 they're deidentified. 85 00:04:24.959 --> 00:04:28.879 I'm like, huh what? Because back then that's how a 86 00:04:28.879 --> 00:04:32.480 lot of people thought. And what's really important is. 87 00:04:32.399 --> 00:04:36.480 That we have all of the firewalls and the measures 88 00:04:36.480 --> 00:04:40.720 in place to protect people who do participate in our 89 00:04:40.759 --> 00:04:44.759 clinical research studies. And that should not in any way 90 00:04:44.839 --> 00:04:47.920 make anybody think that you can't sit down just like 91 00:04:48.000 --> 00:04:53.199 this to learn their experience, their lived experience, their perspective, 92 00:04:53.279 --> 00:04:57.600 their insights. It doesn't mean you can't participate and share 93 00:04:57.680 --> 00:05:03.000 information to the advocacy team that coordinates the patient advisory boards. 94 00:05:03.680 --> 00:05:05.680 The medical Advisory boards which are. 95 00:05:06.519 --> 00:05:10.519 Equal, and Sabina actually knows about that as well. So 96 00:05:10.920 --> 00:05:13.920 it's really important to show the value, and the value 97 00:05:14.040 --> 00:05:18.800 is by bringing in your colleagues across the different functions 98 00:05:18.839 --> 00:05:22.639 of the company and for those who are hesitant people 99 00:05:22.680 --> 00:05:26.079 in advocacy because they know as best they can without 100 00:05:26.120 --> 00:05:30.000 living with a disease themselves, they understand the patient perspective 101 00:05:30.279 --> 00:05:33.160 and they know the kinds of questions that everybody else 102 00:05:33.319 --> 00:05:37.160 should be asking, the kinds of information people should want 103 00:05:37.199 --> 00:05:40.639 to know if they're in clinops, if they're in marketing, 104 00:05:40.720 --> 00:05:44.720 if they're in medical affairs, if they're in legal, if 105 00:05:44.759 --> 00:05:49.079 they're in CMC. And so that's why I believe that 106 00:05:49.240 --> 00:05:55.240 advocacy is really the central node as opposed to engagement, 107 00:05:55.319 --> 00:05:59.759 because the patient advocacy team is the one that is 108 00:05:59.800 --> 00:06:03.160 to developing those relationships which we just heard are so 109 00:06:03.279 --> 00:06:06.920 important as just described in the National nem and pict 110 00:06:06.959 --> 00:06:10.160 Disease Foundation, which I've known and work with for many years, 111 00:06:11.000 --> 00:06:14.800 to understand and to have those relationships with patients and 112 00:06:14.839 --> 00:06:16.319 patient organizations is. 113 00:06:16.240 --> 00:06:18.480 The role of advocacy to then. 114 00:06:18.399 --> 00:06:21.560 Be your central node, to then work with clinical, to 115 00:06:21.680 --> 00:06:25.800 then work with everybody else. But it's just like medical affairs, 116 00:06:25.839 --> 00:06:31.240 there's only one function that owns the physician relationships and 117 00:06:31.279 --> 00:06:34.920 the strategies for the physician and kol relationships, and then 118 00:06:34.959 --> 00:06:37.560 you work cross functionally. And it's the same thing with 119 00:06:37.639 --> 00:06:40.360 patient advocacy. So I hope that answers in. 120 00:06:40.279 --> 00:06:42.399 A roundabuve now it does, and it actually kind of 121 00:06:42.480 --> 00:06:45.160 a nice little segue and maybe build upon for my 122 00:06:45.199 --> 00:06:47.519 next question. You know, we all know it's kind of 123 00:06:47.519 --> 00:06:52.319 a harsh reality that departments work in silos. It's I mean, yeah, 124 00:06:52.399 --> 00:06:55.000 you have clinopsy, medical affairs, you have all these different 125 00:06:56.079 --> 00:06:58.519 different departments within the organization and they're all trying to 126 00:06:58.879 --> 00:07:02.240 reach their own their own goals. How do you And 127 00:07:02.279 --> 00:07:05.360 I know emmic Is is unique in the like in 128 00:07:05.360 --> 00:07:08.759 the fact that you have a way to really kind 129 00:07:08.800 --> 00:07:11.279 of break down those silos when it comes to patient advocacy. 130 00:07:11.639 --> 00:07:13.879 So what are some of the ways that you do that. 131 00:07:14.000 --> 00:07:16.560 Well, I hope we're not unique, because I really believe 132 00:07:16.639 --> 00:07:19.160 this should be the way of the world, and that 133 00:07:19.399 --> 00:07:21.399 is you make sure you're at. 134 00:07:21.240 --> 00:07:24.800 The table with every cross functional meeting. That's it. And 135 00:07:24.839 --> 00:07:26.480 so if a company has. 136 00:07:27.800 --> 00:07:32.160 Brand meetings, matrix meetings, core mean, you know, everybody's got 137 00:07:32.160 --> 00:07:36.680 their lingo, right. But if you've got somebody from every 138 00:07:36.759 --> 00:07:40.199 major function that's going to be touching the program and 139 00:07:40.240 --> 00:07:44.879 they're at that table, then you're not as siloed. The 140 00:07:44.920 --> 00:07:48.959 silos still happen because everybody has their timelines and their 141 00:07:49.000 --> 00:07:51.600 list of things they need to do, and often it's 142 00:07:51.680 --> 00:07:53.000 just heads down and let's. 143 00:07:52.839 --> 00:07:56.000 Get through that checklist. And I think that that also. 144 00:07:55.800 --> 00:07:58.160 Speaks to what if he said at the beginning, which 145 00:07:58.240 --> 00:08:00.879 is you have to put the patient first. If you're 146 00:08:00.920 --> 00:08:03.360 not putting the patient first, and you're looking at a 147 00:08:03.399 --> 00:08:06.399 gant chart and you don't see a patient advisory board 148 00:08:06.680 --> 00:08:08.920 meeting up there, but you see the medical ad board 149 00:08:08.959 --> 00:08:11.439 and you don't see whatever it is, you're like, wait 150 00:08:11.480 --> 00:08:14.759 a minute, guys, stop, you can't do that if advocacy 151 00:08:14.800 --> 00:08:17.759 is not in the room. And so we really have 152 00:08:17.879 --> 00:08:20.439 put into the charters. And that's the other way that 153 00:08:20.480 --> 00:08:23.360 you can embed this, if you will, in the DNA, 154 00:08:23.560 --> 00:08:26.680 is that when these committees, these cross functional teams are 155 00:08:26.720 --> 00:08:29.720 created and then two years later, recreated, in three years 156 00:08:29.759 --> 00:08:32.720 after that, recreated again, and they change the names, but 157 00:08:32.759 --> 00:08:36.840 the functions are similar. You've got those charters and you 158 00:08:36.960 --> 00:08:39.279 need to make sure that advocacy is there. 159 00:08:39.639 --> 00:08:42.279 And I have a favorite quote. 160 00:08:42.320 --> 00:08:43.919 I'm not a big quoter because I like them, but 161 00:08:43.960 --> 00:08:47.159 I always remember them, but Shirley Chisholm. And I hope 162 00:08:47.200 --> 00:08:50.240 everybody here knows who Shirley Chisholm was. She was the 163 00:08:50.279 --> 00:08:53.360 first African American woman to serve in the US House 164 00:08:53.360 --> 00:08:56.919 of Representatives, and she was the first African American to 165 00:08:57.000 --> 00:08:59.759 run for President of the United States. And something that, 166 00:09:00.000 --> 00:09:03.080 as Chisholm said, has always stuck with me. If you're 167 00:09:03.120 --> 00:09:06.960 not given a seat at the table, bring a folding chair. 168 00:09:08.600 --> 00:09:14.519 That's it. And I and I you know, and I 169 00:09:14.600 --> 00:09:15.120 push that. 170 00:09:15.320 --> 00:09:17.519 And fortunately, I've been in the c suite for many 171 00:09:17.600 --> 00:09:22.080 years at Amicus, report directly to the CEO. And even 172 00:09:22.240 --> 00:09:26.080 still there can be a challenge of silos, particularly when 173 00:09:26.120 --> 00:09:30.279 people come from other companies and other corporate cultures and 174 00:09:30.399 --> 00:09:33.759 other experiences where maybe things weren't done quite the same 175 00:09:34.200 --> 00:09:36.960 and they don't always give themselves time to sit back, 176 00:09:37.039 --> 00:09:40.279 observe and learn context, and so they kind of push 177 00:09:40.360 --> 00:09:44.600 ahead and you're like, Okay, chill foot on the brake, 178 00:09:45.159 --> 00:09:48.399 and this is how we do it. And you see 179 00:09:48.440 --> 00:09:52.039 the difference in how people not only react but in 180 00:09:52.159 --> 00:09:57.480 how they appreciate what they can accomplish because they truly 181 00:09:57.519 --> 00:09:59.639 see that it's it's holding hands all around. 182 00:10:00.759 --> 00:10:02.720 No, I appreciate that, and I appreciate you pointing out 183 00:10:02.720 --> 00:10:04.679 that you know you are in the C suite, and 184 00:10:05.080 --> 00:10:07.799 I'm so happy to hear that there are more chief 185 00:10:07.840 --> 00:10:10.159 Patient officers out there, and I know there's several here 186 00:10:10.200 --> 00:10:13.960 within our audience. It's a good step forward, but you 187 00:10:14.039 --> 00:10:16.480 need to be handling it right and really keep pushing 188 00:10:16.480 --> 00:10:18.799 because there's a lot of obstacles in your way. 189 00:10:18.879 --> 00:10:19.679 So thank you for that. 190 00:10:20.440 --> 00:10:20.720 Okay. 191 00:10:20.759 --> 00:10:21.759 So we understand the. 192 00:10:21.679 --> 00:10:26.200 Importance of science and hard data in clinical trials. How 193 00:10:26.200 --> 00:10:30.919 do you balance scientific rigor and the real world needs 194 00:10:30.960 --> 00:10:33.000 and experiences of patients. 195 00:10:34.000 --> 00:10:37.039 It's a really good question and something that I have 196 00:10:37.080 --> 00:10:39.639 said for many years is data does not come from 197 00:10:39.679 --> 00:10:44.440 clinical studies alone. And so at Amicus, in our patient 198 00:10:44.519 --> 00:10:48.240 advocacy department, which is global. So we have a group 199 00:10:48.679 --> 00:10:51.000 based in Princeton, we have a group based in our 200 00:10:51.000 --> 00:10:55.519 international headquarters outside London. We've got somebody in Japan. We 201 00:10:55.600 --> 00:11:00.759 conduct a lot of research. Most recently we have done 202 00:11:00.960 --> 00:11:05.080 really a string of research projects on the lived experience 203 00:11:05.279 --> 00:11:09.320 and focusing in fabric disease, which is a rare lysosomal disorder, 204 00:11:10.240 --> 00:11:16.200 on the experience of females with FABRAE only because over 205 00:11:16.240 --> 00:11:20.799 the years excellent condition not thought to really affect females. 206 00:11:20.879 --> 00:11:22.840 They're just carriers and we're. 207 00:11:22.679 --> 00:11:24.919 Not going to go beyond that, and you will see 208 00:11:24.919 --> 00:11:28.679 that in other EXCELLNK diseases, particularly rare diseases as well. 209 00:11:29.639 --> 00:11:32.799 We wanted to make sure that we were understanding the 210 00:11:33.000 --> 00:11:37.559 experience of females, girls and women living with fabra so 211 00:11:37.600 --> 00:11:40.639 that we could learn where their gaps are in service 212 00:11:40.840 --> 00:11:43.639 in knowledge, et cetera, and where together we could co 213 00:11:43.720 --> 00:11:46.159 create and fill those gaps for the community. 214 00:11:46.679 --> 00:11:50.080 So we've done. We have many, many. 215 00:11:49.879 --> 00:11:54.799 Posters that have been presented. We have manuscripts in other areas. 216 00:11:54.840 --> 00:11:58.399 We just completed a piece that we did in conjunction 217 00:11:58.559 --> 00:12:04.120 with our market access and that was published January twod 218 00:12:04.399 --> 00:12:09.639 in the Journal of Health, Economic Outcomes and Research, and 219 00:12:09.679 --> 00:12:13.240 it's on the lived experience of people with late onset 220 00:12:13.279 --> 00:12:17.840 Pompeage disease. So it's both the emotional and the physical experience. 221 00:12:18.279 --> 00:12:20.000 So this is what I mean when I say that 222 00:12:20.200 --> 00:12:24.039 advocacy goes beyond the clinical studies or that patient engagement, 223 00:12:24.120 --> 00:12:27.320 and what we do goes beyond the clinical studies is 224 00:12:27.360 --> 00:12:30.200 to understand what that lived experience is and to do 225 00:12:30.360 --> 00:12:35.200 rigorous IRB approved research that that data is published or 226 00:12:35.240 --> 00:12:40.759 those manuscripts are published alongside the scientific and the clinical. 227 00:12:40.519 --> 00:12:42.559 Because that really helps medical. 228 00:12:42.159 --> 00:12:47.039 Affairs share their story with physicians and with KOLs, and 229 00:12:47.080 --> 00:12:52.039 it also helps your salespeople as well. So it's again 230 00:12:52.120 --> 00:12:56.120 it's all around disease awareness. It's not around product advocacy 231 00:12:56.159 --> 00:12:59.159 is above product you collaborate. 232 00:12:58.600 --> 00:13:01.200 With and you helped to in warm product development. 233 00:13:02.039 --> 00:13:05.720 But this is where the lived experience and the true 234 00:13:05.759 --> 00:13:09.080 connection I think happens with the patient community. 235 00:13:09.879 --> 00:13:10.080 Yeah. 236 00:13:10.080 --> 00:13:11.480 I think that's going to be a common thread that 237 00:13:11.519 --> 00:13:13.879 we're going to hear throughout this conference, is just looking 238 00:13:13.919 --> 00:13:16.440 at the patient and the caregiver as a whole. Is 239 00:13:16.919 --> 00:13:21.639 again our earlier presenter had touched upon, it's so important 240 00:13:21.639 --> 00:13:25.320 because it's not just it's not just lab numbers. It's 241 00:13:25.360 --> 00:13:28.679 there's so much more to the lived experience and it 242 00:13:28.720 --> 00:13:31.879 really speaks into and it really it does go from 243 00:13:31.919 --> 00:13:34.320 clinical clinical trial design award. 244 00:13:34.559 --> 00:13:37.440 Yeah, because and again it is important for everyone to 245 00:13:37.480 --> 00:13:39.960 know their numbers, right, we all need to know our numbers, 246 00:13:39.960 --> 00:13:42.360 whatever they may be, but we need to know what 247 00:13:42.399 --> 00:13:45.559 they mean, and we need to be able to have 248 00:13:46.200 --> 00:13:51.320 cogent conversations with our healthcare providers about those and sometimes 249 00:13:51.360 --> 00:13:55.879 for a whole list of really, you know, obvious reasons. 250 00:13:56.480 --> 00:14:01.600 Physicians don't always have the time, and so in advocacy, 251 00:14:01.679 --> 00:14:04.879 whether you're in industry, wherever you might be an advocacy, 252 00:14:04.919 --> 00:14:08.960 and particularly in the patient organizations, we are in a 253 00:14:09.039 --> 00:14:12.200 role to help people know what are the things. 254 00:14:11.919 --> 00:14:13.559 To ask about their numbers. 255 00:14:13.559 --> 00:14:16.600 It's not about medical advice, it's just what do you 256 00:14:16.759 --> 00:14:20.279 need to know or what can help you to better 257 00:14:20.360 --> 00:14:23.879 understand your numbers so you can have that conversation with 258 00:14:23.919 --> 00:14:27.919 your healthcare provider and you can have shared decision making, 259 00:14:28.240 --> 00:14:31.960 you can look at appropriate treatments and really be a 260 00:14:32.000 --> 00:14:34.919 partner in your own management. And I think that that's 261 00:14:35.000 --> 00:14:38.600 the piece that's really important too. And we hear from 262 00:14:38.919 --> 00:14:43.159 patients through our advisory boards and our patient champions and 263 00:14:43.279 --> 00:14:48.720 others that there still is this gap in resources to 264 00:14:48.799 --> 00:14:52.279 help them have those best conversations. But we also want 265 00:14:52.320 --> 00:14:55.639 to tell the physicians, here's something that you can initiate 266 00:14:56.200 --> 00:14:59.120 with your patients and your patient families to make the 267 00:14:59.120 --> 00:15:00.159 most of their visits. 268 00:15:02.080 --> 00:15:04.120 Hey, Jane, going back to one of the points you 269 00:15:04.159 --> 00:15:07.759 made earlier about we know that John Crowley was amazing 270 00:15:08.000 --> 00:15:11.840 started with the heart of the patient is so important 271 00:15:12.159 --> 00:15:15.559 right from the beginning. But what about if your a 272 00:15:15.639 --> 00:15:19.600 new employee coming into Amicus, So tell me some of 273 00:15:19.679 --> 00:15:20.759 your strategies there. 274 00:15:21.080 --> 00:15:23.879 So I'm really proud of where we are today. 275 00:15:24.159 --> 00:15:26.120 It took a while to get there. 276 00:15:27.399 --> 00:15:30.960 So together with our chief compliance officer and our Chief 277 00:15:31.000 --> 00:15:35.320 people officer, we've really said, look, guys, you've got to 278 00:15:35.320 --> 00:15:38.279 have advocacy in the beginning, because so many times in 279 00:15:38.399 --> 00:15:42.360 doing interview panels, people say we came because of your 280 00:15:42.440 --> 00:15:46.720 commitment to the patient community, to advocacy, et cetera. And frankly, 281 00:15:46.759 --> 00:15:48.960 we have had many people over the years work at 282 00:15:49.000 --> 00:15:52.360 Amicus who themselves or their families live with a rare 283 00:15:52.480 --> 00:15:54.360 disease and that's part of the attraction. 284 00:15:55.840 --> 00:15:58.240 But the onboarding process. 285 00:15:57.799 --> 00:16:01.799 If you will, we have a separate on our patient 286 00:16:01.879 --> 00:16:07.120 advocacy onboarding session that we do for every new employee. 287 00:16:08.360 --> 00:16:09.440 So that's separate. 288 00:16:09.120 --> 00:16:11.200 From the onboarding they get with how to do your 289 00:16:11.240 --> 00:16:14.320 expense reports, which, when you think about it, you know, 290 00:16:14.320 --> 00:16:16.720 when a new person comes into a company. It's a 291 00:16:16.759 --> 00:16:22.360 lot of the administrative logistical obviously, the compliance training, the 292 00:16:22.399 --> 00:16:26.080 master control, all that stuff, But you want to make 293 00:16:26.120 --> 00:16:28.840 sure that they're not around for two months before they 294 00:16:28.879 --> 00:16:31.159 have the opportunity to attend to lunch and learn they 295 00:16:31.200 --> 00:16:32.720 may have just missed one in the you know, in 296 00:16:32.759 --> 00:16:37.720 the rotation. Oops, And so we do that. Okay, that's 297 00:16:37.720 --> 00:16:41.200 one thing. So that's at the beginning. And again because 298 00:16:41.240 --> 00:16:44.919 we have advocacy at every cross functional team meeting, wherever 299 00:16:45.039 --> 00:16:48.080 that person might be, they're hearing it. If they're more 300 00:16:48.240 --> 00:16:50.519 on the support side, legal finance, etc. 301 00:16:50.960 --> 00:16:51.759 They're getting it. 302 00:16:53.279 --> 00:16:57.240 But also what we did over the last handful of 303 00:16:57.320 --> 00:17:01.879 years is in our performance management we have mission focused 304 00:17:01.960 --> 00:17:06.480 behaviors and the first one is patient first thinking. So 305 00:17:06.759 --> 00:17:09.119 in their goals and objectives in the mid year and 306 00:17:09.200 --> 00:17:12.880 year end, people have to show how they have met 307 00:17:12.960 --> 00:17:16.359 that mission focused behavior of patient first thinking. And we 308 00:17:16.400 --> 00:17:18.960 all know how the scales work and if they've met it, 309 00:17:19.000 --> 00:17:19.599 they've met it. 310 00:17:19.640 --> 00:17:22.400 But if they say they've done more, or their manager 311 00:17:22.440 --> 00:17:24.720 says they've done more, they need to explain it. 312 00:17:25.400 --> 00:17:28.799 And people's bonuses are tied to their mission focused behaviors. 313 00:17:29.000 --> 00:17:33.559 So a certain amount of your bonus is your performance, right, 314 00:17:35.599 --> 00:17:38.480 It's what you do, but it's how you do it 315 00:17:38.519 --> 00:17:42.240 where you can score extra points. So that's one thing 316 00:17:42.880 --> 00:17:45.640 and we're really pleased about that, and we've been able 317 00:17:45.720 --> 00:17:49.200 to even fine tune it over the last couple of years, 318 00:17:49.200 --> 00:17:51.640 so people know that this is a real thing. It's 319 00:17:51.720 --> 00:17:54.519 right up there with compliance and performance and. 320 00:17:54.400 --> 00:17:56.240 Innovation, etc. 321 00:17:57.279 --> 00:18:02.200 And this year, twenty twenty five is the first official year, 322 00:18:02.240 --> 00:18:06.440 if you will, of our PAIR program. It's called Patient 323 00:18:06.640 --> 00:18:13.200 Advocates in Residence. At some larger companies there may be preceptorships, 324 00:18:13.240 --> 00:18:15.599 there may be rotations where people can go in and 325 00:18:15.640 --> 00:18:18.240 out of different functions to learn more about the different 326 00:18:18.240 --> 00:18:22.279 aspects of the business, and advocacy is sometimes included there. 327 00:18:22.559 --> 00:18:24.920 We're too small a company to do that. All of 328 00:18:24.960 --> 00:18:27.920 our individual functional teams are too lean to let somebody 329 00:18:27.960 --> 00:18:31.480 go for four or six months into another department. So 330 00:18:31.720 --> 00:18:35.599 last year we completed the pilot year of PAIR and 331 00:18:36.359 --> 00:18:40.000 we had I think eleven people and they self nominated, 332 00:18:40.079 --> 00:18:42.720 and it was to understand more about what patient first 333 00:18:42.799 --> 00:18:45.440 thinking is and then to think about how they can 334 00:18:45.519 --> 00:18:49.200 integrate it into their own personal and professional development and 335 00:18:49.319 --> 00:18:53.880 into their functions so that they become the advocate in residence. 336 00:18:53.960 --> 00:18:57.319 They're they're almost like an ambassador because they're starting to 337 00:18:57.400 --> 00:19:00.640 think more like an advocate in a formal way. And 338 00:19:00.680 --> 00:19:04.400 then each person or people could team up did projects. 339 00:19:05.759 --> 00:19:08.279 So one of the projects, for example, was done by 340 00:19:08.480 --> 00:19:12.400 legal and it was something we already had started, but 341 00:19:12.480 --> 00:19:15.359 it was to move it even farther, and that is 342 00:19:15.519 --> 00:19:19.720 to put legal documents, particularly consulting agreements and things of 343 00:19:19.759 --> 00:19:23.240 that nature, in lay language, just making you know we 344 00:19:23.599 --> 00:19:24.279 were there. 345 00:19:24.599 --> 00:19:25.519 They took us father. 346 00:19:26.279 --> 00:19:28.160 There was a range of other things, and it was 347 00:19:28.200 --> 00:19:31.039 also that people are starting to think of about their 348 00:19:31.039 --> 00:19:34.480 own volunteerism outside of the company. And one of the 349 00:19:34.519 --> 00:19:38.440 people is now a member of her county's United Wayboard 350 00:19:38.599 --> 00:19:39.759 of Directors. 351 00:19:40.160 --> 00:19:40.960 So that's one thing. 352 00:19:42.000 --> 00:19:45.359 And now this year, yes it's still a self nomination, 353 00:19:45.519 --> 00:19:49.680 but the manager also has to endorse that nomination. And 354 00:19:49.759 --> 00:19:52.839 it's basically a six month program. There is a workshop 355 00:19:52.920 --> 00:19:56.480 once a month. We have external guests who helped to 356 00:19:56.559 --> 00:19:59.839 explain about what patient for us, thinking for them mean 357 00:20:00.480 --> 00:20:02.079 what they want industry to think of. 358 00:20:02.599 --> 00:20:04.599 And then we have internal We have. 359 00:20:04.599 --> 00:20:09.960 Case studies presented by internal colleagues who have demonstrated very 360 00:20:10.000 --> 00:20:13.000 strong patient first thinking, and so we've had people from marketing, 361 00:20:13.400 --> 00:20:16.240 we've had people from medical affairs, and these are people 362 00:20:16.279 --> 00:20:20.880 who sort of either innately or they this is just 363 00:20:20.920 --> 00:20:23.519 how they operate or it's something that they have built 364 00:20:23.599 --> 00:20:26.440 upon during their time at the company. And so we're 365 00:20:26.480 --> 00:20:29.359 really excited about that as well. And then people at 366 00:20:29.400 --> 00:20:31.279 the end of the year get to present their projects 367 00:20:31.359 --> 00:20:33.480 to the whole company and that type of thing. 368 00:20:34.279 --> 00:20:36.559 Now I love to hear how there's just there's simple 369 00:20:36.599 --> 00:20:39.880 ways that you can incorporate the patient voice and caregiver 370 00:20:40.039 --> 00:20:45.359 voice always to more intricate programs such as that. So 371 00:20:45.400 --> 00:20:48.160 it's interesting because I know with lunch and learns you've 372 00:20:48.160 --> 00:20:51.519 had some great experiences. Just real quickly, there was a 373 00:20:51.559 --> 00:20:54.279 story that you told us when we were chatting earlier 374 00:20:54.640 --> 00:20:57.000 about a lunch and learn where you brought at a 375 00:20:57.599 --> 00:20:58.160 patient in. 376 00:21:00.000 --> 00:21:03.559 Because I believe the gentleman and nobody had no it 377 00:21:03.599 --> 00:21:04.839 wasn't a lunch no. 378 00:21:05.279 --> 00:21:07.440 But I want to share this because it really is 379 00:21:07.680 --> 00:21:10.680 was a It was formative to me when I moved 380 00:21:10.680 --> 00:21:17.240 into rare diseases in the nineteen nineties or whatever it was. 381 00:21:17.000 --> 00:21:17.200 It was. 382 00:21:17.400 --> 00:21:20.160 It was a leap of faith and took me totally 383 00:21:20.200 --> 00:21:23.319 out of my comfort zone. I became the executive director 384 00:21:23.920 --> 00:21:27.680 of National TAYSACS and Ally Diseases Association, and I hadn't 385 00:21:27.680 --> 00:21:29.920 been in rare diseases before, and. 386 00:21:29.720 --> 00:21:30.480 We won't go there. 387 00:21:30.519 --> 00:21:33.240 But I was attending a conference for people with late 388 00:21:33.279 --> 00:21:37.480 onset tas acts, which is similar to late onset POMPEII, 389 00:21:37.519 --> 00:21:41.440 it's similar to some of the muscular dystrophees. It's a 390 00:21:42.000 --> 00:21:48.119 progressive neuromuscular disorder and very small community obviously. And we 391 00:21:48.160 --> 00:21:51.640 were in a hotel room much smaller than this in Philadelphia, 392 00:21:52.240 --> 00:21:55.200 and they were waiting for the research session to begin, 393 00:21:55.480 --> 00:21:57.799 and there was somebody who was going to be speaking 394 00:21:58.160 --> 00:22:04.480 and sharing a basic researcher but sharing his latest information 395 00:22:04.640 --> 00:22:09.559 and results of looking at upstream and downstream effects of proteins. 396 00:22:10.119 --> 00:22:13.160 And you had people lined up somewhere in wheelchairs, somewhere 397 00:22:13.200 --> 00:22:17.039 had their walkers, somebody had their service dog. Their family 398 00:22:17.119 --> 00:22:20.000 and friends were seated behind them. There were people from industry, 399 00:22:21.000 --> 00:22:26.440 other physicians. So this doctor came up, nice introduction, and 400 00:22:26.480 --> 00:22:29.559 he looks at the room and he sees the people 401 00:22:29.799 --> 00:22:32.039