I know it's probably too late for me. I made my piece with that I need to know before I die that my children find themselves in this unenviable position. This nation that I probably served will respect their liberties and the right to make their own decision about their medical treatments. Thank you for having me. Matt Billina is a father of three boys and a former U. S Naval aviator in he testified at a congressional hearing to advocate for dying patients who want access to experimental drugs.
At that point, his speech was slightly slurred by his Luke Garrig's disease. It's a fatal condition with no treatment, it destroys the nerve cells and the muscles they control. Slowly, every part of the body stops working. Letting patients like Matt who have no other options use these experimental medicines seems like it should be easy, but it's not that straightforward, and the critics, the people who oppose making them widely available,
aren't always who you would expect. Welcome to Prognosis, a podcast about innovation in medicine and science. I'm your host, Michelle fay Cortes, and today we're going to explore what people do when they're desperate for a chance to try an unapproved drug. Do you want to say something? Yeah? Good? Do you want to say yes? All right? Laura McGlenn is another American who wants access, not for herself but
for her son, Jordan's. You just heard him in a clip from a few years ago, asking lawmakers to make it easier he has to shend Muscular dystrophee, a genetic disease that erodes the growing muscles of young boys and eventually hits the heart and lungs. The average life expectancy is twenty six. Jordan was diagnosed when he was almost four, and at that time he was diagnosed at our local hospital in Indianapolis, which is a pretty big, well known
children's hospital. But we left there pretty much with no hope, and the doctor told us basically, this was a fatal disease, no cure, probably nothing even coming up through the pipeline that would happen in Jordan's lifetime. It turns out the doctors who are wrong. Fast forward now, I mean Jordan's is nine and a half and I think, um, there are more than forty drugs actually in the investigational stages for his disease. So a lot's happened in such a
short time with the science. First, a little background. The easiest way to get access to an experimental drug is in a clinical trial, but many people can't participate in trials. They may be too sick or too young for them. There's something called compassionate use. It's an official program regulated by the Food and Drug Administration, but that's also difficult to navigate. There's paperwork, and you need the f d A s and the drug company's blessing, and it's possible
that drug company may not give it. There's too much risk enter right to try. This is the law that Laura McGlenn and Matt Bolina fought for. Its main difference it cuts out the f d A. Instead, the doctor directly asked the company for a drug. The patients take on all the risk and make their own decisions, but they do still need to get the drug company to go along with it. I'm not gonna lie. I mean, when I started this journey, all I could think about
was my one, my Jordan's. Jordan got lucky, he was accepted into a clinical trial, but Laura didn't give up her fight for right to try. She did it for the other boys, and because the disende community is so close, she's seeing firsthand what happens to those who can't get into trials. We want our boys to access experimental treatments through a clinical trial. Unfortunately, for example Jordan's, he's one of sixteen boys in North America that are able to
try the drug that he's trying. While I watch many of my friends who have sons who are amendable to the same drug, who can't access the drug because they don't qualify for the trial, and so their sons are basically, you know, um, continuing to progress through this disease, which is a muscle wasting disease, and they're losing muscle function and it's it's truly heartbreaking. Mike Smith, a New York attorney,
also wants better access to medicines. So now, I grew up on Long Island, New York, and the town called Sayaucet, Um basically had a up until I was fifteen, frankly had a perfectly normal and uneventful childhood. That's sort of changed on Black Monday, the day of the stock market crash on October seven. The day started with Mike scoring a goal in a soccer game during his gym class. The opposing goalie didn't take it well. He punched Mike in the chest and kicked him in the lower right side.
He immediately fell ill. It was the start of a long and harrowing health odyssey. It took five years just to get a diagnosis, and we were starting to identify that I had a disease or disorder called chronic intestinal pseudo obstruction. It is an extreme spastic disorder of the digestive track. I'd be sitting in a high school classroom, for example, and get a sensation as if my stomach was being tied into a shoel. I snot, you know, And ever since, I really have never been able to
take eating or even drink king for granted. Essentially, his digestive tract stopped working. For several years, he couldn't need at all. He relied on artificial nutrition, and then thankfully new drugs came along. He could eat again. But the two treatments were pulled from the market in the US because of potential side effects, but not in Europe. Mike
was back to square one. Now here's the thing even under right to try he might not get the drugs that he knows work for him, because technically it's only for people with the terminal diagnosis and Mike's condition. Isn't that simple? On the span of my condition at this point? Is it chronic and debilitating? Yes? Absolutely? Can we say fairly on a day to day basis? Is it life threatening? I'm not a doctor. It's not for me to determine whether or not it's life threatening. But who should make
that call? Should patients like Mike have access? Over the past five years, more than forty states passed right to Try laws. Those efforts culminated in the federal legislation. It was a rare bipartisan achievement, pushed by Vice President Mike Pence. Right to Try one support in both Houses of Congress. It was touted by President Donald Trump in his first State of the Union address. He signed it into law
in May. But this is such a great name. For the first day I heard it, It's so perfect right to try, and a lot of that trying is going to be successful. I really believe that. I really believe it. Ibraheim del Pasand is also a believer. More importantly, he's the medical director at Houston's Excel Diagnostics. He's also an expert in nuclear medicine. That means he uses radioactive materials to diagnose and treat patients. Hey, Michelle, Hi, here we go,
we're working now. Okay, very good. Doctor del Pasand is the doctor who is most publicly braced right to try. He felt he had no other option. There was a drug he wanted to give his patients, a form of radiation for neuroendocrine cancer that had been used in Europe for years, but it wasn't approved in the US. Steve Jobs, the late founder of Apple, went to Switzerland to get it. But that's not something that most Americans can do. How
sick were these patients? I mean majority of these patients that I treated, they had a stage four cancer, which means that the cancer has already spread throughout the body and involved different organs such as livers such as lymph notes such as bone, lungs. A majority of these patients they were expecting to leave between three to twelve months. Mix Meanwhile, there were positive reports out of Europe. American patients were reading them and they wanted the drug. There's
a twist here. The treatment isn't a pill, It a radioactive isotope that has to be carefully built in a lab, and Dr del Pasand just happens to be also the CEO of a company who has the equipment to make it. So he got permission from the U S regulators and the company that owned the rights to the therapy and
ramped up production. In that way, I was able to actually provide this necessary treatment to my patients and they were able to get this treatment in US and in Houston versus UH instead of going across the ocean to Europe. Dr del Pasand initially had FDA approval to give the drug to his patients, but in April, the agency told him he was interfering with efforts to get the treatment approved.
So at that point, basically I noted that, you know, I have to stop treating patients because it's going to
be illegal for me to continue treatment. Fortunately, for his cancer patients, a Right to Try law ALWA was passed at the state level in Texas two months later, and as a result of this Right to tryl I was able to UH to treat hundred seventy eight patients until the drug became approved and commercially available in February of two thousand and eighteen a study of the drug used by Dr del pasand showed that patients lived for an
average of thirty months without their cancer progressing. That compared to less than nine months for those given standard care. The doctor got letters from his patients thanking him for his efforts. I asked him to read me excerpts from one. Here it says that when I was diagnosed, my daughter was just graduating high school and my son just finished his first year in college. I have seen both of
them graduate college and settled into their chosen professions. I also have seen each one fall in love with wonderful people. I own a business, and my family did not have to sell it. All this from the hard work of people devorded to helping with the serious challenges in someone's life and being assisted by the common sense right to try law. It's it's just amazing. I think this is the best pay day for when when you read something from a patients, from a human being. Uh, it's just
it's just chriceless. Right to try has now been the law of the land on a national level for about six months. I have to be honest, I tried really hard to find patients, any patients who have gotten an experimental medicine under the federal law. No luck so far. Some patients and families were very candid with me, saying they would never tell if they were getting treat and under Right to Try. There's too much risk of a backlash, and if you're getting the drug, there's absolutely no benefit
to going public. Eventually we will find out if anyone's getting treatment under Right to Try. The companies have to send the f d A a report once a year telling the agency about any medicines they provided under the law. We still don't know exactly when that will be. Not everyone agrees that dying patients should have unfettered access to unapproved drugs. Experimental medicines are precious and unpredictable things. You
have to be careful with both. When the medicines do work, when they really are going to save lives, proving their efficacy and scientific studies has to be the priority. Controlled clinical studies are also the best way to protect patients from harm even death when they don't work. I was surprised about who's opposed to Right to Try. The laws backers believe that cancer patients will really benefit. Otis Browley is the chief medical officer of the American Cancer Society.
He spoke to me from his office in Atlanta, where a fire alarm went off while we were talking. I have come to realize that there truly is something worse than dying from cancer. I've seen people suffer getting drugs for a treatment and then not have benefit. I have seen, even in standard practice, people who were tortured because of treatment. I just don't want to see that um ever again. Dr Browley is concerned that the right to try law
will exploit people who have no other options. One of the problems with people who are in this situation I've talked to many of them is sometimes I fear that they see too much hope in the drug and don't really appreciate what we're talking about in terms of harms. Keep in mind, I'm somebody who wants these people to be able to have access to these drugs if they are well informed and understand. My problem is, I know a large number of people are not well informed and understand.
Dr Browley's point was that sometimes hope can be misleading. Most medicines don't make it through. Is that correct. It is absolutely correct. Most of the medicines that we have theoretic belief that it's going to be beneficial for cancer, so much belief that we actually start clinical trials in those drugs to figure out if they work and figure out if they improve outcome. We find that most of
those drugs actually fail. So most people who are going to be getting these drugs through Right to Try UM are going to get drugs that aren't going to work, but some undoubtedly will work, and that creates another problem. Companies are focused on developing medicines for big groups of people who suffer from a disease. In the early days, they can't make big batches just enough for studies, and
the drugs can be hard to make. It's a particular problem at small biotech companies that are often the engine of innovation. They work on tighter budgets and are more vulnerable to mishaps. Typically, patients are very sick, and giving away treatment to a single patient under Right to Try could jeopardize the entire research effort if something goes wrong.
If someone is taking one of these drugs and you're not in an established clinical trial where people can learn about that experience on the drug, then you're actually slowing down the development of the drug for everybody else. It really is selfish. It's not a theoretical risk. Some companies have trouble in enrolling early stage studies, known as phase one and phase two. Allowing some patients to have the experimental drug without participating in a trial could make the
process harder. I really want people to go into those phase one and phase two clinical trials and really try to help us to move cancer medicine forward as expeditiously and as efficiently as possible. Dr Browley isn't against patients getting fast access to promising new drugs. He just wants to capture all the information possible about how they work, preferably in a monitored setting, and he wants to protect the patients who are taking experimental drugs from unintended harm.
Remember before Right to Try, terminally ill patients could get experimental drugs under the FDA's Compassionate Use Program, which is also known as Expanded Access. In recent years, it's approved more than of applications. F d A Commissioner Scott god Leaves, a fan of Expanded access Right to Try, not so much. He declined to talk to us for this podcast, but he did testify before Congress about the topic in right now, our expanded access program is generally available for patients facing
life threatening conditions and terminal illness. The agency has gotten even more vigilant at trying to move these things through the agency in a in a efficient fashion and approve these The FDA also has access to information that no single doctor or patient has. It knows if complications cropped up with earlier use of the drug or with similar medicines. Such side effects aren't always immediately disclosed. The FDA's guidance
can help make sure experimental drugs are used safely. Drug makers have the last word on their medicines, and it's tempting to point the finger at pharmaceutical companies that say no to a desperate patient, But it's more complicated than that. Drug maker have been grappling with these requests for years. Johnson and Johnson's chief medical officer, Joanne wald Striker says it's one of the hardest and most heartbreaking parts of
her job. Sometimes, before we know everything about our products in advance of getting approvals from the health authorities like the f d A, before they even get to see the data on the product, we sometimes get requests for our products from patients and families around the globe who have exhausted all other options for treatment. There's a long list of issues the company has to consider when evaluating requests.
Is there enough medicine available, We'll giving it to one patient make it harder to enroll others in clinical trials. If J and JA gets more requests than the available supply, who gets the drug and who doesn't? And if things go wrong and if fragile patient dies, will that put the drugs entire development in jeopardy. We have to weigh the need of an individual person and family who's requesting access versus the needs of society, versus our our obligation
to society into public health. And that is a real bioethic challenge that we face. And you can imagine if a person is enrolled in a clinical trial and they have the chance of getting either the drug or the placebo, it may not be fair to that person if we decide to give access to someone else outside of the clinical trial setting, just because they asked and they're not going into a study where they have a chance of getting either drug or placebo. Hanging over the entire issue
is fairness. Some families and patients have the wherewithal to create powerful social media campaigns to shame companies or to try to force them to make an experimental medicine available, especially for children. But if there's one child who needs an experimental drug, there are likely more. We have to treat people in clinical trials early. But also we want
to treat people in all different socioeconomic circumstances fairly. If we get a request of someone who's a public figure, someone who's in the government, someone who's very wealthy, we want to be sure we're treating those requests the same as people who have a need who may not be in those circumstances. We want to treat everyone fairly, and so that's why this is such a challenging issue for us. So the company decided to bring in outside experts to
help deal with all these issues. In twenty fifteen, we approached the Division of Medical Ethics at the n y U School of Medicine to really help us think this through. Instead of physicians like myself at Johnson and Johnson reviewing every request and then having individual teams try to juggle and make this difficult bioethical decision in terms of whether to provide the product. The requests go to this group
called the Compassionate Access Group Compact. You know, I feel very strongly that people who have such important medical needs and who are in such urgent situations should have a level of control over what happens to them. And I sympathize and I understand that they want to be able to access experimental medicines. But on the other hand, at a time like that, when you're desperate to try anything, it's a time when expert input really can help make
a difference. Of course, Jay and J is an outlier. It's the biggest health care company in the world with unmatched resources. Clearly, not every company can afford to do what it does, but it is a start, right to try us put the f d A and the industry on notice that the immediate needs of patients must be taken into account. Laura McGlynn, the woman we met at the beginning of this episode, is taking it day by day.
Her son Jordan, is enrolled in a clinical try aisle, but she believes one day he'll need other medicines, and that's why she keeps fighting. I feel proud to know that, you know, my son played a part in helping create this additional pathway, so rather Jordan is able to access better treatments in the future through clinical trials, through um compassionate use, through right to try, through maybe you know
an f D approved drug. I'm just glad to know that there are options available and to those who feel like right to try as a shortcut a form of cutting in line that could hurt someone else. I don't let my mind go there as far as thinking about what's fair or not fair if a life is saved, or if someone has a chance to try to save their own life and they want to, I don't care. It's one life at a time, and that's to me, that's what matters. And that's it for this week's prognosis.
Thanks for listening. Do you have a story about healthcare in the US or around the world, We want to hear from you. You can email me m Cortes at Bloomberg dot net or find me on Twitter at bay Cortes. If you were a fan of this episode, please take a moment to rate and review us. It helps new listeners find the show. This episode was produced by Lindsay Cratterwell. Our story editor was Cecile Durraant. Thanks also to Drew
Armstrong Francesca Leavi as head of Bloomberg Podcasts. We'll see you next week.