The Patient's Right

00:02 Laura McLinn: I’m Laura McLinn and I am… I have a lot of titles I’m trying to think about what… I’m sorry.

00:09 Landry Ayres: That’s fine.

00:10 Laura McLinn: We can just say, “I’m a mom.” How about that?

00:13 Laura McLinn: Well, how about we start with that? Why don’t you tell me about your son, Jordan?

00:17 Laura McLinn: So Jordan is my ten-​year-​old son.

00:20 Landry Ayres: Jordan McLinn was a small kid even for his age, so his parents decided to bring him to the doctor to see if everything was okay.

00:28 Laura McLinn: So, Jordan was diagnosed just a little bit before his fourth birthday and keep in mind he’s 10 now, so it’s been what, six years ago. Jordan’s journey has been a little bit different. We actually kind of accidentally stumbled upon the diagnosis, because Jordan was doing what we thought was really well, looking back on it now, we realized that he wasn’t moving as fast as his peers, he wasn’t taking the step as easily, but he was also small and we thought it had to do with that. So we actually had gone to an endocrinologist to look at the possibility of growth hormones or to see if there was a growth issue. So we literally went from one day thinking, “Ha! He might need growth hormones or” just something like that to literally almost, gosh, I mean very quickly being told by the GI doctor, actually later saying, “Have you ever heard of muscular dystrophy?” My husband and I have both been very healthy, we haven’t dealt with a lot of illness in our lives. So we actually went home thinking we are going to lose our son.

01:40 Laura McLinn: There was gonna be a three-​week waiting period on the blood test, so we prayed and just cried. We were just literally in mourning and I remember, I actually tried to make a deal with God, and I said, “I’m gonna fast, I’m not gonna eat, I’m not gonna eat any food for three weeks until this blood test comes in” and if I do that, it’s gonna come back to… It’s gonna say he doesn’t have it. And so I remember I went over two weeks that I did not eat anything and I just prayed my heart out and then I did have a close friend that came and talked to me and just kind of reminded me of what the Scripture says and just kind of like just reminded me about my faith.

02:23 Landry Ayres: The doctors, specifically thought Jordan might have a form of Muscular Dystrophy called Duchenne muscular dystrophy. Duchenne muscular dystrophy is a genetic disorder usually found in young boys. Those with Duchenne typically have problems walking, running or playing because a protein that helps muscles regenerate can’t be replaced properly. The disease starts affecting the core and extremities and as early as the teen years can lead to serious respiratory and heart complications. As soon as Jordan’s doctors suspected he might be living with Duchenne, they ordered a blood test. After three weeks of waiting, prayer and uncertainty, the doctor’s diagnosis and Laura’s fears were confirmed. Jordan had Duchenne.

03:14 Laura McLinn: And they told us there was no cure, nothing in the pipeline that would happen for Jordan in time. And to basically go home and just make the best that we can of the time that we have. So, that was pretty devastating. When we left there we went through this mourning phase because it was just so shocking and so I don’t know a little bit after that, I finally got online, started doing my research, found out there were other doctors out there outside of our local hospital. So I sought out the best Duchenne doctor on the planet, found out he was three hours from home. And so I started taking Jordan there, found out there was something in the pipeline and maybe it could even happen within Jordan’s lifetime. And so, that really began my search for just having a whole new level of hope, really. I was determined, there were no other options. I mean, that was it. That really was the only thing. I would have literally dreams that night of breaking into the drug company and getting this drug for my kid.

04:29 Laura McLinn: I mean, I don’t know, it just brought out a different side of me. I’ve always been a pretty quiet, to myself kind of person my whole life, but this really totally changed me because when it’s about your baby, and you know that the clock is ticking, you will do pretty much anything when you see that there is something that can help them. I just became determined to figure it out and make sure that he was gonna get that treatment.

04:58 Landry Ayres: At the time, the only way for Jordan to get this treatment was to be accepted into a clinical research trial as it wasn’t yet approved by the US Food and Drug Administration. However, Laura was concerned, this wouldn’t even happen because Jordan might not be selected even if he did meet the highly selective inclusion criteria.

05:20 Laura McLinn: So I have set our story out there from the beginning even in the days we were waiting for diagnosis. At the time, I chose to just use my Facebook is what I did, that’s the only social media I had at the time, so I just put it out there and realize that for us, that was the way to go. And even though social media can be good and bad, it was a way for me to share Jordan’s story and what I realized is, and even to this day, it’s opened up so many doors because so many people follow our story, and then you know, one thing leads to another, and just the networking and the resourcing that happens with that. And so I put it out there immediately and because I did that…

06:06 Laura McLinn: One of our old neighbors knew what was going on with Jordan, and he actually saw an article on Sunday morning at the Indianapolis Star that said something about Right to Try. I had never heard of it. And he called me and he said, “Hey, Laura, I know you’re trying to get a treatment for Jordan. I don’t even know if this means anything to you, but have you ever heard of this?” And so, I read it, and I was like, “I don’t know, but I’m gonna find out.”

06:31 Landry Ayres: To explain what the Right to Try is, here is Christina Sandefur, Executive Vice President of the Goldwater Institute. Christina helped advocate for the Right to Try from its infancy as an idea to the push to get legislation passed.

06:46 Christina Sandefur: Well, the Right to Try movement in simplest terms is a movement that recognizes that the right to try to save your own life is a fundamental right. In fact it’s perhaps the most fundamental right of all and that government shouldn’t stand in the way of particularly terminally-​ill patients, dying patients getting treatment that they want to try.

07:11 Landry Ayres: Essentially Right to Try allows those living with terminal illnesses, who have exhausted all of their other available options, to be able to try experimental drugs and treatments even if they haven’t been deemed effective by the FDA. After hearing this, Laura jumped into action.

07:31 Laura McLinn: And so, I immediately called the state house. I found out who the sponsor was. I asked about the activity. Keep in mind I knew nothing about politics. And they said there was getting ready to be a hearing, like in a couple of hours. And so, I got Jordan and I put his firefighter suit on him, and we made up a little sheet of paper that told our story. And within two hours, I was standing in front of a microphone asking this committee to please pass this legislation for my son. I mean, that’s how fast it happened.

08:07 Landry Ayres: Well, Laura and Jordan’s involvement with the Right to Try movement happened quickly, they didn’t know just how much work it would take to actually make changes to the drug-​approval process. There are a lot of factors that influence where you as a person have the ability and permission to choose how you manage your own health. I spoke with Jess Flanigan, the Richard L. Morrill Chair in Ethics and Democratic Values Values at the University of Richmond. She’s also the author of Pharmaceutical Freedom: Why patients have a right to self-​medicate. She discusses some of the ways we can choose how to treat ourselves and some of the ways that we can’t.

08:46 Jess Flanigan: People already self-​medicate in a lot of ways, of course. So, they go to the pharmacy and they can get an over-​the-​counter medicine or they could use home remedies, but your rights of self-​medication are limited by the government in most developed countries. People are prohibited outright from using certain drugs, so certain drugs never gained approval or they’re scheduled as recreational drugs, and they can’t be used for recreational purposes. Certain drugs are approved but you need a prescription for it, and so you need to get a permission slip from your doctor if you wanna make that kind of treatment decision for yourself. And other drugs are waiting for approval, and while they’re going through the approval process, people can’t access them until the FDA gives them a seal of approval.

09:31 Landry Ayres: At this point, it becomes important to ask why is the drug approval process like this and how did we get here?

09:40 Christina Sandefur: The FDA has a mission that has really morphed over the years. It began over 100 years ago as an agency that really empowered patients to make their own treatment choices, so it was really focused on making sure that people had all the information they needed to make choices and that drug companies were actually providing products that did what they say they do and they weren’t [10:03] ____. Now, it’s become an organization that is actually replacing the patient as the decision maker.

10:11 Landry Ayres: Over the course of the early 20th century, a series of amendments and court cases slowly expanded the FDA’s purview. But it wasn’t until 1962 that things really took off. After it was discovered, thalidomide, a drug marketed to treat morning sickness was causing physical deformities in newborns, a national uproar prompted passage of the Kefauver Harris Amendments of 1962. These legislative changes mandated drug companies prove treatments to be both safe and effective before marketing them. Prior to this, the question of efficacy, whether a drug really worked or not, was left to clinical researchers. But this shift along with a ballooning bureaucracy at the FDA slowed the timeline of the drug approval process to over a decade on average by the end of the 1970s.

11:07 Christina Sandefur: The FDA is very much of the mindset “better safe than sorry,” which of course, not every patient feels that way when they are approaching certain death. And so, the FDA is not objective. The FDA has its own mission, and it has its own objectives, and it can’t possibly know what the best decision is for a patient because that’s not a scientific question, that’s not a medical question. Ultimately, that’s a question of that individual’s preferences. Over the FDA’s history, it’s really grown into a very bureaucratic organization that really has not put patients and their needs and their desires first. And for decades, patient activists have been trying to get reform, have been trying to break down that bureaucracy so that they can make more of their own choices. There is sort of this bureaucratic inertia in Washington DC, where it’s a very easy to put more and more red tape into place, but it’s very hard to peel it back.

12:04 Landry Ayres: The FDA became rigid and risk-​averse, and it stayed that way. Advocates of the Right to Try looked to past moments to try and find methods of effectively motivating the FDA to act more efficiently and compassionately.

12:04 Christina Sandefur: The really obvious example of a successful movement that everybody knows about is in the 1980s at the head of the AIDS crisis. AIDS advocates started protesting the FDA to allow for broader access to treatments and earlier access to treatments and that was the first time we ever really saw any major FDA reform and even that reform only came because the FDA was essentially publicly shamed. People were dying.

[video playback]

12:57 Landry Ayres: In October of 1988, the AIDS activist group ACT UP hosted a protest at the Food and Drug Administration headquarters.

13:07 Video playback: Fight back! Fight AIDS!

13:08 Video playback: Protesters ring the building demanding the FDA allow the use of experimental drugs by terminally ill AIDS patients. Dozens of demonstrators were arrested when they staged sit-​ins in front of the FDA’s main entrance. Skip?

13:20 Video playback: Don the demonstrators are angry at the FDA for what they say is the agency’s criminally inadequate response to the AIDS crisis. They ringed the FDA headquarters forcing police to turn away most of the agency’s 5000 employees when they arrived for work.

13:37 Video playback: FDA don’t delay, 52 will die today!

13:41 Video playback: 52 people are dying of AIDS today in this country, and they’re holding on to drugs that are being released in other countries.

[background conversation]

13:52 Video playback: We’re here because our friends are dying and there’s 134 or so treatments that the FDA is not releasing to them.

14:00 Christina Sandefur: So you would have AIDS activist lay down on the steps of the FDA, screaming things like, “Hey, hey FDA, how many people did you kill today?” And that was a very public demonstration of how regulatory barriers caused people to die. Just given the right to try knowing that there was a lot of uncertainty about the drug was important to those patients.

14:19 Video playback: Most of the demonstrators did stay behind police barricades, but to illustrate their frustration dozens laid down in the street in front of the main entrance and were arrested by police wearing rubber gloves.

14:30 Video playback: How many more have to die? How many more have to die? How many more have to die?

14:35 Landry Ayres: A line of police officers, some wearing plastic gloves stood in front of the front doors of the building, but one demonstrator leapt atop the oning above them and was able to drop a black banner with the now famous slogan. “Silence equals death.”

14:51 Video playback: But the FDA says it must be sure that a drug is both safe and effective before it is approved for public use.

15:02 Landry Ayres: At the end of the day, 176 people were arrested, there was national news coverage, and the FDA began to include activists in streamlining the drug approval process. At that time AIDS was a death sentence. While the specific drugs these people fought for proved to have mixed results, the seized control of the FDA demonstration was a crucial turning point in making clear to the FDA their slow process was costing people their lives.

15:32 Christina Sandefur: And the FDA was shamed into taking a step, but it was really only political pressure that changed a lot, it did not change the mindset of the FDA, and that’s really the concern, is that you can have some very good people at the FDA. I think that the people that work at the FDA believe in their mission, they believe in their job and they think they’re doing good, they think they’re keeping people safe and keeping people safe from their own decisions. And although the FDA has not gotten in the way of right to try and now that it’s federal law, I don’t really see any big pushes from within the agency to expand on that concept, and to broaden right to try for more people.

16:19 Jess Flanigan: I don’t think that anybody involved in the system is malicious. I don’t think that regulators want to hurt people. I think the people who work for FDA genuinely see their role as protecting people’s health and safety. But if you look at the way that the incentives are lined up, say that there’s a huge drug disaster. So a drug that was approved by FDA turns to be very dangerous and a lot of people will suffer or die from it. Then citizens and also the representatives are mad at the FDA. And FDA’s regulatory mandate rests on congressional approval, and so FDA has incentive to anticipate congressional backlash in these ways. So they have those types of reasons, and also just humanly. People don’t wanna be on the hook for something that looks like they caused it to happen. So they had a lot of incentives to be excessively cautious to try to avoid these big drug disasters. So I think that part of it’s just incentives, and how they think of their role and what their responsibility is, they would think like, “Oh, I feel so bad that we approved the drug that ended up to be dangerous,” but it’s not as salient to them, the people who suffer because of the delay. So I think that’s probably, well intentioned people just set up in this bad system leads to this tragic loss of life, through the approval process.

17:37 Landry Ayres: All of these incentives made it so that, without the right to try it was unlikely Jordan would be able to receive this experimental treatment, even if it could save his life. So once Laura got started, nothing was going to stop her from getting this law passed.

17:57 Laura McLinn: So we started fighting for the right to try for Jordan honestly, that’s what started our journey and I had hoped that that might be a pathway, a different pathway outside of the clinical trial where Jordan might be able to access a treatment. We advocated for, right to try in our home state of Indiana when Mike Pence was our governor at the time, that was in 2015. It passed here with unanimous, bipartisan support, and Jordan actually got invited to be there at the signing ceremony with Governor Pence and our whole family was there, and a lot of firefighters were there. Jordan had been named an honorary firefighter for the Indianapolis Fire Department, he had his own fire suit and he had a locker and like a job assignment, so some of his firefighter family was there.

18:46 Landry Ayres: In the midst of all of this, something amazing happened. Jordan was accepted into a clinical trial. He would no longer need the right to try to access the experimental treatment that could potentially save his life. For Jordan specifically there was no need for Laura to continue this fight but that didn’t stop their attempts to get right to try legislation passed.

19:12 Laura McLinn: Fast forward just a little bit and Jordan got invited to be a guest at President Obama’s final State of the Union address. And he was invited because of the work with Right to Try, and he was invited by an Indiana Congressman, Marlin Stutzman. So, he invited us out. It was the first time we had ever been to Washington DC. I didn’t know anything about politics, and I mean anything. I didn’t even know who my own senator was. But that night at the State of the Union, we actually met one of our Indiana senators Joe Donnelly. And during that trip, we also met with Todd Young who at the time served in the House of Representatives and is now our other Indiana Senator. But anyway, that night at the State of the Union and throughout that trip, we made a lot of connections. So that began our journey of continuing the fight for Right to Try, but this time, at the federal level.

20:13 Laura McLinn: And then, somewhere along the way, Jordan kind of became one of the national faces of this movement. Jordan and I, probably over the last two or three years, have made, I don’t even know, maybe 30 trips to Washington DC together. We’ve testified at hearings. We’ve had private meetings with members of Congress on both sides of the aisle. We met with our senator, Joe Donnelly and asked him personally if he would please sponsor this legislation, and he actually ended up becoming a co-​sponsor, making it bipartisan in the Senate which was amazing. And so, long story short, we did get it done, but it was quite a journey, and Jordan was very, very involved in the process and it was pretty amazing. He even cast a vote on the House Floor at one of the votes, like the yes vote, which was very awesome to watch.

21:09 Christina Sandefur: Bills were introduced in Congress and President Trump signed the federal Right to Try Act into law, making Right to Try the law of the land for patients across the country.

21:21 Laura McLinn: And of course, Jordan was there. The federal law actually bears his name along with three others. And then, one of the others on the law, Matt Bellina, who we got really close with him and his family throughout the journey.

21:35 Landry Ayres: Matt Bellina is another important figure in the Right to Try movement.

21:40 Christina Sandefur: He is a former navy pilot. He’s a father of three. He’s in his mid-​30s. He spent his career fighting for the rights of Americans in the [21:50] ____, and then he was diagnosed with ALS in 2014. Progressive nerve cell disease, and he was in one of those situations that is also common for people diagnosed with terminal illnesses. He had basically progressed too far for one of the FDA-​approved treatments that were out there and not far enough for the others. So, he was kind of in this middle-​ground situation, and he was getting worse and he wasn’t able to access treatments. The treatments that were out there that could help him had not been approved yet by the FDA, and he couldn’t qualify for clinical trials. This is another problem that the clinical trial system is the way that we test and approve drugs and medical treatments.

22:35 Christina Sandefur: Of course, it’s a rigorous and important scientific process, but because it’s a rigorous and important scientific process only certain people qualify. The vast number of terminally patients cannot qualify for clinical trials. And again, they’re left without being able to access this treatment. So, Matt Bellina, although he continued to get sicker, devoted that part of his life advocating for the Right to Try. He knew that it might be too late for him, but he wanted others to have that option. Matt was actually on stage with the president last year when Right to Try was signed into federal law. And at that point, he was wheelchair bound. He was barely speaking. He was unable to sleep at night without a breathing machine, and he was doing quite poorly.

23:19 Landry Ayres: The Right to Try was particularly huge for Matt, as unlike Jordan he actually received an experimental treatment under the legislation, not just from a standard clinical trial.

23:31 Christina Sandefur: After Right to Try passed, Matt was able to get access to a very promising treatment for ALS that’s in the final phases of FDA approval, but again not yet FDA approved.

23:42 Landry Ayres: Matt declined to be interviewed on tape, but he did write to me several times to share his experience. Matt wrote to me in an email. “On the morning of December 27, 2018, American Airlines Flight 1776 flew from Boston to Philadelphia with a small temperature controlled box containing my own personal stem cell treatment.” 10 days later Matt wrote, he felt the overwhelming urge to stand up. He says since then he’s had three more injections and has regained the ability to stand on his own without assistance. His lung capacity is 37% higher than it was, so he no longer needs a breathing machine. He’s gotten enough mobility in his arms to scratch his face and even take his glasses off. All of these are improvements from where he was before the treatment.

24:49 Christina Sandefur: The effect has been incredible. Matt is now able to not only stand up himself but pull himself up to standing with his upper arm strength. He can lift weights. His speech has improved so much. He can speak to his children, and he can sleep at night without a breathing machine. It’s an incredible progress. And Matt knows that we don’t know what the future will bring. We don’t know how long that treatment will last, but it has given him a gift, in his own words, that he just thought was impossible and that would have been impossible without Right to Try. It’s impossible to measure what it means that he’s now able to have conversations with his children again, that he’s able to stand up on his own. And again, that he was able to make that choice for himself to give it one last shot and to not have to beg the government for permission to try to save his own life.

25:11 Landry Ayres: After all of this, Matt didn’t stop. His fight continued long past Right to Try and into advocating treatment for all terminal patients and reforming the clinical trial process, so others who had not gained access to treatments like his would also have a chance. In August of 2019, Matt sent me a message announcing that unless the FDA published their guidance document by September 30th of that year, he would refuse all forms of nutrition which would effectively be a death sentence due to the nature of his disease. He ended the email saying, “It is entirely up to the FDA whether I live or die. And I pray you never find yourself in the same situation.” This drastic step was very much on purpose, to gain attention just like ACT-UP activists had done over three decades prior. Matt put his life on the line to make the image of those dying all the more vivid and real. Jess Flanigan spoke to me about why this sometimes seems like the only way.

26:54 Jess Flanigan: People suffer and die from their diseases because a drug was waiting for approval, it doesn’t look like they were killed based on regulatory delay, even if they were. What it looks like is that they died from their diseases, they died from cancer, they died from LS, something like that. And so it’s not as vivid when we see the victims of regulatory delay. That’s especially true for the people who die because regulatory barriers prevented innovation. So there’s new drugs that could have been created or invented that we’ll never see, having never even heard of, and we’ll never know who the victims of that are because we don’t even know what drugs could have been created had we had a less burdensome regulatory system.

27:34 Christina Sandefur: This comes down to the right to choose, this comes down to the right to pursue happiness which is something that is so fundamentally embodied in our country’s founding. The right to life, liberty, and pursue happiness, and that is what right to try is all about.

28:00 Landry Ayres: Several days after receiving Matt’s email, he posted to Facebook that he had received a message from the Public Affairs Office of the FDA commissioner, and that they would be publishing the guidance document by the end of September, which they did. He and the other members of No More Excuses celebrated this as a victory, claiming the FDA finally felt compelled to move more quickly at the tip of a bayonet. And while many are concerned with the larger aspects of medical paternalism, it was important to remember for those fighting for right to try on a personal level, even if it’s not by any means a cure-​all it has very real impacts on their day-​to-​day lives. You speak a lot and it’s very encouraging to hear you, you mentioned the idea of hope, so often in this conversation. And some people who are critics of Right to Try they’ve stated that it gives people and those seeking these treatments, a false sense of hope because it doesn’t actually guarantee them access to these drugs. Can you tell me what you might say in response to that about your experience with hope?

29:16 Laura McLinn: Well, first of all hope is never a guarantee of anything. Hope is just something that you can wrap your heart around and just not lose sight of what’s possible. And so I think it’s ridiculous when people try to say that something like Right to Try offers false hope. I don’t believe in false hope, I don’t even think it’s a thing. I think it’s the most ridiculous pairing of two words I’ve ever heard in my life. So what I would say to these critics now, and there were some, there were actually congressman who stood up and actually said, “This provides false hope, I’m not gonna support this.” I would love for them to look Matt Bellina in the eye today and dare to use those words, false hope. I just don’t believe in it. I don’t think it should even be a thing that should be allowed to be said. Life-​saving treatments shouldn’t be kept from patients, if there’s a way for patients to access them. And we do have different ways for patients to access investigational treatment. We do have clinical trials, we do have the Compassionate Youth program, but it’s just not enough because there are still so many patients not able to access the treatment. And the science is moving so fast, but the government and the FDA haven’t been able to keep up with the speed of science.

30:40 Laura McLinn: We never would have imagined even five years ago, that Jordan would even be in a clinical trial, and now we have more than 40 clinical trials available for patients with Duchenne. And that’s just Duchenne. We have Jin therapy right around the corner. We are seeing the science move at just a breath-​taking pace right now. And so for patients… It’s one thing when you don’t have hope because nothing exists, but when something does exist and you can’t have it, and you don’t even have the hope to be able to have that, that’s so cruel, and it’s devastating. It’s a whole different kind of mourning in my opinion, when you’re thinking about that. So the Right to Try has just opened another unique pathway for patients to be able to access treatment. And I think the people have spoken, I think they spoke as this Western nation at the state level, and I think the people spoke again when it passed again at the federal level. So, it’s what people want and it’s what patients deserve.

31:53 Landry Ayres: Thanks for listening to the Pursuit. If you like the Pursuit, please rate and subscribe to us on Apple Podcasts, Spotify, or wherever you get your podcasts. The Pursuit is a project of lib​er​tar​i​an​ism​.org and the Cato Institute, music by Cellophane Sam. If you’d like to learn more about Libertarianism, visit us on the web at lib​er​tar​i​an​ism​.org.