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Speaker 1: I know it's probably too late for me. I made

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Speaker 1: my piece with that I need to know before I

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Speaker 1: die that my children find themselves in this unenviable position.

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Speaker 1: This nation that I probably served will respect their liberties

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Speaker 1: and the right to make their own decision about their

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Speaker 1: medical treatments. Thank you for having me. Matt Billina is

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Speaker 1: a father of three boys and a former U. S

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Speaker 1: Naval aviator in he testified at a congressional hearing to

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Speaker 1: advocate for dying patients who want access to experimental drugs.

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Speaker 1: At that point, his speech was slightly slurred by his

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Speaker 1: Luke Garrig's disease. It's a fatal condition with no treatment,

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Speaker 1: it destroys the nerve cells and the muscles they control. Slowly,

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Speaker 1: every part of the body stops working. Letting patients like

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Speaker 1: Matt who have no other options use these experimental medicines

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Speaker 1: seems like it should be easy, but it's not that straightforward,

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Speaker 1: and the critics, the people who oppose making them widely available,

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Speaker 1: aren't always who you would expect. Welcome to Prognosis, a

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Speaker 1: podcast about innovation in medicine and science. I'm your host,

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Speaker 1: Michelle fay Cortes, and today we're going to explore what

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Speaker 1: people do when they're desperate for a chance to try

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Speaker 1: an unapproved drug. Do you want to say something? Yeah? Good?

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Speaker 1: Do you want to say yes? All right? Laura McGlenn

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Speaker 1: is another American who wants access, not for herself but

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Speaker 1: for her son, Jordan's. You just heard him in a

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Speaker 1: clip from a few years ago, asking lawmakers to make

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Speaker 1: it easier he has to shend Muscular dystrophee, a genetic

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Speaker 1: disease that erodes the growing muscles of young boys and

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Speaker 1: eventually hits the heart and lungs. The average life expectancy

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Speaker 1: is twenty six. Jordan was diagnosed when he was almost four,

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Speaker 1: and at that time he was diagnosed at our local

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Speaker 1: hospital in Indianapolis, which is a pretty big, well known

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Speaker 1: children's hospital. But we left there pretty much with no hope,

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Speaker 1: and the doctor told us basically, this was a fatal disease,

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Speaker 1: no cure, probably nothing even coming up through the pipeline

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Speaker 1: that would happen in Jordan's lifetime. It turns out the

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Speaker 1: doctors who are wrong. Fast forward now, I mean Jordan's

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Speaker 1: is nine and a half and I think, um, there

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Speaker 1: are more than forty drugs actually in the investigational stages

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Speaker 1: for his disease. So a lot's happened in such a

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Speaker 1: short time with the science. First, a little background. The

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Speaker 1: easiest way to get access to an experimental drug is

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Speaker 1: in a clinical trial, but many people can't participate in trials.

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Speaker 1: They may be too sick or too young for them.

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Speaker 1: There's something called compassionate use. It's an official program regulated

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Speaker 1: by the Food and Drug Administration, but that's also difficult

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Speaker 1: to navigate. There's paperwork, and you need the f d

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Speaker 1: A s and the drug company's blessing, and it's possible

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Speaker 1: that drug company may not give it. There's too much

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Speaker 1: risk enter right to try. This is the law that

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Speaker 1: Laura McGlenn and Matt Bolina fought for. Its main difference

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Speaker 1: it cuts out the f d A. Instead, the doctor

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Speaker 1: directly asked the company for a drug. The patients take

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Speaker 1: on all the risk and make their own decisions, but

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Speaker 1: they do still need to get the drug company to

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Speaker 1: go along with it. I'm not gonna lie. I mean,

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Speaker 1: when I started this journey, all I could think about

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Speaker 1: was my one, my Jordan's. Jordan got lucky, he was

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Speaker 1: accepted into a clinical trial, but Laura didn't give up

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Speaker 1: her fight for right to try. She did it for

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Speaker 1: the other boys, and because the disende community is so close,

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Speaker 1: she's seeing firsthand what happens to those who can't get

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Speaker 1: into trials. We want our boys to access experimental treatments

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Speaker 1: through a clinical trial. Unfortunately, for example Jordan's, he's one

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Speaker 1: of sixteen boys in North America that are able to

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Speaker 1: try the drug that he's trying. While I watch many

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Speaker 1: of my friends who have sons who are amendable to

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Speaker 1: the same drug, who can't access the drug because they

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Speaker 1: don't qualify for the trial, and so their sons are basically,

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Speaker 1: you know, um, continuing to progress through this disease, which

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Speaker 1: is a muscle wasting disease, and they're losing muscle function

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Speaker 1: and it's it's truly heartbreaking. Mike Smith, a New York attorney,

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Speaker 1: also wants better access to medicines. So now, I grew

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Speaker 1: up on Long Island, New York, and the town called Sayaucet,

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Speaker 1: Um basically had a up until I was fifteen, frankly

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Speaker 1: had a perfectly normal and uneventful childhood. That's sort of

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Speaker 1: changed on Black Monday, the day of the stock market

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Speaker 1: crash on October seven. The day started with Mike scoring

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Speaker 1: a goal in a soccer game during his gym class.

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Speaker 1: The opposing goalie didn't take it well. He punched Mike

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Speaker 1: in the chest and kicked him in the lower right side.

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Speaker 1: He immediately fell ill. It was the start of a

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Speaker 1: long and harrowing health odyssey. It took five years just

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Speaker 1: to get a diagnosis, and we were starting to identify

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Speaker 1: that I had a disease or disorder called chronic intestinal

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Speaker 1: pseudo obstruction. It is an extreme spastic disorder of the

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Speaker 1: digestive track. I'd be sitting in a high school classroom,

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Speaker 1: for example, and get a sensation as if my stomach

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Speaker 1: was being tied into a shoel. I snot, you know,

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Speaker 1: And ever since, I really have never been able to

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Speaker 1: take eating or even drink king for granted. Essentially, his

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Speaker 1: digestive tract stopped working. For several years, he couldn't need

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Speaker 1: at all. He relied on artificial nutrition, and then thankfully

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Speaker 1: new drugs came along. He could eat again. But the

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Speaker 1: two treatments were pulled from the market in the US

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Speaker 1: because of potential side effects, but not in Europe. Mike

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Speaker 1: was back to square one. Now here's the thing even

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Speaker 1: under right to try he might not get the drugs

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Speaker 1: that he knows work for him, because technically it's only

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Speaker 1: for people with the terminal diagnosis and Mike's condition. Isn't

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Speaker 1: that simple? On the span of my condition at this point?

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Speaker 1: Is it chronic and debilitating? Yes? Absolutely? Can we say

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Speaker 1: fairly on a day to day basis? Is it life threatening?

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Speaker 1: I'm not a doctor. It's not for me to determine

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Speaker 1: whether or not it's life threatening. But who should make

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Speaker 1: that call? Should patients like Mike have access? Over the

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Speaker 1: past five years, more than forty states passed right to

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Speaker 1: Try laws. Those efforts culminated in the federal legislation. It

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Speaker 1: was a rare bipartisan achievement, pushed by Vice President Mike Pence.

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Speaker 1: Right to Try one support in both Houses of Congress.

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Speaker 1: It was touted by President Donald Trump in his first

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Speaker 1: State of the Union address. He signed it into law

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Speaker 1: in May. But this is such a great name. For

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Speaker 1: the first day I heard it, It's so perfect right

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Speaker 1: to try, and a lot of that trying is going

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Speaker 1: to be successful. I really believe that. I really believe it.

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Speaker 1: Ibraheim del Pasand is also a believer. More importantly, he's

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Speaker 1: the medical director at Houston's Excel Diagnostics. He's also an

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Speaker 1: expert in nuclear medicine. That means he uses radioactive materials

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Speaker 1: to diagnose and treat patients. Hey, Michelle, Hi, here we go,

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Speaker 1: we're working now. Okay, very good. Doctor del Pasand is

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Speaker 1: the doctor who is most publicly braced right to try.

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Speaker 1: He felt he had no other option. There was a

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Speaker 1: drug he wanted to give his patients, a form of

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Speaker 1: radiation for neuroendocrine cancer that had been used in Europe

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Speaker 1: for years, but it wasn't approved in the US. Steve Jobs,

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Speaker 1: the late founder of Apple, went to Switzerland to get it.

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Speaker 1: But that's not something that most Americans can do. How

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Speaker 1: sick were these patients? I mean majority of these patients

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Speaker 1: that I treated, they had a stage four cancer, which

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Speaker 1: means that the cancer has already spread throughout the body

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Speaker 1: and involved different organs such as livers such as lymph

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Speaker 1: notes such as bone, lungs. A majority of these patients

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Speaker 1: they were expecting to leave between three to twelve months.

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Speaker 1: Mix Meanwhile, there were positive reports out of Europe. American

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Speaker 1: patients were reading them and they wanted the drug. There's

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Speaker 1: a twist here. The treatment isn't a pill, It a

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Speaker 1: radioactive isotope that has to be carefully built in a lab,

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Speaker 1: and Dr del Pasand just happens to be also the

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Speaker 1: CEO of a company who has the equipment to make it.

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Speaker 1: So he got permission from the U S regulators and

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Speaker 1: the company that owned the rights to the therapy and

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Speaker 1: ramped up production. In that way, I was able to

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Speaker 1: actually provide this necessary treatment to my patients and they

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Speaker 1: were able to get this treatment in US and in

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Speaker 1: Houston versus UH instead of going across the ocean to Europe.

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Speaker 1: Dr del Pasand initially had FDA approval to give the

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Speaker 1: drug to his patients, but in April, the agency told

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Speaker 1: him he was interfering with efforts to get the treatment approved.

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Speaker 1: So at that point, basically I noted that, you know,

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Speaker 1: I have to stop treating patients because it's going to

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Speaker 1: be illegal for me to continue treatment. Fortunately, for his

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Speaker 1: cancer patients, a Right to Try law ALWA was passed

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Speaker 1: at the state level in Texas two months later, and

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Speaker 1: as a result of this Right to tryl I was

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Speaker 1: able to UH to treat hundred seventy eight patients until

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Speaker 1: the drug became approved and commercially available in February of

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Speaker 1: two thousand and eighteen a study of the drug used

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Speaker 1: by Dr del pasand showed that patients lived for an

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Speaker 1: average of thirty months without their cancer progressing. That compared

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Speaker 1: to less than nine months for those given standard care.

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Speaker 1: The doctor got letters from his patients thanking him for

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Speaker 1: his efforts. I asked him to read me excerpts from one.

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Speaker 1: Here it says that when I was diagnosed, my daughter

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Speaker 1: was just graduating high school and my son just finished

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Speaker 1: his first year in college. I have seen both of

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Speaker 1: them graduate college and settled into their chosen professions. I

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Speaker 1: also have seen each one fall in love with wonderful people.

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Speaker 1: I own a business, and my family did not have

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Speaker 1: to sell it. All this from the hard work of

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Speaker 1: people devorded to helping with the serious challenges in someone's

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Speaker 1: life and being assisted by the common sense right to

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Speaker 1: try law. It's it's just amazing. I think this is

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Speaker 1: the best pay day for when when you read something

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Speaker 1: from a patients, from a human being. Uh, it's just

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Speaker 1: it's just chriceless. Right to try has now been the

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Speaker 1: law of the land on a national level for about

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Speaker 1: six months. I have to be honest, I tried really

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Speaker 1: hard to find patients, any patients who have gotten an

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Speaker 1: experimental medicine under the federal law. No luck so far.

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Speaker 1: Some patients and families were very candid with me, saying

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Speaker 1: they would never tell if they were getting treat and

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Speaker 1: under Right to Try. There's too much risk of a backlash,

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Speaker 1: and if you're getting the drug, there's absolutely no benefit

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Speaker 1: to going public. Eventually we will find out if anyone's

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Speaker 1: getting treatment under Right to Try. The companies have to

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Speaker 1: send the f d A a report once a year

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Speaker 1: telling the agency about any medicines they provided under the law.

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Speaker 1: We still don't know exactly when that will be. Not

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Speaker 1: everyone agrees that dying patients should have unfettered access to

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Speaker 1: unapproved drugs. Experimental medicines are precious and unpredictable things. You

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Speaker 1: have to be careful with both. When the medicines do work,

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Speaker 1: when they really are going to save lives, proving their

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Speaker 1: efficacy and scientific studies has to be the priority. Controlled

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Speaker 1: clinical studies are also the best way to protect patients

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Speaker 1: from harm even death when they don't work. I was

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Speaker 1: surprised about who's opposed to Right to Try. The laws

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Speaker 1: backers believe that cancer patients will really benefit. Otis Browley

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Speaker 1: is the chief medical officer of the American Cancer Society.

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Speaker 1: He spoke to me from his office in Atlanta, where

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Speaker 1: a fire alarm went off while we were talking. I

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Speaker 1: have come to realize that there truly is something worse

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Speaker 1: than dying from cancer. I've seen people suffer getting drugs

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Speaker 1: for a treatment and then not have benefit. I have seen,

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Speaker 1: even in standard practice, people who were tortured because of treatment.

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Speaker 1: I just don't want to see that um ever again.

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Speaker 1: Dr Browley is concerned that the right to try law

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Speaker 1: will exploit people who have no other options. One of

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Speaker 1: the problems with people who are in this situation I've

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Speaker 1: talked to many of them is sometimes I fear that

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Speaker 1: they see too much hope in the drug and don't

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Speaker 1: really appreciate what we're talking about in terms of harms.

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Speaker 1: Keep in mind, I'm somebody who wants these people to

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Speaker 1: be able to have access to these drugs if they

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Speaker 1: are well informed and understand. My problem is, I know

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Speaker 1: a large number of people are not well informed and understand.

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Speaker 1: Dr Browley's point was that sometimes hope can be misleading.

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Speaker 1: Most medicines don't make it through. Is that correct. It

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Speaker 1: is absolutely correct. Most of the medicines that we have

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Speaker 1: theoretic belief that it's going to be beneficial for cancer,

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Speaker 1: so much belief that we actually start clinical trials in

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Speaker 1: those drugs to figure out if they work and figure

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Speaker 1: out if they improve outcome. We find that most of

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Speaker 1: those drugs actually fail. So most people who are going

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Speaker 1: to be getting these drugs through Right to Try UM

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Speaker 1: are going to get drugs that aren't going to work,

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Speaker 1: but some undoubtedly will work, and that creates another problem.

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Speaker 1: Companies are focused on developing medicines for big groups of

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Speaker 1: people who suffer from a disease. In the early days,

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Speaker 1: they can't make big batches just enough for studies, and

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Speaker 1: the drugs can be hard to make. It's a particular

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Speaker 1: problem at small biotech companies that are often the engine

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Speaker 1: of innovation. They work on tighter budgets and are more

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Speaker 1: vulnerable to mishaps. Typically, patients are very sick, and giving

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Speaker 1: away treatment to a single patient under Right to Try

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Speaker 1: could jeopardize the entire research effort if something goes wrong.

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Speaker 1: If someone is taking one of these drugs and you're

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Speaker 1: not in an established clinical trial where people can learn

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Speaker 1: about that experience on the drug, then you're actually slowing

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Speaker 1: down the development of the drug for everybody else. It

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Speaker 1: really is selfish. It's not a theoretical risk. Some companies

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Speaker 1: have trouble in enrolling early stage studies, known as phase

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Speaker 1: one and phase two. Allowing some patients to have the

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Speaker 1: experimental drug without participating in a trial could make the

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Speaker 1: process harder. I really want people to go into those

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Speaker 1: phase one and phase two clinical trials and really try

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Speaker 1: to help us to move cancer medicine forward as expeditiously

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Speaker 1: and as efficiently as possible. Dr Browley isn't against patients

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Speaker 1: getting fast access to promising new drugs. He just wants

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Speaker 1: to capture all the information possible about how they work,

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Speaker 1: preferably in a monitored setting, and he wants to protect

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Speaker 1: the patients who are taking experimental drugs from unintended harm.

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Speaker 1: Remember before Right to Try, terminally ill patients could get

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Speaker 1: experimental drugs under the FDA's Compassionate Use Program, which is

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Speaker 1: also known as Expanded Access. In recent years, it's approved

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Speaker 1: more than of applications. F d A Commissioner Scott god Leaves,

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Speaker 1: a fan of Expanded access Right to Try, not so much.

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Speaker 1: He declined to talk to us for this podcast, but

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Speaker 1: he did testify before Congress about the topic in right now,

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Speaker 1: our expanded access program is generally available for patients facing

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Speaker 1: life threatening conditions and terminal illness. The agency has gotten

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Speaker 1: even more vigilant at trying to move these things through

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Speaker 1: the agency in a in a efficient fashion and approve

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Speaker 1: these The FDA also has access to information that no

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Speaker 1: single doctor or patient has. It knows if complications cropped

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Speaker 1: up with earlier use of the drug or with similar medicines.

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Speaker 1: Such side effects aren't always immediately disclosed. The FDA's guidance

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Speaker 1: can help make sure experimental drugs are used safely. Drug

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Speaker 1: makers have the last word on their medicines, and it's

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Speaker 1: tempting to point the finger at pharmaceutical companies that say

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Speaker 1: no to a desperate patient, But it's more complicated than that.

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Speaker 1: Drug maker have been grappling with these requests for years.

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Speaker 1: Johnson and Johnson's chief medical officer, Joanne wald Striker says

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Speaker 1: it's one of the hardest and most heartbreaking parts of

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Speaker 1: her job. Sometimes, before we know everything about our products

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Speaker 1: in advance of getting approvals from the health authorities like

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Speaker 1: the f d A, before they even get to see

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Speaker 1: the data on the product, we sometimes get requests for

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Speaker 1: our products from patients and families around the globe who

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Speaker 1: have exhausted all other options for treatment. There's a long

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Speaker 1: list of issues the company has to consider when evaluating requests.

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Speaker 1: Is there enough medicine available, We'll giving it to one

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Speaker 1: patient make it harder to enroll others in clinical trials.

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Speaker 1: If J and JA gets more requests than the available supply,

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Speaker 1: who gets the drug and who doesn't? And if things

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Speaker 1: go wrong and if fragile patient dies, will that put

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Speaker 1: the drugs entire development in jeopardy. We have to weigh

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Speaker 1: the need of an individual person and family who's requesting

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Speaker 1: access versus the needs of society, versus our our obligation

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Speaker 1: to society into public health. And that is a real

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Speaker 1: bioethic challenge that we face. And you can imagine if

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Speaker 1: a person is enrolled in a clinical trial and they

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Speaker 1: have the chance of getting either the drug or the placebo,

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Speaker 1: it may not be fair to that person if we

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Speaker 1: decide to give access to someone else outside of the

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Speaker 1: clinical trial setting, just because they asked and they're not

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Speaker 1: going into a study where they have a chance of

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Speaker 1: getting either drug or placebo. Hanging over the entire issue

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Speaker 1: is fairness. Some families and patients have the wherewithal to

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Speaker 1: create powerful social media campaigns to shame companies or to

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Speaker 1: try to force them to make an experimental medicine available,

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Speaker 1: especially for children. But if there's one child who needs

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Speaker 1: an experimental drug, there are likely more. We have to

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Speaker 1: treat people in clinical trials early. But also we want

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Speaker 1: to treat people in all different socioeconomic circumstances fairly. If

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Speaker 1: we get a request of someone who's a public figure,

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Speaker 1: someone who's in the government, someone who's very wealthy, we

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Speaker 1: want to be sure we're treating those requests the same

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Speaker 1: as people who have a need who may not be

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Speaker 1: in those circumstances. We want to treat everyone fairly, and

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Speaker 1: so that's why this is such a challenging issue for us.

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Speaker 1: So the company decided to bring in outside experts to

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Speaker 1: help deal with all these issues. In twenty fifteen, we

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Speaker 1: approached the Division of Medical Ethics at the n y

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Speaker 1: U School of Medicine to really help us think this through.

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Speaker 1: Instead of physicians like myself at Johnson and Johnson reviewing

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Speaker 1: every request and then having individual teams try to juggle

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Speaker 1: and make this difficult bioethical decision in terms of whether

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Speaker 1: to provide the product. The requests go to this group

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Speaker 1: called the Compassionate Access Group Compact. You know, I feel

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Speaker 1: very strongly that people who have such important medical needs

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Speaker 1: and who are in such urgent situations should have a

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Speaker 1: level of control over what happens to them. And I

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Speaker 1: sympathize and I understand that they want to be able

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Speaker 1: to access experimental medicines. But on the other hand, at

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Speaker 1: a time like that, when you're desperate to try anything,

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Speaker 1: it's a time when expert input really can help make

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Speaker 1: a difference. Of course, Jay and J is an outlier.

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Speaker 1: It's the biggest health care company in the world with

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Speaker 1: unmatched resources. Clearly, not every company can afford to do

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Speaker 1: what it does, but it is a start, right to

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Speaker 1: try us put the f d A and the industry

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Speaker 1: on notice that the immediate needs of patients must be

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Speaker 1: taken into account. Laura McGlynn, the woman we met at

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Speaker 1: the beginning of this episode, is taking it day by day.

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Speaker 1: Her son Jordan, is enrolled in a clinical try aisle,

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Speaker 1: but she believes one day he'll need other medicines, and

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Speaker 1: that's why she keeps fighting. I feel proud to know that,

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Speaker 1: you know, my son played a part in helping create

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Speaker 1: this additional pathway, so rather Jordan is able to access

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Speaker 1: better treatments in the future through clinical trials, through um

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Speaker 1: compassionate use, through right to try, through maybe you know

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Speaker 1: an f D approved drug. I'm just glad to know

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Speaker 1: that there are options available and to those who feel

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Speaker 1: like right to try as a shortcut a form of

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Speaker 1: cutting in line that could hurt someone else. I don't

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Speaker 1: let my mind go there as far as thinking about

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Speaker 1: what's fair or not fair if a life is saved,

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Speaker 1: or if someone has a chance to try to save

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Speaker 1: their own life and they want to, I don't care.

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Speaker 1: It's one life at a time, and that's to me,

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Speaker 1: that's what matters. And that's it for this week's prognosis.

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Speaker 1: Thanks for listening. Do you have a story about healthcare

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Speaker 1: in the US or around the world, We want to

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Speaker 1: hear from you. You can email me m Cortes at

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Speaker 1: Bloomberg dot net or find me on Twitter at bay Cortes.

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Speaker 1: If you were a fan of this episode, please take

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Speaker 1: a moment to rate and review us. It helps new

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Speaker 1: listeners find the show. This episode was produced by Lindsay Cratterwell.

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Speaker 1: Our story editor was Cecile Durraant. Thanks also to Drew

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Speaker 1: Armstrong Francesca Leavi as head of Bloomberg Podcasts. We'll see

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Speaker 1: you next week.