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Life-and-death dilemmas. New medical technologies. Controversial treatments. In playing god? we hear from the patients whose lives were transformed—and sometimes saved—by medical innovations and the bioethicists who help guide complex decisions.
Ventilators can keep critically ill people alive, but when is it acceptable to turn the machines off? Organ transplants save lives, but when demand outpaces supply, how do we decide who gets them? Novel reproductive technologies can help people have babies in ways that are far beyond what nature allows. So, when should these “Brave New World” technologies be introduced, and who should control them?
playing god? is a production of the Berman Institute of Bioethics at Johns Hopkins University, with generous support from The Greenwall Foundation. New episodes drop every Tuesday.
The Berman Institute has created a guide for each episode where you can learn more about the guests, the history, and the ethics issues at: bioethics.jhu.edu/playing-god
00:00:00
Speaker 1: I got the little vial out and it's just like five milliliters of liquid gold. You know, it wasn't in the bottle because it was inside of our son. But I was just looking at that little bottle and teary eyed, you know, like, oh my goodness, it's so small, and yet it's been so big in our lives. And how do you put a price on that? I don't know.
00:00:27
Speaker 2: This is Cheryl Yoda. The liquid gold she's referring to is a medication called nusan Erson that saved the life of her eight year old son, Jace.
00:00:37
Speaker 1: His big thing right now is hunting. We've literally gotten up and he's been out for like an hour stalking around the backyard with his bow and arrow.
00:00:45
Speaker 2: He's practicing for when he's old enough to go deer hunting with his dad and big brothers. But back when Jace was a baby, Cheryl didn't think anything like that would ever be possible for him. When Jace was eight days old, he was diagnosed with rare genetic disease called SMA, or spinal muscular atrophy. SMA interferes with the brain's ability to communicate with muscles over time. People with the most severe form of the disease like Jace lose the ability to swallow and breathe. Doctors told Cheryl it was unlikely Jace would live to see his second birthday. Sadly, Cheryl was already familiar with SMA before Jace. She'd given birth to two boys who didn't have SMA and a daughter, Ariel, who did. Ariel died when she was fifteen months old, and after learning about Jace's diagnosis, she thought he would too.
00:01:42
Speaker 1: It just felt like he had received basically a death sentence, and we were going to kind of enjoy him for a year and a half to two years and then try to help him be as happy and comfortable and have a great life as he could, and then have to say goodbye to him.
00:02:00
Speaker 2: Jace's doctor gave Cheryl a referral to see a pediatric neurologist at Johns Hopkins University School of Medicine. At the appointment, Cheryl says, the doctor gave Jason an exam and then asked her and her husband how soon they could get on an aeroplane. The doctor explained there was a clinical trial getting started for a drug that might be able to stop the symptoms of SMA from ever starting, and he said Jace would be a perfect candidate since he was still presymptomatic, but there were only twenty five spots open. They'd need to act fast and get to one of the research sites in a matter of days. Cheryl and her husband left the appointment completely shocked.
00:02:44
Speaker 1: Just feeling like we had received a gift a lake instead of walking out there with with options alike. You know, when do you want Haspers to come out.
00:02:54
Speaker 3: And visit you?
00:02:55
Speaker 1: That kind of thing we were talking about him living.
00:03:01
Speaker 2: They flew from Baltimore to Chicago later that week, where Jace was screened for the clinical trial and selected. Jace was given four doses of new Sinerson over the course of a few weeks. Cheryl learned that he'd need another dose every four months, probably for the rest of his life. By the time Jace was one and a half in December twenty sixteen, he still wasn't showing any SMA symptoms. Cheryl was overjoyed. Then, two days before Christmas, the rest of the SMA community rejoiced too. New Sinerson was approved by the FDA. That meant the drug treatment would become available to more people, even if they weren't enrolled in a clinical trial, But one week later, the joy turned to outrage. Biogen, the drug company that manufactures the drug, issued an announcement of its own. The drugs trade name would be spin Raza, they said, and it would cost one hundred and twenty five thousand dollars per dose.
00:04:08
Speaker 1: I mean, now I pay anything, But then how can I pay anything? Like, how do I pay one hundred and twenty five thousand dollars a dose? Just get that out three times a year? That's impossible.
00:04:22
Speaker 2: At the time, Cheryl considered herself fortunate. Jase's costs was still being covered by the clinical trial, and the clinical trial kept getting extended and extended, but eventually it came to an end. Earlier this year when Jace turned.
00:04:37
Speaker 1: Eight, and I did have those thoughts like, oh my goodness, now what Like I need to get on the ball and start researching. Maybe there are other studies we could be in. I need to talk to our insurance and find out if they'll cover this drug and that kind of thing. And I just started feeling overwhelmed, like I'm not that sad, I'm not that organized. How am I ever going to do all of this?
00:05:07
Speaker 2: I'm Laurena Rora Hutchinson. I'm the director of the Ideas Lab at the Johns Hopkins Berman Institute of Bioethics. In today's episode, Miracle Drugs, Modern medicine has been on the cusp of a revolution for years. There's a growing list of drugs that can save lives and cure diseases like never before. But the price for these medications can reach six and even seven figures, raising high stakes ethical questions about access and regulation from Pushkin Industries and the Johns Hopkins Berman Institute of Bioethics. This is playing God. Jays got his first commercial dose of Spinraza this year, and Cheryl's insurance company covered the cost, but that's not how it works for all families. It can be a real issue getting insurance companies to cover high cost drugs like spinraza. Even when they do, there's no guarantee coverage will last.
00:06:13
Speaker 4: The best day of my career was December twenty third, twenty sixteen, when the drug was licensed by the FDA. The worst day of my career was December twenty seventh, twenty sixteen, when the price was announced.
00:06:27
Speaker 2: Tom Crawford is a pediatric neurologist at Johns Hopkins University School of Medicine. He's been caring for children with spinal muscular atrophy for over thirty years, and he's very familiar with the challenges his patient's face. It was Tom who told Cheryls she should try to get jase enrolled in the trial.
00:06:46
Speaker 4: I couldn't you know. I was over the top. Oh my god, we have this therapy. It's going to work. And then four days later, one hundred and twenty five thousand dollars a dose, six doses for the first year of life, three doses every year thereafter. Oh my god, none of our patients are ever ever going to be able to pay for it. It's been snatched away from me in all my patients. Then I learned that the drug companies were really, really smart. They realized that that no insurance company is going to let a baby die for one hundred and twenty five thousand dollars, and they would approve the authorization. And so what the new barrier is now not just the price, but the authorization's getting the insurance companies to realize that this child is indeed eligible and falls into the criteria that were used to prove that it works. But now there's a whole game going on because insurance companies will try to not authorize if possible, if the child's not exactly in the group of patients than which the experiment was on, they'll say it's an unproven therapy, and frankly, they're right, because we proved it on a very narrow group of kids that in which the benefit could be seen as quickly as possible, because we wanted to get the drug out, And so the shift in getting drugs to patients that would make a difference to them has now shifted from science to clinical authorizations and letters and pleadings and newspaper articles. This is a different landscape than I would have ever imagined.
00:08:18
Speaker 2: So I'd like to just zoom out here and cover some of the basics. For people that don't really know anything about SMA, could you just tell us what sm is.
00:08:27
Speaker 4: Spinal muskro atrophy is a rare disease, but it's more common than people realize. It's actually, before these new therapies, was the most common genetic cause of death and infants that would kill about one in ten thousand babies. It affects all children of all races around the world with roughly equal prevalence. It's a disease that affects essentially the motor system, the muscles, and so babies who are born with it look normal initially for the most part, and then somewhere around two three four months of age, they start to get weaker and weaker and weaker, to the point where the muscles of breathing no longer work. As near as we can tell, they are completely normal cognitively, and it's a genetic disease, but it's a recessive genetic disease, so when it happens, they don't have a family history. For the most part, it's a surprise, and it comes because both parents were carriers for it. The other thing that's kind of interesting about SMA is that it exists for some complicated genetic reasons across the spectrum, so that there are the babies two thirds of them are the babies that are going to succumb by twelve or fifteen months of age, but the other third are milder forms that show up a little bit later and are milder, and at the mildest end you have adults who are troubled by the fact that they can't go upstairs as well as they would want to otherwise. So it's an absolutely continuous spectrum of decreasing instance to milder and milder expression.
00:09:58
Speaker 2: So could you tell me about y connection to Jace and Cheryl Yoda.
00:10:02
Speaker 4: Jace and his mom are my friends now. I first met Cheryl and jeremy mom and dad when I cared for the first baby, Ariel, and I have over here a picture of Ariel. She lived to be fifteen months of age, and we did the best possible. A few years later, Jace was born and so they brought him to me to see if this is what he had or not. It was right around that time that we were opening up a clinical trial of one of the new therapies called Spinoza now, and he was my first patient. So he got the dose, and I have a picture here as well. I'm looking at the most amazing day of my career. He was four months old and it was clear that he was getting stronger, not weaker, and it was one of the most goosebump moments of my entire life, like, we have a cure for a disease that used to kill babies, And so Jace over the years has gotten stronger and stronger, and it's still for me one of these goosebump moments to interact with him because he's alive. Oh my god.
00:11:05
Speaker 2: Yeah.
00:11:06
Speaker 4: I was taking care of babies that lose and now after this therapy, I get to be the person who helps them not And no one could ever imagine a career that goes from that to where we are now. It's a feeling, an unbelieval ecstasy that we can We don't have to do that anymore. I'm not burying babies.
00:11:31
Speaker 1: Wow.
00:11:32
Speaker 2: What an incredible shift. So I understand your research on how SMA works was crucial for developing the miracle drugs bin Rauser, as well as other drugs for SMA, and you were instrumental in designing the clinical trial to test spinraser. Could you tell me more about that.
00:11:49
Speaker 4: Yeah. So I had the privilege of being carrying for kids with SMA from the very beginning. I was very much involved in designing the clinical trials and the outcome measures necess to do the clinical trials. And then when we had this first idea with spin raza, I was the lead investigator here at Hopkins to give the drug and then follow how well, they do and see if there's any complications of it, and we've since studied quite a number beyond that to show that it has a broader effect and its benefit for this and the other drugs for SME.
00:12:22
Speaker 2: So, can you describe how drugs like spinraza work.
00:12:25
Speaker 4: Spin Rasa and the other therapies are high end therapies that are really designer for a very very specific mutation in a specific genetic defect, and as such a high effect therapy for rare disease.
00:12:41
Speaker 2: What sort of diseases can these therapies treat?
00:12:44
Speaker 4: Well? Right now they're targeting these mostly rare disorders, but we can imagine targeting many of the named diseases like cystic fibrosis or sickle cell disease or hemophilia. There's also the promise that we can extend this to extremely rare diseases, things that affect only a few people in the world at a time. And so when we know the mutation, there's the future prospect that we can design a drug for that mutation in that patient, which is just abtolutely awesome. It used to be the idea that we had to understand why a disease happened and now it looks like we have therapiest where we can skip over that step and repair or replace the specific abdamality at the very start by supplying something that was missing or undermining something that's doing damage.
00:13:34
Speaker 2: So one of the things we've heard is that drugs like spin rasa are incredibly expensive, and that part of what seems to drive the price of these miracle drugs is how much benefit they provide to the patient. But what happens to the price when for some patients the benefit is so much more than it is for other patients. So, for example, patients who have milder forms of SMA, the benefit might be less in comparison to patients who born with the more severe forms.
00:14:01
Speaker 4: This is the amazing quandary, and spinal must Gratupy shows this more than anything, because the price is sort of based upon the fact that a baby is going to live and have a full and impactful life. And what's that worth. Obviously it's worth everything. It's interesting that patients say, I'm not getting better. I'm not like that kid, the baby that got much better. I said, no, the baby's getting better because we stopped degeneration at a time when babies normally climb out of their mom's arms and run around and then and develop and do normally. So we allow that normal development to happen, But what's really happened is we stopped degeneration. And so if we wait to give the drug until after development has already accrued, they actually are not getting better. They're just not declining any further. And that's another place where the quandary between the insurance providers and the doctors and the patients are in clash because evidence is really hard to come by. Spinal weskiatvie is going to be the story of many of the diseases to come. So I think we can learn a lot from what's happened so far, and this tension is going to be a big issue in the next couple decades.
00:15:19
Speaker 2: Tom, thank you so much for joining us today and talking about this topic. Coming up after the break, we'll hear more about how these miracle drugs are priced and why accessing them is so difficult. Cheryl Yoda's experience of getting insurance to cover the cost of her son Jas's medicine is hardly typical. Holly Fernander's Lynch is a professor of medical Ethics and Law at the University of Pennsylvania. She focuses on access to investigational medicines, food and drug administration, policy, and gatekeeping in healthcare.
00:16:03
Speaker 3: I think equity is a huge challenge in this context. So do you have insurance in the first place? How high quality is the insurance coverage that you have access to. But there's a lot of important data showing that even people who have good insurance coverage are often unable to access these high priced medicines because their out of pocket costs are still so high. It's very few people who can pay tens of thousands of dollars for a drug, let alone, you know, the out of pocket costs for a million dollar or two million dollar drug. So equity is going to be a huge issue too.
00:16:41
Speaker 2: The price point for spinras that seems shockingly high to me. How do drug companies arrive at such high prices in the first place.
00:16:51
Speaker 3: So the way that you typically hear companies talking about this is based on the research and development costs, what it takes to actually bring these products to market. And in that context, they're not thinking just about the drug that ultimately got approved, but all the other drugs that failed along the way before they got to human trials or in earlier stages of clinical development, So that's certainly part of it. However, there has been quite a bit of research to demonstrate that it's not the R and D prices that are dictating price that's actually set for these drugs. It's market conditions. It's how much will the market bear. What ultimately happens is that pharmaceutical companies get to decide what the list price is going to be for their drug. They come onto the market with very high prices, and then payers have very little choice but to pay those prices.
00:17:47
Speaker 2: What about relative benefit, Well, the value of a drug. Does that get taken into account when setting the price.
00:17:54
Speaker 3: So value based pricing is really important. What impact are these drugs having on people's lives? In many cases, the drug price has no bearing on that. And that is where we really run into ethical challenges because if a drug is not very good, right, maybe it has a marginal benefit, then we can say, look, we shouldn't be paying high prices for that drug. But if a drug is a cure for a life threatening disease, especially a disease affecting children, the value is going to be really high right, sometimes in the realm of millions of dollars, and you might even say that those are cost effective drugs because it's going to reduce the lifetime costs that you might otherwise have to pay. So that's where those are some of the most ethically challenging issues because their cost effective, high value drugs. But if we had to pay for them for a lot of patients, we won't have enough money or resources to do that.
00:18:51
Speaker 2: So we had earlier in the episode that the price of spin Rouser was announced just after the drug was approved by the FDA. Does the FDA approval process have anything to do with the drug pricing?
00:19:02
Speaker 3: They are completely disconnected. When FDA approves a drug, it is evaluating only the safety and effectiveness of that drug. It has nothing to do with price. It doesn't even know what the price is because the price may not be publicly announced. Yet even if it did know, it could not take that into consideration. Once FDA approves a product, then we get into questions about drug pricing and coverage, and there's some debate about whether that's the right approach.
00:19:36
Speaker 2: So could you talk a bit more about that debate? What concerns does this rise from an ethics perspective.
00:19:41
Speaker 3: Yeah, the debate largely has to do with whose responsibility is it to consider price? And FDA is viewed as a science based agency. Their expertise is evaluating clinical trial data and evaluating is this drug strong enough that the statutory standard has been met? Can we feel confident that patients taking this drug the risk benefit ratio is going to be reasonable for them. The product then goes on the market, and if patients want to take it, then they need to find some way of paying for it. Many people will go to private insurance coverage to pay Lots of people are covered through government programs. Sometimes what you see happening is for these very high priced drugs, even if people have insurance coverage, they are crowd funding to pay for the out of pocket costs. And that's when you get people, you know, kind of pulling their hair out, like, how could it be in this highly privileged nation, we're asking people to go on the internet and ask their friends and family for money to pay for maybe life saving products, but certainly life affecting products.
00:20:53
Speaker 2: So can any federal agency set limits on drug pricing?
00:20:57
Speaker 3: There are lots of federal payers that are involved in coverage. So we have Medicare, Medicaid, and then the Veterans Administration, and they all are a bit different in what their statutory authority allows them to do. So you may have heard about the Inflation Reduction Act. It's a huge piece of legislation that has lots of components to it, two of which have to do with drug pricing. So for a very long time, Medicare was not allowed to negotiate price. So FDA would say this drug is safe and effective. Then Medicare had a decision to make is this drug reasonable and necessary for the Medicare population. So Medicare can say it's not reasonable and necessary or it is. They have an on off switch, are we going to cover it or not? But they can't consider cost or cost effectiveness, so they couldn't negotiate for prices. Under the Inflation Reduction Act, for the first time, Medicare has the ability to start negotiating prices on a very limited set of drugs. To start off with, do you basically have nine to thirteen years as a drug company to profit as much as you want before Medicare could negotiate the price. There are some other exclusions for drugs, for rare diseases, for example. But this is a big development. Medicare will negotiate prices on those ten drugs, and then every year thereafter additional drugs will kind of get added to the negotiation list.
00:22:30
Speaker 2: And so we hear these stories about people that have to fight their insurance companies in the US to get expensive treatment covered, and there's some varying outcomes. Is there a rhymal reason to this when the decisions are made?
00:22:45
Speaker 3: So, what insurance companies are typically doing is evaluating whether a drug is medically necessary, and that might be a little bit different than the broad label or indication for which FDA approves a drug. So sometimes FDA will approve a drug for a very narrow population children up to age two, for example. Sometimes though FDA gives a very broad indication, you know, people with early stage dementia or Alzheimer's disease, for example. And so part of what the insurance company has to do is evaluate is there some more narrow population for which this FDA approved drug is going to truly be medically necessary. Sometimes insurers will say you need to have prior authorization, so we want your doctor to talk to our insurance company doctors to make sure that you can access the drug. Sometimes they will make you try other drugs first, but in many cases, the drugs that are getting approved are the only thing for the disease or condition in question. It's this or nothing. And so sometimes we do see insurance company denying access to patients because they say it's not medically necessary, and then you get into appeals where you know the patient's physician will go to the company and say no, it really is medically necessary. But of course that takes time and resources to go back and forth. It is important for insurers to be able to carefully evaluate should we be paying this price for this drug for this patient, because if they don't carefully analyze that, they're going to run out of resources for their pool. The question is, you know, how far do they need to go in that regard.
00:24:35
Speaker 2: So you've been talking about the US so far. How are these issues addressed outside the US and what different essical issues to these approaches raise.
00:24:46
Speaker 3: Yeah, there's two big differences between the US and elsewhere. In the US, we have private health insurance system that runs alongside public health insurance, right, so a lot of Americans get their coverage through their owls lawyers, and we have lots of different insurers. Outside the US, right we see more typically national health systems and national health insurance provided by the government to the citizens. And then you have the government decide is this drug worth paying for and how much are we going to pay for it? And there's pros and cons to that. So in the US, once a FDA approves a drug, if you can find a way to pay for it, there's no waiting. You could gain access to it the very next day. Elsewhere there can be a pretty substantial lag in between regulatory approval for the drug and when patients are going to access it because the government has to decide whether it's going to pay. But the pro is that it is a wiser use of resources because then the government is negotiating with a company, here's how much we think your product is worth. It's a stronger stewardship of healthcare resources for the government to say this is what this product is worth, and this is all we're going on to be willing to pay. And that's really important because we don't have unlimited resources in any capacity, let alone unlimited money to pay for medicines.
00:26:11
Speaker 2: Could you tell us how big an issue you think this is likely to be in the coming years.
00:26:17
Speaker 3: So you know, if it's spinal muscular atrophy, it's affecting deeply affecting, but affecting us proportionately small part of the population, and so you can find ways to cover very high priced products because it's spread over a broader population that's paying. But once you start to have drugs that are very effective for terrible diseases that affect a much wider part of the population, you're not going to be able to fudge your way through it the way you can with rare disease. And then we're really going to have to face the music to say these drugs are important, they're cost effective, but they're hugely expensive. Are we going to pay for this or are we going to pay for something else that's important to a functional society?
00:27:05
Speaker 2: Yeah, I mean, how do you think we should navigate these kind of questions?
00:27:09
Speaker 3: This is what ethicists work on all the time. These are career spanning ethical challenges, trying to come up with the best arguments for one approach or another. There's not a clear cut response to say this is the right way to spend our money, this is the wrong way in the US at least, this has been politicized in a really terrible way. The minute you start talking about not covering things, there are claims of government death panels that are going to put a price on life, and who wants to live in a world like that. On the other hand, that completely fails to recognize that we have to make judgments about how we're going to spend our resources, and it's reasonable to ask, even if it's life saving, how much does something cost? Can we afford it? And what's the trade off? Very often, if something's life saving, people are going to be willing to accept that trade off. Right the community is going to be willing to accept that trade off. But we can't stick our head in the sand and pretend like we have unlimited resources because we don't.
00:28:21
Speaker 2: Cheryl Yoda is beyond grateful that a drug was developed in time to save her son Jayce's life, especially when so many other less rare diseases need funding to But does that mean she thinks Spinaza's price is fair? She says, the answer isn't so simple.
00:28:38
Speaker 1: What is a good price, or like, what would be a reasonable cost? I have no idea, like I don't feel like I know. I mean, that's the whole question.
00:28:48
Speaker 2: She says, She's been so fortunate already that it would feel almost selfish to hope that the drug's price would come down to her. Calling Spinaza a miracle drug isn't an exaggeration, but still she can't help but dream of a day when it's more easily accessible.
00:29:04
Speaker 1: I know that the science that goes into making it is still pretty complicated, but the more they know about it and the longer they're making it, they get better at it or easier, and it wouldn't have to be so expensive. It's a good dream.
00:29:28
Speaker 2: Next time on Playing God, we look into the future to learn more about a technology that would create human babies without starting with sperm or eggs. It's a long way off, but researchers and biotech companies are already at work or making this possible. If they succeed, it would open up a whole world of new options for how humans procreate. But how can we be sure this technology is introduced safely and ethically. That's next time on Playing God. Thank you to our guests Cheryl Yoder, Holly Fernandez Lynch, and Tom Crawford. Playing God is a co production of Pushkin Industries and the Berman Institute of Bioethics at Johns Hopkins University. Emily Vaughan is our lead producer. This episode was also produced by Sophie Crane and Lucy Sullivan. Our editors are Karen Shakergee and Kate Parkinson Morgan. Mixing by Samir Sengupta, the music by Echo Mountain, Engineering support from Sarah Brugare and Amanda ka Wang. Show art by Sean Carney, fact checking by David jar and Arthur Gompertz. Our executive producer is Justine Lang at the Johns Hopkins Berman Institute of Bioethics. Our executive producers are Jeffrey Kahan and Anna Mastriani, working with Emilia Hood. Specials thanks to Jared Whyland. Funding provided by the Greenwall Foundation. I'm Laurena Rura Hutchinson. Come back next week for more Playing God. Has this show inspired you? Are you interested in studying bioethics? Perhaps you want to become someone shaping this field. We have a Master of Bioethics program at the Johns Hopkins Berman Institute of Bioethics. To find out more, visit Bioethics dot Jhu dot edu. Forward slash MBE scholarships are available.