Katelyn Jetelina: A Visit from Your Local Epidemiologist

Harlan Krumholz: Welcome to Health & Veritas. I’m Harlan Krumholz.‌

Howard Forman: And I’m Howie Forman. We’re physicians and professors at Yale University, and we’re trying to get closer to the truth about health and healthcare. Our guest today is Professor Katelyn Jetelina, but first we like to check in on current hot topics in health and healthcare. And what do you have today, Harlan?‌

Harlan Krumholz: Oh, I got a couple quick things, Howie, but since we have Katelyn Jetelina on, I want to make sure that we don’t spend too much time talking on this segment and we’ll give her a big platform.‌

Howard Forman: Yeah, I’m so looking forward to it.‌

Harlan Krumholz: So first thing, you may have noticed that Elevance, one of the major insurers, reported out their Q2 earnings last week, and I don’t know if you saw, but the headline was that it reduced its diluted earnings per share expectations by 13% for 2025. Mostly a result of the challenges in the ACA and Medicaid markets, and their stock dropped as a result 18%. But it’s going to be interesting to see whether this is a harbinger of what you’re going to hear from the insurance companies as we go forward. But one of the things that interested me about it—that actually wasn’t the reason that they dropped so much, but it still caught my attention, was that they noted on the earnings call that it was seeing pockets of providers using—wait for it, Howie—AI to upcode and increase revenue.‌

Which really was at least a small part of its issue with the ACA was that there’s this shift now in the perception at least of the sickness of populations which are leading them to be in a weakened position. Now, this is interesting because there’s a juxtaposition between this and other things that are going on in the ecosystem. Blue Cross Blue Shield of Kansas City is in the midst of a lawsuit with AdventHealth. So Advent is suing them because Blue Cross Blue Shield is using AI tools to review Advent’s claim submissions for inappropriate codes and remove them. And so they’re basically coming out and saying that ... I’m just juxtaposing this. Elevance is saying the docs now have got these companies that are helping them upcode with AI, and then here’s a health system going after Blue Cross saying that they’re using it to deny claims using all these artificial intelligence devices and actually putting together a lawsuit for it. So this is going to be a continuing thing. We’ve talked about this before, where AI is going to be talking to AI about preauthorization and claims and denials and repeals, and it’s going to be one of these endless loops that’s going to take up a lot of—‌

Howard Forman: It’s very frustrating.‌

Harlan Krumholz: ...a lot of power.‌

Howard Forman: It’s very frustrating. And pointedly, while Elevance is down like 50% from its all-time high and all of the health plans are down considerably, Oscar health is down 40% now. What’s interesting to note though for our listeners is they are all up so much since 2003, when the Medicare Modernization Act passed. They’re up by twice as much as the S&P 500 or more, and it’s just a reminder that it is a very profitable market. And even when their profits go down, they are still enormously profitable.‌

Harlan Krumholz: Yeah, but nobody gives you credit for the history. They all want to look forward.‌

Howard Forman: Yep. That’s exactly right.‌

Harlan Krumholz: I think that’s a very good perspective. Here’s my final thing I just want to get in before we get to Katelyn, is that and then there are groups that don’t even still know exactly what AI is, and there’s debate. So you know Whoop, this wearable that—‌

Howard Forman: Oh, I don’t know it.‌

Harlan Krumholz: They advertise it with a lot of the athletes. Well, they’ve got a blood pressure monitoring feature. They also have an atrial fibrillation feature. The atrial fibrillation feature they took through the FDA and got authorized. So that actually went through as a medical device and approved. But they’re trying to make the case that the blood pressure monitoring feature is not AI, is not a medical device—well, I guess I can’t deny it’s AI, but they’re saying it’s not a medical device and they don’t need to go through the FDA. So Whoop and the FDA are kind of going back and forth about, well, what exactly does need to be regulated among all of these newfangled devices and algorithms, and when should the FDA get involved? And this is interesting because Whoop CEO was actually meeting with RFK Jr. recently as RFK Jr. was promoting the idea that everybody should have wearables. So it’s an interesting week for AI. AI being used to upcode, AI being used to deny claims, and then you’ve got this wearable company that’s saying it’s not even a medical device. “Pay no attention. It doesn’t need to be regulated.” I don’t know. A lot going on.‌

Howard Forman: It’s fascinating, though. And this may be beyond my ability to talk about, but I know that Apple actually got disapproved for, I thought their ... was it their hypertension monitor or something like they had built into their watch and then weren’t allowed to activate it because they didn’t have approval, right?‌

Harlan Krumholz: No. Well, that was about O2 saturation.‌

Howard Forman: Oh yeah, yeah. Sorry.‌

Harlan Krumholz: Because there was a lawsuit from Masimo that said that they had brought them in, said, “We might buy this feature,” and then they developed it on their own and Masimo claim that they basically just stole trade secrets from them.‌

Howard Forman: The ability of wearables to influence us is huge. The ability for them to actually positively change outcomes is uncertain.‌

Harlan Krumholz: But your point brings up another point, which is, what gets regulated, what gets protected, and how is all this going to get sorted out? We’re still in the very early phases.‌

Howard Forman: It is. It’ll be an interesting time always. Hey, so let’s get to Katelyn.‌

Harlan Krumholz: Yep.‌

Howard Forman: Dr. Katelyn Jetelina is an epidemiologist and adjunct professor at the Yale School of Public Health. She’s served as a senior scientific advisor to several government and nonprofit agencies, including the Centers for Disease Control and Prevention, and has been an active researcher on law enforcement, violence prevention, child welfare, and mental health. As the founder and CEO of the public health newsletter, Your Local Epidemiologist, she has worked to translate complex public health issues into easily digestible information for the community. This has exploded, and deservedly so, into an enormous enterprise and will be a big focus of our conversation today. Not surprisingly, last year she was named a Time 100 most influential person in health. Dr. Jetelina received her bachelor’s in physiology from the University of Arizona. She went on to earn both her master’s in public health and PhD in epidemiology and biostatistics from the University of Texas Health Science Center at Houston.‌

So first, we are so excited to welcome you to the podcast. I mean, you are somebody that I look for in my mailbox as many times a week as you’ll show up there, and I get excited for your successes. When I see how much traction you’ve had, it really fills me with joy. But partly because my goal in life is to communicate and explain things well, and nobody does this better than you.‌

Harlan Krumholz: But you did say the biggest thing, Howie, didn’t you, that she’s an adjunct professor at the Yale School of Health too?‌

Howard Forman: I said that. Absolutely.‌

Harlan Krumholz: I just want to make sure everyone knows that.‌

Howard Forman: Right from the top.‌

Harlan Krumholz: That somehow we snagged her to be part of our faculty.‌

Howard Forman: Megan is a very smart curator of talent. and it was not surprising that she found you early on and that you found her. So I really appreciate that. But I want to start off, this is now what, over a hundred thousand people get this newsletter, right?‌

Katelyn Jetelina: Yeah. Four hundred thousand.‌

Howard Forman: How many?‌

Katelyn Jetelina: Four hundred thousand.‌

Howard Forman: Oh, four hundred thousand. Like, oh my God, it’s even bigger. I mean, you have this enormous audience. Where did this idea start from? How did you first conceptualize this, and how is it going for you?‌

Katelyn Jetelina: Yeah. I will just preface by saying none of this was planned, ever. I was a faculty member. I was building a research lab. I was a professor. I was teaching students. In March of 2020, actually, my dean came to me and she’s like, “Katelyn, can you just tell our faculty, staff, and students in a daily email what the heck is going on with this new virus called COVID-19?” And so I did. I started these emails. It was like to, I don’t know, 50 people. Very small. I inserted really ugly Excel graphs. But I just spoke, like I was talking to my sister, like, what I was following in real time, what I was doing as a parent. And I actually signed those emails “Love, Your Local Epidemiologist”—because that’s who I was to them.‌

And then a few days later, one of my students came to me and she’s like, “Dr. Jetelina, can you just please put this on Facebook so I don’t have to keep copying and pasting this for my family and friends?” And I distinctly remember telling my husband, I was like, “What the hell? I only have to do this for six weeks max and then surely someone’s going to pick up the baton and actually explain in real time, in plain language, what is going on.” And I guess that never happened, and it’s gone through this incredible journey where I find myself now with this public health newsletter, a team of 15 behind it, and still growing. So yeah, it’s been, like I said, a wild ride.‌

Howard Forman: Can you just speak a little about what the team looks like? I know you have a couple of people who occasionally will author under the template of Your Local Epidemiologist, but you also have some local newsletters now as well.‌

Katelyn Jetelina: That’s right. So I am building the basic infrastructure of an enterprise. So I have a COO. Also, I have a copy editor that catches all of my grammar mistakes—or tries to, at least. A Spanish translator, social media manager. So the infrastructure there. Also incredible writers and scientific communicators as well that help write. And then you’re right, I have started state-level newsletters. I’ve started in New York, where that is growing exponentially, and actually in two weeks we’re starting a California one. And so the purpose of those is, we all know that public health is local. Local context really matters. What’s happening in New York is not happening in San Diego, for example. And so when we left the COVID emergency, particularly on the national level, I had a really hard time knowing what to talk about because not everyone was talking about the same thing. And so really trying to experiment with those state-level newsletters.‌

Harlan Krumholz: What’s so nice is, you made being an epidemiologist cool again. I don’t know. It’s kind of like for a long time epidemiologists played a central role in remarkable breakthroughs in health in so many different ways. And then I think there was a period where it wasn’t quite as visible, and now what you’ve done is really shown the value of the information and the knowledge. It’s so interesting. So many people think about, how do you get past the cold start problem when you want to build something big and you maybe didn’t have the plan of how big it would be, but you just created a great product. And I don’t mean to diminish it by calling it a “product,” but in the end you created something that people wanted.‌

Katelyn Jetelina: I was just going to say, the first thing is finding a gap. And I stumbled upon this gap of what ended up being people wanting translated information about public health that is actionable, timely, and understandable. The other thing that I think is a bit different the way I approach this than I would say even traditional public health and where I think a reason we’ve lost so much trust in public health is that I would say about 80 to 90% of my content isn’t what I as an epidemiologist think is interesting or important. It’s from questions I get from the audience. So I try to be very responsive, try to understand where the conversation is at, figure out what those information voids are and those questions are—and then answer them. I mean, one great example is the seed oils. I did the seed oils post.‌

Howard Forman: I remember that.‌

Katelyn Jetelina: A couple weeks ago. It went viral because all I did was ask, “What’s your questions about seed oils?,” and I answered them. So I think that there’s this bidirectional relationship that we need to reignite in public health.‌

Harlan Krumholz: So here’s just one follow-up on this because I’m curious how you have thought about this. So in some ways, as I see you, I see that, in my view, that you really aren’t polarizing at all. You’re informing. I’m sure there must be some people on the spectrum of political beliefs that don’t always agree with the kind of things you’re saying, but how do you navigate the politics when almost everything said in public health is seen through a lens of “whose side is this on?” and “what is someone trying to promote?” How are you able to find just the right place, that sweet spot where you’re saying, “I’m just trying to channel science”?‌

Katelyn Jetelina: Well, it’s positive feedback that that’s how it’s coming across because it is really, really, really hard to do that and especially in this place. But the reality is that public health is inherently political. The decisions, the leadership, the budgets—that’s all dependent on politics. So I don’t think that we ignore politics and say “Kumbaya, here we go.” I think my biggest goal, though, is to not turn people off that are curious. And so that means leading with a lot of empathy. It means understanding what their questions and concerns and perspectives are. So I actually started this year to try to understand where others are coming from. I listen to Joe Rogan, I read Free Press. I try to listen from these different perspectives to also help fill their information voids while talking. And so I’m glad to hear that, because my biggest goal right now, especially in this moment, which is so different than COVID, is try to keep a steady head, try not to come off like I’m screaming when you open your inbox email and try to reach people in this middle that are genuinely curious and genuinely want to help protect their own communities and themselves.‌

Howard Forman: Can you speak to the motivation and conception of the PopHIVE that you’ve worked with at the Yale School of Public Health? Because I think that connects to your newsletter, but it’s also an independent project.‌

Katelyn Jetelina: It is. So for anyone listening, PopHIVE is this data democratization project at Yale. So this is the challenge we’re trying to solve, is that data in the United States is incredibly fragmented. There’s no one data set of people’s health in the United States, like, for example, Denmark has. And that makes it really, really hard to understand trends, to understand what’s going on in the landscape to understand who is at most risk for some diseases or some health topics. And so you’re right, so Ann Zink and Megan Ranney and Dan and a few others started putting together PopHIVE, which is basically triangulating all these data sources to look at diseases, whether it’s chronic diseases or injury or infectious diseases. They looped me in, which was an incredible honor, to help translate that data, what I call data translation. How do we make it usable? How do we make it understandable? And so it’s been a very fun project and really needed in this world.‌

Howard Forman: And let me just point, we’re going to make a link in our show notes for PopHIVE, but it’s pophive.org. P-O-P-H-I-V-E.org. And it’s a Yale School of Public Health–sponsored entity, correct?‌

Katelyn Jetelina: Correct.‌

Harlan Krumholz: So another question about how you navigate your communication style. Another issue is that especially when you’re talking about public health threats, that it can seem as if the message is constantly about what you need to be fearful about. What’s around the corner, what terrible thing is about to occur, what people need to worry about. And as I’ve seen your communications, they are a balance of both realistically what can occur and what we need to be attentive to but are not alarmist, don’t actually in any way, I think, give people anxiety. It’s basically, you’re able to hit this tone that is, again, one of information without putting people in a position where they need to be scared about something. How do you think about how you’re able to straddle that: “Here’s the truth, there are things in the world that could be problematic, but you can’t live your life every day just worried about the sky falling.” So how are you able to navigate that?‌

Katelyn Jetelina: Yeah. I mean I want to take a step back and us in public health, I feel like the public only hears when there’s a really terrible thing going on. And the challenge with that is, if we’re always in an emergency, we’re never in an emergency. And the other challenge with that is, I truly think it’s a learned behavior. We live in this cycle of panic and neglect with funding. And so if it’s not panicky, we don’t get funding. And so I think that’s part of the challenge. How do I straddle it? I mean, one is just Risk Communications 101. What do you know, what do you not know, and how are you finding answers? The other thing that’s really important to me is empowering the reader of like, okay, this is great information, but what do I actually do with this? And so there’s this action there. And if there’s no action, if this is just an FYI, I try to tell them this is just an FYI or I don’t even talk about it because I’m a mom. There’s a billion things to be worried about right now in this world, and you know what, this little thing that happened in Arizona, blah blah blah, is just not worth your stress right now. And so I guess I look at it those three angles.‌

Howard Forman: I want to point out one of the things that you do and it’s today’s newsletter you even have it. It’s like you might talk about real challenges, real concerns, and then you’ll come to, “There are some silver linings here and here are things that you can do.” How do you think about ... you have a very broad audience. As you pointed out, it’s now four hundred thousand. Beyond belief. How do you reach them all at where they are so that they feel engaged as opposed to ever feeling that they might be put off?‌

Katelyn Jetelina: It’s hard. I guess my biggest answer is I don’t know. I think that if we’re writing and if we’re communicating, I always try to think about who’s reading it. Who is the audience? And at YLE, at least in the beginning, I always thought I was talking to Joe on the corner, just my sister. Isn’t trained in epidemiology, doesn’t have an M.D. but environmental science. So she kind of gets it. But when I did a survey, the first one, 77,000 people answered my survey to understand who they were and it was very clear they were not Joe on the corner. They are trusted messengers. They’re physicians, they’re academics, they’re policymakers, they’re ... did I already say physicians? Or nurses or school nurses. So I view my audience mainly as trusted messengers. And a great example I always give is, for example, a business leader. The chief financial officer of this huge international bank includes some of my updates and his stock reports to his hundred thousand employees. So what I started realizing is that I’m this node in this massive grassroots information network and how can I help empower those nodes to then go talk to their people who will then go talk to their people.‌

Harlan Krumholz: Curious, how many hours a week does it take for you to do all this content? I know you’ve got a team, but what’s it take from you?‌

Katelyn Jetelina: I would say about 50% of my time is spent really writing. You’re right. Now I’m leading a team, I’m building an enterprise different product, so it’s actually taking more time. And then I do about 50% consulting. So I guess that’s the balance.‌

Harlan Krumholz: Oh, that’s amazing. So listen, I just want to get to a couple of things. Let me just try one. Howie, you can try one, but just since we’ve got you on, might as well get your views on stuff. So what are you thinking about COVID?‌

Katelyn Jetelina: What am I thinking about COVID? My question is—‌

Harlan Krumholz: So we’ve got Nimbus, we’ve got things coming around the pike, and it’s like there are new strains. Here’s the idea. So a lot of people ... I’m just gonna give you a chance to talk to our listeners about this. A lot of people are saying now, “Yeah, it’s just another virus. It’s got the name from the legacy of what it was when it first came out, but it’s just another virus.” I mean by the way, viruses, flu, all these things cause mischief and bad things to happen to people, but that we shouldn’t be paying any extra attention to it that we pay to any other virus that’s circulating around. Or does it deserve a place for us to be monitoring it closely and worried about it going forward?‌

Katelyn Jetelina: I mean, I think for the average American that it is just another virus and that there are things to do to prevent disruptions and that when we’re in a wave, yeah, I think it’s worth putting a mask on while you’re boarding a plane because then you don’t have to get sick on your vacation. And I think it’s the same as flu and with RSV too. I think as epidemiologists, we’re for sure still paying attention. I mean this is an infant of a virus and can still have a lot of surprises up its sleeve, and I think that we can still learn a lot from watching it integrate into our society. I mean this is the first time we’ve ever actually got to really watch that.‌

Harlan Krumholz: So for example, a lot of authoritative sources are still saying “10 days of isolation.” You’re shedding for more than 10 days. You shouldn’t be out seeing people for 10 days. But almost no one I know is actually following that advice. What are you telling people?‌

Katelyn Jetelina: I’m going to get in trouble. But I mean, the same thing. If I don’t have a fever, my kids don’t have a fever and they’ve been home and they’re feeling better or slowly getting better, then I let them go to school. I mean it’s the same thing with norovirus, right? We can shed norovirus for three weeks, but no one stays home. I mean, it’s just not feasible. And so I think that there’s a balance here between feasibility and getting on with our life, and I guess that’s where I stick to it. There’s one exception—‌

Harlan Krumholz: This is, Howie, by the way, the common-sense part that I think everyone loves about her.‌

Katelyn Jetelina: I was just going to say, personally, there’s one exception, and that is my grandparents are in their mid-90s and I would keep a week or two before I go see them at the nursing home. I guess that’s my only—‌

Harlan Krumholz: Makes sense.‌

Howard Forman: Oh, my God. Yeah. I mean my parents are approaching that age, and when I think about me getting sick, the only thing that bothers me is it means I won’t see them for a while. That’s the only thing that bothers me. I want to ask you, because you said, “I’m going to get in trouble for this,” and it reminds me that in one of your other interviews you mentioned that as you became more of a public figure, people would attack you occasionally. I presume mostly just online threats and things. But I’m curious to hear what that has felt like because you are not a polarizing figure. I mean, to me at least, you’re not. If anything, you’re about as middle ground as anyone could ever be as a scientist. What has happened and how do you deal with it and process that?‌

Katelyn Jetelina: I’ll just say, it’s been ugly. Especially during COVID, it was very ugly. I mean, you’re right, it’s online haters, but people did show up at my work, and we got the FBI involved. People still show up at my house in my front yard. I mean, it’s—‌

Howard Forman: Oh my god.‌

Katelyn Jetelina: I think it’s terrible and I was surprised because I am just an epidemiologist. But it’s also what happens when you enter the arena and you get in that public-facing. I will say, though, that despite the death threats and doxing and getting hacked and everything that came around, the worst thing I will say is a thousand paper cuts from colleagues not understanding why I’m doing this, that nuance has to be dropped sometimes, that I’m not a real scientist if I’m doing this communication stuff, that they’re disappointed in me that I can’t find a job. And so I think that that actually is way bigger weight than death threats and people showing up at my house, honestly.‌

Harlan Krumholz: I want to recommend to people who are listening that you put together a really nice primer on Medicaid that sort of laid out, I thought, just very dispassionately, what is the situation, what’s going to happen with the bill, what people can actually do. And one of the I thought most important points that you were making is are we at a high-level thinking about how we’re developing a system that’s going to help us better prevent, give more access, help people be more free from debt, or are we creating a system that’s increasing those bills, debt, actually potentially can make people sicker? And it is an interesting thing for a local epidemiologist to actually then be able to come in and say, “Well, let’s just look at what we think the effects of this are going to be and isn’t really steering us in the right direction.” I don’t know. Did you get much feedback on that piece? It was really so nicely done.‌

Katelyn Jetelina: Oh, thank you. I mean, it comes down to the need. I mean, I was incredibly surprised that there was basically no media out there just explaining in plain language what the heck was going on with Medicaid. Right? Same sort of thing with COVID is that you kind of assume everyone knows, and no one knows. In fact, it actually took me a long time to actually understand what the heck was going on with this whole Medicaid stuff to translate it. But to come to your point, is right now where we find ourselves, at least how I’m viewing, is that public health and the healthcare ecosystem is very good at defending and holding the line and the resistance right now. What we’re incredibly missing, though, is providing an alternative path.‌

What do we see as a healthy world? How do we presume we want to change this? Because the status quo wasn’t good. The status quo wasn’t okay. But because we’re in this defense mode, it looks like we’re defending the status quo. And I think that in order to bring people along for the ride, we really need to think proactively of, what is this health ecosystem that we want to see? What are our values and how would we change this? And that’s where I was trying to make that parallel with Medicaid because Medicaid, to me, in an ideal utopia, it’s not making people sicker. And I think running those parallels is really important right now when we start talking about policy change.‌

Howard Forman: Can I do just really quick rapid-fire questions about just key things you’ve talked about in the newsletter that I think our audience might find interesting? If you’ll let me. Where do we stand right now with H5N1? Should we be worried or not?‌

Katelyn Jetelina: Average Americans should not be worried right now.‌

Howard Forman: Thank you. COVID right now, how is the outbreak compared to prior years at this time? Are you worried, and are there any states in the country that you’re particularly a little worried about?‌

Katelyn Jetelina: COVID is actually very low right now, but it is rising in the south and the west. Am I worried? No. It’s not going to be a tsunami, but people are going to get a bit more sick this time of year.‌

Howard Forman: And last for this segment, measles right now, are you feeling better? Are you feeling worse? Are we going to lose our eradication status?‌

Katelyn Jetelina: I think we are going to lose our eradication status, but to 93% of Americans, they’re not at risk. It’s not that big of a deal. I think it’s a bigger signal of where we’re going as a country.‌

Howard Forman: And by the way, this is what I love about you is you give people answers that they can actually understand. They don’t have to thumb through it and figure out what you mean by it. I mean, you are amazing. You’re a great communicator.‌

Harlan Krumholz: Well, let’s ask one more hard one, then. When parents are asking you for the fall about vaccinations for COVID for kids, what are you saying about that?‌

Katelyn Jetelina: We don’t know. We don’t know what the license and availability is going to be. I think we’re going to know a whole lot more in the next two, three weeks, but right now it’s a big question mark.‌

Howard Forman: Yeah. You have made a huge impact in a positive way in public health communications. I admire what you’re doing, and I hope we can have you back.‌

Harlan Krumholz: And how you’re doing it. What you’re doing and how you’re doing it. It’s incredible. Yeah.‌

Howard Forman: Thank you.‌

Harlan Krumholz: Thank you for joining us.‌

Katelyn Jetelina: Thanks for having me, guys.‌

Harlan Krumholz: Okay. Well, she’s a rock star.‌

Howard Forman: Oh my god. So happy to have her on. We got to have her back.‌

Harlan Krumholz: Oh my god, Howie, we’ve got to study her. We’ve got to figure out how did she get to four hundred thousand? I mean, we’re so close.‌

Howard Forman: We are close, but we’re not there yet, Harlan.‌

Harlan Krumholz: No, we’re not close. We’re not close.‌

Howard Forman: We’re not close.‌

Harlan Krumholz: But we’ve got a great segment every week that you do and that’s going to get us closer, I’m sure. So what’s on your mind this week?‌

Howard Forman: Yeah. I have a topic with many lessons for me and hopefully for our audience, and that is, Duchenne muscular dystrophy is a severe disease affecting mostly boys, leaving them wheelchair-bound by adolescence with a median life expectancy of roughly 30. It’s rare, with varying estimates of prevalence, but roughly one to 3,000, one to 5,000 male births in the United States. So rare, but not unheard of, and there is no cure, but there has been progress made, particularly in understanding the mutation in the dystrophin gene that causes this and has been isolated and it is being better understood over time. And now there’s multiple related gene therapies for this disease, but none of them are particularly effective, and even approval remains controversial, as you’ll hear. One of these drugs is currently in the news and has valuable lessons for us, and that is a drug ... I’m pronouncing it as “Elevidys.”‌

It has this long real name of delandistrogene moxeparvovec, and it’s a gene therapy, and it was approved by FDA senior leadership over the objections of staff. And it has shown some progress in slowing progression and seemingly could reverse the effects in the mutation of the dystrophin gene. The gene therapy is delivered by a viral vector, and it’s been approved and in use for two years now. Nine months ago, a major clinical trial was published that showed that it did not succeed in statistically improving the primary endpoint but showed some promise with secondary endpoints. Things like how you can stand up and how far you can walk. The safety profile was seen as more than acceptable with similar adverse events between the placebo and the drug arm. Although there were more events in the drug arm, none were severe.‌

However, over the last several months, two teenagers died from liver failure after being treated with the drug. And now a 51-year-old man has also died, but from a different drug, but still from a similar mechanism. This has forced the FDA to encourage Elevidys’s removal from the market, and Sarepta, the drug company, has now complied with this request. This is a sad setback for the Duchenne muscular dystrophy community that had a lot of hope in this innovation. And it is not the only time we’ve seen disappointment in this sector. I highlight this case for a few quick reasons I’ll mention. Muscular dystrophy is obviously devastating illness. Efforts to achieve some quality of life improvement are welcome. And while this looks like an uphill battle for this drug, the pharmacology itself does seem to work. The trial showed production of what’s called micro-dystrophin in the treated patients, all of whom were between the ages of four and eight years of age at onset of the trial.‌

A total of 125 people were enrolled in this trial, and the deaths are occurring in an entirely different group, much older individuals. Importantly, this treatment is a one-and-done treatment. So when you give it to a four-, five-, six-, seven- or eight-year-old, it’s not like you’re continuing to give it to them. You give it to them once. I also want to point out this drug is a multi-million-dollar drug. It is roughly $3 million for this treatment. The other big point I want to mention is this is a company that has been public for 28 years. It is well below its initial offering price and is down more than 90% from its all-time high of one year ago. Even at its all-time high, it had already underperformed the S&P 500.‌

So this is yet another example of ambitious scientists and entrepreneurs who have helped make enormous progress in understanding and treating Duchenne muscular dystrophy but have failed to capitalize on it as investors had hoped. We’ve talked about a long line of such startups, and I’m going to continue to emphasize this because I think a lot of other podcasts, news reports, social media, make it just seem so easy for life science companies to make huge windfalls, and my experience following these companies is that it is far, far from easy. I don’t know Sarepta at all—that’s the company—nor anyone who works there, but I am just humbled by their multi-decade efforts, and I certainly hope that their experiences continue to form the foundation for future success in this area.‌

Harlan Krumholz: Yeah, that’s so nice of you to call them out. And it is heroic work to try to actually get something through and make a kind of contribution, especially in a condition like this. It was heartbreaking to see that there had been these deaths and now this option is being taken away. It makes me wonder for a condition like this, what kind of risks would people be willing to take? Of course, this gets even more complicated because it’s about children.‌

Howard Forman: Exactly.‌

Harlan Krumholz: But given the expected trajectory for these individuals, even if there are risks, would some people be willing to try it?‌

Howard Forman: I know. And Harlan, to a point that you’ve raised with me, I wonder whether they made a mistake in pursuing treating teenagers when every bit of evidence they had favored this very young group. It was almost like this was a Hail Mary for the teenagers, and so they went for it. But instead what they’ve done is pointed out a potential risk in older kids that may not even be there in the younger ones.‌

Harlan Krumholz: And I know that a lot of companies, once you get experience, if it doesn’t go well, you always think of what you could have done or how you could have done it differently. But yeah, certainly they make the best decision they can and they try to proceed. So I still hope we’ll make progress against this disease.‌

Howard Forman: I do, too.‌

Harlan Krumholz: Yeah. Thanks for bringing that up. You’ve been listening to Health & Veritas with Harlan Krumholz and Howie Forman.‌

Howard Forman: So how did we do? To give us your feedback or to keep the conversation going, email us at health.veritas@yale.edu or follow us on any of social media.‌

Harlan Krumholz: And we always want to hear your feedback, any questions. Tell us about your experience with these topics. And if you liked the podcast, please rate us. Review us on your favorite app. That helps people find us.‌

Howard Forman: If you have questions about the MBA for Executives program at the Yale School of Management, reach out via email for more information or check out our website at som.yale.edu/emba.‌

Harlan Krumholz: Health & Veritas is produced at Yale School of Management, Yale School of Public Health. Thanks to our amazing superstar researchers, Tobias Liu and Gloria Beck, to our remarkable producer, Miranda Shafer, and to the best partner in the world, Howie Forman.‌

Howard Forman: Right back at all of you. We’re so fortunate.‌

Harlan Krumholz: Talk to you soon, Howie.‌

Howard Forman: Thanks very much, Harlan. Talk to you soon.‌