1 00:00:00,160 --> 00:00:02,400 Speaker 1: I got the little vial out and it's just like 2 00:00:02,560 --> 00:00:08,719 Speaker 1: five milliliters of liquid gold. You know, it wasn't in 3 00:00:08,760 --> 00:00:11,640 Speaker 1: the bottle because it was inside of our son. But 4 00:00:11,760 --> 00:00:15,640 Speaker 1: I was just looking at that little bottle and teary eyed, 5 00:00:15,880 --> 00:00:19,360 Speaker 1: you know, like, oh my goodness, it's so small, and 6 00:00:19,440 --> 00:00:23,640 Speaker 1: yet it's been so big in our lives. And how 7 00:00:23,680 --> 00:00:26,440 Speaker 1: do you put a price on that? I don't know. 8 00:00:27,720 --> 00:00:31,520 Speaker 2: This is Cheryl Yoda. The liquid gold she's referring to 9 00:00:31,840 --> 00:00:35,040 Speaker 2: is a medication called nusan Erson that saved the life 10 00:00:35,040 --> 00:00:36,640 Speaker 2: of her eight year old son, Jace. 11 00:00:37,560 --> 00:00:40,560 Speaker 1: His big thing right now is hunting. We've literally gotten 12 00:00:40,680 --> 00:00:43,280 Speaker 1: up and he's been out for like an hour stalking 13 00:00:43,320 --> 00:00:45,480 Speaker 1: around the backyard with his bow and arrow. 14 00:00:45,920 --> 00:00:48,480 Speaker 2: He's practicing for when he's old enough to go deer 15 00:00:48,560 --> 00:00:51,720 Speaker 2: hunting with his dad and big brothers. But back when 16 00:00:51,800 --> 00:00:54,800 Speaker 2: Jace was a baby, Cheryl didn't think anything like that 17 00:00:54,840 --> 00:00:58,160 Speaker 2: would ever be possible for him. When Jace was eight 18 00:00:58,240 --> 00:01:02,240 Speaker 2: days old, he was diagnosed with rare genetic disease called SMA, 19 00:01:02,960 --> 00:01:07,760 Speaker 2: or spinal muscular atrophy. SMA interferes with the brain's ability 20 00:01:07,800 --> 00:01:11,840 Speaker 2: to communicate with muscles over time. People with the most 21 00:01:11,880 --> 00:01:15,800 Speaker 2: severe form of the disease like Jace lose the ability 22 00:01:15,880 --> 00:01:20,120 Speaker 2: to swallow and breathe. Doctors told Cheryl it was unlikely 23 00:01:20,240 --> 00:01:25,240 Speaker 2: Jace would live to see his second birthday. Sadly, Cheryl 24 00:01:25,280 --> 00:01:29,520 Speaker 2: was already familiar with SMA before Jace. She'd given birth 25 00:01:29,560 --> 00:01:33,320 Speaker 2: to two boys who didn't have SMA and a daughter, Ariel, 26 00:01:33,520 --> 00:01:37,160 Speaker 2: who did. Ariel died when she was fifteen months old, 27 00:01:37,920 --> 00:01:42,720 Speaker 2: and after learning about Jace's diagnosis, she thought he would too. 28 00:01:42,880 --> 00:01:46,920 Speaker 1: It just felt like he had received basically a death sentence, 29 00:01:46,920 --> 00:01:50,120 Speaker 1: and we were going to kind of enjoy him for 30 00:01:50,200 --> 00:01:52,360 Speaker 1: a year and a half to two years and then 31 00:01:53,320 --> 00:01:55,760 Speaker 1: try to help him be as happy and comfortable and 32 00:01:55,840 --> 00:01:58,240 Speaker 1: have a great life as he could, and then have 33 00:01:58,320 --> 00:01:59,320 Speaker 1: to say goodbye to him. 34 00:02:00,400 --> 00:02:03,600 Speaker 2: Jace's doctor gave Cheryl a referral to see a pediatric 35 00:02:03,640 --> 00:02:08,440 Speaker 2: neurologist at Johns Hopkins University School of Medicine. At the appointment, 36 00:02:08,639 --> 00:02:12,000 Speaker 2: Cheryl says, the doctor gave Jason an exam and then 37 00:02:12,040 --> 00:02:14,359 Speaker 2: asked her and her husband how soon they could get 38 00:02:14,360 --> 00:02:19,000 Speaker 2: on an aeroplane. The doctor explained there was a clinical 39 00:02:19,040 --> 00:02:21,600 Speaker 2: trial getting started for a drug that might be able 40 00:02:21,680 --> 00:02:25,520 Speaker 2: to stop the symptoms of SMA from ever starting, and 41 00:02:25,760 --> 00:02:29,240 Speaker 2: he said Jace would be a perfect candidate since he 42 00:02:29,360 --> 00:02:34,320 Speaker 2: was still presymptomatic, but there were only twenty five spots open. 43 00:02:34,760 --> 00:02:36,920 Speaker 2: They'd need to act fast and get to one of 44 00:02:36,960 --> 00:02:40,280 Speaker 2: the research sites in a matter of days. Cheryl and 45 00:02:40,320 --> 00:02:43,400 Speaker 2: her husband left the appointment completely shocked. 46 00:02:44,160 --> 00:02:47,320 Speaker 1: Just feeling like we had received a gift a lake 47 00:02:48,000 --> 00:02:52,560 Speaker 1: instead of walking out there with with options alike. You know, 48 00:02:52,760 --> 00:02:54,560 Speaker 1: when do you want Haspers to come out. 49 00:02:54,440 --> 00:02:55,040 Speaker 3: And visit you? 50 00:02:55,280 --> 00:02:58,360 Speaker 1: That kind of thing we were talking about him living. 51 00:03:01,440 --> 00:03:04,519 Speaker 2: They flew from Baltimore to Chicago later that week, where 52 00:03:04,600 --> 00:03:08,919 Speaker 2: Jace was screened for the clinical trial and selected. Jace 53 00:03:09,040 --> 00:03:12,160 Speaker 2: was given four doses of new Sinerson over the course 54 00:03:12,200 --> 00:03:15,560 Speaker 2: of a few weeks. Cheryl learned that he'd need another 55 00:03:15,639 --> 00:03:19,679 Speaker 2: dose every four months, probably for the rest of his life. 56 00:03:19,880 --> 00:03:22,040 Speaker 2: By the time Jace was one and a half in 57 00:03:22,120 --> 00:03:26,799 Speaker 2: December twenty sixteen, he still wasn't showing any SMA symptoms. 58 00:03:27,440 --> 00:03:32,520 Speaker 2: Cheryl was overjoyed. Then, two days before Christmas, the rest 59 00:03:32,560 --> 00:03:36,640 Speaker 2: of the SMA community rejoiced too. New Sinerson was approved 60 00:03:36,680 --> 00:03:39,920 Speaker 2: by the FDA. That meant the drug treatment would become 61 00:03:39,960 --> 00:03:43,440 Speaker 2: available to more people, even if they weren't enrolled in 62 00:03:43,480 --> 00:03:51,360 Speaker 2: a clinical trial, But one week later, the joy turned 63 00:03:51,400 --> 00:03:56,120 Speaker 2: to outrage. Biogen, the drug company that manufactures the drug, 64 00:03:56,440 --> 00:04:00,400 Speaker 2: issued an announcement of its own. The drugs trade name 65 00:04:00,440 --> 00:04:03,920 Speaker 2: would be spin Raza, they said, and it would cost 66 00:04:04,160 --> 00:04:07,720 Speaker 2: one hundred and twenty five thousand dollars per dose. 67 00:04:08,640 --> 00:04:12,080 Speaker 1: I mean, now I pay anything, But then how can 68 00:04:12,120 --> 00:04:16,040 Speaker 1: I pay anything? Like, how do I pay one hundred 69 00:04:16,040 --> 00:04:19,440 Speaker 1: and twenty five thousand dollars a dose? Just get that 70 00:04:19,560 --> 00:04:21,920 Speaker 1: out three times a year? That's impossible. 71 00:04:22,400 --> 00:04:26,880 Speaker 2: At the time, Cheryl considered herself fortunate. Jase's costs was 72 00:04:26,920 --> 00:04:30,480 Speaker 2: still being covered by the clinical trial, and the clinical 73 00:04:30,560 --> 00:04:34,920 Speaker 2: trial kept getting extended and extended, but eventually it came 74 00:04:34,960 --> 00:04:38,000 Speaker 2: to an end. Earlier this year when Jace turned. 75 00:04:37,839 --> 00:04:41,880 Speaker 1: Eight, and I did have those thoughts like, oh my goodness, 76 00:04:42,120 --> 00:04:45,080 Speaker 1: now what Like I need to get on the ball 77 00:04:45,160 --> 00:04:48,839 Speaker 1: and start researching. Maybe there are other studies we could 78 00:04:48,880 --> 00:04:51,360 Speaker 1: be in. I need to talk to our insurance and 79 00:04:51,400 --> 00:04:54,120 Speaker 1: find out if they'll cover this drug and that kind 80 00:04:54,160 --> 00:04:59,360 Speaker 1: of thing. And I just started feeling overwhelmed, like I'm 81 00:04:59,360 --> 00:05:03,359 Speaker 1: not that sad, I'm not that organized. How am I 82 00:05:03,520 --> 00:05:04,599 Speaker 1: ever going to do all of this? 83 00:05:07,800 --> 00:05:11,359 Speaker 2: I'm Laurena Rora Hutchinson. I'm the director of the Ideas 84 00:05:11,440 --> 00:05:16,440 Speaker 2: Lab at the Johns Hopkins Berman Institute of Bioethics. In 85 00:05:16,480 --> 00:05:21,000 Speaker 2: today's episode, Miracle Drugs, Modern medicine has been on the 86 00:05:21,040 --> 00:05:25,200 Speaker 2: cusp of a revolution for years. There's a growing list 87 00:05:25,240 --> 00:05:28,080 Speaker 2: of drugs that can save lives and cure diseases like 88 00:05:28,279 --> 00:05:33,119 Speaker 2: never before. But the price for these medications can reach 89 00:05:33,240 --> 00:05:37,880 Speaker 2: six and even seven figures, raising high stakes ethical questions 90 00:05:37,920 --> 00:05:43,200 Speaker 2: about access and regulation from Pushkin Industries and the Johns 91 00:05:43,200 --> 00:05:54,039 Speaker 2: Hopkins Berman Institute of Bioethics. This is playing God. Jays 92 00:05:54,080 --> 00:05:57,640 Speaker 2: got his first commercial dose of Spinraza this year, and 93 00:05:57,760 --> 00:06:01,520 Speaker 2: Cheryl's insurance company covered the cost, but that's not how 94 00:06:01,560 --> 00:06:04,400 Speaker 2: it works for all families. It can be a real 95 00:06:04,480 --> 00:06:08,400 Speaker 2: issue getting insurance companies to cover high cost drugs like spinraza. 96 00:06:09,240 --> 00:06:12,560 Speaker 2: Even when they do, there's no guarantee coverage will last. 97 00:06:13,240 --> 00:06:16,719 Speaker 4: The best day of my career was December twenty third, 98 00:06:16,760 --> 00:06:20,200 Speaker 4: twenty sixteen, when the drug was licensed by the FDA. 99 00:06:21,320 --> 00:06:23,800 Speaker 4: The worst day of my career was December twenty seventh, 100 00:06:23,839 --> 00:06:26,440 Speaker 4: twenty sixteen, when the price was announced. 101 00:06:27,160 --> 00:06:31,200 Speaker 2: Tom Crawford is a pediatric neurologist at Johns Hopkins University 102 00:06:31,279 --> 00:06:34,960 Speaker 2: School of Medicine. He's been caring for children with spinal 103 00:06:35,080 --> 00:06:39,120 Speaker 2: muscular atrophy for over thirty years, and he's very familiar 104 00:06:39,160 --> 00:06:42,839 Speaker 2: with the challenges his patient's face. It was Tom who 105 00:06:42,839 --> 00:06:45,680 Speaker 2: told Cheryls she should try to get jase enrolled in 106 00:06:45,680 --> 00:06:46,159 Speaker 2: the trial. 107 00:06:46,800 --> 00:06:49,560 Speaker 4: I couldn't you know. I was over the top. Oh 108 00:06:49,560 --> 00:06:51,360 Speaker 4: my god, we have this therapy. It's going to work. 109 00:06:51,360 --> 00:06:53,720 Speaker 4: And then four days later, one hundred and twenty five 110 00:06:53,760 --> 00:06:56,760 Speaker 4: thousand dollars a dose, six doses for the first year 111 00:06:56,760 --> 00:06:59,520 Speaker 4: of life, three doses every year thereafter. Oh my god, 112 00:06:59,680 --> 00:07:01,600 Speaker 4: none of our patients are ever ever going to be 113 00:07:01,640 --> 00:07:03,719 Speaker 4: able to pay for it. It's been snatched away from 114 00:07:03,720 --> 00:07:07,160 Speaker 4: me in all my patients. Then I learned that the 115 00:07:07,240 --> 00:07:09,800 Speaker 4: drug companies were really, really smart. They realized that that 116 00:07:09,960 --> 00:07:13,320 Speaker 4: no insurance company is going to let a baby die 117 00:07:13,480 --> 00:07:15,360 Speaker 4: for one hundred and twenty five thousand dollars, and they 118 00:07:15,400 --> 00:07:21,480 Speaker 4: would approve the authorization. And so what the new barrier 119 00:07:22,160 --> 00:07:25,520 Speaker 4: is now not just the price, but the authorization's getting 120 00:07:26,160 --> 00:07:29,760 Speaker 4: the insurance companies to realize that this child is indeed 121 00:07:29,840 --> 00:07:33,720 Speaker 4: eligible and falls into the criteria that were used to 122 00:07:34,080 --> 00:07:37,040 Speaker 4: prove that it works. But now there's a whole game 123 00:07:37,080 --> 00:07:41,760 Speaker 4: going on because insurance companies will try to not authorize 124 00:07:41,760 --> 00:07:45,200 Speaker 4: if possible, if the child's not exactly in the group 125 00:07:45,240 --> 00:07:47,760 Speaker 4: of patients than which the experiment was on, they'll say 126 00:07:47,800 --> 00:07:52,320 Speaker 4: it's an unproven therapy, and frankly, they're right, because we 127 00:07:52,560 --> 00:07:55,240 Speaker 4: proved it on a very narrow group of kids that 128 00:07:55,440 --> 00:07:57,960 Speaker 4: in which the benefit could be seen as quickly as possible, 129 00:07:57,960 --> 00:08:00,520 Speaker 4: because we wanted to get the drug out, And so 130 00:08:01,400 --> 00:08:05,440 Speaker 4: the shift in getting drugs to patients that would make 131 00:08:05,440 --> 00:08:09,280 Speaker 4: a difference to them has now shifted from science to 132 00:08:09,960 --> 00:08:15,880 Speaker 4: clinical authorizations and letters and pleadings and newspaper articles. This 133 00:08:15,920 --> 00:08:18,000 Speaker 4: is a different landscape than I would have ever imagined. 134 00:08:18,800 --> 00:08:20,520 Speaker 2: So I'd like to just zoom out here and cover 135 00:08:20,600 --> 00:08:23,360 Speaker 2: some of the basics. For people that don't really know 136 00:08:23,360 --> 00:08:26,720 Speaker 2: anything about SMA, could you just tell us what sm is. 137 00:08:27,080 --> 00:08:30,520 Speaker 4: Spinal muskro atrophy is a rare disease, but it's more 138 00:08:30,720 --> 00:08:34,800 Speaker 4: common than people realize. It's actually, before these new therapies, 139 00:08:34,920 --> 00:08:38,040 Speaker 4: was the most common genetic cause of death and infants 140 00:08:38,320 --> 00:08:42,040 Speaker 4: that would kill about one in ten thousand babies. It 141 00:08:42,040 --> 00:08:46,400 Speaker 4: affects all children of all races around the world with 142 00:08:46,600 --> 00:08:50,840 Speaker 4: roughly equal prevalence. It's a disease that affects essentially the 143 00:08:50,880 --> 00:08:54,240 Speaker 4: motor system, the muscles, and so babies who are born 144 00:08:54,320 --> 00:08:58,040 Speaker 4: with it look normal initially for the most part, and 145 00:08:58,080 --> 00:09:00,559 Speaker 4: then somewhere around two three four months of age, they 146 00:09:00,559 --> 00:09:02,760 Speaker 4: start to get weaker and weaker and weaker, to the 147 00:09:02,800 --> 00:09:06,000 Speaker 4: point where the muscles of breathing no longer work. As 148 00:09:06,040 --> 00:09:09,439 Speaker 4: near as we can tell, they are completely normal cognitively, 149 00:09:10,600 --> 00:09:13,720 Speaker 4: and it's a genetic disease, but it's a recessive genetic disease, 150 00:09:13,880 --> 00:09:16,600 Speaker 4: so when it happens, they don't have a family history. 151 00:09:16,600 --> 00:09:19,640 Speaker 4: For the most part, it's a surprise, and it comes 152 00:09:19,640 --> 00:09:23,000 Speaker 4: because both parents were carriers for it. The other thing 153 00:09:23,040 --> 00:09:26,520 Speaker 4: that's kind of interesting about SMA is that it exists 154 00:09:26,520 --> 00:09:30,560 Speaker 4: for some complicated genetic reasons across the spectrum, so that 155 00:09:30,640 --> 00:09:32,640 Speaker 4: there are the babies two thirds of them are the 156 00:09:32,640 --> 00:09:35,439 Speaker 4: babies that are going to succumb by twelve or fifteen 157 00:09:35,480 --> 00:09:39,040 Speaker 4: months of age, but the other third are milder forms 158 00:09:39,440 --> 00:09:41,600 Speaker 4: that show up a little bit later and are milder, 159 00:09:41,760 --> 00:09:45,000 Speaker 4: and at the mildest end you have adults who are 160 00:09:45,120 --> 00:09:48,559 Speaker 4: troubled by the fact that they can't go upstairs as 161 00:09:48,600 --> 00:09:50,760 Speaker 4: well as they would want to otherwise. So it's an 162 00:09:50,800 --> 00:09:56,120 Speaker 4: absolutely continuous spectrum of decreasing instance to milder and milder expression. 163 00:09:58,360 --> 00:10:00,960 Speaker 2: So could you tell me about y connection to Jace 164 00:10:01,040 --> 00:10:01,959 Speaker 2: and Cheryl Yoda. 165 00:10:02,480 --> 00:10:05,880 Speaker 4: Jace and his mom are my friends now. I first 166 00:10:06,000 --> 00:10:10,240 Speaker 4: met Cheryl and jeremy mom and dad when I cared 167 00:10:10,280 --> 00:10:13,400 Speaker 4: for the first baby, Ariel, and I have over here 168 00:10:13,640 --> 00:10:16,120 Speaker 4: a picture of Ariel. She lived to be fifteen months 169 00:10:16,120 --> 00:10:19,480 Speaker 4: of age, and we did the best possible. A few 170 00:10:19,559 --> 00:10:23,440 Speaker 4: years later, Jace was born and so they brought him 171 00:10:23,440 --> 00:10:25,640 Speaker 4: to me to see if this is what he had 172 00:10:25,760 --> 00:10:27,839 Speaker 4: or not. It was right around that time that we 173 00:10:27,840 --> 00:10:30,320 Speaker 4: were opening up a clinical trial of one of the 174 00:10:30,360 --> 00:10:34,840 Speaker 4: new therapies called Spinoza now, and he was my first patient. 175 00:10:35,120 --> 00:10:38,440 Speaker 4: So he got the dose, and I have a picture 176 00:10:38,559 --> 00:10:41,240 Speaker 4: here as well. I'm looking at the most amazing day 177 00:10:41,280 --> 00:10:44,040 Speaker 4: of my career. He was four months old and it 178 00:10:44,120 --> 00:10:47,880 Speaker 4: was clear that he was getting stronger, not weaker, and 179 00:10:47,920 --> 00:10:50,240 Speaker 4: it was one of the most goosebump moments of my 180 00:10:50,880 --> 00:10:55,480 Speaker 4: entire life, like, we have a cure for a disease 181 00:10:55,520 --> 00:10:58,040 Speaker 4: that used to kill babies, And so Jace over the 182 00:10:58,120 --> 00:11:00,600 Speaker 4: years has gotten stronger and stronger, and it's still for 183 00:11:00,679 --> 00:11:03,280 Speaker 4: me one of these goosebump moments to interact with him 184 00:11:03,280 --> 00:11:05,760 Speaker 4: because he's alive. Oh my god. 185 00:11:05,960 --> 00:11:06,560 Speaker 2: Yeah. 186 00:11:06,800 --> 00:11:11,360 Speaker 4: I was taking care of babies that lose and now 187 00:11:11,520 --> 00:11:15,200 Speaker 4: after this therapy, I get to be the person who 188 00:11:15,480 --> 00:11:19,440 Speaker 4: helps them not And no one could ever imagine a 189 00:11:19,480 --> 00:11:23,120 Speaker 4: career that goes from that to where we are now. 190 00:11:24,040 --> 00:11:27,520 Speaker 4: It's a feeling, an unbelieval ecstasy that we can We 191 00:11:27,640 --> 00:11:30,480 Speaker 4: don't have to do that anymore. I'm not burying babies. 192 00:11:31,960 --> 00:11:32,240 Speaker 1: Wow. 193 00:11:32,400 --> 00:11:36,240 Speaker 2: What an incredible shift. So I understand your research on 194 00:11:36,280 --> 00:11:39,960 Speaker 2: how SMA works was crucial for developing the miracle drugs 195 00:11:40,000 --> 00:11:43,080 Speaker 2: bin Rauser, as well as other drugs for SMA, and 196 00:11:43,120 --> 00:11:46,600 Speaker 2: you were instrumental in designing the clinical trial to test spinraser. 197 00:11:47,000 --> 00:11:48,319 Speaker 2: Could you tell me more about that. 198 00:11:49,000 --> 00:11:52,719 Speaker 4: Yeah. So I had the privilege of being carrying for 199 00:11:52,920 --> 00:11:55,679 Speaker 4: kids with SMA from the very beginning. I was very 200 00:11:55,720 --> 00:11:59,160 Speaker 4: much involved in designing the clinical trials and the outcome 201 00:11:59,160 --> 00:12:03,280 Speaker 4: measures necess to do the clinical trials. And then when 202 00:12:03,320 --> 00:12:05,920 Speaker 4: we had this first idea with spin raza, I was 203 00:12:05,960 --> 00:12:09,640 Speaker 4: the lead investigator here at Hopkins to give the drug 204 00:12:09,679 --> 00:12:11,560 Speaker 4: and then follow how well, they do and see if 205 00:12:11,559 --> 00:12:14,840 Speaker 4: there's any complications of it, and we've since studied quite 206 00:12:14,880 --> 00:12:17,040 Speaker 4: a number beyond that to show that it has a 207 00:12:17,080 --> 00:12:20,720 Speaker 4: broader effect and its benefit for this and the other 208 00:12:20,800 --> 00:12:21,680 Speaker 4: drugs for SME. 209 00:12:22,559 --> 00:12:25,120 Speaker 2: So, can you describe how drugs like spinraza work. 210 00:12:25,679 --> 00:12:30,400 Speaker 4: Spin Rasa and the other therapies are high end therapies 211 00:12:30,400 --> 00:12:34,680 Speaker 4: that are really designer for a very very specific mutation 212 00:12:34,960 --> 00:12:38,760 Speaker 4: in a specific genetic defect, and as such a high 213 00:12:38,800 --> 00:12:41,000 Speaker 4: effect therapy for rare disease. 214 00:12:41,440 --> 00:12:43,760 Speaker 2: What sort of diseases can these therapies treat? 215 00:12:44,880 --> 00:12:49,440 Speaker 4: Well? Right now they're targeting these mostly rare disorders, but 216 00:12:49,559 --> 00:12:53,120 Speaker 4: we can imagine targeting many of the named diseases like 217 00:12:53,200 --> 00:12:57,640 Speaker 4: cystic fibrosis or sickle cell disease or hemophilia. There's also 218 00:12:57,920 --> 00:13:01,920 Speaker 4: the promise that we can extend this to extremely rare diseases, 219 00:13:01,960 --> 00:13:04,600 Speaker 4: things that affect only a few people in the world 220 00:13:04,640 --> 00:13:07,040 Speaker 4: at a time. And so when we know the mutation, 221 00:13:07,800 --> 00:13:10,720 Speaker 4: there's the future prospect that we can design a drug 222 00:13:10,800 --> 00:13:15,840 Speaker 4: for that mutation in that patient, which is just abtolutely awesome. 223 00:13:16,360 --> 00:13:17,960 Speaker 4: It used to be the idea that we had to 224 00:13:18,040 --> 00:13:21,320 Speaker 4: understand why a disease happened and now it looks like 225 00:13:21,360 --> 00:13:23,760 Speaker 4: we have therapiest where we can skip over that step 226 00:13:24,160 --> 00:13:28,559 Speaker 4: and repair or replace the specific abdamality at the very 227 00:13:28,640 --> 00:13:33,120 Speaker 4: start by supplying something that was missing or undermining something 228 00:13:33,160 --> 00:13:34,160 Speaker 4: that's doing damage. 229 00:13:34,960 --> 00:13:37,080 Speaker 2: So one of the things we've heard is that drugs 230 00:13:37,120 --> 00:13:40,080 Speaker 2: like spin rasa are incredibly expensive, and that part of 231 00:13:40,120 --> 00:13:42,439 Speaker 2: what seems to drive the price of these miracle drugs 232 00:13:42,559 --> 00:13:45,400 Speaker 2: is how much benefit they provide to the patient. But 233 00:13:45,480 --> 00:13:48,360 Speaker 2: what happens to the price when for some patients the 234 00:13:48,400 --> 00:13:51,880 Speaker 2: benefit is so much more than it is for other patients. So, 235 00:13:52,040 --> 00:13:56,320 Speaker 2: for example, patients who have milder forms of SMA, the 236 00:13:56,360 --> 00:13:59,720 Speaker 2: benefit might be less in comparison to patients who born 237 00:13:59,760 --> 00:14:01,320 Speaker 2: with the more severe forms. 238 00:14:01,960 --> 00:14:05,199 Speaker 4: This is the amazing quandary, and spinal must Gratupy shows 239 00:14:05,200 --> 00:14:08,440 Speaker 4: this more than anything, because the price is sort of 240 00:14:08,480 --> 00:14:10,240 Speaker 4: based upon the fact that a baby is going to 241 00:14:10,280 --> 00:14:14,920 Speaker 4: live and have a full and impactful life. And what's 242 00:14:14,960 --> 00:14:20,680 Speaker 4: that worth. Obviously it's worth everything. It's interesting that patients say, 243 00:14:20,760 --> 00:14:22,960 Speaker 4: I'm not getting better. I'm not like that kid, the 244 00:14:23,040 --> 00:14:25,280 Speaker 4: baby that got much better. I said, no, the baby's 245 00:14:25,280 --> 00:14:28,400 Speaker 4: getting better because we stopped degeneration at a time when 246 00:14:28,440 --> 00:14:31,880 Speaker 4: babies normally climb out of their mom's arms and run 247 00:14:31,920 --> 00:14:34,400 Speaker 4: around and then and develop and do normally. So we 248 00:14:34,480 --> 00:14:37,600 Speaker 4: allow that normal development to happen, But what's really happened 249 00:14:37,640 --> 00:14:40,800 Speaker 4: is we stopped degeneration. And so if we wait to 250 00:14:41,120 --> 00:14:44,640 Speaker 4: give the drug until after development has already accrued, they 251 00:14:44,680 --> 00:14:47,640 Speaker 4: actually are not getting better. They're just not declining any further. 252 00:14:48,280 --> 00:14:51,440 Speaker 4: And that's another place where the quandary between the insurance 253 00:14:51,480 --> 00:14:55,360 Speaker 4: providers and the doctors and the patients are in clash 254 00:14:55,520 --> 00:15:02,520 Speaker 4: because evidence is really hard to come by. Spinal weskiatvie 255 00:15:02,840 --> 00:15:06,760 Speaker 4: is going to be the story of many of the 256 00:15:06,800 --> 00:15:09,120 Speaker 4: diseases to come. So I think we can learn a 257 00:15:09,160 --> 00:15:13,920 Speaker 4: lot from what's happened so far, and this tension is 258 00:15:13,960 --> 00:15:17,120 Speaker 4: going to be a big issue in the next couple decades. 259 00:15:19,680 --> 00:15:22,120 Speaker 2: Tom, thank you so much for joining us today and 260 00:15:22,200 --> 00:15:26,560 Speaker 2: talking about this topic. Coming up after the break, we'll 261 00:15:26,560 --> 00:15:29,400 Speaker 2: hear more about how these miracle drugs are priced and 262 00:15:29,400 --> 00:15:44,040 Speaker 2: why accessing them is so difficult. Cheryl Yoda's experience of 263 00:15:44,080 --> 00:15:46,800 Speaker 2: getting insurance to cover the cost of her son Jas's 264 00:15:46,840 --> 00:15:51,200 Speaker 2: medicine is hardly typical. Holly Fernander's Lynch is a professor 265 00:15:51,240 --> 00:15:54,320 Speaker 2: of medical Ethics and Law at the University of Pennsylvania. 266 00:15:55,080 --> 00:16:00,200 Speaker 2: She focuses on access to investigational medicines, food and drug administration, policy, 267 00:16:00,560 --> 00:16:02,000 Speaker 2: and gatekeeping in healthcare. 268 00:16:03,440 --> 00:16:06,600 Speaker 3: I think equity is a huge challenge in this context. 269 00:16:06,720 --> 00:16:10,160 Speaker 3: So do you have insurance in the first place? How 270 00:16:10,360 --> 00:16:13,880 Speaker 3: high quality is the insurance coverage that you have access to. 271 00:16:14,520 --> 00:16:17,920 Speaker 3: But there's a lot of important data showing that even 272 00:16:18,040 --> 00:16:21,840 Speaker 3: people who have good insurance coverage are often unable to 273 00:16:22,040 --> 00:16:25,240 Speaker 3: access these high priced medicines because their out of pocket 274 00:16:25,280 --> 00:16:29,920 Speaker 3: costs are still so high. It's very few people who 275 00:16:29,920 --> 00:16:33,680 Speaker 3: can pay tens of thousands of dollars for a drug, 276 00:16:33,760 --> 00:16:35,840 Speaker 3: let alone, you know, the out of pocket costs for 277 00:16:35,880 --> 00:16:39,040 Speaker 3: a million dollar or two million dollar drug. So equity 278 00:16:39,120 --> 00:16:40,480 Speaker 3: is going to be a huge issue too. 279 00:16:41,120 --> 00:16:45,040 Speaker 2: The price point for spinras that seems shockingly high to me. 280 00:16:46,400 --> 00:16:49,400 Speaker 2: How do drug companies arrive at such high prices in 281 00:16:49,440 --> 00:16:50,160 Speaker 2: the first place. 282 00:16:51,200 --> 00:16:54,120 Speaker 3: So the way that you typically hear companies talking about 283 00:16:54,200 --> 00:16:57,360 Speaker 3: this is based on the research and development costs, what 284 00:16:57,440 --> 00:17:01,080 Speaker 3: it takes to actually bring these products to market. And 285 00:17:01,440 --> 00:17:04,080 Speaker 3: in that context, they're not thinking just about the drug 286 00:17:04,119 --> 00:17:07,040 Speaker 3: that ultimately got approved, but all the other drugs that 287 00:17:07,119 --> 00:17:11,360 Speaker 3: failed along the way before they got to human trials 288 00:17:11,560 --> 00:17:15,360 Speaker 3: or in earlier stages of clinical development, So that's certainly 289 00:17:15,480 --> 00:17:18,640 Speaker 3: part of it. However, there has been quite a bit 290 00:17:18,680 --> 00:17:21,760 Speaker 3: of research to demonstrate that it's not the R and 291 00:17:21,840 --> 00:17:26,119 Speaker 3: D prices that are dictating price that's actually set for 292 00:17:26,240 --> 00:17:31,439 Speaker 3: these drugs. It's market conditions. It's how much will the 293 00:17:31,480 --> 00:17:36,000 Speaker 3: market bear. What ultimately happens is that pharmaceutical companies get 294 00:17:36,040 --> 00:17:39,240 Speaker 3: to decide what the list price is going to be 295 00:17:39,280 --> 00:17:41,560 Speaker 3: for their drug. They come onto the market with very 296 00:17:41,640 --> 00:17:45,280 Speaker 3: high prices, and then payers have very little choice but 297 00:17:45,400 --> 00:17:47,280 Speaker 3: to pay those prices. 298 00:17:47,720 --> 00:17:50,439 Speaker 2: What about relative benefit, Well, the value of a drug. 299 00:17:50,920 --> 00:17:53,480 Speaker 2: Does that get taken into account when setting the price. 300 00:17:54,240 --> 00:17:59,120 Speaker 3: So value based pricing is really important. What impact are 301 00:17:59,160 --> 00:18:03,080 Speaker 3: these drugs having on people's lives? In many cases, the 302 00:18:03,160 --> 00:18:06,119 Speaker 3: drug price has no bearing on that. And that is 303 00:18:06,160 --> 00:18:10,720 Speaker 3: where we really run into ethical challenges because if a 304 00:18:10,840 --> 00:18:14,959 Speaker 3: drug is not very good, right, maybe it has a 305 00:18:14,960 --> 00:18:17,720 Speaker 3: marginal benefit, then we can say, look, we shouldn't be 306 00:18:17,760 --> 00:18:20,560 Speaker 3: paying high prices for that drug. But if a drug 307 00:18:20,600 --> 00:18:23,920 Speaker 3: is a cure for a life threatening disease, especially a 308 00:18:24,040 --> 00:18:27,520 Speaker 3: disease affecting children, the value is going to be really 309 00:18:27,600 --> 00:18:30,800 Speaker 3: high right, sometimes in the realm of millions of dollars, 310 00:18:30,880 --> 00:18:33,119 Speaker 3: and you might even say that those are cost effective 311 00:18:33,200 --> 00:18:36,119 Speaker 3: drugs because it's going to reduce the lifetime costs that 312 00:18:36,160 --> 00:18:38,959 Speaker 3: you might otherwise have to pay. So that's where those 313 00:18:39,000 --> 00:18:42,480 Speaker 3: are some of the most ethically challenging issues because their 314 00:18:42,480 --> 00:18:45,919 Speaker 3: cost effective, high value drugs. But if we had to 315 00:18:45,960 --> 00:18:48,600 Speaker 3: pay for them for a lot of patients, we won't 316 00:18:48,640 --> 00:18:50,879 Speaker 3: have enough money or resources to do that. 317 00:18:51,240 --> 00:18:53,480 Speaker 2: So we had earlier in the episode that the price 318 00:18:53,480 --> 00:18:56,159 Speaker 2: of spin Rouser was announced just after the drug was 319 00:18:56,160 --> 00:18:59,879 Speaker 2: approved by the FDA. Does the FDA approval process have 320 00:19:00,080 --> 00:19:01,760 Speaker 2: anything to do with the drug pricing? 321 00:19:02,359 --> 00:19:07,320 Speaker 3: They are completely disconnected. When FDA approves a drug, it 322 00:19:07,359 --> 00:19:12,520 Speaker 3: is evaluating only the safety and effectiveness of that drug. 323 00:19:13,320 --> 00:19:16,720 Speaker 3: It has nothing to do with price. It doesn't even 324 00:19:16,760 --> 00:19:18,960 Speaker 3: know what the price is because the price may not 325 00:19:19,040 --> 00:19:22,439 Speaker 3: be publicly announced. Yet even if it did know, it 326 00:19:22,480 --> 00:19:28,280 Speaker 3: could not take that into consideration. Once FDA approves a product, 327 00:19:28,640 --> 00:19:33,280 Speaker 3: then we get into questions about drug pricing and coverage, 328 00:19:33,720 --> 00:19:36,160 Speaker 3: and there's some debate about whether that's the right approach. 329 00:19:36,280 --> 00:19:38,119 Speaker 2: So could you talk a bit more about that debate? 330 00:19:38,359 --> 00:19:41,639 Speaker 2: What concerns does this rise from an ethics perspective. 331 00:19:41,640 --> 00:19:45,680 Speaker 3: Yeah, the debate largely has to do with whose responsibility 332 00:19:45,840 --> 00:19:50,400 Speaker 3: is it to consider price? And FDA is viewed as 333 00:19:50,440 --> 00:19:55,200 Speaker 3: a science based agency. Their expertise is evaluating clinical trial 334 00:19:55,320 --> 00:20:00,280 Speaker 3: data and evaluating is this drug strong enough that the 335 00:20:00,320 --> 00:20:04,040 Speaker 3: statutory standard has been met? Can we feel confident that 336 00:20:04,119 --> 00:20:07,639 Speaker 3: patients taking this drug the risk benefit ratio is going 337 00:20:07,680 --> 00:20:10,280 Speaker 3: to be reasonable for them. The product then goes on 338 00:20:10,359 --> 00:20:14,280 Speaker 3: the market, and if patients want to take it, then 339 00:20:14,440 --> 00:20:17,600 Speaker 3: they need to find some way of paying for it. 340 00:20:17,800 --> 00:20:21,080 Speaker 3: Many people will go to private insurance coverage to pay 341 00:20:21,560 --> 00:20:25,639 Speaker 3: Lots of people are covered through government programs. Sometimes what 342 00:20:25,680 --> 00:20:28,240 Speaker 3: you see happening is for these very high priced drugs, 343 00:20:28,280 --> 00:20:31,880 Speaker 3: even if people have insurance coverage, they are crowd funding 344 00:20:32,280 --> 00:20:36,280 Speaker 3: to pay for the out of pocket costs. And that's 345 00:20:36,320 --> 00:20:38,400 Speaker 3: when you get people, you know, kind of pulling their 346 00:20:38,440 --> 00:20:41,320 Speaker 3: hair out, like, how could it be in this highly 347 00:20:41,359 --> 00:20:45,080 Speaker 3: privileged nation, we're asking people to go on the internet 348 00:20:45,200 --> 00:20:48,040 Speaker 3: and ask their friends and family for money to pay 349 00:20:48,119 --> 00:20:53,040 Speaker 3: for maybe life saving products, but certainly life affecting products. 350 00:20:53,119 --> 00:20:56,520 Speaker 2: So can any federal agency set limits on drug pricing? 351 00:20:57,080 --> 00:21:00,680 Speaker 3: There are lots of federal payers that are involved in coverage. 352 00:21:00,720 --> 00:21:05,480 Speaker 3: So we have Medicare, Medicaid, and then the Veterans Administration, 353 00:21:05,800 --> 00:21:08,320 Speaker 3: and they all are a bit different in what their 354 00:21:08,359 --> 00:21:12,000 Speaker 3: statutory authority allows them to do. So you may have 355 00:21:12,119 --> 00:21:15,800 Speaker 3: heard about the Inflation Reduction Act. It's a huge piece 356 00:21:15,800 --> 00:21:18,960 Speaker 3: of legislation that has lots of components to it, two 357 00:21:19,000 --> 00:21:22,440 Speaker 3: of which have to do with drug pricing. So for 358 00:21:22,480 --> 00:21:28,679 Speaker 3: a very long time, Medicare was not allowed to negotiate price. 359 00:21:29,359 --> 00:21:31,680 Speaker 3: So FDA would say this drug is safe and effective. 360 00:21:32,160 --> 00:21:35,480 Speaker 3: Then Medicare had a decision to make is this drug 361 00:21:35,640 --> 00:21:40,120 Speaker 3: reasonable and necessary for the Medicare population. So Medicare can 362 00:21:40,160 --> 00:21:44,680 Speaker 3: say it's not reasonable and necessary or it is. They 363 00:21:44,680 --> 00:21:46,760 Speaker 3: have an on off switch, are we going to cover 364 00:21:46,840 --> 00:21:51,359 Speaker 3: it or not? But they can't consider cost or cost effectiveness, 365 00:21:51,400 --> 00:21:55,800 Speaker 3: so they couldn't negotiate for prices. Under the Inflation Reduction Act, 366 00:21:56,280 --> 00:22:00,359 Speaker 3: for the first time, Medicare has the ability to start 367 00:22:00,400 --> 00:22:04,439 Speaker 3: negotiating prices on a very limited set of drugs. To 368 00:22:04,560 --> 00:22:08,640 Speaker 3: start off with, do you basically have nine to thirteen 369 00:22:08,720 --> 00:22:11,320 Speaker 3: years as a drug company to profit as much as 370 00:22:11,359 --> 00:22:14,439 Speaker 3: you want before Medicare could negotiate the price. There are 371 00:22:14,440 --> 00:22:18,200 Speaker 3: some other exclusions for drugs, for rare diseases, for example. 372 00:22:18,960 --> 00:22:22,360 Speaker 3: But this is a big development. Medicare will negotiate prices 373 00:22:22,359 --> 00:22:27,080 Speaker 3: on those ten drugs, and then every year thereafter additional 374 00:22:27,160 --> 00:22:30,119 Speaker 3: drugs will kind of get added to the negotiation list. 375 00:22:30,480 --> 00:22:32,600 Speaker 2: And so we hear these stories about people that have 376 00:22:32,680 --> 00:22:36,359 Speaker 2: to fight their insurance companies in the US to get 377 00:22:36,400 --> 00:22:41,480 Speaker 2: expensive treatment covered, and there's some varying outcomes. Is there 378 00:22:41,520 --> 00:22:45,120 Speaker 2: a rhymal reason to this when the decisions are made? 379 00:22:45,600 --> 00:22:49,960 Speaker 3: So, what insurance companies are typically doing is evaluating whether 380 00:22:50,000 --> 00:22:54,720 Speaker 3: a drug is medically necessary, and that might be a 381 00:22:54,760 --> 00:23:00,119 Speaker 3: little bit different than the broad label or indication for 382 00:23:00,160 --> 00:23:04,040 Speaker 3: which FDA approves a drug. So sometimes FDA will approve 383 00:23:04,040 --> 00:23:07,320 Speaker 3: a drug for a very narrow population children up to 384 00:23:07,359 --> 00:23:11,080 Speaker 3: age two, for example. Sometimes though FDA gives a very 385 00:23:11,080 --> 00:23:15,320 Speaker 3: broad indication, you know, people with early stage dementia or 386 00:23:15,359 --> 00:23:19,159 Speaker 3: Alzheimer's disease, for example. And so part of what the 387 00:23:19,200 --> 00:23:23,080 Speaker 3: insurance company has to do is evaluate is there some 388 00:23:23,320 --> 00:23:27,520 Speaker 3: more narrow population for which this FDA approved drug is 389 00:23:27,520 --> 00:23:32,320 Speaker 3: going to truly be medically necessary. Sometimes insurers will say 390 00:23:32,800 --> 00:23:35,840 Speaker 3: you need to have prior authorization, so we want your 391 00:23:35,960 --> 00:23:39,360 Speaker 3: doctor to talk to our insurance company doctors to make 392 00:23:39,400 --> 00:23:43,200 Speaker 3: sure that you can access the drug. Sometimes they will 393 00:23:43,840 --> 00:23:47,640 Speaker 3: make you try other drugs first, but in many cases, 394 00:23:47,760 --> 00:23:50,680 Speaker 3: the drugs that are getting approved are the only thing 395 00:23:51,119 --> 00:23:56,840 Speaker 3: for the disease or condition in question. It's this or nothing. 396 00:23:57,160 --> 00:24:01,280 Speaker 3: And so sometimes we do see insurance company denying access 397 00:24:01,400 --> 00:24:05,240 Speaker 3: to patients because they say it's not medically necessary, and 398 00:24:05,280 --> 00:24:08,080 Speaker 3: then you get into appeals where you know the patient's 399 00:24:08,080 --> 00:24:10,280 Speaker 3: physician will go to the company and say no, it 400 00:24:10,320 --> 00:24:13,720 Speaker 3: really is medically necessary. But of course that takes time 401 00:24:13,920 --> 00:24:17,520 Speaker 3: and resources to go back and forth. It is important 402 00:24:17,800 --> 00:24:21,840 Speaker 3: for insurers to be able to carefully evaluate should we 403 00:24:22,000 --> 00:24:25,360 Speaker 3: be paying this price for this drug for this patient, 404 00:24:25,880 --> 00:24:28,679 Speaker 3: because if they don't carefully analyze that, they're going to 405 00:24:28,720 --> 00:24:31,840 Speaker 3: run out of resources for their pool. The question is, 406 00:24:32,000 --> 00:24:33,920 Speaker 3: you know, how far do they need to go in 407 00:24:34,040 --> 00:24:35,000 Speaker 3: that regard. 408 00:24:35,240 --> 00:24:38,520 Speaker 2: So you've been talking about the US so far. How 409 00:24:38,600 --> 00:24:42,360 Speaker 2: are these issues addressed outside the US and what different 410 00:24:42,680 --> 00:24:45,800 Speaker 2: essical issues to these approaches raise. 411 00:24:46,600 --> 00:24:49,800 Speaker 3: Yeah, there's two big differences between the US and elsewhere. 412 00:24:50,359 --> 00:24:53,959 Speaker 3: In the US, we have private health insurance system that 413 00:24:54,119 --> 00:24:57,720 Speaker 3: runs alongside public health insurance, right, so a lot of 414 00:24:57,760 --> 00:25:01,119 Speaker 3: Americans get their coverage through their owls lawyers, and we 415 00:25:01,200 --> 00:25:04,919 Speaker 3: have lots of different insurers. Outside the US, right we 416 00:25:04,960 --> 00:25:09,480 Speaker 3: see more typically national health systems and national health insurance 417 00:25:09,560 --> 00:25:12,480 Speaker 3: provided by the government to the citizens. And then you 418 00:25:12,600 --> 00:25:17,080 Speaker 3: have the government decide is this drug worth paying for 419 00:25:17,520 --> 00:25:19,600 Speaker 3: and how much are we going to pay for it? 420 00:25:20,040 --> 00:25:22,520 Speaker 3: And there's pros and cons to that. So in the US, 421 00:25:23,040 --> 00:25:25,600 Speaker 3: once a FDA approves a drug, if you can find 422 00:25:25,640 --> 00:25:28,280 Speaker 3: a way to pay for it, there's no waiting. You 423 00:25:28,280 --> 00:25:32,399 Speaker 3: could gain access to it the very next day. Elsewhere 424 00:25:32,760 --> 00:25:37,159 Speaker 3: there can be a pretty substantial lag in between regulatory 425 00:25:37,200 --> 00:25:40,159 Speaker 3: approval for the drug and when patients are going to 426 00:25:40,200 --> 00:25:42,800 Speaker 3: access it because the government has to decide whether it's 427 00:25:42,840 --> 00:25:45,760 Speaker 3: going to pay. But the pro is that it is 428 00:25:46,080 --> 00:25:49,400 Speaker 3: a wiser use of resources because then the government is 429 00:25:49,520 --> 00:25:52,040 Speaker 3: negotiating with a company, here's how much we think your 430 00:25:52,040 --> 00:25:56,440 Speaker 3: product is worth. It's a stronger stewardship of healthcare resources 431 00:25:56,480 --> 00:25:58,520 Speaker 3: for the government to say this is what this product 432 00:25:58,560 --> 00:26:00,199 Speaker 3: is worth, and this is all we're going on to 433 00:26:00,200 --> 00:26:02,879 Speaker 3: be willing to pay. And that's really important because we 434 00:26:02,920 --> 00:26:07,199 Speaker 3: don't have unlimited resources in any capacity, let alone unlimited 435 00:26:07,359 --> 00:26:09,879 Speaker 3: money to pay for medicines. 436 00:26:11,640 --> 00:26:14,800 Speaker 2: Could you tell us how big an issue you think 437 00:26:14,840 --> 00:26:16,679 Speaker 2: this is likely to be in the coming years. 438 00:26:17,480 --> 00:26:21,480 Speaker 3: So you know, if it's spinal muscular atrophy, it's affecting 439 00:26:21,920 --> 00:26:25,760 Speaker 3: deeply affecting, but affecting us proportionately small part of the population, 440 00:26:26,640 --> 00:26:30,520 Speaker 3: and so you can find ways to cover very high 441 00:26:30,520 --> 00:26:37,200 Speaker 3: priced products because it's spread over a broader population that's paying. 442 00:26:37,359 --> 00:26:40,520 Speaker 3: But once you start to have drugs that are very 443 00:26:40,520 --> 00:26:44,160 Speaker 3: effective for terrible diseases that affect a much wider part 444 00:26:44,200 --> 00:26:46,960 Speaker 3: of the population, you're not going to be able to 445 00:26:46,960 --> 00:26:49,399 Speaker 3: fudge your way through it the way you can with 446 00:26:49,600 --> 00:26:52,080 Speaker 3: rare disease. And then we're really going to have to 447 00:26:52,119 --> 00:26:56,440 Speaker 3: face the music to say these drugs are important, they're 448 00:26:56,440 --> 00:26:59,840 Speaker 3: cost effective, but they're hugely expensive. Are we going to 449 00:26:59,840 --> 00:27:01,639 Speaker 3: pay for this or are we going to pay for 450 00:27:01,720 --> 00:27:05,399 Speaker 3: something else that's important to a functional society? 451 00:27:05,680 --> 00:27:07,840 Speaker 2: Yeah, I mean, how do you think we should navigate 452 00:27:07,880 --> 00:27:08,879 Speaker 2: these kind of questions? 453 00:27:09,280 --> 00:27:11,560 Speaker 3: This is what ethicists work on all the time. These 454 00:27:11,560 --> 00:27:15,600 Speaker 3: are career spanning ethical challenges, trying to come up with 455 00:27:15,640 --> 00:27:18,760 Speaker 3: the best arguments for one approach or another. There's not 456 00:27:18,840 --> 00:27:21,680 Speaker 3: a clear cut response to say this is the right 457 00:27:21,720 --> 00:27:23,920 Speaker 3: way to spend our money, this is the wrong way 458 00:27:24,960 --> 00:27:30,000 Speaker 3: in the US at least, this has been politicized in 459 00:27:30,000 --> 00:27:35,360 Speaker 3: a really terrible way. The minute you start talking about 460 00:27:35,440 --> 00:27:41,840 Speaker 3: not covering things, there are claims of government death panels 461 00:27:42,240 --> 00:27:47,000 Speaker 3: that are going to put a price on life, and 462 00:27:47,080 --> 00:27:49,679 Speaker 3: who wants to live in a world like that. On 463 00:27:49,760 --> 00:27:52,800 Speaker 3: the other hand, that completely fails to recognize that we 464 00:27:52,880 --> 00:27:55,199 Speaker 3: have to make judgments about how we're going to spend 465 00:27:55,320 --> 00:28:00,760 Speaker 3: our resources, and it's reasonable to ask, even if it's 466 00:28:00,800 --> 00:28:04,479 Speaker 3: life saving, how much does something cost? Can we afford it? 467 00:28:04,520 --> 00:28:08,160 Speaker 3: And what's the trade off? Very often, if something's life saving, 468 00:28:09,080 --> 00:28:11,000 Speaker 3: people are going to be willing to accept that trade off. 469 00:28:11,040 --> 00:28:13,199 Speaker 3: Right the community is going to be willing to accept 470 00:28:13,240 --> 00:28:15,600 Speaker 3: that trade off. But we can't stick our head in 471 00:28:15,600 --> 00:28:19,080 Speaker 3: the sand and pretend like we have unlimited resources because 472 00:28:19,080 --> 00:28:20,960 Speaker 3: we don't. 473 00:28:21,840 --> 00:28:24,960 Speaker 2: Cheryl Yoda is beyond grateful that a drug was developed 474 00:28:25,000 --> 00:28:28,640 Speaker 2: in time to save her son Jayce's life, especially when 475 00:28:28,760 --> 00:28:32,840 Speaker 2: so many other less rare diseases need funding to But 476 00:28:32,920 --> 00:28:36,480 Speaker 2: does that mean she thinks Spinaza's price is fair? She says, 477 00:28:36,480 --> 00:28:37,800 Speaker 2: the answer isn't so simple. 478 00:28:38,480 --> 00:28:41,000 Speaker 1: What is a good price, or like, what would be 479 00:28:41,000 --> 00:28:44,000 Speaker 1: a reasonable cost? I have no idea, like I don't 480 00:28:44,000 --> 00:28:46,719 Speaker 1: feel like I know. I mean, that's the whole question. 481 00:28:48,440 --> 00:28:51,160 Speaker 2: She says, She's been so fortunate already that it would 482 00:28:51,160 --> 00:28:54,040 Speaker 2: feel almost selfish to hope that the drug's price would 483 00:28:54,040 --> 00:28:58,200 Speaker 2: come down to her. Calling Spinaza a miracle drug isn't 484 00:28:58,200 --> 00:29:01,960 Speaker 2: an exaggeration, but still she can't help but dream of 485 00:29:02,000 --> 00:29:04,040 Speaker 2: a day when it's more easily accessible. 486 00:29:04,760 --> 00:29:08,320 Speaker 1: I know that the science that goes into making it 487 00:29:08,360 --> 00:29:12,240 Speaker 1: is still pretty complicated, but the more they know about 488 00:29:12,240 --> 00:29:14,640 Speaker 1: it and the longer they're making it, they get better 489 00:29:14,680 --> 00:29:17,440 Speaker 1: at it or easier, and it wouldn't have to be 490 00:29:17,480 --> 00:29:28,280 Speaker 1: so expensive. It's a good dream. 491 00:29:28,560 --> 00:29:31,680 Speaker 2: Next time on Playing God, we look into the future 492 00:29:31,920 --> 00:29:35,320 Speaker 2: to learn more about a technology that would create human 493 00:29:35,360 --> 00:29:39,440 Speaker 2: babies without starting with sperm or eggs. It's a long 494 00:29:39,480 --> 00:29:42,719 Speaker 2: way off, but researchers and biotech companies are already at 495 00:29:42,760 --> 00:29:46,600 Speaker 2: work or making this possible. If they succeed, it would 496 00:29:46,640 --> 00:29:49,120 Speaker 2: open up a whole world of new options for how 497 00:29:49,200 --> 00:29:53,240 Speaker 2: humans procreate. But how can we be sure this technology 498 00:29:53,320 --> 00:29:58,040 Speaker 2: is introduced safely and ethically. That's next time on Playing God. 499 00:30:01,840 --> 00:30:06,280 Speaker 2: Thank you to our guests Cheryl Yoder, Holly Fernandez Lynch, 500 00:30:06,800 --> 00:30:11,480 Speaker 2: and Tom Crawford. Playing God is a co production of 501 00:30:11,560 --> 00:30:15,080 Speaker 2: Pushkin Industries and the Berman Institute of Bioethics at Johns 502 00:30:15,120 --> 00:30:20,240 Speaker 2: Hopkins University. Emily Vaughan is our lead producer. This episode 503 00:30:20,400 --> 00:30:24,360 Speaker 2: was also produced by Sophie Crane and Lucy Sullivan. Our 504 00:30:24,480 --> 00:30:29,320 Speaker 2: editors are Karen Shakergee and Kate Parkinson Morgan. Mixing by 505 00:30:29,360 --> 00:30:34,280 Speaker 2: Samir Sengupta, the music by Echo Mountain, Engineering support from 506 00:30:34,320 --> 00:30:39,480 Speaker 2: Sarah Brugare and Amanda ka Wang. Show art by Sean Carney, 507 00:30:40,480 --> 00:30:45,200 Speaker 2: fact checking by David jar and Arthur Gompertz. Our executive 508 00:30:45,240 --> 00:30:50,960 Speaker 2: producer is Justine Lang at the Johns Hopkins Berman Institute 509 00:30:50,960 --> 00:30:55,400 Speaker 2: of Bioethics. Our executive producers are Jeffrey Kahan and Anna Mastriani, 510 00:30:55,880 --> 00:31:01,040 Speaker 2: working with Emilia Hood. Specials thanks to Jared Whyland. Funding 511 00:31:01,080 --> 00:31:05,760 Speaker 2: provided by the Greenwall Foundation. I'm Laurena Rura Hutchinson. Come 512 00:31:05,760 --> 00:31:19,360 Speaker 2: back next week for more Playing God. Has this show 513 00:31:19,400 --> 00:31:23,640 Speaker 2: inspired you? Are you interested in studying bioethics? Perhaps you 514 00:31:23,680 --> 00:31:26,680 Speaker 2: want to become someone shaping this field. We have a 515 00:31:26,760 --> 00:31:30,240 Speaker 2: Master of Bioethics program at the Johns Hopkins Berman Institute 516 00:31:30,240 --> 00:31:34,840 Speaker 2: of Bioethics. To find out more, visit Bioethics dot Jhu 517 00:31:35,000 --> 00:31:39,400 Speaker 2: dot edu. Forward slash MBE scholarships are available.