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