WEBVTT 1 00:00:00.160 --> 00:00:03.799 Welcome to Pharma Talk Radio. This podcast is focused on 2 00:00:03.879 --> 00:00:07.200 how to better partner with patient organizations from the twenty 3 00:00:07.240 --> 00:00:10.439 twenty five Chief Medical Officer Summer three sixty. For more 4 00:00:10.439 --> 00:00:15.080 information on the CMO Summit, editorials, podcasts, or webcasts, please 5 00:00:15.199 --> 00:00:18.199 visit CMO three sixty dot org. Thank you and enjoy 6 00:00:18.280 --> 00:00:18.960 the podcast. 7 00:00:19.320 --> 00:00:24.600 So I'm Joseph Palumbo, and yeah, I want to introduce 8 00:00:24.640 --> 00:00:26.719 these folks who are sitting next to me. They aren't 9 00:00:26.800 --> 00:00:31.760 quite remarkable individuals. In getting ready for this discussion, hearing 10 00:00:31.879 --> 00:00:36.560 their stories and their perspectives has really been quite fascinating, 11 00:00:36.560 --> 00:00:39.320 and I would like for them to share that with you, 12 00:00:39.520 --> 00:00:42.119 And assuming we have some time, we'll talk a little 13 00:00:42.159 --> 00:00:45.640 bit about my experience as well. But I think theirs 14 00:00:45.679 --> 00:00:48.759 is really wonderful. So let me allow them please to 15 00:00:48.880 --> 00:00:55.240 introduce themselves in the order my left to right, and 16 00:00:55.280 --> 00:00:58.520 then after the introduction, we'll decide who goes first. 17 00:00:58.799 --> 00:01:03.520 Sure to go offhabetical I hope. I'm Marie Abramson. I'm 18 00:01:03.560 --> 00:01:08.519 head of Clinical development and medical Affairs at Precision Biosciences. 19 00:01:09.480 --> 00:01:12.159 I'm Laura Williams. I'm the Chief Medical Officer at our 20 00:01:12.239 --> 00:01:15.519 Daleks Small Biotech just outside Boston. 21 00:01:16.359 --> 00:01:19.920 And I'm Peter Schmidt, I'm the president chief Scientific officer 22 00:01:20.000 --> 00:01:20.760 from BROW. 23 00:01:21.200 --> 00:01:25.079 And Joseph PULLMBO at BIOV. So who's going first? 24 00:01:27.519 --> 00:01:28.159 That's your call? 25 00:01:28.519 --> 00:01:31.000 All right, I'm gonna go Laura first. 26 00:01:34.680 --> 00:01:36.719 Great, So where where do you want me to start? 27 00:01:36.840 --> 00:01:40.079 So talk to us? Why does this make sense? Why 28 00:01:40.120 --> 00:01:43.760 do we want to talk Well that's first of all, 29 00:01:43.760 --> 00:01:47.760 that's the wrong title. But why do we want to 30 00:01:47.799 --> 00:01:52.159 talk to patients and advocacy when we start a study, 31 00:01:52.280 --> 00:01:54.959 or even before we start a study? What makes that 32 00:01:55.040 --> 00:01:56.239 important and useful? 33 00:01:56.599 --> 00:02:00.560 Yeah? I think at the forefront really of everything we 34 00:02:00.640 --> 00:02:04.040 do is the patient, right, and so you know when 35 00:02:04.120 --> 00:02:10.039 I think about the value of the patient's voice, that 36 00:02:10.360 --> 00:02:13.599 sort of reigned supreme in terms of all of the 37 00:02:13.639 --> 00:02:17.639 things that we do. And so for me, not only 38 00:02:17.840 --> 00:02:23.479 partnering with patients, but with organizations advocacy groups that connect 39 00:02:23.479 --> 00:02:29.159 you to those patients is absolutely critical. It's interesting. In 40 00:02:29.280 --> 00:02:33.680 my last drug that we were able to get approved, 41 00:02:33.719 --> 00:02:40.159 it was for patients on maintenance dialysis to treat their hyperfocetemia, 42 00:02:41.039 --> 00:02:43.360 and it was quite a challenge, you know, we went 43 00:02:43.400 --> 00:02:49.919 through a couple of appeals processes. Crl ad come got it. 44 00:02:50.000 --> 00:02:54.039 Approved and I thought that was just great, right, good 45 00:02:54.159 --> 00:02:58.319 votes all of that, But what really was important was 46 00:02:58.479 --> 00:03:02.120 actually trying to make sure patients access to the drug. 47 00:03:02.280 --> 00:03:04.680 And you don't really get a chance to do that 48 00:03:05.080 --> 00:03:10.280 if you haven't done the right partnering with patients as 49 00:03:10.319 --> 00:03:14.599 well as advocacy groups. And so for me, I think 50 00:03:14.599 --> 00:03:18.000 that was an excellent learning. It's not just getting a 51 00:03:18.080 --> 00:03:21.439 drug approved, but it's also making sure that the folks 52 00:03:21.479 --> 00:03:23.199 that you did all. 53 00:03:23.080 --> 00:03:27.280 That work for actually have access to the drug. How 54 00:03:27.280 --> 00:03:28.680 did you engage them? 55 00:03:29.159 --> 00:03:31.400 Yeah, So, I mean I think first and foremost, you 56 00:03:31.479 --> 00:03:34.599 have to identify the groups that you're most interested in. 57 00:03:35.120 --> 00:03:37.520 You have to ask the question, how can I help? 58 00:03:38.280 --> 00:03:42.360 In other words, you know, what are the things that 59 00:03:42.400 --> 00:03:47.439 are most important to you, your patients, the patient advocacy groups, 60 00:03:48.120 --> 00:03:51.599 and then what are the things that would be helpful 61 00:03:51.879 --> 00:03:57.039 to us because it's a shared relationship, right, And once 62 00:03:57.080 --> 00:04:00.479 you identify those things, you have to bring in and 63 00:04:00.560 --> 00:04:05.840 actually be real about. You know your intent about, you 64 00:04:05.879 --> 00:04:08.639 know what it is you want want to gain. It's 65 00:04:08.719 --> 00:04:14.000 not just a one off where again you need this 66 00:04:14.000 --> 00:04:18.079 this from them, and so it's transactional. No, this is 67 00:04:18.120 --> 00:04:22.079 a real relationship. It you start building it early on, 68 00:04:23.240 --> 00:04:29.839 and you utilize it throughout every aspect of your drug 69 00:04:29.879 --> 00:04:32.079 development piece as well as commercialization. 70 00:04:32.399 --> 00:04:35.120 So there's really that invitation to participate. 71 00:04:35.319 --> 00:04:35.959 Absolutely. 72 00:04:36.839 --> 00:04:40.560 Did you put together a patient advisory Absolutely? 73 00:04:40.600 --> 00:04:44.319 I mean for both both our indications. One is an 74 00:04:44.319 --> 00:04:46.360 IBS C, the other one is, as I just said, 75 00:04:46.720 --> 00:04:53.839 in patients on dialysis. We have really strong patient advisory councils. 76 00:04:53.879 --> 00:04:57.199 And it was really obvious to me, and I think 77 00:04:57.199 --> 00:04:59.040 I shared this a couple of years ago when I 78 00:04:59.079 --> 00:05:02.240 was a year how important the patient voice was when 79 00:05:02.279 --> 00:05:06.360 we had the open public forum and one by one 80 00:05:06.920 --> 00:05:09.439 every patient you know, sort of came up in that 81 00:05:10.720 --> 00:05:16.120 our session and basically spoke to the unmet need. And 82 00:05:16.199 --> 00:05:20.639 so just looking at the clinical efficacy, the safety, obviously 83 00:05:20.680 --> 00:05:25.439 all of those things are extremely important, but hearing why 84 00:05:25.480 --> 00:05:29.040 this matters to patients, I think stole the day and I. 85 00:05:28.959 --> 00:05:32.079 Think that has to be very rewarding as a physician. 86 00:05:32.360 --> 00:05:40.800 Absolutely cool, We'll come back, Murray please. You have a 87 00:05:40.839 --> 00:05:41.800 wonderful story. 88 00:05:42.319 --> 00:05:47.240 Yeah, you know, my perspective covers kind of multiple areas 89 00:05:47.240 --> 00:05:51.279 of my own experience, having been fortunate enough to do 90 00:05:51.319 --> 00:05:54.680 a fair bit of my clinical work, but subsequent to 91 00:05:54.680 --> 00:05:58.000 that in drug development, spending a substantial time at MSD, 92 00:05:58.160 --> 00:06:02.519 MERK and infectious diseases vaccines, among others, and then moving 93 00:06:02.519 --> 00:06:06.519 to biogen where there's always an un medical need and 94 00:06:06.560 --> 00:06:13.439 there's a lot of really needing patient populations that haven't 95 00:06:13.480 --> 00:06:17.040 been tapped before. And I think that for me, there 96 00:06:17.040 --> 00:06:20.560 are multiple reasons to think about how to partner with 97 00:06:20.600 --> 00:06:25.959 a patient organization. The practical there are the ones that 98 00:06:26.000 --> 00:06:28.279 I perhaps don't have to convince a physician why it's 99 00:06:28.319 --> 00:06:32.839 important to be patient connected. I think from a practical perspective, 100 00:06:32.959 --> 00:06:35.920 I would say that it falls into multiple categories. One 101 00:06:35.959 --> 00:06:38.680 is the premise that all patients are not the same. 102 00:06:38.839 --> 00:06:42.959 It's not a monolithic group, and I think for anyone 103 00:06:43.120 --> 00:06:45.879 this means that you have to get multiple opinions to 104 00:06:46.000 --> 00:06:51.199 understand whether the feasibility of your protocol makes sense and it's separationable. 105 00:06:51.560 --> 00:06:52.879 That's one that's to be doable. 106 00:06:53.480 --> 00:06:57.720 One two is because patients aren't a monolithic group, you 107 00:06:57.839 --> 00:07:01.839 have to make sure you're addressing a diverse population, bringing 108 00:07:01.920 --> 00:07:04.920 in depending on the phase of your study, the therapeutic area, 109 00:07:05.000 --> 00:07:07.839 that you're bringing in a wide variety of patients into 110 00:07:07.879 --> 00:07:11.120 your study. This may not be fashionable at this moment 111 00:07:11.199 --> 00:07:14.199 in time, but I have believed it for a long 112 00:07:14.240 --> 00:07:15.279 time and I still. 113 00:07:15.079 --> 00:07:16.560 Do better days are ahead. 114 00:07:16.959 --> 00:07:19.920 Yeah, So I think that there are multiple ways to 115 00:07:20.040 --> 00:07:23.000 do this. I think with the limitations of working in 116 00:07:23.000 --> 00:07:25.560 a drug company, you can. It's more difficult to do 117 00:07:25.600 --> 00:07:28.879 it individually, one on one, because you do run into 118 00:07:28.879 --> 00:07:33.560 the issues of one individual representing the entire patient. So 119 00:07:33.600 --> 00:07:36.759 I think patient organizations provide you an opportunity, and there's 120 00:07:36.800 --> 00:07:40.439 a diversity of patient organizations that can do this. There's 121 00:07:40.480 --> 00:07:43.920 some that are more advocacy based, there's some that are 122 00:07:43.920 --> 00:07:46.800 more scientific based, there's some that are more political based. 123 00:07:47.040 --> 00:07:49.120 And I'll give an example to a companies which you 124 00:07:49.160 --> 00:07:53.040 I may And so from my own experience working in 125 00:07:53.079 --> 00:07:56.839 medical research operations, the goal of course is to be 126 00:07:56.879 --> 00:07:59.439 able to enroll your study and have success at the 127 00:07:59.560 --> 00:08:04.000 end with label and so clearly the feasibility component is 128 00:08:04.120 --> 00:08:07.560 very important. Connecting with the right patient group is also 129 00:08:07.720 --> 00:08:11.959 very important. Building your credibility within the industry is also very. 130 00:08:11.879 --> 00:08:14.399 And there's that wording and that credibility right invitation and 131 00:08:14.439 --> 00:08:15.279 the credibility. 132 00:08:15.480 --> 00:08:17.839 And I have to say, based on my own professional 133 00:08:17.839 --> 00:08:21.879 and personal experience, based on health conditions within my family 134 00:08:22.079 --> 00:08:26.759 based on conditions that I've observed within my community. I 135 00:08:26.800 --> 00:08:30.920 think that it's particularly important to reach out to multiple 136 00:08:30.920 --> 00:08:35.080 patient organizations for this reason because what I've observed, For example, 137 00:08:35.159 --> 00:08:37.960 I'll share personally. I think I shared on the line, Yes, 138 00:08:38.159 --> 00:08:41.600 you know, my brother passed with ALS a few years ago, 139 00:08:42.440 --> 00:08:45.039 and I happen to be working at a company that's 140 00:08:45.120 --> 00:08:48.360 pioneering within the ALS space, and it was at this time, 141 00:08:49.320 --> 00:08:52.759 and what I recognized was several things. One, the people 142 00:08:52.759 --> 00:08:57.519 who are being diagnosed with the disease aren't well connected necessarily. 143 00:08:57.879 --> 00:09:02.440 There's a lot of disparity and disconnection within the medical 144 00:09:02.679 --> 00:09:06.320 community that prevents patients from getting access. That's correct, And 145 00:09:06.399 --> 00:09:08.440 so I think as a company, you have to be 146 00:09:08.519 --> 00:09:12.559 creative and thoughtful depending on your therapeutic area, depending on 147 00:09:12.600 --> 00:09:16.080 your disease, depending on the unmet medical need, depending on 148 00:09:16.120 --> 00:09:20.159 the moral urgency of the situation. In the case of ALS, 149 00:09:20.399 --> 00:09:24.360 reaching people earlier, reaching a diverse group, if you're looking 150 00:09:24.360 --> 00:09:29.120 for precision medicine and you're looking specifically for a particular genotype. 151 00:09:28.679 --> 00:09:30.600 All the more reasons you did successfully. 152 00:09:31.000 --> 00:09:33.399 And then when you talk about different patient groups, there 153 00:09:33.399 --> 00:09:36.360 are multiple different ways to do that, and I think 154 00:09:36.440 --> 00:09:39.639 different patients relate and connect to it in different ways. 155 00:09:39.840 --> 00:09:42.600 And again I think it requires a diversity of outreach 156 00:09:43.039 --> 00:09:44.039 to make that possible. 157 00:09:44.159 --> 00:09:47.600 Yeah. Absolutely, and congratulations on having done that the right way. 158 00:09:47.679 --> 00:09:50.039 I don't know if I did make it the right way, 159 00:09:50.080 --> 00:09:52.720 but by tried my best, I mean in the case 160 00:09:52.759 --> 00:09:55.320 of our trials, we were able to enroll them. I 161 00:09:55.360 --> 00:09:59.799 always feel like I've failed because ultimately we haven't solved 162 00:09:59.840 --> 00:10:04.320 the problem yet. Yeah, it's an iterative process, but I 163 00:10:04.360 --> 00:10:07.279 think for when you interact with the patient, at least 164 00:10:07.279 --> 00:10:10.440 for me, I derive energy from that and you realize 165 00:10:10.559 --> 00:10:12.879 it orients you to do the right thing and to 166 00:10:13.000 --> 00:10:16.840 think about a particular problem in a more well, should 167 00:10:16.879 --> 00:10:20.480 I say, a higher level of thinking than just operationally 168 00:10:20.639 --> 00:10:21.879 from point A to point B. 169 00:10:22.039 --> 00:10:25.879 Yeah. Absolutely, it goes both ways. It is energizing to 170 00:10:25.919 --> 00:10:30.919 be able to be a physician. So next to me 171 00:10:31.559 --> 00:10:33.519 is Peter Schmidt, who happens to be one of my 172 00:10:33.639 --> 00:10:39.679 good friends, and he came up here anyway. He has 173 00:10:39.720 --> 00:10:43.000 a very subtle sense of humor. And you guys can't 174 00:10:43.039 --> 00:10:48.200 see this, but he has mermaids on his tie and 175 00:10:48.279 --> 00:10:50.679 he told me he was going for a nautical theme, 176 00:10:51.080 --> 00:10:55.200 and so his braces have pirates, So thank you for that. 177 00:10:58.120 --> 00:11:01.879 So you've been working in a number of fields in 178 00:11:01.960 --> 00:11:05.240 neuroscience for quite a number of years, both as an academic, 179 00:11:06.000 --> 00:11:10.320 a leader in advocacy of course, and as a developer. 180 00:11:10.759 --> 00:11:15.639 So your perspective can, in some ways, I don't know, 181 00:11:15.759 --> 00:11:18.960 sort of go over the top of everything else we've 182 00:11:19.000 --> 00:11:20.600 been talking about here. You've seen it in a lot 183 00:11:20.639 --> 00:11:25.279 of different directions. So comments, what would you what would 184 00:11:25.320 --> 00:11:26.159 you like to tell us? 185 00:11:26.559 --> 00:11:30.679 So I spent about a decade working leading programs for 186 00:11:31.240 --> 00:11:38.279 Parkinson's Disease Advocacy Group, and one of the challenges that 187 00:11:38.360 --> 00:11:41.519 you all will face working with advocacy is that for 188 00:11:41.559 --> 00:11:46.720 the most of these organizations, their exposure to pharma is 189 00:11:46.759 --> 00:11:51.039 through the commercial division. Those are the people who often 190 00:11:51.120 --> 00:11:54.080 go out and first make contact, and they have a 191 00:11:54.200 --> 00:11:57.879 very different set of rules and a very different way 192 00:11:57.919 --> 00:12:02.639 of engaging. And I always tell I'm currently the chairman 193 00:12:02.759 --> 00:12:06.519 of the Davis Finney Foundation for Parkinson's, and I tell 194 00:12:06.559 --> 00:12:11.600 them there is no better engagement than a development stage company. 195 00:12:11.840 --> 00:12:17.919 It's hope, it's opportunity, it's a way that people The 196 00:12:18.519 --> 00:12:23.600 most valuable donation a patient can make is their participation 197 00:12:23.919 --> 00:12:27.799 in a clinical trial. Absolutely that is the number one value. 198 00:12:28.240 --> 00:12:32.120 But many of these advacy groups don't understand that, and 199 00:12:32.200 --> 00:12:37.039 so it's important to go out early and make contact. 200 00:12:37.720 --> 00:12:40.519 You don't have to go out with your checkbook, but 201 00:12:40.759 --> 00:12:44.080 just go out and have someone from your team or 202 00:12:44.519 --> 00:12:47.679 or someone from your office go out and make contact 203 00:12:47.720 --> 00:12:51.600 and talk to them about make sure they understand that 204 00:12:51.720 --> 00:12:55.519 you can that during your clinical development you cannot be 205 00:12:55.639 --> 00:13:01.000 promotional about your molecule. That you can't. They're afraid that 206 00:13:01.080 --> 00:13:06.159 they're going to be railroaded into something, but that's not lawful, 207 00:13:06.759 --> 00:13:08.679 and they don't understand that. So you need to help 208 00:13:08.720 --> 00:13:15.960 them to understand how clinical research represents the most, you know, 209 00:13:16.240 --> 00:13:20.399 the best possible way that an patient advoacy group can 210 00:13:20.480 --> 00:13:26.039 work with industry. And I truly believe that, and that 211 00:13:26.080 --> 00:13:28.600 I tell that to the groups that I advise. The 212 00:13:29.120 --> 00:13:31.600 other thing that you need to watch out for is that, 213 00:13:32.840 --> 00:13:36.879 like in any space, and the politics were discussed this morning, 214 00:13:36.919 --> 00:13:40.279 so I'm not discussing politics. There is a Dunning Krueger 215 00:13:40.320 --> 00:13:44.720 effect that you will see in patient advocacy, just as 216 00:13:44.759 --> 00:13:47.919 you see in so many domains. The people who come 217 00:13:47.960 --> 00:13:50.200 to you with the patients who come to you with 218 00:13:50.240 --> 00:13:53.639 the greatest confidence are the ones who are going to 219 00:13:53.639 --> 00:13:58.320 have preset ideas about which scientific avenues are going to 220 00:13:58.320 --> 00:14:01.039 be productive and which one they don't want to get 221 00:14:01.080 --> 00:14:06.440 engaged in the staff of Patient advocacy groups can help 222 00:14:06.519 --> 00:14:09.879 you get past that and find the people who hesitate. 223 00:14:10.279 --> 00:14:11.000 I used to be. 224 00:14:11.120 --> 00:14:15.360 On a panel for the Department of Defense funding Parkinson's research, 225 00:14:16.000 --> 00:14:18.879 and I got a friend of mine with a patient 226 00:14:18.919 --> 00:14:22.279 with Parkinson's. He got him appointed to be the patient representative, 227 00:14:22.720 --> 00:14:27.639 and every year he would say, I'm not smart enough. 228 00:14:27.679 --> 00:14:30.000 I don't think I should go back, and I would say, well, 229 00:14:30.039 --> 00:14:32.480 that's why you need to keep staying on this panel, 230 00:14:32.519 --> 00:14:34.679 and they kept him on that panel longer than any 231 00:14:34.759 --> 00:14:39.399 other participant because his insight and input was so valuable. 232 00:14:39.440 --> 00:14:42.440 He has finally said he can't do it anymore. It's 233 00:14:42.519 --> 00:14:44.960 not his diseases progress and he can't travel. But he, 234 00:14:45.600 --> 00:14:51.039 for I think ten years, was a productive represented the 235 00:14:51.080 --> 00:14:55.519 patient voice, and his value came from the fact that 236 00:14:55.759 --> 00:14:59.720 he knew that he was not the embodiment of Parkinson's. 237 00:15:00.080 --> 00:15:05.360 He represented a community and so working. It's great to 238 00:15:05.440 --> 00:15:08.679 engage with these groups and work with the staff to 239 00:15:08.919 --> 00:15:12.440 identify the people who are going to have an open mind, 240 00:15:12.440 --> 00:15:14.600 who are going to listen. One of the things that 241 00:15:14.639 --> 00:15:19.320 we've seen is that, you know, working in neurology, there 242 00:15:19.320 --> 00:15:23.000 are so many new approaches than there used to be. 243 00:15:23.399 --> 00:15:26.679 When I started working in Parkinson's disease, people thought there 244 00:15:26.679 --> 00:15:29.600 were only really two promising avenues, one of which was 245 00:15:29.679 --> 00:15:33.759 going to cure the disease stem cells and gene therapy 246 00:15:34.720 --> 00:15:40.679 dope minergic gene therapy, and those are not really major folks. 247 00:15:40.679 --> 00:15:44.799 I Now in clinical development, there are a ton of 248 00:15:44.879 --> 00:15:49.159 different molecular pathways that we're focusing on, and the Parkinson's 249 00:15:49.240 --> 00:15:52.799 community does not understand them, and so it's crucial that 250 00:15:53.919 --> 00:15:59.200 well doing outreach, that we bring scientific training. We teach 251 00:15:59.240 --> 00:16:02.480 people what are what's going to be productive, what's going 252 00:16:02.519 --> 00:16:06.279 to why we think that this avenue might be effective. 253 00:16:06.639 --> 00:16:09.519 And you can always get an independent SCIENTI as somebody 254 00:16:09.559 --> 00:16:12.039 who's not on your team, but at a university near 255 00:16:12.080 --> 00:16:16.039 where the headquarters is there always you can always find 256 00:16:16.080 --> 00:16:19.919 somebody who has the understanding and the independence to be 257 00:16:19.960 --> 00:16:23.519 able to talk about it honestly with that group. And 258 00:16:23.759 --> 00:16:27.279 that's what you'll need is just an honest discussion, Yes, 259 00:16:27.399 --> 00:16:32.000 this is something that's interesting, Yes it needs human trials 260 00:16:32.039 --> 00:16:35.000 for us to truly understand if this is the breakthrough 261 00:16:35.120 --> 00:16:36.720 that we hope it will be. 262 00:16:37.360 --> 00:16:44.000 And again that's honesty credibility one more time. So before 263 00:16:44.039 --> 00:16:48.000 I say anything, you've all had an opportunity to talk 264 00:16:48.039 --> 00:16:51.480 to each other. He would you ask each other any 265 00:16:51.559 --> 00:16:56.799 questions based on what you've just heard? 266 00:17:00.279 --> 00:17:04.440 Have lots of questions, you know, I guess from my 267 00:17:04.559 --> 00:17:08.720 vantage point. You know, patient organizations are as different as 268 00:17:08.759 --> 00:17:13.279 the patients themselves, and so I guess from a Parkinson's perspective, 269 00:17:13.880 --> 00:17:16.640 are there and you don't have to name specific ones, 270 00:17:16.680 --> 00:17:19.440 but what I've observed in the ALF space and I 271 00:17:19.480 --> 00:17:23.680 know that Parkinson's space a bit coming from biogen, are 272 00:17:23.680 --> 00:17:28.400 there organizations that are more focused on advocacy or patient 273 00:17:28.480 --> 00:17:38.920 engagement that probably men amend themselves more to pharmaceutical development, education, 274 00:17:39.440 --> 00:17:41.839 patient awareness feasibility than others. 275 00:17:42.799 --> 00:17:46.039 So I think in every domain, so that we used 276 00:17:46.079 --> 00:17:46.640 to say. 277 00:17:46.519 --> 00:17:47.119 What are they? 278 00:17:47.160 --> 00:17:50.119 Actually that was the real question is you know, could 279 00:17:50.160 --> 00:17:52.039 you share with me some of those thoughts? 280 00:17:52.680 --> 00:17:54.720 So in every it used to be that in every 281 00:17:54.759 --> 00:17:57.960 disease you had one organization that was focused on care 282 00:17:58.119 --> 00:18:02.480 and one organization that's focused on search. But they will 283 00:18:02.519 --> 00:18:06.000 probably both be good targets. It depends on them, how 284 00:18:06.319 --> 00:18:10.359 on the personalities the people you talk to, It depends 285 00:18:10.400 --> 00:18:17.200 on the on You can't know in advance whether or 286 00:18:17.240 --> 00:18:21.119 not an organization has funded something, and sometimes if you 287 00:18:21.160 --> 00:18:24.240 look at their websites, you can see they're really pushing 288 00:18:24.319 --> 00:18:27.319 one concept and that's not what I'm doing, and that's 289 00:18:27.359 --> 00:18:31.519 probably not the right group to reach out to. Sometimes 290 00:18:31.559 --> 00:18:38.599 they they are aligned, and they will be. Sometimes their 291 00:18:38.720 --> 00:18:43.240 research initiative aligns with your therapeutic approach, and then that's 292 00:18:43.279 --> 00:18:46.279 a great probably a great organization, unless they decide that 293 00:18:46.319 --> 00:18:49.160 there's the subtlety of the difference in what they're investing 294 00:18:49.200 --> 00:18:52.640 in is different. There are a lot of organizations who 295 00:18:52.799 --> 00:18:55.799 have taken a queue from the Cystic Fibrosis Foundation and 296 00:18:55.839 --> 00:19:01.119 think they're going to make billions by investing in a drug. 297 00:19:02.240 --> 00:19:09.359 And I don't know how firewall that is from programs 298 00:19:09.400 --> 00:19:13.559 at different places, but you may find that their challenge 299 00:19:13.559 --> 00:19:15.200 is there if they're invested in something. 300 00:19:15.400 --> 00:19:18.400 Yeah, yeah, I observed it. I observe that too. There's 301 00:19:18.440 --> 00:19:22.680 some that are profit motivated, some are, you know, and 302 00:19:22.720 --> 00:19:25.279 it also depends on the disease, how organized they are, 303 00:19:25.319 --> 00:19:28.839 at what stage they are. I've interacted with some spaces, 304 00:19:28.880 --> 00:19:32.240 let's say in mitochondrial disease, which is still very nascent 305 00:19:32.400 --> 00:19:35.799 because there's not adequate therapy in those spaces, And they're 306 00:19:35.960 --> 00:19:40.200 very different perspective of organization and structure than maybe in 307 00:19:40.240 --> 00:19:45.000 Alzheimer's or dementia program, which is vastly different in size, 308 00:19:45.000 --> 00:19:48.880 but also in focus and professional experience. 309 00:19:49.680 --> 00:19:55.359 Groups that engage parents of children with childhood diseases can 310 00:19:55.920 --> 00:20:00.680 are often really great to work with. They want any benefit, 311 00:20:00.799 --> 00:20:04.720 anything is, they're interested in anything. They don't just want 312 00:20:05.519 --> 00:20:08.799 Holy Grail cure. They want anything that can make their 313 00:20:08.880 --> 00:20:09.519 child better, and. 314 00:20:09.440 --> 00:20:11.480 Those good points to work. It's a really good point. 315 00:20:12.200 --> 00:20:14.759 Any Yeah, I mean I think it depends on the disease. 316 00:20:14.799 --> 00:20:18.400 I mean when I look at again and patients in 317 00:20:18.759 --> 00:20:22.000 the renal space, and we're talking about chronic kidney disease 318 00:20:22.079 --> 00:20:25.799 more specifically kidney failure. These are folks with chronic diseases. 319 00:20:26.200 --> 00:20:28.519 And when you think about some of the advocacy groups 320 00:20:28.519 --> 00:20:31.920 that come to my you know, National Kidney Foundation, American 321 00:20:32.000 --> 00:20:38.640 Kidney Fund, their focus is multi factorial, right, It's it's 322 00:20:38.720 --> 00:20:43.279 it's disease awareness. I mean, specifically with something like chronic 323 00:20:43.359 --> 00:20:46.119 kidney disease. We're up to ninety percent of folks don't 324 00:20:46.119 --> 00:20:50.240 even know they have it, right, and so disease awareness education, 325 00:20:51.680 --> 00:20:56.279 you know, championing you know, more transplants as opposed to 326 00:20:56.880 --> 00:21:01.640 maintenance dialysis, actually providing financial so to some of those 327 00:21:01.680 --> 00:21:07.319 patients who actually don't despite you know, you know, sort 328 00:21:07.359 --> 00:21:11.640 of our antiquated payment system for those patients they don't 329 00:21:11.680 --> 00:21:16.640 have enough to cover just you know, simple things. And 330 00:21:16.680 --> 00:21:20.519 so I think it depends on the disease. It depends 331 00:21:20.519 --> 00:21:25.480 on the organization. But they've been great to work with, 332 00:21:25.720 --> 00:21:28.079 at least in the renal space. When I look at 333 00:21:28.119 --> 00:21:33.759 the IBS space, not as many advocacy groups, but there 334 00:21:33.799 --> 00:21:34.680 needs to be more. 335 00:21:35.000 --> 00:21:38.799 So absolutely thank you for that. I want to leave 336 00:21:38.799 --> 00:21:42.559 about five minutes for question and answering, so I'll take 337 00:21:42.559 --> 00:21:44.920 a couple of minutes and sort of talk about my experience. 338 00:21:45.000 --> 00:21:50.960 I've been mostly working in the rare disease space, orphan 339 00:21:51.039 --> 00:21:54.119 disease since I came to Farmer from academia in the 340 00:21:54.200 --> 00:22:01.960 nineteen nineties. It's wonderful what the belong to a community. 341 00:22:02.000 --> 00:22:07.480 It's engaging in this way reminds us or reminds me 342 00:22:08.720 --> 00:22:15.519 of the fiduciary relationship we have as physicians. It's very 343 00:22:15.559 --> 00:22:19.279 easy in farm it's very easy in academia to put 344 00:22:19.279 --> 00:22:21.880 on your scientific hat, and sometimes informer to put on 345 00:22:21.920 --> 00:22:27.000 your commercial hat and think of, well, I'm doing this 346 00:22:27.119 --> 00:22:31.799 because X. But in speaking to families and speaking with 347 00:22:31.920 --> 00:22:35.240 advocacy and talking to patients and people who don't know 348 00:22:35.279 --> 00:22:40.200 their patients yet about what they want and what's meaningful, 349 00:22:41.240 --> 00:22:45.000 it educates you. It makes you a better physician, it 350 00:22:45.000 --> 00:22:47.880 makes you a better drug developer. It helps you to 351 00:22:48.000 --> 00:22:53.079 really hear the voice of the patient. There have been 352 00:22:53.119 --> 00:22:58.200 times where I brought things forward to global health authorities 353 00:22:58.440 --> 00:23:03.960 and not been sick successful and having the community there 354 00:23:04.160 --> 00:23:07.240 knowing that I had engaged with them and we were 355 00:23:07.319 --> 00:23:11.400 working together really helped to soothe a lot of that 356 00:23:11.559 --> 00:23:15.319 hurt when you know we didn't get to hear what 357 00:23:15.359 --> 00:23:20.400 we wanted to hear. I've taken that into my career 358 00:23:20.680 --> 00:23:24.279 and it's been one of the most fulfilling things to 359 00:23:24.319 --> 00:23:28.119 be able to reflect what the community wants. And I 360 00:23:28.119 --> 00:23:32.839 would say it's not just working with advocacy. I had 361 00:23:32.920 --> 00:23:36.839 the privilege of working in translational research in Japan for 362 00:23:36.880 --> 00:23:39.839 a number of years and one of the ways that 363 00:23:39.880 --> 00:23:42.599 we were able to advance molecules was to go into 364 00:23:42.640 --> 00:23:48.240 the laboratory and talk to the individual scientists about the 365 00:23:48.279 --> 00:23:52.720 cares they have in their families, the diseases, the conditions 366 00:23:52.759 --> 00:23:57.759 that are still unmet, because for many preclinical scientists, they 367 00:23:57.920 --> 00:24:02.559 read the literature, but they kind I do forget or 368 00:24:02.720 --> 00:24:07.359 unaware that they're there to produce medicines and to return 369 00:24:07.440 --> 00:24:10.240 it to them and say, Okay, here's an opportunity to 370 00:24:10.319 --> 00:24:13.799 do something for someone you care about. Are you interested 371 00:24:13.839 --> 00:24:16.759 in making that molel you are interested in developing that program. 372 00:24:18.880 --> 00:24:24.240 It's been really a privilege to work with these communities. 373 00:24:25.119 --> 00:24:29.160 So we have about five minutes left, and I'm hopeful 374 00:24:29.200 --> 00:24:31.759 that those of you who've worked in this field or 375 00:24:32.000 --> 00:24:36.000 or have thoughts will share those thoughts and experiences, because 376 00:24:36.000 --> 00:24:40.039 it's not just about bringing the advocacy group to the 377 00:24:40.119 --> 00:24:44.759 advisory panel. At the end of the day, it's about 378 00:24:44.839 --> 00:24:50.000 creating medicines. So any thoughts, Yeah, I had a question 379 00:24:50.200 --> 00:24:50.359 for you. 380 00:24:51.799 --> 00:24:55.680 So many of us here in small biotech companies, so 381 00:24:55.799 --> 00:24:58.960 we don't have a big organization. We don't have anybody 382 00:24:59.079 --> 00:25:04.720 in patient advocacy necessarily at our companies. And if you're 383 00:25:04.759 --> 00:25:07.039 going into a rare disease where maybe there isn't a 384 00:25:07.119 --> 00:25:12.279 well defined patient organization or advocacy group. Do you have 385 00:25:12.359 --> 00:25:15.440 any suggestions of how we get to the patients, how 386 00:25:15.480 --> 00:25:16.960 we get the voice of the patients. 387 00:25:17.480 --> 00:25:19.599 So let me first start by saying, we have about 388 00:25:19.599 --> 00:25:22.440 ten people in my company. Small company. It can be 389 00:25:22.519 --> 00:25:25.119 done with. Let me these folks provide you with some 390 00:25:25.240 --> 00:25:25.880 advice please. 391 00:25:26.400 --> 00:25:28.880 I was whispering, but I guess not in my microphone. 392 00:25:30.039 --> 00:25:32.240 I was just saying, at my current company, it's a 393 00:25:32.359 --> 00:25:35.200 very small company, right, it's a gene editing company, and 394 00:25:35.200 --> 00:25:38.759 they're working in various areas. One of the areas happens 395 00:25:38.759 --> 00:25:42.200 to be mitochondrial disease. And so that is an example 396 00:25:42.240 --> 00:25:46.519 that you're talking about. It's not well organized, but if 397 00:25:46.559 --> 00:25:48.119 you compare to big pharma and all of that, that 398 00:25:48.200 --> 00:25:51.079 actually I get the opportunity to dip into it and 399 00:25:51.160 --> 00:25:54.000 get to meet and interact, and it's a very rewarding 400 00:25:54.160 --> 00:25:58.799 thing because they actually don't come to you with preconceived 401 00:25:58.839 --> 00:26:02.279